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Cited by 493Muscle Physiology and DisordersNeurogenetic and Muscular Disorders ResearchAdrenal Hormones and Disorders

Corticosteroids for the treatment of Duchenne muscular dystrophy

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Matthews, Emma, Brassington, Ruth, Kuntzer, Thierry, Jichi, Fatima, Manzur, Adnan Y·2016·Cochrane Database of Systematic Reviews
DOI 10.1002/14651858.cd003725.pub4PMC PMC8580515
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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 c

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Authors
Matthews, E., Brassington, R., Kuntzer, T., Jichi, F., & Manzur, A. Y.
Affiliations
  • National Hospital for Neurology and Neurosurgery (GB)
  • CHU Vaudois and University of Lausanne; Nerve-Muscle Unit, Service of Neurology; Lausanne Switzerland 1011
  • University College London (GB)
  • Great Ormond Street Hospital (GB)
ORCID
Matthews, Emma
Venue
Cochrane Database of Systematic Reviews
Publisher
Wiley
Published
2016-05-05
Volume
2016
Issue
6
Pages
CD003725-CD003725
ISSN
  • 1361-6137 (ISSN-L)
  • 1465-1858
DOI
10.1002/14651858.cd003725.pub4
PMC
PMC8580515
OpenAlex
W2431474207
Cited by
493
References
144
Citations by year
2026: 37 · 2025: 52 · 2024: 56 · 2023: 47 · 2022: 47 · 2021: 48 · 2020: 74 · 2019: 41 · 2018: 39 · 2017: 45 · 2016: 6 · 2015: 1
FWCI
3.95
Type
journal article
Language
en
Open access
Locations
  • Cochrane Database of Systematic Reviews (publishedVersion)
  • PubMed Central (publishedVersion)
  • PubMed (publishedVersion)
  • SERVAL (Université de Lausanne) (publishedVersion, other-oa)
  • IRIS (submittedVersion)
  • UCL Discovery (University College London) (submittedVersion)
Funders
  • National Institute for Health and Care Research
  • European Commission (036825)
  • Medical Research Council
  • Evidence Synthesis Programme
Topics
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Muscle Physiology and Disorders
  • Health Sciences › Medicine › Genetics › Neurogenetic and Muscular Disorders Research
  • Health Sciences › Medicine › Endocrinology, Diabetes and Metabolism › Adrenal Hormones and Disorders
Keywords
Duchenne muscular dystrophy, corticosteroids, prednisone, prednisolone, deflazacort, walking ability, muscle strength, functional capacity, quality of life, adverse events, corticosteroid dosing regimens, long-term treatment outcomes, treatment initiation timing, children and adolescents
MeSH terms
Adrenal Cortex Hormones, Glucocorticoids, Humans, Male, Prednisolone, Prednisone, Pregnenediones, Quality of Life, Randomized Controlled Trials as Topic, Walking, Muscular Dystrophy, Duchenne, Muscle Strength

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