Prolonged Survival and End-of-Life Care Among End-Stage Patients With Duchenne Muscular Dystrophy (DMD)

Birnkrant, Jennifer M.; Bennett, David S.; Noritz, Garey; Harrington, Michael A.; Birnkrant, David J.

doi:10.1378/chest.1114744

Chest

2011

DOI: 10.1378/chest.1114744

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Prolonged Survival and End-of-Life Care Among End-Stage Patients With Duchenne Muscular Dystrophy (DMD)

Jennifer M. Birnkrant¹,

David S. Bennett²,

Garey Noritz³

et al.

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“…Although there is presently no cure for DMD, advancements to the standard of care, including the introduction of systemic corticosteroids in the 1990s, have helped slow disease progression and improve survival [10][11][12]. However, the impact of these changes in standard of care across the full range of clinically-relevant disease progression milestones experienced by those with DMD has not been fully characterized.…”

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confidence: 99%

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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mentioning

confidence: 99%

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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Prolonged Survival and End-of-Life Care Among End-Stage Patients With Duchenne Muscular Dystrophy (DMD)

Cited by 1 publication

References 0 publications

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

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

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