Development and Validation of a New Scoring System to Predict Survival in Patients With Myotonic Dystrophy Type 1.

ABSTRACT:

Importance

Life expectancy is greatly shortened in patients presenting with myotonic dystrophy type 1 (DM1), the most common neuromuscular disease. A reliable prediction of survival in patients with DM1 is critically important to plan personalized health supervision.

Objective

To develop and validate a prognostic score to predict 10-year survival in patients with DM1.

Design, setting, and participants

In this longitudinal cohort study, between January 2000 and November 2014, we enrolled 1296 adults referred to 4 tertiary neuromuscular centers in France for management of genetically proven DM1, including 1066 patients in the derivation cohort and 230 in the validation cohort. Data were analyzed from December 2016 to March 2017.

Main outcomes and measures

Factors

SUBMITTER: Wahbi K 

PROVIDER: S-EPMC5885178 | biostudies-literature | 2018 May

REPOSITORIES: biostudies-literature