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

被引：31

作者：

Khaleva, Ekaterina ^{[1
]}

Rattu, Anna ^{[1
]}

Brightling, Chris ^{[2
]}

Bush, Andrew ^{[3
,4
]}

Bossios, Apostolos ^{[5
,6
]}

Bourdin, Arnaud ^{[7
]}

Chung, Kian Fan ^{[8
]}

Chaudhuri, Rekha ^{[9
]}

Coleman, Courtney ^{[10
]}

Dahlen, Sven-Erik ^{[5
,6
]}

Djukanovic, Ratko ^{[1
,11
]}

Deschildre, Antoine ^{[12
,13
]}

Fleming, Louise ^{[8
]}

Fowler, Stephen J. ^{[14
]}

Gupta, Atul ^{[15
]}

Hamelmann, Eckard ^{[16
]}

Hashimoto, Simone ^{[17
,18
]}

Hedlin, Gunilla ^{[19
,20
]}

Koppelman, Gerard H. ^{[21
,22
]}

Melen, Erik ^{[23
]}

Murray, Clare S. ^{[14
]}

Pilette, Charles ^{[24
,25
]}

Porsbjerg, Celeste ^{[26
]}

Pike, Katharine C. ^{[27
]}

Rusconi, Franca ^{[28
]}

Williams, Clare

Ahrens, Birgit ^{[29
]}

Alter, Peter ^{[30
]}

Anckers, Freja ^{[31
]}

van den Berge, Maarten ^{[32
]}

Blumchen, Katharina ^{[33
]}

Brusselle, Guy ^{[34
]}

Clarke, Graham W. ^{[35
]}

Cunoosamy, Danen ^{[36
]}

Dahlen, Barbro ^{[5
,6
]}

Dixey, Piers ^{[8
,37
]}

Exley, Andrew ^{[38
]}

Frey, Urs ^{[39
]}

Gaillard, Erol A. ^{[40
]}

Giovannini-Chami, Lisa ^{[41
,42
]}

Grigg, Jonathan ^{[43
]}

Hartenstein, Diana ^{[29
]}

Heaney, Liam G. ^{[44
]}

Karadag, Bulent ^{[45
]}

Kaul, Susanne ^{[29
]}

Kull, Inger ^{[23
]}

Licari, Amelia ^{[46
]}

Maitland-van der Zee, Anke H. ^{[17
,18
]}

Mahler, Vera ^{[29
]}

Schoos, Ann-Marie M. ^{[47
,48
]}

机构：

[1] Univ Southampton, Clin & Expt Sci & Human Dev & Hlth, Fac Med, Southampton, Hants, England

[2] Univ Leicester, Inst Lung Hlth, Leicester NIHR Biomed Res Ctr, Leicester, Leics, England

[3] Imperial Coll, Royal Brompton Hosp, Ctr Paediat & Child Hlth, London, England

[4] Imperial Coll, Royal Brompton Hosp, Natl Heart & Lung Inst, London, England

[5] Karolinska Inst, Karolinska Univ Hosp, Dept Resp Med & Allergy, Stockholm, Sweden

[6] Karolinska Inst, Dept Med, Stockholm, Sweden

[7] Univ Montpellier, PhyMedExp, Montpellier, France

[8] Imperial Coll London, Natl Heart & Lung Inst, London, England

[9] Univ Glasgow, Inst Infect Immun & Inflammat, Glasgow, Lanark, Scotland

[10] European Lung Fdn, Sheffield, S Yorkshire, England

[11] Univ Hosp Southampton NHS Fdn Trust, NIHR Southampton Biomed Res Ctr, Southampton, Hants, England

[12] CHU Lille, Unite Pneumol & Allergol Pediat, Hop Jeanne Flandre, Lille, France

[13] Univ Lille, U1019, UMR 8204, CIIL, Lille, France

[14] Univ Manchester, Manchester Univ NHS Fdn Trust, Sch Biol Sci, Div Infect Immun & Resp Med,Fac Biol Med & Hlth,N, Manchester, Lancs, England

[15] Kings Coll Hosp London, Dept Paediat Resp Med, London, England

[16] Univ Bielefeld, Dept Pediat, Childrens Ctr Bethel, Bielefeld, Germany

[17] Amsterdam UMC Locat Univ Amsterdam, Emma Childrens Hosp, Dept Pediat Resp Med & Allergy, Amsterdam, Netherlands

[18] Univ Amsterdam, Amsterdam UMC, Dept Resp Med, Amsterdam, Netherlands

[19] Karolinska Inst, Dept Womens & Childrens Hlth, Stockholm, Sweden

[20] Karolinska Inst, Ctr Allergy Res, Stockholm, Sweden

[21] Univ Groningen, Beatrix Childrens Hosp, Dept Pediat Pulmonol & Pediat Allergol, Univ Med Ctr Groningen, Groningen, Netherlands

[22] Univ Groningen, Groningen Res Inst Asthma & COPD GRIAC, Univ Med Ctr Groningen, Groningen, Netherlands

[23] Karolinska Inst, Dept Clin Sci & Educ, Sodersjukhuset, Stockholm, Sweden

[24] UCLouvain, Inst Expt Clin Res IREC, Dept Pulmonol, Clin Univ St Luc, Brussels, Belgium

[25] UCLouvain, Inst Expt & Clin Res IREC, Pole Pulmonol ENT & Dermatol, Brussels, Belgium

