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ATHN 7: A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People with Hemophilia-Demographics and Preliminary Results

1University of Colorado School of Medicine, Hemophilia and Thrombosis Center, Aurora, United States, 2American Thrombosis and Hemostasis Network, Rochester, United States, 3Boston Children's Hospital, Harvard Medical School, Boston Hemophilia Center, Boston, United States, 4Emory University School of Medicine, Hemophilia of Georgia Center for Bleeding & Clotting Disorders, Atlanta, United States, 5Versiti Blood Center of Wisconsin, Comprehensive Center for Bleeding Disorders, Milwaukee, United States, 6University of Iowa Stead Family Children's Hospital, Iowa Hemophilia and Thrombosis Center, Iowa City, United States

Abstract Number: PB1018

Meeting: ISTH 2020 Congress

Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical

Background: Introduction of non-factor molecules mimicking the effect of activated factor VIII or inhibiting endogenous anticoagulants mandates longitudinal understanding of their safety and effectiveness. Utilizing the national infrastructure of the American Thrombosis and Hemostasis Network (ATHN), ATHN 7: Hemophilia Natural History Study aims to collect such data in a cohort of participants with hemophilia.

Aims: The primary objective of ATHN 7: Hemophilia Natural History Study is to determine the safety of current FDA-approved hemophilia therapies. Safety will be measured by those events in the European Haemophilia Safety Surveillance (EUHASS) outcomes and other adverse events of special interest. Secondary objectives include determining the real-world effectiveness of therapies including patient-reported outcomes.

Methods: This longitudinal cohort study, currently open in 25 ATHN-affiliated sites in the United States, was approved by institutional review boards. All participants signed informed consent. Inclusion criteria included the presence of congenital hemophilia A or B. Participants were excluded if they had any other known bleeding disorder. See Figure 1 for data collection procedures.

Results: As of August 2019, 152 participants were enrolled. The mean age of participants was 19.1 years (range less than one to 80 years) with 144 having hemophilia A and 5 having hemophilia B. For three participants, a diagnosis was not provided. Ten (6.6%) participants had inhibitors at the time of enrollment. Fifty percent of participants were utilizing non-factor products, 45% recombinant factor, and 5% plasma-derived factor. To date, no safety events were reported.

Conclusions: ATHN 7: Hemophilia Natural History Study demonstrates the capability of ATHN´s national infrastructure to prospectively collect longitudinal data to determine the safety and effectiveness of current therapies for those with hemophilia. The study is early in accrual, so final conclusions as to the safety and effectiveness of current hemophilia therapies cannot yet be drawn.


[Figure 1: ATHN 7 Data Collection Procedures]

To cite this abstract in AMA style:

Buckner T, Daoud N, Croteau SE, Kempton CL, Malec L, Staber J, Wang M, Watson C, Recht M. ATHN 7: A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People with Hemophilia-Demographics and Preliminary Results [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/athn-7-a-natural-history-cohort-study-of-the-safety-effectiveness-and-practice-of-treatment-for-people-with-hemophilia-demographics-and-preliminary-results/. Accessed September 24, 2023.

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