Hemophilia - Impact of Recent Advances on Management.

Abstract

There have been numerous advances in the field of hemophilia management in the past decade, including long acting factor products, non-factor products, and potentially curative interventions such as gene therapy. Each of these interventions introduces exciting treatment modalities to patients with both hemophilia A and B, however they also pose a daunting array of possible management options. Adverse reactions to novel agents are being reported as more patients are treated and long-term sustainability of interventions such as gene therapy is yet to be determined. The practicing hematologist should be aware of the intricacies involved in customizing care for their individual patients and be aware of the monitoring strategies for each interventional strategy to avoid adverse events. Upfront cost vs. long term benefit should be considered as choices of treatment strategies are made, especially in resource poor countries. The goal of the newer agents is to decrease annualized bleed rates and avoid debilitating arthropathy. This article looks at current treatment models for prophylaxis and management of inhibitors, reviews the recent advances in the field (with bioengineered factor products, non-factor products and gene therapy) and summarizes the incorporation of these new interventions in the treatment plan for patients with hemophilia.