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 language | English (US) |
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Pages (from-to) | 55-67 |
Number of pages | 13 |
Journal | Muscle and Nerve |
Volume | 48 |
Issue number | 1 |
DOIs | |
State | Published - Jul 1 2013 |
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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
Research output: Contribution to journal › Article
}
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 -