Premium
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
Author(s) -
McDonald Craig M.,
Henricson Erik K.,
Abresch R. Ted,
Han Jay J.,
Escolar Diana M.,
Florence Julaine M.,
Duong Tina,
Arrieta Adrienne,
Clemens Paula R.,
Hoffman Eric P.,
Cnaan Avital
Publication year - 2013
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.23807
Subject(s) - natural history study , natural history , duchenne muscular dystrophy , medicine , clinical trial , physical therapy , anthropometry , observational study , neuromuscular disease , longitudinal study , quality of life (healthcare) , physical medicine and rehabilitation , disease , pathology , nursing
Contemporary natural history data in Duchenne muscular dystrophy (DMD) is needed to assess care recommendations and aid in planning future trials. Methods 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. Results 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). Conclusions 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. Muscle Nerve , 2013