Connective tissue disease related interstitial lung diseases and idiopathic pulmonary fibrosis: provisional core sets of domains and instruments for use in clinical trials

被引：104

作者：

Saketkoo, Lesley Ann ^{[1
]}

Mittoo, Shikha ^{[2
]}

Huscher, Doerte ^{[3
,4
]}

Khanna, Dinesh ^{[5
]}

Dellaripa, Paul F. ^{[6
]}

Distler, Oliver ^{[7
]}

Flaherty, Kevin R. ^{[5
]}

Frankel, Sid ^{[8
]}

Oddis, Chester V. ^{[9
]}

Denton, Christopher P. ^{[10
]}

Fischer, Aryeh ^{[11
]}

Kowal-Bielecka, Otylia M. ^{[12
]}

LeSage, Daphne ^{[13
]}

Merkel, Peter A. ^{[14
]}

Phillips, Kristine ^{[5
]}

Pittrow, David ^{[15
]}

Swigris, Jeffrey ^{[11
]}

Antoniou, Katerina ^{[16
]}

Baughman, Robert P. ^{[17
]}

Castelino, Flavia V. ^{[18
]}

Christmann, Romy B. ^{[19
]}

Christopher-Stine, Lisa ^{[20
]}

Collard, Harold R. ^{[21
]}

Cottin, Vincent ^{[22
]}

Danoff, Sonye ^{[20
]}

Highland, Kristin B. ^{[23
]}

Hummers, Laura ^{[20
]}

Shah, Ami A. ^{[20
]}

Kim, Dong Soon ^{[24
]}

Lynch, David A. ^{[11
]}

Miller, Frederick W. ^{[25
]}

Proudman, Susanna M. ^{[26
]}

Richeldi, Luca ^{[27
]}

Ryu, Jay H. ^{[28
]}

Sandorfi, Nora ^{[14
]}

Sarver, Catherine ^{[29
]}

Wells, Athol U. ^{[30
,31
]}

Strand, Vibeke ^{[32
]}

Matteson, Eric L. ^{[28
]}

Brown, Kevin K. ^{[11
]}

Seibold, James R. ^{[33
]}

机构：

[1] Louisiana State Univ, Hlth Sci Ctr, New Orleans, LA 70112 USA

[2] Univ Toronto, Toronto, ON, Canada

[3] German Rheumatism Res Ctr, Berlin, Germany

[4] Charite, D-13353 Berlin, Germany

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

[6] Brigham & Womens Hosp, Boston, MA 02115 USA

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

[8] Univ Manitoba, Winnipeg, MB R3T 2N2, Canada

[9] Univ Pittsburgh, Pittsburgh, PA USA

[10] Royal Free Hosp, London NW3 2QG, England

[11] Natl Jewish Hlth, Denver, CO USA

[12] Med Univ Bialystok, Bialystok, Poland

[13] Patient Res Partner, Off Publ Hlth, New Orleans, LA USA

[14] Univ Penn, Philadelphia, PA 19104 USA

[15] Univ Dresden, Dresden, Germany

[16] Univ Crete, Iraklion, Greece

[17] Univ Cincinnati, Cincinnati, OH USA

[18] Massachusetts Gen Hosp, Boston, MA 02114 USA

[19] Boston Univ, Sch Med, Boston, MA 02118 USA

[20] Johns Hopkins Univ, Baltimore, MD USA

[21] Univ Calif San Francisco, San Francisco, CA 94143 USA

[22] Univ Lyon 1, F-69365 Lyon, France

[23] Cleveland Clin, Cleveland, OH 44106 USA

[24] Univ Ulsan, Asan Med Ctr, Ulsan 680749, South Korea

[25] NIEHS, NIH, Bethesda, MD USA

[26] Univ Adelaide, Adelaide, SA, Australia

[27] Univ Southampton, Resp Biomed Res Unit, Southampton SO9 5NH, Hants, England

[28] Mayo Clin, Coll Med, Rochester, MN USA

[29] Patient Res Partner, Bethesda, MD USA

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

[31] Natl Heart & Lung Inst, London, England

[32] Stanford Univ, Palo Alto, CA 94304 USA

[33] Scleroderma Res Consultants LLC, Avon, CT USA

来源：

THORAX | 2014年 / 69卷 / 05期

基金：

美国国家卫生研究院;

关键词：

GEORGES RESPIRATORY QUESTIONNAIRE; QUALITY-OF-LIFE; SYSTEMIC-SCLEROSIS; SCLERODERMA LUNG; END-POINT; VALIDITY; IPF; MORTALITY; COUGH; CYCLOPHOSPHAMIDE;

D O I：

10.1136/thoraxjnl-2013-204202

中图分类号：

R56 [呼吸系及胸部疾病];

学科分类号：

摘要：

Rationale Clinical trial design in interstitial lung diseases (ILDs) has been hampered by lack of consensus on appropriate outcome measures for reliably assessing treatment response. In the setting of connective tissue diseases (CTDs), some measures of ILD disease activity and severity may be confounded by non-pulmonary comorbidities. Methods The Connective Tissue Disease associated Interstitial Lung Disease (CTD-ILD) working group of Outcome Measures in Rheumatology-a non-profit international organisation dedicated to consensus methodology in identification of outcome measures-conducted a series of investigations which included a Delphi process including >248 ILD medical experts as well as patient focus groups culminating in a nominal group panel of ILD experts and patients. The goal was to define and develop a consensus on the status of outcome measure candidates for use in randomised controlled trials in CTD-ILD and idiopathic pulmonary fibrosis (IPF). Results A core set comprising specific measures in the domains of lung physiology, lung imaging, survival, dyspnoea, cough and health-related quality of life is proposed as appropriate for consideration for use in a hypothetical 1-year multicentre clinical trial for either CTD-ILD or IPF. As many widely used instruments were found to lack full validation, an agenda for future research is proposed. Conclusion Identification of consensus preliminary domains and instruments to measure them was attained and is a major advance anticipated to facilitate multicentre RCTs in the field.

引用

页码：428 / 436

页数：9