[26] Bispebjerg Hosp, Resp Res Unit, Dept Resp Med, Copenhagen, Denmark

[27] Bristol Royal Hosp Children, Dept Paediat Resp Med, Bristol, Avon, England

[28] Azienda USL Toscana Nord Ovest, Dept Mother & Child Hlth, Pisa, Italy

[29] Fed Inst Vaccines & Biomed, Div Allergol, Paul Ehrlich Inst, Langen, Germany

[30] Philipps Univ Marburg UMR, Dept Med Pulm & Crit Care Med, Marburg, Germany

[31] 3TR Resp Patient Working Grp, Brussels, Belgium

[32] Univ Groningen, Univ Med Ctr Groningen, Dept Pulm Dis, Groningen, Netherlands

[33] Goethe Univ, Univ Hosp Frankfurt, Div Pneumol, Dept Children & Adolescent Med,Allergol,Cyst Fibr, Frankfurt, Germany

[34] Ghent Univ Hosp, Dept Resp Med, Ghent, Belgium

[35] AstraZeneca, Res & Early Dev, Resp & Immunol, BioPharmaceut R&D,Translat Sci & Expt Med, Gothenburg, Sweden

[36] Sanofi Genzyme, Global Med Affairs Resp Allergy & GI, Cambridge, MA USA

[37] Royal Brompton Hosp, London, England

[38] Adept Biol Consulting Ltd, London, England

[39] Univ Basel, Univ Childrens Hosp Basel, Basel, Switzerland

[40] Univ Leicester, Leicester NIHR Biomed Res Ctr Resp Theme, Dept Resp Sci, Leicester, Leics, England

[41] Hop Pediat Nice CHU Lenval, Pediat Pulmonol & Allergol Dept, Nice, France

[42] Univ Cote dAzur, Nice, France

[43] Queen Mary Univ London, Blizard Inst, Ctr Genom & Child Hlth, London, England

[44] Queens Univ Belfast, Wellcome Wolfson Ctr Expt Med, Sch Med Dent & Biomed Sci, Belfast, Antrim, North Ireland

[45] Marmara Univ, Fac Med, Div Pediat Pulmonol, Istanbul, Turkiye

[46] Univ Pavia, Pediat Clin, Fdn IRCCS Policlin San Matteo, Pavia, Italy

[47] Univ Copenhagen, Herlev & Gentofte Hosp, COpenhagen Prospect Studies Asthma Childhood COPS, Copenhagen, Denmark

[48] Slagelse Sygehus, Dept Pediat, Slagelse, Denmark

[49] Birmingham Childrens Hosp, Dept Resp Med, Birmingham, W Midlands, England

[50] Univ Birmingham, Inst Inflammat & Ageing, Birmingham, W Midlands, England

来源：

EUROPEAN RESPIRATORY JOURNAL | 2023年 / 61卷 / 04期

关键词：

QUALITY-OF-LIFE; CONTROL QUESTIONNAIRE; SYSTEMATIC REVIEWS; DIFFICULT ASTHMA; VALIDATION; CHILDREN; BURDEN; IMPAIRMENT; GUIDELINES; CHILDHOOD;

D O I：

10.1183/13993003.00606-2022

中图分类号：

R56 [呼吸系及胸部疾病];

学科分类号：

摘要：

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.

引用

页数：20

共 40 条

[31] Development of a core outcome set for clinical trials in childhood asthma: a survey of clinicians, parents, and young people
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Trials, 13
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Gallagher, Ruairi
Williamson, Paula R.
Smyth, Rosalind L.
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Sato, Emiko
Murano, Haruka
Laurent, Thomas
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Mannion, Anne F.
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Aydin, Sibel Z.
Cronholm, Peter F.
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Easley, Ebony
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Mahr, Alfred
McAlear, Carol A.
Milman, Nataliya
Robson, Joanna C.
Tomasson, Gunnar
Direskeneli, Haner
Merkel, Peter A.
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Jones, Stephanie
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Doumouchtsis, Stergios K.
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[36] Core Outcome Measures for Palliative and End-of-Life Research After Severe Stroke Mixed-Method Delphi Study
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Boyd, Kirsty
Doubal, Fergus
Barber, Mark
Brady, Marian
Cowey, Eileen
Visvanathan, Akila
Lewis, Steff
Gallacher, Katie
Morton, Sarah
Mead, Gillian E.
STROKE, 2021, 52 (11) : 3507 - 3513
[37] Development of Core Outcome Sets for trials on the management of Atrial fiBrillAtion in Critically Unwell patientS (COS-ABACUS): a protocol
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Blackwood, Bronagh
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Monteiro, Karolinne Souza
Azevedo, Matheus de Paiva
Patino, Cecilia M.
Ahmed, Sara
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[40] Development of 'Core Outcome Sets' for Meningioma in Clinical Studies (The COSMIC Project): protocol for two systematic literature reviews, eDelphi surveys and online consensus meetings
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Armstrong, Terri S.
Barrington, Heather
Bell, Sabrina
Brodbelt, Andrew R.
Bulbeck, Helen
Crofton, Anna
Dirven, Linda
Georgious, Theo
Grundy, Paul L.
Islim, Abdurrahman I.
Javadpour, Mohsen
Keshwara, Sumirat M.
Koszdin, Shelli D.
Marson, Anthony G.
McDermott, Michael W.
Meling, Torstein R.
Oliver, Kathy
Plaha, Puneet
Preusser, Matthias
Santarius, Thomas
Srikandarajah, Nisaharan
Taphoorn, Martin J. B.
Turner, Carole
Watts, Colin
Weller, Michael
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Zadeh, Gelareh
Zamanipoor Najafabadi, Amir H.
Jenkinson, Michael D.
BMJ OPEN, 2022, 12 (05): : e057384

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