Real-world provider experiences with hemophilia A gene therapy: administration of valoctocogene roxaparvovec.

Severe hemophilia A is a bleeding condition caused by a deficiency in clotting factor VIII (FVIII ≤1 IU/dL) that results in spontaneous and excessive posttraumatic bleeding. The current standard of care is prophylaxis with exogenous FVIII or bispecific antibodies that mimic FVIII function. Valoctocogene roxaparvovec is a gene therapy approved for adults with severe hemophilia A that enables endogenous FVIII production to protect against bleeding. As a newer treatment modality, workflows for gene therapy administration and postinfusion care are still unfamiliar to many health care professionals and may be perceived as implementation barriers. We are 10 hemophilia gene therapy providers across the United States, Italy, and Germany sharing our experiences of administering valoctocogene roxaparvovec in clinical practice to >25 patients with severe hemophilia A. In this study, we provide our insights into patient preparation, establishment of treatment centers and multidisciplinary teams, postinfusion management, and patient follow-up to facilitate open communication and collaboration among the treatment community.

© 2026 The Author(s).

PMID: 42232249