New Guidance Addresses Management of Patients With Congenital Hemophilia

A new International Society on Thrombosis and Haemostasis clinical practice guideline published in the Journal of Thrombosis and Haemostasis strongly recommends prophylactic over episodic treatment in patients with severe and moderately severe hemophilia A and B.

The recommendations for prophylactic treatment, which constitute 2 of a total 13 statements, are the sole strong recommendations, based on moderate-certainty evidence, in the guidance. The remaining 11 recommendations are conditional and based on very low-certainty evidence.

The advice for prophylactic over episodic treatment in patients with severe and moderately severe hemophilia aligns with nearly 3 decades of guidance from the World Health Organization and the World Federation of Hemophilia, researchers explained. 

“This also corroborates the results of a recent systematic review that concluded that prophylaxis, as compared with episodic treatment, may reduce bleeding frequency in previously treated individuals with hemophilia,” wrote corresponding author Frits R. Rosendaal, MD, PhD, of Leiden University Medical Center, Leiden, the Netherlands, and guideline coauthors.

The remaining conditional recommendations focus on episodic treatment with factor (F)VIII concentrates, bypassing agents, and emicizumab for hemophilia A (with and without inhibitors); immune tolerance induction in patients with hemophilia A; and prophylactic and episodic treatment of bleeding events with FIX concentrates in patients with hemophilia B.

The guidance resulted from a systematic review of evidence that followed the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) approach. Evidence from randomized clinical trials formed the basis for 7 of the recommendations. Data from observational studies guided 3 of the recommendations, and findings from indirect comparisons guided 3 recommendations.

“Future research should focus on direct treatment comparisons and the treatment of hemophilia B with and without inhibitors,” researchers wrote. “Future updates of this guideline will provide an updated evidence synthesis on the current questions and focus on new FVIII and FIX concentrates, novel nonfactor therapies, and gene therapy for severe and nonsevere hemophilia A and B.”

Reference
Rezende SM, Neumann I, Angchaisuksiri P, et al. International society on thrombosis and haemostasis clinical practice guideline for treatment of congenital hemophilia A and B based on the grading of recommendations assessment, development, and evaluation methodology. J Thromb Haemost. 2024;22(9):2629-2652. doi:10.1016/j.jtha.2024.05.026