TY - JOUR
T1 - The Cooperative International Neuromuscular Research Group Duchenne Natural History Study-A Longitudinal Investigation In The Era Of Glucocorticoid Therapy
T2 - Design Of Protocol And The Methods Used
AU - McDonald, Craig M.
AU - Henricson, Erik K.
AU - Abresch, R. Ted
AU - Han, Jay J.
AU - Escolar, Diana M.
AU - Florence, Julaine M.
AU - Duong, Tina
AU - Arrieta, Adrienne
AU - Clemens, Paula R.
AU - Hoffman, Eric P.
AU - Cnaan, Avital
AU - the Cooperative International Neuromuscular Research Group (CINRG) Investigators
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 - Joyce, Nanette
AU - Nicorici, Alina
AU - Kumar, Suresh
AU - Skalsky, Andrew
AU - Witt, Amanda
AU - Lipa, Bethany
AU - Eliasoph, Laila
AU - Hosaki, Elizabeth
AU - Gonzales, Angela
AU - Harris, Vivien
AU - Chiu, Angela
AU - Goia, Edit
AU - Thannhauser, Jennifer
AU - Walker, Lori
AU - Wright, Caitlin
AU - Yousefi, Mehrnaz
AU - Alhander, Ann Christine
AU - Berglund, Lisa
AU - Ekstrom, Ann Berit
AU - Kroksmark, Anna Karin
AU - Sterky, Ulrika
AU - Birkmeier, Marissa
AU - Kaminski, Sarah
AU - Parker, Katie
AU - Carroll, Kate
AU - DeValle, Katy
AU - Kennedy, Rachel
AU - Villano, Dani
AU - Leve, Adina Bar
AU - Shurr, Itai
AU - Wisband, Elana
AU - Yaffe, Debbie
AU - Andreone, Luz
AU - Corderi, Jose
AU - Mesa, Lilia
AU - Levi, Lorena
AU - Coleman-Wood, Krista
AU - Hoffman, Ann
AU - Korn-Petersen, Wendy
AU - Selcen, Duygu
AU - Abdel-Hamid, Hoda
AU - Bise, Christopher
AU - Craig, Ann
AU - Hache, Lauren
AU - Hughes, Sarah
AU - Nguyen, Casey
AU - Weimer, Jason
AU - Golumbak, Paul
AU - Lopate, Glenn
AU - Malane, Justin
AU - Malkus, Betsy
AU - Nozaki, Kenkicki
AU - Renna, Renee
AU - Schierbacker, Jeanine
AU - Seiner, Catherine
AU - Wulf, Charlie
AU - Blair, Susan
AU - Grillo, Barbara
AU - Jones, Karen
AU - Monasterio, Eugenio
AU - Clift, Judy
AU - Feliciano, Cassandra
AU - Igarashi, Masanori
AU - Young, Rachel
AU - Rose, Kristy
AU - Webster, Richard
AU - Wicks, Stephanie
AU - Chen, Lucia
AU - Kennedy, Cameron
AU - Deliz, Brenda
AU - Espada, Sheila
AU - Fuste, Pura
AU - Luciano, Carlos
AU - Capone, Luca
AU - Beneggi, Maria
AU - Morettini, Valentina
AU - Gupta, Anjali
AU - McNeil, Robert
AU - Erickson, Amy
AU - Margolis, Marcia
AU - Naughton, Cameron
AU - Parry, Gareth
AU - Walk, David
AU - Bartley, Naomi
AU - Canelos, Paola
AU - Casper, Robert
AU - Hache, Lauren
AU - Ingram, Corinne
AU - Hu, Fengming
AU - Ahmed, Mohammad
AU - Zimmerman, Angela
PY - 2013/7
Y1 - 2013/7
N2 - 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.
AB - 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.
KW - Adolescent
KW - Adult
KW - Child/preschool
KW - Follow-up study
KW - Health status
KW - Human
KW - Locomotion
KW - Male
KW - Muscle strength/physiology
KW - Muscular dystrophies/classification
KW - Muscular dystrophies/Duchenne/physiopathology
KW - Muscular dystrophies/therapy
KW - Phenotype
KW - Quality of life/psychology
KW - Respiratory function test
UR - http://www.scopus.com/inward/record.url?scp=84879554058&partnerID=8YFLogxK
U2 - 10.1002/mus.23807
DO - 10.1002/mus.23807
M3 - Review Article
C2 - 23677550
VL - 48
SP - 32
EP - 54
JO - Muscle & Nerve
JF - Muscle & Nerve
SN - 0148-639X
IS - 1
ER -
