以磁共振评分评估重型血友病A患儿规律低剂量预防治疗的长期关节结局  

Study on long-term joint outcome of regular low dose prophylaxis in children with severe hemophilia A with magnetic resonance imaging score

作　　者：武跃芳 肖娟[1] 李卓[1] 周寅[1] 陆菁菁[2] 刘淑芬[3] 刘颖[3] WU Yuefang;XIAO Juan;LI Zhuo;ZHOU Yin;LU Jingjing;LIU Shufen;LIU Ying(Department of Pediatrics,Peking Union Medical College Hospital,Chinese Academy of Medical Sciences& Peking Union Medical College,Beijing 100730,China;Department of Radiology,Peking Union Medical College Hospital,Chinese Academy of Medical Sciences& Peking Union Medical College,Beijing 100730,China;Department of Rehabilitation Physiotherapy,Peking Union Medical College Hospital,Chinese Academy of Medical Sciences& Peking Union Medical College,Beijing 100730,China)

机构地区：[1]中国医学科学院北京协和医学院北京协和医院儿科,北京100730 [2]中国医学科学院北京协和医学院北京协和医院放射科,北京100730 [3]中国医学科学院北京协和医学院北京协和医院康复理疗科,北京100730

出　　处：《中国小儿血液与肿瘤杂志》2019年第3期131-135,共5页Journal of China Pediatric Blood and Cancer

摘　　要：目的对规律低剂量预防治疗重型血友病A患儿长期随访,采取磁共振等方法评估患儿的长期关节结局,并对影响关节结局的因素进行初步分析。方法 2008年1月-2011年12月就诊于北京协和医院血友病中心,接受规律低剂量FVIII预防治疗的重型血友病A患儿,进行长达10年的长期随访,收集其临床病例资料,统计患儿开始低剂量预防治疗年龄、随访时间、预防时间(总周数)、预防剂量等,应用年关节出血次数(AJBR)、靶关节数、关节磁共振评分(IPSG MRI评分)等评估患儿在长期规律低剂量预防治疗方案下关节的状态及其损伤程度。结果 21例患儿入组年龄为1岁9个月~17岁11个月,随访时间为6~10年,其关节MRI评分为2~24分(13.52±6.17),21/21(100%)有关节损伤,19/21例(91%)患儿有关节软骨损伤,17/21例(81%)患儿有骨质损伤。IPSG MRI评分越高,关节损害越严重;关节MRI评分与年关节出血次数、开始预防年龄有明显相关性(P<0.05)。结论(1)低剂量预防治疗下,长期随访100%患儿仍然有关节受损,说明低剂量方案有待提高;(2)儿童尽早开始低剂量预防性治疗,可有效减少血友病A患儿关节出血次数,可能改善关节结局;(3)关节MRI检查敏感发现关节结构破坏,可能是评估关节早期损伤的首选检查方法。Objective To evaluate the long-term joint outcome of children with severe hemophilia A by regular low dose prevention,we used some methods like IPSG Magnetic resonance imaging scale to make a preliminary analysis of the influencing factors.Methods Children with severe hemophilia A who received regular low-dose FVIII prophylaxis in the Hemophilia center of Peking Union Medical Hospital from January 2008 to December 2011 were followed up for 10 years,we collected their clinical data,such as the age,follow-up time and prevention time(total number of weeks),preventive dose,etc.We applied the annual joint hemorrhage Rate(AJBR),number of target joints,joint MRI score(IPSG MRI score)to evaluate severe hemophilia A children’s joint state and degree of injury who were under the longterm regular low-dose prophylaxis treatment.Results The age of 21 children in the group was from 1 year 9 months old to 17 year 11 months old,the follow-up time was 6~10 years,and the MRI scores of the joints were divided into 2~24 points(13.52±6.17),21/21 cases(100%)had joint injury,19/21 cases(91%)of children had articular cartilage and 17/21 cases(81%)had bone injury.The higher the IPSG MRI score was,the more serious of joint damage,The MRI score of joint was significantly correlated with the number of annual joint hemorrhage and the age of initiation prevention(P<0.05).Conclusions(1)After long-term follow up,we found that 100% cases have joint damage even with low dose preventive treatment in severe hemophilia A.The results indicate that the low dose program needs to be improved.(2)Early initiation of low-dose preventive treatment can effectively reduce the number of joint bleeding in children with hemophilia A,which may improve joint outcomes.(3)Joint MRI examination is sensitive to the discovery of joint structural damage,it may be the preferred test method for the evaluation of early joint injury.

关 键 词：重型血友病A 儿童 低剂量预防治疗 关节MRI评分 关节结局 

分 类 号：R725.5[医药卫生—儿科] R445.2[医药卫生—临床医学]