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Meta-Analysis
. 2016 May 5;2016(5):CD003725.
doi: 10.1002/14651858.CD003725.pub4.

Corticosteroids for the treatment of Duchenne muscular dystrophy

Emma Matthews  1 , Ruth Brassington, Thierry Kuntzer, Fatima Jichi, Adnan Y Manzur
Affiliations
Meta-Analysis

Corticosteroids for the treatment of Duchenne muscular dystrophy

Emma Matthews et al. Cochrane Database Syst Rev. .

Abstract

Background: Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy of childhood. Untreated, this incurable disease, which has an X-linked recessive inheritance, is characterised by muscle wasting and loss of walking ability, leading to complete wheelchair dependence by 13 years of age. Prolongation of walking is a major aim of treatment. Evidence from randomised controlled trials (RCTs) indicates that corticosteroids significantly improve muscle strength and function in boys with DMD in the short term (six months), and strength at two years (two-year data on function are very limited). Corticosteroids, now part of care recommendations for DMD, are largely in routine use, although questions remain over their ability to prolong walking, when to start treatment, longer-term balance of benefits versus harms, and choice of corticosteroid or regimen.We have extended the scope of this updated review to include comparisons of different corticosteroids and dosing regimens.

Objectives: To assess the effects of corticosteroids on prolongation of walking ability, muscle strength, functional ability, and quality of life in DMD; to address the question of whether benefit is maintained over the longer term (more than two years); to assess adverse events; and to compare efficacy and adverse effects of different corticosteroid preparations and regimens.

Search methods: On 16 February 2016 we searched the Cochrane Neuromuscular Specialised Register, CENTRAL, MEDLINE, EMBASE, CINAHL Plus, and LILACS. We wrote to authors of published studies and other experts. We checked references in identified trials, handsearched journal abstracts, and searched trials registries.

Selection criteria: We considered RCTs or quasi-RCTs of corticosteroids (e.g. prednisone, prednisolone, and deflazacort) given for a minimum of three months to patients with a definite DMD diagnosis. We considered comparisons of different corticosteroids, regimens, and corticosteroids versus placebo.

Data collection and analysis: The review authors followed standard Cochrane methodology.

Main results: We identified 12 studies (667 participants) and two new ongoing studies for inclusion. Six RCTs were newly included at this update and important non-randomised cohort studies have also been published. Some important studies remain unpublished and not all published studies provide complete outcome data.

Primary outcome measure: one two-year deflazacort RCT (n = 28) used prolongation of ambulation as an outcome measure but data were not adequate for drawing conclusions.

Secondary outcome measures: meta-analyses showed that corticosteroids (0.75 mg/kg/day prednisone or prednisolone) improved muscle strength and function versus placebo over six months (moderate quality evidence from up to four RCTs). Evidence from single trials showed 0.75 mg/kg/day superior to 0.3 mg/kg/day on most strength and function measures, with little evidence of further benefit at 1.5 mg/kg/day. Improvements were seen in time taken to rise from the floor (Gowers' time), timed walk, four-stair climbing time, ability to lift weights, leg function grade, and forced vital capacity. One new RCT (n = 66), reported better strength, function and quality of life with daily 0.75 mg/kg/day prednisone at 12 months. One RCT (n = 28) showed that deflazacort stabilised muscle strength versus placebo at two years, but timed function test results were too imprecise for conclusions to be drawn.One double-blind RCT (n = 64), largely at low risk of bias, compared daily prednisone (0.75 mg/kg/day) with weekend-only prednisone (5 mg/kg/weekend day), finding no overall difference in muscle strength and function over 12 months (moderate to low quality evidence). Two small RCTs (n = 52) compared daily prednisone 0.75 mg/kg/day with daily deflazacort 0.9 mg/kg/day, but study methods limited our ability to compare muscle strength or function.

