H3094- Section on Advances in Therapeutics and Technology Program

Research Abstract Program and Award for Pediatric Innovation

Topic: Advances in Therapeutics and Technology

Sponsors: Section on Advances in Therapeutics and Technology (SOATT)

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2011

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This program will provide information on new research in the area of pediatric innovation. The third annual section Award for Pediatric Innovation also will be presented.

12:00PM Welcome
12:05PM Top Three Research Paper Presentations, Question & Answer Session
1:00PM Section Award for Pediatric Innovation Presentation
Supported by Pfizer
1:30PM Reception
2:00PM Adjourn

Abstracts

12:00PM - 2:00PM
12:00PM - 2:00PM
12:20PM - 12:30PM

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1- A Novel Nonsteroidal, Topical Anti-inflammatory, Phosphodiesterase 4 Inhibitor, Crisaborole Ointment, Reduced Pruritus and Signs of Atopic Dermatitis in 2 Phase 3 Studies in Children and Adults

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Background: Atopic dermatitis (AD) is an inflammatory skin disease that presents with intense pruritus and red, inflamed lesions. Crisaborole Topical Ointment, 2% (Anacor Pharmaceuticals, Palo Alto, CA), is an investigational, nonsteroidal, topical anti-inflammatory, phosphodiesterase 4 inhibitor for the treatment of AD.
Objective: To evaluate the additional predefined efficacy endpoints on the impact of crisaborole ointment on pruritus and signs of AD from 2 Phase 3 studies (AD-301: NCT02118766; AD-302: NCT02118792).
Methods: Patients ≥2 years old with mild-to-moderate AD were enrolled in 2 multicenter, double-blind, vehicle-controlled, identically designed Phase 3 studies. Patients were randomly assigned 2:1 for treatment with crisaborole or vehicle twice daily for 28 days. Exploratory efficacy endpoints evaluated severity of signs of AD (measured weekly: erythema, induration/papulation, exudation, excoriation, and lichenification) and pruritus (measured twice daily) on a 4-point scale (None [0] to Severe [3]). Achievement of None (0) or Mild (1) with ≥1-grade improvement from baseline defined Success for each sign or symptom. Time to Success in Pruritus was examined using Kaplan-Meier analysis.
Results: Studies AD-301 and AD-302 enrolled 503:256 and 513:250 crisaborole:vehicle patients, respectively. Across treatment groups/studies, baseline severity of signs and symptoms was generally balanced. Success in Pruritus was achieved sooner by crisaborole-treated patients than vehicle-treated patients (pooled data: median 1.37 vs 1.70 days; P = 0.001). A greater proportion of crisaborole-treated patients achieved Success for all clinical signs of AD than vehicle-treated patients in both studies ([AD-301, crisaborole vs vehicle; AD-302, crisaborole vs vehicle] erythema: 62.8% vs 46.1%, 54.9% vs 33.9%; induration/papulation: 57.7% vs 54.8%, -51.9% vs 40.2%; exudation: 41.0% vs 33.3%, 38.1% vs 27.2%; excoriation: 63.0% vs 51.8%, 57.2% vs 44.2%; lichenification: 51.7% vs 46.5%, 51.4% vs 35.3%).
Conclusion: Crisaborole Topical Ointment, 2%, demonstrated improvement in all measured signs of AD and early relief of pruritus in 2 large Phase 3 studies and may represent an efficacious and safe treatment for patients with mild to moderate AD as young as 2 years.

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2- Harmonics of High Frequency Ventilation at 3hz

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Introduction: High frequency ventilation has allowed clinicians to oxygenate and ventilate patients when conventional ventilation is not sufficient, particularly in premature babies. High frequency ventilation (HFOV) incorporates lung protective strategies including adequate mean airway pressures to sustain alveolar recruitment, and small tidal volumes to avoid over distention. However, reviews of randomized controlled clinical trials have yet to confirm improved outcomes (Cools 2009). The premature lung exhibits mechanical and spatial heterogeneity, and may benefit from oscillation with multiple simultaneous frequencies compared with traditional single frequency HFOV (Kaczka 2015). The term “harmonic” is used across various disciplines and is applied when considering the frequencies of repeating signals, such as sinusoidal waves, as we see in HFOV. In this case, a harmonic is a signal whose frequency is a multiple of the frequency of some other given signal, or “daughter frequency”. We theorized that the oscillator may provide multi-frequency ventilation, and that we would be able to pick up harmonics at multiple frequencies that may provide useful ventilation and, may help explain the potential benefits of multi-frequency ventilation in the premature lung.
Materials and Methods: We used the Sensormedics 3100A High Frequency Oscillator and the RD020 test lung for this study. We studied different flow and pressure dynamics at varying ventilator i-times (IT) from 0.30 (30%) to 0.50 (50%) at bias flows of 20 and 30L. In addition, we varied Amplitude and flow at a frequency of 3hz. Baseline flow and pressure measurements were obtained at those settings with typical HFOV tubing configuration. Easy sense UPC2100 software was used to obtain our measurements. Sampling was at 1000 Hz. 10 seconds of data or 10,000 measurements were taken at each setting.
Results: With the varying Amplitude and IT at 30 and 40, 0.33, 0.4, and 0.49 respectively, studied together with a fixed MAP, we were able to uncover the harmonics at multiples of the fundamental frequencies of 3Hz. Clear spikes in pattern occurred at 3.296 Hz, 6.531Hz, 9.827Hz, and 19.65 Hz.
Conclusion: Harmonics have been studied in various disciplines, particularly music, and may now have a place in the use of high frequency ventilation. At 3 Hz, we were able to detect consistent harmonics, or daughter frequencies, reliably across variable iTime, amplitude, and flow. There are functional differences in the magnitude of the harmonic frequencies that may provide evidence of variation in the ventilation effect. Further delineation of this effect is a necessary precursor to understanding the potential benefit of multi-frequency oscillator ventilation prior to amplification of individual harmonic frequencies. There remains a gap in the understanding of the contribution of the baseline harmonics in high frequency ventilation.

Variation in frequencies at 9Hz
This graph demonstrates the variation in frequencies present at different iTimes at a set frequency of 9 Hz.
Variation in frequencies at 6Hz
This graph demonstrates the variation in frequencies present at different iTimes at a set frequency of 6 Hz.

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3- Phase 3 Studies Treating Children and Adult Patients with Mild to Moderate Atopic Dermatitis with Crisaborole Ointment, a Novel, Nonsteroidal, Topical Anti-inflammatory, Phosphodiesterase 4 Inhibitor

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Background: Atopic dermatitis (AD) is an inflammatory skin disease affecting children and adults, and up to 90% of patients present with mild to moderate AD. Crisaborole Topical Ointment, 2% (Anacor Pharmaceuticals, Palo Alto, CA), is an investigational nonsteroidal, topical, anti-inflammatory inhibitor of phosphodiesterase 4.
Objective: To evaluate the efficacy and safety of Crisaborole Topical Ointment, 2%, in children and adults with mild to moderate AD in 2 Phase 3 clinical studies (AD-301: NCT02118766; AD-302: NCT02118792).
Method: Patients ≥2 years old with mild to moderate AD affecting ≥5% of body surface area (BSA) were enrolled in 2 multicenter, double-blind, vehicle-controlled studies of identical design. Patients were randomly assigned 2:1 to receive crisaborole or vehicle twice daily for 28 days and evaluated on days 8, 15, 22, and 29. The primary endpoint defined Success in the Investigator’s Static Global Assessment (ISGA) as “clear/0”or “almost clear/1” with ≥2-grade improvement from baseline at day 29. Secondary endpoints measured the percentage of patients achieving “clear/0” or “almost clear/1” on ISGA and time to Success in ISGA.
Results: AD-301 and AD-302 enrolled 503:256 and 513:250 patients to crisaborole:vehicle, respectively. There were no significant differences in key baseline characteristics across all groups/studies (pooled data: mean age, ~12 years; mean BSA, ~18%; ISGA, ~60% “moderate/3” and 40% “mild/2”). More patients achieved Success in ISGA with crisaborole than vehicle at day 29 (AD-301: 32.8% vs 25.4%, P = 0.038; AD-302: 31.4% vs 18.0%, P < 0.001), with a greater percentage of “clear/0” or “almost clear/1” ISGA scores (51.7% vs 40.6%, P = 0.005; 48.5 vs 29.7%, P < 0.001). Patients achieved Success in ISGA earlier when treated with crisaborole than vehicle (P < 0.001). Treatment-related adverse events (AE) were mostly mild and included application site pain (pooled data, crisaborole vs vehicle: 4.4% vs 1.2%) and upper respiratory tract infections (3.0% vs 3.0%). Discontinuation rates due to AEs were 1.2% for both crisaborole- and vehicle-treated patients.
Conclusion: Crisaborole Topical Ointment, 2%, demonstrated favorable efficacy and safety in patients as young as 2 years old with mild to moderate AD in 2 large Phase 3 studies. Crisaborole may represent a safe and efficacious treatment for AD in children and adults.

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4- Advances in Imaging Technology and Increases in Healthcare Accessibility Have Led to Shifts in the Initial Staging of Pediatric Cancers in the Us

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Background and Objectives: The State Children’s Health Insurance Program (CHIP) was created in 1997 to expand affordable health insurance coverage for United States children and successfully enrolled more than 8 million children by 2012, halving the uninsured rate from 14% to 7%. Concordant with this increase in insured children was an increase in preventive care visits, a doubling of the CT and MRI devices per capita as well as scans per capita, and the commercial availability of the first PET/CT in 2001. Over this same time period, no changes were made to pediatric cancer screening guidelines. This study’s objective was to determine if this increase in healthcare and imaging utilization would correspond with an increase in the detection of early stage rather than late stage pediatric cancers.

Methods: The Surveillance, Epidemiology, and End Results Program (SEER) registries was queried for all children (age < 18) diagnosed and staged with cancer from 1997-2012. Individual SEER registries were excluded if they did not start collecting data before the ratification of CHIP in 1997. Cancer types that did not have a staging system were excluded from the analysis. Solid tumors were classified as “early stage” if they were localized without evidence of regional invasion. Lymphomas were classified as “early stage” if they were Ann Arbor Stage I.

Results: The SEER registries queried contained 16,569 records of children that were staged with cancer from 1997-2012. Of this population, the proportion of stageable cancers that were detected at an early stage increased modestly from 24.3% in the 4-year period from 1997-2000 to 26.3% in 2009-2012 (p=0.036). When stratifying by primary sites across the same time period, the proportion of lymphomas detected at an early stage decreased significantly from 25.2% to 19.6% (p=0.007). The proportion of soft tissue/cardiac primaries detected at an early stage increased significantly from 43.5% to 51.4% (p=0.027). The proportion of bone/joint, and renal cancers that were detected at an early stage did not change significantly in the time periods studied.

Conclusions: From 1997-2012, the proportion of stageable pediatric cancers detected at an early stage increased from 1997-2012. This increase was most notable in soft tissue sarcomas and cardiac primaries. Contrary to the general trend, lymphomas experienced a significant decrease in the proportion of patients detected at an early stage of disease, likely due to the highly metastatic nature of lymphoma and the new availability of highly sensitive imaging modalities such as PET/CT. These trends in initial staging will likely continue due to efforts to universalize health insurance as well as advancements in imaging such as PET/MRI and ultra-high resolution CT.

Figure 1: Pediatric Lymphoma Staging By Year
Initial Ann Arbor Staging of patients 0-17 years of age by 4 year periods. Significant changes from initial and ending time periods denoted by bolding.
Figure 2: Pediatric Soft Tissue Sarcoma Staging By Year
Initial tumor involvement of patients 0-17 years of age by 4 year periods. Significant changes from initial and ending time periods denoted by bolding.

