Extended Half-life Versus Standard Half-life Factor Replacement Therapy in Hemophilia A and B: A Systematic Review and Meta-analysis

Abstract

Hemophilia is a congenital bleeding disorder that requires frequent factor replacement therapies to manage bleeding episodes. This systematic review and meta-analysis compares the efficacy of extended half-life (EHL) and standard half-life (SHL) factor replacement therapies in patients with haemophilia A or B. Despite advances in hemophilia treatment, recurrent bleeding episodes, particularly joint bleeds, continue to significantly impact patients' health. While SHL therapies are effective, they require frequent infusions, leading to poor adherence and increased bleeding risks. EHL therapies, developed to extend factor persistence, have shown promise in reducing bleeding events and infusions, improving patient adherence, and enhancing quality of life. In this review Pubmed, Google scholar and Clinicaltrial.gov were searched and six (6) studies that met the inclusion criteria were included with a total 259 participants, who were all male aged 20 to 40 years (214 participants were suffering from hemophilia A and 45 participants were suffering from hemophilia B) featured. The data synthesized from the studies revealed a pooled Standardized Mean Difference (SMD) of -0.37 in annualized bleeding rate (ABR) favoring EHL therapies (95% CI: -0.69, -0.05). These results underscore EHL's potential in reducing bleeding episodes and infusion frequency. However, heterogeneity across studies suggests the need for further research on factors such as patient demographics and treatment regimens. This study highlights the clinical advantages of EHL therapies and calls for greater global access to these treatments, particularly in regions where SHL remains the primary therapy. Future research should explore randomized studies to enhance evidence and Policymakers should consider facilitating access to EHL therapies, particularly in resource-limited settings.