Adeno-associated virus-based gene therapy for hemophilia A and B: a systematic review and meta-analysis.

ABSTRACT:

Abstract

Adeno-associated virus (AAV)-based gene therapy is an emerging treatment for hemophilia A (HA) and hemophilia B (HB). In this systematic review and meta-analysis, we searched for studies of adult males with severe or moderately severe HA or HB who received AAV-based gene therapy. Annualized bleeding rate (ABR), annualized infusion rate (AIR), total factor use, factor levels, and adverse events (AEs) were extracted. Eight HA trials representing 7 gene therapies and 211 patients and 12 HB trials representing 9 gene therapies and 184 patients were included. For HA, gene therapy resulted in an annualized decrease of 7.58 bleeding events (95% confidence interval [CI], -11.50 to -3.67) and 117.2 factor infusions (95% CI, -151.86 to -82.53) compared with before gene therapy. Fact

SUBMITTER: Deshpande SR 

PROVIDER: S-EPMC11629206 | biostudies-literature | 2024 Dec

REPOSITORIES: biostudies-literature