title: Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up.
abstract: Outcomes sensitive to change over time in non-ambulatory boys/men with Duchenne muscular dystrophy (DMD) are not well-established.
  
  
  
  Subjects (n = 91; 16.8 ± 4.5 years old) were assessed at baseline and 6-month intervals for 2 years. We analyzed all subjects using an intent-to-treat model and a subset of stronger subjects with Brooke Scale score ≤4, using repeated measures.
  
  
  
  Eight patients (12-33 years old) died during the study. Sixty-six completed 12-month follow-up, and 51 completed 24-month follow-up. Those taking corticosteroids performed better at baseline, but rates of decline were similar. Forced vital capacity percent predicted (FVC% predicted) declined significantly only after 2 years. However, Brooke and Egen Klassifikation (EK) Scale scores, elbow flexion, and grip strength declined significantly over both 1 and 2 years.
  
  
  
  Brooke and EK Scale scores, elbow flexion, and grip strength were outcomes most responsive to change. FVC% predicted was responsive to change over 2 years. Corticosteroids benefited non-ambulatory DMD subjects but did not affect decline rates of measures tested here. Muscle Nerve 54: 681-689, 2016.
authors.affiliation: Department of Neurology, Washington University School of Medicine, Saint Louis, Missouri, USA. connollya@neuro.wustl.edu.|Department of Pediatrics, Washington University School of Medicine, Saint Louis, Missouri, USA. connollya@neuro.wustl.edu.
authors.forename: Anne M
authors.initials: AM
authors.lastname: Connolly
authors.affiliation: Department of Neurology, Washington University School of Medicine, Saint Louis, Missouri, USA.
authors.forename: Julaine M
authors.initials: JM
authors.lastname: Florence
authors.affiliation: Department of Neurology, Washington University School of Medicine, Saint Louis, Missouri, USA.|Department of Pediatrics, Washington University School of Medicine, Saint Louis, Missouri, USA.
authors.forename: Craig M
authors.initials: CM
authors.lastname: Zaidman
authors.affiliation: Department of Neurology, Washington University School of Medicine, Saint Louis, Missouri, USA.|Department of Pediatrics, Washington University School of Medicine, Saint Louis, Missouri, USA.
authors.forename: Paul T
authors.initials: PT
authors.lastname: Golumbek
authors.affiliation: Department of Pediatrics, Ohio State University, and the Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
authors.forename: Jerry R
authors.initials: JR
authors.lastname: Mendell
authors.affiliation: Department of Pediatrics, Ohio State University, and the Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
authors.forename: Kevin M
authors.initials: KM
authors.lastname: Flanigan
authors.affiliation: Department of Neurology, University of Minnesota, Minneapolis, Minnesota, USA.
authors.forename: Peter I
authors.initials: PI
authors.lastname: Karachunski
authors.affiliation: Department of Neurology, Stanford University, Stanford, California, USA.
authors.forename: John W
authors.initials: JW
authors.lastname: Day
authors.affiliation: Department Physical Medicine and Rehabilitation, University of California, Davis Medical Center, Sacramento, California, USA.
authors.forename: Craig M
authors.initials: CM
authors.lastname: McDonald
authors.affiliation: Department of Neurology, Harvard University, Boston Children's Hospital, Boston, Massachusetts, USA.
authors.forename: Basil T
authors.initials: BT
authors.lastname: Darras
authors.affiliation: Division of Pediatric Neurology, University of Florida College of Medicine, Gainesville, Florida, USA.
authors.forename: Peter B
authors.initials: PB
authors.lastname: Kang
authors.affiliation: Department of Neurology, Washington University School of Medicine, Saint Louis, Missouri, USA.
authors.forename: Catherine A
authors.initials: CA
authors.lastname: Siener
authors.affiliation: Department of Neurology, Washington University School of Medicine, Saint Louis, Missouri, USA.
authors.forename: Rebecca K
authors.initials: RK
authors.lastname: Gadeken
authors.affiliation: Department of Neurology, Washington University School of Medicine, Saint Louis, Missouri, USA.
authors.forename: Pallavi
authors.initials: P
authors.lastname: Anand
authors.affiliation: Department of Neurology, Washington University School of Medicine, Saint Louis, Missouri, USA.
authors.forename: Jeanine R
authors.initials: JR
authors.lastname: Schierbecker
authors.affiliation: Department of Neurology, Washington University School of Medicine, Saint Louis, Missouri, USA.
authors.forename: Elizabeth C
authors.initials: EC
authors.lastname: Malkus
authors.affiliation: Department of Pediatrics, Ohio State University, and the Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
authors.forename: Linda P
authors.initials: LP
authors.lastname: Lowes
authors.affiliation: Department of Pediatrics, Ohio State University, and the Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
authors.forename: Lindsay N
authors.initials: LN
authors.lastname: Alfano
authors.affiliation: Department Physical Medicine and Rehabilitation, University of California, Davis Medical Center, Sacramento, California, USA.
authors.forename: Linda
authors.initials: L
authors.lastname: Johnson
authors.affiliation: Department Physical Medicine and Rehabilitation, University of California, Davis Medical Center, Sacramento, California, USA.
authors.forename: Alina
authors.initials: A
authors.lastname: Nicorici
authors.affiliation: Department of Neurology, University of Minnesota, Minneapolis, Minnesota, USA.
authors.forename: Jason M
authors.initials: JM
authors.lastname: Kelecic
authors.affiliation: Department of Neurology, Harvard University, Boston Children's Hospital, Boston, Massachusetts, USA.
authors.forename: Janet
authors.initials: J
authors.lastname: Quigley
authors.affiliation: Department of Neurology, Harvard University, Boston Children's Hospital, Boston, Massachusetts, USA.
authors.forename: Amy E
authors.initials: AE
authors.lastname: Pasternak
authors.affiliation: Division of Biostatistics, Washington University School of Medicine, Saint Louis, Missouri, USA.
authors.forename: J Philip
authors.initials: JP
authors.lastname: Miller
mesh_terms: D000293:Adolescent; D000305:Adrenal Cortex Hormones / Q000627:therapeutic use; D000328:Adult; D002648:Child; D005500:Follow-Up Studies; D018737:Hand Strength / Q000502:physiology; D006801:Humans; D008297:Male; D051346:Mobility Limitation; D020388:Muscular Dystrophy, Duchenne / Q000175:diagnosis* / Q000188:drug therapy / Q000503:physiopathology*; D010358:Patient Participation / Q000379:methods*; D016059:Range of Motion, Articular / Q000502:physiology; D014797:Vital Capacity / Q000502:physiology; D055815:Young Adult
chemical_list: D000305:Adrenal Cortex Hormones
keywords: Brooke Scale; Duchenne muscular dystrophy; Egen Klassifikation Scale; clinical trial; force vital capacity; non-ambulatory; outcomes