TY - JOUR

T1 - Development of Core Outcome Measures sets for paediatric and adult Severe Asthma (COMSA)

AU - Khaleva, Ekaterina

AU - Rattu, Anna

AU - Brightling, Chris

AU - Bush, Andrew

AU - Bossios, Apostolos

AU - Bourdin, Arnaud

AU - Chung, Kian Fan

AU - Chaudhuri, Rekha

AU - Coleman, Courtney

AU - Dahlén, Sven Erik

AU - Djukanovic, Ratko

AU - Deschildre, Antoine

AU - Fleming, Louise

AU - Fowler, Stephen J.

AU - Gupta, Atul

AU - Hamelmann, Eckard

AU - Hashimoto, Simone

AU - Hedlin, Gunilla

AU - Koppelman, Gerard H.

AU - Melén, Erik

AU - Murray, Clare S.

AU - Pilette, Charles

AU - Porsbjerg, Celeste

AU - Pike, Katharine C.

AU - Rusconi, Franca

AU - Williams, Clare

AU - Ahrens, Birgit

AU - Alter, Peter

AU - Anckers, Freja

AU - van den Berge, Maarten

AU - Blumchen, Katharina

AU - Brusselle, Guy

AU - Clarke, Graham W.

AU - Cunoosamy, Danen

AU - Dahlén, Barbro

AU - Dixey, Piers

AU - Exley, Andrew

AU - Frey, Urs

AU - Gaillard, Erol A.

AU - Giovannini-Chami, Lisa

AU - Grigg, Jonathan

AU - Hartenstein, Diana

AU - Heaney, Liam G.

AU - Karadag, Bülent

AU - Kaul, Susanne

AU - Kull, Inger

AU - Licari, Amelia

AU - Maitland-van der Zee, Anke-Hilse

AU - Mahler, Vera

AU - Schoos, Ann Marie M.

AU - Nagakumar, Prasad

AU - Negus, Jenny

AU - Nielsen, Hanna

AU - Pijnenburg, Mariëlle

AU - Ramiconi, Valeria

AU - Romagosa Vilarnau, Sofia

AU - Principe, Stefania

AU - Rutjes, Niels W. P.

AU - Saglani, Sejal

AU - Seddon, Paul C

AU - Singer, Florian

AU - Staudinger, Heribert

AU - Turner, Steve

AU - Vijverberg, Susanne J

AU - Winders, Tonya

AU - Yasinska, Valentyna

AU - Roberts, Graham

AU - COMSA Working Group in the 3TR Consortium

A2 - Sverrild, Asger

A2 - Lapperre, Therese

N1 - Publisher Copyright: Copyright © The authors 2023.

PY - 2023

Y1 - 2023

N2 - 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.

AB - 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.

U2 - 10.1183/13993003.00606-2022

DO - 10.1183/13993003.00606-2022

M3 - Review

C2 - 36229046

AN - SCOPUS:85148996792

VL - 61

JO - The European respiratory journal

JF - The European respiratory journal

SN - 0903-1936

IS - 4

M1 - 2200606

ER -