摘要
目的探讨重型血友病A患儿低、中剂量重组人凝血因子Ⅷ预防治疗的临床意义。方法收集2010年3月至2016年8月进行预防治疗且预防治疗时间≥4个月的重型血友病A患儿72例，分为低剂量预防治疗组42例[10U／（kg·次），2次／周]，中剂量预防治疗组30例[15U／（kg·次），2次／周]，另选取同期重型血友病患）L30例为按需治疗组。比较三组患儿治疗前后出血次数、严重出血次数、关节出血次数、凝血因子用量及关节功能FISH评分和日常生活功能、心理状态评分。结果三组患儿治疗前年龄、体重、血友病家族史、首次治疗年龄和疾病起始信息等比较，差异均无统计学意义（p〉0．05）。三组患儿治疗4个月内，低、中剂量预防组总出血次数、关节出血总次数与按需治疗组比较，均显著减少（P〈0．05），且中剂量预防组总出血次数和关节出血总次数降低最显著（P〈0．05），按需治疗组和低剂量预防组凝血因子4个月内总用量比较差异无统计学意义（P〉0．05）；在关节功能FJSH评分、日常生活状态及心理状态评分方面，中剂量预防组改善最佳，低剂量预防组次之，差异有统计学意义（P〈0．05）。三组患儿均未出现严重不良事件，且未发现FⅧ抗体阳性病例。结论低中剂量预防治疗可显著减少重型血友病患儿的出血次数，改善关节功能、提高生活质量，经济条件允许前提下应采用中剂量预防治疗方案。
Objective To explore the clinical significance of short-term preventive treatment with low and intermediate dose of recombinant coagulation factorⅧ for children with severe hemophilia A. Methods 72 cases of children with severe hemophilia A were collected from March 2010 to August 2010 in our hospital with preventive treatment at least 4 months, and they were divided into the low dose group, 42 cases （ 10 u/kg/times, 2 times/week ） , the intermediate dose group of 30 patients （ 15 u/kg/times, 2-3times/week ）. Other 30 patients received on-demand treatment screening as the control, who were matched by age and weight.The number of annual bleeding, annual joint bleeding and annual severe bleeding event, the dosage of clotting factors, joint function FISH score, daily life function and psychological state were collected and compared among the three groups. Results There were no statistically significant difference of the baseline data such as age, weight, family history of hemophilia, age and disease treatment among the three groups of child patients （ P〉0.05 ） . The total number of bleeding and joint bleeding in the low and medium dose prevention groups of child patients in the treatment of 4 months were significantly reduced, compared with on-demand treatment group （ P〈0.05 ） , and the total number of bleeding and joint bleeding in the medium dose prevention group were reduced the most significant （ P〈0.05 ） . There was no significant difference of the total amount of clotting factor in 4 month between the on-demand treatment group and low dose prevention group （ 414.52 ± 112.53 vs 456.14 ± 126.14, P〉0.05 ） . In joint function FISH scale, daily life and mental state score, the medium dose prevention group were improved better than low dose prevention group （ P〈0.05 ） . No serious adverse events and F Ⅷ antibody positive cases were found in the process of research of the three groups. Conclusions Preventive treatment of children with severe hemophilia A with low or medium dose of clotting factor both can significantly decrease the number of hemorrhage, improve joint function and improve the quality of life. Tne intermediate-dose regimen should be used in the premise of economic conditions.
出处
《浙江临床医学》
2017年第7期1220-1221,1224,共3页
Zhejiang Clinical Medical Journal
基金
国家自然科学基金（81560350）