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5- Effect of krn23, a Fully Human anti-fgf23 Monoclonal Antibody, on Rickets in Children with X-linked Hypophosphatemia (xlh): 40-week Interim Results from a Randomized, Open-label Phase 2 Study

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Purpose: In patients with XLH, high circulating FGF23 induces renal phosphate (Pi) wasting, hypophosphatemia, rickets, and short stature. Current treatment with oral phosphate/active vitamin D is suboptimal in many patients. Methods: In a Phase 2 study, we randomized 52 children with XLH (ages 5-12 years, ≤Tanner 2) to receive KRN23 subcutaneously, either biweekly (Q2W) or monthly (Q4W). Serum Pi was measured biweekly. KRN23 dose was titrated (maximum 2mg/kg) to achieve age-appropriate serum Pi concentrations. The first 36 subjects received standard-of-care treatment for a mean of 6.6 years before enrolment. Results: With KRN23 treatment, serum Pi increased from baseline in all subjects to near normal levels (mean increase 0.30 mmol/L at 38 weeks; p < 0.001) and was more stable with Q2W dosing; hyperphosphatemia did not occur in any subject. KRN23 significantly improved rickets, assessed using the radiographic Thacher Rickets Severity Score (RSS), with greater improvements seen with Q2W dosing (44% reduction; p=0.013) and particularly in subjects with higher-severity rickets at baseline (RSS ≥1.5) (59% reduction; p < 0.0001). Using the Radiographic Global Impression of Change (RGI-C; 3=worsening; +3=complete healing), Q2W dosing improved rickets by +1.6 (p < 0.0001) with the higher-severity rickets subset showing substantial healing (+2.0; p < 0.0001). Serum alkaline phosphatase decreased. Most treatment-related adverse events (AE) were mild; transient injection site reactions occurred most frequently (39%). One child experienced a serious AE, was hospitalized for fever/muscle pain that improved, and continues in the trial. No clinically meaningful changes occurred in serum or urine calcium, serum iPTH, or renal ultrasound. Conclusion: KRN23 improved phosphorus homeostasis and healed rickets in children with XLH.

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6- Removing the Active Exhalation Phase from High Frequency Oscillatory Ventilation

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Introduction: High frequency ventilation allows clinicians to treat patients when conventional ventilation is not sufficient. Various mechanical methods are used to achieve high frequency ventilation and these differences can have clinical significance. For example, the difference between passive and active exhalation is very significant when comparing the High Frequency Oscillatory Ventilator (HFOV) with the High Frequency Jet Ventilator (HFJV). The HFOV diaphragm creates an oscillatory pressure wave with both positive and negative pressures and gas flows (active ventilation). HFJV uses a flow interrupter to create high frequency pulse “breaths” without generating any negative pressure (passive ventilation). We theorized that using a one-way valve in-line with the HFOV we could deliver high frequency positive ventilation with reduced active negative flow and change the pressure curve properties (skew and kurtosis) in response to the change from active to passive exhalation.
Methods: We used the Sensormedics 3100A HFOV and an RD020 test lung for this study. Frequency, MAP, Amplitude and bias flow were fixed at 10 Hz, 20 cm H2O, 20 cm H2O and 20 LPM respectively. Baseline flow and pressure measurements were obtained at those settings with typical HFOV configuration. A one-way valve was then placed in-line with the circuit and repeat measurements were recorded. Flow and pressure dynamics were studied at varying ventilator i-times (IT) from 0.30 (30%) to 0.50 (50%). Easy sense UPC2100 software was used to obtain our measurements. Sampling was at 1000 Hz and 10 seconds of data were taken at each setting.
Results: Measured flow was compared over each IT with and without the one-way valve. The mean minimum flow, with and without the one-way valve were 1.3 ± 0.2 LPM vs -12.6 ± 0.8 LPM (p < 0.001) [image 1]. The pressure data was collected and compared between the baseline and one-way valve configurations. The waveforms were similar but had significant differences in compared skew and kurtosis. With the valve in place, the pressure skew was consistently less positive (more normally distributed) at each IT compared to the control. At lower IT, the valve pressure wave kurtosis was more negative (flat) than the control, however, as the IT was increased, the control kurtosis decreased more significantly and became more negative at higher IT [image 2].
Conclusion: Significantly higher average flow and reduced negative flow were demonstrated with the one-way valve in place. This implies that using a HFO ventilator with a one-way valve in-line with the circuit may be a novel way to provide ventilation with essentially eliminated negative flows seen when using traditional HFOV. In addition, the changes to the pressure waveform skew and kurtosis demonstrate that the pressure waveform characteristics are also novel and can be modified by changing the HFOV IT.

Image 1
Distribution of flow occurrences (10,000) without valve (BLUE) and with valve (RED)
Image 2
Pressure waveform skew and kurtosis. Control vs valve at increasing i-times.

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7- A Comparison of Patients with Acute vs Chronic Headache Presenting to a Pediatric Emergency Department

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Purpose
Headache is a frequent and problematic complaint in pediatric emergency departments (ED) that causes distress for the patients, family, and staff. Our purpose was to investigate the management of children presenting to an urban pediatric ED with headache complaints.

Methods
We conducted a retrospective chart review of patients presenting between January 1 and December 31, 2013. Patients between the ages of 5 and 17 years with a discharge code of headache, migraine, or pseudotumor cerebri were included. Children with head trauma were excluded. We compared two groups of children: those with acute (ACUTE) headache of < one month duration, and those with chronic (CHRONIC) symptoms of headache for > 1 month. Charts were limited to the patients’ first visit when there were multiple occurrences within the one-year span.

Results
A total of 151 patients met the criteria for inclusion. Twelve children were excluded because they left the ED against medical advice or before being evaluated. This left a total of 139 (92%) for analysis. Study patients had a median age of 13 years with the majority being female (56%). The mean pre medication administration (RX) pain score for all patients was 5.8 (SD=3.1) out of a maximum score of 10 and 1.9 (SD=2.3) post RX. In our study sample, 71% of patients were ACUTE, while 29% were CHRONIC. Median length of stay (LOS) was more than double in the CHRONIC (6 hours: 59 minutes) vs. ACUTE (3 hours:11 minutes). When adjusted for non-normality, significant associations were seen among ACUTE for (a) positive patient history of headache (p < .05) and (b) not receiving a CT or MRI (p=.05). Significantly more ACUTE consulted their pediatrician prior to ED presentation vs. CHRONIC (ACUTE 79% vs. CHRONIC 21%, p < .001). There were no significant differences in disposition. However, the only patients admitted (n=3) to the medical center were ACUTE patients. Both ibuprofen (p < .05) and metoclopramide (p < .05) were more frequently dispensed to ACUTE patients. Females were more likely to receive toradol (p < .05). Amitriptyline, topirimate, or prochlorperazine were not prescribed to any patient in either group. Narcotics were only prescribed within the ACUTE group (n=3). Please see figures 1 and 2 for distribution of pain scores pre and post RX.

Conclusion
Our principal findings are: (1) children with chronic headaches are significantly less likely than those with acute headache to consult their pediatricians prior to seeking help at the ED; (2) neuroimaging is used judiciously in the evaluation of acute headache; (3) ibuprofen, ketorolac and metoclopramide reduced headache pain in children with both chronic and acute headache; (4) amitriptyline, topirimate, and prochlorperazine were not utilized in the management of headache in the ED.

Pediatric Pain Scores Pre and Post for ACUTE Headache
Pediatric Pain Scores Pre and Post for CHRONIC Headache

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8- Clinical Characteristics of Adolescent and Adult Patients with Refractory Chronic Idiopathic Urticaria (ciu) in Three Phase Iii Studies with Omalizumab

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Purpose: Information on the clinical profile of chronic idiopathic urticaria (CIU) in adolescents is limited. Baseline characteristics and demographics of adolescent and adult subgroups of patients with CIU enrolled in randomized, placebo-controlled omalizumab trials are evaluated.
Methods: This is a post hoc analysis of pooled baseline data from 3 omalizumab trials (ASTERIA I, ASTERIA II and GLACIAL) in patients with CIU who remained symptomatic despite H1-antihistamine treatment (and H2-antihistamines and/or leukotriene receptor antagonists [LTRA] in one study). Demographics, disease characteristics and medical history are summarized for adolescent (≥12 to < 18 years) and adult (≥18 years) patients.
Results: In total, 39 adolescents (mean [SD] age: 15.1 [1.5] years) and 936 adults (43.4 [13.2] years) were investigated. The majority of patients were female (adolescents: 69.2%; adults: 73.6%) and white (84.6%; 85.5%), with a mean (SD) BMI of 24.4 (4.7) kg/m2 and 29.9 (7.3) kg/m2, respectively. A positive CU Index™ test was seen in 17.9% of adolescents compared with 29.0% of adults. Most clinical disease characteristics were similar, including urticaria activity score over 7 days (mean [SD] adolescents: 28.6 [5.7]; adults: 31.0 [6.7]), weekly itch severity score (13.3 [3.4]; 14.1 [3.6]) and weekly number of hives score (15.3 [4.0]; 16.9 [4.3]). Baseline angioedema was present less frequently in adolescents (28.2%) compared with adults (48.0%). Both groups reported similar levels of impairment in quality of life as assessed by Dermatology Life Quality Index (overall score [SD] adolescents: 12.0 [5.2]; adults: 13.3 [6.6]), Chronic Urticaria Quality of Life Questionnaire (36.1 [17.5]; 44.1 [17.5]) and European Quality of Life-5 Dimensions index (both groups: 0.7 [0.3]). Allergic rhinitis (61.5%; 43.2%) and asthma (56.4%; 22.3%) were experienced more frequently in adolescents compared with adults, while conditions associated with increased age, such as hypertension (7.7%; 21.5%) and coronary artery disease (0.0%; 1.8%) were more likely to have been experienced by adults. Diabetes mellitus was reported in a similar percentage of adolescents and adults (5.1% and 5.7%, respectively). Adolescents tended not to have experienced CIU for as long as adults (mean [SD] 3.2 [3.7] vs 7.1 [9.2] years, respectively) and had received fewer previous CIU medications (mean [SD] 4.2 (2.3) vs 5.0 [2.8], respectively). With the exception of one adult, all patients received H1-antihistamines. Concomitant medications included: H2-antihistamines (adolescents: 48.7%; adults: 48.4%); LTRAs (35.9%; 34.4%) and systemic steroids (33.3%; 46.3%).
Conclusion: The number of adolescents across these studies suggests that CIU is either of low incidence or misdiagnosed, highlighting the need for larger-scale studies in this population. The demographic and clinical differences observed between adolescent and adult populations will aid in improving our understanding of CIU in adolescents and may help guide clinical practice.

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9- Extracorporeal Membrane Oxygenation Bridge to Pediatric Lung Transplantation: Trends in the United States

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Introduction: In the United States, extracorporeal membrane oxygenation (ECMO) has been increasingly used to bridge adult patients to lung transplantation (LTx). Use of ECMO in pediatric LTx has been shown to have acceptable survival outcomes, yet recent trends in ECMO bridge to pediatric LTx are unknown.

Materials and Methods: The United Network for Organ Sharing was queried for LTx performed between 5/2005 and 3/2015 to define center experience with ECMO bridge to LTx and center mix of pediatric and adult LTx. Pediatric patients (age < 18 years) bridged to first-time LTx with ECMO were selected for analysis and divided into early (2005-2012) and recent (2013-2015) eras based on the year of LTx. Comparisons among eras were performed using Fisher’s exact test for categorical data and Mann-Whitney U-test for continuous data.

Results: Twenty-two children bridged to LTx with ECMO were identified since May 2005, 11 of whom received LTx in 2012 or earlier. An average of 3 pediatric LTx involved ECMO support since 2009. No statistically significant trend in ECMO bridge to LTx as a proportion of pediatric LTx was identified (p = 0.243 by Cochran-Armitage test). Comparisons of patient and center characteristics between early and recent eras revealed no significant differences in patient age, waitlist time, indication for LTx, center ECMO expertise, or center mix of pediatric and adult LTx.

Conclusions: ECMO is infrequently used to bridge children to LTx, and has not shared in the growth of ECMO as bridge to LTx in the adult population.

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10- Standardized Patterned Somatosensory Oral Stimulation in Very Preterm Infants: A Multicenter Randomized Controlled Trial

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Background: Very preterm infants often have delayed establishment of full oral feeding (FOF), a rate-limiting step for discharge, prolonging hospital stay.

