Patient-centred composite scores as tools for assesment of response to biological therapy for paediatric and adult severe asthma
Authors: Ekaterina Khaleva, Chris 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 A 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 J.H. Vijverberg, Peter A B Wark, Michael E. Wechsler, Valentyna Yasinska, Graham Roberts
Journal: European Respiratory Journal (2024)

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 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). Methods International 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. Results Five 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&lt;0.0001)). Conclusions We have developed novel patient-centred paediatric and adult CONFiRMs which include QoL 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.