Adverse effects: excessive weight gain, behavioural abnormalities, cushingoid appearance, and excessive hair growth were all previously shown to be more common with corticosteroids than placebo; we assessed the quality of evidence (for behavioural changes and weight gain) as moderate. Hair growth and cushingoid features were more frequent at 0.75 mg/kg/day than 0.3 mg/kg/day prednisone. Comparing daily versus weekend-only prednisone, both groups gained weight with no clear difference in body mass index (BMI) or in behavioural changes (low quality evidence for both outcomes, one study); the weekend-only group had a greater linear increase in height. Very low quality evidence suggested less weight gain with deflazacort than with prednisone at 12 months, and no difference in behavioural abnormalities. Data are insufficient to assess the risk of fractures or cataracts for any comparison.Non-randomised studies support RCT evidence in showing improved functional benefit from corticosteroids. These studies suggest sustained benefit for up to 66 months. Adverse effects were common, although generally manageable. According to a large comparative longitudinal study of daily or intermittent (10 days on, 10 days off) corticosteroid for a mean period of four years, a daily regimen prolongs ambulation and improves functional scores over the age of seven, but with a greater frequency of side effects than an intermittent regimen.

Authors' conclusions: Moderate quality evidence from RCTs indicates that corticosteroid therapy in DMD improves muscle strength and function in the short term (twelve months), and strength up to two years. On the basis of the evidence available for strength and function outcomes, our confidence in the effect estimate for the efficacy of a 0.75 mg/kg/day dose of prednisone or above is fairly secure. There is no evidence other than from non-randomised trials to establish the effect of corticosteroids on prolongation of walking. In the short term, adverse effects were significantly more common with corticosteroids than placebo, but not clinically severe. A weekend-only prednisone regimen is as effective as daily prednisone in the short term (12 months), according to low to moderate quality evidence from a single trial, with no clear difference in BMI (low quality evidence). Very low quality evidence indicates that deflazacort causes less weight gain than prednisone after a year's treatment. We cannot evaluate long-term benefits and hazards of corticosteroid treatment or intermittent regimens from published RCTs. Non-randomised studies support the conclusions of functional benefits, but also identify clinically significant adverse effects of long-term treatment, and a possible divergence of efficacy in daily and weekend-only regimens in the longer term. These benefits and adverse effects have implications for future research and clinical practice.

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Conflict of interest statement

Dr Emma Matthews has no conflicts of interest.

Dr Ruth Brassington is Managing Editor of Cochrane Neuromuscular, of which The National Institute for Health Research (NIHR) is the largest single funder. The NIHR provided an incentive award to Cochrane Neuromuscular for the updating of this review (see Acknowledgements). A grant from the Motor Neurone Disease Association to Cochrane Neuromuscular contributed to her salary in 2011‐2015. She has no financial conflicts of interest. She withdrew from the later stages of the editorial process of this review.

Dr Thierry Kuntzer has no conflicts of interest.

Fatima Jichi has no known conflicts of interest.

Dr Adnan Y Manzur, at the time of preparation and submission of the protocol for this review was the principal investigator of a proposed UK multicentre trial of prednisolone in Duchenne muscular dystrophy. However, this trial was not funded. Currently, Dr Manzur is the lead clinician of the UK North Star Clinical Network for Neuromuscular Disorders. The clinicians on this clinical network have a consensus on approach to use of corticosteroids (prednisolone) and plans for future collaboration to audit and modify clinical practice in line with available evidence.

