SynthesisQuality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation2020
Characterizing health state utilities associated with Duchenne muscular dystrophy: a systematic review.
Synthesis in Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers, 3 of them syntheses that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
21 citing papers in PubMed, 3 syntheses or guidelines pooled it.
- Health-related quality of life and health state utility value in idiopathic pulmonary fibrosis: a systematic review and meta-analysis.Health and quality of life outcomes · 2025Pooled it
- Quality appraisal for systematic literature reviews of health state utility values: a descriptive analysis.BMC medical research methodology · 2022Pooled it
- Health-related quality of life in adult patients with asthma according to asthma control and severity: A systematic review and meta-analysis.Frontiers in pharmacology · 2022Pooled it
- Exploring the relationship between North Star Ambulatory Assessment and Health Utilities Index scores in Duchenne muscular dystrophy.Health and quality of life outcomes · 2023Trial
- Factors associated with the health-related quality of life among people with Duchenne muscular dystrophy: a study using the Health Utilities Index (HUI).Health and quality of life outcomes · 2022Trial
- Estimating Utility Values Using the DMD-QoL: A Disease-Specific Preference-Based Measure for Duchenne Muscular Dystrophy (DMD).Advances in therapy · 2026Article
- Caregiver burden in Duchenne muscular dystrophy in Europe, Japan, and the United States - a real-world study.BMC neurology · 2026Article
- Systematic Literature Reviews of Health-State Utility Values, Costs, and Healthcare Resource Use in Duchenne Muscular Dystrophy.Journal of health economics and outcomes research · 2026Article
- The Paradox of Life Extension with Traditional Cost-Effectiveness Analysis: A Case Study with Duchenne Muscular Dystrophy.Journal of health economics and outcomes research · 2026Article
- Remaining Burden of Spinal Muscular Atrophy Among Treated Patients: A Survey of Patients and Caregivers.Annals of clinical and translational neurology · 2025Article
- Psychometric analysis of the patient-reported outcomes measurement information system parent proxy physical function-upper extremity item bank for children with Duchenne muscular dystrophy.Frontiers in neurology · 2025Article
- Cataloging health state utility estimates for Duchenne muscular dystrophy and related conditions.Health and quality of life outcomes · 2024Article
- Quality Appraisal in Systematic Literature Reviews of Studies Eliciting Health State Utility Values: Conceptual Considerations.PharmacoEconomics · 2024Article
- "You Take This Day by Day, Come What May": A Qualitative Study of the Psychosocial Impacts of Living with Duchenne Muscular Dystrophy.Advances in therapy · 2024Article
- Estimating health state utilities in Duchenne muscular dystrophy using the health utilities index and EQ-5D-5L.Journal of patient-reported outcomes · 2023Article
- Real-world evidence of eteplirsen treatment effects in patients with Duchenne muscular dystrophy in the USA.Journal of comparative effectiveness research · 2023Article
- A comprehensive qualitative framework for health-related quality of life in Duchenne muscular dystrophy.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2023Article
- Pain characteristics among individuals with Duchenne muscular dystrophy according to their clinical stage.BMC musculoskeletal disorders · 2022Article
- Quality of life and informal care burden associated with duchenne muscular dystrophy in Portugal: the COIDUCH study.Health and quality of life outcomes · 2022Article
- A qualitative study on the impact of caring for an ambulatory individual with nonsense mutation Duchenne muscular dystrophy.Journal of patient-reported outcomes · 2021Article
Corrections and comments
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Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundPreferences for health states for Duchenne muscular dystrophy (DMD) are necessary to assess costs and benefits of novel therapies. Because DMD progression begins in childhood, the impact of DMD on health-related quality-of-life (HRQoL) affects preferences of both DMD patients and their families. The objective of this review was to synthesize published evidence for health state utility from the DMD patient and caregiver perspectives.
methodsA systematic review was performed using MEDLINE and Embase, according to best practices. Data were extracted from studies reporting DMD patient or caregiver utilities; these included study and patient characteristics, health states considered, and utility estimates. Quality appraisal of studies was performed.
resultsFrom 888 abstracts, eight publications describing five studies were identified. DMD utility estimates were from preference-based measures presented stratified by ambulatory status, ventilation, and age. Patient (or patient-proxy) utility estimates ranged from 0.75 (early ambulatory DMD) to 0.05 (day-and-night ventilation). Caregiver utilities ranged from 0.87 (for caregivers of adults with DMD) to 0.71 (for caregivers of predominantly childhood patients). Both patient and caregiver utilities trended lower with higher disease severity. Variability in utilities was observed based on instrument, respondent type, and country. Utility estimates for health states within non-ambulatory DMD are under reported; nor were utilities for DMD-related health states such as scoliosis or preserved upper limb function identified.
conclusionPublished health state utilities document the substantial HRQoL impacts of DMD, particularly with disease progression. Additional research in patient utilities for additional health states, particularly in non-ambulatory DMD patients, is warranted.
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