This Plain Language Summary of Publication article (PLSP) from Expert Review of Haematology, discusses hemophilia A, a bleeding disorder caused by low levels of factor VIII (FVIII), and the new treatments for it, including emicizumab and valoctocogene roxaparvovec gene therapy. Both therapies were shown to reduce bleeding in separate clinical trials (HAVEN 3 and GENEr8-1) compared to traditional FVIII replacement therapy. However, these treatments have not been directly compared in a clinical trial. To address this, an unanchored matching-adjusted indirect comparison (MAIC) method was used to compare bleeding rates between the two therapies across different studies with varying trial structures.

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This PLSP is based on an article called ‘Matching-adjusted indirect comparison of bleeding outcomes in severe haemophilia A: Comparing valoctocogene roxaparvovec gene therapy, emicizumab prophylaxis, and FVIII replacement prophylaxis’ and was published in Haemophilia.

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