title: Can outcomes in Duchenne muscular dystrophy be improved by public reporting of data?
abstract: OBJECTIVE
  To review current approaches for obtaining patient data in Duchenne muscular dystrophy (DMD) and consider how monitoring and comparing outcome measures across DMD clinics could facilitate standardized and improved patient care.
  
  
  METHODS
  We reviewed annual standardized data from cystic fibrosis (CF) clinics and DMD care guidelines and consensus statements; compared current approaches to obtain DMD patient data and outcome measures; and considered the best method for implementing public reporting of outcomes, to drive improvements in health care delivery.
  
  
  RESULTS
  Current methods to monitor DMD patient information (MD STARnet, DuchenneConnect, and TREAT-NMD) do not yet provide patients with comparative outcome data. The CF patient registry allows for reporting of standard outcomes across clinics and is associated with improved CF outcomes. A similar patient registry is under development for the Muscular Dystrophy Association (MDA) clinic network. Suggested metrics for quality care include molecular diagnosis, ambulatory status and age at loss of ambulation, age requiring ventilator support, and survival.
  
  
  CONCLUSIONS
  CF longevity has increased by almost 33% from 1986 to 2010, in part due to a CF patient registry that has been stratified by individual care centers since 1999, and publically available since 2006. Implementation of outcome reporting for MDA clinics might promote a similar benefit to patients with DMD.
authors: University of Rochester Medical Center, Rochester, NY, USA.
authors: Michele A
authors: MA
authors: Scully
authors: Valerie A
authors: VA
authors: Cwik
authors: Bruce C
authors: BC
authors: Marshall
authors: Emma
authors: E
authors: Ciafaloni
authors: Jodi M
authors: JM
authors: Wolff
authors: Thomas S
authors: TS
authors: Getchius
authors: Robert C
authors: RC
authors: Griggs
mesh_terms: D006801:Humans; D020388:Muscular Dystrophy, Duchenne; D017063:Outcome Assessment, Health Care / Q000379:methods* / Q000592:standards; D012042:Registries; D012107:Research Design / Q000592:standards*
references: Acta Myol. 2009 Jul;28(1):12-5
references: 19772190
references: Cochrane Database Syst Rev. 2008 Jan 23;(1):CD003725
references: 18254031
references: Neurology. 2005 Jan 11;64(1):13-20
references: 15642897
references: MMWR Morb Mortal Wkly Rep. 2009 Oct 16;58(40):1119-22
references: 19834452
references: J Child Neurol. 2010 Nov;25(11):1319-24
references: 20207610
references: Lancet. 2011 Mar 26;377(9771):1057-9
references: 20674966
references: Neurology. 1991 Dec;41(12):1874-7
references: 1745340
references: Birth Defects Res A Clin Mol Teratol. 2006 Nov;76(11):793-7
references: 17036307
references: Thorax. 2011 Dec;66(12):1106-8
references: 21813618
references: Lancet Neurol. 2010 Feb;9(2):177-89
references: 19945914
references: J Pediatr. 2005 Sep;147(3):295-301
references: 16182664
references: Genet Med. 2011 Nov;13(11):942-7
references: 21836521
references: Lancet Neurol. 2010 Jan;9(1):77-93
references: 19945913
references: Int J Technol Assess Health Care. 2011 Jul;27(3):247-52
references: 21756412
references: Neuromuscul Disord. 2007 Jun;17(6):470-5
references: 17490881
references: Curr Opin Pediatr. 2010 Jun;22(3):296-301
references: 20414114
references: Contemp Clin Trials. 2012 Mar;33(2):302-11
references: 22155025
references: Neurology. 2010 Sep 14;75(11):982-9
references: 20837966
references: Neuromuscul Disord. 2002 Dec;12(10):926-9
references: 12467747
references: PLoS Curr. 2012 Feb 29;4:RRN1309
references: 22453902
references: J Child Neurol. 2010 Sep;25(9):1098-102
references: 20817884
references: Chest. 2005 Oct;128(4):2347-54
references: 16236893
references: J Pediatr. 2008 Dec;153(6):733-5
references: 19014811