Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy

for the MDA DMD Clinical Research Network

doi:10.1002/mus.26441

Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy

for the MDA DMD Clinical Research Network

Pediatrics

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

35 Scopus citations

Abstract

Introduction: Glucocorticosteroids (GC) are effective in slowing weakness in boys with Duchenne muscular dystrophy (DMD). Methods: This is a multisite, 1-year, open-label trial of twice-weekly prednisolone (5 mg/kg/dose) in infants/young boys (0.4–2.4 years) with DMD. We compared changes in Bayley III Scales of Infant Development (Bayley-III) with untreated boys followed for 1 year (historical control cohort [HCC]). Twenty-three of 25 participants completed the study. Results: Treated boys gained an average of 0.5 points on the Bayley-III gross motor scaled score (GMSS) compared with the HCC who, on average, declined 1.3 points (P = 0.03). All boys maintained linear growth, and none developed Cushingoid features. Excessive weight gain occurred in 13 of 23 (56%) boys. Discussion: This study provides evidence that twice-weekly GC is well tolerated in infants and young boys with DMD and improves GMSS. Excessive weight gain is a potential risk. Longer follow-up is required to determine whether early GC initiation is feasible in most infants/boys with DMD. Muscle Nerve 59:650–657, 2019.

Original language	English (US)
Pages (from-to)	650-657
Number of pages	8
Journal	Muscle and Nerve
Volume	59
Issue number	6
DOIs	https://doi.org/10.1002/mus.26441
State	Published - Jun 2019

Funding

Abbreviations: AE, adverse event; Bayley-III, Bayley-III Scales of Infant Development; CDC, US Centers for Disease Control and Prevention; CE, clinical evaluator; DMD, Duchenne muscular dystrophy; FMSS, fine motor scaled score; GC, glucocorticosteroids; GMSS, gross motor scaled score; HCC, historical control cohort; TC, treated cohort Key words: Bayley-III Scales of Infant and Toddler Development; DMD; Duchenne muscular dystrophy; GC; infants; twice-weekly glucocorticosteroid Funding: All phases of this study were supported by a grant from the Muscular Dystrophy Association USA (Grant 266053). Conflicts of Interest: No author has relevant disclosures for this work. Clinical Trial Registration: ClinicalTrials.gov ID NCT02167217 “Historically controlled Trial of Corticosteroids in Young boys with Duchenne Muscular Dystrophy” ClinicalTrials.gov PRS Protocol ID 201308062

Keywords

Bayley-III Scales of Infant and Toddler Development
DMD
Duchenne muscular dystrophy
GC
infants
twice-weekly glucocorticosteroid

ASJC Scopus subject areas

Clinical Neurology
Physiology (medical)
Cellular and Molecular Neuroscience
Physiology

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10.1002/mus.26441

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title = "Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy",

abstract = "Introduction: Glucocorticosteroids (GC) are effective in slowing weakness in boys with Duchenne muscular dystrophy (DMD). Methods: This is a multisite, 1-year, open-label trial of twice-weekly prednisolone (5 mg/kg/dose) in infants/young boys (0.4–2.4 years) with DMD. We compared changes in Bayley III Scales of Infant Development (Bayley-III) with untreated boys followed for 1 year (historical control cohort [HCC]). Twenty-three of 25 participants completed the study. Results: Treated boys gained an average of 0.5 points on the Bayley-III gross motor scaled score (GMSS) compared with the HCC who, on average, declined 1.3 points (P = 0.03). All boys maintained linear growth, and none developed Cushingoid features. Excessive weight gain occurred in 13 of 23 (56%) boys. Discussion: This study provides evidence that twice-weekly GC is well tolerated in infants and young boys with DMD and improves GMSS. Excessive weight gain is a potential risk. Longer follow-up is required to determine whether early GC initiation is feasible in most infants/boys with DMD. Muscle Nerve 59:650–657, 2019.",

keywords = "Bayley-III Scales of Infant and Toddler Development, DMD, Duchenne muscular dystrophy, GC, infants, twice-weekly glucocorticosteroid",

