ISTH Releases Hemophilia Treatment Guideline

In a clinical practice guideline released by the International Society on Thrombosis and Haemostasis and published online on June 13 in the Journal of Thrombosis and Haemostasis, new recommendations are provided for the treatment of congenital hemophilia. The guideline, which was crafted by Suely M. Rezende, M.D., Ph.D., from the Universidade Federal de Minas Gerais in Belo Horizonte, Brazil, alongside her colleagues, is evidence-based and aims to provide clarity in the management of hemophilia.

The multidisciplinary panel that formulated the guideline addressed thirteen key questions, with eleven focusing on hemophilia A and two on hemophilia B. For hemophilia A, the queries revolved around both prophylactic and episodic treatments using factor VIII concentrates, bypassing agents, and nonfactor therapy such as emicizumab, both with and without inhibitors. The guideline also addresses immune tolerance induction. 

Regarding hemophilia B, the panel examined questions about prophylactic and episodic treatments of bleeding events with factor IX concentrates. Across all thirteen recommendations, the panel reached a consensus. For individuals with severe and moderately severe hemophilia A and B who do not have inhibitors, prophylaxis is strongly recommended over the episodic treatment of bleeding events. For hemophilia A, the panel recommends prophylaxis using either emicizumab or factor VIII concentrates. For hemophilia B patients without inhibitors, the guideline recommends prophylaxis with either purified plasma-derived factor IX or standard or extended half-life recombinant factor IX concentrates.

Rezende emphasized the complexity of managing hemophilia and highlighted that the guideline aims to guide treatment decisions with evidence-based insights. However, she also stressed the importance of tailoring treatment decisions to align with patients' individual risks, values, and preferences.

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