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Research & Innovation

For many children with rare or hard-to-treat conditions, research provides an opportunity for patients, families, providers, and scientists to better understand the disease, how to manage it, and potentially to develop new treatment options. There are dozens of clinical trials currently open and listed on clinicaltrials.gov for patients with bleeding disorders. We offer clinical studies in hemophilia, von Willebrand disease, and young women with bleeding disorders that are developed and led at our center, as well as those sponsored by other institutions and pharmaceutical companies.

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Learn about our patients and their families whose experiences with bleeding disorders offer hope and help to others.

Read their stories

Gene therapy trials in hemophilia

Gene Therapy provides an opportunity to provide the body with instructions on how to make a particularly protein. In the case of hemophilia, these proteins are factor VIII or factor IX.

Observational bleeding disorders studies

Hemophilia and associated bleeding disorders

  • CDC Public Health Surveillance for Bleeding Disorders: The purpose of this project is to monitor the health status of people with hemophilia and other bleeding disorders. This will help HTCs and CDC better understand the health issues of people with bleeding disorders.

Key clinical trial, clinical care innovation publications involving or led by BBDC team members

Understanding bleeding disorders

  • The eTHINK study: cognitive and behavioral outcomes in children with hemophilia. The Journal of Pediatrics. 2024. doi:10.1016/j.jpeds.2024.114089
  • Natural History Study of Factor IX Deficiency with Focus on Treatment and Complications (B-Natural). Haemophilia. 2020. doi:10.1111/hae.14139
  • Posttraumatic stress disorder and posttraumatic stress symptoms among adults with hemophilia A and B. Research and Practice in Thrombosis and Haemostasis. 2023. doi:10.1016/j.rpth.2023.102241
  • Low von Willebrand Factor in Pediatric Patients: Retrospective analysis of 293 cases informs Diagnostic and Therapeutic Decision-Making. Pediatric Blood & Cancer. 2020. doi:10.1002/pbc.28497
  • Outcomes of long-term von Willebrand factor prophylaxis use in von Willebrand disease: A systematic literature review. Haemophilia. 2022 May. doi:10.1111/hae.14550
  • Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: facilitating research through infrastructure, workforce, resources and funding. Expert Review of Hematology. 2023. doi:10.1080/17474086.2023.2181781
  • Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities in health services; diversity, equity, and inclusion; and implementation science. Expert Review of Hematology. 2023. doi:10.1080/17474086.2023.2183836

Expanding treatment options

  • Clinical Trials Update on hemophilia, VWD, and rare bleeding disorders, American Journal of Hematology. 2025. doi:10.1002/ajh.27602
  • Surgical outcomes in people with hemophilia A taking emicizumab prophylaxis: experience from the HAVEN 1-4 studies. Blood Advances. 2022. doi:10.1182/bloodadvances.2022007458
  • Emicizumab for preventing intracranial hemorrhage in infants with severe hemophilia A: a cost-effectiveness analysis, Blood Advances. 2025. doi:10.1182/bloodadvances.2025016822
  • Discussing investigational AAV gene therapy with hemophilia patients: A guide. Blood Reviews 2021. doi:10.1016/j.blre.2020.100759
  • Benefits and risks of non-factor therapies: redefining haemophilia treatment goals in the era of new technologies. Haemophilia. 2024. doi:10.1111/hae.14976

Guidelines and health resource utilization

  • ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease. Blood Advances. 2021. doi:10.1182/bloodadvances.2020003264
  • ASH ISTH NHF WFH 2021 guidelines on the diagnosis of von Willebrand disease. Blood Advances. 2021. doi:10.1182/bloodadvances.2020003265
  • Health care costs and resource utilization among commercially insured adult patients with hemophilia A managed with FVIII prophylaxis in the United States. Journal of Managed Care and Specialty Pharmacy. 2022. doi:10.18553/jmcp.2021.21368