Objective: To evaluate the effect of standardized patterned somatosensory oral stimulation on transition to FOF and on length of stay (LOS) in very preterm infants.

Design/Methods: This multicenter randomized controlled trial (RCT) was conducted in five centers between 2011 and 2015. Eligible infants born between 26 0/7 and 30 6/7 weeks of gestational age (GA) were enrolled, randomized, and stratified by GA. The experimental group (n=109) received programmed patterned somatosensory oral stimulation delivered by a pulsatile pneumatically charged pacifier (NTrainer, Innara Health, KS) and the sham control group (n=101) received a non-pulsatile pacifier. Interventions were performed 3-4 times daily during tube feeding for 10-14 days beginning at 30 0/7-32 6/7 weeks post menstrual age (PMA). Oral feeding was initiated as early as 32 0/7 weeks PMA and advanced based on a standardized feeding protocol. The median days to and PMA at FOF and median LOS between the groups were compared using the Mann-Whitney rank sum test. The mean days to FOF, LOS, PMA at FOF and PMA at discharge were compared using t-test.

Results: There were no differences in the GA and birth weight between the two groups. The number of days and PMA to FOF and discharge are shown in table.

Conclusions: This RCT shows that oral stimulation shortens LOS. The magnitude of reduction in LOS compared to FOF suggests that impact of entrainment is not limited to enhancing oral feeding but also in promoting developmental maturity in these infants.

Table 1: Outcomes

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11- Enhancing Pediatric Clinical Trial Feasibility Through the Use of Bayesian Statistics

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Pediatric trials are becoming increasingly challenging. Common hurdles include an increasingly crowded research landscape – with numerous treatments in late-stage development and newly approved treatments for adults, most with regulatory commitments for pediatric trials, and limited pools of pediatric patients and investigators. For adult clinical trials, such challenges can be addressed by expanding the number of sites or countries; however, that approach is unrealistic in pediatric trials due to variable regulatory environments and a dearth of appropriately skilled or interested sites. Site availability is also often hindered by a perception that pediatric trials are not scientifically stimulating or offer low return on investment due to the small number of patients to be enrolled per year. Improving the practicability of pediatric trials will take a multifactorial approach, including conducting advance feasibility assessments, utilizing pediatric networks, collaborating across institutions, and using alternative statistical approaches.

Using a Bayesian statistical approach, we demonstrated the potential to decrease the number of subjects required for a pediatric trial. We chose to examine type 2 diabetes because enrollment into pediatric trials for this indication is particularly challenging, and the disease pathophysiology is considered to be similar in adolescents and adults, making it a good candidate for this statistical approach. We ran simulations using prior adult data from six unique anti-hyperglycemic agents from three different, recently approved classes.

Results show that giving the adult prior and pediatric data equal weight consistently reduced the projected sample size for pediatric studies by approximately 75% at 90% power. To decrease the likelihood of incorrectly concluding that a drug works in children, we also carried out simulation modifications that can be used to decrease false positives while retaining a large part of the reduction in sample size.

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12- Early Morning Functioning in Stimulant-treated Children and Adolescents with Attention-deficit Hyperactivity Disorder and Its Impact on Caregivers

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Purpose: To examine the temporal occurrence and severity of inadequate attention-deficit/hyperactivity disorder (ADHD) symptom control throughout the day and associated functional impairments and the emotional impact on caregivers specifically during the early morning routine (EMR) before school, in children and adolescents with ADHD currently treated with stable doses of stimulant medications.

Methods: An on-line, primary caregiver-completed questionnaire (n=201) was designed to determine from parent self-reports if inadequately controlled ADHD symptoms exist in children and adolescents with ADHD currently treated with stable doses of stimulants. Caregivers who identified inadequately controlled ADHD symptoms during the EMR (Likert severity rating ≥2) were asked to continue the survey by answering a series of questions on the EMR temporal period, defined as: “from the moment the child/adolescent awakens to the time they leave for school”.

Results: On a 10-point scale (1=no ADHD symptoms and 10=significant ADHD symptoms), inadequately controlled ADHD symptoms were rated as most severe during the early morning routine (6.5) and the evening homework time (6.5). The average level of overall functional impairment due to ADHD symptoms during the EMR time of day was rated by respondents as 6.1 on a 10-point scale, where 1 indicated mild impairment and 10 indicated severe impairment. Majorities of caregivers reported early morning ADHD symptoms (74%) and impairment of early morning functioning (EMF) (76%) as moderate to severe (ADHD symptom score 5-10). Easily distracted (74%), does not listen (73%), and unable to sustain attention on tasks (66%) were the ADHD symptoms reported most frequently during the EMR. Respondents reported that being impulsive (49%), fails to finish things (49%), and time awareness (44%) were the most frequent unwanted behaviors that appeared during the EMR. Parents reported that they often: felt overwhelmed and exhausted (41%), raised their voice more (37%) and felt constantly stressed (30%) as a result of their child’s ADHD symptoms during the EMR. A majority of respondents (79%) reported having previously discussed their child’s EMF impairments with their primary ADHD physician. Almost half (48%) reported that they had previously woken up early in order to administer ADHD medication to their child/adolescent in attempt to help mitigate early morning ADHD symptoms.

Conclusion: The results strongly suggest that despite early morning administration of stimulants, caregivers of children and adolescents with ADHD report a high prevalence of inadequately controlled, early morning ADHD symptoms and related functional impairments. Importantly, often the parent’s relationship with their child or adolescent with ADHD was negatively affected by these symptoms (42%). These results suggest that pharmacologic management of EMF impairments due to ADHD symptoms remains a significant unmet need in school-age children with ADHD.

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13- Safety and Efficacy of Canakinumab in Patients with Caps: Interim Results from the Beta-confident Registry

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Cryopyrin-associated periodic syndrome (CAPS) is a rare autoinflammatory disease encompassing a spectrum of 3 phenotypes with an estimated prevalence of 1-3 per million. The β-confident registry (NCT01213641) is a multicenter, long-term, observational study with an enrollment period of 5 years and a 1-year follow-up period to monitor the long-term safety and efficacy of canakinumab (CAN) for CAPS. The primary objective of the registry is to monitor the overall safety of CAN, focusing on serious adverse events (SAEs) including serious infections, vertigo, malignancies, and hypersensitivity reactions. Here we report interim data of CAN in CAPS patients from the enrollment period.

The registry protocol does not mandate any visits or procedures; however, all observed and reported AEs and SAEs or AEs potentially related to CAN are recorded. Cumulative safety data are reported as incidence rate per 100 patient-years (IR) from the enrollment of the first patient (Nov 19, 2009) until the interim data cut-off date (Dec 31, 2014). The enrollment is now complete with follow-up through Dec 2015. Although only partial data are available for 11 patients owing to the cut-off date for the analysis, the data will be updated later. Efficacy was measured using physician global assessments (PGA).

Overall, 288 patients were enrolled at 39 sites across 13 countries with mean patient exposure duration of 2.8 years. Of these, 21 (7.3%) discontinued CAN: 5 each due to AEs, poor efficacy, and patient preference; and 6 due to unknown reasons. The IR for overall AEs was 100.0. Patients with familial cold Autoinflammatory syndrome (FCAS) had the lowest IR (60.9) compared to patients with Muckle–Wells syndrome (MWS) (IR 107.2), and neonatal onset multisystem inflammatory disease (NOMID)/chronic infantile neurologic cutaneous and articular (CINCA) syndrome (IR 120.3). The most common AEs were infections and infestations (IR 36.7). Vertigo was reported by 19 patients (IR3.7). Overall, 117 SAEs were reported by 62 patients (IR15.0), most commonly, infections (IR 4.1). One death (metastatic rectal adenocarcinoma in a 76-year-old MWS patient) was reported. Of 18 patients that received pneumococcal vaccination (PPV), 13 (72%) reported local post-PPV injection site reactions (5 were considered serious). Based on PGA, nearly half the patients had no disease activity, whereas most of the others experienced mild/moderate disease activity. Similarly, disease activity was mostly absent in patients with NLRP3 mutation negative CAPS (n=14) treated with CAN. No evidence of loss of effect was observed with time.

The β-confident registry is the largest CAPS cohort documented in a registry. Canakinumab demonstrated a safety profile consistent with that observed in the clinical trial program and provided continued effectiveness in patients with CAPS for up to 5 years. Canakinumab therapy was also effective in patients with NLRP3 mutation negative CAPS.

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14- Safety and Efficacy of Tiotropium Respimat® Add-on Therapy in Pre-school Children with Symptomatic Persistent Asthma

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Purpose: Asthma is the most common chronic disease of childhood (GINA. Diagnosis and management of asthma in children 5 years and younger. 2015). The preferred controller medication in children aged ≤5 years is a low dose of inhaled corticosteroids (ICS). However, treatment options for these patients are limited if their asthma symptoms are not then well controlled on ICS. The aim of this study was to evaluate the safety and efficacy of once-daily tiotropium Respimat® in patients aged 1-5 years with symptomatic persistent asthma, as add-on to ICS with or without further maintenance therapy.
Methods: A Phase II/III, randomized, double-blind, placebo-controlled, parallel-group trial (NCT01634113) in patients aged 1-5 years with symptomatic persistent asthma. Patients received tiotropium 5 µg (two puffs of 2.5 µg), tiotropium 2.5 µg (two puffs of 1.25 µg), or placebo (two puffs), administered once daily in the afternoon for 12 weeks via the Respimat® device, each as add-on to usual maintenance therapy of ICS with or without other controller medication. The primary objective was to determine the safety of tiotropium Respimat®. Safety data included post hoc analyses of three composite exacerbation endpoints derived from adverse events (AEs), with treatment comparisons reported as hazard ratios (95% confidence interval [HR (95% CI)]). The primary efficacy endpoint was change in the weekly mean combined daytime asthma symptom score from baseline at Week 12 (response). The co-primary endpoint in 5-year-olds was peak forced expiratory volume in 1 second within 3 hours post-dose (peak FEV1(0-3h)) response. Treatment comparisons for efficacy endpoints were exploratory, and no formal hypothesis testing was performed.
Results: Of the 102 patients randomized, 101 were treated and completed the 12-week treatment period: tiotropium 5 µg, n=31; tiotropium 2.5 µg, n=36; placebo, n=34. Safety results were comparable between all treatment groups, with a lower proportion of patients reporting any AEs in the tiotropium groups (5 µg: 58.1%; 2.5 µg: 55.6%) than in the placebo group (73.5%). No AEs leading to treatment discontinuation or death were reported, and AEs were of mild or moderate intensity. Serious AEs were reported in three patients (8.8%), all in the placebo group. In post hoc analyses, both tiotropium doses reduced the risk of asthmatic exacerbation over 12 weeks compared with placebo (e.g. see exacerbation [broad] with pneumonia plus worsening in the figure). Tiotropium and placebo were associated with comparable improvements in weekly mean combined daytime asthma symptom score and peak FEV1(0-3h) responses (exploratory analyses); treatment comparisons were not performed for peak FEV1(0-3h) response because of insufficient patient numbers in each treatment group.
Conclusion: Once-daily tiotropium Respimat® add-on to ICS with or without further maintenance therapy is safe and well tolerated, and may reduce exacerbations in pre-school children with symptomatic persistent asthma.

