Research Studies

This study looked at how people with inherited bleeding disorders reported using cannabis to help manage symptoms. The researchers used survey answers from 88 adults in the Community Voices in Research registry. They wanted to learn why people used cannabis and whether users reported differences in pain, anxiety, depression, sleep, coping, and social support.

About 3 out of 10 people in this study said they used cannabis for symptom relief. People reported using it most often for pain, anxiety, and sleep problems. Many said it helped them, especially with sleep and anxiety. Cannabis use was reported by people with different types of bleeding disorders, not just one diagnosis.

The study also found that people who reported using cannabis had somewhat higher anxiety scores than people who did not report using cannabis. Among people taking prescription pain medicine, opioid use was more common in those who also reported cannabis use. The study did not find clear differences between groups in age, sex, diagnosis, depression scores, pain measures, coping, or social support.

These findings suggest that cannabis use may be fairly common in some people with inherited bleeding disorders, especially for symptom relief. But this study was small and based on self-reported survey answers, so it cannot show that cannabis is safe or effective for everyone. Patients and families should talk openly with their care team about pain, sleep, anxiety, depression, and any cannabis use so they can make informed decisions together.

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The National Research Blueprint is a proposal for a new Bleeding Disorders Research Collaborative (BDRC) doing the research people with bleeding disorders need and want. The people who live with a disorder, and their close family members affected by it, are Lived Experience Experts. The new collaborative will place Lived Experience Experts at the center of research. What research is done, how it is done, and how the results are used and shared must be decided in partnership with lived experience expertise. The collaborative must also advance health equity for all. Every initiative and project must improve diversity, inclusion, and belonging.

This paper proposes infrastructure and workforce development processes for the new research collaborative. Recommendations were developed by groups of clinical, research, lived experience, and health equity experts. The groups made sure everyone was able to contribute meaningfully and confidently. Every voice was heard and valued. This is also how the collaborative must operate, with shared leadership and teams that are trained to work well together. Education and processes are proposed to develop a diverse, inclusive interdisciplinary workforce, reflecting the community it serves and integrating lived experience expertise throughout. The proposed infrastructure is a network of expertise, resources, facilities, and processes, all connected by a platform. It is designed to start small, with just enough of each component to support a few simple projects. All projects will be evaluated to learn what works well and what can be improved. The whole collaborative will improve with learnings from each success and shortcoming.

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Previous work led by the U.S. National Bleeding Disorders Foundation (NBDF) concluded that Lived Experience Experts (LEEs), people, who live with inheritable bleeding disorders and their impacted caregivers and family members, develop unique knowledge about their disorder. It also recommended a national research collaborative guided by the principles of health equity, diversity, and inclusion (HEDI). Now, in four accompanying papers, seven expert working groups propose Research and Development, Workforce, Infrastructure, LEE, HEDI, Community Engagement, and Policy elements for a new Bleeding Disorders Research Collaborative. These groups all included lots of input from LEEs and HEDI experts, as well as researchers and healthcare providers. They made sure the insights and needs of all people with inheritable bleeding disorders were valued in every part of the plan.

In this paper, we present an overview of the whole collaborative. It will start out small and develop by learning from every project. The infrastructure and workforce will grow progressively; partnerships will be key in bringing together all the resources and expertise needed. LEE insights will be integrated into every stage of every research project and in every part of the organization and governance. HEDI principles will be respected in every study and in how the collaborative runs. Education, training, and mentorship will help everyone gain the skills they need to work well together. Shared leadership of the collaborative will ensure transparency, accountability, and the guiding principles of mutual respect, authentic partnership, and respectful communication. If the inheritable bleeding disorders community joins forces and works together, we can accelerate research that advances health justice for all.

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The National Research Blueprint (NRB) was a U.S. National Bleeding Disorders Foundation (NBDF) initiative to better understand all facets impacting research and to set the foundation for what and how inheritable bleeding disorders research should be done in the future. The personal journeys of Lived Experience Experts (LEEs), individuals living with disorders, are highly valuable. It is critical to incorporate them throughout all stages of future research. The NRB made sure LEEs were heard from the very beginning of the development process and throughout. The goal was to place diverse LEEs from across the community at the center of research, with all collaborative partners recognizing LEEs as equal partners.

One NRB working group developed a list of priorities to make future inheritable bleeding disorders research more inclusive and reflective of the community. They combined community input and medical, research, and lived experience expertise to choose 327 top research priorities. They ensured all priorities were feasible.

