A novel treatment regimen for Duchenne muscular dystrophy

Abstract

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.

Superpulsed Low-Level Laser Therapy Protects Skeletal Muscle of mdx Mice against Damage, Inflammation and Morphological Changes Delaying Dystrophy Progression

“…Furthermore, CK activity was significantly lower in the LLLT group compared with the placebo-control group and this suggests that LLLT reduced the progression of muscle damage. Reduction of CK activity has also been reported after successful glucocorticoid therapy and is associated with clinical benefit [52].…”

Efficacy and safety of glucocorticoids in the treatment of progressive muscular dystrophy in children: a systematic review and meta-analysis

“…Progressive muscular dystrophy (PMD), also known as Duchenne muscular dystrophy (DMD), is a group of muscular degeneration caused by genetic mutations. The main clinical manifestations are progressive muscle atrophy with central nervous system, cardiac, skeletal, respiratory, and gastrointestinal involvement (1,2). Progressive muscular dystrophy is a chronic disease, most children around the age of 3 to 5 years of onset, gradually appear some clinical symptoms.…”

Efeitos do Tempol, um mimético da superóxido dismutase, em fibras musculares distróficas de camundongos mdx

“…Ainda, no grupo mdxT apresentou redução significativa na densidade de microvasos em relação ao grupo mdxP (47,7%) (Figura 23-B). A Prednisona é um glicocorticoide sintético que vem sendo vastamente investigado e aceito como tratamento para a DMD por apresentar excelentes resultados na melhora da condição danosa destes pacientes (Brooke, Fenichel et al 1987, Fenichel, Florence et al 1991, Li, Cai et al 2013. Porém a Prednisona pode apresentar efeitos adversos como, ganho de peso, desenvolvimento de catarata e glaucoma, retardo no desenvolvimento ósseo e surgimento de osteoporose, decorrente da perda de osso trabecular, e alterações no metabolismo da glicose, com indução ou agravamento de quadro de diabetes, quando administrados por um longo período (Ricotti, Ridout et al 2013).…”