Post hoc Cox regression analysis and Kaplan-Meier estimates of the time to first composite endpoint asthma exacerbation (broad) with pneumonia plus asthma worsening (treated set)

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15- The Impact of the State of Hydration on the Degree of Hydronephrosis in Children: Towards a Uniform Standard of Ultrasonographic Assessment

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Background:
Hydronephrosis (HN) is an imaging state affected by multiple factors, including: hydrodynamic inflow (e.g., hydration), hydrodynamic outflow (e.g., obstruction) and renal tissue compliance (e.g., elasticity). Changes in the degree of HN significantly influence clinical and surgical decision-making. Without standardized assessment, there may be risk of false positive/negative changes in degree of HN. We hypothesize that the degree of hydronephrosis seen on ultrasound is impacted by the state of relative hydration at the time of the study.
Objective:
To determine if the degree of HN seen on renal ultrasound (US) is impacted by the state of hydration at the time of the study. We propose a protocol to control outflow resistance, and provoke maximal physiologic hydration before ultrasonographic imaging for HN.
Methods:
After Research Ethics Board approval, we conducted a prospective study of children up to 18 years of age with known hydronephrosis seen for follow-up imaging in the Pediatric Urology clinic over a period of 8 months. Once consent was obtained, the initial ultrasound examination was performed. Then, the children were rapidly hydrated orally with 15-20 mL/kg fluid. Breast fed children were simply allowed to drink as much as tolerable. Children’s weight pre and post hydration has been recorded.After hydration, a second, more focused ultrasound was performed within 30-60 minutes.. The effect of bladder filling was controlled by having toilet trained children void just prior to each study, and by measuring the bladder volume sonographically in all children (Figure 1). The unaffected kidneys studied represented the control group. The change in hydronephrosis, represented by the antero-posterior diameter (APD) of the renal pelvis, was assessed statistically using a Wilcoxon paired, two-tailed t-test, and significance was determined if p < 0.05.
Results:
We aim to enroll a total of 200 patients. So far, 21 patients (mean age 74.9 ± 50.1 months), totaling 26 kidneys were included in the analysis. The unaffected 16 kidneys became the control group. Pre-hydration, the median APD was 8.1 mm (IQR 5-11), and post-hydration was 10.3 mm (IQR 7.5-16). The mean values pre- and post-hydration were 10.5 and 14.2 mm, respectively, with a mean increase of 34.8% following hydration (p < 0.001). Also, 8/26 kidneys (30.8%) had an increase of >50%, 13/26 (50%) increased by >25% while 22/26 (84.6%) showed some degree of increase. Additionally, 4/16 (25%) of the control kidneys showed de novo hydronephrosis upon hydration, with a maximum increase to 8 mm dilation of the APD. Bladder volumes were negligible in all studies.
Conclusion:
The degree of hydronephrosis is significantly impacted by hydration status at the time of the US study. We suggest that a standardized protocol of hydration should be considered when assessing renal dilatation sonographically to avoid discrepancies on the estimation of the degree of hydronephrosis.

Figure 1. Study protocol flow chart
JPEG
Figure 2. Change in AP diameter pre and post-hydration
JPEG

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16- Prospective Clinical Trial to Investigate a Novel Otc Treatment of ear Pain in Children: the Clearpop Device

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Background: The need for OTC remedies for acute otitis media (AOM) is imperative. While antibiotics remain the initial yet not preferred therapy of choice, the AAP advocates “watchful waiting”. This leaves the medical community without valid treatment options. Parents become frustrated while their child experiences pain from a blocked Eustachian tube. Study data from Clearpop® (Try This First, Inc., Walnut Creek, CA), a novel lollipop-like consumable intra-oral device, indicates a safe and effective option. Clearpop, containing primarily Vitamin C, Xylitol and natural sweeteners, works in a bio-mechanical fashion to induce saliva production thus lubricating the Eustachian tube, eliciting sucking to create negative pressure in the oral cavity. Mucus is pulled into throat while jaw movement opens the Eustachian tube.

Purpose: The purpose of the clinical trial was to demonstrate that a single use of a Clearpop device provides a safe and effective method to open up the inflamed Eustachian tube, mechanically clearing pressure and alleviating pain in children with AOM.

Methods: Children ages 3-12 were enrolled in a prospective trial at two busy pediatric clinics (Modesto, CA, Gresham, OR). When presenting with ear pain subsequently diagnosed as AOM, subjects were offered the opportunity to receive the device in-clinic on the day of presentation. Subjects were excluded if other medical conditions that might require antibiotics (e.g. strep throat) were present. The study was conducted in strict accordance with Good Clinical Practices and under ethics approval. Once enrolled, the subject was instructed to lay down with infected ear facing up, the Clearpop was placed flat side on tongue and subject instructed to suck hard until the pop dissolved, typically 20 minutes. Patients were evaluated immediately after finishing the pop, with a 2 day follow-up phone call to assess whether resolution of pain was maintained.

Results: 26 patients diagnosed with AOM were enrolled and completed detailed questionnaires pre- and post-consumption of the device in-clinic. Of these patients, 88.5% (23/26) reported immediate pain relief compared to pre-treatment pain level. At 48 hour follow-up, 78.3% (18/23) reported sustained relief and indicated they did not fill their prescription for antibiotics. All patients reported liking the taste of Clearpop and 88% of parents said they would use Clearpop again.

Conclusions: The use of a biomechanically designed, intra-oral lozenge may present an effective treatment option for the resolution of pain and fluid buildup caused by AOM.

Clearpop Device
Clearpop consumable intra-oral device
Clearpop Package
Clearpop sold OTC in package of 2 pops

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17- The Effects of a 3d Printed Prosthetic Device on Muscular Echo Intensity and Morphology in a Pediatric Amputee

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Introduction
Children's prosthetic needs are complex due to their small size, constant growth, and psychosocial development. Family finances play a crucial role in the prescription of prosthetics for their children, especially when private insurance and public funding are insufficient. For these reasons, prosthetic devices are not accessible to children from low income, uninsured families, or to children from developing countries. Advancements in computer-aided design (CAD) programs and additive manufacturing (3D printing) offer the possibility of designing and printing prosthetic hands at a very low cost. Creighton University has developed a low cost, fully customizable, wrist-driven 3D printed prosthetic device, the Cyborg Beast, to help overcome these obstacles. Over the past few years, Creighton University has designed and printed 3D prosthetic devices for nearly two-dozen children in the Omaha area, as well as for six children in Chile.

Case Report
This study employed ultrasonography (US) to analyze muscular echo intensity (EI), muscle fiber pennation angle, and muscle compartment thickness as a means to quantify the effects of using the Cyborg Beast over time. Previous studies have shown that muscle echo intensity directly reflects muscle quality, with higher densities found in healthier tissue. Similarly, muscle fiber pennation angle is a factor that determines the functional force of muscle. We captured both sagittal and coronal ultrasound images of the affected and unaffected (control) forearms. Sagittal ultrasound images were analyzed with an image-editing program (ImageJ, version 1.46, NIH) to obtain the mean echo intensity of the muscles of interest. Coronal ultrasound images were analyzed with the program Tracker (Tracker, version 4.91, OSP) to determine muscle fiber pennation angle and muscle compartment thickness. Data was gathered during three visits over the course of one year.

Discussion
After using the Cyborg Beast prosthetic device for 18 months, our subject showed an increase in EI in both affected and non-affected extensor and flexor compartments. His muscle thickness increased each visit in both limbs and in all compartments, except his non-affected extensor compartment. Lastly, our subject’s muscle fiber pennation angle showed an overall increase at each visit, except for his affected extensor digitorum muscle, which showed an overall decline in angle.

Conclusion
A prepubescent child is likely to experience both longitudinal and circumferential muscular growth, producing the increases in EI and pennation angle seen here. By tracking these changes over time, we can assess the cyborg beast in creating muscular changes, helping our users improve in strength and activities of daily living. The case study has helped develop an efficient, systematic approach to US analysis of Cyborg Beast users, which we are employing to expand our data collection and analysis to our entire Cyborg Beast population to determine the effects of using the device over time.

Subject wearing Cyborg Beast
Subject shown wearing his custom, wrist-driven, low cost, 3D printed device.

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18- A Single Dose Monoclonal Antibody Immunoprophylaxis Strategy to Prevent Respiratory Syncytial Virus Disease in All Infants: Results of the First in Infant Study with medi8897

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Purpose: RSV is the most common cause of lower respiratory tract infection (LRTI) among infants. A significant unmet need exists for RSV prevention in healthy infants. Our goal is to develop a monoclonal antibody with an extended half-life (t½) to protect infants through an entire RSV season with a single intramuscular (IM) dose. This study was conducted to evaluate the safety profile, pharmacokinetics (PK), and anti-drug antibody (ADA) responses for MEDI8897 in healthy 32 – 35 weeks’ gestation preterm infants.

Methods: Infants were randomized to receive a single IM injection of MEDI8897 10 mg (n=8), 25 mg (n=31), 50 mg (n=32) or placebo (n=18) and will be followed for 360 days. Enrollment occurred during the 2015 RSV seasons in the US, South Africa, and Chile. Blood was collected at multiple timepoints. Infants who met criteria for a medically attended (MA) LRTI had nasal samples obtained for RSV RT-PCR.

Results: At the interim analysis 30 days after the last subject was dosed, 88 of 89 infants enrolled remained in the study. Adverse events (AEs) were reported in 12/18 (66.7%) placebo and 51/71 (71.8%) MEDI8897 recipients. One serious AE (LRTI, RSV negative) was reported in a MEDI8897 recipient. Model-based simulations predict MEDI8897 median t½ to range from 83 - 94 days for the 50 mg dose in infants with normal clearance. ADA post-dose was detected in 12.5% of placebo and 4.4% of MEDI8897 recipients. MA-LRTI was reported in 4 MEDI8897 recipients, and 1 (10 mg group) was RSV positive.

Conclusions: In healthy preterm infants, the safety profile of MEDI8897 was favorable. The interim PK results support the feasibility of a single IM dose to provide protection for the duration of the RSV season.

This study was sponsored by MedImmune.

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19- Long-term Efficacy and Safety of Canakinumab in Active Hyperimmunoglobulinemia D with Periodic Fever Syndrome

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Hyperimmunoglobulinemia D with periodic fever syndrome (HIDS) is a recessively inherited disorder characterized by periodic episodes of high fever, abdominal distress, joint pain, and skin rashes; it is also known as a subtype of mevalonate kinase deficiency. Previous reports suggested interleukin (IL)-1 blockade as a potential therapy in HIDS. Here we examine the efficacy and safety of the anti-IL-1β human monoclonal antibody canakinumab in patients with active HIDS and biallelic mevalonate kinase mutations.

This open-label single treatment arm 36 month study had a 6-month treatment period (TP), an up-to 6-month follow-up period (FP) without canakinumab treatment, and a 24-month long-term treatment period (LTTP; NCT01303380). The TP included canakinumab administration (4 mg/kg q6w, max 300 mg, subcutaneously), with one permissible dose up-titration to 6 mg/kg (max, 450 mg) if a flare occurred in the first 6 weeks. In the LTTP, patients received the same canakinumab dose administered at the last visit of the TP. The primary objective was to assess reduction in frequency of flares during the TP compared with a 6-month historical treatment-free period. Secondary objectives evaluated for the entire study included assessment of reduction in frequency of flares, changes in flare duration, changes in physician’s global assessment (PGA) of flare severity and disease control, changes in plasmatic inflammatory markers, and adverse events (AEs).

All enrolled patients (n=9) completed both the TP and FP, and eight patients completed the LTTP. The median number of flares decreased from 5 (3-12) in the historical period to 0 (0-2) during TP, and persisted (0-3) until study end. During the LTTP, the median flare duration was 3.5 days (2-8) in the first year and 8.5 days (6-11) in the second. PGA of flare severity was “mild” to “moderate” at baseline (n=9) and remained the same through the TP (n=2) and FP (n=7). Flare severity reduced to “mild” or “minimal” and “mild” or “without signs/symptoms” in the first (n=4) and second years (n=2) of the LTTP, respectively. PGA disease control scores improved in all patients from either “no” or “poor” control at baseline to “good” or “excellent” control by Day 4, which persisted until study end. Plasma levels of C-reactive protein and serum amyloid A normalized by Day 15 and remained with normal values until study end. The most frequent AEs were infections. No deaths were registered. No AE led to study discontinuation. Four patients experienced 14 serious AEs (mild to moderate); none considered as drug-related.

Canakinumab treatment substantially reduced the frequency of flares in active HIDS and provoked a rapid control of signs and symptoms as well as normalization of serum inflammatory markers. No unexpected safety findings were observed through the study, and the safety profile was consistent with those reported previously.

