Considerations for the Use of AAV-based Gene Therapy in HIV-Positive Individuals With Haemophilia.

INTRODUCTION: There is a high prevalence of human immunodeficiency virus (HIV) infection among the haemophilia community due to treatment in the 1970s and 1980s with contaminated clotting factor. Lifelong treatment regimens for haemophilia and HIV are burdensome alone and pose a particular challenge for individuals living with both conditions. Adeno-associated virus (AAV)-based gene therapy restores endogenous factor expression and offers an alternative to routine prophylaxis for haemophilia that individuals living with haemophilia and HIV infection may find uniquely appealing to alleviate the treatment burden for at least one of their chronic conditions.

AIM: The aim of this article is to provide guidance on clinical practice to health care professionals considering gene therapy as a treatment option for individuals with haemophilia and HIV.

METHODS: We compile available safety and efficacy evidence from participants living with HIV who participated in gene therapy trials for haemophilia. Then, based on this evidence, we provide several HIV-specific considerations for individuals with haemophilia and HIV comorbidity.

CONCLUSION: As part of a shared decision-making approach, it is important to not only evaluate if gene therapy is appropriate but also to offer recommendations on what to expect when navigating the treatment journey. The available evidence to date indicates modern antiretroviral therapy (ART) regimens may not cause complications when combined with AAV-based gene therapies. Accordingly, HIV infection should not be considered a general contraindication for gene therapy to restore factor expression in haemophilia. However, a careful consideration of the individual's life context, especially hepatotoxic drug effects or interactions, is warranted.

© 2026 The Author(s). Haemophilia published by John Wiley & Sons Ltd.

PMID: 42033288