Glucocorticoids for Duchenne Muscular Dystrophy: A Meta-Analysis_Chinese Journal of Evidence-Based Medicine

Authors：

 HUJun ¹ ,  JIANGLi ² , HONGSi-qi ² , KONGMin ² , YEYuan-zhen ²

1. Department of Pediatrics, Union Hospital, Fujian Medical University, Fuzhou 350001, China;
2. Department of Neurology, Children's Hospital Affiliated Chongqing Medical University, Chongqing 400014, China;

Corresponding author：

JIANGLi, Email: Dr.jiangl@gmail.com

Keywords：

Glucocorticoid; Duchenne muscular dystrophy; Therapeutic effect; Safety; Meta-analysis; Randomized controlled trial

DOI：

10.7507/1672-2531.20140143

Video：

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Objective To systematically review the therapeutic effects and safety of glucocorticoids (GC) for Duchenne muscular dystrophy (DMD). Methods Databases such as PubMed, EMbase, CENTRAL, CNKI, WanFang Data, VIP and CBM were electronically searched from the establishment of the databases till December 2011. All randomized controlled trials (RCTs) about GC for DMD were included. Two reviewers independently screened literature according to the inclusion and exclusion criteria, extracted data, and evaluated the methodological quality of the included studies. Then meta-analysis was performed using RevMan 5.0.2 software. Results A total of 6 studies involving 303 DMD children were included. The results of meta-analysis showed that after 6 month treatment of GC (deflazacort), patients' symptoms were obviously improved in average muscle strength, lift weight ability, forced vital capacity (FVC) of the lung, emotional factor scores and total scores in Quality of life (QoL), Gower's time, nine meters walking time (T9 m), and four-stair climbing time (T4 s). However, the trial group showed more weight gain, behavioural changes, increased appetite, cushingoid appearance, and excessive hair growth. The incidences of osteoporosis/fracture, hypertension, diabetes, and cataract were not increased. Conclusion GC could improve muscle strength and function, stabilize pulmonary function, prolong independent walk time, and improve QoL of DMD patients. However, adverse reaction caused by GC should be taken caution.

Citation： HUJun, JIANGLi, HONGSi-qi, KONGMin, YEYuan-zhen. Glucocorticoids for Duchenne Muscular Dystrophy: A Meta-Analysis. Chinese Journal of Evidence-Based Medicine, 2014, 14(7): 867-873. doi: 10.7507/1672-2531.20140143 Copy

