The cooperative international neuromuscular research group Duchenne natural history study: Glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures

the CINRG Investigators

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110 Citations (Scopus)

Abstract

Introduction: Glucocorticoid (GC) therapy in Duchenne muscular dystrophy (DMD) has altered disease progression, necessitating contemporary natural history studies. Methods: The Cooperative Neuromuscular Research Group (CINRG) DMD Natural History Study (DMD-NHS) enrolled 340 DMD males, ages 2-28 years. A comprehensive battery of measures was obtained. Results: A novel composite functional "milestone" scale scale showed clinically meaningful mobility and upper limb abilities were significantly preserved in GC-treated adolescents/young adults. Manual muscle test (MMT)-based calculations of global strength showed that those patients <10 years of age treated with steroids declined by 0.4±0.39 MMT unit/year, compared with -0.4±0.39 MMT unit/year in historical steroid-naive subjects. Pulmonary function tests (PFTs) were relatively preserved in steroid-treated adolescents. The linearity and magnitude of decline in measures were affected by maturational changes and functional status. Conclusions: In DMD, long-term use of GCs showed reduced strength loss and preserved functional capabilities and PFTs compared with previous natural history studies performed prior to the widespread use of GC therapy.

Original languageEnglish (US)
Pages (from-to)55-67
Number of pages13
JournalMuscle and Nerve
Volume48
Issue number1
DOIs
StatePublished - Jul 1 2013

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Duchenne Muscular Dystrophy
Natural History
Glucocorticoids
Disease Progression
Outcome Assessment (Health Care)
Clinical Trials
Muscles
Respiratory Function Tests
Steroids
Research
Therapeutics
Upper Extremity
Young Adult

Keywords

  • Adolescent
  • Adult
  • Child/preschool
  • Follow-up studies
  • Health status
  • Humans
  • Locomotion
  • Male
  • Muscle strength/physiology
  • Muscular dystrophies/Duchenne/physiopathology
  • Muscular dystrophies/classification
  • Muscular dystrophies/therapy
  • Phenotype
  • Quality of life/psychology
  • Respiratory function tests

Cite this

@article{8b5d0755e77a463daffc2a2977693b59,
title = "The cooperative international neuromuscular research group Duchenne natural history study: Glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures",
abstract = "Introduction: Glucocorticoid (GC) therapy in Duchenne muscular dystrophy (DMD) has altered disease progression, necessitating contemporary natural history studies. Methods: The Cooperative Neuromuscular Research Group (CINRG) DMD Natural History Study (DMD-NHS) enrolled 340 DMD males, ages 2-28 years. A comprehensive battery of measures was obtained. Results: A novel composite functional {"}milestone{"} scale scale showed clinically meaningful mobility and upper limb abilities were significantly preserved in GC-treated adolescents/young adults. Manual muscle test (MMT)-based calculations of global strength showed that those patients <10 years of age treated with steroids declined by 0.4±0.39 MMT unit/year, compared with -0.4±0.39 MMT unit/year in historical steroid-naive subjects. Pulmonary function tests (PFTs) were relatively preserved in steroid-treated adolescents. The linearity and magnitude of decline in measures were affected by maturational changes and functional status. Conclusions: In DMD, long-term use of GCs showed reduced strength loss and preserved functional capabilities and PFTs compared with previous natural history studies performed prior to the widespread use of GC therapy.",
keywords = "Adolescent, Adult, Child/preschool, Follow-up studies, Health status, Humans, Locomotion, Male, Muscle strength/physiology, Muscular dystrophies/Duchenne/physiopathology, Muscular dystrophies/classification, Muscular dystrophies/therapy, Phenotype, Quality of life/psychology, Respiratory function tests",
author = "{the CINRG Investigators} and Henricson, {Erik K.} and Abresch, {R. Ted} and Avital Cnaan and Fengming Hu and Tina Duong and Adrienne Arrieta and Jay Han and Escolar, {Diana M.} and Florence, {Julaine M.} and Clemens, {Paula R.} and Hoffman, {Eric P.} and McDonald, {Craig M.} and V. Vishwanathan and S. Chidambaranathan and Biggar, {W. Douglas} and Mah, {Jean K.} and Mar Tulinius and Robert Leshner and Carolina Tesi-Rocha and Andrew Kornberg and Monique Ryan and Yoram Nevo and Alberto Dubrovsky and Nancy Kuntz and Sherilyn Driscoll and Anne Connolly and Alan Pestronk and Jean Teasley and Tulio Bertorini and Kathryn North and Hanna Kolski and Jose Carlo and Ksenija Gorni and Timothy Lotze and John Day and Josh Benditt and Louis Boitano and David Birnkrant and David Connuck and Jonathan Finder and Veronica Hinton and Katherine Mathews and Richard Moxley and Susan Sparks and Erynn Gordon and Michelle Cregan and Erica Goude and Merete Glick and Linda Johnson and David Walk",
year = "2013",
month = "7",
day = "1",
doi = "10.1002/mus.23808",
language = "English (US)",
volume = "48",
pages = "55--67",
journal = "Muscle and Nerve",
issn = "0148-639X",
publisher = "John Wiley and Sons Inc.",
number = "1",

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TY - JOUR

T1 - The cooperative international neuromuscular research group Duchenne natural history study

T2 - Glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures

AU - the CINRG Investigators

AU - Henricson, Erik K.

