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Adult People with Hemophilia A Have Low Annualized Bleeding Rate, However the Arthropathy Remains a Burden: A Retrospective Cohort Study

Eréndira Pacheco Zavala, Carlos Vargas Oliva, Karla Edith Santibañez Bedolla,Blanca Olivia Murillo Ortíz,Octavio Martínez Villegas,Lauro Fabián Amador Medina

Indian Journal of Hematology and Blood Transfusion(2024)SCI 4区

Mexican Institute of Social Security | University of Guanajuato

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Abstract
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
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Hemophilia A,Annualized bleeding rate,People with hemophilia,Hemophilia Treatment Center,Hemophilic arthropathy
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要点】:本研究分析了墨西哥Bajio地区一家治疗中心对先天性血友病A成年患者的治疗结果,发现尽管年出血率低,但关节病变仍然是一个负担。创新点在于在非 Hemophilia Treatment Centers (HTCs) 地区,对成年先天性血友病A患者进行观察性回顾性研究。

方法】:采用观察性回顾性研究方法,对2022年6月至2023年6月在墨西哥Bajio地区一家三级医院接受治疗的82名成年先天性血友病A患者进行分析。

实验】:研究发现,患者的中位年出血率为每年一次(IQR 0-3次/年),其中关节出血为零(IQR 0-3次/年)。8.5%的患者检测出高反应性抑制剂,总体抑制剂发生率为14.6%。在单变量分析中,抑制剂(OR 21.10;CI 95% 1.20–370.3;P = 0.03)和临床关节病变(OR 6.14;CI 95% 2.13–17.68;P = 0.001)在严重血友病患者中显著升高。71.9%的患者出现临床显著关节病变。关节超声检查显示,主要是软骨退化受影响。10.9%的患者检测出与输血相关的病毒感染。尽管在治疗中心中,目前使用止血剂可以适当控制年出血率,但仍有大多数患者出现关节损伤,这在一定程度上可以解释为预防性治疗是在最近几年才引入的。