Journal article
Evaluation of effects of continued corticosteroid treatment on cardiac and pulmonary function in non‐ambulatory males with Duchenne muscular dystrophy from MD STARnet
Muscle & nerve, Vol.66(1), pp.15-23
07/2022
DOI: 10.1002/mus.27490
PMCID: PMC9197945
PMID: 34994466
Abstract
Introduction/Aims
Corticosteroids have been shown to improve muscle strength and delay loss of ambulation (LOA) in Duchenne muscular dystrophy (DMD) and are considered standard of care despite significant side‐effects. The objective of this study is to evaluate whether corticosteroid treatment after LOA is beneficial for cardiac or pulmonary functions among boys with DMD.
Methods
We used the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) to characterize associations between corticosteroid use and onset of abnormal left ventricular (LV) function or abnormal percent predicted forced vital capacity (ppFVC) among 398 non‐ambulatory boys with DMD. Kaplan–Meier curve estimation was used to compare time to onset by corticosteroid use groups; Cox proportional hazards modeling was used to estimate hazard ratios (HRs) and corresponding 95% confidence intervals.
Results
We found no differences in time to onset of abnormal LV function by corticosteroid use groups. We observed a longer time from LOA to first abnormal ppFVC in boys that were treated with corticosteroid ≥1 y beyond LOA compared with those with no corticosteroid use or those who stopped corticosteroid use within 1 y of LOA.
Discussion
Our findings show no association of corticosteroid use beyond LOA with the onset of abnormal LV function, but a significant association with a delay in onset of abnormal ppFVC. Prospective studies of corticosteroid use in boys with DMD who have lost ambulation may identify benefits and can better elucidate risks, allowing for more effective counseling of patients on continuing treatment after LOA.
See Editorial on pages 1‐2 in this issue.
Details
- Title: Subtitle
- Evaluation of effects of continued corticosteroid treatment on cardiac and pulmonary function in non‐ambulatory males with Duchenne muscular dystrophy from MD STARnet
- Creators
- Russell J. Butterfield - University of UtahSergey Kirkov - University of UtahKristin M. Conway - University of IowaNicholas Johnson - Virginia Commonwealth UniversityDennis Matthews - University of Colorado BoulderHan Phan - National Center on Birth Defects and Developmental DisabilitiesBo Cai - University of South CarolinaPangaja Paramsothy - National Center on Birth Defects and Developmental DisabilitiesShiny Thomas - New York State Department of HealthMarcia L. Feldkamp - University of Utah
- Resource Type
- Journal article
- Publication Details
- Muscle & nerve, Vol.66(1), pp.15-23
- DOI
- 10.1002/mus.27490
- PMID
- 34994466
- PMCID
- PMC9197945
- NLM abbreviation
- Muscle Nerve
- ISSN
- 0148-639X
- eISSN
- 1097-4598
- Publisher
- John Wiley & Sons, Inc
- Number of pages
- 9
- Grant note
- National Institute of Neurological Disorders and Stroke (K08 NS097631) Centers for Disease Control and Prevention (DD000187; DD000189; DD000190; DD000191; DD000392)
- Language
- English
- Date published
- 07/2022
- Academic Unit
- Epidemiology
- Record Identifier
- 9984627220502771
Metrics
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