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20- Compliance with Recommended Follow up Eye Exam After Spot Photoscreening

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Purpose: Determine follow up eye exam compliance rate for infants/toddlers referred for eye examinations based on photoscreening failures at well child checks (WCC). Determine factors contributing to compliance by analyzing patient demographics.
Methods: Infants/ toddlers (6-35 months of age) were screened using the SPOT- photoscreener (Welch Allyn, Inc.) at a pediatric outpatient clinic as part of their WCC from 7/18/2014 - 2/18/2016. Children who failed the screening were offered a follow-up eye examination at the Visual Development Laboratory (VDL) at no charge, as part of a research study. Parents of children who did not want their child to participate in a research study were referred to a community eye doctor. The number of children referred due to failed photoscreening was determined. VDL records were examined to determine the percentage of referred children who had a follow-up eye examination. We compared gender, primary language (English vs. Not English), and distance from child’s home to the VDL calculated from zip code using Chi square and t-test analyses for children who were and were not compliant with receiving a follow up eye examination.
Results: 1092 children were screened, with 295 being referred to VDL for a failed screen. Of the 295 children, only 76 (25.8%) followed up for an eye examination at the VDL. There was no significance in follow up rate by gender for children who were or were not compliant with follow up, 56.6% vs. 45.7% were female respectively, (p=0.11). English was the primary language overall, with no significant difference in the percentage of English speakers across groups who were and were not compliant with follow-up (88.5% vs. 84.2%, respectively, p=0.32). Distance to the VDL was determined for each child excluding distances more than 2 SDs above the mean (out of state addresses). The mean distance to the VDL did not significantly differ for children who were and were not compliant with a follow-up exam, (10.97 miles vs. 8.74 miles, respectively, p=0.22). Of the 76 eye exams performed at the VDL, 30 were true positives (met American Association for Pediatric Ophthalmology and Strabismus (AAPOS) referral criteria based on cycloplegic exam), 43 were false positives and 3 were incorrectly referred (referral criteria incorrectly programmed into SPOT). After excluding the 3 incorrectly referred, the true positive referral rate was 42%.

Conclusions: In this clinic population, infant-toddler follow up rate is 25.8%. The majority (74%) failed to get the recommended follow-up examination. Results indicate that gender, primary language and distance from clinic were not significant factors. Our results may have underestimated follow up rates due to families choosing to follow up at providers other than the VDL, but results suggest that additional studies are needed to determine parental barriers to follow up.

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21- A Novel Training Course in Ultrasound Peripheral Iv Access for Pediatrics Residents

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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In the pediatric population, obtaining peripheral intravenous (PIV) access can be uniquely challenging. Difficult PIV access can often lead to delayed administration of fluids and important medications. Furthermore, each additional attempt increases patient discomfort, anxiety, and risk of complications. US guided PIV placement has been shown in previous studies to increase first pass success, thus requiring fewer attempts. Pediatric residents typically have limited training and minimal experience using either a landmark based approach or Ultrasound (US) guidance for obtaining PIV access. Only 5% of participants in our study felt prepared to use US out of medical school, and 89% had never placed a PIV via US. Yet, pediatrics residents are often called upon to gain PIV access when attempts by other medical providers have been unsuccessful. Resident comfort and ability to successfully complete the procedure therefore suffers. We hypothesize that a course comprising of a focused didactic and subsequent hands-on PIV US simulation will improve resident comfort and success with PIV placement in the pediatric population. In addition, our goal is to increase procedural efficiency amongst pediatric residents through exposure to these techniques. In order to accomplish these goals and test the stated hypothesis, our team created a two-tiered course. The initial step is an introductory lecture covering basic US guided PIV techniques. Following the didactic lecture, residents will participate in a training session that utilizes PIV phantoms containing gel medium to practice US visualization of needle insertion. Of the 40-50 residents attending didactics, 32 expressed interest in the simulation course, and 19 participated in the session led by Emergency Medicine US fellows and staff. Pre and post course surveys were conducted, which demonstrated drastically improved resident comfort level with both US operation (from 11% to 68% comfortable) and use of US to obtain PIV (from 0% to 63% comfortable) following simulation. The ability to differentiate arteries and veins also improved following simulation (fro 33% to 95% comfortable). After completion of the course, all 19 participants either agreed or strongly agreed that the PIV phantom is a useful tool in practicing US PIV placement. Every participant agreed PIV access is a common issue in pediatrics. Use of the phantoms for US PIV insertion was reported to be an effective teaching method by all of the participants. Furthermore, every participant expressed interest in learning more about US PIV placement and general uses for US. Ultimately, training residents in US guided PIV insertion can hopefully prove to be a valuable skill to cultivate in pediatric training, given that timely administration of fluids and medications are needed not only in places like the Emergency Department, but also on the pediatric wards when back-up options are not readily available.

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22- The Role of Intravenous Access During Oral Food Challenges in Food Protein-induced Enterocolitis Syndrome

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Purpose
Food protein-induced enterocolitis syndrome (FPIES) is a non-IgE mediated food hypersensitivity syndrome characterized by profuse vomiting and diarrhea (V/D), which can lead to lethargy, dehydration, and hypotension. Given the potential severity of reactions, resolution of FPIES is confirmed via a physician-supervised oral food challenge (OFC) during which intravenous (IV) access is recommended in case IV fluids (IVF) and steroids are required. The objectives of this study are to evaluate the necessity for IV access during OFC and to analyze predictors for IV requirement.

Methods
Retrospective chart review was conducted on patients with ICD-9 codes 558.3/558.9 seen in Allergy & Immunology Clinic from 1/2000-10/2015. Patients without documented FPIES OFC were excluded. OFC reaction severity was scored on a Likert scale (1=mild; 3=severe). Demographics, IV placement and treatment frequency, and OFC outcomes were evaluated, and Fisher’s exact test and Wilcoxon rank sum statistical analyses performed.

Results
Of 184 patients reviewed, 28 met inclusion/exclusion criteria with 39 FPIES OFCs performed. The median age of onset was 6 months, milk was the most common food trigger, and 57% had 1 trigger. All presented with V/D, and only one (4%) had worsening severity of symptoms over time. Median age at OFC was 2.6 years, 2.2 (0.3-8.5) years since symptom onset. Of 39 OFCs, IV access was obtained in 33%, 33% experienced a reaction, but IV treatment (IVT+), including IVF and/or steroids, was required in only 7.7%. Thirty-eight (97.4%) OFCs were of equal or lesser severity than historical reactions. Median severity of presenting reaction (3(IVT+):1(IVT-); p=0.05), as well as OFC reaction (3(IVT+):0(IVT-); p=0.002), was greater in those requiring IV treatment. Additionally, patients requiring IV treatment underwent OFCs significantly younger (15(IVT+):32(IVT-) months; p=0.034) and sooner from time of diagnosis (8(IVT+):28(IVT-) months; p=0.018) compared to those without IV treatment.

Conclusion
Though FPIES can potentially elicit severe symptomatology, patients most commonly experience only V/D, which often resolves with minimal treatment. Reactions generally do not worsen over time. Only 10% of patients challenged required IV treatment, all young with severe FPIES. Therefore, it is reasonable to consider age, length of time from historical reactions, and reaction severity when evaluating the necessity of IV placement in patients undergoing FPIES OFC.

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23- Long-term Safety of Crisaborole, a Novel, Nonsteroidal, Topical, Anti-inflammatory, Phosphodiesterase 4 Inhibitor in Children and Adults with Mild to Moderate Atopic Dermatitis

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Background: Atopic dermatitis (AD) is a chronic inflammatory skin disease affecting children and adults. Approximately 85% of patients develop AD by age 5 years, and long-term topical treatment is often required. Unfortunately, topical therapies are associated with potential safety concerns and have not changed over the past 15 years. Crisaborole Topical Ointment, 2% (Anacor Pharmaceuticals, Inc., Palo Alto, CA), is a novel nonsteroidal, topical, anti-inflammatory phosphodiesterase 4 (PDE4) inhibitor currently being investigated for the treatment of AD to address the need for a more targeted and safe long-term treatment.
Objective: To assess the long-term safety results of patients as young as 2 years of age with mild to moderate AD who were included in an open-label extension study.
Methods: After completing a 28-day Phase 3 pivotal study (301: NCT02118766; 302: NCT02118792), patients who opted to continue treatment were enrolled in a multicenter, open-label, long-term, 48-week safety study (303). Every 4 weeks patients were assessed for AD severity using the Investigator’s Static Global Assessment (ISGA) scale and were treated as needed with 4-week cycles of crisaborole. Investigators initiated each on-treatment period based on severity of AD (ISGA ≥2 [Mild]). Safety measures included assessment of adverse events (AEs), serious adverse events (SAEs), vital signs, physical examinations, and clinical laboratory results.
Results: Study 303 enrolled 517 patients, who had a mean age of 11.7 years. During the open-label extension and the pivotal studies, ≥1 treatment-emergent AE (TEAE) was reported by 65% of patients; most of these were considered unrelated to treatment (93.1%) and were mild (51.2%) or moderate (44.6%) in severity. Analysis of the frequency and severity of TEAEs indicated a favorable safety profile amongst pediatric, adolescent, and adult patients. Analysis of the frequency and severity of TEAEs over time (four 12-week treatment periods) was well balanced, indicating a favorable safety profile for long-term treatment of crisaborole. None of the 9 treatment-emergent SAEs (7 of which occurred in the extension study) were considered treatment related. Treatment-related AEs were reported by 10.2% of patients; with atopic dermatitis (3.1%), application site pain (burning/stinging, 2.3%), and application site infection (1.2%) as the most frequently reported events. Only 33 patients (6.4%) interrupted or discontinued treatment because of TEAEs, and only 9 patients (1.7%) discontinued the study because of TEAEs during the long-term period. Review of clinical laboratory and vital sign results did not identify any safety signals. There were no cutaneous adverse reactions reported, such as telangiectasia, application site atrophy, or hypopigmentation. Crisaborole demonstrated a similar safety profile across age groups.
Conclusion: Crisaborole demonstrated a favorable safety profile for the long-term treatment of patients ≥2 years or older with mild to moderate AD.

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24- A Treatment Optimization Study of hld200 in Children with Attention-deficit/hyperactivity Disorder

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Purpose: This 6-week open-label, treatment optimization phase for an 11-week Phase III Clinical Endpoint Evaluation Study (CEES), examined the safety and efficacy of HLD200, a delayed- and extended-release formulation of methylphenidate (MPH) designed to be taken in the evening, before bedtime to control early morning attention-deficit/hyperactivity disorder (ADHD) symptoms before school and throughout the day, in pediatric subjects.

Methods: Males and females with ADHD (ages 6-12) were enrolled. At baseline (Visit 2), subjects initiated HLD200 at their previous MPH dose equivalent for 1 week and were dose optimized over five weeks to determine: a) optimal daily dose, and b) optimal evening dosage administration time; prior to the start of double-blind, placebo-controlled test phase at the end of Visit 8. Optimal dose was defined as safe and well-tolerated and allows for improvement from baseline of greater than or equal to 30% on the ADHD Rating Scale (ADHD-RS-IV), assessing frequency of DSM-IV ADHD symptom criteria. Similarly, the optimal evening dosage administration time was defined as one that is well-tolerated and allows for improvement from baseline (Visit 2) of greater than or equal to 30% on the Before School Functioning Questionnaire (BSFQ), a validated instrument measuring ADHD behaviors and related functioning during the early morning routine in children. This analysis will report the ADHD-RS-IV, BSFQ and the Daily Parent Ratings of Evening and Morning Behavior–Revised (DPREMB-R [AM] and [PM]) results for the dose optimization period.

Results: Forty-three subjects were included in this analysis (20 girls and 23 boys). The mean HLD200 starting dose at Visit 2 was 33.0 mg, and the mean optimal dose achieved was 65.6 mg at Visit 8. Modal evening administration time was 9 p.m. Mean baseline ADHD-RS-IV scores (±SD) at Visit 2 were 38.2±8.9 compared to mean Visit 8 scores of 12.5±6.6 (p < 0.0001). Mean BSFQ scores (±SD) at Visit 2 were 36.2±13.3 compared to mean Visit 8 scores of 10.1±7.3 (p < 0.0001). DPREMB-R AM and PM scores (±SD) also showed statistically significant differences, with an AM mean of 4.9±2.4 at Visit 2 and 1.2±1.2 at Visit 8 (p < 0.0001) and a PM mean of 15.1±5.9 at Visit 2 and 7.7±5.7 at Visit 8 (p < 0.0001). The vast majority (99.2%) of treatment-emergent adverse events (TEAEs) were judged by the investigator as mild or moderate in severity and no TEAEs led to early withdrawal. No subject suffered altered sleep patterns or breakthrough insomnia related to study drug.