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2.	Emery AE. The muscular dystrophies. Lancet, 2002, 359(1):687-695.
3.	Kunkel LM, Hejtmancik JF, Caskey CT, et al. Analysis of deletions in DNA from patients with Becker and Duchenne muscular dystrophy. Nature, 1986, 322(6074):73-77.
4.	Petrof BJ, Shrager JB, Stedman HH, et al. Dystrophin protects the sarcolemma from stresses developed during muscle contraction. Proc Natl Acad Sci USA, 1993, 90(8):3710-3714.
5.	Chakkalakal JV, Thompson J, Parks RJ, et al. Molecular, cellular, and pharmacological therapies for Duchenne/Becker muscular dystrophies. FASEB J, 2005, 19(8):880-891.
6.	Porter JD, Khanna S, Kaminski HJ, et al. A chronic inflammatory response dominates the skeletal muscle molecular signature in dystrophin-deficient mdx mice. Hum Mol Genet, 2002, 11(3):263-272.
7.	Dudley RWR, Danialou G, Govindaraju K, et al. Sarcolemmal damage in dystrophin deficiency is modulated by synergistic interactions between mechanical and oxidative/nitrosative stresses. Am J Pathol, 2006, 168(4):1276-1287.
8.	Moxley RT 3rd, 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(1):13-20.
9.	Manzur AY, Kuntzer T, Pike M, et al. Glucocorticoid corticosteroids for Duchenne muscular dystrophy. Cochrane Database Syst Rev, 2008, 23(1):CD003725.
10.	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(1):77-93.
11.	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(2):177-189.
12.	Moxley RT 3rd, Pandya S, Ciafaloni E, et al. Change in natural history of Duchenne muscular dystrophy with long-term corticosteroid treatment:implications for management. J Child Neurol, 2010, 25(9):1116-1129.
13.	Julian PT, Sally G (editors). Cochrane Handbook for Systematic Reviews of Interventions Version 5.1.0[updated March 2011]. Available at:http://www.cochrane-handbook.org.
14.	Mendell JR, Moxley RT 3rd, Griggs RC, et al. Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy. N Engl J Med, 1989, 320(24):1592-1597.
15.	Griggs RC, Moxley RT 3rd, 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(4):383-388.
16.	Angelini C, Pegoraro E, Turella E, et al. Deflazacort in Duchenne dystrophy:study of long-term effect. Muscle Nerve, 1994, 17(4):386-391.
17.	Bäckman E, Henriksson KG. Low-dose prednisolone treatment in Duchenne and Becker muscular dystrophy. Neuromuscul Disord, 1995, 5(3):233-241.
18.	Rahman MM, Hannan MA, Mondol BA, et al. Prednisolone in Duchenne muscular dystrophy. Bangladesh Med Res Counc Bull, 2001, 27(1):38-42.
19.	Beenakker EA, Fock JM, Van Tol MJ, et al. Intermittent prednisone therapy in Duchenne muscular dystrophy-a randomized controlled trial. Arch Neurol, 2005, 62(1):128-132.
20.	Griggs RC, Moxley RT 3rd, Mendell JR, et al. Duchenne dystrophy:randomized, controlled trial of prednisone (18 months) and azathioprine (12 months). Neurology, 1993, 43(3 Pt 1):520-527.
21.	Brooke MH, Fenichel GM, Griggs RC, et al. Clinical investigation in Duchenne dystrophy:2. Determination of the "power" of therapeutic trials based on the natural history. Muscle Nerve, 1983, 6(2):91-103.
22.	Brooke MH, Griggs RC, Mendell JR, et al. Clinical trial in Duchenne dystrophy. 1. The design of the protocol. Muscle Nerve, 1981, 4(3):186-197.
23.	Connolly MA, Johnson JA. Measuring quality of life in paediatric patients. Pharmacoeconomics, 1999, 16(6):605-625.
24.	Muntoni F, Fisher I, Morgan JE, et al. Steroids in Duchenne muscular dystrophy-from clinical trials to genomic research. Neuromuscul Disord, 2002, 12(Suppl 1):S162-S165.
25.	King WM, Ruttencutter R, Nagaraja HN, et al. Orthopedic outcomes of long-term daily corticosteroid treatment in Duchenne muscular dystrophy. Neurology, 2007, 68(19):1607-1613.
26.	Biggar WD, Harris VA, Eliasoph L, et al. Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade. Neuromuscul Disord, 2006, 16(4):249-255.
27.	Escolar DM, Hache LP, Clemens PR, et al. Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy. Neurology, 2011, 77(5):444-452.

