Boston Hemophilia Center | 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 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 Studies

Hemophilia

• 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

Hemophilia

• PK Tailored Approach to Prophylaxis: Medical Decision Making and Outcomes. Croteau, SE, Wheeler, AP, Khan, O, Haley, K, Borst, AJ, Lattimore, S, Yeung, CHT, Iorio, A., Res Pract Thromb Haemost. 2020; 4(2): 326-333.
• 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, 3(24): 4166-4176.
• Application of Web Accessible Population Pharmacokinetic Service – Hemophilia (WAPPS-Hemo): Patterns of blood sampling and patient characteristics among clinician users. McEneny-King, A, Yeung, CHT, Edginton, AN, Iorio, A, Croteau, SE. Haemophilia, 2019 Jan; 26(1):56-63.
• Awareness, Care, and Treatment in Obesity Management to inform Hemophilia Obesity Patient Empowerment (ACTION-TO-HOPE): Results of a Survey of US Patients with Hemophilia and Obesity (PwHO) and Their Partners and Caregivers, Haemophilia. Croteau, SE, Cutter, S, Hernandez, G, Wicklund, B, Dreyer, ML, Gillette, D, Haugstad, K, Cooper, DL, Ostrow, V, Nadglowski, J. 2020; 26: 3– 19. 
• Neuropsychological function in children with hemophilia: A review of the Hemophilia Growth and Development Study and introduction of the current eTHINK study. Buranahirun, C, Walsh, K, Mrakotsky, C, Croteau, SE, Rajpurkar, M, Kearney, S, Hannemann, C, Wilkening, GN, Shapiro, KA, Holbrook, J, Cooper, DL., Pediatric Blood & Cancer, 2020; Jan;67(1):e28004. Epub 2019 Oct 8. 
• 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; epub Apr 17.
• Focusing in on use of PK profiles in routine hemophilia care. Croteau, SE, Callaghan, MU, Davis, J, Dunn, AL, Guerrera, M, Khan, O, Neufeld, EJ, Raffini, LJ, Recht, M, Wang, M, Iorio, A.  Research and Practice in Thrombosis and Haemostasis, 2018; 2:607–614.

Von Willebrand Disease (VWD)

• 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.