PB0683 - Current landscape of prophylaxis and tolerisation in patients with Haemophilia A in Australia

Monday, July 11, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Presenting Author(s)

Sumit PARIKH, PhD

Allied Health Professional
Australian Haemophilia Centre Director's Organisation
Malvern East, Victoria, Australia

Co-Author(s)

SP

Stephanie P’NG

Haematologist
Fiona Stanley Hospital, Perth, WA, Australia
Perth, Western Australia, Australia
SB

Simon BROWN

Director
Queensland Children's Hospital
Brisbane, Queensland, Australia
CB

Chris BARNES

Clinical Director
Royal Children Hospital, Melbourne, VIC, Australia
Melbourne, Victoria, Australia
CT

Chee Wee TAN

Clinical Director
SA Pathology, Adelaide, SA, Australia
Adelaide, South Australia, Australia
TC

Tina CARTER

Director
Perth Children's Hospital
Perth, Western Australia, Australia
HT

Huyen Tran

Professor
Australian Centre for Blood Diseases, Monash University
Melbourne, Victoria, Australia

Background: Prescribing prophylaxis in patients with Haemophilia A (HA) is a well-established standard of care, helping to reduce or abolish frequency of bleeds, maintain joint health and improve quality of life. Emerging trends and availability of various factor (F8), including Standard Half-Life (SHL) and Extended Half-Life (EHL), and non-factor replacement (emicizumab) therapies provide an opportunity to assess changing landscape of prophylaxis and tolerisation in the Australian cohort.

Aims: To characterise current practice in HA patients prescribed prophylaxis and/or Immune Tolerance Therapy (ITT) in Australia.

Methods: Data was derived from the Australian Bleeding Disorder Registry (ABDR) on consented HA patients. Data was obtained on patient diagnosis, severity, age group, treatment product, treatment regimen and inhibitor status. Patient demographics and results presented are based on ABDR data up until December 2021.

Results: There are currently 2346 active HA patients in the ABDR. 87.1% (637/731) of severe HA patients and 35.7% (86/241) of moderate HA patients are prescribed prophylaxis. Breakdown of severe and moderate HA patients on prophylaxis are 61.6%, 21.3% and 17.1% for using emicizumab, EHL & SHL respectively. 9 non-inhibitor HA patients (all severe) are prescribed both, F8 and emicizumab. A significant proportion (76.2%) of paediatric patients with severe HA are prescribed emicizumab as prophylaxis compared to 48.1% of severe adult HA population with the majority of all patients on fortnightly dosing. Among the 70 patients with F8 inhibitors, 17 are on-demand treatment, 3 are on ITT using F8 only and 50 are on emicizumab (including 4 that are also prescribed F8). There were no serious adverse events reported in the ABDR for Year 2021 caused by any F8 product or emicizumab.

Conclusion(s): Improved availability of various treatment options has led to an increased uptake of HA patients prescribed prophylaxis in the last few years. Further information is required to examine treatment guidelines and clinical outcomes.