Decision-Making And Selection Bias in Four Observational Studies on Duchenne and Becker Muscular Dystrophy.

ABSTRACT:

Background

Natural history data are essential for trial design in Duchenne (DMD) and Becker muscular dystrophy (BMD), but recruitment for observational studies can be challenging.

Objective

We reviewed reasons why patients or caregivers declined participation, and compared characteristics of participants and non-participants to assess possible selection bias in four observational studies, three on DMD and one on BMD.

Methods

Three pediatric DMD studies focused on cross-sectional cognitive function and brain MRI (DMDbrain, n = 35 and DMDperfusion, n = 12), and on longitudinal upper extremity function and muscle MRI (DMDarm, n = 22). One adult BMD study assessed longitudinal functioning (n = 36). Considerations for non-participation were retrospectively reviewed from sc

SUBMITTER: Naarding KJ 

PROVIDER: S-EPMC7902964 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature