Evidence mapPaperPMID 41961170Full record

ArticleJournal of patient-reported outcomes2026

Estimating health-state utility values for family-caregivers of patients with Duchenne muscular dystrophy using time trade-off valuation.

Oktawia Borecka, Samuel Llewellyn, Ione Woollacott, Lucy Richardson, Alasdair Fellows, Jack Lawrence, Catherine Bottomley, Alice M Biggane

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Article in Journal of patient-reported outcomes, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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1 · What the graph read from it

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2 · The registry

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3 · Its place in the literature

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4 · The record

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5 · Who and what money

Authors and funding

8 authors.

Oktawia BoreckaVitaccess, 2nd Floor Nucleus House, 2 Lower Mortlake Road, Richmond, London, TW9 2JA, UK.
Samuel LlewellynVitaccess, 2nd Floor Nucleus House, 2 Lower Mortlake Road, Richmond, London, TW9 2JA, UK. samuel.llewellyn@vitaccess.com.ORCID http://orcid.org/0000-0001-5216-4216
Ione WoollacottPfizer Ltd., Tadworth, UK.
Lucy RichardsonPfizer Ltd., Tadworth, UK.
Alasdair FellowsVitaccess, 2nd Floor Nucleus House, 2 Lower Mortlake Road, Richmond, London, TW9 2JA, UK.
Jack LawrenceVitaccess, 2nd Floor Nucleus House, 2 Lower Mortlake Road, Richmond, London, TW9 2JA, UK.
Catherine BottomleyVitaccess, 2nd Floor Nucleus House, 2 Lower Mortlake Road, Richmond, London, TW9 2JA, UK.
Alice M BigganePfizer Ltd., Tadworth, UK.

Funding

Pfizer UK N/A
6 · The paper itself

Abstract

objectivesDuchenne muscular dystrophy (DMD) is a rare, progressive neuromuscular disease. Long-term care is primarily provided by unpaid family-caregivers. This time trade-off (TTO) study aimed to generate family-caregiver utility values associated with different stages of DMD.

methodsEight vignettes were developed to reflect the experience of caring for patients with DMD across health states (HS) defined in the Project HERCULES model. Vignettes were informed by literature and input from family-caregivers and healthcare professionals. TTO interviews were conducted online with members of the UK general public. Participants reviewed all vignettes and completed HS ranking, visual analog scale (VAS) ratings, and TTO valuation tasks.

results200 participants (mean age 44.0 years, 51% female) completed interviews. Mean utility scores were highest for HS1 (early ambulatory: 0.717) and lowest for HS8 (no hand-to-mouth function, full-time ventilation: 0.477). VAS scores showed a similar pattern, with HS1 scoring 65.49 and HS8 scoring 31.665.

conclusionsThis study highlights the increasing burden on family-caregivers as DMD progresses, with declining health-related quality of life across HS. The resulting utility values support inclusion of family-caregiver outcomes in future DMD cost-effectiveness models.

Indexed as

CaregiversFamilyHealth StatusMuscular Dystrophy, DuchenneQuality of LifeAdultCost of IllnessFemaleHumansMaleMiddle AgedUnited Kingdom

Identifiers

PMID41961170
PMCPMC13076743

What Socratic holds

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.