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Duchenne Muscular Dystrophy: Canadian Paediatric Neuromuscular Physicians Survey

Published online by Cambridge University Press:  02 December 2014

Hugh J. McMillan ,
Craig Campbell  and
Jean K. Mah
Hugh J. McMillan
Affiliation:
Division of Neurology, Department of Paediatrics, Children's Hospital of Eastern Ontario, Ottawa
Craig Campbell
Affiliation:
Departments of Paediatrics, Clinical Neurological Sciences and Epidemiology, University of Western Ontario, London, Ontario
Jean K. Mah*
Affiliation:
Division of Paediatric Neurology, Department of Paediatrics and Clinical Neurosciences, University of Calgary, Calgary, Alberta, Canada
*
Alberta Children's Hospital, 2888 Shaganappi Trail NW, Calgary, Alberta, T3B 6A8, Canada.
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Abstract

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Background:

Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy in childhood.

Method:

To assess the current care of paediatric DMD patients in Canada, a questionnaire was mailed to 17 physicians who were members of the Canadian paediatric neuromuscular group. Areas of enquiry included; 1) multidisciplinary team composition; 2) means of DMD diagnosis; 3) corticosteroid use; surveillance and management for: 4) orthopaedic, 5) respiratory and 6) cardiac complications and 7) health maintenance (nutrition & immunizations).

Results:

Completed surveys were returned by 14/17 (82%) of physicians. Twelve respondents followed DMD patients. All centres had multidisciplinary teams, including respirology (11/12), child neurology or physiatry (11), physiotherapy (9), occupational therapy (9) and orthopaedic surgery (7). Deflazacort 0.9mg/kg/d was used at all centres, which was continued after loss of independent ambulation (11), along with routine calcium and vitamin D supplementation (10). Night splints were prescribed at all centres. Routine surveillance studies included pulmonary function testing (11), sleep studies (10), EKG/echocardiogram (10), bone density (DEXA) scans (10), spine radiography (9), and dietician referral (4).

Conclusion:

Paediatric DMD patients are receiving relatively consistent care in multidisciplinary clinics across Canada, in accordance with recommended guidelines for DMD.

Type
Research Article
Information
Canadian Journal of Neurological Sciences , Volume 37 , Issue 2 , March 2010 , pp. 195 - 205
Copyright
Copyright © The Canadian Journal of Neurological 2010

