Deflazacort Use in Duchenne Muscular Dystrophy: An 8-Year Follow-Up

Houde, Sylvie; Filiatrault, Michèle; Fournier, Anne; Dubé, Julie; D’Arcy, Sylvie; Bérubé, Denis; Brousseau, Yves; Lapierre, G.; Vanasse, Michel

doi:10.1016/j.pediatrneurol.2007.11.001

Cited by 147 publications

(134 citation statements)

References 30 publications

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“…The use of differing treatment regimens and medications exists within these studies (21,25,26); however, the overall results appear positive regardless of the regimen used. There is little consensus on the exact dose of steroids required.…”

Section: Interventions Before Surgerymentioning

confidence: 97%

“…There is evidence that using steroids in patients with DMD can have numerous positive effects including prolonged independent ambulation, decreased progression of scoliosis, increased muscle strength, respiratory function, cardiac function and quality of life (18,(21)(22)(23)(24)(25)(26). The use of steroid therapy has been associated with a reduction in the incidence of scoliosis in DMD because prolonging ambulation means that some boys will undergo their pubertal growth spurt before loss of ambulation.…”

Section: Interventions Before Surgerymentioning

confidence: 99%

“…There is also a risk of vertebral compression fractures related to cumulative dose for those boys on steroid treatment (30). Other potential complications include the development of cataracts (24,26), the risk of spine and long bone fractures (22,25), and a decreased bone density (25). With regards to cataracts, both articles, while reporting differing incidence rates of cataracts, noted that those that required surgical correction for this was minimal (24,26).…”

Section: Interventions Before Surgerymentioning

confidence: 99%

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Duchenne muscular dystrophy: the management of scoliosis

et al. 2016

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This study summaries the current management of scoliosis in patients with Duchenne Muscular Dystrophy.A literature review of Medline was performed and the collected articles critically appraised. This literature is discussed to give an overview of the current management of scoliosis within Duchenne Muscular Dystrophy.Importantly, improvements in respiratory care, the use of steroids and improving surgical techniques have allowed patients to maintain quality of life and improved life expectancy in this patient group.

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Section: Interventions Before Surgerymentioning

confidence: 97%

Section: Interventions Before Surgerymentioning

confidence: 99%

Section: Interventions Before Surgerymentioning

confidence: 99%

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Duchenne muscular dystrophy: the management of scoliosis

et al. 2016

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“…[17][18][19] Osteopenia was common in boys with DMD, and the added effect of oral steroids increased the incidence of osteoporosis and vertebral and other fractures. 20,21 The bone mineral density of these children was measured by dual-energy X-ray absorptiometry In children with joint hypermobility, heritable disorders of connective tissue, such as Ehlers-Danlos syndrome, were considered.…”

Section: Skeletalmentioning

confidence: 99%

Manejo de longo prazo em crianças com transtornos neuromusculares

Strehle¹

2009

J. Pediatr. (Rio J.)

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Objective: Duchenne muscular dystrophy is the commonest genetic myopathy but there exist a large number of inherited neuromuscular diseases which individually are very rare and where clinical information is not widely available. This review is based on the author's experience in a pediatric muscle clinic and provides practical guidance and treatment plans for frequently encountered problems. Sources:A MEDLINE search was conducted to retrieve recent articles relevant to the management of children with inherited myopathies and neuropathies. A patient cohort (n = 200) was evaluated using descriptive statistics. Summary of the findings:Duchenne muscular dystrophy accounted for almost half of the diagnoses, followed by spinal muscular atrophy (12%), Becker muscular dystrophy and myotonic dystrophy (7% each). Sixteen patients (9%) had an unknown myopathy. Conclusions:As with other chronic illnesses, these patients should be regularly reviewed by health professionals from an early age to increase life expectancy and improve quality of life. It is useful for physicians to take a structured approach when looking after children with neuromuscular disorders and to monitor all affected organ systems. J Pediatr (Rio J). 2009;85(5): 379 IntroductionInherited neuromuscular disorders (NMDs) affect approximately one in 3,500 children worldwide, and Xlinked Duchenne muscular dystrophy (DMD) has the highest incidence among them. Many conditions present with muscle weakness and wasting and can be diagnosed by genetic mutation analysis alone, but muscle, skin or nerve biopsy still remain important diagnostic tools. Although various strategies for genetic therapies of these diseases are being explored, the cornerstones of current treatment are symptomatic and supportive healthcare. 1,2 Comprehensive reviews on specific NMDs have been published but it can be a challenge for the novice or the busy clinician to keep up to date with the latest literature when looking after children suffering from a wide range of rare disorders. [3][4][5] However, when monitoring these chronically ill patients over a prolonged period, certain recurring themes can be observed that are pertinent to most NMDs. These themes can be ascribed to the specific muscle or nerve pathology and its effect on other systems of the body.Here, the author describes his experience of following up 200 children presenting to a pediatric muscle clinic during a 3-year period. Long-term management of children with neuromuscular disordersEugen-Matthias Strehle* 380 Jornal de Pediatria -Vol. 85, No. 5, 2009 Disease n (%) Becker muscular dystrophy 12 (7)Myotonic dystrophy 11 (7)Merosin-negative congenital muscular dystrophy 5 The remaining 170 patients presented with 18 different diagnoses ( Table 1). Systems MuscularAt each 1-hour assessment a detailed medical history with emphasis on mobility covering the preceding months was obtained, and any concerns raised by the parent/caregiver or patient were addressed. Muscle strength was measured SkeletalDespite regular physi...

