儿童与成人重型血友病 A 患者低剂量预防治疗效果的比较

摘要

Objective To evaluate the efficacies of low -dose prophylaxis on children and adults with severe he-mophilia A.Methods Sixty -four patients with hemophilia A were registered in this retrospective analysis.The adults group and the children group were distinguished by age.In addition, they were divided into prophylaxis group and on -de-mand group according to the treatment regimen.The median annual bleeding rate (ABR), the median annual joint bleed-ing rate (AJBR), the numbers of target joints, the functional independence score in hemophilia (FISH) and the quality of life (QoL) were compared between each groups.Results The ABR of children prophylaxis group (19.7 ±11.1) were significantly lower than that of children on -demand group (54.7 ±20.5) (P =0.000).However, there was no signifi-cant difference in the QoL between these two groups (P =0.928).The ABR of adults prophylaxis group (13.9 ±7.0) were significantly lower than that of adults on -demand group (36.5 ±14.7) (P =0.000).The QoL of adults prophylax-is group were significantly higher than the adults on -demand group (P =0.001).The comparison of efficacies on proph-ylaxis between children and adults showed that it was better in children than in adults in reducing the rate of bleeding.But in improving the quality of life, it was more significant in adult.Conclusion The main effects of prophylaxis therapy in children are to reduce the bleeding of joints and maintain the long -term function of joints.But in adults, whose joints are stable or the damage has been formed, the roles of prophylaxis therapy are in improving the function of damaged joints, self -care ability and quality of life.%目的：评价儿童与成人重型血友病 A 患者低剂量预防治疗疗效差异。方法回顾性分析血友病中心登记在册的重型血友病 A 患者64例。根据年龄和治疗方案划分为儿童按需组、儿童预防组、成人按需组、成人预防组，分别比较各组患者年出血次数、年关节出血次数、靶关节数目、FISH 评分、生活质量评分以及儿童与成人预防治疗下出血情况和生活质量的改善率。结果儿童预防组年平均关节出血次数（9．2±8．4）显著低于儿童按需组（34．8±18．8），差异有统计学意义（P ＝0．000），在生活质量上两者差异无统计学意义（P ＝0．928）。成人预防组年平均关节出血次数（12．2±6．4）显著低于成人按需组（29．2±12．9），差异有统计学意义（P ＝0．000），且在生活质量上较成人按需组显著提高（P ＝0．001）。儿童按需组年均关节出血次数显著高于成人按需组，但儿童预防组与成人预防组间差异无统计学意义（P ＞0．05）。儿童预防组在年均关节出血次数的改善率方面优于成人预防组（73．6％ vs 58．2％）。儿童按需组 SF －36评分显著高于成人按需组（P ＜0．05），但儿童预防组与成人预防组间差异无统计学意义（P ＞0．05）。成人预防组在生活质量改善率方面优于儿童预防组（5．0％ vs 48．9％）。结论预防治疗对于儿童主要作用为减少关节出血，有利于保持长期关节功能。而对于关节稳定或关节损害已形成的成人而言，预防治疗的作用突出表现在其改善受损的关节功能，提高生活质量方面。