Long‐term benefit from prednisone therapy in Duchenne muscular dystrophy

Fenichel, Gerald M.; Florence, J.; Pestronk, Alan; Mendell, Jerry R.; Moxley, Richard T.; Griggs, Robert C.; Brooke, Michael H.; Miller, J. Philip; Robison, Justin; King, Wendy; Signore, L.; Pandya, Shree; Schierbecker, Jeanine; Wilson, Brad

doi:10.1212/wnl.41.12.1874

Cited by 234 publications

(136 citation statements)

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“…Although the effects of CLA in humans remain controversial, two studies have found that CLA reduced body fat mass in overweight humans, 1,13 suggesting that it may be an adjunctive therapy for attenuating corticosteroid-induced fat gain in boys with DMD. 2,10 The ability of CLA, CrM, and ␣-LA to significantly reduce the retroperitoneal fat pad weight may ultimately prove helpful in attenuating body fat gains in DMD patients treated with corticosteroids.…”

Section: Discussionmentioning

confidence: 99%

“…9,41 There are few therapeutic options available to boys with DMD; however, corticosteroid treatment has resulted in improved function and strength. 2,10,30 The exact mechanism by which corticosteroids exert their beneficial effect is unclear, but does not appear to involve a direct suppression of the inflammatory response. 16 Unfortunately, corticosteroids produce well-known and often serious side effects, 2,10,30 and alternative therapeutic strategies are therefore important.…”

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confidence: 99%

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Nutritional therapy improves function and complements corticosteroid intervention in mdx mice

et al. 2005

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Corticosteroid therapy for Duchenne muscular dystrophy is effective but associated with long-term side effects. To determine the potential therapeutic benefit from four nutritional compounds (creatine monohydrate, conjugated linoleic acid, ␣-lipoic acid, and ␤-hydroxy-␤-methylbutyrate) alone, in combination, and with corticosteroids (prednisolone), we evaluated the effects on several variables in exercising mdx mice. Outcome measures included grip strength, rotarod performance, serum creatine kinase levels, muscle metabolites, internalized myonuclei, and retroperitoneal fat pad weight. In isolation, each nutritional treatment showed some benefit, with the combination therapy showing the most consistent benefits. Prednisolone and the combination therapy together provided the most consistent evidence of efficacy; increased peak grip strength (P Ͻ 0.05), decreased grip strength fatigue (P Ͻ 0.05), decreased number of internalized myonuclei (P Ͻ 0.01), and smaller retroperitoneal fat pad stores (P Ͻ 0.001). This study provided evidence for therapeutic benefit from a four-compound combination therapy alone, and in conjunction with corticosteroids in the mdx model of DMD.

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Section: Discussionmentioning

confidence: 99%

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confidence: 99%

Nutritional therapy improves function and complements corticosteroid intervention in mdx mice

et al. 2005

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“…There are no curative therapies, but multiple independent clinical trials since 1974 [3] and meta-analyses firmly establish that chronic corticosteroids at a dose equivalent to 0.75/mg/kg/day of prednisone slow the progression of muscle weakness [4][5][6][7][8][9][10].…”

Section: Introductionmentioning

confidence: 99%

Online Self-Report Data for Duchenne Muscular Dystrophy confirms natural history and can be used to assess for therapeutic benefits

Wang

Fadlon

Ulm

et al. 2014

Preprint

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To assess the utility of online patient self-report outcomes in a rare disease, we attempted to observe the effects of corticosteroids in delaying age at fulltime wheelchair use in Duchenne muscular dystrophy (DMD) using data from 1,057 males from DuchenneConnect, an online registry. Data collected were compared to prior natural history data in regard to age at diagnosis, mutation spectrum, and age at loss of ambulation. Because registrants reported differences in steroid and other medication usage, as well as age and ambulation status, we could explore these data for correlations with age at loss of ambulation. Using multivariate analysis, current steroid usage was the most significant and largest independent predictor of improved wheelchair-free survival. Thus, these online self-report data were sufficient to retrospectively observe that current steroid use by patients with DMD is associated with a delay in loss of ambulation. Comparing commonly used steroid drugs, deflazacort prolonged ambulation longer than prednisone (median 14 years and 13 years, respectively). Further, use of Vitamin D and Coenzyme Q10, insurance status, and age at diagnosis after 4 years were also significant, but smaller, independent predictors of longer wheelchair-free survival. Nine other common supplements were also individually tested but had lower study power. This study demonstrates the utility of DuchenneConnect data to observe therapeutic differences, and highlights needs for improvement in quality and quantity of patient-report data, which may allow exploration of drug/therapeutic practice combinations impractical to study in clinical trial settings.

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“…More recently the EK scale [20] was developed for non-ambulatory patients with Duchenne's muscular dystrophy (DMD) and SMA, the Wee Fim was utilized to quantify function in children with SMA in Hong Kong [11] and the functional research scale for ALS (FRS-ALS) scale was utilized in clinical trials with adult patients with ALS [21,22]. These scales are typically used as primary outcome measures for a treatment trial only when direct measure of power is not possible, though their use as a secondary measure is common [5,6,[23][24][25][26]. Though each of these tests were developed to assess functional skills in weak patients with neuromuscular disorders, construct of test items often precludes their use in assessing younger children.…”

Section: Introductionmentioning

confidence: 99%

A modified Hammersmith functional motor scale for use in multi-center research on spinal muscular atrophy

Krosschell

Maczulski²,

Crawford

et al. 2006

Neuromuscular Disorders

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The Hammersmith functional motor scale for children with spinal muscular atrophy was modified to establish a standard measure of functional ability in children with non-ambulant spinal muscular atrophy types 2 and 3 in a longitudinal multi-center clinical trial. This study assessed the intra-and interrater reliability and the test-retest stability of a modified version of the scale. Both intra-and interrater reliability were established. Results indicate that the scale is reliable and stable over a 6 month period. Reliability was maintained when patient sample criteria were expanded to include children younger than 30 months and children with popliteal angles greater than 20°. These data establish the modified Hammersmith functional motor scale for children with spinal muscular atrophy as a reliable instrument for use in multi-center treatment trials in non-ambulant spinal muscular atrophy children. Our data provides additional support for the use of original scale items in terms of ease of administration, usefulness and reliability, while incorporating modifications to optimize its use in a multi-center clinical research setting.

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Long‐term benefit from prednisone therapy in Duchenne muscular dystrophy

Cited by 234 publications

References 0 publications

Nutritional therapy improves function and complements corticosteroid intervention in mdx mice

Nutritional therapy improves function and complements corticosteroid intervention in mdx mice

Online Self-Report Data for Duchenne Muscular Dystrophy confirms natural history and can be used to assess for therapeutic benefits

A modified Hammersmith functional motor scale for use in multi-center research on spinal muscular atrophy

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