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.
Highlights
Duchenne muscular dystrophy (DMD) is a rare X-linked severe progressive myopathy caused by mutations in the gene for dystrophin, with an estimated birth prevalence of approximately 1:5000 males [1, 2]
A systematic literature review (SLR) was performed to identify and critically appraise published evidence on utility values for health states describing the health-related quality-of-life (HRQoL) impact of DMD, for both patients and caregivers
Of the remaining 46 articles, 38 were excluded on full-text review; most (n = 33; 87%) because they did not present utility values, with the remainder being excluded for study design (n = 1), being a duplicate (n = 1), or other reasons
Summary
Duchenne muscular dystrophy (DMD) is a rare X-linked severe progressive myopathy caused by mutations in the gene for dystrophin, with an estimated birth prevalence of approximately 1:5000 males [1, 2]. The indirect multiattribute health status classification systems assign respondents to a set of non-disease-specific health states based on an individual’s combination of responses provided to a series of questions that measure different attributes of health. Such indirect preference-weighted measures have been widely recommended for use in economic evaluations in part because they incorporate the societal perspective in that the scoring functions for these measures are based on preference scores obtained from representative samples of the general population. 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. Additional research in patient utilities for additional health states, in non-ambulatory DMD patients, is warranted
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