Identifying a core outcome set for pulmonary sarcoidosis research – the Foundation for Sarcoidosis Research – Sarcoidosis Clinical OUtcomes Taskforce (SCOUT)
Main Article Content
Keywords
pulmonary sarcoidosis, core outcome set, outcomes, trial design
Abstract
Background
Pulmonary sarcoidosis is a rare granulomatous disease of unknown aetiology. Heterogeneity in the outcomes measured in trials of treatment for pulmonary sarcoidosis has impacted on the ability to systematically compare findings, contributing to research inefficiency. The FSR-SCOUT study has aimed to address this heterogeneity by developing a core outcome set that represents a patient and health professional consensus on the most important outcomes to measure in future research for the treatment of pulmonary sarcoidosis.
Research design and methods
A systematic review of trial registries, narrative synthesis of published qualitative literature on the patient experience and results of a patient survey contributed to the development of a comprehensive list of outcomes that were rated in a two round online Delphi survey. The Delphi survey was completed by patients/carers and health professionals and the results discussed and ratified at an online consensus meeting.
Results
259 patients/carers and 51 health professionals completed both rounds of the Delphi survey. A pre-agreed definition of consensus was applied and the results discussed at an online consensus meeting attended by 17 patients and 7 health professionals). Fifteen outcomes, across five domains (physiological/clinical, treatment, resource use, quality of life, and death), reached the definition of consensus and were included in the core outcome set.
Conclusions
The core outcome set represents a patient and health professional consensus on the most important outcomes for pulmonary sarcoidosis research. The use of the core outcome set in future trials, and efforts to validate its components, will enhance the relevance of trials to stakeholders and will increase the opportunity for the research to contribute to evidence synthesis.
References
2. James WE, Koutroumpakis E, Saha B, Nathani A, Saavedra L, Yucel RM et al. Clinical Features of Extrapulmonary Sarcoidosis Without Lung Involvement. Chest. 2018;154(2):349-56. doi:https://doi.org/10.1016/j.chest.2018.02.003.
3. Culver DA, Judson MA. New advances in the management of pulmonary sarcoidosis. BMJ. 2019;367:l5553. doi:10.1136/bmj.l5553.
4. 4. Harman NL, Gorst SL, Williamson PR, Barnathan ES, Baughman RP, Judson MA et al. Scout - sarcoidosis outcomes taskforce. A systematic review of outcomes to inform the development of a core outcome set for pulmonary sarcoidosis. Sarcoidosis, vasculitis, and diffuse lung diseases : official journal of WASOG. 2021;38(3):e2021034-e. doi:10.36141/svdld.v38i3.10737.
5. Paramothayan NS, Lasserson TJ, Jones PW. Corticosteroids for pulmonary sarcoidosis. The Cochrane database of systematic reviews. 2005;2005(2):Cd001114. doi:10.1002/14651858.CD001114.pub2.
6. Paramothayan S, Lasserson TJ, Walters EH. Immunosuppressive and cytotoxic therapy for pulmonary sarcoidosis. The Cochrane database of systematic reviews. 2006(3):Cd003536. doi:10.1002/14651858.CD003536.pub2.
7. Williamson PR, Altman DG, Bagley H, Barnes KL, Blazeby JM, Brookes ST et al. The COMET Handbook: version 1.0. Trials. 2017;18(3):280. doi:10.1186/s13063-017-1978-4.
8. Baughman RP, Barriuso R, Beyer K, Boyd J, Hochreiter J, Knoet C et al. Sarcoidosis: patient treatment priorities. ERJ Open Res. 2018;4(4):00141-2018. doi:10.1183/23120541.00141-2018.
9. Kampstra NA, Grutters JC, van Beek FT, Culver DA, Baughman RP, Renzoni EA et al. First patient-centred set of outcomes for pulmonary sarcoidosis: a multicentre initiative. BMJ Open Respiratory Research. 2019;6(1):e000394. doi:10.1136/bmjresp-2018-000394.
10. Kirkham JJ, Davis K, Altman DG, Blazeby JM, Clarke M, Tunis S et al. Core Outcome Set-STAndards for Development: The COS-STAD recommendations. PLOS Medicine. 2017;14(11):e1002447. doi:10.1371/journal.pmed.1002447.
