Professor and Vice Chair for Research University of Washington Seattle, Washington
Rationale: In June 2020 the U.S. Food and Drug Administration approved fenfluramine (FINTEPLA) for the treatment of seizures associated with Dravet syndrome (DS). An interim analysis of a long-term open label extension study of fenfluramine (FFA) indicated that FFA provides meaningful reductions in seizure frequency for up to two years in patients with DS. The purpose of this study was to collect rich qualitative data to further understand in greater detail how FFA treatment affects DS patients and their families. Methods: Study participants were parents caring for a child with DS who had participated in the FFA clinical trial. The caregivers participated in one-on-one semi-structured interviews to discuss the benefits of FFA on the child with DS, the parent, and the family unit. They also provided information about their child with DS and completed a self-report survey about their own health and quality of life. Results: A total of 21 parent caregivers have participated in interviews to date. Average age of caregivers was 47 years, 76% were female, and 91% lived with a partner or spouse. The mean age of the child with DS was 14 years (range, 4-31 years) and they had been on the FFA regimen an average of ten months (range, 5-25 months).
When asked to describe the benefits they observed with their child on FFA, responses could be grouped into seizure-related benefits and non-seizure-related benefits. The four most commonly cited seizure-related benefits noted by parents/caregivers were: a reduction in seizure activity, fewer seizure triggers, shorter post-ictal recovery times, and improved post-seizure function (Figure 1). The most commonly cited non-seizure-related benefits included those related to: cognition, alertness, problem solving, education, child happiness, motor function, sleep quality, and speech (Figure 2). When describing the impact of FFA treatment on their own lives, parents reported feeling less overwhelmed (71%), less stress (71%), and less anxiety and depression (67%). The quality of their sleep improved (71%) and they had more time to do things they enjoy (57%). About 50% of parents reported that the relationship with their spouse or partner improved, and 77% of employed parents missed less work. Parents also reported less family stress (76%), improved relationship between the child with DS and their siblings (71%), improved behavior and/or mood of siblings (77%), and that it was easier for their family to do things with others (62%). Conclusions: Parents with a child with DS reported a large number of both seizure-related and non-seizure-related benefits of FFA treatment for their child, for themselves and for their family. Many reported feeling hope for the first time since the child was born or diagnosed with DS. The study found that FFA treatment is associated with meaningful increases in many quality of life domains for patients with DS who receive FFA, as well as their families. Funding: Please list any funding that was received in support of this abstract.: Research reported in this abstract was supported by a contract awarded to the University of Washington by Zogenix, Inc., under contract #ZXIIS2019-001. Click here to view image/table