For many years the Center has been actively involved in clinical research studies to help cultivate advances in the medical conditions that we treat. If a patient is interested in being involved in a research study at the Center, they would discuss with the doctors and nurses involved what the study is about and what the expectations of them would be. Typically, research activities and patient care are integrated. It is important that patients derive benefit from their participation in research at the Center. To learn more about opportunities to become involved in research, please ask any staff member at the Center. A partial list of studies currently underway at the Hemophilia Center includes the following:
Hemophilia Studies
Hemophilia Study – IRB#: 2056-B Kouides
Title: ATHN 11 – Observational Cohort Study – Liver Transplantation Outcomes Study
Description: To examine the impact of sustained increase in Factor VIII and Factor IX levels after liver transplantation on quality of life (QoL) as measured by pain, health outcomes, work, activity, and mental and physical health in adults with Hemophilia A and B.
Hemophilia Study – IRB#: 2071-A Kouides
Title: Age and Hemophilia Carrier Phenotype Study – Elucidating the age-dependent variation of clinical and laboratory phenotype of hemophilia carriers along with prescription trends of hemostatic therapies – Analysis of the American Thrombosis and Hemostasis Network (ATHN) dataset
Description: Characterize the phenotypic variability of female symptomatic and asymptomatic carriers of hemophilia across the entire age-spectrum, womb to tomb
Hemophilia Study – IRB#: 2032-B Kouides
Title: ATHN 10 – Leveraging the ATHNdataset to Document the State of Rare Coagulation Disorders in the United States
Description: 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-affiliated network.
https://athn.org/what-we-do/national-projects/athn10.html
Von Willebrand Studies
vWD Study – IRB#: 1958-A Kouides
Title: ATHN 9 – A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment of People with Severe von Willebrand Disease
Description: To assess the safety of various von Willebrand Factor (VWF) regimens for different indications (on-demand, surgery and prophylaxis) in adult and pediatric participants with clinically severe congenital VWD.
vWD Study – IRB#: 1911-B-19 Kouides
Title: ATHENA1 – Characterizing the impact and treatment of reproductive tract bleeding in women and post-menarchal girls with bleeding disorders
Description: Characterize reproductive tract bleeding in a cohort of women/girls with bleeding disorders cared for at US HTCs. Characterize the treatment strategies for and the impact of heavy menstrual bleeding in a cohort of females with bleeding disorders cared for at HTCs.
vWD Study – IRB #: 1732-A-17 Kouides
Title: Genotype and Phenotype Analysis of Adolescents with Heavy Menstrual Bleeding and Low von Willebrand Activity
Description: Study the genotype of adolescent females with heavy menstrual bleeding and low vWF activity.
vWD Study – IRB#: 1807-B-18 Kouides
Title: Pregnancy Loss in von Willebrand Disease: A Multi-Centre Cohort Study
Description: Determine the rate of pregnancy loss in women with VWD, compared to a non-bleeding disorder
vWD Study – IRB #: 2084-B Kouides
Title: The von Willebrand Disease Aging and Bleeding Correlation (VWD ABC) Study
Description: Determine if age in patients with type 1 VWD is associated with increased VWF antigen levels and lower VWD bleeding scores
Hemochromatosis Studies
Hemochromatosis Studies – IRB #: 1331-A-11-1 Phatak R24 Grant
Title: Genetic Modifiers of Iron status in Hemochromatosis HFE C28ZY Homozygotes
Description: To determine the genetic and other factors that may influence iron absorbtion and account for some of this variation among those already known to be homozygous (2copies) for the C282Y HFE mutation.
Hemochromatosis Studies – IRB #: 1081-A-08-1 Phatak
Title: The Role of Hepcidin and Hemojuvelin in Iron Regulation (UCLA Study)
Description: To evaluate familie with non-HFE hemochromatosis to determine how to recently discovered proteins, hepcidin and hemojuvelin, play a role in iron metabolism.
Hemochromatosis Studies – IRB #: 749-B-03-1 Phatak
Title: Genotypic and Environmental Determinates of Penetrance in Hereditary Hemochromatosis
Description: To explore dietary and genetic factors that may modulate iron absorption rates and thereby influence penetrance in these subjects.
