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

Ekaterina Khaleva; Anna Rattu; 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 Koppelman; Erik 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; Vera Mahler; Ann-Marie M Schoos; COMSA working group in the 3TR consortium

doi:10.1183/13993003.00606-2022

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

Ekaterina Khaleva, Anna Rattu, 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 Koppelman, Erik MelénClare 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, Vera Mahler, Ann-Marie M Schoos, COMSA working group in the 3TR consortium

38 Citationer (Scopus)

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.

Originalsprog	Engelsk
Artikelnummer	2200606
Tidsskrift	The European respiratory journal
Vol/bind	61
Udgave nummer	4
ISSN	0903-1936
DOI	https://doi.org/10.1183/13993003.00606-2022
Status	Udgivet - apr. 2023

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10.1183/13993003.00606-2022Licens: CC BY-NC

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Khaleva, E., Rattu, A., Brightling, C., Bush, A., Bossios, A., Bourdin, A., Chung, K. F., Chaudhuri, R., Coleman, C., Dahlén, S.-E., Djukanovic, R., Deschildre, A., Fleming, L., Fowler, S. J., Gupta, A., Hamelmann, E., Hashimoto, S., Hedlin, G., Koppelman, G. H., ... COMSA working group in the 3TR consortium (2023). Development of Core Outcome Measures sets for paediatric and adult Severe Asthma (COMSA). The European respiratory journal, 61(4), Artikel 2200606. https://doi.org/10.1183/13993003.00606-2022

Khaleva, E, Rattu, A, Brightling, C, Bush, A, Bossios, A, Bourdin, A, Chung, KF, Chaudhuri, R, Coleman, C, Dahlén, S-E, Djukanovic, R, Deschildre, A, Fleming, L, Fowler, SJ, Gupta, A, Hamelmann, E, Hashimoto, S, Hedlin, G, Koppelman, GH, Melén, E, Murray, CS, Pilette, C, Porsbjerg, C, Pike, KC, Rusconi, F, Williams, C, Ahrens, B, Alter, P, Anckers, F, van den Berge, M, Blumchen, K, Brusselle, G, Clarke, GW, Cunoosamy, D, Dahlén, B, Dixey, P, Exley, A, Frey, U, Gaillard, EA, Giovannini-Chami, L, Grigg, J, Hartenstein, D, Heaney, LG, Karadag, B, Kaul, S, Kull, I, Licari, A, Maitland-van der Zee, AH, Mahler, V, Schoos, A-MM & COMSA working group in the 3TR consortium 2023, 'Development of Core Outcome Measures sets for paediatric and adult Severe Asthma (COMSA)', The European respiratory journal, bind 61, nr. 4, 2200606. https://doi.org/10.1183/13993003.00606-2022

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title = "Development of Core Outcome Measures sets for paediatric and adult Severe Asthma (COMSA)",

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.",

keywords = "Adult, Anti-Asthmatic Agents/therapeutic use, Asthma/drug therapy, Child, Disease Progression, Humans, Outcome Assessment, Health Care, Quality of Life, Reproducibility of Results",

author = "Ekaterina Khaleva and Anna Rattu and Chris Brightling and Andrew Bush and Apostolos Bossios and Arnaud Bourdin and Chung, {Kian Fan} and Rekha Chaudhuri and Courtney Coleman and Sven-Erik Dahl{\'e}n and Ratko Djukanovic and Antoine Deschildre and Louise Fleming and Fowler, {Stephen J} and Atul Gupta and Eckard Hamelmann and Simone Hashimoto and Gunilla Hedlin and Koppelman, {Gerard H} and Erik Mel{\'e}n and Murray, {Clare S} and Charles Pilette and Celeste Porsbjerg and Pike, {Katharine C} and Franca Rusconi and Clare Williams and Birgit Ahrens and Peter Alter and Freja Anckers and {van den Berge}, Maarten and Katharina Blumchen and Guy Brusselle and Clarke, {Graham W} and Danen Cunoosamy and Barbro Dahl{\'e}n and Piers Dixey and Andrew Exley and Urs Frey and Gaillard, {Erol A} and Lisa Giovannini-Chami and Jonathan Grigg and Diana Hartenstein and Heaney, {Liam G} and B{\"u}lent Karadag and Susanne Kaul and Inger Kull and Amelia Licari and {Maitland-van der Zee}, {Anke H} and Vera Mahler and Schoos, {Ann-Marie M} and {COMSA working group in the 3TR consortium}",

note = "Copyright {\textcopyright}The authors 2022. For reproduction rights and permissions contact [email protected].",

year = "2023",

month = apr,

doi = "10.1183/13993003.00606-2022",

language = "English",

volume = "61",

journal = "The European respiratory journal",

issn = "0903-1936",

publisher = "European Respiratory Society",

number = "4",

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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 H

AU - Mahler, Vera

AU - Schoos, Ann-Marie M

AU - COMSA working group in the 3TR consortium

PY - 2023/4

Y1 - 2023/4

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.

KW - Adult

KW - Anti-Asthmatic Agents/therapeutic use

KW - Asthma/drug therapy

KW - Child

KW - Disease Progression

KW - Humans

KW - Outcome Assessment, Health Care

KW - Quality of Life

KW - Reproducibility of Results

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U2 - 10.1183/13993003.00606-2022

DO - 10.1183/13993003.00606-2022

M3 - Review

C2 - 36229046

SN - 0903-1936

VL - 61

JO - The European respiratory journal

JF - The European respiratory journal

IS - 4

M1 - 2200606

ER -

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

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