Patient-centred composite scores as tools for assesment of response to biological therapy for paediatric and adult severe asthma
Authors: Khaleva Ekaterina , Brightling Chris , Eiwegger Thomas , Altraja Alan , Bégin Philippe , Blumchen Katharina , Bossios Apostolos , Bourdin Arnaud , Ten Brinke Anneke , Brusselle Guy , Silvia Bumbacea Roxana , Bush Andrew , Casale Thomas B , Clarke Graham W. , Chaudhuri Rekha , Fan Chung Kian , Coleman Courtney , Corren Jonathan , Dahlén Sven-Erik , Deschildre Antoine , Djukanovic Ratko , Eger Katrien , Exley Andrew , Fleming Louise , Fowler Stephen J , Gaillard Erol A , Gappa Monika , Gupta Atul , Michael Haitchi Hans , Hashimoto Simone , Heaney Liam G , Hedlin Gunilla , Henderson Markaya , Hua Wen , Jackson David J , Karadag Bülent , Helen Katelaris Constance , Koh Mariko S. , Volkmar Kopp Matthias , Koppelman Gerard H. , Kull Inger , Kurukulaaratchy Ramesh J , Lee Ji-Hyang , Mahler Vera , Mäkelä Mika , Masoli Matthew , Mathioudakis Alexander G. , Mazon Angel , Melén Erik , Milger Katrin , Moeller Alexander , Murray Clare S , Nagakumar Prasad , Nair Parameswaran , Negus Jenny , Nieto Antonio , Papadopoulos Nikolaos G. , Paton James , Pijnenburg Mariëlle W , Pike Katharine C , Porsbjerg Celeste , Rattu Anna , Rupani Hitasha , Rusconi Franca , Rutjes Niels W , Saglani Sejal , Seddon Paul , Siddiqui Salman , Singer Florian , Tajiri Tomoko , Turner Steve , Upham John W , Vijverberg Susanne J.H. , Wark Peter A B , Wechsler Michael E. , Yasinska Valentyna , Roberts Graham 
Published: November 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<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.