Figures

1
1
Study flow diagram.
2
2
Risk of bias summary: review authors' judgements about each risk of bias item for each included study.
3
3
Forest plot of comparison: 1 Glucocorticoid corticosteroids versus placebo, outcome: 1.1 MRC ‐ Average muscle score after 6 months of treatment ‐ prednisone.
4
4
Forest plot of comparison: 1 Glucocorticoid corticosteroids versus placebo, outcome: 1.7 Nine‐metre walking/running time after 6 months of treatment ‐ prednisone.
5
5
Forest plot of comparison: 1 Glucocorticoid corticosteroids versus placebo, outcome: 1.11 Four‐stair climbing time after 6 months of treatment ‐ prednisone.
6
6
Forest plot of comparison: 3 Deflazacort versus prednisone, outcome: 3.1 Weight gain (%).
1.1
1.1. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 1 Change in MRC index (%) after 6 months of treatment ‐ deflazacort 2 mg/kg alternate days.
1.2
1.2. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 2 MRC ‐ Average muscle score after 6 months of treatment ‐ prednisone.
1.3
1.3. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 3 Change in MRC index (%) after 24 months of treatment ‐ deflazacort 2 mg/kg alternate days.
1.4
1.4. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 4 Lower limb muscle strength grade after 6 months of treatment ‐ prednisone.
1.5
1.5. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 5 Lower limb muscle strength grade after 12 months of treatment ‐ prednisone.
1.6
1.6. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 6 Time taken to rise from floor after 6 months of treatment ‐ prednisone.
1.7
1.7. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 7 Change in time taken to rise from floor after 6 months of treatment ‐ deflazacort 2 mg/kg alternate days.
1.8
1.8. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 8 Time taken to rise from floor after 12 months of treatment (daily prednisone 0.75 mg/kg/day).
1.9
1.9. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 9 Change in time taken to rise from floor after 24 months of treatment ‐ deflazacort 2 mg/kg alternate days.
1.10
1.10. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 10 9‐metre walking/running time after 6 months of treatment ‐ prednisone.
1.11
1.11. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 11 Timed walk (assumed in seconds) ‐ after 6 months of treatment ‐ deflazacort 2 mg/kg alternate days.
1.12
1.12. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 12 10‐metre walking time 6 months post‐treatment (daily prednisone 0.75 mg/kg).
1.13
1.13. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 13 10‐metre walk time 1 year post‐treatment (daily prednisone 0.75 mg/kg/day).
1.14
1.14. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 14 Timed walk (assumed in seconds) ‐ after 24 months of treatment ‐ deflazacort 2 mg/kg alternate days.
1.15
1.15. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 15 Lifting weight (kg) after 6 months of treatment ‐ prednisone.
1.16
1.16. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 16 Four‐stair climbing time after 6 months of treatment ‐ prednisone.
1.17
1.17. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 17 Timed function: stair climb after 6 months of treatment ‐ deflazacort 2 mg/kg alternate days.
1.18
1.18. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 18 Four‐stair climbing time after 12 months of treatment (daily prednisone 0.75 mg/kg/day).
1.19
1.19. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 19 Change in timed stair climb after 24 months of treatment ‐ deflazacort 2 mg/kg alternate days.
1.20
1.20. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 20 Dynamometry ‐ total muscle force after 6 months of treatment ‐ prednisone.
1.21
1.21. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 21 Leg function grade after 6 months of treatment ‐ prednisone.
1.22
1.22. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 22 Forced vital capacity after 6 months of treatment ‐ prednisone.
1.23
1.23. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 23 Quality of life after six months of treatment (daily prednisone 0.75 mg/kg/day).
1.24
1.24. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 24 Quality of life after 12 months of treatment (daily prednisone 0.75 mg/kg/day).
1.25
1.25. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 25 Mean % weight gain ‐ prednisone ‐ daily dose regimen.
