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

the CINRG Investigators

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73 Citations (Scopus)

Abstract

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.

Original languageEnglish (US)
Pages (from-to)32-54
Number of pages23
JournalMuscle and Nerve
Volume48
Issue number1
DOIs
StatePublished - Jul 1 2013

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Duchenne Muscular Dystrophy
Natural History
Glucocorticoids
Longitudinal Studies
Research
Muscle Strength
Therapeutics
Walking
Observational Studies
Quality of Life
Research Personnel
Clinical Trials
Muscles
Lung

Keywords

  • Adolescent
  • Adult
  • Child/preschool
  • Follow-up study
  • Health status
  • Human
  • Locomotion
  • Male
  • Muscle strength/physiology
  • Muscular dystrophies/Duchenne/physiopathology
  • Muscular dystrophies/classification
  • Muscular dystrophies/therapy
  • Phenotype
  • Quality of life/psychology
  • Respiratory function test

Cite this

@article{d9a6227130534da6ac676d3991fa42f0,
title = "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",
abstract = "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.",
keywords = "Adolescent, Adult, Child/preschool, Follow-up study, Health status, Human, Locomotion, Male, Muscle strength/physiology, Muscular dystrophies/Duchenne/physiopathology, Muscular dystrophies/classification, Muscular dystrophies/therapy, Phenotype, Quality of life/psychology, Respiratory function test",
author = "{the CINRG Investigators} and McDonald, {Craig M.} and Henricson, {Erik K.} and Abresch, {R. Ted} and Han, {Jay J.} and Escolar, {Diana M.} and Florence, {Julaine M.} and Tina Duong and Adrienne Arrieta and Clemens, {Paula R.} and Hoffman, {Eric P.} and Avital Cnaan and V. Vishwanathan and S. Chidambaranathan and Biggar, {W. Douglas} and Mah, {Jean K.} and Mar Tulinius and Robert Leshner and Carolina Tesi-Rocha and Andrew Korn-berg and Monique Ryan and Yoram Nevo and Alberto Dubrovsky and Nancy Kuntz and Sherilyn Driscoll and Anne Connolly and Alan Pestronk and Jean Teasley and Tulio Bertorini and Kathryn North and Hanna Kolski and Jose Carlo and Ksenija Gorni and Timothy Lotze and John Day and Josh Benditt and Louis Boitano and David Birnkrant and David Connuck and Jonathan Finder and Veronica Hinton and Katherine Mathews and Richard Moxley and Susan Sparks and Erynn Gordon and Michelle Cregan and Erica Goude and Merete Glick and Linda Johnson and Nanette Joyce and David Walk",
year = "2013",
month = "7",
day = "1",
doi = "10.1002/mus.23807",
language = "English (US)",
volume = "48",
pages = "32--54",
journal = "Muscle and Nerve",
issn = "0148-639X",
publisher = "John Wiley and Sons Inc.",
number = "1",

}

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 - the CINRG Investigators

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 - Vishwanathan, V.

AU - Chidambaranathan, S.

AU - Biggar, W. Douglas

AU - Mah, Jean K.

AU - Tulinius, Mar

AU - Leshner, Robert

AU - Tesi-Rocha, Carolina

AU - Korn-berg, 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 - Walk, David

PY - 2013/7/1

Y1 - 2013/7/1

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/Duchenne/physiopathology

KW - Muscular dystrophies/classification

KW - Muscular dystrophies/therapy

KW - Phenotype

KW - Quality of life/psychology

KW - Respiratory function test

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U2 - 10.1002/mus.23807

DO - 10.1002/mus.23807

M3 - Article

C2 - 23677550

AN - SCOPUS:84879554058

VL - 48

SP - 32

EP - 54

JO - Muscle and Nerve

JF - Muscle and Nerve

SN - 0148-639X

IS - 1

ER -