A Comparison of Caregiver and Patient Preferences for Treating Duchenne Muscular Dystrophy.

ABSTRACT:

Background and objectives

Caregivers routinely inform medical and regulatory decision making in rare pediatric diseases. While differences in treatment preferences across caregivers and patients have been observed for Duchenne muscular dystrophy, this evidence was limited by small samples of patients and results were confounded by patient age and disease progression. We tested caregiver and patient preference concordance for treating Duchenne.

Methods

Preferences and demographic/clinical information from 115 caregivers and 107 patients were collected in an international study (response = 80%) using a previously developed discrete-choice experiment consisting of 12 experimentally controlled choice tasks. Each task presented two profiles that varied across four attributes: dis

SUBMITTER: Crossnohere NL 

PROVIDER: S-EPMC8894129 | biostudies-literature | 2022 Sep

REPOSITORIES: biostudies-literature