摘要
目的运用综合评估手段比较分析不同剂量FⅧ治疗下青少年重型血友病A患者的临床表型、关节结构与功能变化规律。方法43例青少年(4~18岁)重型血友病A患者,分为按需组(n=7),低剂量预防组[FⅧ10~15 U/kg,2~3次/周,且≤30 U/(kg·周),n=17],中剂量预防组[FⅧ15~20 U/kg,2~3次/周,且45~60 U/(kg·周),n=19]。比较分析其24月间临床出血表型[年出血率/年关节出血率/最严重单个关节年出血率]、关节影像评分(超声HEAD-US评分,IPSG MRI评分)、血友病关节健康评分量表(HJHS)、血友病功能独立性评分(FISH)及变化规律。使用Kruskal-Wallis检验、Wilcoxon和重复测量的方差分析进行统计检验。结果低中剂量组的年出血率较按需组明显减少(P1=0.004,P2=0.000),中剂量较低剂量组减少32.87%。按需组、低剂量组和中剂量组的年关节出血率差异有统计学意义(P1=0.001,P2=0.000,P3=0.001)。按需组、低剂量组和中剂量组的最严重单个关节年出血差异有统计学意义(P1=0.016,P2=0.000,P3=0.005),按需组出血次数渐快增加,低剂量组维持不变,中剂量组则有减缓趋势。3组患者最严重单个关节影像学评分均有进展。低中剂量组的超声评分变化值进展较按需组延缓(P1=0.002,P2=0.000),按需组评分按原速率增长,低、中剂量组均有减缓趋势。低中剂量组的MRI评分变化值进展较按需组均有延缓(P1=0.041,P2=0.000),按需、低剂量组评分增长加快,中剂量组速率保持不变。按需组的HJHS评分变化值较低中剂量组增加(P1=0.003,P2=0.000);按需组保持原有增长速度,低、中剂量组评分均出现负值趋势。按需组FISH评分降低0.29±3.09分,低中剂量组均回升,与按需组差异有统计学意义(P1=0.000,P2=0.000)。结论两年中期随访结果显示,中国青少年低中剂量FⅧ的预防治疗在保护关节结构和功能方面明显优于按需治疗,中剂量可能稍优于低剂量预防。
Objective To explore the clinical phenotype and changes in joint structure and function in adolescent patients with severe hemophilia A under different doses of FⅧ. Methods Forty-three adolescents with severe hemophilia A aged 4-18 years were divided into on-demand group(n=7), low-dose group(FⅧ dose of 10-15 U/kg, 2-3 times a week, and≤30 U/kg a week;n=17), and intermediate-dose group(FⅧ dose of 15-20 U/kg, 2-3 times a week, and 45-60 U/kg a week)(n=19). The 3 groups were compared for their clinical bleeding phenotype, annual bleeding rate(ABR), annual joint bleeding rate(AJBR),annual the most severe joint bleeding rate, joint imaging scores(ultrasound HEAD-US score and IPSG MRI score), Hemophilia Joint Health Score(HJHS) and Functional Independence Score in Hemophilia(FISH) within 24 months. Results Compared with that in on-demand group, the ABR was significantly reduced in the low-and intermediate-dose groups(P=0.004 and0.000, respectively), and was reduced by 32.87% in the intermediate-dose group as compared with the low-dose group. The AJBR(P<0.01) and annual the most severe joint bleeding rate(P<0.05) also differed significantly among the 3 groups. The number of bleeding episodes increased progressively with time in the on-demand group, remained stable in the low-dose group, and tended to decrease in the intermediate-dose group. The imaging scores of the most severe joints showed lesion progression in all the 3 groups. The ultrasound scores, which increased steadily in the on-demand group, showed significantly lowered increment rates in the low-and intermediate-dose groups(P=0.002 and 0.000, respectively). The MRI scores showed also delayed increment in the low-and intermediate-dose groups as compared with the on-demand group(P=0.041 and 0.000,respectively), and the increment was accelerated in the on-demand and low-dose group but remained stable in the intermediate-dose group. The increment of the HJHS scores was significantly higher in the on-demand group than in the lowand intermediate-dose groups
作者
孟蕾
庄金木
孙雪岩
柳竹琴
周璇
刘昊
周芳
李颖嘉
孙竞
MENG Lei;ZHUANG Jinmu;SUN Xueyan;LIU Zhuqing;ZHOU Xuan;LIU Hao;ZHOU Fang;LI Yingjia;SUN Jing(Department of Hematology,Nanfang Hospital,Southern Medical University,Guangzhou 510515,China;Department of Ultrasound,Nanfang Hospital,Southern Medical University,Guangzhou 510515,China;Department of Diagnostic Imaging,Nanfang Hospital,Southern Medical University,Guangzhou 510515,China)
出处
《南方医科大学学报》
CAS
CSCD
北大核心
2021年第4期536-542,共7页
Journal of Southern Medical University
基金
南方医科大学临床重点研究项目(LC2016ZD016)
南方医院临床研究专项(2018CR052)。
关键词
重型血友病A
青少年
关节结构与功能
剂量
severe hemophilia A
adolescent
joint structure and function
dose
