OBJECTIVES: Caregivers commonly report utility on behalf of patients with DMD. As the alignment between these perspectives has not been described, the objective was to compare patient- and caregiver-derived visual analogue scale (VAS) and utility values for DMD health states.

METHODS: Thirty-one patient-caregiver dyads were recruited through a US-based patient organization to complete the EQ-5D and Health Utilities Index (HUI). Patients were classified into one of nine DMD-related health states according to level of lower/upper limb function, daytime/nighttime ventilation, and cardiomyopathy. Median (IQR) EQ-5D, VAS and HUI2 values were stratified by health state and respondent type (patient vs caregiver). Correlations were calculated for ‘ambulatory/transitional’ dyads and ‘non-ambulatory’ dyads separately. Mixed-effects regression was used to model the relationship between caregiver and patient utility values, accounting for ambulatory status and within-dyad correlation.

RESULTS: Mean (SD) patient age was 14 (5.9) years and 21 (67%) were non-ambulatory. Correlations for ambulatory/transitional dyads were r=0.97 (EQ-5D), r=0.88 (HUI2) and r=0.90 (VAS); and for non-ambulatory dyads, r=0.66 (EQ-5D), r=0.70 (HUI2), and r=0.60 (VAS). Across health states, caregivers reported lower EQ-5D (p<0.001) and VAS (p=0.04) values than patients; HUI2 utilities did not differ by respondent type. As an example, for non-ambulatory patients with mildly-impaired upper limb function, without nighttime/daytime ventilation or symptomatic cardiomyopathy, median (IQR) estimates (n=7) were: EQ-5D: 0.30 (0.24-0.35; patient-report) vs 0.15 (0.07-0.18; caregiver-report); HUI2: 0.51 (0.44-0.52; patient-report) vs. 0.47 (0.47-0.55; caregiver-report); and VAS: 84 (74-96; patient-report) vs. 80 (75-87; caregiver-report).

CONCLUSIONS: Utility and VAS values from caregiver-patient dyads were highly correlated, particularly for ‘ambulatory/transitional’ dyads. Across health states, patient-reported values were higher than caregiver-reported values for the EQ-5D and VAS; diverging more as patient status worsened. HUI2 utilities did not differ significantly between dyads. Though numeric differences should be noted, high correlations support reliance on caregivers when patient-reported data are unavailable and can inform utility instrument selection.