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- Volume 83,Issue Suppl 1
- AB1239 MEASURING THE HUMANISTIC BURDEN OF EGPA: A SYSTEMATIC REVIEW
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Vasculitis, small and medium size vessels
AB1239 MEASURING THE HUMANISTIC BURDEN OF EGPA: A SYSTEMATIC REVIEW
- J. Rowell1,
- S. Lucas2,
- P. Dolin3
- 1AstraZeneca, Health Economics and Payer Evidence, Cambridge, United Kingdom
- 2Health Economics and Outcomes Research Ltd, Cardiff, United Kingdom
- 3AstraZeneca, BioPharmaceuticals Medical Evidence, Cambridge, United Kingdom
Abstract
Background: Eosinophilic granulomatosis with polyangiitis (EGPA) is a rare autoimmune disease characterised by eosinophil-rich granulomatous inflammation and vasculitis which can lead to organ damage. EGPA has a significant impact on health-related quality of life (HRQoL), due to substantial fatigue and limitations to daily activities associated with symptom burden. Further, people with EGPA may experience disease manifestations affecting multiple organ systems, contributing to the overall reduction in HRQoL that an individual may experience.
Objectives: To conduct a systematic literature review (SLR) to understand the humanistic burden of EGPA in patients and caregivers.
Methods: Searches were performed in Ovid MEDLINE, Ovid Embase, the Cochrane Library and the International Network Agencies for Health Technology Assessments from January 2013 to July 2023. Relevant conferences (2020–2023), SLR bibliographies and the US Food and Drug Administration website were hand searched. No language restrictions were applied. Risk of bias assessments were conducted on the full-text publication for each included study using validated tools. This review has been registered with PROSPERO (registration number: CRD42023432751).[1]
Results: Of 783 records screened, 25 publications were included that reported 18 unique studies. All studies were observational, most commonly retrospective cohort studies (six studies). The number of patients enrolled ranged from 5 to 276. The mean age of patients ranged from 42 to 62 years and 28.0–90.9% were male. All studies identified reported HRQoL outcomes from the patient perspective. There were no studies identified that reported utility or disutility outcomes, or that reported HRQoL from the caregiver perspective.
The most frequently reported generic HRQoL instrument was Short Form 36 (SF-36; four studies); the most commonly reported asthma-specific instrument was the Asthma Control Questionnaire (ACQ; six studies) and the most reported symptom-specific instrument was the Sino-nasal Outcomes Test (SNOT-22; six studies).
Compared with the general population, patients with active disease experienced reduced HRQoL as measured by both generic (SF-36) and specific (PROMIS and SNOT-22) instruments. Additionally, 34% of patients with EGPA reported an impairment in daily activities assessed by the Work Productivity and Activity Impairment General Health (WPAI-GH) questionnaire.
Eight studies assessed an intervention, with asthma-specific instruments commonly used to assess the impact of treatment on symptoms and associated HRQoL. Across the studies reporting validated instruments (Asthma Quality of Life Questionnaire [AQLQ], Asthma Control Test [ACT] and ACQ), asthma control improved following treatment with the eosinophil-depleting therapies (benralizumab, mepolizumab and reslizumab).
Conclusion: This review confirms that people with active EGPA experience reduced HRQoL compared with the general population. No studies were identified that used EGPA-specific patient-reported outcome (PRO) instruments. In the absence of PRO measures designed for patients with EGPA, a variety of generic and symptom-specific instruments were used to quantify the humanistic burden of EGPA, with the impact of asthma the most frequently measured. No studies were identified that reported on utility/disutility values or caregiver burden. These are potential avenues for further research to determine the full humanistic impact of EGPA on patients and their families.
REFERENCES: [1] National Institute for Health and Care Research. Health-related quality of life of adult patients with eosinophilic granulomatosis with polyangiitis (EGPA) (PROSPERO registration number: CRD42023432751). 2023. Available from: https://www.crd.york.ac.uk/prospero/display_record.php?RecordID=432751 [accessed 15 November 2023].
Acknowledgements: The authors thank Victoria Richardsdóttir Fife and Bianca Kennedy Hall from AstraZeneca for project support, and Sukhvir Rai and Kerrie Ford of Health Economics and Outcomes Research Ltd (Cardiff, UK) for medical writing support, which was funded by AstraZeneca (Cambridge, UK) in accordance with Good Publication Practice (GPP 2022) guidelines (https://www.ismpp.org/gpp-2022).
Disclosure of Interests: Jennifer Rowell owns share options in AstraZeneca and Roche, employee of AstraZeneca, but previously employed by Roche, Amgen and QLT Phototherapeutics, Sara Lucas an employee of Health Economics and Outcomes Research Ltd. Health Economics and Outcomes Research Ltd. received funding for data analysis and medical writing support from AstraZeneca for this work, Paul Dolin contractor for AstraZeneca.
- Rare/orphan diseases
- Quality of life
- Patient Reported Outcome Measures
- Systematic review
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- Rare/orphan diseases
- Quality of life
- Patient Reported Outcome Measures
- Systematic review
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