Pulmonary sarcoidosis clinical trial end-points: a Delphi study.

Area of Science:

• Pulmonary Medicine
• Clinical Trial Design
• Rheumatology

Background:

• Corticosteroids are the primary treatment for sarcoidosis but cause significant long-term toxicity.
• Developing new therapies to reduce or eliminate corticosteroid use requires precise clinical trial endpoints.
• Current consensus on trial endpoints for pulmonary sarcoidosis is lacking.

Purpose of the Study:

• To establish consensus on clinical trial endpoints for pulmonary sarcoidosis.
• To identify specific endpoints for trials evaluating novel steroid-sparing therapies.

Main Methods:

• A Delphi survey was conducted with an expert panel of 30 sarcoidosis investigators.
• An initial 97 statements on potential endpoints were refined through anonymous voting in Round 2.
• Stakeholders, including 70 additional individuals, reviewed and voted on the refined statements.

Main Results:

• Consensus was achieved on combination endpoints and 38 specific statements across six domains.
• Key endpoints include 50% prednisone reduction or discontinuation.
• Improvement in FVC%, FEV1%, DLCO%, and the Kings Sarcoidosis Questionnaire (KSQ) were also agreed upon.

Conclusions:

• The Delphi technique facilitated international expert consensus on 38 clinical trial endpoints for pulmonary sarcoidosis.
• These endpoints are crucial for developing innovative therapies that reduce or eliminate corticosteroid dependence.
• The consensus provides a foundation for future clinical trials in pulmonary sarcoidosis management.