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.
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.
Authors: Raymond S Douglas; Angelo Tsirbas; Mark Gordon; Diana Lee; Nicole Khadavi; Helene Chokron Garneau; Robert A Goldberg; Kenneth Cahill; Peter J Dolman; Victor Elner; Steve Feldon; Mark Lucarelli; Jimmy Uddin; Michael Kazim; Terry J Smith; Dinesh Khanna Journal: Arch Ophthalmol Date: 2009-09
Authors: Paula R Williamson; Douglas G Altman; Heather Bagley; Karen L Barnes; Jane M Blazeby; Sara T Brookes; Mike Clarke; Elizabeth Gargon; Sarah Gorst; Nicola Harman; Jamie J Kirkham; Angus McNair; Cecilia A C Prinsen; Jochen Schmitt; Caroline B Terwee; Bridget Young Journal: Trials Date: 2017-06-20 Impact factor: 2.279
Authors: John D Pauling; Joana Caetano; Corrado Campochiaro; Giacomo De Luca; Ana Maria Gheorghiu; Maria Grazia Lazzaroni; Dinesh Khanna Journal: J Scleroderma Relat Disord Date: 2019-11-25
Authors: Shikha Mittoo; Sid Frankel; Daphne LeSage; Vibeke Strand; Ami A Shah; Lisa Christopher-Stine; Sonye Danoff; Laura K Hummers; Jeffery J Swigris; Dörte Huscher; Angela M Christensen; Sophia L Cenac; Jen K Erbil; Sancia Ferguson; Ignacio Garcia-Valladares; Harmanjot K Grewal; Ana-Maria Orbai; Katherine Clegg Smith; Maithy Tran; Clifton O Bingham; Flavia V Castelino; Aryeh Fischer; Lesley Ann Saketkoo Journal: Curr Respir Med Rev Date: 2015-06