University of Rochester School of Medicine and Dentistry, Rochester, NY, USA
| [b] New York State Department of Health, Albany, NY, USA
| [c] RTI International, Research Triangle Park, NC, USA
University of Colorado-Denver, Denver, CO, USA
University of South Carolina School of Medicine, Columbia, SC, USA
Correspondence to: David R. Weber, MD, MSCE, University of Rochester Medical Center, 601 Elmwood Avenue, Box
690, Rochester, NY 14642, USA. Tel.: +1 585 275 7744; Fax: +1 585 244 6097;
E-mail: [email protected].
Abstract: 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.