NBDF funds a broad range of research programs that seek to increase our understanding of the science behind bleeding disorders, how they affect people's lives, and pathways to better treatments and cures.

In vivo Selection of Hematopoietic Stem Cells that are Genetically-Modified to Express Platelet-FVIII for Hemophilia A Gene Therapy

In vivo Selection of Hematopoietic Stem Cells that are Genetically-Modified to Express Platelet-FVIII for Hemophilia A Gene Therapy

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Gene Therapy
Platelets
Hemophilia A (Factor VIII/F8)
Author(s):
Yingyu Chen

The goal of Dr. Chen's research is to examine a method for selectively expanding hematopoietic stem cells expressing the factor VIII transgene. She will also examine the immune consequences of this approach, based on the idea that gene transfer in platelets evades immune recognition. This research has the potential to elicit important clues to developing an approach for gene therapy of hemophilia A and hemophilia A with inhibitors.

Dr. Chen earned a PhD in hematology from Fujian Medical University in China. She already has more than 27 papers published in the Chinese medical literature. Her research in hemophilia and gene therapy will be under the mentorship of Dr. Qizhen Shi, MD, PhD, Associate Investigator at the Blood Research Institute and Assistant Professor of Pediatric Hematology at the Medical College of Wisconsin.

Lorraine Flaherty

Identifying Fall Risk in Patients with Hemophilia

Year:
-
Grants:
Physical Therapy Excellence Fellowship
Author(s):
Lorraine Flaherty

This research project will examine fall history and fall risk in patients with hemophilia. Multiple risk factors for falls identified in the general population are prevalent in the hemophilia population. Existing data suggest that fall rates may be higher and that fall risk may begin at an earlier age in patients with hemophilia. Identification of fall risk enables early intervention, thereby preventing injury and fear of physical activity, both of which have been associated with falling and may carry an increased risk in patients with bleeding disorders.

Identification and Amelioration of T-cell Mediated Inflammatory Cytokines that Contribute to Anti-Factor VIII Inhibitor Formation in Hemophilia A

Identification and Amelioration of T-cell Mediated Inflammatory Cytokines that Contribute to Anti-Factor VIII Inhibitor Formation in Hemophilia A

Year:
-
Grants:
Career Development Award
Hemophilia A (Factor VIII/F8)
Inhibitors
Author(s):
Keri C. Smith

Dr. Smith's research focuses on better understanding the development of factor VIII inhibitors. Specifically, she will focus on the effects of chemical signals, or cytokines, secreted by helper T cells on the development of inhibitor antibodies. She hypothesizes that certain cells, called Th17 cells, play an important role in the development of these antibodies by stimulating inflammation and driving the immune response toward inhibitor production. Dr. Smith received a BS from the University of Delaware before earning her Ph.D. from Montana State University. Prior to her appointment as an Assistant Professor at the University of Texas Medical School at Houston, she spent four years as a postdoctoral research fellow at the University of Michigan.

FXa Variants for Treatment of Hemophilia

FXa Variants for Treatment of Hemophilia

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Hemophilia A (Factor VIII/F8)
Hemophilia B (Factor IX/F9)
Author(s):
Lacramioara Ivanciu
Per Dr. Ivanciu, the JGP award was the starting point of her career in the field of hemophilia. By receiving this award, she was able to advance her understanding of bleeding disorders and gained the necessary experience in state-of-the-art techniques in animals, molecular biology and biochemistry of clotting factors. All of this has prepared her for the next phase of her career.
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A Web-Based, Real-Time Menstrual Tracking Tool

Year:
-
Grants:
Nursing Excellence Fellowship
Author(s):
Carolyn Solomon

Menstrual cycles for females with bleeding disorders can present special challenges not experienced by other girls and women. Having your period can be physically, social and emotionally exhausting. A new web-based application is now available and can be downloaded to your personal mobile device or desktop computer. With this application you can track how long your period lasts, how much you are bleeding, any interventions utilized and how it is affecting your daily life. This is a new web-based application you can download that is free and available to all girls and women with bleeding disorders through their federally funded hemophilia treatment center (HTC).

