Patient-reported impact of symptoms in myotonic dystrophy type 1 (PRISM-1).

ABSTRACT:

Objective

To determine the most critical symptoms in a national myotonic dystrophy type 1 (DM1) population and to identify the modifying factors that have the greatest effect on the severity of these symptoms.

Methods

We performed a cross-sectional study of 278 adult patients with DM1 from the national registry of patients with DM1 between April and August 2010. We assessed the prevalence and relative significance of 221 critical DM1 symptoms and 14 disease themes. These symptoms and themes were chosen for evaluation based on prior interviews with patients with DM1. Responses were categorized by age, CTG repeat length, gender, and duration of symptoms.

Results

Participants with DM1 provided symptom rating survey responses to address the relative frequency and importan

SUBMITTER: Heatwole C 

PROVIDER: S-EPMC3400095 | biostudies-literature | 2012 Jul

REPOSITORIES: biostudies-literature