Corticosteroid Treatment and Functional Improvement in Duchenne Muscular Dystrophy

Department of Physical Medicine and Rehabilitation, Gulhane Military Medical Academy, Etlik-Ankara, Turkey.
American Journal of Physical Medicine & Rehabilitation (Impact Factor: 2.2). 12/2005; 84(11):843-50. DOI: 10.1097/01.phm.0000184156.98671.d0
Source: PubMed


To determine and compare the long-term effects of prednisone and deflazacort on the functional status of children with Duchenne muscular dystrophy.
A total of 49 boys with Duchenne muscular dystrophy, between the age of 12 and 15 yrs, who were observed over a 7-yr period were reviewed retrospectively. Eighteen had been treated with prednisone, 12 with deflazacort, and 19 had no drug treatment. All boys treated with steroids received medication for >2 yrs before losing their ambulation. Lower and upper limb motor functions, pulmonary function, prevalence of surgery for scoliosis, and side effects were compared.
Boys in the steroid groups were significantly more functional and performed better on all tests than boys not treated (P < 0.05). There was no significant difference between the deflazacort- and prednisone-treated groups (P > 0.05). The number of boys having scoliosis surgery in treated groups was significantly less than nontreated boys (P < 0.05). The control group's pulmonary capacity was decreasing and significantly less than both prednisone- and deflazacort-treated boys. Both deflazacort and prednisone had beneficial effect on pulmonary function and scoliosis. Cataracts, hypertension, behavioral changes, excessive weight gain, and vertebral fracture were noted as serious side effects.
Prednisone and deflazacort have a significant beneficial effect on slowing the disease progress. Their usage in Duchenne muscular dystrophy may prolong ambulation and upper limb function with similar potency. Both steroids also improve pulmonary function, in addition to delaying the need for spinal interventions, with similar therapeutic profiles.

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    • "Third, PSL regimes (dose, age at commencement and duration) in our study may possibly have differed from those in related studies. A few previous studies only enrolled patients treated with GC for >1 [4] or >2 [8] years before LOA. Strictly controlled clinical trials have shown a more marked improvement in ambulation. "
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    ABSTRACT: We evaluated the long-term efficacy of prednisolone (PSL) therapy for prolonging ambulation in Japanese patients with genetically confirmed Duchenne muscular dystrophy (DMD). There were clinical trials have shown a short-term positive effect of high-dose and daily PSL on ambulation, whereas a few study showed a long-term effect. Especially in Japan, "real-life" observation was lacking. We utilized the national registry of muscular dystrophy in Japan for our retrospective study. We compared the age at loss of ambulation (LOA) between patients in PSL group and those in without-PSL group. Out of 791 patients' in the Remudy DMD/BMD registry from July 2009 to June 2012, 560 were matched with inclusion criteria. Of the 560, all were genetically confirmed DMD patients, 245 (43.8 %) of whom were treated with PSL and 315 (56.2 %) without PSL. There was no difference between the two groups regarding their mutational profile. The age at LOA was significantly greater (11 month on average) in the PSL group than in the without-PSL group (median, 132 vs. 121 months; p = 0.0002). Although strictly controlled clinical trials have shown that corticosteroid therapies achieved a marked improvement in ambulation, discontinuation of the drug due to intolerable side effects led to exclusion of clinical trial participants, which is considered as unavoidable. In our study, patients were not excluded from the PSL group, even if they discontinued the medication shortly after starting it. The results of our study may provide evidence to formulate recommendations and provide a basis for realistic expectations for PSL treatment of DMD patients in Japan, even there are certain limitations due to the retrospectively captured data in the registry.
    Full-text · Article · Sep 2013 · Journal of Neurology
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    • "Corticosteroids are the only recommended treatment for DMD. e drug increases muscle strength and slows the onset of complications [1]. As muscles have an endocrine function and due to the fact that this disease causes muscle degeneration, the study of hormone receptors in these organs "
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    ABSTRACT: Introduction: Several evidences show that muscles have an endocrine function. Glucocorticoid, estrogen, progesterone, and testosterone receptors have already been found in normal skeletal muscles, but not in dystrophic muscles. Methods: The gene expression of hormone receptors was compared between dystrophic and healthy muscles in mdx and C57BL6 mice strains. Results: The mdx mice showed a significant increase in the steroid receptors mRNA when compared to the C57BL6 mice: levels of androgen(s) receptors in the heart, estrogen receptors alpha in the EDL, and estrogen receptors beta in the quadriceps were increased. In addition, significant lowered levels of some other hormone receptors were found: corticosteroid receptors in the EDL and estrogen receptors alpha in the quadriceps. Conclusion: Dystrophic muscles bear significant differences in the expression of hormone receptors when compared to the C57BL6 mice strain. The importance of such differences is yet to be better understood.
    Full-text · Article · Mar 2013
    • "In this study, we test the effects of glucocorticoid treatment on the severity of thoracolumbar kyphosis in the mdx mouse. Current research suggests glucocorticoid treatment slows the progression of spinal deformation in humans, but it is unclear if it delays onset.121314 The aim of our study is to determine if glucocorticoid treatment can be administered to delay the appearance of dystrophinopathic spinal deformation. "
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    ABSTRACT: The development of spinal curvature deformities is a hallmark of muscular dystrophy. While glucocorticoid treatment has been shown to prolong muscle function in dystrophic mice, its effects on the development of dystrophinopathic spinal deformation are poorly understood. In this study, we test the effects of glucocorticoid treatment on the onset of thoracolumbar kyphosis in the dystrophin-deficient mdx mouse using voluntary running exercise to exacerbate muscle fibrosis. We measure the kyphotic index, erector spinae muscle fibrosis, and vertebral bone histomorphometry in 4-month-old mdx mice in four groups: sedentary control, exercise-treated (continuous voluntary access to an activity wheel), glucocorticoid-treated, and glucocorticoid + exercise-treated. Exercise treated mice were found to have significantly lower kyphotic index (i.e., greater kyphosis) and greater muscle fibrosis relative to controls (p < 0.05). However, the deleterious effect of exercise on KI and muscle fibrosis was prevented by glucocorticoid treatment. Some differences in bone histological parameters were observed between treatment groups, suggesting there is a complex relationship between dystrophic muscular changes and vertebral bone mass. Our findings indicate glucocorticoid treatment delays the onset of thoracodorsal spinal deformation in mdx mice.
    No preview · Article · Oct 2012 · PLoS Currents
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