The Association between Patient- and Caregiver-Reported Utility Values in Duchenne Muscular Dystrophy (DMD)

Author(s)

Szabo S1, Filipovic Audhya I2, Bever A1, O’Sullivan F1, Malone DC3, Feeny D4, Neumann P5, Iannaccone ST6, Jayasinghe P1, Gooch KL2
1Broadstreet Health Economics & Outcomes Research, Vancouver, BC, Canada, 2Sarepta Therapeutics, Cambridge, MA, USA, 3The University of Utah, Salt Lake City, UT, USA, 4McMaster University, Hamilton, ON, Canada, 5Tufts University, Boston, MA, USA, 6University of Texas Southwestern Medical Center, Dallas, TX, USA

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.

Conference/Value in Health Info

2023-05, ISPOR 2023, Boston, MA, USA

Value in Health, Volume 26, Issue 6, S2 (June 2023)

Acceptance Code

P31

Topic

Methodological & Statistical Research, Patient-Centered Research

Topic Subcategory

Health State Utilities, Instrument Development, Validation, & Translation, PRO & Related Methods

Disease

no-additional-disease-conditions-specialized-treatment-areas

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