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Development of a provisional core set of response measures for clinical trials of systemic sclerosis
  1. Dinesh Khanna (dkhanna{at}mednet.ucla.edu)
  1. UCLA, United States
    1. Daniel J Lovell
    1. Cincinnati Children’s Medical Center, United Kingdom
      1. Edward Giannini
      1. Cincinnati Children’s Medical Center, United States
        1. Philip J Clements
        1. UCLA, United States
          1. Peter A Merkel
          1. Boston University, United States
            1. James R Seibold
            1. University of Michigan, United States
              1. Marco Matucci-Cerinic
              1. University of Florence, Italy
                1. Chris P Denton
                1. Royal Free Hospital, United Kingdom
                  1. Maureen D Mayes
                  1. University of Texas at Houston, United States
                    1. Virginia D Steen
                    1. Georgetown University, United States
                      1. John Varga
                      1. Northwestern University, United States
                        1. Daniel E Furst (defurst{at}mednet.ucla.edu)
                        1. UCLA, United States

                          Abstract

                          Objective: To develop a provisional core set of response measures for clinical trials of systemic sclerosis (SSc).

                          Methods: The Scleroderma Clinical Trial Consortium (SCTC) conducted a structured, 3-round Delphi exercise to reach consensus on a core set of measures for clinical trials of SSc. Round 1 asked the SCTC investigators to list items in 11 pre-defined domains (skin, musculoskeletal, cardiac, pulmonary, cardio-pulmonary, gastrointestinal, renal, Raynaud’s and digital ulcers, health-related quality of life and function, global health, and biomarkers) for SSc clinical trials. Round 2 asked respondents to rate the importance of the chosen items and was followed by a meeting during which Steering Committee discussed the feasibility, reliability, redundancy and validity of the items. Round 3 sought to obtain broader consensus on the core set measures. Members also voted on items that had data on feasibility but lacked data on reliability and validity but may still be useful outcome measures in future trials.

                          Results: Fifty SCTC investigators participated in Round 1, providing 212 unique items for the 11 domains. Forty-six (92%) participants responded in Round 2 and rated 177 items. The ratings of 177 items were reviewed by the Steering Committee and 31 items from the 11 domains were judged to be appropriate for inclusion in a 1-year multicenter clinical trial. Forty SCTC investigators completed Round 3 and ranked 30 of 31 items as acceptable for inclusion in the core set. The Steering Committee also proposed 14 items for a research agenda.

                          Conclusion: Using a Delphi exercise, we developed a provisional core set of measures for assessment of disease activity and severity in clinical trials of SSc.

                          • clinical trials
                          • provisional core set
                          • response measures
                          • systemic sclerosis

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