References

1. Mendell, JR, Moxley, RT, Griggs, RC, Brooke, MH, Fenichel, GM, Miller, JP, et al. Randomized, double-blind six-month trial of prednisone in Duchenne’s muscular dystrophy. N Engl J Med. 1989;320(24):1592–7.CrossRefGoogle ScholarPubMed
2. Griggs, RC, Moxley, RT 3rd, Mendell, JR, Fenichel, GM, Brooke, MH, Pestronk, A, 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(4):383–8.CrossRefGoogle ScholarPubMed
3. Fenichel, GM, Mendell, JR, Moxley, RT 3rd, Griggs, RC, Brooke, MH, Miller, JP, et al. A comparison of daily and alternate-day prednisone therapy in the treatment of Duchenne muscular dystrophy. Arch Neurol. 1991;48(6):575–9.CrossRefGoogle ScholarPubMed
4. Mesa, LE, Dubrovsky, AL, Corderi, J, Marco, P, Flores, D. Steroids in Duchenne muscular dystrophy-deflazacort trial. Neuromuscul Disord. 1991;1(4):261–6.CrossRefGoogle ScholarPubMed
5. Biggar, WD, Gingras, M, Fehlings, DL, Harris, VA, Steele, CA. Deflazacort treatment of Duchenne muscular dystrophy. J Pediatr. 2001;138(1):45–50.CrossRefGoogle ScholarPubMed
6. Schara, U, Mortier, , Mortier, W. Long-term steroid therapy in Duchenne muscular dystrophy-positive results versus side effects. J Clin Neuromuscul Dis. 2001;2(4):179–83.CrossRefGoogle ScholarPubMed
7. King, WM, Ruttencutter, R, Nagaraja, HN, Matkovic, V, Landoll, J, Hoyle, C, et al. Orthopedic outcomes of long-term daily corticosteroid treatment in Duchenne muscular dystrophy. Neurology. 2007;68(19):1607–13.CrossRefGoogle ScholarPubMed
8. DeSilva, S, Drachman, DB, Mellits, D, Kuncl, RW. Prednisone treatment in Duchenne muscular dystrophy. Long-term benefit. Arch Neurol. 1987;44(8):818–22.CrossRefGoogle ScholarPubMed
9. 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(4):386–91.CrossRefGoogle ScholarPubMed
10. Silversides, CK, Webb, GD, Harris, VA, Biggar, DW. Effects of deflazacort on left ventricular function in patients with Duchenne muscular dystrophy. Am J Cardiol. 2003;91(6): 769–72.CrossRefGoogle ScholarPubMed
11. Markham, LW, Spicer, RL, Khoury, PR, Wong, BL, Mathews, KD, Cripe, LH. Steroid therapy and cardiac function in Duchenne muscular dystrophy. Pediatr Cardiol. 2005;26(6):768–71.CrossRefGoogle ScholarPubMed
12. Markham, LW, Kinnett, K, Wong, BL, Woodrow Benson, D, Cripe, LH. Corticosteroid treatment retards development of ventricular dysfunction in Duchenne muscular dystrophy. Neuromuscul Disord. 2008;18(5):365–70.CrossRefGoogle ScholarPubMed
13. Alman, BA, Raza, SN, Biggar, WD. Steroid treatment and the development of scoliosis in males with Duchenne muscular dystrophy. J Bone Joint Surg Am. 2004;86-A(3):519–24.CrossRefGoogle Scholar
14. Kinali, M, Main, M, Eliahoo, J, Messina, S, Knight, RK, Lehovsky, J, et al. Predictive factors for the development of scoliosis in Duchenne muscular dystrophy. Eur J Paediatr Neurol. 2007;11 (3):160–6.CrossRefGoogle ScholarPubMed
15. Bonifati, MD, Ruzza, G, Bonometto, P, Berardinelli, A, Gorni, K, Orcesi, S, et al. A multicenter, double-blind, randomized trial of deflazacort versus prednisone in Duchenne muscular dystrophy. Muscle Nerve. 2000;23(9):1344–7.3.0.CO;2-F>CrossRefGoogle ScholarPubMed
16. Moxley, RT 3rd, Ashwal, S, Pandya, S, Connolly, A, Florence, J, Mathews, K, 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(1):13–20.CrossRefGoogle Scholar
