Comparing Spinal Muscular Atrophy (SMA) outcomes between a patient-reported membership database and a clinical data registry (P2-8.012)

Abstract

<h3>Objective:</h3> The purpose of this study is to compare patient reported data collected in the Cure SMA membership database to clinician entered and electronic medical registry (EMR) derived data from the Cure SMA Clinical Data Registry (CDR) among patients who are members in both databases. <h3>Background:</h3> Cure SMA currently maintains the largest patient-reported database of persons with all types of SMA and documents selected aspects of disease such as type of SMA, date of diagnosis, SMN2 copy number, gender, date of birth and mortality. There are currently 9789 affected individuals with SMA in the membership database. In October 2018, Cure SMA launched a clinical data registry (CDR) to collect real-world data about SMA care. Data from the CDR is comprised of electronic medical records (EMR) sourced data from US hospitals and is linked to a clinician-entered electronic case report form (eCRF) to gather additional SMA-specific information that is not easily found in the EMR. There are currently 734 affected individuals with SMA in the CDR. <h3>Design/Methods:</h3> All data included in the analysis will be previously collected data and patients will not be contacted to provide additional information. Patients will be merged from both databases by first name, last name, birthdate, gender, and US state of residence. <h3>Results:</h3> Current age, race/ethnicity, SMA type, SMN2 copy number, age at SMA diagnosis, and use of FDA-approved SMA treatments will be compared at patient level. <h3>Conclusions:</h3> Findings from this study will provide support and validity to patient-reported data. Additionally, differences in outcomes between databases will highlight any gaps needed in communication between a patient and their healthcare team. <b>Disclosure:</b> Lisa Belter has received personal compensation for serving as an employee of Cure SMA. Mrs. Whitmire has received personal compensation for serving as an employee of Cure SMA. Mrs. Whitmire has received personal compensation for serving as an employee of Novo Nordisk Inc.. Mary Curry has received personal compensation for serving as an employee of Cure SMA. Dr. Schroth has received personal compensation for serving as an employee of Cure SMA.