Real-world outcomes of long-term prednisone and deflazacort use in patients with Duchenne muscular dystrophy: experience at a single, large care center
Publication: Journal of Comparative Effectiveness Research
Abstract
Aim: To assess outcomes among patients with Duchenne muscular dystrophy receiving deflazacort or prednisone in real-world practice. Methods: Clinical data for 435 boys with Duchenne muscular dystrophy from Cincinnati Children’s Hospital Medical Center were studied retrospectively using time-to-event and regression analyses. Results: Median ages at loss of ambulation were 15.6 and 13.5 years among deflazacort- and prednisone-initiated patients, respectively. Deflazacort was also associated with a lower risk of scoliosis and better ambulatory function, greater % lean body mass, shorter stature and lower weight, after adjusting for age and steroid duration. No differences were observed in whole body bone mineral density or left ventricular ejection fraction. Conclusion: This single center study adds to the real-world evidence associating deflazacort with improved clinical outcomes.
Lay abstract
This retrospective study described outcomes for boys with Duchenne muscular dystrophy treated at Cincinnati Children’s Hospital Medical Center with the glucocorticoids deflazacort (∼95% daily dosing) or prednisone (∼68% daily dosing). Patients receiving deflazacort had lower risk of losing ambulation, lost ambulation at older ages than those receiving prednisone, and had lower risk of scoliosis. Across clinic visits, deflazacort was associated with greater preservation of ambulatory and pulmonary function, shorter stature, lower bodyweight and greater % lean body mass. This study adds to the evidence associating real-world dosing of deflazacort with improved outcomes for patients with Duchenne muscular dystrophy.
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References
Papers of special note have been highlighted as: • of interest •• of considerable interest
1.
Genetic and Rare Diseases Information Center. Duchenne muscular dystrophy. https://rarediseases.info.nih.gov/diseases/6291/duchenne-muscular-dystrophy
2.
National Organization for Rare Disorders. Duchenne muscular dystrophy. https://rarediseases.org/rare-diseases/duchenne-muscular-dystrophy/
3.
Emery AE, Muntoni F, Quinlivan RC. Duchenne Muscular Systrophy. Oxford University Press, Oxford, UK (2015).
4.
Birnkrant DJ, Bushby K, Bann CM et al. Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management. Lancet Neurol. 17(4), 347–361 (2018).
5.
Ishikawa Y, Miura T, Ishikawa Y et al. Duchenne muscular dystrophy: survival by cardio-respiratory interventions. Neuromuscul. Disord. 21(1), 47–51 (2011).
6.
Lomauro A, D’Angelo MG, Aliverti A. Assessment and management of respiratory function in patients with Duchenne muscular dystrophy: current and emerging options. Ther. Clin. Risk. Manag. 11, 1475–1488 (2015).
7.
Morgenroth VH, Hache LP, Clemens PR. Insights into bone health in Duchenne muscular dystrophy. Bonekey Rep. 1, 9 (2012).
8.
Pandya S, James KA, Westfield C et al. Health profile of a cohort of adults with Duchenne muscular dystrophy. Muscle Nerve 58(2), 219–223 (2018).
9.
Bello L, Gordish-Dressman H, Morgenroth LP et al. Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study. Neurology 85(12), 1048–1055 (2015).
•• Real-world, observational study that provided class 4 evidence regarding the efficacy of prednisone and deflazacort in delaying the loss of ambulation in patients with Duchenne muscular dystrophy (DMD).
10.
Mcdonald CM, Gordish-Dressman H, Henricson EK et al. Longitudinal pulmonary function testing outcome measures in Duchenne muscular dystrophy: long-term natural history with and without glucocorticoids. Neuromuscul. Disord. 28(11), 897–909 (2018).
•• Longitudinal study that compared changes in pulmonary function (10 years follow-up) among patients with DMD who received glucocorticoid treatment (daily prednisone, daily deflazacort, alternative regimens) versus untreated patients with DMD.
11.
Mcdonald CM, Henricson EK, Abresch RT et al. 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. Muscle Nerve 48(1), 32–54 (2013).
12.
Buckner JL, Bowden SA, Mahan JD. Optimizing bone health in Duchenne muscular dystrophy. Int. J. Endocrinol. 2015, 928385 (2015).
