Article

Corticosteroid treatment and functional improvement in Duchenne muscular dystrophy long-term effect

Department of Physical Medicine and Rehabilitation, Gulhane Military Medical Academy, Etlik-Ankara, Turkey.
American Journal of Physical Medicine & Rehabilitation (Impact Factor: 2.01). 12/2005; 84(11):843-50. DOI: 10.1097/01.phm.0000184156.98671.d0
Source: PubMed

ABSTRACT 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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    • "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.
    01/2013; 2013:604635. DOI:10.1155/2013/604635
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    • "to previous published observations for both corticosteroid treated DMD boys [2,15–17] and steroid naive DMD boys [17] [29]. However, boys on corticosteroids are at increased risk of vertebral fractures [8] [9] [13] [15] [16]. This increased risk may be due to GIOP and disease progression with loss of muscle and increased fat mass. "
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    ABSTRACT: Quality of life in Duchenne Muscular Dystrophy (DMD) has improved significantly with corticosteroid treatment. However, corticosteroids decrease bone mass and increase vertebral fragility fracture risk. We report on bone health in 39 boys with DMD on long-term deflazacort (0.9mg/kg/day) therapy. Bone health was defined by lumbar (L(1)-L(4)) bone mineral density (BMD), long-bone and/or symptomatic vertebral fractures. Lumbar BMD was reported as height-adjusted Z-scores at initiation of deflazacort (T(0)) and 1-2year intervals thereafter. Subcapital body fat percentage and ambulatory status were recorded. At T(0), 39 boys, aged 6.6±1.6years had height-adjusted BMD Z-score -0.5±0.8, and 23.5±5.0% body fat. Height-adjusted Z-scores remained stable with years of deflazacort until loss of ambulation and accrual of body fat. Nine long-bone fractures occurred in eight ambulating boys, two before T(0). Seven vertebral fractures occurred in six non-ambulatory boys after ⩾5years of deflazacort with height-adjusted Z-score -1.8±0.7, and 47.8±12% body fat. Bone health in DMD is influenced by disease progression, corticosteroids, BMD Z-scores and fat mass accumulation. Adjustments for short stature must be considered during BMD interpretation. Percent body fat and ambulatory status are useful bone health indicators. Routine use of height adjusted Z-scores is advocated for use in routine clinical practice.
    Neuromuscular Disorders 07/2012; 22(12). DOI:10.1016/j.nmd.2012.06.354 · 3.13 Impact Factor
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    • "A study determined and compared the long-term effects of prednisone and deflazacort on 49 boys, aged between 12 and 15 years, with DMD over a 7-year follow up period (Balaban et al., 2005). Eighteen boys were treated with prednisone, 12 with deflazacort, and 19 had no drug treatment. "
    MUSCULAR DYSTROPHY, 2012 edited by Madhuri Hegde, Arunkanth Ankala, 05/2012: chapter 19: pages 349-380; InTech., ISBN: 978-953-51-0603-6
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