Effectiveness of Gene Therapies for Hemophilia: A Systematic Review of Clinical Trials
DOI:
https://doi.org/10.22399/ijcesen.4626Keywords:
Gene therapy, hemophilia, systematic review, clinical trials, efficacy, safetyAbstract
This systematic review evaluates the effectiveness and safety of gene therapies for hemophilia by analyzing data from 11 clinical trials involving patients with severe hemophilia A or B. The findings demonstrate that adeno-associated virus (AAV)-mediated and lentiviral-mediated gene therapies consistently enable sustained endogenous production of clotting factors, with mean factor activity levels rising to 18.4–42.6% in hemophilia A and 5–41.9% in hemophilia B. These improvements translated into clinically significant reductions in annualized bleeding rates (71–90%) and near-elimination of prophylactic factor concentrate use in most patients. The most common adverse event was transient, manageable elevation of liver aminotransferases, with no reported inhibitor development. However, gradual declines in factor expression—particularly for factor VIII—and exclusion criteria related to pre-existing AAV immunity highlight ongoing challenges. Gene therapy represents a transformative, durable treatment that substantially improves quality of life, though long-term monitoring and strategies to broaden patient eligibility remain priorities.
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