13.
Hawker GA, Ridout R, Harris VA, Chase CC, Fielding LJ, Biggar WD. Alendronate in the treatment of low bone mass in steroid-treated boys with Duchennes muscular dystrophy. Arch. Phys. Med. Rehabil. 86(2), 284–288 (2005).
14.
Bonifati MD, Ruzza G, Bonometto P et al. A multicenter, double-blind, randomized trial of deflazacort versus prednisone in Duchenne muscular dystrophy. Muscle Nerve 23(9), 1344–1347 (2000).
15.
Griggs RC, Miller JP, Greenberg CR et al. Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy. Neurology 87(20), 2123–2131 (2016).
16.
Cunniff T, Wanaski S, Dubow J, Meyer J. Effect of deflazacort and prednisone versus placebo on muscle strength in boys with Duchenne muscular dystrophy who have lost ambulation: results from the Deflazacort Clinical Trial Program (S28.006). Neurology 86(Suppl. 16), S28.006 (2016).
17.
Shieh PB, Mcintosh J, Jin F et al. Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: a post hoc analysis from the ACT DMD trial. Muscle Nerve 58(5), 639–645 (2018).
•• A post-hoc analysis that assessed clinical outcomes from the placebo arm (prednisone/prednisolone and deflazacort) of a 48-week Phase III trial.
18.
Mcdonald CM, Sajeev G, Yao Z et al. Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: a meta-analysis of disease progression rates in recent multicenter clinical trials. Muscle Nerve (2019).
19.
Wong BL, Rybalsky I, Shellenbarger KC et al. Long-term outcome of interdisciplinary management of patients with Duchenne muscular dystrophy receiving daily glucocorticoid treatment. J. Pediatr. 182, 296–303 (2017).
• Real-world, retrospective study that evaluated clinical outcomes and side effects associated with daily glucocorticoid treatment (deflazacort or prednisone) among patients with DMD (ages 10 to <16 years for a mean of 8.5 years).
20.
Mcdonald CM, Henricson EK, Abresch RT et al. Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study. Lancet 391(10119), 451–461 (2018).
•• Longitudinal, prospective, cohort study that examined functional outcomes of patients with DMD (ages 2–28 years for 10 years) who were treated with glucocorticoids (prednisone/prednisolone or deflazacort) versus untreated patients with DMD.
21.
Bach JR, Martinez D, Saulat B. Duchenne muscular dystrophy: the effect of glucocorticoids on ventilator use and ambulation. Am. J. Phys. Med. Rehabil. 89(8), 620–624 (2010).
22.
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. 16(4), 249–255 (2006).
23.
Archer JE, Gardner AC, Roper HP, Chikermane AA, Tatman AJ. Duchenne muscular dystrophy: the management of scoliosis. J. Spine Surg. 2(3), 185–194 (2016).
24.
Alman BA, Raza SN, Biggar WD. Steroid treatment and the development of scoliosis in males with duchenne muscular dystrophy. J. Bone Joint Surg. Am. 86(3), 519–524 (2004).
25.
Lebel DE, Corston JA, McAdam LC, Biggar WD, Alman BA. Glucocorticoid treatment for the prevention of scoliosis in children with Duchenne muscular dystrophy: long-term follow-up. J. Bone Joint Surg. Am. 95(12), 1057–1061 (2013).
26.
Mcdonald CM, Henricson EK, Abresch RT et al. The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study. Muscle Nerve 48(3), 343–356 (2013).
27.
Mayhew AG, Cano SJ, Scott E et al. Detecting meaningful change using the North Star Ambulatory Assessment in Duchenne muscular dystrophy. Dev. Med. Child Neurol. 55(11), 1046–1052 (2013).
28.
Wong B, Signorovitch J, Staunton H et al. Estimating clinically meaningful 48-week change thresholds in the north star ambulatory assessment and four-stair climb in Duchenne muscular dystophy. Presented at: Action Duchenne International Conference. Birmingham, UK (2018).
29.
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. 25(9), 1116–1129 (2010).
• Highlights studies that documented the clinical effects associated with long-term (>3 years) corticosteroid treatment in patients with DMD.
