Abstract：
Juvenile systemic sclerosis (SSc) is an orphan disease, associated with high morbidity and mortality. New treatment strategies are much needed, but clearly defining appropriate outcomes is necessary if successful therapies are to be developed. Our objective here was to propose such outcomes.
This proposal is the result of 4 face-to-face consensus meetings with a 27-member multidisciplinary team of pediatric rheumatologists, adult rheumatologists, dermatologists, pediatric cardiologists, pulmonologists, gastroenterologists, a statistician, and patients. Throughout the process, we reviewed the existing adult data in this field, the more limited pediatric literature for juvenile SSc outcomes, and data from 2 juvenile SSc patient cohorts to assist in making informed, data-driven decisions. The use of items for each domain as an outcome measure in an open label 12-month clinical trial of juvenile SSc was voted and agreed upon using a nominal group technique.
After voting, the domains agreed on were global disease activity, skin, Raynaud\'s phenomenon, digital ulcers, musculoskeletal, cardiac, pulmonary, renal, and gastrointestinal involvement, and quality of life. Fourteen outcome measures had 100% agreement, 1 item had 91% agreement, and 1 item had 86% agreement. The domains of biomarkers and growth/development were moved to the research agenda.
We reached consensus on multiple domains and items that should be assessed in an open label, 12-month clinical juvenile SSc trial as well as a research agenda for future development.
This proposal is the result of 4 face-to-face consensus meetings with a 27-member multidisciplinary team of pediatric rheumatologists, adult rheumatologists, dermatologists, pediatric cardiologists, pulmonologists, gastroenterologists, a statistician, and patients. Throughout the process, we reviewed the existing adult data in this field, the more limited pediatric literature for juvenile SSc outcomes, and data from 2 juvenile SSc patient cohorts to assist in making informed, data-driven decisions. The use of items for each domain as an outcome measure in an open label 12-month clinical trial of juvenile SSc was voted and agreed upon using a nominal group technique.
After voting, the domains agreed on were global disease activity, skin, Raynaud\'s phenomenon, digital ulcers, musculoskeletal, cardiac, pulmonary, renal, and gastrointestinal involvement, and quality of life. Fourteen outcome measures had 100% agreement, 1 item had 91% agreement, and 1 item had 86% agreement. The domains of biomarkers and growth/development were moved to the research agenda.
We reached consensus on multiple domains and items that should be assessed in an open label, 12-month clinical juvenile SSc trial as well as a research agenda for future development.
摘要：
目的：青少年系统性硬化症（jSSc）是一种孤儿病，与高发病率和死亡率相关。非常需要新的治疗策略，但如果要开发成功的治疗方法,就必须明确定义适当的结局.这里,提出了这样的结果。
方法：这项建议是与27名儿科风湿病专家的多学科团队进行四次面对面共识会议的结果，成人风湿病学家，皮肤科医生,儿科心脏病学家,肺病学家,胃肠病学家,统计学家和患者。在整个过程中,我们回顾了这个领域现有的成人数据，关于jSSc结局的儿科文献和来自两个jSSc患者队列的数据更有限，数据驱动的决策。在公开的12个月的jSSc临床试验中使用每个领域的项目作为结果度量被投票并使用名义组技术达成一致。
结果：投票后，商定的领域是:全球疾病活动，皮肤,雷诺现象,数字溃疡,肌肉骨骼，心脏,肺,肾,胃肠,和生活质量。十四个结果指标100%达成一致，一项有91%的同意，一项有86%的同意。生物标志物和生长/发育领域已移至研究议程。
结论：我们就多个领域和项目达成了共识，这些领域和项目应在开放标签的12个月临床jSSc试验以及未来发展的研究议程中进行评估。本文受版权保护。保留所有权利。
方法：这项建议是与27名儿科风湿病专家的多学科团队进行四次面对面共识会议的结果，成人风湿病学家，皮肤科医生,儿科心脏病学家,肺病学家,胃肠病学家,统计学家和患者。在整个过程中,我们回顾了这个领域现有的成人数据，关于jSSc结局的儿科文献和来自两个jSSc患者队列的数据更有限，数据驱动的决策。在公开的12个月的jSSc临床试验中使用每个领域的项目作为结果度量被投票并使用名义组技术达成一致。
结果：投票后，商定的领域是:全球疾病活动，皮肤,雷诺现象,数字溃疡,肌肉骨骼，心脏,肺,肾,胃肠,和生活质量。十四个结果指标100%达成一致，一项有91%的同意，一项有86%的同意。生物标志物和生长/发育领域已移至研究议程。
结论：我们就多个领域和项目达成了共识，这些领域和项目应在开放标签的12个月临床jSSc试验以及未来发展的研究议程中进行评估。本文受版权保护。保留所有权利。
