Publication Date:
2020-11-05
Description:
Introduction: ATHN 8: U.S. Cohort Study of Previously Untreated Patients (PUPs) with Congenital Hemophilia is sponsored by the American Thrombosis and Hemostasis Network (ATHN) and is being conducted at ATHN-affiliated sites in the United States. The ATHN 8: PUPs Study collects detailed demographic, diagnosis, treatment, bleed, and inhibitor data on children with moderate and severe hemophilia born on or after January 1, 2010 and followed at an ATHN-affiliated hemophilia treatment center (HTC). The endpoint for the overall study is inhibitor development or achieving 50 exposure days. A confirmed inhibitor is defined as two consecutive positive inhibitor titers (〉0.5 Nijmegen Bethesda Units (BU) for hemophilia A and 〉0.3 Nijmegen BU for hemophilia B) which results in change in treatment. Available family history is also collected. For this interim analysis, we hypothesized that children with severe hemophilia A (HA) and a family history (FH) of hemophilia would have earlier age of diagnosis. Methods: Available data through April 30, 2020 were analyzed for participants with severe HA (factor VIII
Print ISSN:
0006-4971
Electronic ISSN:
1528-0020
Topics:
Biology
,
Medicine
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