Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy

Neurology

Volumn 77, Issue 5, 2011, Pages 444-452

  Escolar, D M ^a   Hache, L P ^b   Clemens, P R ^b,c   Cnaan, A ^a   McDonald, C M ^d   Viswanathan, V ^e   Kornberg, A J ^f   Bertorini, T E ^g   Nevo, Y ^h   Lotze, T ⁱ   Pestronk, A ^j   Ryan, M M ^k   Monasterio, E ^l   Day, J W ^m   Zimmerman, A ^a   Arrieta, A ⁿ   Henricson, E ^a   Mayhew, J ^l   Florence, J ^j   Hu, F ^a   Connolly, A M ^j   more..

^a CHILDREN'S NATIONAL MEDICAL CENTER   (United States)

^b UNIVERSITY OF PITTSBURGH   (United States)

^c VETERANS AFFAIRS MEDICAL CENTER   (United States)

^d UNIVERSITY OF CALIFORNIA   (United States)

^e KANCHI KAMAKOTI CHILDS TRUST HOSPITAL   (India)

^f ROYAL CHILDREN'S HOSPITAL   (Australia)

^g UNIVERSITY OF TENNESSEE HEALTH SCIENCE CENTER   (United States)

^h HADASSAH HEBREW UNIVERSITY MEDICAL CENTER   (Israel)

ⁱ TEXAS CHILDREN S HOSPITAL   (United States)

^j WASHINGTON UNIVERSITY   (United States)

^k CHILDREN S HOSPITAL AT WESTMEAD   (Australia)

^l Children's Hospital   (United States)

^m UNIVERSITY OF MINNESOTA   (United States)

ⁿ NONE

more..

Author keywords

[No Author keywords available]

Indexed keywords

CALCIUM; PLACEBO; PREDNISONE; RANITIDINE; TUMS; VITAMIN D;

ACUTE APPENDICITIS; ARTICLE; CHILD; CONTROLLED STUDY; CORTICOSTEROID THERAPY; DOUBLE BLIND PROCEDURE; DRUG DOSE COMPARISON; DRUG EFFICACY; DRUG FEVER; DRUG SAFETY; DRUG SCREENING; DRUG WITHDRAWAL; DUCHENNE MUSCULAR DYSTROPHY; HUMAN; INFLUENZA; LACERATION; LIPID DIET; MAJOR CLINICAL STUDY; MALE; MULTICENTER STUDY; OUTCOME ASSESSMENT; PRESCHOOL CHILD; PRIORITY JOURNAL; PROTEIN DIET; RANDOMIZED CONTROLLED TRIAL; SCHOOL CHILD; SCORING SYSTEM; SKIN DISEASE; VOMITING; WEAKNESS;

EID: 80051500586     PISSN: 00283878     EISSN: 1526632X     Source Type: Journal    
DOI: 10.1212/WNL.0b013e318227b164     Document Type: Article

References (40)

