title: Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: results from the Muscular Dystrophy Association DMD Clinical Research Network.
abstract: Therapeutic trials in Duchenne Muscular Dystrophy (DMD) exclude young boys because traditional outcome measures rely on cooperation. The Bayley III Scales of Infant and Toddler Development (Bayley III) have been validated in developing children and those with developmental disorders but have not been studied in DMD. Expanded Hammersmith Functional Motor Scale (HFMSE) and North Star Ambulatory Assessment (NSAA) may also be useful in this young DMD population. Clinical evaluators from the MDA-DMD Clinical Research Network were trained in these assessment tools. Infants and boys with DMD (n = 24; 1.9 ± 0.7 years) were assessed. The mean Bayley III motor composite score was low (82.8 ± 8; p ≤ .0001) (normal = 100 ± 15). Mean gross motor and fine motor function scaled scores were low (both p ≤ .0001). The mean cognitive comprehensive (p=.0002), receptive language (p ≤ .0001), and expressive language (p = .0001) were also low compared to normal children. Age was negatively associated with Bayley III gross motor (r = -0.44; p = .02) but not with fine motor, cognitive, or language scores. HFMSE (n=23) showed a mean score of 31 ± 13. NSAA (n = 18 boys; 2.2 ± 0.4 years) showed a mean score of 12 ± 5. Outcome assessments of young boys with DMD are feasible and in this multicenter study were best demonstrated using the Bayley III.
authors.affiliation: Department of Neurology, Washington University School of Medicine, Saint Louis, MO 63110, USA. connollya@wustl.edu
authors.forename: Anne M
authors.initials: AM
authors.lastname: Connolly
authors.forename: Julaine M
authors.initials: JM
authors.lastname: Florence
authors.forename: Mary M
authors.initials: MM
authors.lastname: Cradock
authors.forename: Elizabeth C
authors.initials: EC
authors.lastname: Malkus
authors.forename: Jeanine R
authors.initials: JR
authors.lastname: Schierbecker
authors.forename: Catherine A
authors.initials: CA
authors.lastname: Siener
authors.forename: Charlie O
authors.initials: CO
authors.lastname: Wulf
authors.forename: Pallavi
authors.initials: P
authors.lastname: Anand
authors.forename: Paul T
authors.initials: PT
authors.lastname: Golumbek
authors.forename: Craig M
authors.initials: CM
authors.lastname: Zaidman
authors.forename: J
authors.initials: J
authors.lastname: Philip Miller
authors.forename: Linda P
authors.initials: LP
authors.lastname: Lowes
authors.forename: Lindsay N
authors.initials: LN
authors.lastname: Alfano
authors.forename: Laurence
authors.initials: L
authors.lastname: Viollet-Callendret
authors.forename: Kevin M
authors.initials: KM
authors.lastname: Flanigan
authors.forename: Jerry R
authors.initials: JR
authors.lastname: Mendell
authors.forename: Craig M
authors.initials: CM
authors.lastname: McDonald
authors.forename: Erica
authors.initials: E
authors.lastname: Goude
authors.forename: Linda
authors.initials: L
authors.lastname: Johnson
authors.forename: Alina
authors.initials: A
authors.lastname: Nicorici
authors.forename: Peter I
authors.initials: PI
authors.lastname: Karachunski
authors.forename: John W
authors.initials: JW
authors.lastname: Day
authors.forename: Joline C
authors.initials: JC
authors.lastname: Dalton
authors.forename: Janey M
authors.initials: JM
authors.lastname: Farber
authors.forename: Karen K
authors.initials: KK
authors.lastname: Buser
authors.forename: Basil T
authors.initials: BT
authors.lastname: Darras
authors.forename: Peter B
authors.initials: PB
authors.lastname: Kang
authors.forename: Susan O
authors.initials: SO
authors.lastname: Riley
authors.forename: Elizabeth
authors.initials: E
authors.lastname: Shriber
authors.forename: Rebecca
authors.initials: R
authors.lastname: Parad
authors.forename: Kate
authors.initials: K
authors.lastname: Bushby
authors.forename: Michelle
authors.initials: M
authors.lastname: Eagle
mesh_terms: D000367:Age Factors; D002648:Child; D002657:Child Development / Q000502:physiology; D002675:Child, Preschool; D002986:Clinical Trials as Topic; D003071:Cognition / Q000502:physiology*; D002658:Developmental Disabilities / Q000150:complications / Q000503:physiopathology / Q000628:therapy; D006801:Humans; D007223:Infant; D008297:Male; D009043:Motor Activity / Q000502:physiology*; D020388:Muscular Dystrophy, Duchenne / Q000150:complications / Q000503:physiopathology / Q000628:therapy*; D017063:Outcome Assessment, Health Care / Q000379:methods