Journal article
Selected clinical and demographic factors and all-cause mortality among individuals with Duchenne muscular dystrophy in the Muscular Dystrophy Surveillance, Tracking, and Research Network
Neuromuscular disorders : NMD, Vol.32(6), pp.468-476
04/30/2022
DOI: 10.1016/j.nmd.2022.04.008
PMCID: PMC9214635
PMID: 35597713
Abstract
•Estimated 50% survival until age 23.7 years and 12.5 years after loss of ambulation•After loss of ambulation, glucocorticoid users had lower risk of death than non-users•Lower risk of death observed among ever-users of cough assist and scoliosis surgery•Non-Hispanic Black males had higher risk of death than Non-Hispanic White males•Cardiac medication and bilevel positive airway pressure were not associated with death
Population-based estimates of survival among individuals with Duchenne muscular dystrophy (DMD) living in the United States are lacking. It is also unclear whether the association between glucocorticoid use and all-cause mortality persists in the context of other common treatments (cardiac medication, cough-assist, bilevel positive airway pressure, and scoliosis surgery) observed to delay mortality. Among 526 individuals identified by the Muscular Dystrophy Surveillance, Tracking, and Research Network, the estimated median survival time from birth was 23.7 years. Current glucocorticoid users had a lower hazard of mortality than non-users. Individuals who ever had scoliosis surgery had a lower hazard of mortality than individuals who did not have scoliosis surgery. Individuals who ever used cough assist had a lower hazard of mortality than individuals who never used cough assist. Non-Hispanic Black individuals had a higher hazard of mortality than non-Hispanic White individuals. No differences in hazards of mortality were observed between ever versus never use of cardiac medication and ever versus never use of bilevel positive airway pressure. The glucocorticoid observation is consistent with the 2018 Care Considerations statement that glucocorticoid use continues in the non-ambulatory phase. Our observations may inform the clinical care of individuals living with DMD.
Details
- Title: Subtitle
- Selected clinical and demographic factors and all-cause mortality among individuals with Duchenne muscular dystrophy in the Muscular Dystrophy Surveillance, Tracking, and Research Network
- Creators
- Pangaja Paramsothy - National Center on Birth Defects and Developmental DisabilitiesYinding Wang - McKing Consulting Corporation Consultant to Centers for Disease Control and Prevention, 2900 Chamblee Tucker Rd. Building 10, Ste. 100. Atlanta, GA 30341, United States of AmericaBo Cai - University of South CarolinaKristin M Conway - Department of Epidemiology, College of Public Health, The University of Iowa, 145 N Riverside Drive, S416 CPHB, Iowa City, IA 52242, United States of AmericaNicholas E Johnson - Virginia Commonwealth UniversityShree Pandya - University of RochesterEmma Ciafaloni - University of RochesterKatherine D Mathews - Departments of Pediatrics and Neurology, University of Iowa Carver College of Medicine, 200 Hawkins Dr., Iowa City, IA 52242, United StatesPaul A Romitti - Department of Epidemiology, College of Public Health, The University of Iowa, 145 N Riverside Drive, S416 CPHB, Iowa City, IA 52242, United States of AmericaJames F Howard - University of North Carolina at Chapel HillCatharine Riley - National Center on Birth Defects and Developmental Disabilities
- Resource Type
- Journal article
- Publication Details
- Neuromuscular disorders : NMD, Vol.32(6), pp.468-476
- DOI
- 10.1016/j.nmd.2022.04.008
- PMID
- 35597713
- PMCID
- PMC9214635
- NLM abbreviation
- Neuromuscul Disord
- ISSN
- 0960-8966
- eISSN
- 1873-2364
- Publisher
- Elsevier B.V
- Grant note
- DOI: 10.13039/100006087, name: National Center on Birth Defects and Developmental Disabilities
- Language
- English
- Date published
- 04/30/2022
- Academic Unit
- Neurology; Stead Family Department of Pediatrics; Epidemiology; Iowa Neuroscience Institute; Biostatistics; Neurology (Pediatrics)
- Record Identifier
- 9984258956402771
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