Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn, Bonn, Germany.
Haemophilia. 2014 Jan;20(1):83-91. doi: 10.1111/hae.12263. Epub 2013 Nov 19.
Most studies on immune tolerance induction (ITI) therapy in haemophilia A patients are focused on primary ITI in children. Here we report on the ITI outcome in a large retrospective cohort, including adults and patients with rescue ITI, treated with a pdFVIII/VWF concentrate. Retrospective data from haemophilic patients (FVIII< 2%) with inhibitors from 22 centres in Spain, Italy and Germany, who underwent primary or rescue ITI with pdFVIII/VWF concentrate, were collected. Complete success (CS), partial success (PS) and failure were defined based on the criteria of the consensus recommendations of the 2006 International ITI Workshop. A total of 41 cases of primary ITI (32 children and 9 adults) and 19 cases of rescue ITI (17 children and 2 adults) were evaluated. Success (CS+PS) rate of 87% was achieved in primary ITI and 74% in the higher risk profile of rescue ITI. Eight of nine (85%) patients with poorest prognosis (three or more of the known risk factors of poor response to ITI) achieved success (CS+PS). CS of 100% was observed in eight primary ITI patients with titre at start of ITI ≤2.5 BU and inhibitor peak ≤25 BU. The favourable response rates in primary and rescue ITI in children and in adult patients, even in the presence of poor prognostic factors, should be encouraged for broadening the indication of immune tolerance therapy in haemophilia A patients with inhibitors.
大多数关于血友病 A 患者免疫耐受诱导 (ITI) 治疗的研究都集中在儿童的原发性 ITI 上。在这里，我们报告了一项大型回顾性队列的 ITI 结果，该队列包括接受 pdFVIII/VWF 浓缩物治疗的成年人和接受挽救性 ITI 的患者。收集了来自西班牙、意大利和德国 22 个中心的血友病患者（FVIII<2%）的回顾性数据，这些患者因抑制剂接受了 pdFVIII/VWF 浓缩物的原发性或挽救性 ITI。根据 2006 年国际 ITI 研讨会共识建议的标准，定义了完全成功 (CS)、部分成功 (PS) 和失败。共评估了 41 例原发性 ITI（32 例儿童和 9 例成人）和 19 例挽救性 ITI（17 例儿童和 2 例成人）。原发性 ITI 的成功率（CS+PS）为 87%，风险较高的挽救性 ITI 为 74%。在预后最差的 8 名患者（对 ITI 反应不良的已知危险因素中有 3 个或更多）中，有 85%（8 名）获得了成功（CS+PS）。在开始 ITI 时抑制剂滴度≤2.5 BU 和抑制剂峰值≤25 BU 的 8 名原发性 ITI 患者中观察到 100%的 CS。儿童和成年患者的原发性和挽救性 ITI 的良好反应率，即使存在预后不良因素，也应该鼓励扩大血友病 A 患者抑制剂的免疫耐受治疗的适应证。
