OBJECTIVES: The DMD-QoL is a novel disease-specific preference-based measure (PBM) used to estimate patient utility. This study examined how DMD patient utility values estimated using the DMD-QoL differ to utilities measured via the generic PBM, the Health Utilities Index (HUI).

METHODS: Sixty-three individuals with DMD were recruited through a US-based advocacy organization. Patients 12-40 years of age self-reported current health status and completed the DMD-QoL and HUI. DMD-QoL attributes include mobility, hand function, difficulty breathing, pain, tiredness, worry, participation, and feeling good about oneself. The HUI categorizes health status according to two systems; the HUI2 is presented here, and considers sensation, mobility, emotion, cognition, self-care, pain, and fertility (optional). Median (interquartile, IQR) DMD-QoL and HUI utility values were estimated for DMD-relevant progressive health states.

RESULTS: Mean (standard deviation, SD) patient age was 14.9 (6.9) years and 70% were non-ambulatory. Median (IQR) DMD-QoL utility values ranged from 0.77 (0.73-0.86) for ambulatory individuals with preserved upper limb function (n=10), 0.69 (0.58-0.82) for non-ambulatory with mildly impaired upper limb function (n=16), and 0.20 (-0.02-0.41) for non-ambulatory with loss of upper limb function, symptomatic cardiomyopathy, and nighttime/daytime ventilation (n=2). Median (IQR) HUI2 utilities for these health states were 0.96 (0.86-0.99); 0.51 (0.44-0.55); and 0.32 (0.23-0.42). While DMD-QoL mobility and hand function scores are lower with disease progression, this drop in scores was not observed until loss of upper limb function. Mobility and self-care were the HUI2 attributes reflecting the greatest impact, with a worsening of levels observed earlier in DMD progression compared to on the DMD-QoL.

CONCLUSIONS: Compared to HUI2, DMD-QoL utility values remained relatively stable across health states until loss of upper limb function precipitated lower values. Mobility was a driver of utility in both instruments, yet differences in scores may reflect the DMD-QoL’s broader conceptualization of mobility. Future research should compare findings to other commonly-used PBMs.