Clinical importance of patient-reported outcome measures in severe asthma: results from U-BIOPRED

Roy Meys; Frits M E Franssen; Alex J Van 't Hul; Per S Bakke; Massimo Caruso; Barbro Dahlén; Stephen J Fowler; Thomas Geiser; Peter H Howarth; Ildikó Horváth; Norbert Krug; Annelie F Behndig; Florian Singer; Jacek Musial; Dominick E Shaw; Paolo Montuschi; Anke H Maitland-van der Zee; Peter J Sterk; Graham Roberts; Nazanin Z Kermani; Raffaele A Incalzi; Renaud Louis; Lars I Andersson; Scott S Wagers; Sven-Erik Dahlén; Kian Fan Chung; Ian M Adcock; Martijn A Spruit; U-BIOPRED Study Group

doi:10.1186/s12955-024-02321-3

Clinical importance of patient-reported outcome measures in severe asthma: results from U-BIOPRED

Health Qual Life Outcomes. 2024 Dec 20;22(1):109. doi: 10.1186/s12955-024-02321-3.

Authors

Roy Meys^{1

2

3}, Frits M E Franssen^{4

5

6}, Alex J Van 't Hul⁷, Per S Bakke⁸, Massimo Caruso⁹, Barbro Dahlén¹⁰, Stephen J Fowler¹¹, Thomas Geiser¹², Peter H Howarth¹³, Ildikó Horváth^{14

15}, Norbert Krug¹⁶, Annelie F Behndig¹⁷, Florian Singer^{18

19}, Jacek Musial²⁰, Dominick E Shaw²¹, Paolo Montuschi^{22

23}, Anke H Maitland-van der Zee²⁴, Peter J Sterk²⁴, Graham Roberts^{13

25}, Nazanin Z Kermani²³, Raffaele A Incalzi²⁶, Renaud Louis²⁷, Lars I Andersson^{28

