Core Outcome Measures in Effectiveness Trials

Foundation for Sarcoidosis Research (FSR) Endpoints Initiative

General Information

Sarcoidosis is a poorly understood disease that most commonly affects the lungs but can also involve almost every part of the body including the heart. The exact cause of sarcoidosis is unknown. Granulomas appear to be the result of an immune system response to an unidentified trigger. Infectious agents as well as environmental exposures are thought to be possible precipitants for this
immune response; however, no clear triggers or causes have been identified. Genetic mutations in white blood cell proteins (called human leukocyte antigens, or HLA) as well as chemicals that control inflammation (called cytokines) have also been linked to sarcoidosis.

Pulmonary involvement, found in over 90% of sarcoidosis patients, is the most frequent manifestation of sarcoidosis. Attention may be first drawn by abnormalities on routine chest X-ray. Systemic symptoms such as fatigue, night sweats, and weight loss are common. In addition to pulmonary disease, sarcoidosis may cause cardiac, neurologic, ocular, cutaneous, hepatic, and renal involvement. The management of sarcoidosis aims at preventing/controlling organ damage, relieving symptoms, and improving the patient’s quality of life.

Learning Objectives
1. Review current findings from all individual endpoints that have been studied and compared in various pulmonary sarcoidosis trials
2. Continue to engage the current FSR Endpoints Steering Committee focused on pulmonary sarcoidosis endpoints and identify all pulmonary stakeholders in the United States that may help enumerate the various potential endpoints and vote on the relative importance of these endpoints as primary and secondary endpoints in future pulmonary sarcoidosis clinical trials
3. Adopt Delphi process to develop and document process around establishing endpoints in pulmonary sarcoidosis
4. Develop consensus around the various potential endpoints in pulmonary sarcoidosis, including physiologic, chest imaging, and health-related quality of life
5. Educate pulmonary clinicians and researchers focused on pulmonary sarcoidosis on recommended endpoints

The Delphi Approach
The Delphi Process is often used to develop and document consensus recommendations in situations where evidence is not available. In the Delphi approach, a group of expert clinicians and other appropriate stakeholders collaborate in a coordinated multistep process in constructing Clinical Outcome Sets (COS). Experts in the disease field and other appropriate stakeholders will be chosen by the SC Chairs.

Steering Committee Co-Chairs/Key External Experts:
Marc Judson, MD
Jan Grutters, MD

Noopur Singh, Research Manager at FSR
Ginger Spitzer, Executive Director at FSR

Further Study Information

Current Stage:
July 2018 - October 2018
Funding source(s):
Foundation for Sarcoidosis Research (FSR)

Health Area

Disease Category
Lungs & airways

Disease Name

Target Population

Age Range
0 - 100


Nature / type of Intervention


Delphi process
Literature review
Systematic review

Stakeholders Involved

Clinical experts
Consumers (patients)
Journal editors
Patient/ support group representatives
Pharmaceutical industry representatives
Regulatory agency representatives

Study Type

COS for clinical trials or clinical research
COS for practice

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