AU - Abresch, R. Ted

AU - Cnaan, Avital

AU - Hu, Fengming

AU - Duong, Tina

AU - Arrieta, Adrienne

AU - Han, Jay

AU - Escolar, Diana M.

AU - Florence, Julaine M.

AU - Clemens, Paula R.

AU - Hoffman, Eric P.

AU - McDonald, Craig M.

AU - Vishwanathan, V.

AU - Chidambaranathan, S.

AU - Biggar, W. Douglas

AU - Mah, Jean K.

AU - Tulinius, Mar

AU - Leshner, Robert

AU - Tesi-Rocha, Carolina

AU - Kornberg, Andrew

AU - Ryan, Monique

AU - Nevo, Yoram

AU - Dubrovsky, Alberto

AU - Kuntz, Nancy

AU - Driscoll, Sherilyn

AU - Connolly, Anne

AU - Pestronk, Alan

AU - Teasley, Jean

AU - Bertorini, Tulio

AU - North, Kathryn

AU - Kolski, Hanna

AU - Carlo, Jose

AU - Gorni, Ksenija

AU - Lotze, Timothy

AU - Day, John

AU - Benditt, Josh

AU - Boitano, Louis

AU - Birnkrant, David

AU - Connuck, David

AU - Finder, Jonathan

AU - Hinton, Veronica

AU - Mathews, Katherine

AU - Moxley, Richard

AU - Sparks, Susan

AU - Gordon, Erynn

AU - Cregan, Michelle

AU - Goude, Erica

AU - Glick, Merete

AU - Johnson, Linda

AU - Walk, David

PY - 2013/7/1

Y1 - 2013/7/1

N2 - Introduction: Glucocorticoid (GC) therapy in Duchenne muscular dystrophy (DMD) has altered disease progression, necessitating contemporary natural history studies. Methods: The Cooperative Neuromuscular Research Group (CINRG) DMD Natural History Study (DMD-NHS) enrolled 340 DMD males, ages 2-28 years. A comprehensive battery of measures was obtained. Results: A novel composite functional "milestone" scale scale showed clinically meaningful mobility and upper limb abilities were significantly preserved in GC-treated adolescents/young adults. Manual muscle test (MMT)-based calculations of global strength showed that those patients <10 years of age treated with steroids declined by 0.4±0.39 MMT unit/year, compared with -0.4±0.39 MMT unit/year in historical steroid-naive subjects. Pulmonary function tests (PFTs) were relatively preserved in steroid-treated adolescents. The linearity and magnitude of decline in measures were affected by maturational changes and functional status. Conclusions: In DMD, long-term use of GCs showed reduced strength loss and preserved functional capabilities and PFTs compared with previous natural history studies performed prior to the widespread use of GC therapy.

AB - Introduction: Glucocorticoid (GC) therapy in Duchenne muscular dystrophy (DMD) has altered disease progression, necessitating contemporary natural history studies. Methods: The Cooperative Neuromuscular Research Group (CINRG) DMD Natural History Study (DMD-NHS) enrolled 340 DMD males, ages 2-28 years. A comprehensive battery of measures was obtained. Results: A novel composite functional "milestone" scale scale showed clinically meaningful mobility and upper limb abilities were significantly preserved in GC-treated adolescents/young adults. Manual muscle test (MMT)-based calculations of global strength showed that those patients <10 years of age treated with steroids declined by 0.4±0.39 MMT unit/year, compared with -0.4±0.39 MMT unit/year in historical steroid-naive subjects. Pulmonary function tests (PFTs) were relatively preserved in steroid-treated adolescents. The linearity and magnitude of decline in measures were affected by maturational changes and functional status. Conclusions: In DMD, long-term use of GCs showed reduced strength loss and preserved functional capabilities and PFTs compared with previous natural history studies performed prior to the widespread use of GC therapy.

KW - Adolescent

KW - Adult

KW - Child/preschool

KW - Follow-up studies

KW - Health status

KW - Humans

KW - Locomotion

KW - Male

KW - Muscle strength/physiology

KW - Muscular dystrophies/Duchenne/physiopathology

KW - Muscular dystrophies/classification

KW - Muscular dystrophies/therapy

KW - Phenotype

KW - Quality of life/psychology

KW - Respiratory function tests

UR - http://www.scopus.com/inward/record.url?scp=84879551615&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84879551615&partnerID=8YFLogxK

U2 - 10.1002/mus.23808

DO - 10.1002/mus.23808

M3 - Article

C2 - 23649481

AN - SCOPUS:84879551615

VL - 48

SP - 55

EP - 67

JO - Muscle and Nerve

JF - Muscle and Nerve

SN - 0148-639X

IS - 1

ER -