Prednisone 10 days on/10 days off in patients with Duchenne muscular dystrophy

Straathof, C.S.M.; Overweg-Plandsoen, W. C. G.; Burg, Gert Jan van den; Kooi, Anneke J. van der; Verschuuren, Jan J.; Groot, I. de

doi:10.1007/s00415-009-5012-y

Cited by 27 publications

(18 citation statements)

References 24 publications

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“…Bone strength, or mechanical properties, are compromised in these patients as evident by the occurrence of fragility fractures upon falling from standing or sitting height [4], [5], [6], [7]. Compromised bone strength in DMD is multi-factorial, likely including effects of failure to accumulate peak bone strength during growth as well as declines in bone health secondary to the muscle disease.…”

Section: Introductionmentioning

confidence: 99%

Low Intensity, High Frequency Vibration Training to Improve Musculoskeletal Function in a Mouse Model of Duchenne Muscular Dystrophy

et al. 2014

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The objective of the study was to determine if low intensity, high frequency vibration training impacted the musculoskeletal system in a mouse model of Duchenne muscular dystrophy, relative to healthy mice. Three-week old wildtype (n = 26) and mdx mice (n = 22) were randomized to non-vibrated or vibrated (45 Hz and 0.6 g, 15 min/d, 5 d/wk) groups. In vivo and ex vivo contractile function of the anterior crural and extensor digitorum longus muscles, respectively, were assessed following 8 wks of vibration. Mdx mice were injected 5 and 1 days prior to sacrifice with Calcein and Xylenol, respectively. Muscles were prepared for histological and triglyceride analyses and subcutaneous and visceral fat pads were excised and weighed. Tibial bones were dissected and analyzed by micro-computed tomography for trabecular morphometry at the metaphysis, and cortical geometry and density at the mid-diaphysis. Three-point bending tests were used to assess cortical bone mechanical properties and a subset of tibiae was processed for dynamic histomorphometry. Vibration training for 8 wks did not alter trabecular morphometry, dynamic histomorphometry, cortical geometry, or mechanical properties (P≥0.34). Vibration did not alter any measure of muscle contractile function (P≥0.12); however the preservation of muscle function and morphology in mdx mice indicates vibration is not deleterious to muscle lacking dystrophin. Vibrated mice had smaller subcutaneous fat pads (P = 0.03) and higher intramuscular triglyceride concentrations (P = 0.03). These data suggest that vibration training at 45 Hz and 0.6 g did not significantly impact the tibial bone and the surrounding musculature, but may influence fat distribution in mice.

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Section: Introductionmentioning

confidence: 99%

Low Intensity, High Frequency Vibration Training to Improve Musculoskeletal Function in a Mouse Model of Duchenne Muscular Dystrophy

et al. 2014

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“…Muscle weakness is a main clinical symptom and the target of treatment in DMD patients. Many treatment regimens have been reported previously, including daily [21] or intermittent dose regimens (on alternate days or 10 days on/10 days off [15]), with daily administration having a more favorable profile [22]. The most commonly used prednisone dosage was 0.75 mg/kg/day; a higher dose of 1.5 mg/kg/day was no more effective [23] and a lower dose of 0.3 mg/kg/day was less beneficial.…”

Section: Discussionmentioning

confidence: 99%

“…Recently, a randomized, blinded, clinical trial showed that 10 mg/kg prednisone administered only on weekends is as safe and effective over a 12-month treatment period as daily administration of 0.5 mg/kg prednisone in 4-year-old to 10-year-old boys with DMD [14]. Some studies have suggested that intermittent corticosteroid therapy over a 2-year period improves ambulatory function and causes relatively few adverse effects [15,16]. Even among patients who have lost ambulation ability, continued treatment is helpful in reducing the risk of progressive scoliosis and stabilizing pulmonary function [17,18].…”

Section: Introductionmentioning

confidence: 96%

A novel treatment regimen for Duchenne muscular dystrophy

Cai²,

Zhong³

et al. 2013

NeuroReport

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Duchenne muscular dystrophy (DMD) is the most common, X-linked genetic, skeletal muscle disease, with various regimens of treatment. The objective of this study was to determine the safety and efficacy of a novel treatment regimen for this disease. Thirty boys with DMD were administered prednisone according to the following regimen: in the first year, 1.5 mg/kg/day for the first 3 months, 1.0 mg/kg/day for the next 3 months, 0.75 mg/kg/day for the next 3 months, and 0.5 mg/kg/day for the last 3 months. In the second year, prednisone was administered 0.5 mg/kg on the alternate day for 12 months. The muscle strength (Medical Research Council sum score and Gower's sign), serum enzymes (creatine kinase, creatine kinase isoenzyme-2, and lactate dehydrogenase), pulmonary function (forced vital capacity, maximum voluntary ventilation), body weight, height, and BMI were determined before treatment and 3, 6, 9, 12, and 24 months after treatment. The results showed that the patients' mean Medical Research Council sum score increased from 46.1 at the baseline to 53.6 at 12 months and was maintained at 24 months. Gower's sign disappeared in 22 (73.3%) patients at 12 months and 21 (70.0%) at 24 months. The serum levels of creatine kinase, creatine kinase isoenzyme-2, and lactate dehydrogenase decreased and pulmonary function improved after 24 months of treatment. Significantly increased weight gain, osteoporosis, and cushingoid features were not observed. Our results suggested that this novel prednisone regimen for DMD has similar efficacy and safety as other regimens.

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“…The effect of an alternate, 10 days on/10 days off, 0.75 mg/kg prednisone regime has been investigated in a group of 35 DMD patients [14]. The treatment was initiated during the ambulant phase and the median period of treatment was 27 months.…”

Section: Steroid Treatment In Duchenne Muscular Dystrophymentioning

confidence: 99%