The International Society on Thrombosis and Haemostasis (ISTH) has issued an evidence-based clinical practice guideline aimed at revolutionizing the treatment landscape for congenital haemophilia A and B. Hemophilia, a rare bleeding disorder caused by deficiencies in blood coagulation factors, affects individuals worldwide and demands intricate management strategies. Until now, a comprehensive guideline based on the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) approach has been absent, leaving patients, caregivers, and clinicians without a standardized framework for treatment decisions.
The objective of this new guideline, published in the Journal of Thrombosis and Haemostasis, is to offer a comprehensive overview of evidence to inform treatment decisions. The ISTH assembled a multidisciplinary panel comprising physicians and patient representatives from around the globe, meticulously balanced to mitigate potential biases. This diverse panel prioritized key clinical questions and outcomes crucial for both clinicians and patients.
Thirteen questions were selected by the panel: 11 addressed the treatment of haemophilia A and 2 addressed haemophilia B treatment. For haemophilia A, the panel examined prophylactic and episodic treatment with factor VIII concentrates, bypassing agents, nonfactor therapy (emicizumab) with and without inhibitors, as well as immune tolerance induction. For haemophilia B, questions focused on prophylactic and episodic treatment of bleeding events with factor IX concentrates.
The recommendations underwent rigorous investigation, including public commentary, ensuring transparency and inclusivity, with the purpose of providing a set of 13 crafted recommendations, addressing critical aspects of haemophilia A and B treatment. Among these recommendations, strong endorsements advocate for prophylactic treatment over episodic interventions for severe and moderately severe haemophilia A and B, marking a significant paradigm shift in clinical practice.
For severe and moderately severe haemophilia A without inhibitors, prophylaxis with emicizumab or factor VIII concentrates is strongly recommended. For haemophilia B without inhibitors, prophylaxis with purified plasma-derived factor IX or standard or extended half-life recombinant factor IX concentrates is strongly recommended.
Most notably, 54 percent of the recommendations are grounded in evidence from randomized clinical trials, providing clinicians with robust data-driven insights. This comprehensive guideline promises to significantly enhance the quality of care for individuals living with congenital haemophilia A and B, providing a standardized framework to guide treatment decisions and improve patient outcomes.
The full guidelines is available here.
Disclosures: This article was created by the touchHAEMATOLOGY team utilizing AI as an editorial tool (ChatGPT (GPT-4o) [Large language model]. https://chat.openai.com/chat.) The content was developed and edited by human editors. No funding was received in the publication of this article.