Another working group conducted a survey of the current workforce at bleeding disorders centers, often called hemophilia treatment centers (HTC). They asked the different professionals about their interest, capacity, and barriers in doing research. Based on the results, they proposed training and resources needed to develop a diverse workforce that can ensure successful future research.

There is a great potential for collaborative research across the country. HTCs can act as hubs in a network of national partnerships. The incorporation of LEEs as valued partners in this Bleeding Disorders Research Collaborative is imperative. Cross-training of LEEs, HTC professionals, and other researchers will be necessary to ensure its success.

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Over the past several years, bleeding disorders advocates and other community leaders have worked together to create the National Research Blueprint for a new U.S. Bleeding Disorders Research Collaborative. Its goal is for research to be more inclusive and to better meet the needs of all people with inheritable bleeding disorders. The National Research Blueprint was developed through the support of the U.S. National Bleeding Disorders Foundation.

The Bleeding Disorders Research Collaborative will be driven by people with inheritable bleeding disorders, and their impacted caregivers and family members; a group referred to as Lived Experience Experts. A Research Ambassador Program will train diverse Lived Experience Experts to contribute to all research teams and projects. Their insights will be integrated into all areas including, but not limited to, choosing and designing studies, running them, and sharing information about research with the community. Researchers will be trained to communicate and collaborate effectively with Lived Experience Experts, so they can work well together.

Everything the Bleeding Disorders Research Collaborative does will firmly apply the principles of health equity, diversity, and inclusion. The National Bleeding Disorders Foundation and the Bleeding Disorders Research Collaborative must partner with others who share this vision for research that prioritizes and respects the needs and perspectives of all people with inheritable bleeding disorders. Together they must secure policies and funding supporting this way of doing research. The success of the proposed Bleeding Disorders Research Collaborative requires the collective backing and engagement of the entire community: researchers, healthcare providers, Lived Experience Experts, advocacy organizations, regulators, policymakers, funders, and industry.

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The Bleeding Disorders Research Collaborative (BDRC) aims to advance an accessible standard of care and quality of life for all people living with inheritable bleeding disorders. This goal will be achieved through collaborative and meaningful scientific inquiry, coordinated by an efficient research infrastructure, and undertaken by a diverse, capacitated workforce in partnership with an engaged community. The BDRC is supported by facilitative research policy and grounded in the principles of health equity, diversity, inclusion, accessibility, and belonging, striving for dignity, safety, well-being, and opportunities leading to health justice. Importantly, the initiative is fully informed by Lived Experience Experts, people affected by inheritable bleeding disorders, who are key members in the research development, implementation, and dissemination team.

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Dr. Luisanna Sánchez is a pediatric hematologist at Innovative Hematology and the Indiana Hemophilia & Thrombosis Center in Indianapolis, where she cares for children and adolescents with inherited blood disorders. She is board certified in Pediatric Hematology/Oncology and is deeply committed to advancing clinical research that improves care and long-term outcomes for patients and families affected by blood disorders. Dr. Sánchez’s clinical and research work focuses on sickle cell disease, iron deficiency anemia, and expanding access to evidence-based care for young people with blood disorders. She has led national and international research collaborations, contributed to clinical guidelines and education initiatives, and is dedicated to developing patient-centered research that translates scientific discoveries into better everyday care.

Dr. Sánchez’s NBDF-supported research studies why some children with sickle cell disease respond better than others to hydroxyurea, a medication that helps prevent pain, organ damage, and other serious complications. Her project examines clinical and blood cell patterns to better understand how this treatment works and why responses differ between patients. The goal is to help doctors predict which patients will benefit most and how treatment can be optimized earlier in life. By supporting more personalized treatment approaches, this work aims to reduce complications, improve quality of life, and help families feel more confident that treatments are working, advancing NBDF’s mission to prevent complications and improve outcomes across the bleeding and blood disorders community.

I received by BS in Biochemistry from the University of Arizona in 2012. I then received my PhD at the University of Maryland, Baltimore County while studying under Dr. Elsa D. Garcin. I am currently a postdoctoral scholar at Western Washington University under Dr. P. Clint Spiegel. Our research focuses on the structure/function of activated coagulation factor VIII and factor IX and how the two proteins bind to lipid membranes to form the intrinsic tenase complex. The results from this research will elucidate the mechanism behind hemophilia A/B-associated missense mutations and how factor replacement therapeutics can be rationally designed for increased pharmacokinetic properties.

Describe the self-reported comorbidities (SRC) in PWBD, using data from Community Voices in Research (CVR), a registry aimed at collecting the experience of persons affected by these disorders and supported by National Bleeding Disorders Foundation.

