Details:

Alternative Title:
Muscle Nerve
Publisher's site:
http://dx.doi.org/10.1002/mus.27490
Personal Author:
Butterfield, Russell J ; Krikov, Sergey ; Conway, Kristin M. ; Johnson, Nicholas ; Matthews, Dennis ; ... More +
Butterfield, Russell J ; Krikov, Sergey ; Conway, Kristin M. ; Johnson, Nicholas ; Matthews, Dennis ; Phan, Han ; Cai, Bo ; Paramsothy, Pangaja ; Thomas, Shiny ; Feldkamp, Marcia L. ; Less -
Description:
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 hazards ratios (HR)s 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 year beyond LOA compared to those with no corticosteroid use or those who stopped corticosteroid use within 1 year 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.
Subject:
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Adrenal Cortex Hormones
Article
Cardiomyopathy
Corticosteroid
Duchenne Muscular Dystrophy
Humans
Male
Muscular Dystrophy, Duchenne
Proportional Hazards Models
Prospective Studies
Walking

Source:
Muscle Nerve. 66(1):15-23 ;
Pubmed ID:
34994466
Pubmed Central ID:
PMC9197945
Document Type:
Journal Article ;
Funding:
DD000392/CC/CDC HHSUnited States/ ; DD000191/CC/CDC HHSUnited States/ ; K08 NS097631/NS/NINDS NIH HHSUnited States/ ; U01 DD001247/DD/NCBDD CDC HHSUnited States/ ; DD000190/CC/CDC HHSUnited States/ ; ... More +
DD000392/CC/CDC HHSUnited States/ ; DD000191/CC/CDC HHSUnited States/ ; K08 NS097631/NS/NINDS NIH HHSUnited States/ ; U01 DD001247/DD/NCBDD CDC HHSUnited States/ ; DD000190/CC/CDC HHSUnited States/ ; DD000187/CC/CDC HHSUnited States/ ; U01 DD001248/DD/NCBDD CDC HHSUnited States/ ; DD000189/CC/CDC HHSUnited States/ ; DD000187/CC/CDC HHSUnited States/ ; DD000189/CC/CDC HHSUnited States/ ; DD000190/CC/CDC HHSUnited States/ ; DD000191/CC/CDC HHSUnited States/ ; DD000392/CC/CDC HHSUnited States/ ; K08 NS097631/NS/NINDS NIH HHSUnited States/ ; Less -
Collection(s):
CDC Public Access
Main Document Checksum:
[+]
urn:sha256:d1c1e59f00e8409d75734315ae678956becad1e6853a77a3e38589488f1f1046
File Type:

• Supporting Files:
  No Additional Files

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