Preliminary core sets of measures for disease activity and damage assessment in juvenile systemic lupus erythematosus and juvenile dermatomyositis
Preliminary core sets of measures for disease activity and damage assessment in juvenile systemic lupus erythematosus and juvenile dermatomyositis
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DOI：
10.1093/rheumatology/keg403
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发表时间：
2003-12-01
期刊：
影响因子：
5.5
通讯作者：
Martini, A
中科院分区：
文献类型：
--
作者：
Ruperto, N;Ravelli, A;Martini, A
目标。确定青少年系统性红斑狼疮(JSLE)和青少年皮肌炎(JDM)疾病活动性和损害评估的初步核心结果变量集。两份问卷调查邮寄给来自46个不同国家的267名医生，要求每个成员选择并排列在评估JSLE或JDM患者的临床反应时使用的反应变量。接下来，来自34个国家的40名儿科风湿病专家开会，使用名义分组技术，选择要包括在JSLE和JDM的疾病活动和损害核心集合中的域。通过问卷调查，共筛选出41个JSLE反应变量和37个JDM反应变量并进行排序。在共识会议上，为JSLE和JDM活动或损害核心集选择的领域包括医生和父母/患者主观评估和全球评分工具。JSLE活性的特异性区域是免疫学试验和肾功能参数。对于JDM，功能能力和肌肉力量评估都是针对活动和损伤核心组进行的，而血清肌酶仅包括在活动核心组中。在这两种疾病的疾病损害核心集合中引入了一个特定的儿科领域，称为生长和发育，并建议对健康相关的生活质量进行评估，以了解疾病对患者生活方式的影响。我们制定了JSLE和JDM疾病活动性和损害评估的初步核心措施集。核心组的预期验证工作正在进行中。
Objective. To identify preliminary core sets of outcome variables for disease activity and damage assessment in juvenile systemic lupus erythematosus (JSLE) and juvenile dermatomyositis (JDM).Methods. Two questionnaire surveys were mailed to 267 physicians from 46 different countries asking each member to select and rank the response variables used when assessing clinical response in patients with JSLE or JDM. Next, 40 paediatric rheumatologists from 34 countries met and, using the nominal group technique, selected the domains to be included in the disease activity and damage core sets for JSLE and JDM.Results. A total of 41 response variables for JSLE and 37 response variables for JDM were selected and ranked through the questionnaire surveys. In the consensus conference, domains selected for both JSLE and JDM activity or damage core sets included the physician and parent/patient subjective assessments and a global score tool. Domains specific for JSLE activity were the immunological tests and the kidney function parameters. Concerning JDM, functional ability and muscle strength assessments were indicated for both activity and damage core sets, whereas serum muscle enzymes were included only in the activity core set. A specific paediatric domain called 'growth and development' was introduced in the disease damage core set for both diseases and the evaluation of health-related quality of life was advised in order to capture the influence of the disease on the patient lifestyle.Conclusions. We developed preliminary core sets of measures for disease activity and damage assessment in JSLE and JDM. The prospective validation of the core sets is in progress.