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Retrospective cohort study comparing the efficacy of prednisolone and deflazacort in children with muscular dystrophy

A 6 years’ experience in a South Indian teaching hospital

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Indian Journal of Orthopaedics Aims and scope Submit manuscript

Abstract

Background: Muscular dystrophies are inherited myogenic disorders characterized by progressive muscle wasting and weakness of variable distribution and severity. They are a heterogeneous group characterized by variable degree of skeletal and cardiac muscle involvement. The most common and the most severe form of muscular dystrophy is DMD. Currently, there is no curative treatment for muscular dystrophies. Several drugs have been studied to retard the progression of the muscle weakness. There is much controversy about steroid usage in muscular dystrophy with respect to regimen, adverse effects, and whether long term benefits outweigh side effects. This study is to assess steroid efficacy in children with muscular dystrophy.

Materials and Methods: All children with diagnosed muscular dystrophy by muscle biopsy, immunohistochemistry and/or genetic test were enrolled in the study. They were started on either prednisolone (0.75 mg/kg/day) or deflazacort 0.9 mg/kg/day based on afford ability. All were followed up every 6 months with clinical assessment, quality of life questionnaire and clinical and laboratory assessment of side effects. Outcome measures of children on deflazacort and prednisolone at 1 year followup were summarized as numbers and percentages and were compared using Fisher’s exact test.

Results: Twenty two children with muscular dystrophy were included (prednisolone group: 10 and deflazacort group: 12). The mean age was 7.7 years at an average followup of 26.4 months. Twenty children were diagnosed to have Duchenne’s; one had Becker’s muscular dystrophy while one had sarcoglycanopathy by Type 2C. All children from prednisolone group maintained their ambulatory status at 2 and 4 years followups while three on deflazacort lost their ability to walk at an average age of 11.3 years. All activities of daily living were found to be better in prednisolone group. Muscle function and time taken to walk improved in prednisolone group. Weight gain in children on prednisolone was three times more.

Conclusions: Prednisolone is more beneficial than deflazacort at doses of 0.75 mg/kg/day and 0.9 mg/kg/day, respectively however it is associated with adverse effects.

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References

  1. Hoffman EP, Brown RH Jr., Kunkel LM. Dystrophin: The protein product of the Duchenne muscular dystrophy locus. Cell 1987;51:919–28.

    Article  CAS  Google Scholar 

  2. 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;12:926–9.

    Article  Google Scholar 

  3. Karpati G, editor. Structural and molecular basis of skeletal muscle diseases: Structural and molecular basis of skeletal muscle diseases. Basel: ISN Neuropath Press; 2002. p. 312.

    Google Scholar 

  4. Angelini C, Nardetto L, Fanin M, Nascimbeni AC, Tasca E. Heterogeneous pathogenesis of LGMD2: Consequences for therapy. Basic Appl Myol 2007;17:173–9.

    Google Scholar 

  5. Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, et al. Diagnosis and management of Duchenne muscular dystrophy, part 2: Implementation of multidisciplinary care. Lancet Neurol 2010;9:177–89.

    Article  CAS  Google Scholar 

  6. Alman BA. Duchenne muscular dystrophy and steroids: Pharmacologic treatment in the absence of effective gene therapy. J Pediatr Orthop 2005;25:554–6.

    Article  Google Scholar 

  7. Beenakker EA, Fock JM, Van Tol MJ, Maurits NM, Koopman HM, Brouwer OF, er al. Intermittent prednisone therapy in Duchenne muscular dystrophy: A randomized controlled trial. Arch Neurol 2005;62:128–32.

    Article  Google Scholar 

  8. 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.

    Article  CAS  Google Scholar 

  9. 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;48:383–8.

    Article  CAS  Google Scholar 

  10. Takeuchi F, Yonemoto N, Nakamura H, Shimizu R, Komaki H, Mori-Yoshimura M, et al. Prednisolone improves walking in Japanese Duchenne muscular dystrophy patients. J Neurol 2013;260:3023–9.

    Article  CAS  Google Scholar 

  11. Sato Y, Yamauchi A, Urano M, Kondo E, Saito K. Corticosteroid therapy for duchenne muscular dystrophy: Improvement of psychomotor function. Pediatr Neurol 2014;50:31–7.

    Article  Google Scholar 

  12. Bonifati DM, Witchel SF, Ermani M, Hoffman EP, Angelini C, Pegoraro E. The glucocorticoid receptor N363S polymorphism and steroid response in Duchenne dystrophy. J Neurol Neurosurg Psychiatry 2006;77:1177–9

    Article  CAS  Google Scholar 

  13. Manzur AY, Kuntzer T, Pike M, Swan A. Glucocorticoid corticosteroids for Duchenne muscular dystrophy. Cochrane Database Syst Rev. 2004; (2)

    Google Scholar 

  14. Manzur AY, Kuntzer T, Pike M, Swan A. Glucocorticoid corticosteroids for Duchenne muscular dystrophy. Cochrane Database Syst Rev. 2008;(1):CD003725.doi: 10.1002/14651858. CD003725.pub3.