1.26
1.26. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 26 Weight gain ‐ prednisone ‐ intermittent, given 1st 10 days every month.
1.27
1.27. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 27 Mean % weight gain ‐ deflazacort 2 mg/kg alternate days.
1.28
1.28. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 28 Body weight at 6 months (prednisone 0.75 mg/kg/day).
1.29
1.29. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 29 Body weight at 12 months (prednisone 0.75 mg/kg/day).
1.30
1.30. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 30 BMI at 6 months (daily prednisone 0.75 mg/kg/day).
1.31
1.31. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 31 BMI at 12 months (daily prednisone 0.75 mg/kg/day).
1.32
1.32. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 32 Excessive hair growth ‐ prednisone.
1.33
1.33. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 33 Behavioural changes ‐ prednisone.
1.34
1.34. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 34 Cushingoid appearance ‐ prednisone.
1.35
1.35. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 35 Acne ‐ prednisone.
1.36
1.36. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 36 Increased appetite ‐ prednisone.
1.37
1.37. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 37 Height at 6 months (daily prednisone 0.75 mg/kg/day).
1.38
1.38. Analysis
Comparison 1 Corticosteroids versus placebo, Outcome 38 Height at 12 months (daily prednisone 0.75 mg/kg/day).
2.1
2.1. Analysis
Comparison 2 Weekend‐only versus daily prednisone, Outcome 1 Muscle strength (change from baseline to 12 months).
2.2
2.2. Analysis
Comparison 2 Weekend‐only versus daily prednisone, Outcome 2 Functional outcome measures (change from baseline to 12 months).
2.3
2.3. Analysis
Comparison 2 Weekend‐only versus daily prednisone, Outcome 3 Change in mobility function (lower extremity score ‐ Vignos).
2.4
2.4. Analysis
Comparison 2 Weekend‐only versus daily prednisone, Outcome 4 Change in mobility function (upper extremity score ‐ Brooke).
2.5
2.5. Analysis
Comparison 2 Weekend‐only versus daily prednisone, Outcome 5 FVC % predicted.
2.6
2.6. Analysis
Comparison 2 Weekend‐only versus daily prednisone, Outcome 6 FEV1 % predicted.
2.7
2.7. Analysis
Comparison 2 Weekend‐only versus daily prednisone, Outcome 7 Maximal inspiratory pressure.
2.8
2.8. Analysis
Comparison 2 Weekend‐only versus daily prednisone, Outcome 8 Maximal voluntary ventilation.
2.9
2.9. Analysis
Comparison 2 Weekend‐only versus daily prednisone, Outcome 9 Weight (BMI kg/m2).
2.10
2.10. Analysis
Comparison 2 Weekend‐only versus daily prednisone, Outcome 10 Weight (kg).
2.11
2.11. Analysis
Comparison 2 Weekend‐only versus daily prednisone, Outcome 11 Child Behavior Checklist: total problems (higher = more severe).
2.12
2.12. Analysis
Comparison 2 Weekend‐only versus daily prednisone, Outcome 12 Child Behavior Checklist: internalising.
2.13
2.13. Analysis
Comparison 2 Weekend‐only versus daily prednisone, Outcome 13 Child Behavior Checklist: externalising.
2.14
2.14. Analysis
Comparison 2 Weekend‐only versus daily prednisone, Outcome 14 Child Behavior Checklist: anxious/depressed.
2.15
2.15. Analysis
Comparison 2 Weekend‐only versus daily prednisone, Outcome 15 Child Behavior Checklist: somatic complaints.
2.16
2.16. Analysis
Comparison 2 Weekend‐only versus daily prednisone, Outcome 16 Child Behavior Checklist: withdrawn/depressed.
2.17
2.17. Analysis
Comparison 2 Weekend‐only versus daily prednisone, Outcome 17 Child Behavior Checklist: attention problems.
2.18
2.18. Analysis
Comparison 2 Weekend‐only versus daily prednisone, Outcome 18 Child Behavior Checklist: aggressive behaviour.
2.19
2.19. Analysis
Comparison 2 Weekend‐only versus daily prednisone, Outcome 19 Osteoporosis: lumbar spine Z scores (DEXA).
2.20
2.20. Analysis
Comparison 2 Weekend‐only versus daily prednisone, Outcome 20 Height (m).
2.21
2.21. Analysis
Comparison 2 Weekend‐only versus daily prednisone, Outcome 21 Mean growth in cm.
3.1
3.1. Analysis
Comparison 3 Deflazacort versus prednisone, Outcome 1 Weight gain (%).
3.2
3.2. Analysis
Comparison 3 Deflazacort versus prednisone, Outcome 2 Adverse events at six months.
3.3
3.3. Analysis
Comparison 3 Deflazacort versus prednisone, Outcome 3 Adverse events at 1 year.
See this image and copyright information in PMC

