Identifying responders to elamipretide in Barth syndrome: Hierarchical clustering for time series data.

ABSTRACT:

Background

Barth syndrome (BTHS) is a rare genetic disease that is characterized by cardiomyopathy, skeletal myopathy, neutropenia, and growth abnormalities and often leads to death in childhood. Recently, elamipretide has been tested as a potential first disease-modifying drug. This study aimed to identify patients with BTHS who may respond to elamipretide, based on continuous physiological measurements acquired through wearable devices.

Results

Data from a randomized, double-blind, placebo-controlled crossover trial of 12 patients with BTHS were used, including physiological time series data measured using a wearable device (heart rate, respiratory rate, activity, and posture) and functional scores. The latter included the 6-minute walk test (6MWT), Patient-Reported Outcom

SUBMITTER: Van den Eynde J 

PROVIDER: S-EPMC10088720 | biostudies-literature | 2023 Apr

REPOSITORIES: biostudies-literature