Utilizing national electronic data infrastructure to longitudinally follow the United States (US) bleeding disorders population

Background – The US Hemophilia Treatment Center (HTC) Network along with the Centers for Disease Control and Prevention are creating the next generation bleeding and clotting disorder surveillance tool, building upon their previous experience with robust, adaptive data systems. Purpose - To share the iterative and collaborative community-based development strategy, testing process and methods as applied to key outcomes for surveillance utilizing electronic health records (EHR). Methods – Two surveillance tools, HTC Population Profile and Registry for Bleeding Disorders, developed with broad community, scientific and administrative input, leverage a national, multipurpose web-based infrastructure to create a HIPAA compliant limited data set drawn from 14,000+ patients with rare bleeding and clotting disorders. The HTC Population Profile collects limited, de-identified data describing the overall HTC population. The Registry collects more comprehensive data from a subset of HTC patients with eligible disorders. These surveillance tools are prepopulated from an EHR system housed under the stewardship of the American Thrombosis and Hemostasis Network. This patient-level record captures demographics; clinical information (e.g., diagnosis, severity, treatment); key outcomes and complications (chronic pain and related opioid use, inhibitory antibodies, HIV, hepatitis); genetics; and patient reported outcomes (bleeding, activity limitations). Results – Feasibility studies of both tools have been completed. To date, over 10,300 population profiles representing patients from 56 of 130 treatment centers have been extracted for use. These data, when combined with data from the Registry, will enable the network to: 1) provide descriptive knowledge about demographics, diagnoses, complications, and health service utilization of populations with bleeding disorders; and 2) monitor over time medical practices/outcomes including use of preventive therapies, immunization rates, and adverse events. Conclusion – Use of a standardized electronic national data infrastructure may help in monitoring key health indicators and emerging issues among persons with rare bleeding and clotting disorders and improving outcomes.