Centre de traitement des maladies hémorragiques, CHU de Rennes, Rennes, France; Inserm, EHESP, IRSET (institut de recherche en santé, environnement et travail) - UMR_S 1085, Université Rennes, CHU de Rennes, 35000 Rennes, France.
Département de médecine interne et immunologie clinique, CHU de Caen-Normandie, Caen, France.
Rev Med Interne. 2022 Mar;43(3):139-144. doi: 10.1016/j.revmed.2021.07.003. Epub 2021 Jul 31.
Acquired hemophilia (AH) is a rare, serious bleeding disorder most often associated with older age and life-threatening complications. The patient care pathway for AH is complex because of the different types of bleeding, the presence of comorbidities, and the heterogeneity of medical specialists who care for these patients.
This observational study used the French national PMSI (Programme de médicalisation des systèmes d'information) database to characterize patients with AH in real-life practice and analyze their hospital pathway. In total, 180 patients with AH were identified over a 5-year study period (January 2010 to December 2014), based on three criteria: bypassing agent use, International Classification of Diseases, 10th revision code allocation, and aged over 65 years. Comparison of the incidence rate of AH versus registry data validated the PMSI as an epidemiological database.
Rituximab was prescribed more often (60/180; 33.3%) than expected following guidelines and was associated in half of cases to early infections (32/60; 53.3%), surgery procedures were frequently performed during the year before AH onset (29/159; 18.2%), which may suggest a triggering effect, extended hospital stays (median: 20 days) and mortality remaining high (66/180; 36.7%) that occurred mainly during the first month after AH diagnosis. Median costs and number of injections were comparable between recombinant activated factor VII and plasma-derived activated prothrombin complex concentrate.
These findings could inform future medico-economic approaches in this AH population (duration of stays, bypassing agents, rituximab use, comorbidities, hospitalizations with infections).
获得性血友病（AH）是一种罕见且严重的出血性疾病，通常与年龄较大和危及生命的并发症有关。由于不同类型的出血、合并症的存在以及治疗这些患者的医学专家的异质性，AH 的患者护理路径较为复杂。
本观察性研究使用法国国家 PMSI（Programme de médicalisation des systèmes d'information）数据库，根据三种标准（旁路制剂的使用、国际疾病分类第 10 版编码分配和年龄超过 65 岁），在 5 年的研究期间（2010 年 1 月至 2014 年 12 月）确定了 180 例 AH 患者，以描述真实世界中 AH 患者的情况并分析其住院途径。共 180 例患者中，有 60 例（60/180；33.3%）使用利妥昔单抗的频率高于指南预期，且其中一半与早期感染有关（32/60；53.3%），在 AH 发病前一年内经常进行手术（29/159；18.2%），这可能提示存在触发作用，住院时间延长（中位数：20 天），死亡率仍然很高（66/180；36.7%），主要发生在 AH 诊断后的第一个月内。与血浆衍生的激活的凝血酶原复合物浓缩物相比，重组激活的因子 VII 和血浆衍生的激活的凝血酶原复合物浓缩物的中位费用和注射次数相当。
这些发现可能为该 AH 人群的未来医学经济学方法提供信息（住院时间、旁路制剂、利妥昔单抗的使用、合并症、感染相关住院）。
