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) |
|---|---|
| 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 -