Guidelines and Outcomes; Standardizing Care for Duchenne Muscular Dystrophy (P04.087)

Abstract

Objective: To review evidence-based and consensus guidelines for the treatment of Duchenne Muscular Dystrophy (DMD) and consider how outcome measures could standardize and improve patient care. Background There are no evidence-based quality care indicators to standardize outcomes across Muscular Dystrophy Association (MDA) clinics. We reviewed the Cystic Fibrosis (CF) model for standardizing CF clinic outcomes and consider potential outcomes to assess the quality of care in DMD clinics. For forty years, the CF foundation has maintained a nationwide patient registry and acquired patient data from each CF clinic. The data, analyzed yearly, is reviewable on the CF website. Comparing individual and national clinic outcomes challenges clinics to improve clinical care. This data also provides potential patient populations for future investigations, including clinical trials. Design/Methods: We reviewed: published guidelines and consensus statements on the care of DMD; current efforts to obtain outcomes from DMD clinics; standardized data obtained annually from CF clinics; and published approaches to improving outcomes by attention to the fidelity of health care delivery. Results: There are multiple approaches within the DMD field for surveillance and collection of patient information, including MDSTARNet, Parent Project Muscular Dystrophy, and MDA DMD Clinical Research Network. Based on CF analyzed data, a similar patient registry and nationwide reporting of standard outcomes across MDA clinics appears feasible. Monitoring data including molecular diagnosis, age at loss of ambulation, FVC, and survival, could provide metrics for quality of care. Conclusions: There are logistic challenges to reporting nationwide outcomes; however the influence this model has had on outcomes for CF patients has changed survival. In 2009, the median life expectancy for a patient with CF was 35.9 years, compared to 27 years in 1985. Implementation of clinical data monitoring in MDA clinics will determine if similar benefit accrues in patients with Duchenne Muscular Dystrophy. Disclosure: Dr. Scully has nothing to disclose. Dr. Cwik has nothing to disclose. Dr. Marshall has nothing to disclose. Dr. Ciafaloni has nothing to disclose. Dr. Getchius has nothing to disclose. Dr. Griggs has nothing to disclose.