Bone Health and Endocrine Care of Boys with Duchenne Muscular Dystrophy: Data from the MD STARnet

Details

• Alternative Title:
  J Neuromuscul Dis
• Personal Author:
  Weber, David R. ; Thomas, Shiny ; Erickson, Stephen W. ; Fox, Deborah ; Oleszek, Joyce ; Pandya, Shree ; Venkatesh, Yedatore ; Westfield, Christina ; Ciafaloni, Emma
• Corporate Authors:
  Muscular Dystrophy Tracking and Research Network (MD STARnet)
• Description:
  Background:
  
  Patients with Duchenne muscular dystrophy (DMD) are at high risk of endocrine and bone health complications resulting from the high glucocorticoid (GC) doses used to treat this condition. There are limited data characterizing the clinical management of these complications.
  
  Objective:
  
  To determine the frequency of bone health screening, endocrinologist evaluation, and use of endocrine and bone health pharmacotherapy in the clinical care of males with DMD.
  
  Methods:
  
  A population based cohort study using data from the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) was conducted. Clinical data was abstracted from the medical records of 683 males with DMD at five surveillance sites across the US.
  
  Results:
  
  A DXA scan had been documented in 24% of cases; the percentage of cases with DXA varied across surveillance sites from 13% to 43%, p < 0.001. History of fracture and greater disease duration were associated with greater odds of having a DXA. Only 4.7% of cases had documentation of an endocrinologist evaluation. The frequency of documented endocrine and bone health pharmacotherapy use included calcium (42.8%), vitamin D (36.6%), bisphosphonates (13.3%), growth hormone (1.9%), testosterone (1.7%), insulin (1.2%), and metformin (0.3%)
  
  Conclusions:
  
  A low percentage of DMD males had record of DXA scan, endocrinologist evaluation, or treatment with endocrine or bone health pharmacotherapy. Endocrine and bone health care may represent an unmet need in the DMD population.
  
  
• Keywords:
  Absorptiometry, Photon Adolescent Bisphosphonates Bone And Bones Bone Diseases Child Cohort Studies Databases, Factual Duchenne Endocrine System Diseases Glucocorticoids Humans Hypogonadism Male Medical Records Muscular Dystrophy Muscular Dystrophy, Duchenne Osteoporosis

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• Source:
  J Neuromuscul Dis. 5(4):497-507
• Pubmed ID:
  30149461
• Pubmed Central ID:
  PMC6277257
• Document Type:
  Journal Article
• Funding:
  U01 DD000187/DD/NCBDD CDC HHSUnited States/ ; U01 DD001123/DD/NCBDD CDC HHSUnited States/ ; U01DD001126/ACL/ACL HHSUnited States/ ; U01 DD000191/DD/NCBDD CDC HHSUnited States/ ; U01 DD001119/DD/NCBDD CDC HHSUnited States/ ; K23 DK114477/DK/NIDDK NIH HHSUnited States/ ; U01DD001117/ACL/ACL HHSUnited States/ ; U01 DD001117/DD/NCBDD CDC HHSUnited States/ ; U01 DD001126/DD/NCBDD CDC HHSUnited States/ ; U01DD001116/ACL/ACL HHSUnited States/ ; U01DD001123/ACL/ACL HHSUnited States/ ; U01 DD000189/DD/NCBDD CDC HHSUnited States/ ; U01 DD001116/DD/NCBDD CDC HHSUnited States/ ; U01 DD001255/DD/NCBDD CDC HHSUnited States/ ; U01 DD000190/DD/NCBDD CDC HHSUnited States/
• Volume:
  5
• Issue:
  4
• Collection(s):
  CDC Public Access
• Main Document Checksum:
  urn:sha256:7b78421c02369ae2f6300bd0f6b857e6b2e8743a2ab615d0943031e120404b7e
• Download URL:
  https://stacks.cdc.gov/view/cdc/61234/cdc_61234_DS1.pdf
• File Type:
  [PDF - 1.20 MB ]