The American College of Rheumatology Provisional Composite Response Index for Clinical Trials in Early Diffuse Cutaneous Systemic Sclerosis

被引：122

作者：

Khanna, Dinesh ^{[1
]}

Berrocal, Veronica J. ^{[1
]}

Giannini, Edward H. ^{[2
]}

Seibold, James R. ^{[3
]}

Merkel, Peter A. ^{[4
]}

Mayes, Maureen D. ^{[5
]}

Baron, Murray ^{[6
,7
]}

Clements, Philip J. ^{[8
]}

Steen, Virginia ^{[9
]}

Assassi, Shervin ^{[5
]}

Schiopu, Elena ^{[1
]}

Phillips, Kristine ^{[1
]}

Simms, Robert W. ^{[10
]}

Allanore, Yannick ^{[11
,12
]}

Denton, Christopher P. ^{[13
]}

Distler, Oliver ^{[14
]}

Johnson, Sindhu R. ^{[15
,16
]}

Matucci-Cerinic, Marco ^{[17
,18
]}

Pope, Janet E. ^{[19
,20
]}

Proudman, Susanna M. ^{[21
,22
]}

Siegel, Jeffrey ^{[23
]}

Wong, Weng Kee ^{[8
]}

Wells, Athol U. ^{[24
]}

Furst, Daniel E. ^{[8
]}

机构：

[1] Univ Michigan, Ann Arbor, MI 48109 USA

[2] Cincinnati Childrens Hosp, Cincinnati, OH USA

[3] Scleroderma Res Consultants, Litchfield, CT USA

[4] Univ Penn, Philadelphia, PA 19104 USA

[5] Univ Texas Hlth Sci Ctr Houston, Houston, TX 77030 USA

[6] McGill Univ, Jewish Gen Hosp, Montreal, PQ H3T 1E2, Canada

[7] McGill Univ, Montreal, PQ, Canada

[8] Univ Calif Los Angeles, Los Angeles, CA USA

[9] Georgetown Univ, Washington, DC USA

[10] Boston Univ, Boston, MA 02215 USA

[11] Paris Descartes Univ, Paris, France

[12] Cochin Hosp, AP HP, Paris, France

[13] Royal Free & Univ Coll London Med Sch, London, England

[14] Univ Zurich Hosp, CH-8091 Zurich, Switzerland

[15] Toronto Western Hosp, Toronto, ON M5T 2S8, Canada

[16] Univ Toronto, Toronto, ON, Canada

[17] Azienda Osped Univ Careggi, Florence, Italy

[18] Univ Florence, Florence, Italy

[19] Univ Western Ontario, Schulich Sch Med, London Campus, London, ON, Canada

[20] St Josephs Hlth Care, London, ON, Canada

[21] Royal Adelaide Hosp, Adelaide, SA 5000, Australia

[22] Univ Adelaide, Adelaide, SA, Australia

[23] Genentech Roche, San Francisco, CA USA

[24] Royal Brompton Hosp, London SW3 6LY, England

来源：

ARTHRITIS & RHEUMATOLOGY | 2016年 / 68卷 / 02期

基金：

加拿大健康研究院;

关键词：

SKIN THICKNESS SCORE; MEASURING DISEASE-ACTIVITY; PRELIMINARY DEFINITION; OUTCOME MEASURES; DOUBLE-BLIND; SCLERODERMA; METAANALYSIS; IMPROVEMENT; PLACEBO; MULTICENTER;

D O I：

10.1002/art.39501

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Objective. Early diffuse cutaneous systemic sclerosis (dcSSc) is characterized by rapid changes in the skin and internal organs. The objective of this study was to develop a composite response index in dcSSc (CRISS) for use in randomized controlled trials (RCTs). Methods. We developed 150 paper patient profiles with standardized clinical outcome elements (core set items) using patients with dcSSc. Forty scleroderma experts rated 20 patient profiles each and assessed whether each patient had improved or not improved over a period of 1 year. Using the profiles for which raters had reached a consensus on whether the patients were improved versus not improved (79% of the profiles examined), we fit logistic regression models in which the binary outcome referred to whether the patient was improved or not, and the changes in the core set items from baseline to followup were entered as covariates. We tested the final index in a previously completed RCT. Results. Sixteen of 31 core items were included in the patient profiles after a consensus meeting and review of test characteristics of patient-level data. In the logistic regression model in which the included core set items were change over 1 year in the modified Rodnan skin thickness score, the forced vital capacity, the patient and physician global assessments, and the Health Assessment Questionnaire disability index, sensitivity was 0.982 (95% confidence interval 0.982-0.983) and specificity was 0.931 (95% confidence interval 0.930-0.932), and the model with these 5 items had the highest face validity. Subjects with a significant worsening of renal or cardiopulmonary involvement were classified as not improved, regardless of improvements in other core items. With use of the index, the effect of methotrexate could be differentiated from the effect of placebo in a 1-year RCT (P=0.02). Conclusion. We have developed a CRISS that is appropriate for use as an outcome assessment in RCTs of early dcSSc.

引用

页码：299 / 311

页数：13

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