Systemic sclerosis - continuing progress in developing clinical measures of response

Daniel Furst1, Dinesh Khanna, Marco Matucci-Cerinic

  • 1University of California at Los Angeles, Los Angeles, California 90025, USA. defurst@mednet.ucla.edu

Insights

Systemic sclerosis (SSc) research faces challenges in evaluating treatment effects due to limited randomized controlled trials (RCTs). Ongoing efforts focus on developing validated outcome measures and composite indices for better assessment in SSc clinical trials.

Area of Science:

  • Rheumatology
  • Clinical Trials
  • Outcome Measures

Background:

  • Systemic sclerosis (SSc) presents challenges for evaluating treatment efficacy due to limited randomized controlled trials (RCTs).
  • Existing validated outcome measures include skin score, Raynaud's measures, pulmonary function tests, and patient-reported outcomes.
  • Recent RCTs have enabled analysis of organ-specific endpoints in SSc.

Purpose of the Study:

  • To review and discuss validated outcome measures for systemic sclerosis (SSc).
  • To explore the development of new outcome measures and composite indices for SSc.
  • To facilitate consensus on outcome assessment in SSc clinical research.

Main Methods:

  • Review of existing literature and meta-analyses on SSc outcome measures.
  • Analysis of data presented at OMERACT 8 regarding lung imaging and functional tests.
  • Initiation of Delphi exercises to establish consensus on outcome measures for pulmonary arterial hypertension (PAH) and a composite SSc response index.

Main Results:

  • Established validated outcome measures include skin score, Raynaud's, pulmonary function tests, blood pressure, pain, HAQ, and SF-36.
  • Data on high-resolution CT, 6-minute walk test, and patient-reported outcomes were presented for validation.
  • Delphi exercises are underway to develop consensus for PAH measures and a composite SSc response index.

Conclusions:

  • Validated outcome measures exist for SSc, but challenges remain in evaluating treatment effects.
  • Development of composite responder indices may be beneficial given the multi-system nature of SSc.
  • Further validation of proposed outcome measures in future RCTs is essential.