Dutta Anupam, Dutta Taniya S, Borbouah Luish, Duseja Yash, Bora Juhi, Gogoi Papori
Department of General Medicine, Assam Medical College and Hospital, Dibrugarh, IND.
Department of Pediatrics, Assam Medical College and Hospital, Dibrugarh, IND.
Cureus. 2024 Dec 2;16(12):e74948. doi: 10.7759/cureus.74948. eCollection 2024 Dec.
Background and objective Hemophilia A (HA) is a genetic bleeding disorder caused by a lack of factor VIII (FVIII) and is associated with frequent bleeding and joint damage. Traditional intravenous treatments for this condition are cumbersome and can lead to complications. Emicizumab, a bispecific monoclonal antibody, offers a promising subcutaneous alternative with potential safety and efficacy-related benefits. This study aimed to evaluate the impact of emicizumab prophylaxis on bleeding rates, joint health, functional activity, and quality of life (QoL) in patients with congenital HA. Methods A noninterventional, prospective observational study was conducted at the Assam Medical College, a tertiary care center in northeastern India, involving 40 patients with HA (PwHA), who were either on FVIII therapy or newly on emicizumab. Emicizumab was given subcutaneously at 3 mg/kg weekly for the first month, followed by 6 mg/kg every four weeks. Endpoints included changes in annual bleeding rate (ABR), Hemophilia Joint Health Score (HJHS), Functional Independence Score in Hemophilia (FISH), and QoL via European Quality of Life 5-Dimensions 5-Levels (EQ-5D-5L) and visual analog scale (VAS) scores at 24 weeks. Results At 24 weeks, HJHS improved from 12.8 to 4.8 (p<0.001), FISH from 27.5 to 30.6 (p<0.001), and ABR decreased from 11.36 to 0.195. Quality of life scores also improved (EQ-5D-5L index from 0.79 to 0.96, VAS from 72.18 to 92.75, both p<0.001). All 30 patients with target joints had resolved bleeds, and adherence to emicizumab was 100%. Conclusions Based on our findings, emicizumab significantly reduces bleeding, enhances joint health, and improves the quality of life in PwHA. It is associated with high adherence, suggesting its feasibility as a treatment, especially in resource-limited settings. However, long-term studies are needed to validate these results.
背景与目的 甲型血友病（HA）是一种由于缺乏凝血因子VIII（FVIII）引起的遗传性出血性疾病，常伴有出血和关节损伤。针对这种疾病的传统静脉治疗方法繁琐且可能导致并发症。艾美赛珠单抗是一种双特异性单克隆抗体，提供了一种有前景的皮下替代疗法，具有潜在的安全性和疗效相关益处。本研究旨在评估艾美赛珠单抗预防治疗对先天性HA患者出血率、关节健康、功能活动和生活质量（QoL）的影响。方法 在印度东北部的三级医疗中心阿萨姆医学院进行了一项非干预性前瞻性观察研究，纳入40例HA患者（PwHA），这些患者要么正在接受FVIII治疗，要么新开始使用艾美赛珠单抗。艾美赛珠单抗在第一个月每周皮下注射3mg/kg，随后每四周注射6mg/kg。终点指标包括24周时年度出血率（ABR）、血友病关节健康评分（HJHS）、血友病功能独立性评分（FISH）的变化，以及通过欧洲五维五级生活质量量表（EQ-5D-5L）和视觉模拟量表（VAS）评分评估的生活质量变化。结果 在24周时，HJHS从12.8改善至4.8（p<0.001），FISH从27.5改善至30.6（p<0.001），ABR从11.36降至0.195。生活质量评分也有所改善（EQ-5D-5L指数从0.79提高至0.96，VAS从72.18提高至92.75，均p<0.001）。所有30例有目标关节的患者出血均已缓解，对艾美赛珠单抗的依从性为100%。结论 根据我们的研究结果，艾美赛珠单抗可显著减少出血、改善关节健康并提高PwHA的生活质量。它具有高依从性，表明其作为一种治疗方法的可行性，尤其是在资源有限的环境中。然而，需要长期研究来验证这些结果。
