TY - JOUR
T1 - Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy
T2 - Results from the Muscular Dystrophy Association DMD Clinical Research Network
AU - Connolly, Anne M.
AU - Florence, Julaine M.
AU - Cradock, Mary M.
AU - Malkus, Elizabeth C.
AU - Schierbecker, Jeanine R.
AU - Siener, Catherine A.
AU - Wulf, Charlie O.
AU - Anand, Pallavi
AU - Golumbek, Paul T.
AU - Zaidman, Craig M.
AU - Philip Miller, J.
AU - Lowes, Linda P.
AU - Alfano, Lindsay N.
AU - Viollet-Callendret, Laurence
AU - Flanigan, Kevin M.
AU - Mendell, Jerry R.
AU - McDonald, Craig M.
AU - Goude, Erica
AU - Johnson, Linda
AU - Nicorici, Alina
AU - Karachunski, Peter I.
AU - Day, John W.
AU - Dalton, Joline C.
AU - Farber, Janey M.
AU - Buser, Karen K.
AU - Darras, Basil T.
AU - Kang, Peter B.
AU - Riley, Susan O.
AU - Shriber, Elizabeth
AU - Parad, Rebecca
AU - Bushby, Kate
AU - Eagle, Michelle
N1 - Funding Information:
This work was funded by the Muscular Dystrophy Association DMD-center grants to AMC, JDM, CMM, JWD, and BTD. This publication was also made possible by Grant No. UL1 RR024992 from the National Center for Research Resources (NCRR) , a component of the National Institutes of Health (NIH) , and NIH Roadmap for Medical Research . Its contents are solely the responsibility of the authors and do not necessarily represent the official view of NCRR or NIH.
PY - 2013/7
Y1 - 2013/7
N2 - 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.
AB - 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.
KW - Bayley III Scales of Infant and Toddler Development
KW - Duchenne Muscular Dystrophy
KW - Hammersmith Functional Motor Scale Extended
KW - Infant
KW - North Star Ambulatory Assessment
UR - http://www.scopus.com/inward/record.url?scp=84878983799&partnerID=8YFLogxK
U2 - 10.1016/j.nmd.2013.04.005
DO - 10.1016/j.nmd.2013.04.005
M3 - Article
C2 - 23726376
AN - SCOPUS:84878983799
SN - 0960-8966
VL - 23
SP - 529
EP - 539
JO - Neuromuscular Disorders
JF - Neuromuscular Disorders
IS - 7
ER -