Evaluation of effects of continued corticosteroid treatment on cardiac and pulmonary function in non-ambulatory males with Duchenne Muscular Dystrophy from MD STARnet

Details

• Alternative Title:
  Muscle Nerve
• Personal Author:
  Butterfield, Russell J ; Krikov, Sergey ; Conway, Kristin M. ; Johnson, Nicholas ; Matthews, Dennis ; Phan, Han ; Cai, Bo ; Paramsothy, Pangaja ; Thomas, Shiny ; Feldkamp, Marcia L.
• 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.
  
  
• Subjects:
  Adrenal Cortex Hormones Article Cardiomyopathy Corticosteroid Duchenne Muscular Dystrophy Humans Male Muscular Dystrophy, Duchenne Proportional Hazards Models Prospective Studies Walking

  More +
• Source:
  Muscle Nerve. 66(1):15-23
• Pubmed ID:
  34994466
• Pubmed Central ID:
  PMC9197945
• Document Type:
  Journal Article
• Funding:
  DD000392/CC/CDC HHSUnited States/ ; U01 DD000187/DD/NCBDD CDC HHSUnited States/ ; R21 TR003184/TR/NCATS NIH HHSUnited States/ ; U01 DD001246/DD/NCBDD CDC HHSUnited States/ ; U01 DD000189/DD/NCBDD CDC HHSUnited States/ ; U01 DD001247/DD/NCBDD CDC HHSUnited States/ ; U01DD001119/ACL/ACL HHSUnited States/ ; R01 FD006071/FD/FDA HHSUnited States/ ; U01 DD000191/DD/NCBDD CDC HHSUnited States/ ; U01 DD001248/DD/NCBDD CDC HHSUnited States/ ; DD000189/CC/CDC HHSUnited States/ ; U01 DD000190/DD/NCBDD CDC HHSUnited States/ ; U01DD001117/ACL/ACL HHSUnited States/ ; U01 DD001117/DD/NCBDD CDC HHSUnited States/ ; U01 DD000392/DD/NCBDD CDC HHSUnited States/ ; U01 DD001126/DD/NCBDD CDC HHSUnited States/ ; U01 DD001123/DD/NCBDD CDC HHSUnited States/ ; R01 NS104010/NS/NINDS NIH HHSUnited States/ ; DD000191/CC/CDC HHSUnited States/ ; K08 NS097631/NS/NINDS NIH HHSUnited States/ ; U01DD001123/ACL/ACL HHSUnited States/ ; U01DD001108/ACL/ACL HHSUnited States/ ; DD000190/CC/CDC HHSUnited States/ ; DD000187/CC/CDC HHSUnited States/ ; CC999999/ImCDC/Intramural CDC HHSUnited States/ ; U01DD001126/ACL/ACL HHSUnited States/ ; U01 DD001249/DD/NCBDD CDC HHSUnited States/ ; U01 DD001119/DD/NCBDD CDC HHSUnited States/ ; U01 DD001108/DD/NCBDD CDC HHSUnited States/
• Volume:
  66
• Issue:
  1
• Collection(s):
  CDC Public Access
• Main Document Checksum:
  urn:sha256:d1c1e59f00e8409d75734315ae678956becad1e6853a77a3e38589488f1f1046
• Download URL:
  https://stacks.cdc.gov/view/cdc/118369/cdc_118369_DS1.pdf
• File Type:
  [PDF - 702.09 KB ]