Matching-adjusted indirect comparison of bleeding outcomes in severe haemophilia A: Comparing valoctocogene roxaparvovec gene therapy, emicizumab prophylaxis, and FVIII replacement prophylaxis.

Jan AstermarkTyler W BucknerLaurent FrenzelAnthony J HatswellXiaojun YouHai LiuErin GoodmanSandra SantosCharles HawesDavid HindsRobert Klamroth

Published in: Haemophilia : the official journal of the World Federation of Hemophilia (2023)

Valoctocogene roxaparvovec provided generally lower bleeding rates and higher probability of no bleeds, including treated joint bleeds, than emicizumab. Emicizumab was more effective than FVIII prophylaxis regimens used in 270-902.

Keyphrases

gene therapy
atrial fibrillation
early onset
weight loss
skeletal muscle
glycemic control
clinical evaluation