Organization issues clinical practice guideline for the treatment of hemophilia

In a clinical practice guideline issued by the International Society on Thrombosis and Haemostasis and published online June 13 in the Journal of Thrombosis and Haemostasis, recommendations are presented for the treatment of congenital hemophilia.

Suely M. Rezende, M.D., Ph.D., from the Universidade Federal de Minas Gerais in Belo Horizonte, Brazil, and colleagues developed an evidence-based clinical practice guideline for hemophilia treatment. Thirteen questions were selected by a multidisciplinary panel: 11 addressed the treatment of hemophilia A and two addressed hemophilia B treatment.

For hemophilia A, the panel addressed questions relating to prophylactic and episodic treatment with factor VIII concentrates, bypassing agents, nonfactor therapy (emicizumab) (with and without inhibitors), as well as immune tolerance induction. Questions on prophylactic and episodic treatment of bleeding events with factor IX concentrates were addressed for hemophilia B.

For all 13 recommendations, agreement was reached. For severe and moderately severe hemophilia A and B without inhibitors, prophylaxis is strongly recommended over episodic treatment of bleeding events. Prophylaxis with emicizumab or with factor VIII concentrates is recommended for hemophilia A, and prophylaxis with purified plasma-derived factor IX or standard or extended half-life recombinant factor IX concentrates is recommended for hemophilia B without inhibitors.

"With hemophilia management being complex, our guideline provides clarity, guiding treatment decisions with evidence-based insight, yet highlighting the importance of making treatment decisions consistent with patients' individual risks, values, and preferences," Rezende said in a statement.

Several authors disclosed ties to the pharmaceutical industry.

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