17. American Thoracic Society. Respiratory care of the patient with Duchenne muscular dystrophy. Am J Crit Care Med. 2004;170: 456–65.CrossRefGoogle Scholar
18. American Academy of Pediatrics. Cardiovascular health supervision for individuals affected by Duchenne or Becker muscular dystrophy. Pediatrics. 2005;166(6):1569–73.Google Scholar
19. TREAT-NMD.eu [homepage on the Internet]. United Kingdom. [cited 2009 Aug 17]. Available from: http://www.treatnmd.eu/userfiles/file/general/TREAT-NMD_DMD_interim_recommendations.pdf Google Scholar
20. Bushby, K BJ, Bullock, R, Eagle, M, Gibson, M, Quinby, J. The Multidisciplinary management of Duchenne muscular dystrophy. Curr Pediatr. 2005;15:292–300.CrossRefGoogle Scholar
21. Muntoni, F, Bushby, K, van Ommen, G. 128th ENMC International workshop on ‘preclinical optimization and phase I/II clinical trials using antisense oligonucleotides in Duchenne Muscular Dystrophy’ 22-24 October 2004, Naarden, The Netherlands. Neuromuscul Disord. 2005;15(6):450–7.CrossRefGoogle ScholarPubMed
22. Griggs, RC, Moxley, RT 3rd, Mendell, JR, Fenichel, GM, Brooke, MH, Pestronk, A, et al. Duchenne dystrophy: randomized, controlled trial of prednisone (18 months) and azathioprine (12 months). Neurology. 1993;43(3 Pt 1):520–7.CrossRefGoogle ScholarPubMed
23. Backman, E, Henriksson, KG. Low-dose prednisolone treatment in Duchenne and Becker muscular dystrophy. Neuromuscul Disord. 1995;5(3):233–41.CrossRefGoogle ScholarPubMed
24. Rahman, MM, Hannan, MA, Mondol, BA, Bhoumick, NB, Haque, A. Prednisolone in Duchenne muscular dystrophy. Bangladesh Med Res Counc Bull. 2001;27(1):38–42.Google ScholarPubMed
25. Beenakker, EA, Fock, JM, Van Tol, MJ, Maurits, NM, Koopman, HM, Brouwer, OF, et al. Intermittent prednisone therapy in Duchenne muscular dystrophy: a randomized controlled trial. Arch Neurol. 2005;62(1):128–32.CrossRefGoogle ScholarPubMed
26. Reitter, B. Deflazacort vs. prednisone in Duchenne muscular dystrophy: trends of an ongoing study. Brain Dev 1995;17 Suppl: 39–43.CrossRefGoogle ScholarPubMed
27. Reitter, B. Motor performance of DMD boys treated with prednisone or deflazacort: interim results of a double-blind study [abstract]. Dev Med Child Neurol. 1995;37 Suppl 72:107.Google Scholar
28. Campbell, C, Jacob, P. Deflazacort for the treatment of Duchenne Dystrophy: a systematic review. BMC Neurol. 2003;3:7.CrossRefGoogle ScholarPubMed
29. Manzur, AY, Kuntzer, T, Pike, M, Swan, A. Glucocorticoid corticosteroids for Duchenne muscular dystrophy. Cochrane Database Syst Rev. 2008(1):CD003725.CrossRefGoogle ScholarPubMed
30. Merlini, L, Cicognani, A, Malaspina, E, Gennari, M, Gnudi, S, Talim, B, et al. Early prednisone treatment in Duchenne muscular dystrophy. Muscle Nerve. 2003;27(2):222–7.CrossRefGoogle ScholarPubMed
31. Kinali, M, Mercuri, E, Main, M, Muntoni, F, Dubowitz, V. An effective, low-dosage, intermittent schedule of prednisolone in the long-term treatment of early cases of Duchenne dystrophy. Neuromuscul Disord. 2002;12 Suppl 1:S169–74.CrossRefGoogle ScholarPubMed
32. Bachrach, LK. Taking steps towards reducing osteoporosis in Duchenne muscular dystrophy. Neuromuscul Disord. 2005;15 (1):86–7.CrossRefGoogle ScholarPubMed
