低收入国家血友病的非替代疗法：来自科特迪瓦前瞻性研究的经验
Nonreplacement therapy for hemophilia in low-income countries: experience from a prospective study in Ivory Coast.
作者信息
Lambert Catherine, Meité N'Dogomo, Kouassi Gustave Koffi, Dohoma Alexis Silué, Bognini Akou Sara Adélaide, Sanogo Ibrahima, Hermans Cedric
机构信息
Hemostasis and Thrombosis Unit, Division of Haematology, Cliniques Universitaires Saint-Luc, Brussels, Belgium.
Division of Clinical Haematology, Centre Hospitalier Universitaire de Yopougon, Abidjan, Ivory Coast.
出版信息
Res Pract Thromb Haemost. 2022 Dec 28;7(1):100033. doi: 10.1016/j.rpth.2022.100033. eCollection 2023 Jan.
BACKGROUND
Hemophilia management has fundamentally evolved over the last decades with the development of ground-breaking therapies. Because of their mode of action and biochemical properties, these innovative therapies that are available in developed countries could be readily implemented among people from low-income countries who are either not or inadequately treated with clotting factor concentrates (CFCs).
OBJECTIVES
We aimed at evaluating the impact of prophylaxis with emicizumab, a bispecific monoclonal antibody mimicking the FVIII activity administered subcutaneously, among boys with severe hemophilia A (HA) from the Ivory Coast, where access to CFCs is limited to humanitarian aid.
METHODS
We prospectively collected data on the implementation and outcomes of prophylaxis with emicizumab, in 33 Ivorian boys aged 2 to 13 years with severe HA (with and without inhibitors). Bleeds, CFC consumption, quality of life and satisfaction of the patients and their parents were assessed.
RESULTS
Overall, 12 months after initiating emicizumab, a 99% reduction in bleeding rates was observed, with a raise from 18% to 100% of boys having zero spontaneous joint bleeds. Three boys required a single FVIII infusion following a traumatic bleed. Health-related quality of life measures significantly improved, and perception of treatment efficacy was positively rated in children and parents. Acceptance, tolerance, and adherence were excellent. Emicizumab was instrumental in successfully implementing uninterrupted, highly efficacious, and well-tolerated prophylaxis in 72% of the Ivorian children aged ≤ 13 years identified with severe hemophilia A.
CONCLUSION
These data illustrate how innovative and disruptive nonreplacement therapies that are already accessible in developed countries could potentially provide equity in care by profoundly and rapidly modifying hemophilia burden with a magnified impact in low-income countries.
背景
在过去几十年里，随着开创性疗法的发展，血友病的治疗方式发生了根本性的演变。由于其作用方式和生化特性，这些在发达国家可用的创新疗法可以很容易地应用于那些未接受或未充分接受凝血因子浓缩物（CFCs）治疗的低收入国家人群。
目的
我们旨在评估emicizumab（一种皮下注射的模拟FVIII活性的双特异性单克隆抗体）预防对来自科特迪瓦患有严重甲型血友病（HA）男孩的影响，在该国，CFCs仅限于人道主义援助。
方法
我们前瞻性地收集了33名年龄在2至13岁患有严重HA（有或无抑制剂）的科特迪瓦男孩使用emicizumab预防的实施情况和结果数据。评估了出血情况、CFCs消耗量、患者及其父母的生活质量和满意度。
结果
总体而言，开始使用emicizumab 12个月后，出血率降低了99%，自发性关节出血为零的男孩比例从18%升至100%。三名男孩在创伤性出血后需要单次输注FVIII。与健康相关的生活质量指标显著改善，儿童和父母对治疗效果的评价为积极。接受度、耐受性和依从性都很好。emicizumab有助于在72%被确诊为严重甲型血友病的13岁及以下科特迪瓦儿童中成功实施不间断、高效且耐受性良好的预防措施。
结论
这些数据表明，发达国家已经可以获得的创新型和颠覆性非替代疗法如何通过深刻而迅速地改变血友病负担，在低收入国家产生更大影响，从而有可能实现医疗公平。
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