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Resilience and Quality of Life in Individuals Aging with Hemophilia

Year:
-
Grants:
Social Work Excellence Fellowship
Author(s):
Karen Allen, Ellen Kachalsky

Individuals with hemophilia are living longer than previously expected because of advances in treatment, including the development of clotting factor replacement. However, we do not understand the psychological and emotional impact of aging with hemophilia, how this affects quality of life, and how resilience and healthy coping skills are developed and maintained. For example, we do not know if persons with hemophilia: (a) retire early because of having a disability, although this may be a reasonable assumption; (b) if they are financially secure; and (c) how much joint damage impacts the ability to move, walk, and perform everyday activities. We also do not know what special challenges arise from aging with hemophilia, and if this leads to more divorces, an increase in living alone, and a greater need for help to remain independent. There is little research showing which traits help persons with hemophilia continue to mature, and keep an emotional balance while dealing with a lifelong, chronic condition and how all these traits improve or reduce quality of life.

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Gait Parameters of People with Hemophilia Compared to Normal Control Subjects

Year:
-
Grants:
Physical Therapy Excellence Fellowship
Author(s):
Nancy Durben

The objective of this study is to collect a large data base of gait parameters on individuals from the age of 12 months to 99 years old who do not have neuromotor deficits. This ongoing study will be the basis of comparison for people with hemophilia as a means to identify gait deficits that occur due to joint or muscle involvement from the bleeding disorder as compared to gender and age matched controls. We are using the GaitRite electronic walkway evaluation system as the quantitative measurement tool for gait analysis. Enrolled subjects are consented to this study and to the storage of data in a gait repository for future studies. This data will be available to other Hemophilia Treatment Centers upon written request to our Gait Repository Guardian (currently Dave Oleson, PT at the Hemophilia Treatment Center at CDRC in Portland, OR).

Fine Structure-Function Mapping VWF-FVIII Interaction

Fine Structure-Function Mapping VWF-FVIII Interaction

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Von Willebrand Disease
Hemophilia A (Factor VIII/F8)
Author(s):
Andrew Yee

Dr. Yee earned his undergraduate Credentials from Rensselaer Polytechnic Institute before receiving his doctorate from Rice University. His eventual goal is to operate an independent research laboratory in the field of bioengineering with a focus on understanding molecular mechanisms of hemostasis, thrombosis and vascular diseases. Dr. Yee's research project deals with factor VIII and its adhesion to its chaperone protein, von Willebrand factor. In these experiments, fragments of von Willebrand factor are screened for optimal factor VIII stabilization. By further studying the architecture of the "Factor VIII-von Willebrand Factor" complex, Dr. Yee seeks to gain insight to improve designs of novel therapeutics.

A New Approach for a New Generation

A New Approach for a New Generation

Year:
-
Grants:
Nursing Excellence Fellowship
Author(s):
Linda Grooms
The primary purpose of our project will be to provide education that is specific to adolescent females diagnosed with a bleeding disorder. Most of the information available in the bleeding community is gender specific to males, age specific to adults and focused on von Wilebrand Disease and not platelet dysfunctions. In order for these young girls to form a concrete knowledge base that they can build upon through their adult life, they need additional educational resources for their diagnosis and prescribed therapy as well as support.
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Socialization of Adult Men with Hereditary Bleeding Disorders and the Role of Spouse and Significant Other

Year:
-
Grants:
Social Work Excellence Fellowship
Author(s):
Christine Mitchell

The purpose of this pilot study is to characterize social integration of persons with congenital bleeding disorders (PWCBD), specifically adult males with hemophilia A or B. Due to early onset of bleeding symptoms, they are predisposed to acute, life-threatening bleeding, bleeding complications, risk for long-term disabling hemophilic arthropathy, and blood-borne infections. A European study has shown that adult males with hemophilia have inferior socialization as measured by work, work disability and health-related quality of life when compared to adult men in the general population. We hypothesize that a broader view of socialization components also contribute to quality of life. We also propose that the role of the spouse or significant other may have relevance for current social work goals in providing comprehensive care for adult PWCBD.

