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Recent advances of glucocorticoids in the treatment of Duchenne muscular dystrophy (Review).

ABSTRACT: Duchenne muscular dystrophy (DMD) is the most common degenerative neuromuscular disease. The incidence of DMD in live births is 1/3,600-1/6,000. Although glucocorticoid-dependent medication is the mainstay treatment option for DMD, a standard treatment regimen has yet to be determined. The present review discusses the literature on the timing, methods and courses of glucocorticoid treatment for DMD. The review highlights the importance of the immediate commencement of glucocorticoid treatment following the diagnosis of DMD, with weekend-only administration being advantageous. Adherence to long-term single-glucocorticoid therapy can delay the loss of ambulation ability, and the side effects of the treatment are controllable. However, the standard medication for patients of different ages and stages of disease development, and the use of combination therapy require further investigation.

SUBMITTER: Zhang T

PROVIDER: S-EPMC7967797 | biostudies-literature | 2021 May

REPOSITORIES: biostudies-literature

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Recent advances of glucocorticoids in the treatment of Duchenne muscular dystrophy (Review).

Zhang Tianyuan T Kong Xiangdong X

Experimental and therapeutic medicine 20210301 5

Duchenne muscular dystrophy (DMD) is the most common degenerative neuromuscular disease. The incidence of DMD in live births is 1/3,600-1/6,000. Although glucocorticoid-dependent medication is the mainstay treatment option for DMD, a standard treatment regimen has yet to be determined. The present review discusses the literature on the timing, methods and courses of glucocorticoid treatment for DMD. The review highlights the importance of the immediate commencement of glucocorticoid treatment fo ...[more]

PMID: 33777191

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Project description:BackgroundDuchenne Muscular Dystrophy (DMD) is a rapidly progressive, lethal neuromuscular disorder, present from birth, which occurs almost exclusively in males. We have reviewed contemporary evidence of burden, epidemiology, illness costs and treatment patterns of DMD. This systematic review adhered to published methods with information also sought from the web and contacting registries. Searches were carried out from 2005 to June 2015. The population of interest was individuals with clearly defined DMD or their carers.ResultsNine thousand eight hundred fifty titles were retrieved from searches. Fifty-eight studies were reviewed with three assessed as high, 33 as medium and 22 as low quality. We found two studies reporting birth and four reporting point prevalence, three reporting mortality, 41 reporting severity and/or progression, 18 reporting treatment patterns, 12 reporting quality of life, two reporting utility measures, three reporting costs of illness and three treatment guidelines. Birth prevalence ranged from 15.9 to 19.5 per 100,000 live births. Point prevalence per 100,000 males was for France, USA, UK and Canada, 10.9, 1.9, 2.2 and 6.1 respectively. A study of adult DMD patients at a centre in France found median survival for those born between 1970 and 1994 was 40.95 years compared to 25.77 years for those born between 1955 and 1969. Loss of ambulation occurred at a median age of 12 and ventilation starts at about 20 years. There was international variation in use of corticosteroids, scoliosis surgery, ventilation and physiotherapy. The economic cost of DMD climbs dramatically with disease progression - rising as much as 5.7 fold from the early ambulatory phase to the non-ambulatory phase in Germany.ConclusionsThis is the first systematic review of treatment, progression, severity and quality of life in DMD. It also provides the most recent description of the burden, epidemiology, illness costs and treatment patterns in DMD. There are evidence gaps, particularly in prevalence and mortality. People with DMD seem to be living longer, possibly due to corticosteroid use, cardiac medical management and ventilation. Future research should incorporate registry data to improve comparability across time and between countries and to investigate the quality of life impact as the condition progresses.

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Recent advances of glucocorticoids in the treatment of Duchenne muscular dystrophy (Review).

Publications

Recent advances of glucocorticoids in the treatment of Duchenne muscular dystrophy (Review).

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