四川省儿童血友病A预防治疗临床分析

目的分析我院儿童血友病A的病例资料,了解我省儿童血友病A的预防治疗实施现状及成效。方法2008年1月1日至2015年8月1日在我院注册的血友病A患儿156例,61例(39.1%)进行过预防治疗。预防治疗开始年龄:6.8±4.7岁(0.7~16.6岁);预防治疗剂量:15.0±5.9 IU/Kg(5~33 IU/Kg);预防治疗频率:1.3±0.6次/周(0.25~2次/周)。初级预防治疗:7例(11.5%);次级预防治疗:26例(42.6%);三级预防治疗:7例(11.5%);阶段性预防治疗:21例(34.4%)。结果预防治疗前后关节出血次数分别为1.9±1.7次/月、0.8±1.2次/月,两者比较,差异具有统计学意义(P=0.000)。预防治疗后严重出血事件显著减少,差异具有统计学意义(χ2=5.536,P=0.034)。预防治疗前后分别有7例(11.5%)、16例(26.2%)患儿有血友病骨关节病,差异具有统计学意义(χ2=4.340,P=0.037)。其中,次级预防治疗期间有9例患儿(34.6%)共16个关节发展为血友病骨关节病,骨关节病的发生与预防治疗期间关节出血情况明显相关(r=-0.532,P=0.005)。研究截止时间,阶段性预防治疗有7例患儿(33.3%)共12个关节发展为血友病骨关节病。44例(72.1%)患儿进行了输血相关病毒检查,无一例感染相关病毒。38例进行了抑制物筛查,7例(18.4%)出现了抑制物。按需治疗和预防治疗每月所需费用分别为131.1元/kg、308.7元/kg,预防治疗费用是按需治疗的2.4倍。结论我省血友病A患儿实施预防治疗比例低、开始年龄晚、治疗维持时间短、药物剂量小、注射频率低,终止预防比例高。中小剂量的预防治疗(5-25 IU/Kg,1~2次/周)能明显改善关节出血、减少严重出血事件及血友病骨关节病的发生,但次级预防治疗及阶段性预防治疗并不能阻止血友病骨关节病的发生。

Clinical Analysis of Prophylaxis in Childhood Hemophilia A in Sichuan Province

Objective To analyze the prophylaxis data of Children with hemophilia A characteristic and effectiveness in Sichuan Province. Methods There were 156 cases of hemophilia A were registered in our hospital between January 1,2008 and August 1,2015,in which 61 cases(39. 1%)were treated by prophylax-is. The age of prophylaxis:6. 8 ± 4. 7 years(0. 7~16. 6 years) ,The dose:15. 0 ± 5. 9 IU/ Kg(5-33 IU/ Kg) , The frequency:1. 3 ± 0. 6 times per week ( 0. 25 ~ 2 times per week ) . ) Primary prophylaxis:7 cases (11. 5%) , Secondary prophylaxis:26 cases ( 42. 6%) , Tertiary prophylaxis:7 cases ( 11. 5%) , Short-term prophylaxis:21 cases(34. 4%). Results Joint bleeding before prophylaxis was 1. 9 ± 1. 7 times per month, significantly decreased to 0. 8 ± 1. 2 times per month after prophylaxis(P=0. 000). The severe bleeding event also significantly decreased during the prophylaxis period ( P <0. 05 ) . Before and after prophylaxis the inci-dences of hemophilic arthropathy were 11. 5% and 26. 2% respectively(χ2 =4. 340,P=0. 037). During the secondary prophylaxis,9 patients(34. 6%) ,16 joints had developed to hemophilic arthropathy. The occurrence of arthropathy was significantly associated with the joint bleeding(P=0. 005,r=-0. 532). During the short-term prophylaxis,7 patients(34. 6%),12 joints developed to arthropathy. The virus infection rate related to blood infusion was zero. The incidence of inhibitor were 18. 4%( 7/38 ) . The medical cost of prophylaxis was 308. 7 yuan/ kg per month, 2. 4 times than that in on-demand therapy, which was 131. 1 yuan/kg per month. Conclusion In Sichuan province, only a small percentage of children with hemophilia A received prophylaxis therapy. The age of prophylaxis was older,with short duration,small dosage and low frequency. The rate of giving up prophylaxis was high. Low-dose or medium-dose prophylaxis(5-25 IU/ kg,1~2 times week-ly) could significantly reduce the incidence of joint bleeding,severe bleeding events,and hemophilic arthropa-thy,but the secondary prophylaxis and the tertiary prophylaxis did not prevent the occurrence of the arthropa-thy.