Research Studies

Dr. Zia earned a MBBS degree from King Edward Medical College, Pakistan. Following an internship in pediatrics at the Children's Hospital, University of Oklahoma Health Sciences Center, she completed her second and third years of pediatric residency at the Children's Hospital of Michigan, Wayne State University in Detroit. Since becoming a hematology/oncology fellow in 2009, Dr. Zia presented at the American Society of Hematology and has had five abstracts accepted for presentation at other major hematology meetings. Dr. Zia will continue her specialized training in pediatric hemostasis-thrombosis under the directorship of Dr. Jeanne Lusher. The NHF-Baxter Clinical Fellowship award will foster Dr. Zia's development as a clinical scientist, allowing her to study clinical research design, statistical analysis as well as pursue research concerning the thrombogenicity of hormonal supplementation in adolescent girls under the mentorship of Drs. Madhvi Rajpurkar and Michael Callaghan, a previous NHF/Baxter clinical fellowship recipient.

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.

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.

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).

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.

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).

Dr. Grace joined Children's Hospital Boston / Dana Farber Cancer Institute as a Pediatric Resident after receiving both her undergraduate and doctoral degrees from Brown University. She has since gone on to become a fellow in the institution's Hematology/Oncology program and was chief fellow in her final year of fellowship. As Dr. Grace begins her NHF-Baxter fellowship, she will be on staff as a Hematologist at Children's Hospital Boston / Dana Farber Cancer Institute. As an NHF-Baxter fellow, Dr. Grace will spend two years developing advanced skills in clinical hemostasis and clinical trial design. Under the mentorship of Dr. Ellis Neufeld, she will continue her work on several ongoing clinical studies. Dr. Grace is currently the Co-Investigator of a study on the genetic modifiers of chronic ITP. Dr. Grace's goal is to become a clinical expert in hemostasis and thrombosis as well as a productive independent clinical investigator. She hopes to contribute towards improving the treatment, outcomes and quality of life of pediatric patients with bleeding and clotting disorders through a career in clinical research.

Since 2009, Dr. Branchford has been a hematology/oncology/bone marrow transplant fellow at the Children's Hospital of Denver. Prior to this post, he served as Chief Resident at the Children's Hospital of Wisconsin-Milwawukee. Dr. Branchford received his doctoral degree from the University of Wisconsin School of Medicine and Public Health and his BA in Biology from Lawrence University. He is also a member of the American Academy of Pediatrics and Colorado Medical Society as well as the American Societies of Hematology, Clinical Oncology and Pediatric Hematology/Oncology. As an NHF-Baxter Clinical Fellow, Dr. Branchford will be continuing both his clinical and research training under the mentorship of Dr. Marilyn Manco-Johnson. In addition to this, he will be working in the lab of Dr. Jorge DiPaola on a project researching the microfluidic properties of von Willebrand Disease as well as the role of the TAM pathway in platelet activation and thrombus formation. In the long-term, Dr. Branchford's career goal is to serve as Director of a Hemophilia/Thrombosis center, providing clinical care as well as basic or translational research within the structure of an academic pediatric department.

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.

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.

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.

Dr. Boles is currently a hematology and oncology fellow with the University of North Carolina at Chapel Hill, where he received both his BS and MD. His fellowship mentor at UNC will be Dr. Nigel Key. Also involved in Dr. Boles' training will be Dr. Raj Kasthuri, a former NHF Clinical Fellowship recipient. The co-author of a number of publications, including many in the field of neuroscience, Dr. Boles has also been honored for his teaching skills. Training received during the course of the program will include rigorous laboratory research as well as career guidance in hemostasis. Dr. Boles hopes to continue his career in hematology post-fellowship and conduct research that will improve the lives of patients with hemophilia and other bleeding disorders.

Dr. Pels joined the hemophilia treatment center at Yale University as a pediatric hematology/oncology resident in 2004 after receiving her undergraduate degree from Johns Hopkins and her MD from the University of Vermont College of Medicine. Her recently-authored poster abstract on immune thrombocytopenia was selected for presentation at the 2009 Plenary Scientific Session of the Hemophilia and Thrombosis Research Society's Annual Meeting. As an NHF-Baxter Clinical Fellow, Dr. Pels will spend two years developing advanced skills in clinical hemostasis and learning rigorous methodologies of clinical trial design, completion and monitoring. She will also continue her work on several ongoing studies under the mentorship of Dr. Diana Beardsley. Dr. Pels intends to pursue a career that includes both academics and research, including clinical work in the broad areas of hemostasis and thrombosis. She is hopeful that this work will lead to better therapies for patients with hemophilia as well as other bleeding and clotting disorders.

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."

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.

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.