author = "{for the MDA DMD Clinical Research Network} and Connolly, {Anne M.} and Zaidman, {Craig M.} and Golumbek, {Paul T.} and Cradock, {Mary M.} and Flanigan, {Kevin M.} and Kuntz, {Nancy L.} and Finkel, {Richard S.} and McDonald, {Craig M.} and Iannaccone, {Susan T.} and Pallavi Anand and Siener, {Catherine A.} and Florence, {Julaine M.} and Lowes, {Linda P.} and Alfano, {Lindsay N.} and Johnson, {Linda B.} and Alina Nicorici and Nelson, {Leslie L.} and Mendell, {Jerry R.}",

note = "Publisher Copyright: {\textcopyright} 2019 Wiley Periodicals, Inc.",

year = "2019",

month = jun,

doi = "10.1002/mus.26441",

language = "English (US)",

volume = "59",

pages = "650--657",

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T1 - Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy

AU - for the MDA DMD Clinical Research Network

AU - Connolly, Anne M.

AU - Zaidman, Craig M.

AU - Golumbek, Paul T.

AU - Cradock, Mary M.

AU - Flanigan, Kevin M.

AU - Kuntz, Nancy L.

AU - Finkel, Richard S.

AU - McDonald, Craig M.

AU - Iannaccone, Susan T.

AU - Anand, Pallavi

AU - Siener, Catherine A.

AU - Florence, Julaine M.

AU - Lowes, Linda P.

AU - Alfano, Lindsay N.

AU - Johnson, Linda B.

AU - Nicorici, Alina

AU - Nelson, Leslie L.

AU - Mendell, Jerry R.

PY - 2019/6

Y1 - 2019/6

N2 - Introduction: Glucocorticosteroids (GC) are effective in slowing weakness in boys with Duchenne muscular dystrophy (DMD). Methods: This is a multisite, 1-year, open-label trial of twice-weekly prednisolone (5 mg/kg/dose) in infants/young boys (0.4–2.4 years) with DMD. We compared changes in Bayley III Scales of Infant Development (Bayley-III) with untreated boys followed for 1 year (historical control cohort [HCC]). Twenty-three of 25 participants completed the study. Results: Treated boys gained an average of 0.5 points on the Bayley-III gross motor scaled score (GMSS) compared with the HCC who, on average, declined 1.3 points (P = 0.03). All boys maintained linear growth, and none developed Cushingoid features. Excessive weight gain occurred in 13 of 23 (56%) boys. Discussion: This study provides evidence that twice-weekly GC is well tolerated in infants and young boys with DMD and improves GMSS. Excessive weight gain is a potential risk. Longer follow-up is required to determine whether early GC initiation is feasible in most infants/boys with DMD. Muscle Nerve 59:650–657, 2019.

AB - Introduction: Glucocorticosteroids (GC) are effective in slowing weakness in boys with Duchenne muscular dystrophy (DMD). Methods: This is a multisite, 1-year, open-label trial of twice-weekly prednisolone (5 mg/kg/dose) in infants/young boys (0.4–2.4 years) with DMD. We compared changes in Bayley III Scales of Infant Development (Bayley-III) with untreated boys followed for 1 year (historical control cohort [HCC]). Twenty-three of 25 participants completed the study. Results: Treated boys gained an average of 0.5 points on the Bayley-III gross motor scaled score (GMSS) compared with the HCC who, on average, declined 1.3 points (P = 0.03). All boys maintained linear growth, and none developed Cushingoid features. Excessive weight gain occurred in 13 of 23 (56%) boys. Discussion: This study provides evidence that twice-weekly GC is well tolerated in infants and young boys with DMD and improves GMSS. Excessive weight gain is a potential risk. Longer follow-up is required to determine whether early GC initiation is feasible in most infants/boys with DMD. Muscle Nerve 59:650–657, 2019.

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KW - Duchenne muscular dystrophy

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KW - infants

KW - twice-weekly glucocorticosteroid

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Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy

Abstract

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Keywords

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