Corticosteroid treatment and growth patterns in ambulatory males with duchenne muscular dystrophy

Journal of Pediatrics

Volumn 173, Issue , 2016, Pages 207-213.e3

  Lamb, Molly M ^a   West, Nancy A ^a,b   Ouyang, Lijing ^c   Yang, Michele ^d   Weitzenkamp, David ^d   James, Katherine ^a   Ciafaloni, Emma ^e   Pandya, Shree ^e   Diguiseppi, Carolyn ^a   Cunniff, Chris ^f   Meaney, John ^f   Andrews, Jennifer ^f   Pettit, Kathleen ^f   Pettygrove, Sydney ^f   Miller, Lisa ^f   Matthews, Dennis ^f   Montgomery, April ^f   Donnelly, Jennifer ^f   Bolen, Julie ^f   Street, Natalie ^f   Lyles, Bobby ^f   Mann, Sylvia ^f   Romitti, Paul ^f   Mathews, Katherine ^f   Caspers Conway, Kristin ^f   Puzhankara, Soman ^f   Foo, Florence ^f   Westfield, Christina ^f   Druschel, Charlotte ^f   Campbell, Kim ^f   Fox, Deborah ^f   more..

^a Food Science and Human Nutrition   (United States)

^b UNIVERSITY OF MISSISSIPPI MEDICAL CENTER   (United States)

^c CENTERS FOR DISEASE CONTROL AND PREVENTION   (United States)

^d CHILDREN S HOSPITAL COLORADO   (United States)

^e UNIVERSITY OF ROCHESTER   (United States)

^f NONE

Author keywords

BMI; height; weight

Indexed keywords

DEFLAZACORT; PREDNISONE; GLUCOCORTICOID; PREGNANE DERIVATIVE;

ARTICLE; BODY MASS; CHILD; COMPARATIVE STUDY; CORTICOSTEROID THERAPY; DEMOGRAPHY; DUCHENNE MUSCULAR DYSTROPHY; ETHNICITY; GROWTH CURVE; HUMAN; MAJOR CLINICAL STUDY; MALE; MOBILIZATION; PRIORITY JOURNAL; SHORT STATURE; TREATMENT DURATION; UNITED STATES; ADOLESCENT; BODY HEIGHT; BODY WEIGHT; CASE CONTROL STUDY; DOSE RESPONSE; DRUG EFFECTS; MUSCULAR DYSTROPHY, DUCHENNE; PRESCHOOL CHILD;

ADOLESCENT; BODY HEIGHT; BODY MASS INDEX; BODY WEIGHT; CASE-CONTROL STUDIES; CHILD; CHILD, PRESCHOOL; DOSE-RESPONSE RELATIONSHIP, DRUG; GLUCOCORTICOIDS; HUMANS; MALE; MUSCULAR DYSTROPHY, DUCHENNE; PREDNISONE; PREGNENEDIONES;

EID: 84962094373     PISSN: 00223476     EISSN: 10976833     Source Type: Journal    
DOI: 10.1016/j.jpeds.2016.02.067     Document Type: Article

References (47)

