One year outcome of boys with duchenne muscular dystrophy using the bayley-III scales of infant and toddler development

Anne M. Connolly; Julaine M. Florence; Mary M. Cradock; Michelle Eagle; Kevin M. Flanigan; Craig M. McDonald; Peter I. Karachunski; Basil T. Darras; Kate Bushby; Elizabeth C. Malkus; Paul T. Golumbek; Craig M. Zaidman; J. Philip Miller; Jerry R. Mendell

doi:10.1016/j.pediatrneurol.2014.02.006

One year outcome of boys with duchenne muscular dystrophy using the bayley-III scales of infant and toddler development

Anne M. Connolly, Julaine M. Florence, Mary M. Cradock, Michelle Eagle, Kevin M. Flanigan, Craig M. McDonald, Peter I. Karachunski, Basil T. Darras, Kate Bushby, Elizabeth C. Malkus, Paul T. Golumbek, Craig M. Zaidman, J. Philip Miller, Jerry R. Mendell

Neurology

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Abstract

Background The pathogenesis of Duchenne muscular dystrophy starts before birth. Despite this, clinical trials exclude young boys because traditional outcome measures rely on cooperation. We recently used the Bayley-III Scales of Infant and Toddler Development to study 24 infants and boys with Duchenne muscular dystrophy. Clinical evaluators at six centers were trained and certified to perform the Bayley-III. Here, we report 6- and 12-month follow-up of two subsets of these boys. Patients Nineteen boys (1.9 ± 0.8 years) were assessed at baseline and 6 months. Twelve boys (1.5 ± 0.8 years) were assessed at baseline, 6, and 12 months. Results Gross motor scores were lower at baseline compared with published controls (6.2 ± 1.7; normal 10 ± 3; P < 0.0001) and revealed a further declining trend to 5.7 ± 1.7 (P = 0.20) at 6 months. Repeated measures analysis of the 12 boys monitored for 12 months revealed that gross motor scores, again low at baseline (6.6 ± 1.7; P < 0.0001), declined at 6 months (5.9 ± 1.8) and further at 12 months (5.3 ± 2.0) (P = 0.11). Cognitive and language scores were lower at baseline compared with normal children (range, P = 0.002-<0.0001) and did not change significantly at 6 or 12 months (range, P = 0.89-0.09). Fine motor skills, also low at baseline, improved >1 year (P = 0.05). Conclusion Development can reliably be measured in infants and young boys with Duchenne muscular dystrophy across time using the Bayley-III. Power calculations using these data reveal that motor development may be used as an outcome measure.

Original language	English (US)
Pages (from-to)	557-563
Number of pages	7
Journal	Pediatric Neurology
Volume	50
Issue number	6
DOIs	https://doi.org/10.1016/j.pediatrneurol.2014.02.006
State	Published - Jun 2014

Bibliographical note

Funding Information:
This work was funded by the Muscular Dystrophy Association DMD-center grants to A.M.C., J.D.M., C.M.M., J.W.D., and B.T.D. 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
Duchenne muscular dystrophy
clinical trial outcomes
infant development

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Connolly, A. M., Florence, J. M., Cradock, M. M., Eagle, M., Flanigan, K. M., McDonald, C. M., Karachunski, P. I., Darras, B. T., Bushby, K., Malkus, E. C., Golumbek, P. T., Zaidman, C. M., Miller, J. P., & Mendell, J. R. (2014). One year outcome of boys with duchenne muscular dystrophy using the bayley-III scales of infant and toddler development. Pediatric Neurology, 50(6), 557-563. https://doi.org/10.1016/j.pediatrneurol.2014.02.006

Connolly, AM, Florence, JM, Cradock, MM, Eagle, M, Flanigan, KM, McDonald, CM, Karachunski, PI, Darras, BT, Bushby, K, Malkus, EC, Golumbek, PT, Zaidman, CM, Miller, JP & Mendell, JR 2014, 'One year outcome of boys with duchenne muscular dystrophy using the bayley-III scales of infant and toddler development', Pediatric Neurology, vol. 50, no. 6, pp. 557-563. https://doi.org/10.1016/j.pediatrneurol.2014.02.006

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title = "One year outcome of boys with duchenne muscular dystrophy using the bayley-III scales of infant and toddler development",

abstract = "Background The pathogenesis of Duchenne muscular dystrophy starts before birth. Despite this, clinical trials exclude young boys because traditional outcome measures rely on cooperation. We recently used the Bayley-III Scales of Infant and Toddler Development to study 24 infants and boys with Duchenne muscular dystrophy. Clinical evaluators at six centers were trained and certified to perform the Bayley-III. Here, we report 6- and 12-month follow-up of two subsets of these boys. Patients Nineteen boys (1.9 ± 0.8 years) were assessed at baseline and 6 months. Twelve boys (1.5 ± 0.8 years) were assessed at baseline, 6, and 12 months. Results Gross motor scores were lower at baseline compared with published controls (6.2 ± 1.7; normal 10 ± 3; P < 0.0001) and revealed a further declining trend to 5.7 ± 1.7 (P = 0.20) at 6 months. Repeated measures analysis of the 12 boys monitored for 12 months revealed that gross motor scores, again low at baseline (6.6 ± 1.7; P < 0.0001), declined at 6 months (5.9 ± 1.8) and further at 12 months (5.3 ± 2.0) (P = 0.11). Cognitive and language scores were lower at baseline compared with normal children (range, P = 0.002-<0.0001) and did not change significantly at 6 or 12 months (range, P = 0.89-0.09). Fine motor skills, also low at baseline, improved >1 year (P = 0.05). Conclusion Development can reliably be measured in infants and young boys with Duchenne muscular dystrophy across time using the Bayley-III. Power calculations using these data reveal that motor development may be used as an outcome measure.",

