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Research & Innovation | Overview

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 for patients with bleeding disorders. We offer clinical studies in hemophiliavon Willebrand disease and young women with bleeding disorders that are developed and lead at our Center, as well as those sponsored by other institutions and pharmaceutical companies.

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.

  • Phase I/II dose escalation study of a recombinant adeno-associated viral vector to drive expression of a B-domain-deleted human factor VIII transgene to raise the circulating activity levels of endogenous FVIII in adult patients with severe hemophilia A
  • A Multi-Center Evaluation of the Long-Term Safety and Efficacy of SPK-8011 [Adeno-Associated Viral Vector with B-Domain Deleted Human Factor VIII Gene] in Males with Hemophilia A

Observational bleeding disorders studies


  • 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.

Women with bleeding disorders 

  • ATHENA: Characterizing the impact and treatment of reproductive tract bleeding on women and post-menarchal girls with bleeding disorders

Publications highlighting prior clinical trials and clinical-care innovations


  • Decreased Platelet Surface Phosphatidylserine Predicts Increased Bleeding in Patients with Severe Factor VIII Deficiency. Croteau SE, Frelinger AL, Michelson AD. Journal of Thrombosis and Haemostasis, 2020. doi: 10.1111/jth.15223.
  • Spontaneous Bleeding and Poor Bleeding Response with Extended Half-life Factor IX Products: A Survey of Select US Hemophilia Treatment Centers. Malec LA, Croteau SE, Callaghen M, Sidonio R. Haemophilia, 2020. doi: 10.1111/hae.13943
  • Utility of Blood Cultures and Empiric Antibiotics in Febrile Pediatric Hemophilia Patients with Central Venous Access Devices. Al-Samkari H, Landschaft A, Kimia R, Harper HB, Ozonoff AJ, Croteau SE, Kimia AA. Pediatric Emergency Care, 2020. doi: 10.1097/PEC.0000000000002106
  • The Impact of Extended Half-Life Factor Concentrates on Prophylaxis for Severe Hemophilia in The United States. Malec LA, Cheng D, Witmer CM, Jaffray J, Kouides PA, Haley KM, Sidonio R, Johnson K, Recht M, White G, Croteau SE, Ragni M. American Journal of Hematology. 2020. doi: 10.1002/ajh.25844.
  • Natural History Study of Factor IX Deficiency with Focus on Treatment and Complications (B-Natural. Shapiro AD, Ragni M, Borhany M, Abajas Y, Tarantino M, Holstein K, Croteau SE, Liesner R, Tarango C, Carvalho M, McGuinn C, Funding E, Kempton CL, Bidlingmaier C, Cohen A, Oldenburg J, Kearney S, Knoll C, Kuriakose P, Acharya S, Reiss U, Kulkarni R, Shullick M, Lethagen S, Donfield S, LeBeau P, Berntorp E, Astermark J.  Haemophilia, 2020. doi: 10.1111/hae.14139.
  • Bioengineering hemophilia A-specific microvascular grafts for delivery of full-length factor VIII into the bloodstream. Neumeyer, J, Wang, K, Croteau, SE, Hua, T, Lin, RZ, Melero-Martin, JM. Blood Advances, 2019. doi:10.1182/bloodadvances.2019000848
  • Regional Variation and Cost Implications of Prescribed Extended Half-Life Factor Concentrates among U.S. Hemophilia Treatment Centers for Patients with Moderate and Severe Hemophilia. Croteau, SE, Cheng, D, Cohen, AJ, Holmes, CE, Malec, LM, Silvey, M, Thornburg, C, Wheeler, AP, Kouides, PA, Raffini, LJ, Neufeld, EJ. Haemophilia. 2019. doi: 10.1111/hae.13758

Von Willebrand disease (VWD)

  • Shui M, D’Angelo L, Croteau SE. Low von Willebrand Factor in Pediatric Patients: Retrospective analysis of 293 cases informs Diagnostic and Therapeutic Decision-Making. Pediatr Blood Cancer. 2020; e28497, epub 23 Jun. doi:10.1002/pbc.28497 
  • Fifth Åland Island conference on von Willebrand disease, 22–24 September 2016: meeting report. Berntorp, E, Ågren, A, Aledort, L, Blombäck, M, Cnossen, MH, Croteau, SE, von Depka, M, Federici, AB, Goodeve, A, Goudemand, J, Mannucci, PM, Mourik, M, Önundarson, PT, Rodeghiero, F, Szántó, T, Windyga, J. Haemophilia. 2018;24(Suppl. 4):5–19. doi:10.1111/hae.13475

Women with bleeding disorders

  • The Spectrum of Bleeding in Women and Girls with Hemophilia B. Staber, J*, Croteau, SE*, Davis, J, Grabowski, EF, Kouides, PA, Sidonio, RF. Haemophilia. 2018; 24:180-185. doi: 10.1111/hae.13376