Pacheco Zavala Eréndira, Vargas Oliva Carlos, Santibañez Bedolla Karla Edith, Murillo Ortíz Blanca Olivia, Martínez Villegas Octavio, Amador Medina Lauro Fabián
High Specialty Medical Unit UMAE No.1, Bajio, Mexican Institute of Social Security, Leon, Guanajuato, Mexico.
Department of Medicine and Nutrition, University of Guanajuato, Leon, Guanajuato, Mexico.
Indian J Hematol Blood Transfus. 2024 Jul;40(3):487-493. doi: 10.1007/s12288-023-01730-6. Epub 2024 Jan 15.
Congenital Hemophilia A is a complex disease to treat, especially in places without access to hemophilia treatment centers (HTCs). The primary aim of this study was to analyze the outcomes of a cohort of adult people with congenital hemophilia A in an HTC localized in the Bajio region of Mexico. Observational retrospective study of a cohort of 82 adult people with congenital hemophilia A treated in a tertiary-level hospital in the Bajio region of Mexico, between June 2022 and June 2023. The median age of the patients was 29.5 years, 60.9% with severe hemophilia A, 53.6% were under some factor VIII prophylaxis regimen, and 52.4% had home therapy. The median annualized bleeding rate (ABR) was one bleed/year (IQR 0-3 bleeds/year) including a median of zero joint bleeds/year (IQR 0-3 bleeds/year). The presence of high-response inhibitors was detected in 8.5%, with an overall incidence of inhibitors of 14.6% of the cohort. Univariate analysis showed that inhibitors (OR 21.10; CI 95% 1.20-370.3; = 0.03) and clinical arthropathy (OR 6.14; CI 95% 2.13-17.68; = 0.001) were significantly higher in severe hemophilia. Clinically significant arthropathy was found in 71.9% of patients. Ultrasonography of the target joints showed that mainly cartilage degeneration was affected. Blood transfusion-associated viral infections were detected in 10.9% of patients. In our HTC, current treatment with hemostatic agents allows adequate control of ABR with acceptable inhibitor rates. However, we still have joint damage in most patients, which is partly explained by the fact that prophylaxis was introduced only in recent years.
先天性血友病A是一种难以治疗的复杂疾病，在没有血友病治疗中心（HTC）的地区尤其如此。本研究的主要目的是分析墨西哥巴希奥地区一家HTC中一组成年先天性血友病A患者的治疗结果。对2022年6月至2023年6月期间在墨西哥巴希奥地区一家三级医院接受治疗的82名成年先天性血友病A患者进行观察性回顾性研究。患者的中位年龄为29.5岁，60.9%为重度血友病A，53.6%接受某种VIII因子预防方案治疗，52.4%进行家庭治疗。中位年化出血率（ABR）为每年1次出血（四分位间距为每年0 - 3次出血），其中关节出血的中位数为每年0次（四分位间距为每年0 - 3次出血）。8.5%的患者检测到高反应性抑制物，该队列中抑制物的总体发生率为14.6%。单因素分析显示，重度血友病患者中抑制物（比值比21.10；95%置信区间1.20 - 370.3；P = 0.03）和临床关节病（比值比6.14；95%置信区间2.13 - 17.68；P = 0.001）的发生率显著更高。71.9%的患者发现有临床意义的关节病。目标关节的超声检查显示主要是软骨退变受到影响。10.9%的患者检测到输血相关病毒感染。在我们的HTC中，目前使用止血剂进行治疗能够以可接受的抑制物发生率充分控制ABR。然而，我们大多数患者仍存在关节损伤，部分原因是预防措施仅在近年来才开始采用。
