title: The Performance of the Upper Limb scores correlate with pulmonary function test measures and Egen Klassifikation scores in Duchenne muscular dystrophy.
abstract: The Performance of the Upper Limb scale was developed as an outcome measure specifically for ambulant and non-ambulant patients with Duchenne muscular dystrophy and is implemented in clinical trials needing longitudinal data. The aim of this study is to determine whether this novel tool correlates with functional ability using pulmonary function test, cardiac function test and Egen Klassifikation scale scores as clinical measures. In this cross-sectional study, 43 non-ambulatory Duchenne males from ages 10 to 30 years and on long-term glucocorticoid treatment were enrolled. Cardiac and pulmonary function test results were analyzed to assess cardiopulmonary function, and Egen Klassifikation scores were analyzed to assess functional ability. The Performance of the Upper Limb scores correlated with pulmonary function measures and had inverse correlation with Egen Klassifikation scores. There was no correlation with left ventricular ejection fraction and left ventricular dysfunction. Body mass index and decreased joint range of motion affected total Performance of the Upper Limb scores and should be considered in clinical trial designs.
authors.affiliation: Division of Pediatric Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
authors.forename: Ha Neul
authors.initials: HN
authors.lastname: Lee
authors.affiliation: Division of Pulmonary Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
authors.forename: Hemant
authors.initials: H
authors.lastname: Sawnani
authors.affiliation: Division of Pediatric Neurology and Biostatistics & Epidemiology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
authors.forename: Paul S
authors.initials: PS
authors.lastname: Horn
authors.affiliation: Division of Pediatric Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
authors.forename: Irina
authors.initials: I
authors.lastname: Rybalsky
authors.affiliation: Division of Pediatric Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
authors.forename: Lani
authors.initials: L
authors.lastname: Relucio
authors.affiliation: Division of Pediatric Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA. Electronic address: brenda.wong@cchmc.org.
authors.forename: Brenda L
authors.initials: BL
authors.lastname: Wong
mesh_terms: D000293:Adolescent; D000328:Adult; D015992:Body Mass Index; D002648:Child; D003430:Cross-Sectional Studies; D005938:Glucocorticoids / Q000627:therapeutic use; D006801:Humans; D008168:Lung / Q000503:physiopathology; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000175:diagnosis* / Q000188:drug therapy / Q000503:physiopathology*; D009465:Neuromuscular Agents / Q000627:therapeutic use; D016059:Range of Motion, Articular; D012129:Respiratory Function Tests; D012720:Severity of Illness Index; D034941:Upper Extremity / Q000503:physiopathology*; D055815:Young Adult
chemical_list: D005938:Glucocorticoids; D009465:Neuromuscular Agents
keywords: Duchenne muscular dystrophy; Egen Klassifikation; Long term glucocorticoid treatment; Non-ambulatory; Pulmonary function test; The Performance of the Upper Limb