Development of Core Outcome Measures sets for paediatric and adult Severe Asthma (COMSA)

Ekaterina Khaleva, Anna Rattu, COMSA Working Group in the 3TR Consortium, Chris Brightling, Andrew Bush, Apostolos Bossios, Arnaud Bourdin, Kian Fan Chung, Rekha Chaudhuri, Courtney Coleman, Sven Erik Dahlén, Ratko Djukanovic, Antoine Deschildre, Louise Fleming, Stephen J. Fowler, Atul Gupta, Eckard Hamelmann, Simone Hashimoto, Gunilla Hedlin, Gerard H. KoppelmanErik Melén, Clare S. Murray, Charles Pilette, Celeste Porsbjerg, Katharine C. Pike, Franca Rusconi, Clare Williams, Birgit Ahrens, Peter Alter, Freja Anckers, Maarten van den Berge, Katharina Blumchen, Guy Brusselle, Graham W. Clarke, Danen Cunoosamy, Barbro Dahlén, Piers Dixey, Andrew Exley, Urs Frey, Erol A. Gaillard, Lisa Giovannini-Chami, Jonathan Grigg, Diana Hartenstein, Liam G. Heaney, Bülent Karadag, Susanne Kaul, Inger Kull, Amelia Licari, Anke H. Maitland-Van der Zee, Mariëlle Pijnenburg, Martijn Nawijn

Research output: Contribution to journalReview articleAcademicpeer-review

25 Citations (Scopus)
28 Downloads (Pure)

Abstract

Background Effectiveness studies with biological therapies for asthma lack standardised outcome measures. The COMSA (Core Outcome Measures sets for paediatric and adult Severe Asthma) Working Group sought to develop Core Outcome Measures (COM) sets to facilitate better synthesis of data and appraisal of biologics in paediatric and adult asthma clinical studies. Methods COMSA utilised a multi-stakeholder consensus process among patients with severe asthma, adult and paediatric clinicians, pharmaceutical representatives, and health regulators from across Europe. Evidence included a systematic review of development, validity and reliability of selected outcome measures plus a narrative review and a pan-European survey to better understand patients’ and carers’ views about outcome measures. It was discussed using a modified GRADE (Grading of Recommendations Assessment, Development and Evaluation) Evidence to Decision framework. Anonymous voting was conducted using predefined consensus criteria. Results Both adult and paediatric COM sets include forced expiratory volume in 1 s (FEV1) as z-scores, annual frequency of severe exacerbations and maintenance oral corticosteroid use. Additionally, the paediatric COM set includes the Paediatric Asthma Quality of Life Questionnaire and Asthma Control Test or Childhood Asthma Control Test, while the adult COM set includes the Severe Asthma Questionnaire and Asthma Control Questionnaire-6 (symptoms and rescue medication use reported separately). Conclusions This patient-centred collaboration has produced two COM sets for paediatric and adult severe asthma. It is expected that they will inform the methodology of future clinical trials, enhance comparability of efficacy and effectiveness of biological therapies, and help assess their socioeconomic value. COMSA will inform definitions of non-response and response to biological therapy for severe asthma.

Original languageEnglish
Article number2200606
JournalEuropean Respiratory Journal
Volume61
Issue number4
DOIs
Publication statusPublished - 1 Apr 2023

Bibliographical note

Funding Information:
Support statement: This project has received funding from the Innovative Medicines Initiative (IMI) 2 Joint Undertaking ( JU) under grant agreement number 831434 (3TR). The JU receives support from the European Union’s Horizon 2020 research and innovation program and the European Federation of Pharmaceutical Industries and Associations (EFPIA). Further details about the 3TR project and IMI funding programme are available on their websites: www.3tr-imi.eu and www.imi.europa.eu. Funding information for this article has been deposited with the Crossref Funder Registry.

Funding Information:
The COMSA initiative is registered on the Core Outcome Measures in Effectiveness Trials (COMET) database (www.comet-initiative.org/Studies/Details/1698). The approach was adapted from the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) initiative to select outcome measurement instruments for the COM set [26] and is reported in accordance with the Core Outcome Set-STAndards for Reporting (COS-STAR) statement (supplementary table S1) [27]. Approval was gained from the Ethics Committee of the University of Southampton (Southampton, UK) (ERGO 56181). This project is part of the 3TR (Taxonomy, Treatments, Targets and Remission) Consortium (https://3tr-imi.eu) funded by the European Commission’s Innovative Medicines Initiative 2.

Publisher Copyright:
Copyright © The authors 2023.

Fingerprint

Dive into the research topics of 'Development of Core Outcome Measures sets for paediatric and adult Severe Asthma (COMSA)'. Together they form a unique fingerprint.

Cite this