Update of

References

References to studies included in this review

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Angelini 2007 {published data only}
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Balaban 2005 {published data only}
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Dubowitz 2002 {published data only}
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Fenichel 1991a {published data only}
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Fenichel 1991b {published data only}
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Flanigan 2012 {published data only}
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Griggs 1993 {published data only}
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Griggs 2013 {published data only}
    1. Griggs RC, Herr BE, Reha A, Elfring G, Atkinson L, Cwik V, et al. Corticosteroids in Duchenne muscular dystrophy: major variations in practice. Muscle & Nerve 2013; Vol. 48, issue 1:27‐31. - PubMed
Henricson 2013 {published data only}
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Houde 2008 {published data only}
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Kinali 2002 {published data only}
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Kinali 2007 {published data only}
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King 2007 {published data only}
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Mayhew 2013 {published data only}
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Mazzone 2013 {published data only}
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McAdam 2012 {published data only}
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Mesa 1991 {published data only}
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Pandya 2001 {published data only}
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Parreira 2007 {published data only}
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Resende 2001 {published data only}
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Ricotti 2013 {published data only}
    1. Ricotti V, Ridout DA, Scott E, Quinlivan R, Robb SA, Manzur AY, et al. NorthStar Clinical Network. Long‐term benefits and adverse effects of intermittent versus daily glucocorticoids in boys with Duchenne muscular dystrophy. Journal of Neurology, Neurosurgery & Psychiatry 2013;84(6):698‐705. - PubMed
Sansome 1993 {published data only}
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Siegel 1974 {published data only}
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Silversides 2003 {published data only}
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Simon 2011 {published data only}
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Takeuchi 2013 {published data only}
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Tunca 2001 {published data only}
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Vasanth 1996 {published data only}
    1. Vasanth A, Gourie‐Devi M, Rajaram P, Taly AB, Venkataram BS, Ravishankar D, et al. Duchenne muscular dystrophy: therapeutic options and rehabilitation. European Journal of Neurology 1996;3(Suppl 2):20.
Wong 2002 {published data only}
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Yilmaz 2004 {published data only}
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    1. Yilmaz O, Karaduman A, Topaloğlu H. Prednisolone therapy in Duchenne muscular dystrophy prolongs ambulation and prevents scoliosis. European Journal of Neurology 2004;11(8):541‐4. - PubMed

References to studies awaiting assessment

ACTRN12605000075684 {published data only}
    1. ACTRN12605000075684. A randomized phase III study to evaluate the effectiveness of two different dosing regimens (high dose vs daily) of prednisone for boys with Duchenne muscular dystrophy in improving muscle strength and function and minimising side effects. http://apps.who.int/trialsearch/Trial2.aspx?TrialID=ACTRN12605000075684 (accessed 26 April 2016).
Bello 2015 {published data only}
    1. Bello L, Gordish‐Dressman H, Morgenroth L, Henricson E, Duong T, Hoffman E, et al. Prednisone/prednisolone and deflazacort differ in long term outcomes on ambulation and side effects in the CINRG Duchenne Natural History Study. Neurology 2015;84(14 Suppl). - PMC - PubMed
    1. Bello L, Gordish‐Dressman H, Morgenroth LP, Henricson EK, Duong T, Hoffman EP, et al. CINRG Investigators. Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study. Neurology 2015;9(22):1048‐55. - PMC - PubMed
    1. Bello L, Kesari A, Gordish‐Dressman H, Cnaan A, Morgenroth LP, Punetha J, et al. Cooperative International Neuromuscular Research Group Investigators. Genetic modifiers of ambulation in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study. Annals of Neurology 2015;77(4):684‐96. - PMC - PubMed
Pane 2015 {published data only}
    1. Pane M, Mazzone ES, Sivo S, Sormani MP, Messina S, D'Amico A, et al. Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36‐month changes. PLoS One 2014;9(10):e108205. - PMC - PubMed

References to ongoing studies

CTRI/2009/091/000738 {published data only}
    1. CTRI/2009/091/000738. A clinical trial to compare the two ways of giving steroids (daily versus intermittent) in ambulatory patients with Duchenne muscular dystrophy. http://apps.who.int/trialsearch/Trial2.aspx?TrialID=CTRI/2009/091/000605 accessed 28 April 2016.
Guglieri 2015 {published data only}
    1. Guglieri M, Ruiten HJA, Speed C, Hart K, Watson G, McColl E, et al. FOR‐DMD: Double‐blind randomised trial to optimise corticosteroid regime in Duchenne muscular dystrophy (DMD). Developmental Medicine and Child Neurology 2015; Vol. 57, issue Suppl s1:25‐6.
    1. NCT01603407. Finding the optimum regimen for Duchenne muscular dystrophy (FOR‐DMD). www.clinicaltrials.gov/ct2/show/NCT01603407 (accessed 14 December 2015).

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References to other published versions of this review

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