5例存在良好风险抑制物阳性重型血友病A患儿应用小剂量免疫耐受诱导的效果  

Therapeutic Efficacy of low-dose Immune Tolerance Induction in 5 Chinese Severe Hemophilia A Children with Inhibitor in good risk

作　　者：李杨[1] 李刚[1] 陈振萍[1] 吴心怡[1] 甄英姿[1] 唐凌[1] 吴润晖[1] 

机构地区：[1]首都医科大学附属北京儿童医院,北京100045

出　　处：《血栓与止血学》2017年第3期372-375,共4页Chinese Journal of Thrombosis and Hemostasis

基　　金：北京市自然科学基金;(7162059);诺和诺德血友病基金

摘　　要：目的免疫耐受诱导(immune tolerance induction,ITI)是消除血友病抑制物的有效方式,从经济条件出发,本文探讨了小剂量ITI的可行性。方法回顾性分析我院血友病门诊应用小剂量方案进行ITI治疗的5例病例资料。包括、基线数据;抑制物发现时间、滴度峰值、ITI治疗前出血情况;ITI治疗开始时间、应用方案、持续时间、治疗结局;ITI治疗期间出血情况。结果 5例患儿发生抑制物的中位月龄为57(18~65)月龄,有记录的抑制物滴度峰值为13.6 BU/ml(3.9-20.2 BU/ml),抑制物产生后平均出血次数约为6.31次/月/人;开始ITI治疗时中位月龄为64(范围28~91)月,从发现抑制物到开始ITI治疗间期中位月龄为20(范围7々26)月;小剂量ITI方案为25-50 U/kg,隔天一次;治疗成功的4例ITI治疗时间分别为13个月、4个月、8个月、9个月;4例经小剂量ITI治疗抑制物转阴性,随访至今未复发;1例患儿由于治疗3个月时抑制物滴度明显升高而停止治疗;ITI治疗治疗期间仅有2例患儿出血,平均出血次数为0.21次/月/人。结论对存在良好风险抑制物阳性血友病A的患儿,小剂量ITI治疗方案能有效地消除抑制物,控制出血。Objective Immune tolerance induced（ITI） is an effective way to eliminate the hemophilia inhibitor,From the economic conditions, this paper discusses the feasibility of low-dose ITI. Methods Retrospectively collected clinical information of 5 patients at our hemophilia clinic who accepted low dose ITI. Clinical information included the baseline data; Inhibitor found time, degree of peak, ITI bleeding before treatment;ITI treatment start time, application solutions, duration and treatment outcome;bleeding during ITI treatment. Results Inhibitor occurred at 57 （18-65） months. The peak titer was 13.6 BU/ml（3.9-20.2 BU/ ml）. After the appearance of inhibitor, the average bleeding rate （ABR） was 6.31 times/person/month. ITI treatment started at 64 month-old（28-91 ） , and interval between inhibitor appearance to ITI treatment was 20 months（range,7-26）. Low dose ITI was 25 to 50 u/kg Qod. For 4 successful cases,inhibitor turned negative at 13 months, 4 months, 8 months and 9 months respectively and remain negative till now. The other case suspended ITI because of inhibitor titer increasing after 3 months. Only 2 cases bleed during ITI treatment,the ABR was 0. 21. Conclusion Low dose ITI benefits hemophilia A patients with inhibitor and was effective to eliminate inhibitor and control hemorrhage.

关 键 词：儿童 重型血友病A 抑制物 小剂量免疫耐受诱导 

分 类 号：R554.1[医药卫生—血液循环系统疾病]