Conclusions: Initial results from this 6-week treatment optimization phase indicate that when taken at bedtime, HLD200 produces statistically significant improvements of ADHD symptoms and functioning, in the early morning before school and throughout the day, as measured by the BSFQ, DPREMB-R and ADHD-RS-IV. HLD200 was generally well-tolerated, with no apparent impact on sleep.

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25- A Phase Iii Study of hld200 in Children with Attention-deficit/hyperactivity Disorder: Safety Results

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Purpose: This exploratory study consisted of a 6-week open-label, treatment optimization phase followed by a 1-week randomized, double-blind, placebo-controlled, parallel-group test phase to assess HLD200, a delayed- and extended-release formulation of methylphenidate (MPH) designed to be taken in the evening, before bedtime to control early morning attention-deficit/hyperactivity disorder (ADHD) symptoms before school and throughout the day, in pediatric subjects.

Methods: Males and females with ADHD, ages 6-12 were enrolled. At baseline (visit 2 [V2]), subjects initiated HLD200 at their previous MPH dose equivalent for 1 week and were dose optimized over five weeks to achieve optimal symptom control. At V8, subjects were randomly assigned (1:1 ratio) to double-blind HLD200 or placebo treatment for a period of 1 week. Safety endpoints were treatment-emergent adverse events (TEAEs), including elicited sleep disturbance and appetite suppression, vital signs, ECG parameters, clinical laboratory tests, physical examination and the Columbia-Suicide Severity Rating Scale (C-SSRS).

Results: There were a total of 23 males and 20 females enrolled; mean age was 9.7 years. There were no reports of TEAEs leading to early withdrawal and no treatment emergent serious AEs during the course of the study. The majority of these TEAEs were judged by the Investigator as mild or moderate in severity (99%) and possibly to certainly related to the study drug (65%). During the treatment optimization phase, 121 TEAEs were reported in 38 subjects (88%). The most commonly reported TEAEs (>10% of subjects) included: decreased appetite (35%), insomnia (23%), headache (16%), abdominal pain upper (14%) and irritability (12%). Fifteen subjects (35%) experienced a total of 15 events of decreased appetite and 10 subjects (23%) experienced a total of 11 insomnia events. In the double-blind phase, a total of 7 HLD200-(32%) and 7 placebo-treated subjects (33%) reported a TEAE. These TEAEs were judged by the Investigator to be mild or moderate in severity and possibly to certainly related to the study drug in 67% and 33% of HLD200- and placebo-treated subjects, respectively. The most commonly reported TEAE was headache (HLD200: 9%; Placebo: 10%) with no other TEAE reported more than once in any subject. No subjects experienced appetite-related TEAEs, and no HLD200-treated subjects experienced sleep-related TEAEs, whereas two placebo-treated subjects (10%) experienced insomnia-related TEAEs. Vital signs (blood pressure, temperature, respiration) were comparable between HLD200- and placebo-treated subjects. ECG measurements were within normal limits for 18 subjects in the HLD200 group and 19 subjects in the placebo group. However, there were 2 HLD200- and 1 placebo-treated subjects that demonstrated abnormal, but not clinically significant ECGs. One HLD200-treated subject experienced abnormal and clinically significant ECG results (an incomplete transient right bundle branch block).

Conclusions: When taken at bedtime, HLD200 demonstrated a favorable tolerability and safety profile in pediatric ADHD subjects.

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26- A Multifaceted Waveform High Frequency Ventilator System

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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High frequency ventilation has been in clinical use on the neonatal intensive care unit for well over a quarter century. Despite increased familiarity with ventilator mechanics and an understanding of the mechanism of function, various aspects of high frequency ventilation have not yet been completely elucidated. In particular, the optional frequency, or optional set of frequencies have not been well defined. Work by Pillow et al (AJRC, Vol. 176, No. 1 (2007), pp. 63-69) has defined the use and potential for multifaceted frequency delivery (i.e., noisy ventilation). However, there is not a current ventilator on the US market that is capable of delivering more than one frequency and harmonics of that frequency simultaneously. Purpose: We asked if by superimposing high frequency jet and oscillating ventilators, a dual "primary" frequency ventilator could be configured. Methods: A Bunnell life pulse Jet and Sensormedic 3100 oscillating ventilator were set up in tandem and connected to a copper wire test lung of known compliance. The Sensormedics 3100 was configured to deliver at frequency of 5 Hz with MAP 10 cm H20 and amplitude that was varied from 10-20 cm H20. The Jet was configured to deliver a fixed IT of 0.02 s, 240 breaths per minute, and a PIP of 20-50 cm H2O. Using varied settings of the oscillating ventilator and on the Jet, Validyne (Northridge, CA) flow and pressure sensors were used to collect data in EasySense for the PC. A MATLAB (R2014B) script was used to examine the resulting Fast Fourier Transform for peak waveform frequencies for each sample. Samples were examined for consistency of waveform between samples and the effect of manipulating jet settings. Results: Homologous multifaceted waveforms were identified as shown in the graphics. Flow and Pressure waveforms demonstrated novel composite waveforms related to the introduction of the Jet and Jet harmonics (p < 0.001). Discussion: Tandem mode ventilation may provide a consistent means of generating the requisite waveform dynamics for multifaceted waveform generation. No evidence of wave cancellation was evident in the sample. The presence of multiple harmonics of both primary frequencies may be important for the development of "noisy" ventilation. Further, this setup may be a better way to advance the state of the art than the development of a completely new ventilator. Conclusion: Multifaceted waveform ventilation is practical and achievable with conventional equipment. Clinical utility has yet to be defined.

Oscillator Alone
Oscillator at 5 Hz
Oscillator and Jet Tandem Ventilation
Both Oscillator at 5 Hz and Jet at 240 BPM or 4 Hz

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27- Impact of Telemedicine on Severity of Illness and Outcomes Among Children Transferred from Referring Emergency Departments to a Children’s Hospital Pediatric Intensive Care Unit

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Background and Objective: Telemedicine is increasingly utilized by hospitals to address disparities in the access to pediatric subspecialists. While many tele-emergency programs have been implemented in the recent past, few studies have evaluated the impact of these programs on patient outcomes. The objective of this study was to compare the severity of illness and outcomes among children admitted to a Children’s Hospital Pediatric Intensive Care Unit (PICU) from referring Emergency Departments (EDs) with and without access to a pediatric critical care consultations using telemedicine.
Methods: We analyzed data on pediatric patients ( < 18 years old) admitted to the PICU at a tertiary care Children’s Hospital directly from a referring ED between 2010 and 2014 using the Virtual PICU Performance System (VPS) database. We compared demographic factors, illness severity as measured by the PRISM III score and clinical outcomes among children receiving care in EDs with and without access to pediatric telemedicine, and in a sub-cohort of children admitted from EDs before and after the implementation of telemedicine.
Results: 582 patients from 15 EDs with telemedicine and 524 patients from 60 EDs without telemedicine were transferred and admitted to the PICU. Children transferred from EDs with telemedicine were significantly younger (5.6 vs. 6.9 years, p < 0.001), had been transported over a greater distance (72.4 vs. 63.1 miles, p < 0.05) and were less likely to be admitted during nighttime hours (56.0% vs. 63.9%, p < 0.05). However, children transferred from EDs with telemedicine had similar rates of admission during the weekend (31.8% vs. 29.2%, p=0.35) as well as mortality (4.4% vs. 2.4%, p=0.07) compared to children transferred from EDs without telemedicine. Further, patients transferred from EDs with telemedicine arrived to the PICU less ill (PRISM III score 3.2 vs. 4.0, p < 0.05) compared to patients transferred from EDs without telemedicine. After adjusting for age, transport distance, and time and day of admission, children admitted from EDs with telemedicine had lower PRISM III scores on presentation than children admitted from EDs without telemedicine (β = -0.74, 95% CI= -1.46 to -0.01). Among transfers from EDs that established telemedicine programs during the study period, children arrived significantly less sick (mean PRISM III scores 1.2 units lower, p=0.03) after the implementation of telemedicine (N=43) than before the implementation of telemedicine (N=95).
Conclusions: The implementation of a telemedicine program designed to assist in the care of seriously ill children receiving care in referring EDs was associated with lower illness severity upon admission to the PICU. Telemedicine programs assist referring EDs in the care of critically ill children and could result in improved clinical outcomes.

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28- A Randomized, Double-blind, Vehicle-controlled Study of the Ovicidal Efficacy of a New Head Lice Therapy, Abametapir Lotion 0.74%, Administered for the Treatment of Head Lice Infestation

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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PURPOSE
Current head lice treatments primarily target hatched lice, leaving the eggs relatively unaffected. Consequently, a second application is typically required 7-10 days after the first treatment to kill subsequently hatched lice. Abametapir is a novel compound that inhibits various metalloproteinases; these enzymes are critical for both egg development and the survival of crawling lice. As such, abametapir lotion has the potential to be effective in eliminating both live lice and nits with a single 10 minute application. The objective of this clinical study was to assess the ovicidal efficacy against head louse eggs of abametapir lotion 0.74% following a single 10-minute application.

METHODS
This double-blind, randomized, vehicle-controlled, single-dose study enrolled subjects aged ≥3 years with an active head lice infestation, defined as a minimum of 3 live head lice and ≥10 undamaged and unhatched head lice eggs. On Day 0, ≥5 viable eggs were removed from each subject to serve as a control. Subjects were randomized to either abametapir lotion 0.74% or vehicle treatment, which was applied to dry hair for 10 minutes before rinsing with warm water. No nit combing was performed. After treatment, the random egg collection process was repeated. Eggs deemed viable were incubated for 14 days (30°C/60% relative humidity), and hatch rate was assessed. The proportion of hatched eggs from pre-treatment versus post-treatment collection was compared between groups. Subjects were assessed for the presence or absence of lice at Days 1 and 7 post-treatment, primarily for ethical reasons (to allow rescue therapy if necessary).

RESULTS
Overall, 50 subjects were randomized 1:1, with ages ranging from 3 to 17 years (mean 8.5 years). Following treatment with abametapir lotion and the 14-day incubation period, 100% of treated eggs remained unhatched, compared with 64% of vehicle-treated eggs. When comparing egg hatch rate before versus after treatment, the abametapir lotion group had a 92.9% reduction in hatch rate (95% CI: 86.5-99.4) compared with 42.3% (95% CI: 30.2-54.4) for the vehicle group. The treatment difference in the absolute reduction in hatch rate (abametapir lotion minus vehicle) was 50.6% (p < 0.0001, 95% CI: 36.9-64.3). Post-treatment lice assessment showed 22 subjects (88%) in the abametapir group were free of lice at the Day 1 and 7 follow-up visits, compared with only 8 subjects (32%) in the vehicle-treated group.

CONCLUSION
Abametapir lotion 0.74% inhibited 100% of treated head lice eggs from hatching following a single 10 minute application. These results, in conjunction with data obtained in two pivotal Phase 3 studies, confirm that abametapir possesses both significant lousicidal and ovicidal activity―considered critical for the overall treatment of head lice infestations―with a single application.

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29- Antibiotic Resistance Among Ocular Bacterial Pathogens from Pediatric Patients in the Armor Surveillance Study 2009-2015

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Purpose: Antibiotic resistance may be problematic in the treatment of bacterial eye infections. Data from large, multi-center, multi-year surveillance studies are useful for tracking resistance to commonly used antibiotics. The Antibiotic Resistance Monitoring in Ocular Microorganisms (ARMOR) study is the only ongoing nationwide antibiotic resistance surveillance program specific to ocular bacteria. We report antimicrobial susceptibility data for isolates collected from pediatric patients from 2009 through 2015.

