不同治疗方案对中重型血友病A患儿出血及关节活动能力的影响  

Effect of different treatment regimens on bleeding and joint mobility in children with moderate to severe hemophilia A

作　　者：申开慧 刘叶 王智怡 袁敏 汪希勇 班灵英 金皎 SHEN Kaihui;LIU Ye;WANG Zhiyi;YUAN Min;WANG Xiyong;BAN Lingying;JIN Jiao(Department of Pediatric Hematology,the Affiliated Hospital of Guizhou Medical University,Guiyang 550001,Guizhou,China;School of Clinical Medicine,Guizhou Medical University,Guiyang 550001,Guizhou,China;Department of Ultrasound,the Affiliated Hospital of Guizhou Medical University,Guiyang 550001,Guizhou,China)

机构地区：[1]贵州医科大学附属医院儿科血液专科,贵州贵阳550001 [2]贵州医科大学临床医学院,贵州贵阳550001 [3]贵州医科大学附属医院超声科,贵州贵阳550001

出　　处：《贵州医科大学学报》2024年第11期1651-1657,共7页Journal of Guizhou Medical University

基　　金：贵州省卫健委自然科学基金(gzwkj2023-414)。

摘　　要：目的探讨不同治疗方案对中重型血友病A(HA)患儿出血及关节活动能力的治疗效果。方法56例中重型HA患儿据治疗方式不同分为按需治疗组(n=6)、按需转预防组(n=42)及预防治疗组(n=8),治疗并随访2年,收集各组HA患者随访前的一般临床资料[年龄、血友病分型、确诊至开始治疗的时间、治疗年限、FⅧ抑制物、体质量指数(BMI)及治疗方案转换];于随访开始和结束时,记录患者的年出血率(ABR)和年关节出血率(AJBR),采用血友病关节超声评估量表(HEAD-US-C)、血友病关节健康评分量表(HJHS)及血友病功能独立性评分量表(FISH)分别评估患者关节结构、关节功能及关节活动能力,同时判断患者的疗效,采用Spearson相关方法分析HA患儿FISH评分与一般临床特征、HEAD-US-C、HJHS的关系。结果随访开始与随访结束时比较,按需转预防治疗组HA患者ABR、AJBR比较、差异有统计学意义(P<0.05),预防治疗组HA患者ABR、HJHS评分比较、差异有统计学意义(P<0.05),其余各量表评分组内比较差异无统计学意义(P>0.05);HEAD-US-C评分标准下疾病改善及维持组与进展组HA患者年龄比较,差异有统计学意义(P<0.05);FISH评分与HJHS评分呈负相关(P<0.05),进一步分析FISH评分的各个维度、自我照顾能力与HJHS评分呈负相关(P<0.05),行走能力与HEAD-US-C、HJHS评分呈负相关(P<0.05),关节功能状态是HA患者活动能力的影响因素。结论按需转预防治疗方式对HA患者ABR及AJBR的治疗改善最优,其次是预防性治疗方式,最差是按需治疗方式;关节结构及功能状况可影响HA患者关节活动能力。Objective To explore the therapeutic effect of different treatment regimens on bleeding and joint mobility in children with hemophilia A(HA).Methods A total of 56 children with moderate to severe HA were divided into on-demand treatment group(n=6),on-demand to prophylaxis group(from on-demand conversion to prophylaxis,n=42)and prophylaxis group(n=8)according to different treatment regimens.They were treated and followed up for 2 years.Each group was collected for general clinical data of HA patients before follow-up[age,classification of hemophilia,time from diagnosis to treatment initiation,treatment duration,FⅧinhibitors,body mass index(BMI),and treatment regimen switching].Annual bleeding rate(ABR)and annual joint bleeding rate(AJBR)of the patients were recorded.Haemophilic Early Arthropathy Detection with UltraSound in China(HEAD-US-C),Hemophilia Joint Health Score(HJHS),and Hemophilia Functional Independence Score for Hemophilia(FISH)were used to evaluate the joint structure,joint function and joint mobility of the patients,respectively,and the therapeutic efficacy of the patients was simultaneously evaluated.Spearman correlation was used to analyze the association of FISH score with general clinical characteristics,HEAD-US-C and HJHS in the children with HA.Results There were a statistically significant differences in ABR and AJBR scores in on-demand to prophylaxis group between at the beginning and end of the follow-up(P<0.05).There were statistically significant differences(P<0.05)in ABR and HJHS scores between patients in prophylaxis group.There were no statistically significant differences in other scores within intragroup(P>0.05).There was a statistically significant difference in HA patients’age among disease improvement,maintenance and progression groups under HEAD-US-C scoring criteria(P<0.05).FISH score was negatively correlated with HJHS score(P<0.05).Further analysis showed that each dimension of FISH score and self-care ability were negatively associated with HJHS score(P<0.05).Walking abili

关 键 词：血友病A 关节 儿童 影响因素分析 关节结构及功能 活动功能 

分 类 号：R725.5[医药卫生—儿科]