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Louise Baca, MSN, RN, has over 40 years of healthcare experience, primarily specializing in oncology. She has worked in various settings, including inpatient oncology, outpatient infusion, clinical research, and as a nursing instructor. For the past 14 years, she has served in an administrative role, currently as Senior Director at Maine Medical Center, where she leads the MaineHealth Bleeding Disorders program. Under her leadership, the program has grown significantly, expanding from 2.5 to 15 FTEs and enhancing patient care in Maine and New Hampshire. Louise also serves on several committees, including the Hemophilia Alliance Board of Directors and the New England Regional Executive Committee, and has presented nationally on healthcare topics.

This project is a nurse led project (APNs and RN’s) and focuses solely on developing relationships with nurses and other clinicians in rural areas. As the only HTC in the state, it is the responsibility of the MaineHealth Bleeding Disorder nurses to educate other nurses in rural parts of Maine who will be the first point of contact. This work will create a blueprint for nurses, providing them with comprehensive information and practical ideas that can easily be implemented and assist them in caring for patients living in rural areas. These successful strategies will allow for the sharing of best practices ultimately elevating the standard of nursing care across all HTCs.

CC is a Hematology Social Worker & Health Equity Specialist at the Utah Center for Bleeding and Clotting Disorders at Primary Children’s Hospital in Salt Lake City, UT. She works primarily with children and adolescents with bleeding disorders, and she has a special interest in the sickle cell disease population. In May 2024, CC obtained a master's degree in both Social Work and Public Health from the University of Utah. In her current position, she is looking to strengthen her clinical skills, learn about her patients' needs, and advocate for better health outcomes for the most vulnerable members of the community.

The purpose of this project, "Identifying and Addressing Gaps in Care Among Menstruating Individuals in Utah," is to better understand the gaps that exist in caring for individuals in Utah with heavy or prolonged menstrual bleeding, as well as implement interventions to address these gaps. Such interventions may include working with primary care providers to screen for individuals with heavy periods, educating medical professionals on the treatment that is available for the management of heavy or prolonged periods, and ensuring that referrals made to specialists by primary care physicians are efficient and appropriate. 

John “Jack” DeLoach, DPT, specializes in outpatient pediatrics and early intervention. He has worked at UF Health Rehab Center and Kids on the Move in Gainesville for the past three years, treating a wide range of patients in clinical, home, and telehealth settings. In addition to his clinical work, John serves as an adjunct faculty member at the University of Florida and is the physical therapist for the Hemophilia and Hemostasis Treatment Center. His expertise includes piloting telehealth programs, advancing joint health clinics, and providing care in critical care settings for both pediatric and adult patients.

People with hemophilia receive regular clotting factor infusions in order to prevent bleeds, as well get routine blood draws during clinic visits. Vascular access can be difficult in children with hemophilia and can become a stressor, impacting compliance with medication schedules and attitudes toward healthcare systems and procedures. This project aims to explore the impact of exercise on the ease of vascular access. We hope to show that regular exercise, as well as exercise immediately before an infusion/blood draw, will make accessing veins easier. We will also collect data on the impact of our protocol on fear of needles and fear of movement/exercise, in hopes that easier vascular access will decrease fear.  

Dr. Tam E. Perry is an associate professor at Wayne State University School of Social Work.  Her research addresses urban aging from a life course perspective, focusing on how underserved older adults navigate their social and built environments in times of instability and change. She is co-director of the NIH funded Community Liaison and Recruitment Core of the Michigan Center for African American Aging Research. She also currently serves as research chair for a multi-agency coalition, Senior Housing Preservation Detroit. She has recently been selected to be a fellow in the Gerontological Society of America and is the past-president of the Association for Gerontology Education in Social Work (AGESW).

She became interested in the hemophilia community as she learned more its history, resilence and advocacy and vulnerability. She is excited that she is meeting more individuals touched by this research topic and has enjoyed sharing the work in a variety of venues.

Dr. Sara L. Schwartz, PhD, MSW is an Associate Teaching Professor in The University of Southern California Suzanne Dworak-Peck School of Social Work. In this role, Dr. Schwartz teaches across the master and doctoral programs and recently founded the Visual Social Work Certificate Program. Her scholarship focuses on the experiences of aging with hemophilia and HIV/AIDS from a tauma informed perspective; the intersectional experiences of religion, race and history; and building a visual social work methodology. For the past five years, Dr. Schwartz has served on the Board of the Directors for the National AIDS Memorial and previously served on the Board of The HIV Story Project in San Francisco.

