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

for the MDA DMD Clinical Research Network

Research output: Contribution to journalArticle

4 Citations (Scopus)

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, 2019.

Original languageEnglish (US)
JournalMuscle and Nerve
DOIs
StatePublished - Jan 1 2019

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Duchenne Muscular Dystrophy
Child Development
Weight Gain
Prednisolone
Muscles
Growth

Keywords

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

ASJC Scopus subject areas

  • Physiology
  • Clinical Neurology
  • Cellular and Molecular Neuroscience
  • Physiology (medical)

Cite this

Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy. / for the MDA DMD Clinical Research Network.

In: Muscle and Nerve, 01.01.2019.

Research output: Contribution to journalArticle

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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, 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.}",
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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 - 2019/1/1

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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, 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, 2019.

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KW - twice-weekly glucocorticosteroid

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