Monthly Roundup: May 2024
Presented by: Johnny Mahlangu, BSc, MBBCh, MMed, FCPath
The four-year follow-up study on valoctocogene roxaparvovec, a gene therapy for severe hemophilia A, showed sustained safety and efficacy. A single infusion enabled durable Factor VIII production, with no new safety signals and mild ALT elevations being the most common adverse event. Mean Factor VIII levels were 16.1% when measured by chromogenic assay and 27.1% by one-stage assay, with 62% of participants in the mild hemophilia range. The annualized bleed rate decreased by 81% to 0.9, and 74% of patients experienced no treated bleeds. Factor VIII infusion rates dropped by 92%, and quality of life significantly improved. These results confirm durable bleeding protection, reduced treatment needs, and improved health outcomes.