1. B. Balaban, D.J. Matthews, G.H. Clayton, and T. Carry Corticosteroid treatment and functional improvement in Duchenne muscular dystrophy: long-term effect Am J Phys Med Rehabil 84 2005 843 850
2. E.A. Beenakker, J.M. Fock, M.J. Van Tol, N.M. Maurits, H.M. Koopman, O.F. Brouwer, and et al. Intermittent prednisone therapy in Duchenne muscular dystrophy: a randomized controlled trial Arch Neurol 62 2005 128 132
3. W.D. Biggar, V.A. Harris, L. Eliasoph, and B. Alman Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade Neuromuscul Disord 16 2006 249 255
4. E.K. Henricson, R.T. Abresch, A. Cnaan, F. Hu, T. Duong, A. Arrieta, and et al. The cooperative international neuromuscular research group Duchenne natural history study: glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures Muscle Nerve 48 2013 55 67
5. C. Angelini, E. Pegoraro, E. Turella, M.T. Intino, A. Pini, and C. Costa Deflazacort in Duchenne dystrophy: study of long-term effect Muscle Nerve 17 1994 386 391
6. G.M. Fenichel, J.M. Florence, A. Pestronk, J.R. Mendell, R.T. Moxley III, R.C. Griggs, and et al. Long-term benefit from prednisone therapy in Duchenne muscular dystrophy Neurology 41 1991 1874 1877
7. J.R. Mendell, R.T. Moxley, R.C. Griggs, M.H. Brooke, G.M. Fenichel, J.P. Miller, and et al. Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy N Engl J Med 320 1989 1592 1597
8. M.M. Rahman, M.A. Hannan, B.A. Mondol, N.B. Bhoumick, and A. Haque Prednisolone in Duchenne muscular dystrophy Bangladesh Med Res Counc Bull 27 2001 38 42
9. S. DeSilva, D.B. Drachman, D. Mellits, and R.W. Kuncl Prednisone treatment in Duchenne muscular dystrophy. Long-term benefit Arch Neurol 44 1987 818 822
10. S. Houde, M. Filiatrault, A. Fournier, J. Dube, S. D'Arcy, D. Berube, and et al. Deflazacort use in Duchenne muscular dystrophy: an 8-year follow-up Pediatr Neurol 38 2008 200 206
11. W.M. King, R. Ruttencutter, H.N. Nagaraja, V. Matkovic, J. Landoll, C. Hoyle, and et al. Orthopedic outcomes of long-term daily corticosteroid treatment in Duchenne muscular dystrophy Neurology 68 2007 1607 1613
12. S. Pradhan, D. Ghosh, N.K. Srivastava, A. Kumar, B. Mittal, C.M. Pandey, and et al. Prednisolone in Duchenne muscular dystrophy with imminent loss of ambulation J Neurol 253 2006 1309 1316
13. O. Yilmaz, A. Karaduman, and H. Topaloglu Prednisolone therapy in Duchenne muscular dystrophy prolongs ambulation and prevents scoliosis Eur J Neurol 11 2004 541 544
14. B.A. Alman, S.N. Raza, and W.D. Biggar Steroid treatment and the development of scoliosis in males with Duchenne muscular dystrophy J Bone Joint Surg Am 86-A 2004 519 524
15. D.E. Lebel, J.A. Corston, L.C. McAdam, W.D. Biggar, and B.A. Alman Glucocorticoid treatment for the prevention of scoliosis in children with Duchenne muscular dystrophy: long-term follow-up J Bone Joint Surg Am 95 2013 1057 1061
16. C.K. Silversides, G.D. Webb, V.A. Harris, and D.W. Biggar Effects of deflazacort on left ventricular function in patients with Duchenne muscular dystrophy Am J Cardiol 91 2003 769 772
17. G. Schram, A. Fournier, H. Leduc, N. Dahdah, J. Therien, M. Vanasse, and et al. All-cause mortality and cardiovascular outcomes with prophylactic steroid therapy in Duchenne muscular dystrophy J Am Coll Cardiol 61 2013 948 954
18. L.W. Markham, K. Kinnett, B.L. Wong, D. Woodrow Benson, and L.H. Cripe Corticosteroid treatment retards development of ventricular dysfunction in Duchenne muscular dystrophy Neuromuscul Disord 18 2008 365 370
19. K. Bushby, R. Finkel, D.J. Birnkrant, L.E. Case, P.R. Clemens, L. Cripe, and et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management Lancet Neurol 9 2010 77 93
20. R.C. Griggs, R.T. Moxley III, J.R. Mendell, G.M. Fenichel, M.H. Brooke, A. Pestronk, and et al. Duchenne dystrophy: randomized, controlled trial of prednisone (18 months) and azathioprine (12 months) Neurology 43 1993 520 527
21. R.T. Moxley III, S. Ashwal, S. Pandya, A. Connolly, J. Florence, K. Mathews, and 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 64 2005 13 20
22. D.J. Matthews, K.A. James, L.A. Miller, S. Pandya, K.A. Campbell, E. Ciafaloni, and et al. Use of corticocorticosteroids in a population-based cohort of boys with Duchenne and Becker muscular dystrophy J Child Neurol 25 2010 1319 1324
23. L.C. McAdam, A.L. Mayo, B.A. Alman, and W.D. Biggar The Canadian experience with long-term deflazacort treatment in Duchenne muscular dystrophy Acta Myol 31 2012 16 20
24. B.H. Nagel, W. Mortier, M. Elmlinger, H.A. Wollmann, K. Schmitt, and M.B. Ranke Short stature in Duchenne muscular dystrophy: a study of 34 patients Acta Paediatr 88 1999 62 65
