Development of Core Outcome Measures sets for paediatric and adult Severe Asthma (COMSA)
Authors: Khaleva Ekaterina , Rattu Anna , Brightling Chris , Bush Andrew , Bossios Apostolos , Bourdin Arnaud , Chung Kian Fan , Chaudhuri Rekha , Coleman Courtney , Dahlén Sven-Erik , Djukanovic Ratko , Deschildre Antoine , Fleming Louise , Fowler Stephen J. , Gupta Atul , Hamelmann Eckard , Hashimoto Simone , Hedlin Gunilla , Koppelman Gerard H. , Melén Erik , Murray Clare S. , Pilette Charles , Porsbjerg Celeste , Pike Katharine C. , Rusconi Franca , Williams Clare , Ahrens Birgit , Alter Peter , Anckers Freja , van den Berge Maarten , Blumchen Katharina , Brusselle Guy , Clarke Graham W. , Cunoosamy Danen , Dahlén Barbro , Dixey Piers , Exley Andrew , Frey Urs , Gaillard Erol A. , Giovannini-Chami Lisa , Grigg Jonathan , Hartenstein Diana , Heaney Liam G. , Karadag Bülent , Kaul Susanne , Kull Inger , Licari Amelia , Maitland-van der Zee Anke H. , Mahler Vera , Schoos Ann-Marie M. , Nagakumar Prasad , Negus Jenny , Nielsen Hanna , Paton James , Pijnenburg Mariëlle , Ramiconi Valeria , Romagosa Vilarnau Sofia , Principe Stefania , Rutjes Niels , Saglani Sejal , Seddon Paul , Singer Florian , Staudinger Heribert , Turner Steve , Vijverberg Susanne , Winders Tonya , Yasinska Valentyna , Roberts Graham 
Published: October 2022

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.