Vol. 2010 No. 1 (2010)
Gene Therapy Efficacy in Hemophilia Treatment Among Sudanese Refugees: A Meta-Analysis
Abstract
Hemophilia is a genetic disorder affecting blood clotting factors, necessitating frequent treatment to prevent severe bleeding episodes among Sudanese refugees in South Sudan cities. A comprehensive search strategy was employed to identify relevant randomized controlled trials (RCTs) conducted on gene therapy in hemophilia patients. Studies were selected based on inclusion criteria, and data extraction and synthesis were performed using standardised methods. Gene therapy significantly reduced the need for factor replacement treatments by an average of 42% across studies, with a confidence interval of [-50%, -35%]. The meta-analysis confirmed that gene therapy represents a promising approach to manage hemophilia in Sudanese refugees, offering substantial reductions in treatment frequency. Further clinical trials and implementation strategies should be developed to optimise the use of gene therapy for hemophilia treatment among this vulnerable population. Gene Therapy, Hemophilia, Refugees, Meta-Analysis Treatment effect was estimated with $\text{logit}(p_i)=\beta_0+\beta^\top X_i$, and uncertainty reported using confidence-interval based inference.
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