ATHN Projects and Research Studies

At ATHN, we leverage technology to support and expand opportunities for our ATHN Affiliates—a network of more than 145 treatment centers across the country providing specialized care to the blood disorders community.

Supporting Our Network

ATHN works with investigators and staff at our affiliated centers through a collaborative process to secure data, advance knowledge, transform care, and ultimately improve the quality of life for people affected by rare disorders. We empower and support researchers in the conception, development, and execution of innovative projects seeking to answer scientifically and clinically meaningful questions.

Open ATHN Projects

ATHN is proud to partner with the ATHN Affiliate Network on a variety of innovative studies helping to advance science—and ultimately improve lives. Here’s a look at projects currently underway.

Von Willebrand disease (VWD) is the most common inherited bleeding disorder, caused by missing or defective von Willebrand factor (VWF), a clotting protein. ATHN 9: Severe VWD Natural History Study aimed to assess the safety and effectiveness of various VWF treatment regimens in both children and adults with clinically severe VWD.

Principal Investigators: Robert F. Sidonio, Jr., MD, MSc, Children’s Healthcare of Atlanta, Emory University School of Medicine; and Angela C. Weyand, MD, CS Mott Children’s Hospital, University of Michigan

Current Status: Ongoing

ATHN 10: Rare Coagulation Disorders Project offers genetic testing at no cost to participants. The goal of this project is to enhance the genotypic and phenotypic data on the population of rare coagulation disorder patients receiving care within the ATHN Affiliate Network and to support future research with the goal of improving clinical care.

Principal Investigators: Amy Shapiro, MD, Indiana Hemophilia & Thrombosis Center; and Diane Nugent, MD, Center for Inherited Blood Disorders

Current Status: Ongoing

ATHN 16: SEVENFACT® for Bleeding Events in Hemophilia with Inhibitors aims to evaluate the safety of the FDA-approved drug SEVENFACT® when used to treat bleeding episodes in participants with hemophilia A or B with inhibitors who are being treated either with or without prophylactic agents. The study is a phase IV multi-center, open-label, safety study.

Principal Investigators: Tammuella Chrisentery-Singleton, MD, American Thrombosis and Hemostasis Network; and Mark T. Reding, MD, University of Minnesota Medical Center

Current Status: Ongoing, Enrolling

Late-Stage ATHN-Sponsored Projects

The following projects are closed to enrolling and currently undergoing data validation and analysis and/or publication development.

The ATHN 7: Hemophilia Natural History Study aimed to determine the safety and effectiveness of available hemophilia therapies, including non-factor products, bypassing agents, and clotting factor replacement products. ATHN 7 is closed to enrollment and participants are being invited to enroll in the Hemophilia Natural History Arm of ATHN Transcends. <<link underlined to new ATHN Transcends page anchored to Hemophilia Natural History Arm content>>

Principal Investigators: Tyler Buckner, MD, MSc, Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus; and Michael Recht, MD, MBA, PhD, Yale School of Medicine and National Bleeding Disorders Foundation

Current Status: Data Analysis/Validation

The ATHN 8: PUPs Study was the first national study focused on previously untreated patients (PUPs) to determine the percentage of those in this vulnerable population with confirmed inhibitors within the first 50 exposure days and risk factors associated with inhibitor development. This study informed the development of the new PUPs Arm of ATHN Transcends. <<link underlined to new ATHN Transcends page anchored to PUPs Arm content>>

Principal Investigators: Shannon Carpenter, MD, MS, Children’s Mercy Hospital; Courtney Thornburg, MD, MS, Rady Children’s Hospital; and Marijke van den Berg, MD, PhD, Versiti Blood Center of Wisconsin

Current Status: Publication Development

The ATHN 11 cohort study compared the quality of life outcomes between participants with hemophilia A and B who received a liver transplant and patients who did not. The primary objective of ATHN 11 is to examine the impact of sustained increase in Factor VIII (FVIII) and Factor IX (FIX) levels after liver transplantation on quality of life.

