Abstract
Objective. To assess the validation status of echocardiography with continuous Doppler (echo-Doppler) as an outcome measure in pulmonary arterial hypertension associated with systemic sclerosis (PAH-SSc).
Methods. Structured literature review on full-text English articles was performed using the PubMed and Cochrane databases. Assessment of validation of echo-Doppler was based on the OMERACT filter criteria with the domains truth (face, content, construct, and criterion validity), discrimination, and feasibility.
Results. Out of 35 studies eligible for analysis, only 5 included well defined PAH-SSc subgroups (World Health Organization criteria). Echo was considered as having face validity based on expert opinion and high number of studies using echo for evaluation of patients with SSc. Echo was considered partially validated with respect to criterion validity based on significant correlations between echo measures and right-heart catheterization in patients with SSc at risk of PAH/PH. However, echo was found to lack specificity (lack of content validity), since measurements of echo pulmonary pressure may be influenced by left-heart disease and interstitial lung disease. Data from general populations of patients with scleroderma indicate that evaluation of pulmonary artery pressure by echo might not be available in all PAH-SSc patients because of technical factors. No studies enabling evaluation of the discriminant capacity over time and treatment of echo in PAH-SSc could be identified.
Conclusion. Further studies are needed to fully validate echo-Doppler as an outcome measure in PAH-SSc. These studies would include cross-sectional analysis of baseline measures and longitudinal data of placebo and verum groups in randomized controlled trials of patients with PAH-SSc.
- SCLERODERMA
- HYPERTENSION
- ECHO-DOPPLER
- EXPERT PANEL ON OUTCOMES MEASURES IN PULMONARY ARTERIAL HYPERTENSION
- SYSTEMIC SCLEROSIS
- OUTCOMES
- CLINICAL TRIALS
Footnotes
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This study was partially supported by unrestricted educational grants from Actelion, Encysive, GSK, Pfizer, Bayer Schering, United Therapeutics. Dr. Kowal-Bielecka has received research funding from Actelion and Encysive via the EPOSS project. Dr. Pittrow has received honoraria for consultancy from Actelion, Encysive, Bayer Schering Healthcare, GSK and Pfizer in the area of pulmonary arterial hypertension and associated diseases. Dr. Huscher has received research funding from Actelion and Encysive via the EPOSS project. Dr. Denton has received consultancy fees or honoraria from Actelion, Encysive, Pfizer, GSK, BioVitrum, Aspreva and Dyax and Genzyme. He has received unrestricted research funding from Encysive, Actelion and Genzyme. Dr. Humbert has relationships with drug companies including AB Science, Actelion, Altair, Asmacure, Astrazeneca, Bayer Schering, Chiesi, GSK, MSD, Novartis, Nycomed, Pfizer, and United Therapeutics. Relationships include consultancy service and membership of scientific advisory boards. Dr. Nash has received research grants, clinical trial funding, given advice, or lectured on behalf of Actelion, Pfizer and Bayer. Prof. Rubin has received consulting income from Actelion and Encysive, and he has served as an investigator for Actelion and Encysive. Dr. Seibold has consultancy relationships and research funding from Actelion, Pfizer, Encysive, FibroGen, Centocor, Bristol-Myers Squibb, Genzyme, Lilly, Gilead, and United Therapeutics in the area of potential treatments of scleroderma and its complications. He has received lecture honoraria from Actelion, Pfizer, Encysive and United Therapeutics. His spouse is a full-time employee of Actelion. Dr. Strand has consultancy relationships with Pfizer, FibroGen, and Bristol-Myers Squibb in the area of potential treatments of scleroderma and its complications. Prof. Furst has had a consultancy relationship, has been a member of scientific advisory boards, and/or has received research funding from Abbott, Actelion, Amgen, BMS, BiogenIdec, Centocor, Genentech, Gilead, GSK, Merck, Nitec, Novartis, Roche, UCB, Wyeth, and Xoma. He has received lecture honoraria and/or lectured on behalf of Abbott, Actelion, Amgen, BMS, Biogen, Biogenidec, Centocor, Genentech, Gilead, Merck, Nitec, and UCB. Dr. Distler has consultancy relationships and/or has received research funding from Actelion, Pfizer, Encysive, FibroGen, Ergonex, NicOx, and Biovitrum in the area of potential treatments of scleroderma and its complications. He has received lecture honoraria from Actelion, Pfizer, Encysive and Ergonex.
- Accepted for publication August 20, 2009.