33. Biggar, WD, Bachrach, LK, Henderson, RC, Kalkwarf, H, Plotkin, H, Wong, BL. Bone health in Duchenne muscular dystrophy: a workshop report from the meeting in Cincinnati, Ohio, July 8, 2004. Neuromuscul Disord. 2005;15(1):80–5.CrossRefGoogle Scholar
34. Tarnopolsky, M, Mahoney, D, Thompson, T, Naylor, H, Doherty, TJ. Creatine monohydrate supplementation does not increase muscle strength, lean body mass, or muscle phosphocreatine in patients with myotonic dystrophy type 1. Muscle Nerve. 2004;29(1): 51–8.CrossRefGoogle ScholarPubMed
35. Fowler, EG, Graves, MC, Wetzel, GT, Spencer, MJ. Pilot trial of albuterol in Duchenne and Becker muscular dystrophy. Neurology. 2004;62(6):1006–8.CrossRefGoogle ScholarPubMed
36. Skura, CL, Fowler, EG, Wetzel, GT, Graves, M, Spencer, MJ. Albuterol increases lean body mass in ambulatory boys with Duchenne or Becker muscular dystrophy. Neurology. 2008;70 (2):137–43.CrossRefGoogle ScholarPubMed
37. Sharma, KR, Mynhier, MA, Miller, RG. Cyclosporine increases muscular force generation in Duchenne muscular dystrophy. Neurology. 1993;43(3 Pt 1):527–32.CrossRefGoogle ScholarPubMed
38. Fenichel, GM, Griggs, RC, Kissel, J, Kramer, TI, Mendell, JR, Moxley, RT, et al. A randomized efficacy and safety trial of oxandrolone in the treatment of Duchenne dystrophy. Neurology. 2001;56(8):1075–9.CrossRefGoogle ScholarPubMed
39. Stern, LM, Fewings, JD, Bretag, AH, Ballard, FJ, Tomas, FM, Cooper, DM, et al. The progression of Duchenne muscular dystrophy: clinical trial of allopurinol therapy. Neurology. 1981;31(4): 422–6.CrossRefGoogle ScholarPubMed
40. Mok, E, Letellier, G, Cuisset, JM, Denjean, A, Gottrand, F, Alberti, C, et al. Lack of functional benefit with glutamine versus placebo in Duchenne muscular dystrophy: a randomized crossover trial. PLoS One. 2009;4(5):e5448.CrossRefGoogle ScholarPubMed
41. Gamstorp, I, Gustavson, KH, Hellstrom, O, Nordgren, B. A trial of selenium and vitamin E in boys with muscular dystrophy. J Child Neurol. 1986;1(3):211–4.CrossRefGoogle ScholarPubMed
42. Smith, AD, Koreska, J, Moseley, CF. Progression of scoliosis in Duchenne muscular dystrophy. J Bone Joint Surg Am. 1989;71 (7):1066–74.CrossRefGoogle ScholarPubMed
43. Miller, F, Moseley, CF, Koreska, J. Spinal fusion in Duchenne muscular dystrophy. Dev Med Child Neurol. 1992;34(9):775–86.CrossRefGoogle ScholarPubMed
44. Kurz, LT, Mubarak, SJ, Schultz, P, Park, SM, Leach, J. Correlation of scoliosis and pulmonary function in Duchenne muscular dystrophy. J Pediatr Orthop. 1983;3(3):347–53.CrossRefGoogle ScholarPubMed
45. Galasko, CS, Delaney, C, Morris, P. Spinal stabilisation in Duchenne muscular dystrophy. J Bone Joint Surg Br. 1992;74(2):210–4.CrossRefGoogle ScholarPubMed
46. Miller, RG, Chalmers, AC, Dao, H, Filler-Katz, A, Holman, D, Bost, F. The effect of spine fusion on respiratory function in Duchenne muscular dystrophy. Neurology. 1991;41(1):38–40.CrossRefGoogle ScholarPubMed
47. Lankester, BJA WM, Gargan, MF. Duchenne muscular dystrophy. Curr Orthopaed. 2007;21:298–300.CrossRefGoogle Scholar
48. Shapiro, F, Sethna, N, Colan, S, Wohl, ME, Specht, L. Spinal fusion in Duchenne muscular dystrophy: a multidisciplinary approach. Muscle Nerve. 1992;15(5):604–14.CrossRefGoogle ScholarPubMed
49. Marsh, A, Edge, G, Lehovsky, J. Spinal fusion in patients with Duchenne’s muscular dystrophy and a low forced vital capacity. Eur Spine J. 2003;12(5):507–12.CrossRefGoogle Scholar