Methods: Clinical centers across the United States submitted ocular isolates of Streptococcus pneumoniae, Haemophilus influenzae, Staphylococcus aureus, coagulase-negative staphylococci (CoNS), and Pseudomonas aeruginosa from the cornea, conjunctiva, and aqueous or vitreous humor. Minimum inhibitory concentrations (MICs) were determined by broth microdilution methodology for up to 15 antibiotics according to the Clinical and Laboratory Standards Institute guidelines. Isolates were categorized as susceptible or non-susceptible (intermediate and resistant) based on breakpoints for systemic isolates, where available.

Results: A total of 137 S. pneumoniae, 261 H. influenzae, 241 S. aureus, and 186 CoNS isolates were collected from pediatric patients ≤ 17 years of age during the 7-year period. Resistance in S. pneumoniae was most notable for azithromycin (38%) and penicillin (36%), while resistance in H. influenzae was very low. Among the staphylococci (S. aureus and CoNS), 56-71% were resistant to azithromycin and 27-48% were methicillin-resistant (MR) ; multidrug resistance to ≥ 3 drug classes (MDR) was particularly prevalent among MR strains (47% and 70%, respectively). Among the patient subpopulation aged 0-3 years, besifloxacin was the most potent ophthalmic agent against pneumococcal and staphylococcal strains and was comparable to other fluoroquinolones against H. influenzae (Table). Many S. aureus and S. pneumoniae strains appear to be highly resistant to commonly used topical antibiotics.

Conclusion: Based on MIC data, antibiotic resistance appears common among S. pneumoniae and S. aureus isolates collected from pediatric patients, except for besifloxacin, moxifloxacin, and gatifloxacin. Because culture of common eye infections is rarely performed, these susceptibility data maybe especially important when selecting second-line agents for treatment in pediatric patients. The role of S. aureus in routine pediatric eye infections warrants further investigation.

MIC90 values (in µg/mL) for common ophthalmic agents against bacterial isolates collected from patients 0-3 years of age
BES – besifloxacin; MXF – moxifloxacin; GAT – gatifloxacin; CIP – ciprofloxacin; LVX – levofloxacin; OFX – ofloxacin; AZM – azithromycin; PB – polymyxin B; TOB – tobramycin; TMP – trimethoprim

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30- Metabolomic Differences Between Mothers’ Own Breast Milk and Donor Breast Milk

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Background:
Human milk is nutritionally complete, is the ideal food for a newborn infant, and conveys many benefits to all infants including sick preterm infants. Mothers’ own breast milk (MBM) has many advantages as it is tailored for the mother-infant couple. Donor breast milk (DBM) has been thought to be a reasonable alternative to MBM when MBM is unavailable. To our knowledge, there have been no reports comparing donor breast milk to mothers’ own breast milk at the biochemical and micronutrient level. In this study, we compared the composition of mothers’ own breast milk to donor breast milk using a metabolomic approach.

Methods:
Human milk samples were collected from mothers who were breastfeeding or providing milk to their healthy term or preterm infants at around two weeks after delivery (n=48). The milk samples were frozen and stored at -80°C until analysis. Donor breast milk samples (n=8) were taken from four different lots supplied to our NICU. Human milk analysis was performed by Metabolon Inc. (Durham, NC). Global metabolomic profiles were analyzed and relative abundance of compounds were determined.

Results:
A total of 720 biochemicals were identified in the breast milk samples, 565 of known identity and 155 of unknown structural identity. In principal component analysis, DBM samples tended to form a separate, but neighboring, population to the MBM groups. Using a threshold of significance at p < 0.05, preterm MBM, compared to DMB, showed 165 compounds that had elevated abundance and 128 compounds that had decreased abundance; term MBM showed 181 biochemicals that had elevated abundance and 183 biochemicals that had decreased abundance. The majority of differences were seen in the lipid pathways. Medium-chain fatty acids and long-chain fatty acids were decreased in abundance in term MBM compared to DBM; no significant differences were observed between preterm MBM and DBM for these biochemicals. Carnitine-conjugated fatty acids and complex lipids also had elevated abundance but monoacylglycerols had decreased abundance in MBM compared to DBM.

Conclusions:
In summary, this comprehensive metabolomics study shows that the biochemical profiles of donor breast milk is distinctively different compared to MBM, particularly in the lipid metabolites. While the significance of these biological differences remains to be determined, this information provides the fundamental basis for future studies of human milk.

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31- Quantification of the Distribution of Azelastine Hcl/fluticasone Propionate Nasal Spray in an Anatomical Model of the Human Nasal Cavity

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Purpose. In vitro evaluations using an anatomical model of the human nasal cavity quantified the distribution of azelastine HCl (AZ) and fluticasone propionate (FP) in a single nasal spray (Dymista) compared to sequential sprays of marketed azelastine (Astelin) and either Flonase or generic fluticasone.
Methods. The cast was divided into 4 sections from anterior to posterior of the cast. A single spray of AZ/FP (0.137 mL [137 mcg of azelastine/50 mcg of fluticasone propionate]) or sequential single sprays of azelastine (0.137 mL) followed by generic fluticasone propionate or Flonase nasal spray (0.100 mL) were manually actuated into the model. A vacuum (15 mL/min) was applied during actuation to simulate nasal inhalation. Following extraction from the nasal cast, HPLC was used to quantify drug deposition on the different sections of the cast. Each experiment was repeated three times.
Results. A single spray of AZ/FP showed a uniform distribution of close to 100% of applied drug within the nose/nasal valve and turbinates (first 2 sections of the cast); the average % AZ was 61.4% in section 1 and 38.6% in section 2 and the average % FP was 65.4% and 34.6% in sections 1 and 2, respectively. In comparison, single sprays of the individual agents showed uneven distribution of AZ and FP and a substantial amount of dripping from sequential administration.
Conclusions. Application of AZ/FP in a single spray provided more uniform distribution and greater retention in the nasal cavity than sequential sprays of the individual components, potentially allowing for better local absorption of medication.

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32- Multichannel Electroencephalography in the Neonatal Intensive Care Unit Using Microeeg, an Ultra-compact Wireless Device

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Purpose: First, to determine the feasibility of an ultra-compact wireless device (microEEG) to obtain multichannel electroencephalographic (EEG) recording in the Neonatal Intensive Care Unit (NICU). Second, to identify problem areas in order to improve wireless EEG performance.

Methods: 28 subjects (gestational age 24-30 weeks, postnatal age < 30days) were recruited at 2 sites as part of an ongoing study of neonatal apnea and wireless EEG. Infants underwent 8-9 hour EEG recordings every 2-4 weeks using an electrode cap (ANT-Neuro) connected to the wireless EEG device (Bio-Signal Group). A 23 electrode configuration was used incorporating the International 10-20 System. The device transmitted recordings wirelessly to a laptop computer for bedside assessment. The recordings were assessed by a pediatric neurophysiologist for interpretability.

Results: A total of 84 EEGs were recorded from 28 neonates. 61 EEG studies were obtained in infants prior to 35 weeks corrected gestational age (CGA). NICU staff placed all electrode caps and initiated all recordings. Of these recordings 6 (10%) were uninterpretable due to artifacts and one study could not be accessed. The remaining 54 (89%) EEG recordings were acceptable for clinical review and interpretation by a pediatric neurophysiologist. Of the recordings obtained at 35 weeks corrected gestational age or later only 11 out of 23 (48 %) were interpretable.

Conclusion: Wireless EEG devices can provide practical, high-quality, continuous, multichannel EEG monitoring in preterm neonates. Their small size and ease of use could overcome obstacles associated with EEG recording and interpretation in the NICU.

The microEEG device
Flow diagram of the EEG recording process.

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Oral (Podium)

33- Patient Characteristics and Treatment Patterns Among Pediatric Patients with Asthma

Monday, October 24
12:00PM - 2:00PM
Moscone West, 2nd Floor Foyer

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Objective: Asthma prevalence, especially among children, is increasing globally. Nearly 7.1 million children in the US currently have asthma. Given limited research on treatment patterns in the pediatric population, the aim of this study was to examine treatment regimens and adherence to the National Heart Lung and Blood Institute (NHLBI) stepwise approach for asthma management among pediatric patients.
Methods: The study population included asthma patients < 12 years old initiating controller medication between 1/1/2011 and 2/28/2015 from a large US administrative claims database. Asthma was defined based on the presence of ICD-9 codes 493.00-493.02, 493.10-493.12, 493.82 or 493.90-493.92 on ≥2 separate service dates. Patients had ≥2 years of continuous health plan enrollment after controller initiation; those with cystic fibrosis, bronchitis, emphysema, chronic obstructive asthma, or chronic airway obstruction were excluded. Lines of therapy (LOTs) were identified based on ICS dose and controller medication class. A LOT continued until a change in ICS dose or a controller was added or removed. Controller medications within a LOT were used to classify regimens into a NHLBI Step according to the NHLBI Expert Panel Report 3 Stepwise Approach for Managing Asthma. Regimens not corresponding to a NHLBI Step were categorized as “Other.”
Results: The pediatric asthma population included 10,309 patients aged 0-4 years and 20,136 patients aged 5-11 years. Among patients aged 0-4 years, mean ± standard deviation age was 2.9±1.1 years and 64.4% were male. Among 5-11 year olds, mean age was 8.4±2.0 years and 62.2% were male. Among patients aged 0-4 and 5-11 years, just 48.9% and 60.1% were on controller medication(s) and had a LOT at the beginning of year 2, respectively. For the LOT at the beginning of year 2, the proportion of regimens classified within each NHLBI Step for 0-4 and 5-11 year olds, respectively, was: Step 2 (48%, 59%), 3 (16%, 13%), 4 (3%, 3%), 5/6 (6%, 7%) and “Other” (27%, 18%). Among regimens classified as “Other,” 78.4% of LOTs among 0-4 year olds, and 73.4% among 5-11 year olds were high-dose ICS monotherapy, with average LOT durations [ALD] of 229 and 164 days, respectively; while 7.9% of LOTs among 0-4 year olds, and 16.1% among 5-11 year olds were on combination regimens including high-dose ICS, with ALD of 117 and 180 days, respectively.
Conclusion: In this real-world population of pediatric asthma patients on controller therapy, 27% of 0-4 year old and 18% of 5-11 year old patients were on regimens inconsistent with the NHLBI Stepwise Approach for Managing Asthma, of which a large proportion of patients were on high-dose ICS monotherapy. Given the potential risks associated with long-term use of high doses of ICS in children, these data suggest a need for additional controller medications for young asthmatics.

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Oral (Podium)

A Randomized Controlled Trial on the Effectiveness of Oral Zinc Supplementation in Augmenting Tuberculin Skin Test Induration Among Malnourished Children with Pulmonary Tuberculosis Infection

Monday, October 24
12:10PM - 12:20PM
Moscone West, 3020

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Tuberculin skin test is central in diagnosing Tuberculosis, especially in malnourished children who are prone to false-negative skin test results. Zinc supplementation plays a role in the generation and functioning of the immune system and correct false negative tuberculin skin test reactions by correcting Zinc deficiency, commonly seen in malnourished children. The study aimed to determine the effectiveness of Zinc in augmenting tuberculin skin test induration among malnourished children with Pulmonary Tuberculosis infection. It is community-based, double blind, placebo-controlled, randomized study. Fifty malnourished children aged 2-7 years old with BMI of z-score below -2 were included and randomly divided by into two groups. One group was given 5 ml oral glucose water, once a day, for two weeks. The other group was given Zinc syrup (20 mg elemental zinc sulfate) 5 ml, once a day, for two weeks. After two weeks, the investigator injected Tuberculin (5TU in 0.1 ml) on the proximal volar surface of the forearm of each participant. A health worker read the tuberculin skin test result in a blinded manner, 48-72 hours after administration. A 5 mm induration was considered positive. 47 participants came back for Tuberculin skin test reading. Twenty-one (45%) had positive tuberculin skin test reaction, 15 (65.2%) participants belonging to Zinc group and 6 (25%) belonging to placebo. In terms of induration size, those belonging to Zinc group had more significant induration (7± 5.9 mm) as compared to placebo (2.2±4.2 mm). Based on Mantel Haenzel test, there is a significant difference in the outcome response between the two groups with p value set at < 0.05. In conclusion, Oral Zinc supplementation enhanced the tuberculin skin test response as seen by higher positive responders and bigger mean induration size as compared to placebo.

