Natural history of Lafora disease: a prognostic systematic review and individual participant data meta-analysis.

ABSTRACT:

Background

Lafora disease (LD) is a rare fatal autosomal recessive form of progressive myoclonus epilepsy. It affects previously healthy children or adolescents, causing pharmacoresistant epilepsy, myoclonus and severe psychomotor deterioration. This work aims to describe the clinical course of LD and identify predictors of outcome by means of a prognostic systematic review with individual participant data meta-analysis.

Methods

A search was conducted on MEDLINE and Embase with no restrictions on publication date. Only studies reporting genetically confirmed LD cases were included. Kaplan-Meier estimate was used to assess probability of death and loss of autonomy. Univariable and multivariable Cox regression models with mixed effects (clustered survival data) were performed

SUBMITTER: Pondrelli F 

PROVIDER: S-EPMC8365996 | biostudies-literature | 2021 Aug

REPOSITORIES: biostudies-literature