11. Gorst SL HNJM, Grutters J , Barnathan E, Baughman R, Culver D, Junk H, Kampstra N, Singh N, Sullivan E, Victorson D, Walton MK, Williamson PR. SCOUT: Sarcoidosis Clinical OUTcomes task force: protocol for the development of a core outcome set in pulmonary sarcoidosis. 2020. https://www.stopsarcoidosis.org/wp-content/uploads/SCOUT-Protocol-V2.0-4-5-20202.pdf. Accessed 19-11-2020.
12. Dodd S, Clarke M, Becker L, Mavergames C, Fish R, Williamson PR. A taxonomy has been developed for outcomes in medical research to help improve knowledge discovery. Journal of clinical epidemiology. 2018;96:84-92. doi:10.1016/j.jclinepi.2017.12.020.
13. Initative C. http://www.comet-initiative.org/delphimanager/. Accessed 10-11 2020.
14. Guyatt GH, Oxman AD, Kunz R, Atkins D, Brozek J, Vist G et al. GRADE guidelines: 2. Framing the question and deciding on important outcomes. Journal of clinical epidemiology. 2011;64(4):395-400. doi:10.1016/j.jclinepi.2010.09.012.
15. Kirkham JJ, Gorst S, Altman DG, Blazeby JM, Clarke M, Devane D et al. Core Outcome Set-STAndards for Reporting: The COS-STAR Statement. PLoS medicine. 2016;13(10):e1002148-e. doi:10.1371/journal.pmed.1002148.
16. Dodd S, Clarke M, Becker L, Mavergames C, Fish R, Williamson PR. A taxonomy has been developed for outcomes in medical research to help improve knowledge discovery. Journal of Clinical Epidemiology. 2018;96:84-92. doi:https://doi.org/10.1016/j.jclinepi.2017.12.020.
17. Baughman RP, Nagai S, Balter M, Costabel U, Drent M, du Bois R et al. Defining the clinical outcome status (COS) in sarcoidosis: results of WASOG Task Force. Sarcoidosis, vasculitis, and diffuse lung diseases : official journal of WASOG. 2011;28(1):56-64.
18. Voortman M, Hendriks CMR, Elfferich MDP, Bonella F, Møller J, De Vries J et al. The Burden of Sarcoidosis Symptoms from a Patient Perspective. Lung. 2019;197(2):155-61. doi:10.1007/s00408-019-00206-7.
19. Moor CC, van Manen MJG, van Hagen PM, Miedema JR, van den Toorn LM, Gür-Demirel Y et al. Needs, Perceptions and Education in Sarcoidosis: A Live Interactive Survey of Patients and Partners. Lung. 2018;196(5):569-75. doi:10.1007/s00408-018-0144-4.
20. Harrison NK. Cough, sarcoidosis and idiopathic pulmonary fibrosis: raw nerves and bad vibrations. Cough. 2013;9(1):9-. doi:10.1186/1745-9974-9-9.
21. Pietinalho A, Ohmichi M, Hiraga Y, Löfroos AB, Selroos O. The mode of presentation of sarcoidosis in Finland and Hokkaido, Japan. A comparative analysis of 571 Finnish and 686 Japanese patients. Sarcoidosis, vasculitis, and diffuse lung diseases : official journal of WASOG. 1996;13(2):159-66. doi:10.1007/bf00389839.
22. Michielsen HJ, Peros-Golubicic T, Drent M, De Vries J. Relationship between symptoms and quality of life in a sarcoidosis population. Respiration; international review of thoracic diseases. 2007;74(4):401-5. doi:10.1159/000092670.
23. Brignall K, Jayaraman B, Birring SS. Quality of life and psychosocial aspects of cough. Lung. 2008;186 Suppl 1:S55-8. doi:10.1007/s00408-007-9034-x.
24. Cox CE, Donohue JF, Brown CD, Kataria YP, Judson MA. The Sarcoidosis Health Questionnaire. American Journal of Respiratory and Critical Care Medicine. 2003;168(3):323-9. doi:10.1164/rccm.200211-1343OC.
25. Patel AS, Siegert RJ, Creamer D, Larkin G, Maher TM, Renzoni EA et al. The development and validation of the King's Sarcoidosis Questionnaire for the assessment of health status. Thorax. 2013;68(1):57-65. doi:10.1136/thoraxjnl-2012-201962.
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