Ellen Kachalsky has served the hemophilia community for many decades.  Previously received funding from NHF with Dr. Karen Allen, in the area of aging and hemophilia from the practitioner context and we co-authored an article on "Aging with Hemophilia - Implications for Social Work Practice." Also published a number of articles on related topics such as pain management with her HTC colleagues at Henry Ford Health in collaboration with Munson Medical Center, as well as actual and ideal roles of practitioners in hemophilia treatment centers with a group of HTC Social Workers from across the United States.

Ellen has her Masters of Social Work degree from Adelphi University in New York, but then moved to Michigan, with her husband.  Ellen has worked at Henry Ford Hospital for over 25 years, including Bone Marrow Transplant and Hem-Onc, the Multi-Disciplinary Clinics for Breast Cancer, Head and Neck Cancer, and Brain Tumors.  She has worked in Hemophilia since 1999, with prior experience also in Family Practice, ICU and Step-Down units at other hospitals in the metropolitan Detroit area. She also worked for 3 years as the Resident Service Coordinator at HUD-subsidized Senior congregate living, where she helped residents maintain their independence by linking them with community resources and providing support, counseling and programs.
 
She enjoys helping clients with learning and growing with their conditions, as well as teaching about the ins and outs of insurance so that they can get the maximum benefit from their insurance plans, financial and work/career considerations.  Her goals include helping clients realize their full potential in education and employment so that they may lead happier and productive lives, in spite of issues related to bleeding disorders, and to help them adapt to changes as they mature and change

Dr. Qian Liang received the MBBS degree in 2010 from Sichuan University and the M.S. degree in 2013 from Shanghai Jiaotong University in China. Dr. Liang worked as a Research Fellow in the Department of Laboratory Medicine in Shanghai Ruijin Hospital, which is a Heamophilia Treatment Centre for hard-to-treat patients in the Southeastern region of China. She received her Ph.D. degree in Laboratory Medicine in 2022, and is currently a visiting postdoctoral fellow in Professor Renhao Li’s lab in the Aflac Cancer and Blood Disorder Center, Department of Pediatrics at Emory University School of Medicine. Her research work focuses on the structure and function of von Willebrand factor, as well as the development of related diagnostics and therapeutics, and she has published 6 research papers.

Tomasz received his Pharm.D. degree in 2019 in Pharmacology and Toxicology science from Medical University of Bialystok, Poland, and immediately started his postdoctoral appointment at Vascular Medicine Institute at the University of Pittsburgh, PA. Tomasz's research focuses on the innate immune mechanisms in platelets and neutrophils as well as thrombo-inflammation pathophysiology. He uses cutting-edge intravital microscopy techniques to image in real-time the interplay between neutrophils and platelets during the initial stages of immune system activation. His work has authentic interdisciplinary nature since he studies cross-talk between innate immune signaling in neutrophils, Factor VIII deficiency, liver diseases and macroscale proteomics and genomic profiles of neutrophils and platelets under inflammatory stress. Until now, he proved that neutrophil activation seems to be a key player in the hemophilic arthropathy progression, dedifferentiated sinusoidal endothelium impacts liver-directed gene transfer in Hemophilia-a mice, and that liver to lung microemboli NETs promote Gasdermin-D-dependent inflammatory lung injury in Sickle Cell Disease. Tomasz has been appreciated with multiple awards from national and international societies and institutions as well as his research activities were supported by numerous extramural funding sources. In his scientific and personal life, he proudly follows the University of Pittsburgh's motto - Veritas et Virtus.

Web-based registries are essential tools for gathering patient-reported information for research purposes. The National Bleeding Disorders Foundation recognised and responded to the imperative to actively include persons with inheritable bleeding disorders and their immediate family members, as Lived Experience Experts (LEEs), in all aspects of the research process. Consequently, Community Voices in Research (CVR) was developed and launched with the aims of creating a LEE-centred approach to complement existing research efforts, highlighting mechanisms to actively incorporate them, and using the data collected to identify relevant gaps in knowledge.

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Katie Klütz, MSW, LCSW is the social worker for pediatric patients at Orlando Health Arnold Palmer Hospital for Children’s HTC in Orlando, Florida. She earned a bachelor’s degree in Social Work with a double major in Psychology from Florida State University in 2005 and earned a master’s degree in Social Work in 2006 from the same institution. Katie began working with inpatient Hematology/Oncology pediatric patients and their families in 2017 and transitioned to working exclusively with the HTC full time in January 2021.