title: Outcome reliability in non-ambulatory boys/men with Duchenne muscular dystrophy.
abstract: BACKGROUND
  Therapeutic trials in Duchenne muscular dystrophy (DMD) often exclude non-ambulatory individuals. Here we establish optimal and reliable assessments in a multicenter trial.
  
  
  METHODS
  Non-ambulatory boys/men with DMD (N = 91; 16.7 ± 4.5 years of age) were assessed by trained clinical evaluators. Feasibility (percentage completing task) and reliability [intraclass correlation coefficients (ICCs) between morning and afternoon tests] were measured.
  
  
  RESULTS
  Forced vital capacity (FVC), assessed in all subjects, showed a mean of 47.8 ± 22% predicted (ICC 0.98). Brooke Upper Extremity Functional Rating (Brooke) and Egen Klassifikation (EK) scales in 100% of subjects showed ICCs ranging from 0.93 to 0.99. Manual muscle testing, range of motion, 9-hole peg test, and Jebsen-Taylor Hand Function Test (JHFT) demonstrated varied feasibility (99% to 70%), with ICCs ranging from 0.99 to 0.64. We found beneficial effects of different forms of corticosteroids for the Brooke scale, percent predicted FVC, and hand and finger strength.
  
  
  CONCLUSIONS
  Reliable assessment of non-ambulatory boys/men with DMD is possible. Clinical trials will have to consider corticosteroid use.
authors: Department of Neurology, Washington University School of Medicine in Saint Louis, St. Louis, Missouri, 63110, USA; Department of Pediatrics, Washington University School of Medicine in Saint Louis, St. Louis, Missouri, USA.
authors: Anne M
authors: AM
authors: Connolly
authors: Elizabeth C
authors: EC
authors: Malkus
authors: Jerry R
authors: JR
authors: Mendell
authors: Kevin M
authors: KM
authors: Flanigan
authors: J Philip
authors: JP
authors: Miller
authors: Jeanine R
authors: JR
authors: Schierbecker
authors: Catherine A
authors: CA
authors: Siener
authors: Paul T
authors: PT
authors: Golumbek
authors: Craig M
authors: CM
authors: Zaidman
authors: Craig M
authors: CM
authors: Mcdonald
authors: Linda
authors: L
authors: Johnson
authors: Alina
authors: A
authors: Nicorici
authors: Peter I
authors: PI
authors: Karachunski
authors: John W
authors: JW
authors: Day
authors: Jason M
authors: JM
authors: Kelecic
authors: Linda P
authors: LP
authors: Lowes
authors: Lindsay N
authors: LN
authors: Alfano
authors: Basil T
authors: BT
authors: Darras
authors: Peter B
authors: PB
authors: Kang
authors: Janet
authors: J
authors: Quigley
authors: Amy E
authors: AE
authors: Pasternak
authors: Julaine M
authors: JM
authors: Florence
mesh_terms: D000293:Adolescent; D000305:Adrenal Cortex Hormones / Q000627:therapeutic use*; D000328:Adult; D002648:Child; D004185:Disability Evaluation; D006225:Hand / Q000294:innervation / Q000503:physiopathology*; D006801:Humans; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000175:diagnosis* / Q000188:drug therapy* / Q000503:physiopathology; D015203:Reproducibility of Results; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D000305:Adrenal Cortex Hormones
keywords: Duchenne muscular dystrophy; corticosteroids; non-ambulatory; pulmonary function; quality of life; strength