Bhatt Parul, Patel Harsh
General Medicine, GMERS Medical College and Hospital, Sola, Ahmedabad, IND.
Internal Medicine, GMERS Medical College and Hospital, Sola, Ahmedabad, IND.
Cureus. 2025 Feb 28;17(2):e79815. doi: 10.7759/cureus.79815. eCollection 2025 Feb.
Prophylactic treatment for hemophilia in India remains significantly underutilized compared to developed nations, leading to reliance on episodic therapy with limited long-term benefits. This study evaluated the efficacy and safety of glycopegylated extended half-life (EHL) clotting factors in patients with hemophilia A and B. A retrospective observational design was employed, including 25 patients aged ≥18 years with moderate or severe hemophilia A or B who transitioned to EHL therapy. Hemophilia A patients (n=19) switched from standard half-life (SHL) factor VIII (FVIII) to weekly EHL FVIII prophylaxis, while hemophilia B patients (n=6) transitioned from on-demand SHL factor IX (FIX) to glycopegylated EHL FIX prophylaxis every 21 days. Outcomes were assessed through the annual bleeding rate (ABR), functional independence score in hemophilia (FISH score), and hemophilia joint health score (HJHS). A subset analysis for quality of life (QoL) was also performed on hemophilia A patients using the Haem-A-QoL questionnaire in patients with hemophilia A. Glycopegylated EHL FVIII therapy significantly reduced ABR and improved joint health and functional scores over 24 months (p<0.001). Hemophilia B patients demonstrated a significant reduction in ABR and joint bleeding rates at eight months (p<0.05). The study found that prophylaxis with individualized doses of glycopegylated EHL factors is not only effective in reducing bleeding episodes and improving joint health but also safe with improved treatment adherence, offering a viable prophylactic option in resource-constrained settings.
与发达国家相比，印度血友病的预防性治疗仍未得到充分利用，导致人们依赖具有有限长期益处的间歇性治疗。本研究评估了聚乙二醇化延长半衰期（EHL）凝血因子在甲型和乙型血友病患者中的疗效和安全性。采用回顾性观察设计，纳入了25例年龄≥18岁、从标准半衰期（SHL）治疗转换为EHL治疗的中度或重度甲型或乙型血友病患者。19例甲型血友病患者从标准半衰期（SHL）的凝血因子VIII（FVIII）转换为每周一次的EHL FVIII预防性治疗，6例乙型血友病患者从按需使用的SHL凝血因子IX（FIX）转换为每21天一次的聚乙二醇化EHL FIX预防性治疗。通过年出血率（ABR）、血友病功能独立性评分（FISH评分）和血友病关节健康评分（HJHS）评估结果。还使用甲型血友病患者的Haem-A-QoL问卷对甲型血友病患者进行了生活质量（QoL）的亚组分析。聚乙二醇化EHL FVIII治疗在24个月内显著降低了ABR，并改善了关节健康和功能评分（p<0.001）。乙型血友病患者在8个月时ABR和关节出血率显著降低（p<0.05）。研究发现，使用个体化剂量的聚乙二醇化EHL因子进行预防性治疗不仅能有效减少出血发作并改善关节健康，而且安全性高，治疗依从性也有所提高，在资源有限的环境中提供了一种可行的预防性选择。
