TY - JOUR
T1 - Patient-centred composite scores as tools for assessment of response to biological therapy for paediatric and adult severe asthma
AU - behalf of the 3TR Asthma Definition of Response Working Group
AU - Khaleva, Ekaterina
AU - Brightling, Chris
AU - Eiwegger, Thomas
AU - Altraja, Alan
AU - Bégin, Philippe
AU - Blumchen, Katharina
AU - Bossios, Apostolos
AU - Bourdin, Arnaud
AU - Brinke, Anneke Ten
AU - Brusselle, Guy
AU - Bumbacea, Roxana Silvia
AU - Bush, Andrew
AU - Casale, Thomas B.
AU - Clarke, Graham W.
AU - Chaudhuri, Rekha
AU - Chung, Kian Fan
AU - Coleman, Courtney
AU - Corren, Jonathan
AU - Dahlén, Sven Erik
AU - Deschildre, Antoine
AU - Djukanovic, Ratko
AU - Eger, Katrien
AU - Exley, Andrew
AU - Fleming, Louise
AU - Fowler, Stephen J.
AU - Gaillard, Erol A.
AU - Gappa, Monika
AU - Gupta, Atul
AU - Haitchi, Hans Michael
AU - Hashimoto, Simone
AU - Heaney, Liam G.
AU - Hedlin, Gunilla
AU - Henderson, Markaya
AU - Hua, Wen
AU - Jackson, David J.
AU - Karadag, Bülent
AU - Katelaris, Constance Helen
AU - Koh, Mariko S.
AU - Kopp, Matthias Volkmar
AU - Koppelman, Gerard H.
AU - Kull, Inger
AU - Kurukulaaratchy, Ramesh J.
AU - Lee, Ji Hyang
AU - Mahler, Vera
AU - Mäkelä, Mika
AU - Masoli, Matthew
AU - Mathioudakis, Alexander G.
AU - Mazon, Angel
AU - Melén, Erik
AU - Milger, Katrin
N1 - Publisher Copyright:
Copyright ©The authors 2025.
PY - 2025
Y1 - 2025
N2 - Background We have previously developed Core Outcome Measures sets for Severe Asthma (COMSA) by multi-stakeholder consensus. There are no patient-centred tools to quantify response to biological therapies for severe asthma. We aimed to develop paediatric and adult CompOsite iNdexes For Response in asthMa (CONFiRM) incorporating clinical parameters and patient-reported quality of life. Methods International expert healthcare professionals and patients with severe asthma were invited to 1) develop consensus levels of clinically relevant changes for each outcome measure within COMSA, 2) use multicriteria decision analysis to develop the CONFiRM scores and 3) assess their internal validity. A separate group of healthcare professionals evaluated CONFiRM’s external validity. Results Five levels of change for each COMSA outcome were agreed. Severe exacerbations and maintenance oral corticosteroid use were rated as the most important in determining both paediatric and adult CONFIRM scores. There was strong agreement between healthcare professionals and patients, although patients assigned greater importance to quality of life. The CONFiRM score quantified response to a biologic from −31 (deterioration) to 69 (best possible response). Paediatric and adult CONFiRMs had good discriminative ability for a sufficient (area under the curve ≥0.92) and a substantial (area under the curve ≥0.95) response to biologics. Both CONFiRMs demonstrated excellent external validity (Spearman correlation coefficients 0.9 and 0.8 for paediatric and adult, respectively; p<0.0001). Conclusions We have developed novel patient-centred paediatric and adult CONFiRMs that include quality of life measures. CONFiRMs should allow a more holistic understanding of response for the patient and a standardised assessment of the effectiveness of biologics between studies. Further research is needed to prospectively validate CONFiRM scores.
AB - Background We have previously developed Core Outcome Measures sets for Severe Asthma (COMSA) by multi-stakeholder consensus. There are no patient-centred tools to quantify response to biological therapies for severe asthma. We aimed to develop paediatric and adult CompOsite iNdexes For Response in asthMa (CONFiRM) incorporating clinical parameters and patient-reported quality of life. Methods International expert healthcare professionals and patients with severe asthma were invited to 1) develop consensus levels of clinically relevant changes for each outcome measure within COMSA, 2) use multicriteria decision analysis to develop the CONFiRM scores and 3) assess their internal validity. A separate group of healthcare professionals evaluated CONFiRM’s external validity. Results Five levels of change for each COMSA outcome were agreed. Severe exacerbations and maintenance oral corticosteroid use were rated as the most important in determining both paediatric and adult CONFIRM scores. There was strong agreement between healthcare professionals and patients, although patients assigned greater importance to quality of life. The CONFiRM score quantified response to a biologic from −31 (deterioration) to 69 (best possible response). Paediatric and adult CONFiRMs had good discriminative ability for a sufficient (area under the curve ≥0.92) and a substantial (area under the curve ≥0.95) response to biologics. Both CONFiRMs demonstrated excellent external validity (Spearman correlation coefficients 0.9 and 0.8 for paediatric and adult, respectively; p<0.0001). Conclusions We have developed novel patient-centred paediatric and adult CONFiRMs that include quality of life measures. CONFiRMs should allow a more holistic understanding of response for the patient and a standardised assessment of the effectiveness of biologics between studies. Further research is needed to prospectively validate CONFiRM scores.
UR - https://www.scopus.com/pages/publications/105004022308
U2 - 10.1183/13993003.00691-2024
DO - 10.1183/13993003.00691-2024
M3 - Article
C2 - 39510551
AN - SCOPUS:105004022308
SN - 0903-1936
VL - 65
JO - European Respiratory Journal
JF - European Respiratory Journal
IS - 3
M1 - 2400691
ER -
SDG 3 Good Health and Well-being