Sakai M, Shima M, Shirahata A
Department of Pediatrics, School of Medicine, University of Occupational and Environmental Health, Kitakyushu, Japan.
Haemophilia. 2005 May;11(3):285-9. doi: 10.1111/j.1365-2516.2005.01093.x.
We encountered a 2-year-old boy with acquired haemophilia, which rarely occurs in children, who was complicated with nephrotic syndrome. In mid-August 2001, he was diagnosed to have nephrotic syndrome based on the presence of massive proteinuria and hypoalbuminaemia. Activated partial thromboplastin time (APTT) was normal at 42.4 s at that time. After starting prednisone administration of 2 mg kg(-1) day(-1), the proteinuria disappeared immediately. However, in early October the same year, subcutaneous ecchymosis and intramuscular bleeding occurred for no apparent reason, and from the examination results his APTT was 106.4 s, factor VIII (FVIII) activity was <1%, and the anti-FVIII inhibitor titre was 6.9 BU ml(-1). As a result, he was diagnosed to have acquired haemophilia. The anti-nuclear antibody and anti-phospholipid antibody were negative. With recombinant activated FVII, haemostasis was obtained, and after administering three courses of steroid pulse therapy (methyl prednisolone: 20 mg kg(-1) day(-1) x 3 days), the anti-FVIII inhibitory activity disappeared. An analysis of the immunological and coagulation properties of his FVIII autoantibodies showed the anti-FVIII inhibitory activity to be mediated by IgG(1) antibody. In other words, his FVIII inhibitor was a Th1 dominant and it provided a good response to treatment. These findings correlate with those of previous reports. The patient thereafter frequently demonstrated a recurrence of nephrotic syndrome. As a result, he is presently being managed with cyclosporine. However, no recurrence of the anti-FVIII titre has been observed.
我们遇到一名患有获得性血友病的2岁男孩，该病在儿童中很少见，且该患儿并发了肾病综合征。2001年8月中旬，根据大量蛋白尿和低白蛋白血症，他被诊断为肾病综合征。当时活化部分凝血活酶时间（APTT）正常，为42.4秒。开始给予泼尼松2 mg·kg⁻¹·d⁻¹治疗后，蛋白尿立即消失。然而，同年10月初，他无故出现皮下瘀斑和肌肉出血，检查结果显示其APTT为106.4秒，凝血因子VIII（FVIII）活性<1%，抗FVIII抑制剂滴度为6.9 BU/ml⁻¹。因此，他被诊断为获得性血友病。抗核抗体和抗磷脂抗体均为阴性。使用重组活化FVII后实现了止血，在给予三个疗程的类固醇冲击治疗（甲泼尼龙：20 mg·kg⁻¹·d⁻¹×3天）后，抗FVIII抑制活性消失。对其FVIII自身抗体的免疫和凝血特性分析表明，抗FVIII抑制活性由IgG(1)抗体介导。换句话说，他的FVIII抑制剂以Th1为主，对治疗反应良好。这些发现与先前的报道一致。此后，该患者频繁出现肾病综合征复发。因此，他目前正在接受环孢素治疗。然而，未观察到抗FVIII滴度复发。
