OBJECTIVES : Duchenne muscular dystrophy (DMD) is a severe progressive genetic disease causing early loss of ambulation and mortality. To accurately assess the costs and benefits of new therapies, utility values for DMD-related health states are required. As DMD progression begins in early childhood, the health-related quality-of-life (HRQoL) of DMD patients, caregivers and families can be affected. The objective was to synthesize published evidence on utilities for health states capturing the HRQoL impact and caregiver burden of DMD.

METHODS : A systematic review was performed using MEDLINE and Embase to identify studies reporting utilities for DMD patients or caregivers. Data were extracted on study and patient characteristics, health states considered, approach to measuring utility and reported utility values. Quality appraisal of included studies was performed using a framework described by Papaionannou et al (2013).

RESULTS : From 888 abstracts, eight publications were identified. DMD utility estimates were identified for a limited number of health states defined by ambulation, ventilation, and age. Estimates of utility values ranged from 0.75 (HUI-3) for early ambulatory DMD to 0.05 (HUI-3) for day-and-night ventilation. Caregiver utilities ranged from 0.87 (for caregivers of adult DMD patients with DMD; measured by the EQ-5D-3L) to 0.71 (for caregivers of predominantly childhood DMD patients; measured by the EQ-5D-3L). Both patient and caregiver utilities tended to be lower with more severe DMD. Variability in utilities was also observed based on the choice of measure, respondent type, and inclusion criteria. No utilities for other DMD-related health states defined by, for example, upper limb function or developmental disability, were identified.

CONCLUSIONS : Published utility values document substantial HRQoL impacts in DMD, particularly with disease progression. Future initiatives to describe the utility of a wider range of health states are necessary to accurately model the cost-effectiveness of therapies with the potential to alter DMD progression.