Pulmonary sarcoidosis clinical trial end-points: a Delphi study

Robert P Baughman¹, Jan C Grutters^{2

3}, Elyse E Lower⁴, Ying Zhou⁵, Marlies Wijsenbeek⁶, Athol U Wells^{7

8

9}, Marcel Veltkamp^{2

3}, Dominique Valeyre¹⁰, Paolo Spagnolo¹¹, Michelle Sharp¹², Jacobo Sellares¹³, Ogugua N Obi¹⁴, Hilario Nunes¹⁰, Marc A Judson¹⁵, Daniel A Culver¹⁶, Francesco Bonella¹⁷, Arata Azuma^{18

19}, Ingrid H E Korenromp²

Affiliations

¹ Department of Medicine, University of Cincinnati, Cincinnati, OH, USA baughmrp@ucmail.uc.edu.
² ILD Center of Excellence, Department of Pulmonology, St Antonius Hospital, Nieuwegein, The Netherlands.
³ Division of Heart and Lungs, University Medical Center Utrecht, Utrecht, The Netherlands.
⁴ Department of Medicine, University of Cincinnati, Cincinnati, OH, USA.
⁵ Department of Pulmonary and Critical Care Medicine, Shanghai Pulmonary Hospital, Tongji University School of Medicine, Shanghai, China.
⁶ Center of Excellence for Interstitial Lung Diseases and Sarcoidoses, Department of Respiratory Medicine, Erasmus Medical Center, Rotterdam, The Netherlands.
⁷ Interstitial Lung Disease Unit, Royal Brompton Hospital, London, UK.
⁸ National Heart and Lung Institute, Imperial College London, London, UK.
⁹ Margaret Turner Warwick Centre for Fibrosing Lung Disease, Imperial College London, London, UK.
¹⁰ Service de Pneumologie, Hôpital Avicenne, Université Sorbonne Paris Nord, Paris, France.
¹¹ Respiratory Disease Unit, Department of Cardiac, Thoracic, Vascular Sciences and Public Health, University of Padova, Padova, Italy.
¹² Division of Pulmonary and Critical Care Medicine, Department of Medicine, Johns Hopkins School of Medicine, Baltimore, MD, USA.
¹³ ILD/Sarcoidosis Unit, Servei de Pneumologia, Hospital Clinic, Barcelona, Spain.
¹⁴ Division of Pulmonary, Critical Care, and Sleep Medicine, Brody School of Medicine at East Carolina University, Greenville, NC, USA.
¹⁵ Department of Medicine, Division of Pulmonary and Critical Care Medicine, Albany Medical College, Albany, NY, USA.
¹⁶ Department of Pulmonary Medicine and Critical Care, Cleveland Clinic, Cleveland, OH, USA.
¹⁷ Center for Interstitial and Rare Lung Diseases, Pneumology Department, Ruhrlandklinik University Hospital, University of Duisburg-Essen, Essen, Germany.
¹⁸ Clinical Research Center, Mihara General Hospital, Saitama, Japan.
¹⁹ Nippon Medical School, Tokyo, Japan.

PMID: 40774809
DOI: 10.1183/13993003.00943-2025

Pulmonary sarcoidosis clinical trial end-points: a Delphi study

Robert P Baughman et al. Eur Respir J. 2025.

. 2025 Oct 7;66(4):2500943.

doi: 10.1183/13993003.00943-2025. Print 2025 Oct.

Authors

Affiliations

¹ Department of Medicine, University of Cincinnati, Cincinnati, OH, USA baughmrp@ucmail.uc.edu.
² ILD Center of Excellence, Department of Pulmonology, St Antonius Hospital, Nieuwegein, The Netherlands.
³ Division of Heart and Lungs, University Medical Center Utrecht, Utrecht, The Netherlands.
⁴ Department of Medicine, University of Cincinnati, Cincinnati, OH, USA.
⁵ Department of Pulmonary and Critical Care Medicine, Shanghai Pulmonary Hospital, Tongji University School of Medicine, Shanghai, China.
⁶ Center of Excellence for Interstitial Lung Diseases and Sarcoidoses, Department of Respiratory Medicine, Erasmus Medical Center, Rotterdam, The Netherlands.
⁷ Interstitial Lung Disease Unit, Royal Brompton Hospital, London, UK.
⁸ National Heart and Lung Institute, Imperial College London, London, UK.
⁹ Margaret Turner Warwick Centre for Fibrosing Lung Disease, Imperial College London, London, UK.
¹⁰ Service de Pneumologie, Hôpital Avicenne, Université Sorbonne Paris Nord, Paris, France.
¹¹ Respiratory Disease Unit, Department of Cardiac, Thoracic, Vascular Sciences and Public Health, University of Padova, Padova, Italy.
¹² Division of Pulmonary and Critical Care Medicine, Department of Medicine, Johns Hopkins School of Medicine, Baltimore, MD, USA.
¹³ ILD/Sarcoidosis Unit, Servei de Pneumologia, Hospital Clinic, Barcelona, Spain.
¹⁴ Division of Pulmonary, Critical Care, and Sleep Medicine, Brody School of Medicine at East Carolina University, Greenville, NC, USA.
¹⁵ Department of Medicine, Division of Pulmonary and Critical Care Medicine, Albany Medical College, Albany, NY, USA.
¹⁶ Department of Pulmonary Medicine and Critical Care, Cleveland Clinic, Cleveland, OH, USA.
¹⁷ Center for Interstitial and Rare Lung Diseases, Pneumology Department, Ruhrlandklinik University Hospital, University of Duisburg-Essen, Essen, Germany.
¹⁸ Clinical Research Center, Mihara General Hospital, Saitama, Japan.
¹⁹ Nippon Medical School, Tokyo, Japan.

