Department of Clinical Medicine and Surgery, Regional Reference Centre for Coagulation Disorders, "Federico II" University, Naples, Italy.
Semin Thromb Hemost. 2013 Oct;39(7):723-31. doi: 10.1055/s-0033-1354422. Epub 2013 Sep 10.
Chronic arthropathy is a major complication in severe hemophilia A (Factor [F] VIII < 1%). Almost all adults with severe hemophilia, who have not received prophylaxis with FVIII since their early childhood, suffer from chronic arthropathy. Patients with moderate hemophilia (FVIII activity 1-5%) usually experience fewer joint bleeds than those with severe hemophilia and are thought to rarely develop a significant degree of chronic arthropathy. However, some patients with moderate hemophilia behave like those with the severe form of the disorder, reporting several joint bleeds per year and significant joint impairment. Currently, only little data are available about the prevalence of arthropathy, the degree of quality of life impairment, and the need for orthopedic care/aids in patients with moderate hemophilia. In this systematic review of literature, the prevalence of overt arthropathy ranges between 15 and 77% in patients with moderate hemophilia and prophylactic replacement treatment is prescribed in approximately 30% of these patients, usually after diagnosis of clinically overt arthropathy. Moreover, because of the lack of imaging studies (magnetic resonance and/or ultrasound), the prevalence of subclinical arthropathy cannot be determined. These data confirm that severity of hemophilia should not be defined only according to FVIII levels and that a relevant proportion of patients with nonsevere hemophilia might benefit from a "tailored early prophylaxis."
慢性关节病是严重血友病 A（因子[F]VIII < 1%）的主要并发症。几乎所有未在儿童早期接受 FVIII 预防治疗的严重血友病成年患者都患有慢性关节病。中度血友病（FVIII 活性 1-5%）患者通常比严重血友病患者发生关节出血少，据认为很少发展为明显程度的慢性关节病。然而，一些中度血友病患者的表现与严重形式相似，每年报告多次关节出血和明显的关节损伤。目前，关于中度血友病患者的关节病患病率、生活质量受损程度以及对矫形护理/辅助器具的需求，仅有少量数据。在这项文献系统评价中，中度血友病患者中明显关节病的患病率在 15%至 77%之间，这些患者中约有 30%接受预防性替代治疗，通常是在诊断出明显的关节病后。此外，由于缺乏影像学研究（磁共振和/或超声），无法确定亚临床关节病的患病率。这些数据证实，血友病的严重程度不应仅根据 FVIII 水平来定义，而且相当一部分非严重血友病患者可能受益于“量身定制的早期预防治疗”。