30.
Mcadam LC, Mayo AL, Alman BA, Biggar WD. The Canadian experience with long-term deflazacort treatment in Duchenne muscular dystrophy. Acta Myol. 31(1), 16–20 (2012).
31.
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 19(6), 777–779 (1996).
32.
Joseph S, Wang C, Bushby K et al. Fractures and linear growth in a nationwide cohort of boys with Duchenne muscular dystrophy with and without glucocorticoid treatment: results from the UK NorthStar Database. JAMA Neurol. 76(6), 701–709 (2019).
33.
Roberto R, Fritz A, Hagar Y et al. The natural history of cardiac and pulmonary function decline in patients with duchenne muscular dystrophy. Spine (Phila Pa 1976) 36(15), E1009–E1017 (2011).
34.
Hoffman EP, Brown RH Jr, Kunkel LM. Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell 51(6), 919–928 (1987).
35.
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. 12(10), 926–929 (2002).
36.
Spurney C, Shimizu R, Morgenroth LP et al. Cooperative International Neuromuscular Research Group Duchenne Natural History Study demonstrates insufficient diagnosis and treatment of cardiomyopathy in Duchenne muscular dystrophy. Muscle Nerve 50(2), 250–256 (2014).
37.
Berkman LF, Glass T. Social integration, social networks, social support, and health. Social Epidemiol. 1, 137–173 (2000).
38.
Jack K, Mclean SM, Moffett JK, Gardiner E. Barriers to treatment adherence in physiotherapy outpatient clinics: a systematic review. Man. Ther. 15(3), 220–228 (2010).
39.
Flanigan KM, Campbell K, Viollet L et al. Anti-dystrophin T cell responses in Duchenne muscular dystrophy: prevalence and a glucocorticoid treatment effect. Hum. Gene Ther. 24(9), 797–806 (2013).
40.
Liu T, Zhang L, Joo D, Sun SC. NF-kappaB signaling in inflammation. Signal Transduct. Target Ther. 2, pii: 17023 (2017).
41.
Thapa RJ, Basagoudanavar SH, Nogusa S et al. NF-kappaB protects cells from gamma interferon-induced RIP1-dependent necroptosis. Mol. Cell. Biol. 31(14), 2934–2946 (2011).
42.
Wesemann DR, Benveniste EN. STAT-1 alpha and IFN-gamma as modulators of TNF-alpha signaling in macrophages: regulation and functional implications of the TNF receptor 1:STAT-1 alpha complex. J. Immunol. 171(10), 5313–5319 (2003).
43.
Nayak S, Acharjya B. Deflazacort versus other glucocorticoids: a comparison. Indian J. Dermatol. 53(4), 167–170 (2008).
44.
Assandri A, Buniva G, Martinelli E, Perazzi A, Zerilli L. Pharmacokinetics and metabolism of deflazacort in the rat, dog, monkey and man. Adv. Exp. Med. Biol. 171, 9–23 (1984).
45.
Bashar T, Apu MNH, Mostaid MS, Islam MS, Hasnat A. Pharmacokinetics and bioavailability study of a prednisolone tablet as a single oral dose in Bangladeshi healthy volunteers. Dose Response 16(3), 1559325818783932 (2018).
46.
Burakiewicz J, Sinclair CDJ, Fischer D, Walter GA, Kan HE, Hollingsworth KG. Quantifying fat replacement of muscle by quantitative MRI in muscular dystrophy. J. Neurol. 264(10), 2053–2067 (2017).
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Received: 13 November 2019
Accepted: 16 December 2019
Published online: 10 January 2020
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Real-world outcomes of long-term prednisone and deflazacort use in patients with Duchenne muscular dystrophy: experience at a single, large care center. (2020) Journal of Comparative Effectiveness Research. DOI: 10.2217/cer-2019-0170
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- D. V. Vlodavets, S. B. Artemyeva, E. V. Tozliyan, A. K. Shulchenko, Vamorolone – a corticosteroid approved by global regulators for the treatment of Duchenne muscular dystrophy. Literature review, Russian Journal of Child Neurology, 10.17650/2073-8803-2025-20-2-40-51, 20, 2, (40-51), (2025).
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