Braunert L, Bruegel M, Pfrepper C, Thiery J, Niederwieser D
Department für Innere Medizin, selbstständige Abteilung für Hämatologie, Internistische Onkologie und Hämostaseologie, Johannisallee 32 A, 04103 Leipzig.
Hamostaseologie. 2010 Nov;30 Suppl 1:S40-3.
Acquired hemophilia A is a rare but potentially life-threatening bleeding disorder. It is caused by the development of autoantibodies directed against coagulation factor VIII in adults or elderly patients, who do not have a personal or family history of bleeding.
A man (age: 76 years) on prednisone and leflunomide for polymyalgia rheumatica developed spontaneous severe haematomas. The patient was diagnosed with acquired factor VIII deficiency (FVIII activity 1.2%, FVIII inhibitor 31.7 BU). Due to the active bleeding diathesis, treatment was administered with activated prothrombin complex concentrates (FEIBA®, Baxter). Immunosuppressive treatment with a combination of oral prednisone (1 mg/kg daily) and cyclophosphamide (1,5 mg/kg daily) was administered to reduce the FVIII inhibitor. However, after two weeks of treatment, FVIII was only 3% and no clinical improvement was observed. Treatment with the anti CD20 monoclonal antibody rituximab intravenously at 375 mg/m2 once weekly for four consecutive weeks was started. The patient showed rapid clinical improvement following rituximab treatment. He achieved a complete remission defined as return to normal FVIII activity and undetectable FVIII inhibitor titer. After a follow-up of six months no relapse occurred.
Rituximab appears an effective and well-tolerated treatment for patients with acquired haemophilia.
获得性血友病A是一种罕见但可能危及生命的出血性疾病。它是由成人或老年患者体内针对凝血因子VIII的自身抗体产生所引起的，这些患者无个人或家族出血史。
一名76岁男性因风湿性多肌痛正在服用泼尼松和来氟米特，出现了自发性严重血肿。该患者被诊断为获得性因子VIII缺乏症（FVIII活性1.2%，FVIII抑制剂31.7 BU）。由于存在活动性出血素质，给予活化凝血酶原复合物浓缩剂（FEIBA®，百特公司）进行治疗。采用口服泼尼松（每日1 mg/kg）和环磷酰胺（每日1.5 mg/kg）联合进行免疫抑制治疗以降低FVIII抑制剂水平。然而，治疗两周后，FVIII仅为3%，且未观察到临床改善。开始静脉注射抗CD20单克隆抗体利妥昔单抗，剂量为375 mg/m2，每周一次，连续四周。利妥昔单抗治疗后患者临床症状迅速改善。他实现了完全缓解，定义为FVIII活性恢复正常且FVIII抑制剂滴度检测不到。随访六个月未出现复发。
利妥昔单抗似乎是获得性血友病患者一种有效且耐受性良好的治疗方法。
