Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: Results from the Muscular Dystrophy Association DMD Clinical Research Network

Anne M. Connolly; Julaine M. Florence; Mary M. Cradock; Elizabeth C. Malkus; Jeanine R. Schierbecker; Catherine A. Siener; Charlie O. Wulf; Pallavi Anand; Paul T. Golumbek; Craig M. Zaidman; J. Philip Miller; Linda P. Lowes; Lindsay N. Alfano; Laurence Viollet-Callendret; Kevin M. Flanigan; Jerry R. Mendell; Craig M. McDonald; Erica Goude; Linda Johnson; Alina Nicorici; Peter I. Karachunski; John W. Day; Joline C. Dalton; Janey M. Farber; Karen K. Buser; Basil T. Darras; Peter B. Kang; Susan O. Riley; Elizabeth Shriber; Rebecca Parad; Kate Bushby; Michelle Eagle

doi:10.1016/j.nmd.2013.04.005

Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: Results from the Muscular Dystrophy Association DMD Clinical Research Network

Anne M. Connolly, Julaine M. Florence, Mary M. Cradock, Elizabeth C. Malkus, Jeanine R. Schierbecker, Catherine A. Siener, Charlie O. Wulf, Pallavi Anand, Paul T. Golumbek, Craig M. Zaidman, J. Philip Miller, Linda P. Lowes, Lindsay N. Alfano, Laurence Viollet-Callendret, Kevin M. Flanigan, Jerry R. Mendell, Craig M. McDonald, Erica Goude, Linda Johnson, Alina NicoriciPeter I. Karachunski, John W. Day, Joline C. Dalton, Janey M. Farber, Karen K. Buser, Basil T. Darras, Peter B. Kang, Susan O. Riley, Elizabeth Shriber, Rebecca Parad, Kate Bushby, Michelle Eagle

Neurology

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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.

Original language	English (US)
Pages (from-to)	529-539
Number of pages	11
Journal	Neuromuscular Disorders
Volume	23
Issue number	7
DOIs	https://doi.org/10.1016/j.nmd.2013.04.005
State	Published - Jul 2013

Bibliographical note

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.

Keywords

Bayley III Scales of Infant and Toddler Development
Duchenne Muscular Dystrophy
Hammersmith Functional Motor Scale Extended
Infant
North Star Ambulatory Assessment

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Connolly, A. M., Florence, J. M., Cradock, M. M., Malkus, E. C., Schierbecker, J. R., Siener, C. A., Wulf, C. O., Anand, P., Golumbek, P. T., Zaidman, C. M., Philip Miller, J., Lowes, L. P., Alfano, L. N., Viollet-Callendret, L., Flanigan, K. M., Mendell, J. R., McDonald, C. M., Goude, E., Johnson, L., ... Eagle, M. (2013). Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: Results from the Muscular Dystrophy Association DMD Clinical Research Network. Neuromuscular Disorders, 23(7), 529-539. https://doi.org/10.1016/j.nmd.2013.04.005

Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: Results from the Muscular Dystrophy Association DMD Clinical Research Network. / Connolly, Anne M.; Florence, Julaine M.; Cradock, Mary M. et al.
In: Neuromuscular Disorders, Vol. 23, No. 7, 07.2013, p. 529-539.

Research output: Contribution to journal › Article › peer-review

Connolly, AM, Florence, JM, Cradock, MM, Malkus, EC, Schierbecker, JR, Siener, CA, Wulf, CO, Anand, P, Golumbek, PT, Zaidman, CM, Philip Miller, J, Lowes, LP, Alfano, LN, Viollet-Callendret, L, Flanigan, KM, Mendell, JR, McDonald, CM, Goude, E, Johnson, L, Nicorici, A, Karachunski, PI, Day, JW, Dalton, JC, Farber, JM, Buser, KK, Darras, BT, Kang, PB, Riley, SO, Shriber, E, Parad, R, Bushby, K & Eagle, M 2013, 'Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: Results from the Muscular Dystrophy Association DMD Clinical Research Network', Neuromuscular Disorders, vol. 23, no. 7, pp. 529-539. https://doi.org/10.1016/j.nmd.2013.04.005

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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.",

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author = "Connolly, {Anne M.} and Florence, {Julaine M.} and Cradock, {Mary M.} and Malkus, {Elizabeth C.} and Schierbecker, {Jeanine R.} and Siener, {Catherine A.} and Wulf, {Charlie O.} and Pallavi Anand and Golumbek, {Paul T.} and Zaidman, {Craig M.} and {Philip Miller}, J. and Lowes, {Linda P.} and Alfano, {Lindsay N.} and Laurence Viollet-Callendret and Flanigan, {Kevin M.} and Mendell, {Jerry R.} and McDonald, {Craig M.} and Erica Goude and Linda Johnson and Alina Nicorici and Karachunski, {Peter I.} and Day, {John W.} and Dalton, {Joline C.} and Farber, {Janey M.} and Buser, {Karen K.} and Darras, {Basil T.} and Kang, {Peter B.} and Riley, {Susan O.} and Elizabeth Shriber and Rebecca Parad and Kate Bushby and Michelle Eagle",

note = "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.",

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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.

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AU - Mendell, Jerry R.

AU - McDonald, Craig M.

AU - Goude, Erica

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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

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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.

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KW - Duchenne Muscular Dystrophy

KW - Hammersmith Functional Motor Scale Extended

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Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: Results from the Muscular Dystrophy Association DMD Clinical Research Network

Abstract

Bibliographical note

Keywords

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