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“…Steroid therapy improves muscle strength within two to four months after beginning the treatment, and it helps to maintain independent walking for at least two more years 13 . In addition, steroid therapy has a positive and significant impact on quality of life (QoL) and on the cost/ benefit relationship of medical care 14,15 . In addition to steroid therapy, non-invasive mechanical ventilation methods are important palliative approaches involving QoL 16 .…”

mentioning

confidence: 99%

Duchenne muscular dystrophy: quality of life among 95 patients evaluated using the Life Satisfaction Index for Adolescents

Simon

Resende

Simon

et al. 2011

Arq. Neuro-Psiquiatr.

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The purpose of this study was to evaluate the quality of life (QoL) of patients with Duchenne muscular dystrophy (DMD) in different stages of the disease, by means of the Life Satisfaction Index for Adolescents (LSI-A). The practicality of this scale was also verified. The LSI-A was applied four times to 95 patients with DMD who were undergoing steroid therapy, at three-month intervals. The patients were divided into four groups according to age. The results from the four applications and the inter and intra-examiner concordance were treated statistically. Comparing the different age groups, patients with DMD did not lose QoL, even with disease progression. We concluded that, in spite of the progressive course of the disease, the QoL in patients with DMD does not get worse. The use of a scale that embraces a great diversity of circumstances in patients' lives, without considering clinical aspects excessively, is a good alternative for assessing the QoL of these patients. Key words: quality of life, Duchenne muscular dystrophy, steroid therapy, questionnaire, Life Satisfaction Index for Adolescents. Distrofia muscular de Duchenne: avaliação da qualidade de vida em 95 pacientes através do Life Satisfaction Index for AdolescentsRESUMO O objetivo deste estudo foi de quantificar a qualidade de vida (QV) em crianças com distrofia muscular de Duchenne (DMD) em diferentes idades através do uso do questionário Life Satisfaction Index for Adolescents (LSI-A). Foi também avaliada a praticidade do questionário. O LSI-A foi aplicado a 95 pacientes com distrofia muscular de Duchenne em corticoterapia, em diferentes idades, e por quatro vezes com intervalos de três meses. Os resultados concernentes às quatro avaliações e a concordância inter e intra-observador foram tratados estatisticamente. Comparando diferentes faixas etárias, mesmo ao longo da progressão da doença, não notamos perda da QV. Concluímos que por não valorizar excessivamente os aspectos clínicos e abranger uma diversidade de circunstâncias cotidianas, O LSI-A é útil na avaliação da QV das crianças com DMD, sendo também de fácil aplicação. Palavras-chave: qualidade de vida, distrofia muscular de Duchenne, corticoterapia, questionário, Life Satisfaction Index for Adolescents.

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Deflazacort Use in Duchenne Muscular Dystrophy: An 8-Year Follow-Up

Cited by 147 publications

References 30 publications

Duchenne muscular dystrophy: the management of scoliosis

Duchenne muscular dystrophy: the management of scoliosis

Manejo de longo prazo em crianças com transtornos neuromusculares

Duchenne muscular dystrophy: quality of life among 95 patients evaluated using the Life Satisfaction Index for Adolescents

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