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Patient-centred composite scores as tools for assesment of response to biological therapy for paediatric and adult severe asthma

Title: Patient-centred composite scores as tools for assesment of response to biological therapy for paediatric and adult severe asthma
Authors: Ekaterina Khaleva; Christopher Brightling; Thomas Eiwegger; Alan Altraja; Philippe Bégin; Katharina Blumchen; Apostolos Bossios; Arnaud Bourdin; Anneke Ten Brinke; Guy Brusselle; Roxana Silvia Bumbacea; Andrew Bush; Thomas B Casale; Graham W Clarke; Rekha Chaudhuri; Kian Fan Chung; Courtney Coleman; Jonathan Corren; Sven-Erik Dahlén; Antoine Deschildre; Ratko Djukanovic; Katrien Eger; Andrew Exley; Louise Fleming; Stephen J Fowler; Erol Gaillard; Monika Gappa; Atul Gupta; Hans Michael Haitchi; Simone Hashimoto; Liam G Heaney; Gunilla Hedlin; Markaya Henderson; Wen Hua; David J Jackson; Bülent Karadag; Constance Helen Katelaris; Mariko S Koh; Matthias Volkmar Kopp; Gerard H Koppelman; Inger Kull; Ramesh J Kurukulaaratchy; Ji-Hyang Lee; Vera Mahler; Mika Mäkelä; Matthew Masoli; Alexander G Mathioudakis; Angel Mazon; Erik Melén; Katrin Milger; Alexander Moeller; Clare S Murray; Prasad Nagakumar; Parameswaran Nair; Jenny Negus; Antonio Nieto; Nikolaos G Papadopoulos; James Paton; Mariëlle W Pijnenburg; Katharine C Pike; Celeste Porsbjerg; Anna Rattu; Hitasha Rupani; Franca Rusconi; Niels W Rutjes; Sejal Saglani; Paul Seddon; Salman Siddiqui; Florian Singer; Tomoko Tajiri; Steve Turner; John W Upham; Susanne JH Vijverberg; Peter AB Wark; Michael E Wechsler; Valentyna Yasinska; Graham Roberts
Publication Year: 2024
Collection: University of Leicester: Figshare
Subject Terms: Biomedical and clinical sciences; Cardiovascular medicine and haematology; biological therapy; conjoint analysis; patient-centred; treatment response; severe asthma
Description: BackgroundWe 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 biologics 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 (QoL).MethodsInternational expert healthcare professionals (HCPs) 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 HCPs evaluated CONFiRM's external validity.ResultsFive levels of change for each COMSA outcome were agreed. Severe exacerbations and maintenance oral corticosteroids use were rated as most important in determining both paediatric and adult CONFiRM scores. There was strong agreement between HCPs and patients, although patients assigned greater importance to QoL. The CONFiRM score quantified response to a biological from −31 (deterioration) to 69 (best possible response). Paediatric and adult CONFiRMs had good discriminative ability for a sufficient (AUC≥0.92) and a substantial (AUC≥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
Document Type: conference object
Language: unknown
Relation: 2381/28264748.v1
Availability: https://figshare.com/articles/conference_contribution/Patient-centred_composite_scores_as_tools_for_assesment_of_response_to_biological_therapy_for_paediatric_and_adult_severe_asthma/28264748
Rights: CC BY-NC 4.0
Accession Number: edsbas.A310955B
Database: BASE