Outcome measurements in scleroderma: results from a delphi exercise.

Area of Science:

• Rheumatology
• Clinical Trials Methodology

Background:

• Scleroderma clinical trials require standardized measures for treatment effect.
• Defining minimal clinically relevant treatment effects is essential for trial design and interpretation.

Purpose of the Study:

• To achieve expert consensus on minimal clinically relevant treatment effect values for key scleroderma outcome measures.
• To inform the design and analysis of future clinical trials in systemic sclerosis.

Main Methods:

• A two-round Delphi consensus-building exercise was conducted with 65 experts from the Scleroderma Clinical Trials Consortium (SCTC).
• Experts responded to surveys presenting various clinical scenarios and outcome measure changes.
• Median responses from the second round were used to determine consensus values.

Main Results:

• A consensus range was achieved for several outcome measures, including Modified Rodnan skin score (3-7.5 units) and HAQ-DI (0.2-0.25 units).
• The Delphi process indicated a movement towards consensus between the two rounds, with reduced standard deviations.
• Respondents adjusted an average of 33% of their initial answers in the second round.

Conclusions:

• This study provides initial consensus values for minimal clinically relevant treatment effects in scleroderma outcome measures.
• These findings address a critical knowledge gap and will aid in the design of more informative clinical trials.
• The established consensus values are subject to refinement as further clinical evidence emerges.