David Roofeh1, Shaney L Barratt2, Athol U Wells3, Leticia Kawano-Dourado4, Donald Tashkin5, Vibeke Strand6, James Seibold7, Susanna Proudman8, Kevin K Brown9, Paul F Dellaripa10, Tracy Doyle11, Thomas Leonard12, Eric L Matteson13, Chester V Oddis14, Joshua J Solomon9, Jeffrey A Sparks10, Robert Vassallo15, Lara Maxwell16, Dorcas Beaton17, Robin Christensen18, Whitney Townsend19, Dinesh Khanna20. 1. Department of Internal Medicine, Division of Rheumatology, Scleroderma Program, University of Michigan, Ann Arbor, Michigan, USA. 2. Academic Respiratory Unit, School of Clinical Sciences, University of Bristol, UK; Bristol Interstitial Lung Disease Service, North Bristol NHS Trust, Southmead, Bristol, UK. 3. Department of Internal Medicine, Division of Pulmonology, Royal Brompton Hospital and National Heart and Lung Institute; London, UK. 4. HCor Research Institute, Hospital do Coração, São Paulo, Brazil; Pulmonary Division, Heart Institute (InCor), University of Sao Paulo Medical School, São Paulo, Brazil. 5. Department of Medicine, Division of Pulmonary and Critical Care Medicine, David Geffen School of Medicine at UCLA, Los Angeles, California, USA. 6. Division of Immunology/Rheumatology, Stanford University, Palo Alto, California, USA. 7. Scleroderma Research Consultants, Aiken, South Carolina, USA. 8. Rheumatology Unit, Royal Adelaide Hospital and Professor Discipline of Medicine, University of Adelaide, Adelaide, AUS. 9. Department of Medicine, Division of Pulmonary, Critical Care and Sleep Medicine, National Jewish Hospital, Denver, Colorado, USA. 10. Department of Medicine, Division of Rheumatology, Inflammation, and Immunity, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA. 11. Department of Internal Medicine, Division of Pulmonary and Critical Care Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA. 12. From Clinical Development and Medical Affairs, Specialty Care Boehringer Ingelheim Pharmaceuticals, Inc. USA. 13. Department of Internal Medicine, Division of Rheumatology, Mayo Clinic, Rochester, Minnesota, USA. 14. Division of Rheumatology and Clinical Immunology, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania, USA. 15. Department of Internal Medicine, Division of Pulmonary and Critical Care Medicine, Mayo Clinic, Rochester, Minnesota, USA. 16. Faculty of Medicine, University of Ottawa, Ottawa, CA. 17. Institute for Work & Health and Institute for Health Policy Management and Evaluation, University of Toronto, Toronto, CA. 18. Musculoskeletal Statistics Unit, the Parker Institute, Bispebjerg and Frederiksberg Hospital, Copenhagen, & Research Unit of Rheumatology, Department of Clinical Research, University of Southern Denmark, Odense University Hospital, Odense, Denmark. 19. Taubman Health Sciences Library, University of Michigan, Ann Arbor, MI, USA. 20. Department of Internal Medicine, Division of Rheumatology, Scleroderma Program, University of Michigan, Ann Arbor, Michigan, USA. Electronic address: khannad@umich.edu.
Abstract
OBJECTIVE: The Outcome Measures in Rheumatology (OMERACT) is a research organization focused on improving health care outcomes for patients with autoimmune and musculoskeletal diseases. The Connective Tissue Disease-Interstitial Lung Disease (CTD-ILD) Working Group on Lung Physiology is a group within OMERACT charged with identifying outcome measures that should be implemented in studies of patients with CTD-ILD. The OMERACT Filter 2.1 is an evidence-based algorithm used to identify outcome measures that are truthful, feasible, and able to discriminate between groups of interest. Our objective was to summate evidence (published literature, key opinion leader input, patient perspectives) that would influence the CTD-ILD Working Group's vote to accept or reject the use of two measures of lung physiology, the forced vital capacity (FVC) and the diffusion capacity of carbon monoxide (DLco) for use in randomized controlled trials (RTCs) and longitudinal observational studies (LOSs) involving patients with systemic sclerosis associated ILD (SSc-ILD). METHODS: Patient Research Partners (those afflicted with SSc-ILD) and the CTD-ILD Working Group on Lung Physiology were polled to assess their opinion on the FVC and DLco in terms of feasibility; the CTD-ILD Working Group was also queried on these instruments' face and content validity. We then conducted a systematic literature review to identify articles in the SSc-ILD population that assessed the following measurement properties of FVC and DLco: (1) construct validity, (2) test-retest reliability, (3) longitudinal construct validity, (4) clinical trial discrimination/sensitivity to detect change in clinical trials, and (5) thresholds of meaning. Results were summarized in a Summary of Measurement Properties (SOMP) table for each instrument. OMERACT CTD-ILD Working Group members discussed and voted on the strength of evidence supporting these two instruments and voted to endorse, provisionally endorse, or not endorse either instrument. RESULTS: Forty Patient Research Partners reported these two measures are feasible (are not an unnecessary burden or represent an infeasible longitudinal assessment of their disease). A majority of the 18 CTD-ILD Working Group members voted that both the FVC and DLco are feasible and have face and content validity. The systematic literature review returned 1,447 non-duplicated articles, of which 177 met eligibility for full text review. Forty-eight studies (13 RCTs, 35 LOSs) were included in the qualitative analysis. The FVC SOMP table revealed high quality, consistent data with evidence of good performance for all five measurement properties, suggesting requisite published evidence to proceed with endorsement. The DLco SOMP table showed a lack of data to support test-retest reliability and inadequate evidence to support clinical trial discrimination. There was unanimous agreement (15 [100%]) among voting CTD-ILD Working Group members to endorse the FVC as an instrument for lung physiology in RCTs and LOSs in SSc-ILD. Based on currently available evidence, DLco did not meet the OMERACT criteria and is not recommended for use in RCTs to represent lung physiology of SSc-ILD. The OMERACT Technical Advisory Group agreed with these decisions. CONCLUSION: The OMERACT Filter 2.1 was successfully applied to the domain of lung physiology in patients with SSc-ILD. The FVC was endorsed for use in RCTs and LOSs based on the Working Group's vote; DLco was not endorsed.
