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Virus-based Gene Therapy Research · Journal article
Current Advances in Medicine. · August 28, 2026
A consensus or society position rather than new primary data.
This is a comprehensive narrative review of hemophilia epidemiology, pathophysiology, diagnosis, and treatment strategies, including both conventional therapies and emerging options. It synthesizes current knowledge on prophylactic therapy efficacy, inhibitor complications, and novel therapeutic approaches (non-factor treatments and gene therapy) but does not present new primary data or comparative evidence synthesis.
Narrative literature review. Hemophilia A and B patients; review encompasses epidemiology, pathophysiology, and clinical management literature without defining a discrete study cohort.. Intervention: Comprehensive overview of hemophilia therapies including prophylactic factor replacement, extended half-life concentrates, non-factor treatments (Emicizumab, Fitusiran, Concizumab), and gene therapy using AAV vectors and CRISPR approaches..
Prophylactic therapy significantly reduces bleeding rates by ~70–90% and prevents joint damage Inhibitor development occurs in ~20–30% of severe hemophilia cases and remains a major management challenge Emerging therapies including Emicizumab, Fitusiran, Concizumab, and AAV/CRISPR gene therapy improve bleeding control and quality of life
Reported figures (70–90% bleeding reduction, 20–30% inhibitor rate) are aggregate observations from literature; original effect sizes, confidence intervals, and p-values not specified Prophylactic therapy significantly reduces bleeding rates by ~70–90% and prevents joint damage
This review provides an evidence-informed overview of modern hemophilia management for clinicians and researchers, highlighting the value of early prophylactic therapy and the emerging role of non-factor and gene therapies in improving outcomes and reducing treatment burden.
A comprehensive narrative review synthesizing epidemiology, pathophysiology, diagnostics, and therapeutic approaches to hemophilia without presenting new primary data or meta-analytic synthesis.
As stated by the source record.
Quoted from the source exactly as published.
This review provides an evidence-informed overview of modern hemophilia management for clinicians and researchers, highlighting the value of early prophylactic therapy and the emerging role of non-factor and gene therapies in improving outcomes and reducing treatment burden.
Graded across the dimensions that decide whether you should act, each from what the source actually supports. There is no single score, and where a dimension was not assessed it says so.
Introduction: Hemophilia is a rare X-linked inherited bleeding disorder caused by a deficiency of coagulation factor VIII (hemophilia A) / factor IX (hemophilia B), resulting in impaired clot formation and recurrent bleeding episodes. This review aims to provide a comprehensive overview of the epidemiology, pathophysiology, diagnostic approaches, and evolving therapeutic strategies for hemophilia. It focuses on current treatments, inhibitor complications, and emerging therapies to improve outcomes. Methods: A comprehensive literature review was conducted using established databases, including PubMed, Scopus, Web of Science, and Google Scholar. Particular emphasis was placed on literature covering gene therapy, extended half-life factor concentrates, non-factor therapies, and inhibitor management. Results: The study found that hemophilia primarily affects males and presents with varying degrees of severity. Prophylactic therapy significantly reduces bleeding rates (by ~70-90%) and prevents joint damage. However, inhibitor development (seen in ~20-30% of severe cases) remains a key challenge. Emerging therapies, including non-factor treatments and gene therapy, show substantial improvement in bleeding control and patient quality of life. Discussion: The findings show that effective management of Hemophilia A and B relies on early prophylactic therapy, including extended half-life factor VIII and IX with pharmacokinetic-guided dosing. Inhibitor development remains a major challenge. Emerging therapies, such as Emicizumab, Fitusiran, Concizumab, and gene therapy using AAV vectors and CRISPR approaches, improve outcomes and reduce treatment burden. Conclusion: Hemophilia is a lifelong disorder requiring individualized and evolving treatment strategies. Integration of conventional factor replacement with emerging therapies has significantly improved outcomes and reduced morbidity. This review highlights key challenges, recent advances, and future directions, providing an updated and concise resource for clinical and research applications.
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