Journal article
Implementation of Duchenne Muscular Dystrophy Care Considerations
Pediatrics (Evanston), Vol.142(1), p.e20174006
07/2018
DOI: 10.1542/peds.2017-4006
PMID: 29925575
Abstract
Duchenne muscular dystrophy (DMD) is an X-linked disorder characterized by progressive muscle weakness and multisystem involvement. Recent advances in management of individuals with DMD have prolonged survival. Lack of standardized care spurred an international collaboration to develop consensus-based care considerations for diagnosis and management. In this study, we evaluate adherence to considerations at selected sites.
We collaborated with the Muscular Dystrophy Surveillance, Tracking, and Research Network. Our sample included males with DMD and Becker muscular dystrophy <21 years as of December 31, 2010, with 1 health care encounter on or after January 1, 2012. We collected data from medical records on encounters occurring January 1, 2012, through December 31, 2014. Adherence was determined when frequency of visits or assessments were at or above recommendations for selected care considerations.
Our analytic sample included 299 individuals, 7% of whom (20/299) were classified as childhood-onset Becker muscular dystrophy. Adherence for neuromuscular and respiratory clinician visits was 65% for the cohort; neuromuscular assessments and corticosteroid side effect monitoring measures ranged from 16% to 68%. Adherence was 83% for forced vital capacity and ≤58% for other respiratory diagnostics. Cardiologist assessments and echocardiograms were found for at least 84%. Transition planning for education or health care was documented for 31% of eligible males.
Medical records data were used to identify areas in which practice aligns with the care considerations. However, there remains inconsistency across domains and insufficiency in critical areas. More research is needed to explain this variability and identify reliable methods to measure outcomes.
Details
- Title: Subtitle
- Implementation of Duchenne Muscular Dystrophy Care Considerations
- Creators
- Jennifer G Andrews - Department of Pediatrics, University of Arizona, Tucson, Arizona; jandrews@peds.arizona.eduKristin Conway - Departments of Epidemiology andChristina Westfield - New York State Department of Health, Albany, New YorkChristina Trout - Pediatrics, University of Iowa, Iowa City, IowaF John Meaney - Department of Pediatrics, University of Arizona, Tucson, ArizonaKatherine Mathews - Pediatrics, University of Iowa, Iowa City, IowaEmma Ciafaloni - Department of Neurology, School of Medicine and Dentistry, University of Rochester, Rochester, New YorkChristopher Cunniff - Department of Pediatrics, Weill Cornell Medical College, New York City, New York; andDeborah J Fox - Bureau of Environmental and Occupational EpidemiologyDennis Matthews - Department of Physical Medicine and Rehabilitation, School of Medicine, University of Colorado, Aurora, ColoradoShree Pandya - Department of Neurology, School of Medicine and Dentistry, University of Rochester, Rochester, New York
- Resource Type
- Journal article
- Publication Details
- Pediatrics (Evanston), Vol.142(1), p.e20174006
- DOI
- 10.1542/peds.2017-4006
- PMID
- 29925575
- NLM abbreviation
- Pediatrics
- ISSN
- 0031-4005
- eISSN
- 1098-4275
- Publisher
- American Academy of Pediatrics; United States
- Grant note
- U01 DD001041 / NCBDD CDC HHS
- Language
- English
- Date published
- 07/2018
- Academic Unit
- Neurology; Stead Family Department of Pediatrics; Epidemiology; Iowa Neuroscience Institute; Neurology (Pediatrics)
- Record Identifier
- 9984070695302771
Metrics
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