The European respiratory journal · 2025
Pulmonary sarcoidosis clinical trial end-points: a Delphi study.
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Baughman Robert P, Grutters Jan C, Lower Elyse E, Zhou Ying, Wijsenbeek Marlies, Wells Athol U, Veltkamp Marcel, Valeyre Dominique, Spagnolo Paolo, Sharp Michelle, Sellares Jacobo, Obi Ogugua N, Nunes Hilario, Judson Marc A, Culver Daniel A, Bonella Francesco, Azuma Arata, Korenromp Ingrid H E
Аннотация
BACKGROUND: Corticosteroids, 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 corticosteroids require pertinent and precise clinical trial end-points. However, consensus is lacking. The aim of the current study is to develop consensus for trial end-points 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 end-points for a clinical trial of symptomatic pulmonary corticosteroid-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 end-points on 38 statements across six domains that supported the following end-points: prednisone reduction by 50% or discontinuation, 10% predicted or greater improvement in forced vital capacity % predicted, forced expiratory volume in 1 s % predicted and diffusing capacity of the lung for carbon monoxide % predicted, and improvement in the King's Sarcoidosis Questionnaire Lung and General Health domains. Additionally, the majority of Stakeholders agreed to all statements which reached consensus by the Expert Panel.
CONCLUSIONS: Utilising the Delphi technique, international sarcoidosis experts successfully achieved consensus for 38 specific clinical trial end-points which can be utilised in the development of novel steroid-sparing and eliminating therapies for 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 end-points for a clinical trial of symptomatic pulmonary corticosteroid-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 end-points on 38 statements across six domains that supported the following end-points: prednisone reduction by 50% or discontinuation, 10% predicted or greater improvement in forced vital capacity % predicted, forced expiratory volume in 1 s % predicted and diffusing capacity of the lung for carbon monoxide % predicted, and improvement in the King's Sarcoidosis Questionnaire Lung and General Health domains. Additionally, the majority of Stakeholders agreed to all statements which reached consensus by the Expert Panel.
CONCLUSIONS: Utilising the Delphi technique, international sarcoidosis experts successfully achieved consensus for 38 specific clinical trial end-points which can be utilised in the development of novel steroid-sparing and eliminating therapies for pulmonary sarcoidosis.