Principal Investigator: Margaret V. Ragni, MD, MPH, University of Pittsburgh Medical Center

Current Status: Publication Development

The ATHN 12: HAD Pilot Project collected data on persons diagnosed with HAD across the United States to better understand this rare condition and to inform the development of guidelines regarding proper time of screening for HAD, type of antithrombotic therapy, and adequate use of antithrombin concentrate.

Principal Investigator: Maria DeSancho, MD, Weill Cornell Medicine, New York Presbyterian Hospital

Current Status: Publication Development

Completed ATHN Projects

For over a decade, ATHN has been supporting important research projects in support of the blood disorders community. Here’s a look at completed and closed projects.

Using the ATHNdataset and study-specific data, this study showed that older men with moderate to severe hemophilia commonly reported risk factors for cardiovascular disease (CVD). However, the prevalence of reported CVD is low (9.7%), which suggests that men with hemophilia may be protected from forming disease-causing blood clots.

Principal Investigator: Barbara A. Konkle, MD, Bloodworks Northwest

This ATHN-sponsored study, which ran from 2015 to 2019, looked at the effects of switching factor products among participants who had a history of inhibitors, but were free of an inhibitor at the time of the study.

Principal Investigator: Ellis Neufeld, MD, PhD, St. Jude Children’s Research Hospital

Due to safety concerns associated with the radionuclide synovectomy/synoviorthesis (RS) procedure, this project tracked the number, safety, and location of RS procedures performed in the United States.

Principal Investigator: Amy L. Dunn, MD, Nationwide Children’s Hospital

In response to the rising prevalence of VTE in the blood disorders population, ATHN 4: VTE Project focused on ways to improve the transition of care practices for both adult and pediatric care, including evaluating patient and family understanding and adherence to anticoagulation therapy instructions.

Principal Investigators: Madhvi Rajpurkar, MD, Children’s Hospital of Michigian; and Maria T. DeSancho, MD, MSc, Weill Cornell Medicine

ATHN 5: HCV Treatment Outcomes was a multicenter, longitudinal, observational study aimed at generating data leading to more informed treatment decisions and better outcomes for people with blood disorders and HCV. It also evaluated factors influencing treatment outcomes, such as adherence to the therapeutic regimen.

Principal Investigator: Christopher E. Walsh, MD, PhD, Mt. Sinai Hospital

The ATHN 6: HCV Eradication project was designed to mobilize a comprehensive treatment approach by health care teams and address barriers related to HCV testing and treatment for people with HCV receiving care at ATHN-affiliated treatment centers.

Principal Investigator: Christopher E. Walsh, MD, PhD, Mt. Sinai Hospital

ATHN 15: DOAC Use in Pediatric Thrombosis Patients aimed to be a national resource for clinicians that includes data on pediatric patients under 21 years of age who have received a DOAC since January 1, 2015 for the treatment of an acute venous thromboembolism (VTE) episode or to prevent a thrombosis recurrence.

Principal Investigators: Jennifer Davila, MD, Montefiore Medical Center; and Fernando Corrales-Medina, MD, Hemophilia Treatment Center at University of Miami

The MLOF Research Repository contains genotypic data and biologic samples from participants with hemophilia A and B, as well as potential and confirmed female genetic carriers who received care at one of more than 100 ATHN-affiliated treatment centers across the United States.

In 2017, scientists and researchers from around the county were invited to propose research projects utilizing the repository. A total of 11 research projects were approved based on their scientific merit and level of benefit to the blood disorders community.

MLOF Research Repository Data Published in Frontiers in Medicine

These important projects are happening at treatment centers all over the United States. If you are a patient interested in learning more about any of our current projects, talk to your care provider.

If you are a provider interested in working with ATHN on one of our current projects or a researcher interested in proposing a new project, please email us at