Health profile of a cohort of adults with Duchenne muscular dystrophy

Pandya, Shree; James, Katherine A.; Westfield, Christina; Thomas, Shiny; Fox, Deborah; Ciafaloni, Emma; Moxley, Richard T.

doi:10.1002/mus.26129

Cited by 15 publications

(29 citation statements)

References 32 publications

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“…Additional impacts include worsening cardiac and pulmonary function, as well as a deterioration in bone health; over time, these disabling declines collectively contribute to mortality by early adulthood. The median survival for boys with DMD is 21.8 years [4][5][6][7][8].…”

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confidence: 99%

Real-world outcomes of long-term prednisone and deflazacort use in patients with Duchenne muscular dystrophy: experience at a single, large care center

et al. 2020

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Aim: To assess outcomes among patients with Duchenne muscular dystrophy receiving deflazacort or prednisone in real-world practice. Methods: Clinical data for 435 boys with Duchenne muscular dystrophy from Cincinnati Children’s Hospital Medical Center were studied retrospectively using time-to-event and regression analyses. Results: Median ages at loss of ambulation were 15.6 and 13.5 years among deflazacort- and prednisone-initiated patients, respectively. Deflazacort was also associated with a lower risk of scoliosis and better ambulatory function, greater % lean body mass, shorter stature and lower weight, after adjusting for age and steroid duration. No differences were observed in whole body bone mineral density or left ventricular ejection fraction. Conclusion: This single center study adds to the real-world evidence associating deflazacort with improved clinical outcomes.

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confidence: 99%

Real-world outcomes of long-term prednisone and deflazacort use in patients with Duchenne muscular dystrophy: experience at a single, large care center

et al. 2020

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“…One study reported the mean age at scoliosis [ 38 ], 2 studies the median age at scoliosis [ 31 , 35 ], and 5 studies the percentage with scoliosis by age (Table 2 ) [ 22 , 30 , 31 , 35 , 37 ]. How scoliosis was defined varied across studies.…”

Section: Resultsmentioning

confidence: 99%

“…3 a) [ 38 ]. The median (range) age at scoliosis surgery among a mixed corticosteroid use sample from MD STARnet was 14.6 (10.2–20.2) years (with surgery observed in 52.4% of n = 208) [ 35 ]. In the remaining study of 274 corticosteroid-treated patients (also from MD STARnet), the median (range) age (by spinal curvature > 30° or surgery) was 14.2 (12.5–15.6) years among the 107 patients with scoliosis [ 31 ].…”

Section: Resultsmentioning

confidence: 99%

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The clinical course of Duchenne muscular dystrophy in the corticosteroid treatment era: a systematic literature review

Szabo¹,

Salhany

Deighton³

et al. 2021

Orphanet J Rare Dis

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Background Duchenne muscular dystrophy (DMD) is a severe rare progressive inherited neuromuscular disorder, leading to loss of ambulation (LOA) and premature mortality. The standard of care for patients with DMD has been treatment with corticosteroids for the past decade; however a synthesis of contemporary data describing the clinical course of DMD is lacking. The objective was to summarize age at key clinical milestones (loss of ambulation, scoliosis, ventilation, cardiomyopathy, and mortality) in the corticosteroid-treatment-era. Methods A systematic review was conducted using MEDLINE and EMBASE. The percentage experiencing key clinical milestones, and the mean or median age at those milestones, was synthesized from studies from North American populations, published between 2007 and 2018. Results From 5637 abstracts, 29 studies were included. Estimates of the percentage experiencing key clinical milestones, and age at those milestones, showed heterogeneity. Up to 30% of patients lost ambulation by age 10 years, and up to 90% by 15 years of age. The mean age at scoliosis onset was approximately 14 years. Ventilatory support began from 15 to 18 years, and up to half of patients required ventilation by 20 years of age. Registry-based estimates suggest that 70% had evidence of cardiomyopathy by 15 years and almost all by 20 years of age. Finally, mortality rates up to 16% by age 20 years were reported; among those surviving to adulthood mortality was up to 60% by age 30 years. Conclusions Contemporary natural history studies from North America report that LOA on average occurs in the early teens, need for ventilation and cardiomyopathy in the late teens, and death in the third or fourth decade of life. Variability in rates may be due to differences in study design, treatment with corticosteroids or other disease-modifying agents, variations in clinical practices, and dystrophin mutations. Despite challenges in synthesizing estimates, these findings help characterize disease progression among contemporary North American DMD patients.

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“…In their article, Pandya et al assessed the health profiles of 208 adults with Duchenne muscular dystrophy (DMD) in 5 states in the USA. Many of the interventions with this condition are needed in the second decade, a critical period as individuals transition from adolescence to adulthood and switch from having pediatric to adult providers.…”

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confidence: 99%

Critical evaluation of the treatment for duchenne muscular dystrophy during the transition from adolescence to adulthood

Fiorentino

Esquinas

2018

Muscle and Nerve

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Health profile of a cohort of adults with Duchenne muscular dystrophy

Abstract: These population-based data provide critical information about late-stage health profiles among adults with DMD for developing appropriate models of care. Muscle Nerve 58: 219-223, 2018.

Cited by 15 publications

References 32 publications

Real-world outcomes of long-term prednisone and deflazacort use in patients with Duchenne muscular dystrophy: experience at a single, large care center

Real-world outcomes of long-term prednisone and deflazacort use in patients with Duchenne muscular dystrophy: experience at a single, large care center

The clinical course of Duchenne muscular dystrophy in the corticosteroid treatment era: a systematic literature review

Critical evaluation of the treatment for duchenne muscular dystrophy during the transition from adolescence to adulthood

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