keywords = "Bayley-III, Duchenne muscular dystrophy, clinical trial outcomes, infant development",

author = "Connolly, {Anne M.} and Florence, {Julaine M.} and Cradock, {Mary M.} and Michelle Eagle and Flanigan, {Kevin M.} and McDonald, {Craig M.} and Karachunski, {Peter I.} and Darras, {Basil T.} and Kate Bushby and Malkus, {Elizabeth C.} and Golumbek, {Paul T.} and Zaidman, {Craig M.} and Miller, {J. Philip} and Mendell, {Jerry R.}",

note = "Funding Information: This work was funded by the Muscular Dystrophy Association DMD-center grants to A.M.C., J.D.M., C.M.M., J.W.D., and B.T.D. 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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T1 - One year outcome of boys with duchenne muscular dystrophy using the bayley-III scales of infant and toddler development

AU - Connolly, Anne M.

AU - Florence, Julaine M.

AU - Cradock, Mary M.

AU - Eagle, Michelle

AU - Flanigan, Kevin M.

AU - McDonald, Craig M.

AU - Karachunski, Peter I.

AU - Darras, Basil T.

AU - Bushby, Kate

AU - Malkus, Elizabeth C.

AU - Golumbek, Paul T.

AU - Zaidman, Craig M.

AU - Miller, J. Philip

AU - Mendell, Jerry R.

N1 - Funding Information: This work was funded by the Muscular Dystrophy Association DMD-center grants to A.M.C., J.D.M., C.M.M., J.W.D., and B.T.D. 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 - 2014/6

Y1 - 2014/6

N2 - Background The pathogenesis of Duchenne muscular dystrophy starts before birth. Despite this, clinical trials exclude young boys because traditional outcome measures rely on cooperation. We recently used the Bayley-III Scales of Infant and Toddler Development to study 24 infants and boys with Duchenne muscular dystrophy. Clinical evaluators at six centers were trained and certified to perform the Bayley-III. Here, we report 6- and 12-month follow-up of two subsets of these boys. Patients Nineteen boys (1.9 ± 0.8 years) were assessed at baseline and 6 months. Twelve boys (1.5 ± 0.8 years) were assessed at baseline, 6, and 12 months. Results Gross motor scores were lower at baseline compared with published controls (6.2 ± 1.7; normal 10 ± 3; P < 0.0001) and revealed a further declining trend to 5.7 ± 1.7 (P = 0.20) at 6 months. Repeated measures analysis of the 12 boys monitored for 12 months revealed that gross motor scores, again low at baseline (6.6 ± 1.7; P < 0.0001), declined at 6 months (5.9 ± 1.8) and further at 12 months (5.3 ± 2.0) (P = 0.11). Cognitive and language scores were lower at baseline compared with normal children (range, P = 0.002-<0.0001) and did not change significantly at 6 or 12 months (range, P = 0.89-0.09). Fine motor skills, also low at baseline, improved >1 year (P = 0.05). Conclusion Development can reliably be measured in infants and young boys with Duchenne muscular dystrophy across time using the Bayley-III. Power calculations using these data reveal that motor development may be used as an outcome measure.

AB - Background The pathogenesis of Duchenne muscular dystrophy starts before birth. Despite this, clinical trials exclude young boys because traditional outcome measures rely on cooperation. We recently used the Bayley-III Scales of Infant and Toddler Development to study 24 infants and boys with Duchenne muscular dystrophy. Clinical evaluators at six centers were trained and certified to perform the Bayley-III. Here, we report 6- and 12-month follow-up of two subsets of these boys. Patients Nineteen boys (1.9 ± 0.8 years) were assessed at baseline and 6 months. Twelve boys (1.5 ± 0.8 years) were assessed at baseline, 6, and 12 months. Results Gross motor scores were lower at baseline compared with published controls (6.2 ± 1.7; normal 10 ± 3; P < 0.0001) and revealed a further declining trend to 5.7 ± 1.7 (P = 0.20) at 6 months. Repeated measures analysis of the 12 boys monitored for 12 months revealed that gross motor scores, again low at baseline (6.6 ± 1.7; P < 0.0001), declined at 6 months (5.9 ± 1.8) and further at 12 months (5.3 ± 2.0) (P = 0.11). Cognitive and language scores were lower at baseline compared with normal children (range, P = 0.002-<0.0001) and did not change significantly at 6 or 12 months (range, P = 0.89-0.09). Fine motor skills, also low at baseline, improved >1 year (P = 0.05). Conclusion Development can reliably be measured in infants and young boys with Duchenne muscular dystrophy across time using the Bayley-III. Power calculations using these data reveal that motor development may be used as an outcome measure.

KW - Bayley-III

KW - Duchenne muscular dystrophy

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KW - infant development

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One year outcome of boys with duchenne muscular dystrophy using the bayley-III scales of infant and toddler development

Abstract

Bibliographical note

Keywords

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