29}, Scott S Wagers³⁰, Sven-Erik Dahlén^{28

29

31}, Kian Fan Chung^{23

32}, Ian M Adcock²³, Martijn A Spruit^{4

5

6}; U-BIOPRED Study Group

Affiliations

¹ Department of Research and Development, Hornerheide 1, 6085 NM, Ciro, Horn, The Netherlands. [email protected].
² NUTRIM School of Nutrition and Translational Research in Metabolism, Faculty of Health, Medicine and Life Sciences, Maastricht University, Maastricht, The Netherlands. [email protected].
³ Department of Respiratory Medicine, Maastricht University Medical Centre (MUMC+), Maastricht, The Netherlands. [email protected].
⁴ Department of Research and Development, Hornerheide 1, 6085 NM, Ciro, Horn, The Netherlands.
⁵ NUTRIM School of Nutrition and Translational Research in Metabolism, Faculty of Health, Medicine and Life Sciences, Maastricht University, Maastricht, The Netherlands.
⁶ Department of Respiratory Medicine, Maastricht University Medical Centre (MUMC+), Maastricht, The Netherlands.
⁷ Department of Pulmonary Diseases, Radboud Institute for Health Sciences, Radboud University Medical Centre, Nijmegen, The Netherlands.
⁸ Department of Clinical Science, University of Bergen, Bergen, Norway.
⁹ Department of Biomedical and Biotechnological Sciences, University of Catania, Catania, Italy.
¹⁰ Lung/Allergy Clinic, Karolinska University Hospital Huddinge, Stockholm, Sweden.
¹¹ Division of Infection, Immunity and Respiratory Medicine, School of Biological Sciences, Faculty of Biology, Medicine and Health, NIHR Biomedical Research Centre, University of Manchester and Manchester Academic Health Science Centre, Manchester University Hospitals NHS Foundation Trust, Manchester, UK.
¹² Department of Pulmonary Medicine, University Hospital and University of Bern, Bern, Switzerland.
¹³ NIHR Southampton Biomedical Research Centre, Faculty of Medicine, University of Southampton, Southampton, UK.
¹⁴ Department of Pulmonology, Semmelweis University, Budapest, Hungary.
¹⁵ National Koranyi Institute for Pulmonology, Budapest, Hungary.
¹⁶ Fraunhofer Institute for Toxicology and Experimental Medicine Hannover, Hannover, Germany.
¹⁷ Department of Public Health and Clinical Medicine, Umeå University, Umeå, Sweden.
¹⁸ Paediatric Respiratory Medicine, Children's University Hospital of Bern, University of Bern, Bern, Switzerland.
¹⁹ Division of Paediatric Pulmonology and Allergology, Department of Paediatrics and Adolescent Medicine, Medical University of Graz, Graz, Austria.
²⁰ Department of Medicine, Jagiellonian University Medical College, Krakow, Poland.
²¹ Respiratory Research Unit, University of Nottingham, Nottingham, UK.
²² Catholic University of the Sacred Heart, Rome, Italy.
²³ National Heart and Lung Institute, Imperial College, London, UK.
²⁴ Amsterdam University Medical Center, University of Amsterdam, Amsterdam, The Netherlands.
²⁵ The David Hide Asthma and Allergy Research Centre, St Mary's Hospital, Newport, UK.
²⁶ Università Campus Bio-Medico IT, Rome, Italy.
²⁷ Department of Respiratory Medicine, GIGA I 3, CHU Sart-TilmanB35, University of Liege, Liege, Belgium.
²⁸ Institute of Environmental Medicine, Karolinska Institutet, Stockholm, Sweden.
²⁹ Centre for Allergy Research, Karolinska Institutet, Stockholm, Sweden.
³⁰ BioSci Consulting, Maasmechelen, Belgium.
³¹ Department of Respiratory Medicine and Allergy, Karolinska University Hospital, Stockholm, Sweden.
³² Respiratory Department, Royal Brompton & Harefield Hospital, London, UK.

Abstract

Rationale: Knowledge about the clinical importance of patient-reported outcome measures (PROMs) in severe asthma is limited.

Objectives: To assess whether and to what extent asthma exacerbations affect changes in PROMS over time and asthma-specific PROMs can predict exacerbations in adult patients with severe asthma in usual care.

Methods: Data of 421 patients with severe asthma (62% female; mean age 51.9 ± 13.4 years; mean FEV₁ 67.5 ± 21.3%pred) from the U-BIOPRED cohort were analyzed. The included PROMs were: Asthma Control Questionnaire (ACQ5); Asthma Quality of Life Questionnaire (AQLQ); Hospital Anxiety and Depression scale (HADS); Epworth Sleepiness Scale (ESS); Medication Adherence Report Scale (MARS); Sino-Nasal Outcomes Test (SNOT20). Participants were assessed at baseline and after 12-18 months of usual care.

Results: PROMs showed very weak to weak correlations with clinical characteristics such as age, body mass index, FEV₁, FeNO and eosinophilic cell count. Patients presenting no exacerbations during follow-up showed a statistically significant improvement in all PROMs (except for MARS), whereas individuals experiencing > 2 exacerbations showed a deterioration. Baseline ACQ5 was a predictor of exacerbations with an AUC of 0.590 (95%CI 0.514-0.666).

Conclusions: The association of PROMs with clinical measures was poor in severe asthmatics. Moreover, PROMs were prone to changes in usual care, with exacerbations playing a key role. PROMs need to be systematically evaluated in severe asthma to improve clinical care based on specific patient's needs.

Keywords: Health status; Obstructive pulmonary diseases; Patient outcome assessment.

MeSH terms

Adult
Asthma* / drug therapy
Asthma* / physiopathology
Asthma* / psychology
Asthma* / therapy
Clinical Relevance
Female
Humans
Male
Middle Aged
Patient Reported Outcome Measures*
Quality of Life*
Severity of Illness Index
Surveys and Questionnaires

Grants and funding

5.1.18.232/Lung Foundation Netherlands