Defining appropriate outcome measures in pulmonary arterial hypertension related to systemic sclerosis: A Delphi consensus study with cluster analysis

Distler, Oliver, Behrens, Frank, Pittrow, David, Huscher, Doerte, Denton, Christopher P., Foeldvari, Ivan, Humbert, Marc, Matucci-Cerinic, Marco, Nash, Peter, Opitz, Christian F., Rubin, Lewis J., Seibold, James R., Furst, Daniel E. and EPOSS-Omeract Group (2008) Defining appropriate outcome measures in pulmonary arterial hypertension related to systemic sclerosis: A Delphi consensus study with cluster analysis. Arthritis Care and Research, 59 6: 867-875. doi:10.1002/art.23718


Author Distler, Oliver
Behrens, Frank
Pittrow, David
Huscher, Doerte
Denton, Christopher P.
Foeldvari, Ivan
Humbert, Marc
Matucci-Cerinic, Marco
Nash, Peter
Opitz, Christian F.
Rubin, Lewis J.
Seibold, James R.
Furst, Daniel E.
EPOSS-Omeract Group
Title Defining appropriate outcome measures in pulmonary arterial hypertension related to systemic sclerosis: A Delphi consensus study with cluster analysis
Journal name Arthritis Care and Research   Check publisher's open access policy
ISSN 2151-464X
2151-4658
Publication date 2008-06
Sub-type Article (original research)
DOI 10.1002/art.23718
Volume 59
Issue 6
Start page 867
End page 875
Total pages 9
Place of publication New York
Publisher John Wiley & Sons
Language eng
Subject 1103 Clinical Sciences
Formatted abstract
Objective
Outcome measures for pulmonary arterial hypertension associated with systemic sclerosis (PAH-SSc) are only partially validated. The aim of the present study was to establish an expert consensus regarding which outcome measures are most appropriate for clinical trials in PAH-SSc.

Methods
Sixty-nine PAH-SSc experts (rheumatologists, cardiologists, pulmonologists) rated a list of disease domains and measurement tools in an Internet-based 3-stage Delphi consensus study. In stages 2 and 3, the medians of domains and measurement tools and frequency distributions of ratings, along with requests for re-ratings, were distributed to respondents to provide feedback. A final score of items was identified by means of cluster analysis.

Results
The experts judged the following domains and tools as most appropriate for randomized controlled trials in PAH-SSc: lung vascular/pulmonary arterial pressure and cardiac function both measured by right heart catheterization and echocardiography, exercise testing measured by 6-minute walking test and oxygen saturation at exercise, severity of dyspnea measured on a visual analog scale, discontinuation of treatment measured by (serious) adverse events, quality of life/activities of daily living measured by the Short Form 36 and Health Assessment Questionnaire disability index, and global state assessed by physician measured by survival.

Conclusion
Among experts in PAH-SSc, a core set of outcome measures has been defined for clinical trials by Delphi consensus methods. Although these outcome measures are recommended by this expert group to be used as an interim tool, it will be necessary to formally validate the present measures, as well as potential research measures, in further studies.
Q-Index Code C1
Q-Index Status Provisional Code
Institutional Status UQ

Document type: Journal Article
Sub-type: Article (original research)
Collections: Excellence in Research Australia (ERA) - Collection
School of Medicine Publications
 
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Created: Thu, 03 Sep 2009, 09:58:43 EST by Mr Andrew Martlew on behalf of Faculty Of Health Sciences