Journal article
The cooperative international neuromuscular research group Duchenne natural history study--a longitudinal investigation in the era of glucocorticoid therapy: design of protocol and the methods used
Muscle & nerve, Vol.48(1), pp.32-54
07/2013
DOI: 10.1002/mus.23807
PMCID: PMC4147958
PMID: 23677550
Abstract
Contemporary natural history data in Duchenne muscular dystrophy (DMD) is needed to assess care recommendations and aid in planning future trials.
The Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study (DMD-NHS) enrolled 340 individuals, aged 2-28 years, with DMD in a longitudinal, observational study at 20 centers. Assessments obtained every 3 months for 1 year, at 18 months, and annually thereafter included: clinical history; anthropometrics; goniometry; manual muscle testing; quantitative muscle strength; timed function tests; pulmonary function; and patient-reported outcomes/health-related quality-of-life instruments.
Glucocorticoid (GC) use at baseline was 62% present, 14% past, and 24% GC-naive. In those ≥6 years of age, 16% lost ambulation over the first 12 months (mean age 10.8 years).
Detailed information on the study methodology of the CINRG DMD-NHS lays the groundwork for future analyses of prospective longitudinal natural history data. These data will assist investigators in designing clinical trials of novel therapeutics.
Details
- Title: Subtitle
- The cooperative international neuromuscular research group Duchenne natural history study--a longitudinal investigation in the era of glucocorticoid therapy: design of protocol and the methods used
- Creators
- Craig M McDonald - Department of Physical Medicine & Rehabilitation, School of Medicine, University of California, Davis, 4860 Y Street, Suite 3850, Sacramento, California 95817, USA. cmmcdonald@ucdavis.eduErik K HenricsonR Ted AbreschJay J HanDiana M EscolarJulaine M FlorenceTina DuongAdrienne ArrietaPaula R ClemensEric P HoffmanAvital CnaanCooperative International Neuromuscular Research Group (CINRG) Investigators
- Contributors
- Katherine D Mathews (Contributor) - University of Iowa, Stead Family Department of Pediatrics
- Resource Type
- Journal article
- Publication Details
- Muscle & nerve, Vol.48(1), pp.32-54
- Publisher
- United States
- DOI
- 10.1002/mus.23807
- PMID
- 23677550
- PMCID
- PMC4147958
- ISSN
- 0148-639X
- eISSN
- 1097-4598
- Grant note
- R01AR062380 / NIAMS NIH HHS\r\nU54HD053177 / NICHD NIH HHS\r\nG12 MD007600 / NIMHD NIH HHS\r\nG12RR003051 / NCRR NIH HHS\r\nR01 AR061875 / NIAMS NIH HHS\r\nU54 HD053177 / NICHD NIH HHS\r\nUL1 RR031988 / NCRR NIH HHS\r\nR01 AR062380 / NIAMS NIH HHS\r\nUL1 TR000448 / NCATS NIH HHS\r\nUL1RR031988 / NCRR NIH HHS\r\nG12 RR003051 / NCRR NIH HHS\r\n1R01AR061875 / NIAMS NIH HHS\r\nU54RR026139 / NCRR NIH HHS\r\nU54 RR026139 / NCRR NIH HHS\r\nUL1RR024992 / NCRR NIH HHS
- Language
- English
- Date published
- 07/2013
- Academic Unit
- Neurology; Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Neurology (Pediatrics)
- Record Identifier
- 9984070713602771
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