Prophylaxis and tolerisation in haemophilia A

SUMIT PARIKH

Sumit Parikh is the AHCDO ABDR Senior Research Fellow

The International Society for Thrombosis and Haemostasis (ISTH) reconvened for their annual scientific meeting this year in July in London, UK. It was the first ISTH Congress with an in-person component since 2019. There was renewed enthusiasm among the health professionals to present and share their ground-breaking research and latest developments. Australian Haemophilia Centre Directors’ Organisation (AHCDO) presented one abstract this year – Current landscape of prophylaxis and tolerisation in patients with haemophilia A in Australia.  This was drawn from Australian Bleeding Disorders Registry (ABDR) data.

Sumit Parikh at ISTH 2022 in London
Photo: Sumit Parikh

Prescribing prophylaxis is a well-established standard of care in patients with haemophilia A in Australia. Emerging trends and availability of various factor VIII (8) concentrates, including Standard Half‑Life (SHL) and Extended Half‑Life (EHL), and non-factor replacement (emicizumab) therapies provided an opportunity to assess the current landscape of prophylaxis and tolerisation in Australia. The main aims of this study were to review current practice in prescribing prophylaxis and tolerisation among haemophilia A patients in Australia and observe the trend over the last 7 years. 

One of the most record-breaking outcomes was that 87.1% of severe haemophilia A patients and 35.7% of moderate haemophilia A patients are currently on prophylaxis in Australia. This demonstrates a rising trend in the proportion of patients prescribed prophylaxis over the last 7 years, when the proportions were 81.8% for severe haemophilia A patients and 25.1% for moderate haemophilia A patients in 2015. A significant proportion (76.2%) of children and young adolescents with severe haemophilia A on prophylaxis are prescribed emicizumab compared to 48.1% of severe haemophilia A adults (> 18 years). With the introduction of emicizumab, most of the inhibitor patients have discontinued factor VIII tolerisation and have continued with emicizumab prophylaxis. 

The current landscape demands increased monitoring and evaluation studies to be able to determine clinical outcomes and quality of life in haemophilia A patients on various factor VIII and non factor products. The emphasis is again on self-recording treatment and bleeds effectively in MyABDR in conjunction with joint health assessment to measure clinical outcomes. The patient perspective is very much central to understand the effectiveness of any treatment product and prescribed regimen. An important milestone in future studies would be to include quality-of-life questionnaire and responses for further evaluation and analysis.          

As mentioned in one of my previous articles, MyABDR is a valuable tool to determine the relationship between prescribed treatment and clinical outcomes. With the number of different therapies and treatments available now and in future, patient self-recording treatment and bleeds is crucial to any monitoring and/or evaluation. 

Stay safe – and keep recording in MyABDR! 
 

REFERENCE

Parikh S, P'ng S, Brown S, Barnes C, Tan C, Carter T, Tran H. Current landscape of prophylaxis and tolerisation in patients with Haemophilia A in Australia [abstract]. ISTH 2022 Congress, 9-13 July 2022, London, UK.