welcome to DuchenneXchange

- a positively charged Duchenne muscular dystrophy community.
  • join today!
Abstracts & Posters

A Pilot Study to Evaluate Adherence to the 2010 Care Considerations for Duchenne Muscular Dystrophy (DMD) at Selected Clinics Identified by the Muscular Dystrophy Surveillance Tracking and Research Network (MD STARnet) Sites. Phase 1:Clinic Director Survey

key information

source: American Academy of Neurology

year: 2016

authors: Shree Pandya, Kristin Caspers-Conway, Christina Trout, Christina Westfield, Deborah Fox


To evaluate adoption of the Care Considerations for DMD through a pilot survey of clinic directors identified by the MD STARnet sites.

Care recommendations for the management of patients with Duchenne Muscular Dystrophy (DMD) were developed by an international panel of experts convened and supported by the Centers for Disease Control and Prevention (CDC) and published in 2010. In 2013, the CDC funded a pilot study to evaluate adherence to these recommendations by selected clinics treating dystrophinopathy patients.

The pilot study included two-phases. In the first phase, a survey was distributed to 9 clinic directors identifiedby the MD STARnet sites in Arizona,Colorado, Iowa and western New York. The survey was comprised of questions about interdisciplinary management and perceived barriers to care at the patient, system, and provider levels.Item response categories were included to reflect recommended care practices and alternative approaches to patient management. The second phase includes medical record abstraction of patient records.

Completed surveyswere received from six clinics. Findings suggest all clinics offer multidisciplinary care, examine patients biannually, and offer all patients corticosteroidtreatment. Monitoring of pulmonary health was consistent with recommendations for non-ambulatory, asymptomatic patients. Monitoring of scoliosis and cardiac health consistent with recommendations were reported by 80% of clinic directors and over one-half reported neurocognitive/ neuropsychological evaluations.Prominent system barriers were funding for ancillary staff and care coordination. Barriers for families included distance to care, lack of resources and out-of-pocket expenses.

A majority of surveyed clinics reported adherence to most of the care recommendations in their management practices. The next phase of the project includes medical record abstraction. The availability of both clinic surveys and medical record abstraction afford a unique perspective on assessing the use of the care recommendations when treating dystrophinopathy patients.


read more