1. Emery AE. Population frequencies of inherited neuromuscular diseases-a world survey. Neuromuscul Disord, 1991, 1(1):19-29.
2. Emery AE. The muscular dystrophies. Lancet, 2002, 359(1):687-695.
3. Kunkel LM, Hejtmancik JF, Caskey CT, et al. Analysis of deletions in DNA from patients with Becker and Duchenne muscular dystrophy. Nature, 1986, 322(6074):73-77.
4. Petrof BJ, Shrager JB, Stedman HH, et al. Dystrophin protects the sarcolemma from stresses developed during muscle contraction. Proc Natl Acad Sci USA, 1993, 90(8):3710-3714.
5. Chakkalakal JV, Thompson J, Parks RJ, et al. Molecular, cellular, and pharmacological therapies for Duchenne/Becker muscular dystrophies. FASEB J, 2005, 19(8):880-891.
6. Porter JD, Khanna S, Kaminski HJ, et al. A chronic inflammatory response dominates the skeletal muscle molecular signature in dystrophin-deficient mdx mice. Hum Mol Genet, 2002, 11(3):263-272.
7. Dudley RWR, Danialou G, Govindaraju K, et al. Sarcolemmal damage in dystrophin deficiency is modulated by synergistic interactions between mechanical and oxidative/nitrosative stresses. Am J Pathol, 2006, 168(4):1276-1287.
8. Moxley RT 3rd, 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(1):13-20.
9. Manzur AY, Kuntzer T, Pike M, et al. Glucocorticoid corticosteroids for Duchenne muscular dystrophy. Cochrane Database Syst Rev, 2008, 23(1):CD003725.
10. 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(1):77-93.
11. 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(2):177-189.
12. Moxley RT 3rd, Pandya S, Ciafaloni E, et al. Change in natural history of Duchenne muscular dystrophy with long-term corticosteroid treatment:implications for management. J Child Neurol, 2010, 25(9):1116-1129.
13. Julian PT, Sally G (editors). Cochrane Handbook for Systematic Reviews of Interventions Version 5.1.0[updated March 2011]. Available at:http://www.cochrane-handbook.org.
14. Mendell JR, Moxley RT 3rd, Griggs RC, et al. Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy. N Engl J Med, 1989, 320(24):1592-1597.
15. Griggs RC, Moxley RT 3rd, 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(4):383-388.
16. Angelini C, Pegoraro E, Turella E, et al. Deflazacort in Duchenne dystrophy:study of long-term effect. Muscle Nerve, 1994, 17(4):386-391.
17. Bäckman E, Henriksson KG. Low-dose prednisolone treatment in Duchenne and Becker muscular dystrophy. Neuromuscul Disord, 1995, 5(3):233-241.
18. Rahman MM, Hannan MA, Mondol BA, et al. Prednisolone in Duchenne muscular dystrophy. Bangladesh Med Res Counc Bull, 2001, 27(1):38-42.
19. Beenakker EA, Fock JM, Van Tol MJ, et al. Intermittent prednisone therapy in Duchenne muscular dystrophy-a randomized controlled trial. Arch Neurol, 2005, 62(1):128-132.
20. Griggs RC, Moxley RT 3rd, Mendell JR, et al. Duchenne dystrophy:randomized, controlled trial of prednisone (18 months) and azathioprine (12 months). Neurology, 1993, 43(3 Pt 1):520-527.
21. Brooke MH, Fenichel GM, Griggs RC, et al. Clinical investigation in Duchenne dystrophy:2. Determination of the "power" of therapeutic trials based on the natural history. Muscle Nerve, 1983, 6(2):91-103.
22. Brooke MH, Griggs RC, Mendell JR, et al. Clinical trial in Duchenne dystrophy. 1. The design of the protocol. Muscle Nerve, 1981, 4(3):186-197.
23. Connolly MA, Johnson JA. Measuring quality of life in paediatric patients. Pharmacoeconomics, 1999, 16(6):605-625.
24. Muntoni F, Fisher I, Morgan JE, et al. Steroids in Duchenne muscular dystrophy-from clinical trials to genomic research. Neuromuscul Disord, 2002, 12(Suppl 1):S162-S165.
25. King WM, Ruttencutter R, Nagaraja HN, et al. Orthopedic outcomes of long-term daily corticosteroid treatment in Duchenne muscular dystrophy. Neurology, 2007, 68(19):1607-1613.
26. Biggar WD, Harris VA, Eliasoph L, et al. Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade. Neuromuscul Disord, 2006, 16(4):249-255.
27. Escolar DM, Hache LP, Clemens PR, et al. Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy. Neurology, 2011, 77(5):444-452.

Chinese Journal of Evidence-Based Medicine

Glucocorticoids for Duchenne Muscular Dystrophy: A Meta-Analysis

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