PMID: 40774809
DOI: 10.1183/13993003.00943-2025

Abstract

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.

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Conflict of interest statement

Conflict of interest: R.P. Baughman reports consultancy fees from aTyr, Xentria, Mallinckrodt and Foresee, payment or honoraria for lectures, presentations, manuscript writing or educational events from Mallinckrodt, support for attending meetings from WASOG, and participation on a data safety monitoring board or advisory board with Molecure. J.C. Grutters reports participation on a data safety monitoring board or advisory board with aTyr (Trial Steering Committee for phase 3 trial on efzofitimod in pulmonary sarcoidosis) and a leadership role with WASOG. E.E. Lower reports payment or honoraria for lectures, presentations, manuscript writing or educational events from WASOG. M. Wijsenbeek reports grants from the Netherlands Organisation for Health Research and Development, Dutch Lung Foundation, Dutch Pulmonary Fibrosis Organization, Sarcoidosis.nl, Boehringer Ingelheim, Hoffman-La Roche and AstraZeneca-Daiichi, consultancy fees from Agomab, AstraZeneca, Avalyn Pharma, Bristol Myers Squibb, Boehringer Ingelheim, Calluna, Galecto, GSK, Hoffman-La Roche, Horizon Therapeutics, Kinevant Sciences, Molecure, Nerre Therapeutics, Novartis, PureTech Health, Thyron, Trevi and Vicore, payment or honoraria for lectures, presentations, manuscript writing or educational events from Boehringer Ingelheim, CSL Behring, Hoffman-La Roche, Novartis and Sanofi, support for attending meetings from Boehringer Ingelheim, GSK and Hoffman-La Roche, and leadership roles with the ILD Assembly of the European Respiratory Society, Netherlands Respiratory Society, European Idiopathic Pulmonary Fibrosis and Related Disorders Federation, European Reference Network for Rare Lung Diseases, and the Dutch Lung Fibrosis and Sarcoidosis Patient Associations. A.U. Wells reports consultancy fees from Boehringer Ingelheim, Roche, Veracyte, Chiesi and CSL Behring, payment or honoraria for lectures, presentations, manuscript writing or educational events from Boehringer Ingelheim and Veracyte, and a leadership role with WASOG. M. Veltkamp reports consultancy fees from Boehringer Ingelheim, payment or honoraria for lectures, presentations, manuscript writing or educational events from Chiesi Pharmaceuticals, and participation on a data safety monitoring board or advisory board with Xentria Pharmaceuticals. D. Valeyre reports payment or honoraria for lectures, presentations, manuscript writing or educational events and support for attending meetings from Boehringer Ingelheim. P. Spagnolo reports grants from PPM Services, Roche, Boehringer Ingelheim and Chiesi, consultancy fees from PPM Services and Novartis, payment or honoraria for lectures, presentations, manuscript writing or educational events from Boehringer Ingelheim, support for attending meetings from PPM Services, participation on a data safety monitoring board or advisory board with AstraZeneca, CSL Behring, Trevi, Merck, Galapagos, Novartis and Structure Therapeutics, and the author's spouse is an employee of AstraZeneca. M. Sharp reports support for the present study from the National Heart, Lung, and Blood Institute of the National Institutes of Health, grants from aTyr Pharma, Kinevant/LabCorp, Novartis, Xentria and Molecure, consultancy fees from KeyQuest Health, payment or honoraria for lectures, presentations, manuscript writing or educational events from MSRD and NYU CME, payment for expert testimony from Veramedica Institute, support for attending meetings from the WASOG Task Force, and a leadership role with the Ann Theodore Foundation Breakthrough Sarcoidosis Initiative. J. Sellares reports grants from Boehringer, consultancy fees from Aflofarm, payment or honoraria for lectures, presentations, manuscript writing or educational events from Boehringer, Aflofarm, Adamed and Atyr, and support for attending meetings from Boehringer. O.N. Obi reports grants from Novartis, Kinevant, aTyr and Proviant, consultancy fees from CSL Behring, payment or honoraria for lectures, presentations, manuscript writing or educational events from the WASOG Clinical Trials Task Force and Ann Theodore Foundation, support for attending meetings from WASOG and Proviant, and participation on a data safety monitoring board or advisory board with the Ann Theodore Foundation and Foundation of Sarcoidosis Research. M.A. Judson reports grants from Atyr Pharmaceuticals and consultancy fees from Sparrow Pharmaceuticals, Priovant Pharmaceuticals and Merck. D.A. Culver reports grants from aTyr, Ann Theodore Foundation, Boehringer Ingelheim, Foundation for Sarcoidosis Research, Kinevant and Molecure, consultancy fees from Merck and Foresee, support for attending meetings from aTyr, participation on a data safety monitoring board or advisory board with Pliant and Fibrogen, leadership roles with WASOG and AASOG, and receipt of equipment, materials, drugs, medical writing, gifts or other services from Boehringer Ingelheim. F. Bonella reports consultancy fees from Boehringer Ingelheim, Savara Pharma and Sanofi, and payment or honoraria for lectures, presentations, manuscript writing or educational events from Boehringer Ingelheim, Savara Pharma and Sanofi. A. Azuma reports consultancy fees from Boehringer Ingelheim International GmbH, Kyorin Pharma Co. and aTyr Pharma Inc, payment or honoraria for lectures, presentations, manuscript writing or educational events from Boehringer Ingelheim International GmbH, and participation on a data safety monitoring board or advisory board with Toray Co. and Pfizer Inc. The remaining authors have no potential conflicts of interest to report.

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Pulmonary sarcoidosis clinical trial end-points: a Delphi study

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Pulmonary sarcoidosis clinical trial end-points: a Delphi study

Authors

Affiliations

Abstract

Conflict of interest statement

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