Josephson Neil
1University of Washington, Seattle, WA; and.
Hematology Am Soc Hematol Educ Program. 2013;2013:261-7. doi: 10.1182/asheducation-2013.1.261.
Outcomes for patients with hemophilia have improved dramatically over the past 50 years. With the increased availability of safe clotting factor concentrates, the primary focus in clinical management is now the prevention of long-term complications, most notably the debilitating hemophilic arthropathy that is associated with severe disease. This article reviews evidence-based approaches for managing both children and adults with hemophilia. Definitive evidence of improved clinical results from primary prophylaxis started in young patients with severe hemophilia A and a minimal bleeding history is presented. Furthermore, recent studies showing benefits for initiating prophylaxis in older adolescents and adults with established joint disease are examined. Inhibitors to factor VIII are the most problematic complication of factor replacement therapy. Patient-specific and treatment-related factors that contribute to the risk of inhibitor formation are discussed and controversies and clinical evidence related to approaches for tolerance induction are reviewed.
在过去50年里，血友病患者的治疗效果有了显著改善。随着安全凝血因子浓缩物供应的增加，目前临床管理的主要重点是预防长期并发症，最显著的是与严重疾病相关的致残性血友病性关节病。本文回顾了针对血友病儿童和成人的循证管理方法。文中给出了在患有严重甲型血友病且出血史轻微的年轻患者中开始进行初级预防可改善临床结果的确切证据。此外，还研究了近期关于在患有已确诊关节疾病的大龄青少年和成人中开始预防有益处的研究。VIII因子抑制剂是因子替代疗法最棘手的并发症。讨论了导致抑制剂形成风险的患者特异性和治疗相关因素，并回顾了与耐受诱导方法相关的争议和临床证据。
