Rare Coagulation Disorders Grant Funded
As steady progress moves the ATHN infrastructure forward, ATHN’s strategic plan encourages research projects that appropriately use the database. With support from a two-year, $200,000 grant from Baxter Bioscience, ATHN has begun work to move its research agenda forward to improve the care and treatment of people with rare coagulation factor deficiencies, a vulnerable sub-population of the HTC patient population. Recognizing that current registries for this sub-population are inadequate, incomplete and not dynamic for this population, this project builds on the infrastructure funded through the contribution of Novo Nordisk to develop:
- An approach to standardized data collection for rare bleeding disorders within our web-based national database platform (Lab Tracker);
- An electronic national system for generation of a “Unique Patient Identification Number” (UPIN) that is compatible with the approach adopted for hemophilia patients; and
- A portal for creation of data collection forms for rare bleeding disorder research protocols.
ATHN’s intention is to leverage the work of experts who have created the rare coagulation disorder resource room through MASAC and the current efforts of the HTC Network coordinating committee in this area. Data elements required to understand rare coagulation disorders will be integrated into the Lab Tracker (web) data dictionary as well as the data collection and reporting templates to make it easier for centers. The resultant data about the care and treatment for people with rare disorders, will help meet the critical need for insight into the:
- Prevalence of these disorders;
- Types and severity of bleeding events experienced with each deficiency;
- Level of replacement required for specific bleeding events;
- Need and outcome of prophylaxis in each of these disorders; and
- Rate of complications and treatment failures associated with presently utilized treatment strategies.
The database is also expected to facilitate progress towards the development of FDA approved products to treat these disorders and improve clinical outcomes.
A variety of U.S. and international agencies and committees have identified adequate national data collection as the first most important step toward improving care and treatment for people with rare coagulation disorders. ATHN’s work further advances a collaborative national response that is harmonized with international efforts to meet the special needs of this vulnerable underserved population.
The rare bleeding disorders working group at the ATHN Data Summit will explore the plan in more detail.
