Research Studies

Dr. Patricia Zerra is currently completing her pediatric hematology/oncology fellowship at Emory University/Children's Healthcare of Atlanta (CHOA) and will begin an additional fellowship in Transfusion Medicine focusing on coagulation in July 2017. Dr. Zerra graduated from Connecticut College followed by two years of research at Boston Children's Hospital. She received her M.D. from Jefferson Medical College in Philadelphia and completed pediatrics residency at the University of Miami/Jackson Memorial Hospital where she served as chief resident with an additional year practicing general pediatrics. As an NHF-Shire Clinical Fellow, Dr. Zerra will work under the mentorship of Dr. Robert Sidonio, Pediatric Director of the Emory/CHOA Hemophilia Treatment Center to focus on the clinical management of children with bleeding disorders. She will also continue her current research under the mentorship of Drs. Shannon Meeks and Sean Stowell, focusing on the immune response to FVIII in an effort to identify initiating immune events that can serve as targets to prevent FVIII inhibitor formation. Her goal is to serve as a pediatric hematologist and clinical/translational researcher focusing on the care of pediatric patients at risk for developing inhibitors.

Dr. Karen Zimowski is a pediatric hematology/oncology fellow at Emory University/Children's Healthcare of Atlanta (CHOA). Dr. Zimowski received her BS in Biochemistry from Clemson University and her MD from the Medical College of Georgia. She completed pediatric residency at Johns Hopkins University. As a NHF-Shire Clinical Fellow, she will receive clinical training under the guidance of Drs. Robert Sidonio and Shannon Meeks in the Comprehensive Bleeding Disorders Clinic at CHOA and Special Coagulation Laboratory at Emory University. In addition, she will continue her current research projects, investigating the structure-function relationships of both coagulation factor V and factor VIII. Dr. Zimowski aims to become an expert physician-scientist in the field of pediatric hemostasis and thrombosis. She seeks to apply the knowledge gained through laboratory analysis to the clinical setting and provide the highest level of medical care to patients with bleeding and coagulation disorders.

Dr. Megan Rost is a postdoctoral fellow at the University of Michigan. She received a B.S in biochemistry and biotechnology from Michigan State University, and her Ph.D. in molecular and developmental biology at the University of Cincinnati - Cincinnati Children's Hospital Medical Center. Her graduate work focused on understanding vascular endothelial development using zebrafish as a model organism. In July 2015, she joined the lab of Dr. Jordan Shavit in the Department of Pediatrics and Hematology/Oncology at University of Michigan. For her 2016 JGP research fellowship project, she will be using the zebrafish model to analyze blood clot structure and function in the presence and absence of von Willebrand Factor. In studying this, Dr. Rost will be elucidating how arterial thrombus formation occurs in the absence of VWF, aiding in uncovering possible new therapeutic targets for VWD treatment.

Swimming is an important life skill that benefits hemophilia patients medically and psychosocially. As a recipient of the NHF " Social work Excellence Grant" our HTC has implemented a swim program for patients with hemophilia and other bleeding disorders. We currently have 17 children enrolled and are also monitoring these children by conducting before and after physical therapy and QOL examinations. 

Objective:

BAY 94-9027 is an extended–half-life recombinant factor VIII (FVIII) product. In the PROTECT VIII study, BAY 94-9027 provided effective protection against bleeds and was well tolerated with twice-weekly, every-5-day, and every-7-day prophylaxis in patients with severe hemophilia A. We report interim safety data from the PROTECT VIII extension study evaluating long-term outcomes in patients using BAY 94-9027 prophylaxis for >5 years .

Methods:

Previously treated patients aged 12 to 65 years with severe hemophilia A were enrolled in PROTECT VIII, in which they received BAY 94-9027 for 36 weeks on demand or as twice-weekly (30–40 IU/kg), every-5-day (45–60 IU/kg), or every-7-day (60 IU/kg) prophylaxis. Patients could subsequently participate in an extension study with the same or a different regimen. Adverse events (AEs), anti-PEG antibodies, inhibitor development, renal safety, and plasma PEG levels were evaluated during the extension phase.

Summary:

One hundred twenty-one of 134 patients from PROTECT VIII continued in the extension study receiving BAY 94-9027 either on demand (n=14) or as prophylaxis (n=107). At data cutoff (January 2018), patients aged 15 to 67 years at time of analysis (median age, 40 y) had a median (range) of 1420 (297–1965) days in the trial since enrollment and a median (range) of 223 (23–563) exposure days . Prophylaxis patients were treated either twice weekly (n=23), every 5 days (n=33), every 7 days (n=23), or switched frequency during the extension (n=28) . Overall, 9 patients (7.4%) experienced treatment-related AEs during the extension classified as either mild (n=4), moderate (n=4), or severe (n=1) . Two patients (1.7%) experienced 3 SAEs considered to be treatment-related (elevated liver function tests in a patient with hepatitis C ; 2 incidences of back pain); these 2 patients discontinued the study. Transient low-titer anti-PEG antibodies were detected at a single visit in 8 patients but were not associated with clinical events. No patients developed FVIII inhibitors or had sustained levels of detectable PEG in plasma . No specific changes in renal parameters were observed.

Conclusions:

During the ongoing PROTECT VIII extension, BAY 94-9027 prophylaxis was well tolerated for >5 years, and no patients developed FVIII inhibitors.