Forced vital capacity and cross-domain late-onset Pompe disease outcomes: an individual patient-level data meta-analysis.

ABSTRACT:

Background

Late-onset Pompe disease (LOPD) is a rare, metabolic disease primarily affecting the musculoskeletal and respiratory systems. Forced vital capacity (FVC) is commonly used to measure pulmonary function; however, associations between FVC and other LOPD outcomes remain unclear.

Methods

A systematic literature review was conducted on November 2015, updated September 2016 and supplemented with clinical trial data from the sponsor. Outcomes included: 6-min walk test distance (6MWT), FVC, maximal inspiratory/expiratory pressure (MIP/MEP), Medical Research Council-skeletal muscle strength score (MRC), 36-item short-form survey-physical component score (SF-36), Rotterdam Handicap Scale (RHS), Fatigue Severity Scale (FSS) and survival. Individual patient data meta-analysis

SUBMITTER: Berger KI 

PROVIDER: S-EPMC6687674 | biostudies-literature | 2019 Sep

REPOSITORIES: biostudies-literature