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Fast and Sensitive: Automated Point-of-care Urine Dips

Monday, October 24
12:20PM - 12:30PM
Moscone West, 3020

Take Evaluation

Purpose
Point-of-care (POC) urine dipstick is a highly utilized test in the pediatric emergency department (PED) due to its fast turn-around time (2 minutes) and inexpensive cost ( < $1). Limited literature exists evaluating the accuracy of POC urine dipstick and urinalysis for the evaluation of children with possible urinary tract infection (UTI) in the PED. Past studies have shown hand-held urine dipsticks and automated urinalysis in children younger than 48 months to be sensitive predictors for UTI. No studies were found comparing current automated urine dipstick analysis technology, urinalysis and urine culture in the PED.

Methods
A retrospective chart review was conducted on patients (ages birth through 18 years) presenting to an urban PED between January 2015 and December 2015. All patients had a POC urine dipstick, urinalysis and urine culture performed as part of their evaluation for UTI. A convenience sample of 120 randomly selected charts was reviewed. A positive POC dipstick was defined as having positive leukocytes or nitrites and a positive urinalysis was defined using the same criteria. Urine culture was used as the gold standard in this study (≥105 colony forming units (cfu)/ml).

Results
The 120 subjects were 85% female, 16% African American, 41% Hispanic with a mean age of 6.8 years (SD 5.4). The sensitivity and positive predictive value were greater for POC dipstick than laboratory urinalysis (Table 1).

Conclusion
Our data suggests that the automated urine dipstick is more sensitive at detecting a UTI when compared to the urinalysis. For this reason the automated POC urine dipstick may be a fast and reliable way to screen children for a possible UTI.

Table1

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Oral (Podium)

Abametapir Lotion 0.74% Demonstrates High Elimination Rates of Head Lice with a Single Application in Phase 3 Trials

Monday, October 24
12:30PM - 12:40PM
Moscone West, 3020

Take Evaluation

PURPOSE
Head lice (pediculosis capitis) affects approximately 6-12 million children in the United States each year, with resistance to currently available head lice treatments increasing. The majority of these treatments require two applications, applied 7-10 days apart. Abametapir lotion 0.74% is a new single-application product for the treatment of head lice infestation. A Phase 2b study conducted in children aged ≥2 years demonstrated that a single application of abametapir lotion (0.74% w/w) resulted in 86% of subjects being lice-free at 14 days post-treatment [1]. These results supported further investigation of abametapir lotion in Phase 3 trials.
Our objective was to evaluate the efficacy of a single application of abametapir lotion 0.74% for the treatment of head lice infestation in subjects as young as 6 months.

METHODS
Two randomized, double-blind, vehicle-controlled, parallel-group studies were conducted at multiple sites in subjects aged ≥6 months. Subjects with active head lice infestation of ≥3 live lice (index subjects) along with household members with ≥1 live louse (non-index subjects) were eligible for enrollment. Households were randomized to receive either abametapir lotion 0.74% or vehicle. Product was applied at home on Day 0 to dry hair for 10 minutes, then rinsed with water. No nit combing was performed. Subjects were inspected for live lice on Days 1, 7, and 14 post-treatment. The primary endpoint was the percentage of index subjects in the intent-to-treat (ITT) population who achieved treatment success, defined as being louse-free at all post-baseline visits through to Day 14.

RESULTS
A total of 704 subjects were enrolled in the two Phase 3 trials, including 216 index subjects (108 in each study, ITT). Treatment groups were similar in age, gender, and hair length, texture, and volume. Treatment success was achieved in significantly more index subjects receiving abametapir lotion compared with vehicle (study 1: 81.1% vs 50.9%, p=0.001; study 2: 81.8% vs 47.2%, p < 0.001). For all subjects (index and non-index), treatment success was 88.2% (165/187) and 81.0% (132/163) with abametapir lotion for the two trials, respectively, compared with 62% (119/192) and 60.5% (98/162) for the vehicle groups (both p < 0.001).

CONCLUSION
Abametapir lotion 0.74% was significantly more effective than vehicle in clearing head lice infestation following a single 10 minute at-home application. These results confirm the Phase 2b findings of high elimination rates of head lice with abametapir in children as young as 6 months of age.

1. Bowles VM, et al. A novel treatment for the control of head lice and their eggs following a single application. AAP National Conference. October 20-23, 2012, New Orleans, LA. Abstract#16372.

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Send Email for Scott Arnold

Juan Ignacio Arostegui

Department of Immunology-CDB, Hospital Clínic, Barcelona, Spain.
Barcelona, Spain

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Lindsay Bengtson, PhD, MPH

Optum
Eden Prairie, MN

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Stanley Block, MD, FAAP

Pediatrician
Professor of Clinical Pediatrics, University of Louisville, and University of Kentucky; the President, Kentucky Pediatric and Adult Research Inc.
Bardstown, Kentucky

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Joseph Bowens, M.S.

Medical Student
Creighton University School of Medicine
Omaha, NE

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John Calcagno, MD

Pediatrician
Calcagno Pediatrics
Gresham, OR

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Maria Ruby Rose Castaneda, MD

Pediatrician
University of Santo Tomas Hospital
Makati City, Philippines

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Joseph Domachowske, MD

SUNY Upstate Medical Universtiy
Syracuse, NY

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Joana Dos Santos, Assistant Professor of Paediatrics

Pediatrician, medical urology staff
The Hospital for Sick Children , University of Toronto
Toronto, ON, Canada

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Kimberly Gerhart, MD

Assistant Professor
University of Arizona
Tucson, AZ

Presentation(s):

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Mitchell Goldstein, MD

Loma Linda University Children's Hospital
Loma Linda, California

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Benjamin Harding, MD

Neonatology Fellow
Loma Linda University Children's Hospital
Loma Linda, CA

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Don Hayes, Jr., MD

Nationwide Children's Hospital and The Ohio State University
Columbus, OH

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Lydie Hazan, MD

Axis Clinical Trials
Los Angeles, CA

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Hal Hoffman, MD

Professor of Pediatrics
University of California at San Diego, La Jolla, CA, United States,
UCSD
La Jolla, CA

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Andrew Huang, MD

Medical Resident, Department of Radiation Oncology
California Pacific Medical Center
San Francisco, CA

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Zachary Ibrahim, MD

Assistant Professor of Pediatrics
SUNY Downstate Medical Center
Brooklyn, NY

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Allison Judkins, MD, FAAP


Loma Linda University Children's Hospital
Loma Linda, CA

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Todd Mahr, MD

Gundersen Lutheran Medical Center
La Crosse, Wisconsin

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Laurie Malia, DO

Pediatric Emergency Medicine Fellow
Connecticut Children's Medical Center
Hartford, CT

Presentation(s):

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Mary Ann McDonnell, PhD

South Shore Psychiatric Services
Marshfield, MA

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Mary Ann McDonnell, PhD

South Shore Psychiatric Services
Marshfield, MA

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Christine Mulvey, MA

Research Associate
Connecticut Children's Medical Center
Bethlehem, CT

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Sudha Rani Narasimhan, MD, IBCLC

Neonatologist
Santa Clara Valley Medical Center
San Jose, California

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Anthony Portale, MD

Professor of Pediatrics
University of California San Francisco, Department of Pediatrics
San Francisco, CA

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Kyra Reed, MD

Indiana University School of Medicine
Indianapolis, IN

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Laura Roberts Pena, MD

Baylor College of Medicine, Department of Pediatrics
Kingwood, TX

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Earl Seltzer

Therapeutic Strategy Lead
Quintiles
Durham, NC

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David Skoner, MD

Director, Allergy and Immunology, Department of Pediatrics, West Virginia University School of Medicine
Allegheny General Hospital
Pittsburgh, PA

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Dongli Song, MD, PhD

Neonatologist
Santa Clara Valley Medical Center
San Jose, California

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Mark Vandewalker, MD

University of Copenhagen, CO

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Jordi Anton

Unitat de Reumatologia Pediàtrica, Hospital Sant Joan de Déu, Barcelona, Spain.
Barcelona, Spain

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William Berger, MD

Allergy and Asthma Associates of Southern California
Mission Viejo, CA

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Hans Bisgaard, MD

University of Copenhagen, Department of Clinical Medicine, Gentofte Hospital, Denmark

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Geetha Chari, MD

Associate Professor of Pediatric Neurology
SUNY Downstate Medical Center
Brooklyn, NY

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Heleen DeCory, PhD

Director, Pharmaceutical Medical Affairs
Bausch + Lomb
Rochester, NY

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Mitchell Goldstein, MD

Loma Linda University Children's Hospital
Loma Linda, California

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Stanley Goldstein, MD

Allergy and Asthma Care of Long Island
New York, NY

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Aaron Goshinska, MS3

University of Arizona
Tucson, AZ

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Danielle Guffey, MS

Research Associate, Biostatistician
Baylor College of Medicine, Institute for Clinical and Translational Research
Houston, TX

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Robert S Haber, MD

Professor of Dermatology and Pediatrics
Case Western University School of Medicine
Clevelend, OH

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Karen Huang, M.S.

Harvard School of Public Health
Long Beach, CA

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Jason Kinchen, PhD

Metabolon Inc.
Durham, NC

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Jasmin Kuemmerle-Deschner

University Hospital Tuebingen, Tuebingen, Germany,
Tuebingen,, Germany

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Jeff Maca, Ph.D.

Principal Scientific Advisor
Quintiles
Durham, NC

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Jessica Marvel, MPH

Novartis Pharmaceuticals Corporation
East Hanover, NJ

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Luyen Pham, MD

Pediatrician
Central Valley Medical Group
Modesto, CA

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David Rayburn, MD

Indiana University School of Medicine
Indianapolis, IN

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Kaitlin Strumph, DO

Resident
Connecticut Children's Medical Center
University of Connecticut
Hartford, CT

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Joseph Tobias, MD

Nationwide Children's Hospital and The Ohio State University
Columbus, Ohio

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Carter Tong, RRT

Loma Linda University Children's Hospital
Loma Linda, California

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Richard Young, MD, MPH

Assistant Professor of Pediatrics and Neurology
University of Connecticut School of Medicine
Hartford, CT

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Fadi Zu'bi, Urology Research Fellow

The Hospital for Sick Children, University of Toronto
Toronto, ON, Canada

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Jorge Zuniga, Ph.D.

Assistant Professor
Creighton University Department of Exercise Science
Omaha, NE

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Samah Abdel Baki, MD

Chief Executive Officer
Biosignal Group
Brooklyn, NY

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John Brancato, MD

Pediatric Emergency Medicine Attending
Connecticut Children's Medical Center
Hartford, CT

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John Brancato, MD

Pediatric Emergency Medicine Attending
Connecticut Children's Medical Center
Hartford, CT

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Inmaculada Calvo-Penedes

Hospital de La Fe, Valencia, Spain.
Valencia, Spain

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Ann Childress, MD

Center for Psychiatry and Behavioral Medicine, Inc.
Las Vegas, NV

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Ann Childress, MD

Center for Psychiatry and Behavioral Medicine, Inc.
Las Vegas, NV

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Elba Fayard, MD

Loma Linda University Children's Hospital
Loma Linda, California

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Maggie Fleita

Undergraduate Student
Creighton University Department of Exercise Science
Omaha, NE

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Erin Harvey, PhD

University of Arizona
Tucson, AZ

Presentation(s):

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Philip Hawkins

Division of Rheumatology, Allergy, and Immunology, University College London Medical School, London, United Kingdom,
London, United Kingdom

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Asal Hojjat, Urology Research Fellow

The Hospital for Sick Children, University of Toronto
Toronto, ON, Canada

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Michael Husseman, MD

Wee Care Pediatrics
Layton, UT

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