用于评估青少年皮肌炎治疗反应的临时儿科风湿病国际试验组织/美国风湿病学会/欧洲抗风湿病联盟疾病活动核心集：一项前瞻性验证研究。

The provisional Paediatric Rheumatology International Trials Organisation/American College of Rheumatology/European League Against Rheumatism Disease activity core set for the evaluation of response to therapy in juvenile dermatomyositis: a prospective validation study.

作者信息

Ruperto Nicolino, Ravelli Angelo, Pistorio Angela, Ferriani Virginia, Calvo Immaculada, Ganser Gerd, Brunner Jurgen, Dannecker Guenther, Silva Clovis Arthur, Stanevicha Valda, Cate Rebecca Ten, van Suijlekom-Smit Lisette W A, Voygioyka Olga, Fischbach Michel, Foeldvari Ivan, Hilario Odete, Modesto Consuelo, Saurenmann Rotraud K, Sauvain Marie-Josephe, Scheibel Iloite, Sommelet Danièle, Tambic-Bukovac Lana, Barcellona Roberto, Brik Riva, Ehl Stephan, Jovanovic Mirjana, Rovensky Jozef, Bagnasco Francesca, Lovell Daniel J, Martini Alberto

机构信息

IRCCS G. Gaslini, Genoa, Italy.

出版信息

Arthritis Rheum. 2008 Jan 15;59(1):4-13. doi: 10.1002/art.23248.

DOI:10.1002/art.23248

PMID:18163404

Abstract

OBJECTIVE

To validate a core set of outcome measures for the evaluation of response to treatment in patients with juvenile dermatomyositis (DM).

METHODS

In 2001, a preliminary consensus-derived core set for evaluating response to therapy in juvenile DM was established. In the present study, the core set was validated through an evidence-based, large-scale data collection that led to the enrollment of 294 patients from 36 countries. Consecutive patients with active disease were assessed at baseline and after 6 months. The validation procedures included assessment of feasibility, responsiveness, discriminant and construct ability, concordance in the evaluation of response to therapy between physicians and parents, redundancy, internal consistency, and ability to predict a therapeutic response.

RESULTS

The following clinical measures were found to be feasible, and to have good construct validity, discriminative ability, and internal consistency; furthermore, they were not redundant, proved responsive to clinically important changes in disease activity, and were associated strongly with treatment outcome and thus were included in the final core set: 1) physician's global assessment of disease activity, 2) muscle strength, 3) global disease activity measure, 4) parent's global assessment of patient's well-being, 5) functional ability, and 6) health-related quality of life.

CONCLUSION

The members of the Paediatric Rheumatology International Trials Organisation, with the endorsement of the American College of Rheumatology and the European League Against Rheumatism, propose a core set of criteria for the evaluation of response to therapy that is scientifically and clinically relevant and statistically validated. The core set will help standardize the conduct and reporting of clinical trials and assist practitioners in deciding whether a child with juvenile DM has responded adequately to therapy.

摘要

目的

验证一套核心结局指标，用于评估青少年皮肌炎（DM）患者的治疗反应。

方法

2001年，建立了一套初步的、基于共识的用于评估青少年DM治疗反应的核心指标集。在本研究中，通过基于证据的大规模数据收集对该核心指标集进行验证，共纳入了来自36个国家的294例患者。对连续的活动性疾病患者在基线和6个月后进行评估。验证程序包括评估可行性、反应性、判别能力和结构效度、医生与家长对治疗反应评估的一致性、冗余性、内部一致性以及预测治疗反应的能力。

结果

发现以下临床指标可行，具有良好的结构效度、判别能力和内部一致性；此外，它们不存在冗余，对疾病活动的临床重要变化有反应，且与治疗结果密切相关，因此被纳入最终的核心指标集：1）医生对疾病活动的整体评估，2）肌肉力量，3）整体疾病活动指标，4）家长对患者健康状况的整体评估，5）功能能力，6）健康相关生活质量。