Recent research explores the theoretical risk of hepatocellular carcinoma (HCC) following adeno-associated virus (AAV) gene therapy in hemophilia patients. While data from clinical trials are reassuring, concerns remain about long-term oncogenic potential. A single case of HCC post AAV infusion in a patient with prior HCV infection raised questions, but further analysis suggested AAV integration did not contribute to tumor development. Monitoring with serum markers and imaging studies is recommended for hemophilia patients receiving AAV gene therapy, especially considering the increased HCC risk in this population. Infants receiving high AAV doses for genetic disorders like SMA may face similar genotoxicity risks, highlighting the importance of long-term surveillance in these cohorts. Understanding the implications of AAV gene therapy vectors for hemophilia patients requires ongoing research and vigilance to ensure patient safety and efficacy of this promising treatment approach.
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