OBJECTIVE: The Outcome Measures in Rheumatology (OMERACT) is a research organization focused on improving health care outcomes for patients with autoimmune and musculoskeletal diseases. The Connective Tissue Disease-Interstitial Lung Disease (CTD-ILD) Working Group on Lung Physiology is a group within OMERACT charged with identifying outcome measures that should be implemented in studies of patients with CTD-ILD. The OMERACT Filter 2.1 is an evidence-based algorithm used to identify outcome measures that are truthful, feasible, and able to discriminate between groups of interest. Our objective was to summate evidence (published literature, key opinion leader input, patient perspectives) that would influence the CTD-ILD Working Group's vote to accept or reject the use of two measures of lung physiology, the forced vital capacity (FVC) and the diffusion capacity of carbon monoxide (DLco) for use in randomized controlled trials (RTCs) and longitudinal observational studies (LOSs) involving patients with systemic sclerosis associated ILD (SSc-ILD). METHODS: Patient Research Partners (those afflicted with SSc-ILD) and the CTD-ILD Working Group on Lung Physiology were polled to assess their opinion on the FVC and DLco in terms of feasibility; the CTD-ILD Working Group was also queried on these instruments' face and content validity. We then conducted a systematic literature review to identify articles in the SSc-ILD population that assessed the following measurement properties of FVC and DLco: (1) construct validity, (2) test-retest reliability, (3) longitudinal construct validity, (4) clinical trial discrimination/sensitivity to detect change in clinical trials, and (5) thresholds of meaning. Results were summarized in a Summary of Measurement Properties (SOMP) table for each instrument. OMERACT CTD-ILD Working Group members discussed and voted on the strength of evidence supporting these two instruments and voted to endorse, provisionally endorse, or not endorse either instrument. RESULTS: Forty Patient Research Partners reported these two measures are feasible (are not an unnecessary burden or represent an infeasible longitudinal assessment of their disease). A majority of the 18 CTD-ILD Working Group members voted that both the FVC and DLco are feasible and have face and content validity. The systematic literature review returned 1,447 non-duplicated articles, of which 177 met eligibility for full text review. Forty-eight studies (13 RCTs, 35 LOSs) were included in the qualitative analysis. The FVC SOMP table revealed high quality, consistent data with evidence of good performance for all five measurement properties, suggesting requisite published evidence to proceed with endorsement. The DLco SOMP table showed a lack of data to support test-retest reliability and inadequate evidence to support clinical trial discrimination. There was unanimous agreement (15 [100%]) among voting CTD-ILD Working Group members to endorse the FVC as an instrument for lung physiology in RCTs and LOSs in SSc-ILD. Based on currently available evidence, DLco did not meet the OMERACT criteria and is not recommended for use in RCTs to represent lung physiology of SSc-ILD. The OMERACT Technical Advisory Group agreed with these decisions. CONCLUSION: The OMERACT Filter 2.1 was successfully applied to the domain of lung physiology in patients with SSc-ILD. The FVC was endorsed for use in RCTs and LOSs based on the Working Group's vote; DLco was not endorsed.
Authors: Donald P Tashkin; Robert Elashoff; Philip J Clements; Jonathan Goldin; Michael D Roth; Daniel E Furst; Edgar Arriola; Richard Silver; Charlie Strange; Marcy Bolster; James R Seibold; David J Riley; Vivien M Hsu; John Varga; Dean E Schraufnagel; Arthur Theodore; Robert Simms; Robert Wise; Fredrick Wigley; Barbara White; Virginia Steen; Charles Read; Maureen Mayes; Ed Parsley; Kamal Mubarak; M Kari Connolly; Jeffrey Golden; Mitchell Olman; Barri Fessler; Naomi Rothfield; Mark Metersky Journal: N Engl J Med Date: 2006-06-22 Impact factor: 91.245
Authors: Christopher J Ryerson; Eric Vittinghoff; Brett Ley; Joyce S Lee; Joshua J Mooney; Kirk D Jones; Brett M Elicker; Paul J Wolters; Laura L Koth; Talmadge E King; Harold R Collard Journal: Chest Date: 2014-04 Impact factor: 9.410
Authors: Rachel K Hoyles; Ross W Ellis; Jessica Wellsbury; Belinda Lees; Pauline Newlands; Nicole S L Goh; Christopher Roberts; Sujal Desai; Ariane L Herrick; Neil J McHugh; Noeleen M Foley; Stanley B Pearson; Paul Emery; Douglas J Veale; Christopher P Denton; Athol U Wells; Carol M Black; Roland M du Bois Journal: Arthritis Rheum Date: 2006-12
Authors: Elizabeth R Volkmann; Donald P Tashkin; Ning Li; Daniel E Furst; Philip J Clements; Robert M Elashoff Journal: Rheumatology (Sunnyvale) Date: 2015-06-30