25. N.A. West, M.L. Yang, D.A. Weitzenkamp, J. Andrews, F.J. Meaney, J. Oleszek, and et al. Patterns of growth in ambulatory males with Duchenne muscular dystrophy J Pediatr 163 2013 1759 1763.e1
26. R.J. Kuczmarski, C.L. Ogden, S.S. Guo, L.M. Grummer-Strawn, K.M. Flegal, Z. Mei, and et al. 2000 CDC Growth Charts for the United States: methods and development Vital Health Stat 11 246 2002 1 190
27. L.A. Miller, P.A. Romitti, C. Cunniff, C. Druschel, K.D. Mathews, F.J. Meaney, and et al. The muscular Dystrophy Surveillance Tracking and Research Network (MD STARnet): surveillance methodology Birth Defects Res Part A Clin Mol Teratol 76 2006 793 797
28. K.D. Mathews, C. Cunniff, J.R. Kantamneni, E. Ciafaloni, T. Miller, D. Matthews, and et al. Muscular Dystrophy Surveillance Tracking and Research Network (MD STARnet): case definition in surveillance for childhood-onset Duchenne/Becker muscular dystrophy J Child Neurol 25 2010 1098 1102
29. M. Bodor, and C.M. McDonald Why short stature is beneficial in Duchenne muscular dystrophy Muscle Nerve 48 2013 336 342
30. M. Zatz, D. Rapaport, M. Vainzof, J.M. Rocha, C. Pavanello Rde, G.M. Colletto, and et al. Relation between height and clinical course in Duchenne muscular dystrophy Am J Med Genet 29 1988 405 410
31. W.D. Biggar, M. Gingras, D.L. Fehlings, V.A. Harris, and C.A. Steele Deflazacort treatment of Duchenne muscular dystrophy J Pediatr 138 2001 45 50
32. Z.E. Davidson, M.M. Ryan, A.J. Kornberg, K. Sinclair, A. Cairns, K.Z. Walker, and et al. Observations of body mass index in Duchenne muscular dystrophy: a longitudinal study Eur J Clin Nutr 68 2014 892 897
33. N. Goemans, and G. Buyse Current treatment and management of dystrophinopathies Curr Treat Options Neurol 16 2014 287
34. R.C. Griggs, R.T. Moxley III, J.R. Mendell, G.M. Fenichel, M.H. Brooke, A. Pestronk, and 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 48 1991 383 388
35. D.M. Escolar, L.P. Hache, P.R. Clemens, A. Cnaan, C.M. McDonald, V. Viswanathan, and et al. Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy Neurology 77 2011 444 452
36. G.M. Fenichel, J.R. Mendell, R.T. Moxley III, R.C. Griggs, M.H. Brooke, J.P. Miller, and et al. A comparison of daily and alternate-day prednisone therapy in the treatment of Duchenne muscular dystrophy Arch Neurol 48 1991 575 579
37. R.C. Griggs, B.E. Herr, A. Reha, G. Elfring, L. Atkinson, V. Cwik, and et al. Corticocorticosteroids in Duchenne muscular dystrophy: major variations in practice Muscle Nerve 48 2013 27 31
38. V. Ricotti, D.A. Ridout, E. Scott, R. Quinlivan, S.A. Robb, A.Y. Manzur, and et al. Long-term benefits and adverse effects of intermittent versus daily glucocorticoids in boys with Duchenne muscular dystrophy J Neurol Neurosurg Psychiatry 84 2013 698 705
39. C.S. Straathof, W.C. Overweg-Plandsoen, G.J. van den Burg, A.J. van der Kooi, J.J. Verschuuren, and I.J. de Groot Prednisone 10 days on/10 days off in patients with Duchenne muscular dystrophy J Neurol 256 2009 768 773
40. Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR-DMD). http://www.ninds.nih.gov/disorders/clinical-trials/NCT01603407.htm. Accessed July 23, 2015.
41. M.D. Bonifati, G. Ruzza, P. Bonometto, A. Berardinelli, K. Gorni, S. Orcesi, and et al. A multicenter, double-blind, randomized trial of deflazacort versus prednisone in Duchenne muscular dystrophy Muscle Nerve 23 2000 1344 1347
42. A.M. Connolly, J. Schierbecker, R. Renna, and J. Florence High dose weekly oral prednisone improves strength in boys with Duchenne muscular dystrophy Neuromuscul Disord 12 2002 917 925
43. V. Dubowitz, M. Kinali, M. Main, E. Mercuri, and F. Muntoni Remission of clinical signs in early Duchenne muscular dystrophy on intermittent low-dosage prednisolone therapy Eur J Paediatr Neurol 6 2002 153 159
44. R.T. Moxley III, S. Pandya, E. Ciafaloni, D.J. Fox, and K. Campbell Change in natural history of Duchenne muscular dystrophy with long-term corticosteroid treatment: implications for management J Child Neurol 25 2010 1116 1129
45. L. Merlini, A. Cicognani, E. Malaspina, M. Gennari, S. Gnudi, B. Talim, and et al. Early prednisone treatment in Duchenne muscular dystrophy Muscle Nerve 27 2003 222 227
46. C.L. Ogden, M.D. Carroll, and K.M. Flegal High body mass index for age among US children and adolescents, 2003-2006 J Am Med Assoc 299 2008 2401 2405
47. S. Kim, K.A. Campbell, D.J. Fox, D.J. Matthews, and R. Valdez Corticosteroid treatments in males with Duchenne muscular dystrophy: treatment duration and time to loss of ambulation J Child Neurol 30 2015 1275 1280