Pulmonary Sarcoidosis Clinical Trial Endpoints: A Delphi Study.

Abstract

BACKGROUND Corticosteroids (CS), the most commonly prescribed treatment for sarcoidosis, are associated with significant toxicity, especially with long term usage. New intervention trials designed to reduce or eliminate CS require pertinent and precise clinical trial endpoints. However, consensus is lacking. The aim of the current study is to develop consensus for trial endpoints in pulmonary sarcoidosis. METHODS A Delphi survey was developed by an Expert Panel of 30 sarcoidosis investigators. For Round 1, the panel created 97 statements regarding potential endpoints for a clinical trial of symptomatic pulmonary CS treated sarcoidosis patients. After anonymous voting in Round 2, the 97 statements were refined by all Stakeholders, including the Expert Panel and an additional 70 individuals interested in sarcoidosis therapies. RESULTS After Round 2, the Expert Panel achieved consensus for combination endpoints and 38 statements across six domains that supported the following endpoints: prednisone reduction by 50% or discontinuation, 10% predicted or greater improvement in FVC%, FEV1%, and DLCO% predicted, and improvement in the Kings Sarcoidosis Questionnaire (KSQ)-Lung and KSQ-General Health. Additionally, the majority of Stakeholders agreed to all statements which reached consensus by the Expert Panel. CONCLUSIONS Utilizing the Delphi technique, international sarcoidosis experts successfully achieved consensus for 38 specific clinical trial endpoints which can be utilized in the development of novel steroid-sparing and eliminating therapies for pulmonary sarcoidosis.