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

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.