    Google Scholar 

  15. Merlini L, Cicognani A, Malaspina E, Gennari M, Gnudi S, Talim B, et al. Early prednisone treatment in Duchenne muscular dystrophy. Muscle Nerve 2003;27:222–7.

    Article  CAS  Google Scholar 

  16. Gorni K, Orcesi S, Berardinelli A, Pini A, Giannotta M, Fazzi E.G.P. 1.05 long term followup of functional performance in children with Duchenne muscular dystrophy treated with deflazacort. Neuromuscul Disord 2007; 17:773.

    Article  Google Scholar 

  17. Angelini C. The role of corticosteroids in muscular dystrophy: A critical appraisal. Muscle Nerve 2007;36:424–35.

    Article  CAS  Google Scholar 

  18. Angelini C, Peterle E. Old and new therapeutic developments in steroid treatment in Duchenne muscular dystrophy. Acta Myol 2012;31:9–15.

    PubMed  PubMed Central  Google Scholar 

  19. 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;23:1344–7.

    Article  CAS  Google Scholar 

  20. Ricotti V, Ridout DA, Scott E, Quinlivan R, Robb SA, Manzur AY, et al. Long term benefits and adverse effects of intermittent versus daily glucocorticoids in boys with Duchenne muscular dystrophy. J Neurol Neurosurg Psychiatry 2013;84:698–705.

    Article  Google Scholar 

  21. Bushby K, Muntoni F, Urtizberea A, Hughes R, Griggs R. Report on the 124th ENMC International Workshop. Treatment of Duchenne muscular dystrophy; defining the gold standards of management in the use of corticosteroids 2-4 April 2004, Naarden, The Netherlands. Neuromuscul Disord 2004;14:526–34.

    Article  CAS  Google Scholar 

  22. Biggar WD, Politano L, Harris VA, Passamano L, Vajsar J, Alman B, et al. Deflazacort in Duchenne muscular dystrophy: A comparison of two different protocols. Neuromuscul Disord 2004;14:476–82.

    Article  CAS  Google Scholar 

  23. Moxley RT 3rd, Pandya S, Ciafaloni E, Fox DJ, Campbell K. Change in natural history of Duchenne muscular dystrophy with long term corticosteroid treatment: Implications for management. J Child Neurol 2010;25:1116–29.

    Article  Google Scholar 

  24. Merlini L, Gennari M, Malaspina E, Cecconi I, Armaroli A, Gnudi S, et al. Early corticosteroid treatment in 4 Duchenne muscular dystrophy patients: 14-year followup. Muscle Nerve 2012;45:796–802.

    Article  CAS  Google Scholar 

  25. Dubowitz V. Muscle Disorders in Childhood. 2nd ed. WB Saunders, London; 1995. p. 552.

    Google Scholar 

  26. 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;23:1344–1347.

    Article  CAS  Google Scholar 

  27. Merlini L, Granata C, Ballestrazzi A, Cornelio F, Tassoni P, Tugnoli S, et al. Growth hormone evaluation in Duchenne muscular dystrophy. Ital J Neurol Sci 1988;9:471–5.

    Article  CAS  Google Scholar 

  28. Desguerre I, Christov C, Mayer M, Zeiler R, Becane HM, Bastuji-Garin S, et al. Clinical heterogeneity of duchenne muscular dystrophy (DMD): Definition of sub-phenotypes and predictive criteria by long term followup. PLoS One 2009;4:e4347.

    Article  Google Scholar 

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Authors and Affiliations

  1. Department of Child Health, CMC, Vellore, Tamil Nadu, India

    Indira Agarwal

  2. Department of Biostatistics, CMC, Vellore, Tamil Nadu, India

    Belavendra Antonisamy

  3. Department of Paediatric Orthopaedics, Christian Medical College, Paul Brand Building, Vellore, 632 004, Tamil Nadu, India

    Harish Petnikota,  Vrisha Madhuri & Sangeet Gangadharan

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  1. Harish Petnikota
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  2. Vrisha Madhuri
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  3. Sangeet Gangadharan
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  4. Indira Agarwal
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  5. Belavendra Antonisamy
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Correspondence to Vrisha Madhuri.

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Petnikota, H., Madhuri, V., Gangadharan, S. et al. Retrospective cohort study comparing the efficacy of prednisolone and deflazacort in children with muscular dystrophy. IJOO 50, 551–557 (2016). https://doi.org/10.4103/0019-5413.189609

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  • DOI: https://doi.org/10.4103/0019-5413.189609

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