50. Bushby, K, Muntoni, F, Bourke, JP. 107th ENMC international workshop: the management of cardiac involvement in muscular dystrophy and myotonic dystrophy. 7th-9th June 2002, Naarden, the Netherlands. Neuromuscul Disord. 2003;13(2):166–72.CrossRefGoogle ScholarPubMed
51. Eagle, M, Baudouin, SV, Chandler, C, Giddings, DR, Bullock, R, Bushby, K. Survival in Duchenne muscular dystrophy: improvements in life expectancy since 1967 and the impact of home nocturnal ventilation. Neuromuscul Disord. 2002;12(10): 926–9.CrossRefGoogle ScholarPubMed
52. Nigro, G, Comi, LI, Politano, L, Bain, RJ. The incidence and evolution of cardiomyopathy in Duchenne muscular dystrophy. Int J Cardiol. 1990;26(3):271–7.CrossRefGoogle ScholarPubMed
53. Duboc, D, Meune, C, Lerebours, G, Devaux, JY, Vaksmann, G, Becane, HM. Effect of perindopril on the onset and progression of left ventricular dysfunction in Duchenne muscular dystrophy. J Am Coll Cardiol. 2005;45(6):855–7.CrossRefGoogle ScholarPubMed
54. Ramaciotti, C, Heistein, LC, Coursey, M, Lemler, MS, Eapen, RS, Iannaccone, ST, et al. Left ventricular function and response to enalapril in patients with Duchenne muscular dystrophy during the second decade of life. Am J Cardiol. 2006;98(6):825–7.CrossRefGoogle ScholarPubMed
55. Ishikawa, Y, Bach, JR, Minami, R. Cardioprotection for Duchenne’s muscular dystrophy. Am Heart J. 1999;137(5):895–902.CrossRefGoogle ScholarPubMed
56. Ogata, H, Ishikawa, Y, Ishikawa, Y, Minami, R. Beneficial effects of beta-blockers and angiotensin-converting enzyme inhibitors in Duchenne muscular dystrophy. J Cardiol. 2009;53(1):72–8.CrossRefGoogle ScholarPubMed
57. Garg, R, Yusuf, S. Overview of randomized trials of angiotensinconverting enzyme inhibitors on mortality and morbidity in patients with heart failure. Collaborative Group on ACE Inhibitor Trials. JAMA. 1995;273(18):1450–6.CrossRefGoogle Scholar
58. Gross, R, Johnston, K. Levels of evidence: taking neurology to the next level. Neurology. 2009;72:8–10.CrossRefGoogle Scholar
59. Reitter, B. 75th European neuromuscular centre international workshop: 2nd workshop on the treatment of muscular dystrophy, 10-12 December, 1999, Naarden, the Netherlands. Neuromusc Disord. 2000;10:313–20.Google Scholar
60. Corrado, G, Lissoni, A, Beretta, S, Terenghi, L, Tadeo, G, Foglia-Manzillo, G, et al. Prognostic value of electrocardiograms, ventricular late potentials, ventricular arrhythmias, and left ventricular systolic dysfunction in patients with Duchenne muscular dystrophy. Am J Cardiol. 2002;89(7):838–41.CrossRefGoogle ScholarPubMed
61. Mirabella, M, Servidei, S, Manfredi, G, Ricci, E, Frustaci, A, Bertini, E, et al. Cardiomyopathy may be the only clinical manifestation in female carriers of Duchenne muscular dystrophy. Neurology. 1993;43(11):2342–5.CrossRefGoogle ScholarPubMed
62. Hoogerwaard, EM, van der Wouw, PA, Wilde, AA, Bakker, E, Ippel, PF, Oosterwijk, JC, et al. Cardiac involvement in carriers of Duchenne and Becker muscular dystrophy. Neuromuscul Disord. 1999;9(5):347–51.CrossRefGoogle ScholarPubMed
63. Willig, TN, Bach, JR, Venance, V, Navarro, J. Nutritional rehabilitation in neuromuscular disorders. Semin Neurol. 1995; 15(1):18–23.CrossRefGoogle ScholarPubMed