The Limb Girdle Muscular Dystrophy Health Index (LGMD-HI)

• Published in: Neuromuscular disorders : NMD 2024-06, Vol.39, p.48-53
• Main Authors: Stouffer, Joy A., Bates, Kameron, Thacker, Leroy R., Heatwole, Chad, Johnson, Nicholas E.
• Format: Article
• Language: English
• Subjects: Adult; Aged; Female; Health index; Humans; Limb girdle muscular dystrophy; Male; Middle Aged; Muscular dystrophies; Muscular Dystrophies, Limb-Girdle - diagnosis; Muscular Dystrophies, Limb-Girdle - genetics; Patient-reported outcome measure; Registries; Reproducibility of Results; Severity of Illness Index; Symptom burden; Young Adult
• ISSN: 0960-8966; 1873-2364; 1873-2364
• DOI: 10.1016/j.nmd.2024.04.008

•Patient reported outcome measure for all limb girdle muscular dystrophies (LGMDs).•This outcome measure is specific to LGMD and measures 15 areas of disease burden.•This measure is designed for use in clinical trials to capture patient experience. Limb girdle muscular dystrophy (LGMD) is a debilitating disease and the fourth most common muscular dystrophy. This study describes the development of the LGMD-Health Index (LGMD-HI). Participants were aged >18 years and recruited from three LGMD registries and GRASP-LGMD consortium. The initial instrument, comprised of 16 thematic subscales and 161 items, underwent expert review resulting in item removal as well as confirmatory factor analysis followed by inter-rater reliability and internal consistency of the subscales. Following expert review, one subscale and 59 items were eliminated. Inter-rater reliability was assessed and five items were removed due to Cohen's kappa