Pharmacogenomics of Hemophilia Therapy: Genetics of Inhibitor Antibody Response

Year:
-
Grants:
Career Development Award
Inhibitors
Author(s):
Jay Lozier
Studies of VWF Function in VWF-Platelet and VWF-FVIII Interactions

Studies of VWF Function in VWF-Platelet and VWF-FVIII Interactions

Year:
-
Grants:
Career Development Award
Von Willebrand Disease
Author(s):
Veronica Flood

Dr. Flood earned her undergraduate degree from Harvard University in 1995 and her doctorate from Tufts University in 1999. She is board certified in pediatrics as well as pediatric hematology/oncology. She has been serving as an Assistant Professor at the Medical College of Wisconsin, where she has been performing research and seeing patients since 2006.

As part of her NHF Career Development Award, Dr. Flood will study the common variants and interactions of the von Willebrand factor molecule as a means to enhance overall understanding and eventually improve upon current methods of von Willebrand disease testing. Using mouse models, she will investigate diagnostic alternatives by examining the interaction between ristocetin and its ability to bind to von Willebrand factor. As Dr. Flood states in her summary, "Improved testing for VWD will prevent patients with normal VWF function from receiving unnecessary treatment, while allowing more accurate assessment of patients with true defects in VWD."

Regulatory Mechanisms in the Activation of Blood Coagulation Factors V and VIII

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Factor V
Hemophilia A (Factor VIII/F8)
Author(s):
Mettine Bos
Qizen Shi

Immune Response in Platelet-Derived FVIII Gene Therapy of Murine Hemophilia A

Year:
-
Grants:
Career Development Award
Gene Therapy
Hemophilia A (Factor VIII/F8)
Platelets
Author(s):
Qizhen Shi

Recombinant Factor VIIa and RL Platelets as a Hemophilia Therapy

Year:
-
Grants:
Career Development Award
Platelets
Hemophilia A (Factor VIII/F8)
Hemophilia B (Factor IX/F9)
Factor VII/F7
Author(s):
Alisa Wolberg
Use of Emergency Medical Identification in the Pediatric Hemophilia Population: A National Study

Use of Emergency Medical Identification in the Pediatric Hemophilia Population: A National Study

Year:
-
Grants:
Nursing Excellence Fellowship
Author(s):
Jocelyn Bessette Gorlin
Nurses and health care providers recommend Emergency Medical Identification (EMI) such as Medic Alert for children of all ages with hemophilia and other bleeding disorders, but most are unsure of what type should be used and where they should be located. This is because there are no guidelines on how EMI should be worn. This project will develop guidelines for the use of Emergency Medical Identification (EMI) and develop educational information for families of children with hemophilia. Guidelines will show recommended ages to wear EMI, its use with infants, and where it should be worn or if it should be carried. In addition, this project will review barriers for not wearing EMI based on our research, including perceived lack of need, sizing concerns for infants, and the fear of being different as expressed by adolescents.
Women with Severe and Moderate Hemophilia A and B and Other Bleeding Disorders: A Grounded Theory Study

Women with Severe and Moderate Hemophilia A and B and Other Bleeding Disorders: A Grounded Theory Study

Year:
-
Grants:
Social Work Excellence Fellowship
Author(s):
Jacqueline Lefkowitz
This study will collect data from interviews with women who have moderate or severe hemophilia or another congenital bleeding disorder. Participants will be asked to express the impact that a congenital bleeding disorder has on the development and maintenance of identity and social relationships. It will provide women a chance to share how they feel about the impact that living with a bleeding disorder has on their lives. The results of this study will be of benefit to treatment providers and the entire community as we come to better understand the unique experiences of this segment of the bleeding disorders population.

Targeting Dendritic Cells to Induce Immune Tolerance to Factor VIII

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Hemophilia A (Factor VIII/F8)
Inhibitors
Author(s):
Ruijin Su

Proficient AAV Vectors for the Treatment of Hemophilia B (2007)

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Hemophilia B (Factor IX/F9)
Gene Therapy
Author(s):
Jyoti Mathur

Self-Regulating HIV Vectors for Hemophilia A Gene Therapy

Year:
-
Grants:
Career Development Award
Gene Therapy
HIV/AIDS
Hemophilia A (Factor VIII/F8)
Author(s):
Yashuhiro Ikeda