1. Emery AE. Population frequencies of inherited neuromuscular diseases: a world survey. Neuromuscul Disord 1991;1:19-29.
2. Tuffery S, Chambert S, Bareil C, et al. Mutation analysis of the dystrophin gene in Southern French DMD or BMD families: from Southern blot to protein truncation test. Hum Genet 1998;102:334-342. (Pubitemid 28185225)
3. Escolar DM, Buyse G, Henricson E, et al. CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy. Ann Neurol 2005;58:151-155. (Pubitemid 40962472)
4. Fenichel G, Pestronk A, Florence J, Robison V, Hemelt V. A beneficial effect of oxandrolone in the treatment of Duchenne muscular dystrophy: a pilot study. Neurology 1997;48:1225-1226. (Pubitemid 27240538)
5. Tarnopolsky MA, Mahoney DJ, Vajsar J, et al. Creatine monohydrate enhances strength and body composition in Duchenne muscular dystrophy. Neurology 2004;62:1771-1777. (Pubitemid 38661733)
6. Angelini C, Pegoraro E, Turella E, Intino MT, Pini A, Costa C. Deflazacort in Duchenne dystrophy: study of long-term effect. Muscle Nerve 1994;17:386-391. (Pubitemid 24096533)
7. Biggar WD, Harris VA, Eliasoph L, Alman B. Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade. Neuromuscul Disord 2006;16:249-255.
8. Griggs RC, Moxley RT III, Mendell JR, et al. Prednisone in Duchenne dystrophy: a randomized, controlled trial defining the time course and dose response: Clinical Investigation of Duchenne Dystrophy Group. Arch Neurol 1991;48:383-388.
9. Griggs RC, Moxley RT III, Mendell JR, et al. Duchenne dystrophy: randomized, controlled trial of prednisone (18 months) and azathioprine (12 months). Neurology 1993; 43:520-527. (Pubitemid 23088485)
10. Manzur AY, Kuntzer T, Pike M, Swan A. Glucocorticoid corticosteroids for Duchenne muscular dystrophy. Cochrane Database Syst Rev 2008;CD003725.
11. Markham LW, Kinnett K, Wong BL, Woodrow BD, Cripe LH. Corticosteroid treatment retards development of ventricular dysfunction in Duchenne muscular dystrophy. Neuromuscul Disord 2008;18:365-370.
12. Mendell JR, Moxley RT, Griggs RC, et al. Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy. N Engl J Med 1989;320:1592-1597. (Pubitemid 19154573)
13. Moxley RT, III, Ashwal S, Pandya S, et al. Practice parameter: corticosteroid treatment of Duchenne dystrophy: report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society. Neurology 2005;64:13-20.
14. Bushby K, Finkel R, Birnkrant DJ, et al. Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care. Lancet Neurol 2010;9:177-189.
15. Bushby K, Finkel R, Birnkrant DJ, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol 2010;9:77-93.
16. Henricson E, McDonald C, Abresch RT, et al. A Cooperative International Neuromuscular Research Group (CINRG) study of the relationship between impairment, activity limitation, participation and quality of life in persons with confirmed dystrophinopathies: one year follow-up of skeletal muscle strength and timed motor performance. Neurology 2010;74:A110. Abstract.
17. McDonald C, Henricson E, Abresch RT, et al. Functional motor performance characteristics of boys with duchenne muscular dystrophy by age groups and steroid use: oneyear data from the CINRG longitudinal study project. Neurology 2010;74:A219. Abstract.
18. Connolly AM, Schierbecker J, Renna R, Florence J. High dose weekly oral prednisone improves strength in boys with Duchenne muscular dystrophy. Neuromuscul Disord 2002;12:917-925. (Pubitemid 35414370)
19. Escolar DM, Henricson EK, Mayhew J, et al. Clinical evaluator reliability for quantitative and manual muscle testing measures of strength in children. Muscle Nerve 2001;24:787-793. (Pubitemid 32476525)
20. Mayhew JE, Florence JM, Mayhew TP, et al. Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophy. Muscle Nerve 2007;35:36-42. (Pubitemid 46052646)
21. Vignos PJ Jr, Spencer GE Jr, Archibald KC. Management of progressive muscular dystrophy in childhood. JAMA 1963;184:89-96.
22. Brooke MH, Griggs RC, Mendell JR, Fenichel GM, Shumate JB, Pellegrino RJ. Clinical trial in Duchenne dystrophy: I: the design of the protocol. Muscle Nerve 1981; 4:186-197. (Pubitemid 11098499)
23. Achenbach TM, Rescorla LA. Manual for ASEBA School-Age Forms and Profiles. Burlington, VT: University of Vermont, Research Center for Children, Youth and Families; 2001.
24. Fitzmaurice G, Davidian M, Verbeke G, Molenberghs G. Longitudinal Data Analysis. Boca Raton, FL: Chapman &Hall/CRC Press; 2009.
25. Cancer Therapy Evaluation Program, Common Terminology Criteria for Adverse Events, Version 3.0, DCTD, NCI, NIH, DHHS. March 31, 2003. Available at: http:// ctep.cancer.gov. Publication date: August 9, 2006.
26. SAS. Cary, NC: SAS Institute Inc.; 2009.
27. EquivTest/PK test. Saugus, MA: Statistical Solutions Ltd.; 2009.
28. Brooke MH, Fenichel GM, Griggs RC, et al. Clinical investigation of Duchenne muscular dystrophy: interesting results in a trial of prednisone. Arch Neurol 1987;44:812-817.
29. Fenichel GM, Mendell JR, Moxley RT III, et al. A comparison of daily and alternate-day prednisone therapy in the treatment of Duchenne muscular dystrophy. Arch Neurol 1991;48:575-579.
30. Beenakker EA, Fock JM, Van Tol MJ, et al. Intermittent prednisone therapy in Duchenne muscular dystrophy: a randomized controlled trial. Arch Neurol 2005;62:128-132. (Pubitemid 40105045)
31. Sansome A, Royston P, Dubowitz V. Steroids in Duchenne muscular dystrophy: pilot study of a new low-dosage schedule. Neuromuscul Disord 1993;3:567-569. (Pubitemid 24050839)
32. Bianchi ML, Mazzanti A, Galbiati E, et al. Bone mineral density and bone metabolism in Duchenne muscular dystrophy. Osteoporos Int 2003;14:761-767. (Pubitemid 37295839)
33. Mei Z, Grummer-Strawn LM, Pietrobelli A, Goulding A, Goran MI, Dietz WH. Validity of body mass index compared with other body-composition screening indexes for the assessment of body fatness in children and adolescents. Am J Clin Nutr 2002;75:978-985. (Pubitemid 34547525)
34. Rapaport D, Colletto GM, Vainzof M, Duaik MC, Zatz M. Short stature in Duchenne muscular dystrophy. Growth Regul 1991;1:11-15.
35. McDonald CM, Abresch RT, Carter GT, et al. Profiles of neuromuscular diseases: Duchenne muscular dystrophy. Am J Phys Med Rehabil 1995;74:S70-S92.
36. Bachrach LK. Taking steps towards reducing osteoporosis in Duchenne muscular dystrophy. Neuromuscul Disord 2005;15:86-87. (Pubitemid 41556638)
37. Aparicio LF, Jurkovic M, DeLullo J. Decreased bone density in ambulatory patients with Duchenne muscular dystrophy. J Pediatr Orthop 2002;22:179-181. (Pubitemid 34297676)
38. Douvillez B, Braillon P, Hodgkinson I, Berard C. Pain, osteopenia and body composition of 22 patients with Duchenne muscular dystrophy: a descriptive study. Ann Readapt Med Phys 2005;48:616-622. (Pubitemid 41526969)
39. Larson CM, Henderson RC. Bone mineral density and fractures in boys with Duchenne muscular dystrophy. J Pediatr Orthop 2000;20:71-74. (Pubitemid 30013402)
40. Palmieri GM, Bertorini TE, Griffin JW, Igarashi M, Karas JG. Assessment of whole body composition with dual energy x-ray absorptiometry in Duchenne muscular dystrophy: correlation of lean body mass with muscle function. Muscle Nerve 1996;19:777-779. (Pubitemid 26134591)