Glanzmann Thrombasthenia beyond bleeding: Insights from lived experience experts

Glanzmann Thrombasthenia beyond bleeding: Insights from lived experience experts

Year: 2024
Grants:
N/A
CVR
Author(s):
Maria Santaella, Samantha Carlson, Denise Kurta, Quinn Kennedy, Peter Zdziarski, Eliza VanZweden, Esmeralda Vázquez

Glanzmann Thrombasthenia (GT) is a rare inherited platelet disorder caused by a qualitative or quantitative defect of the glycoprotein IIb/IIIa complex. The bleeding phenotype, psychosocial impact, and patient perspectives in GT remain inadequately characterized. This abstract explores disease burden, unmet medical and psychological needs, and gaps in care associated with GT.

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The relationship between different pain measures, depression, and social support and race and ethnicity in persons with hemophilia

The relationship between different pain measures, depression, and social support and race and ethnicity in persons with hemophilia

Year: 2024
Grants:
N/A
CVR
Author(s):
Maria E Santaella, Matthew Hartnett, Luke Luckey, Cynthia D Nichols

Disparities in healthcare and health outcomes within minoritized and marginalized populations have been extensively documented in the literature. However, despite growing evidence that race/ethnicity may have a negative impact, few studies in BDs account for these variables when analyzing results. This analysis describes the relationship between age, birth sex, pain intensity, impact, and interference; depression, and social support and race/ethnicity in people with hemophilia A and B participating in CVR.

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The National Hemophilia Foundation’s State of the Science Research Summit: the foundation of a national research blueprint for inherited bleeding disorders

The National Hemophilia Foundation’s State of the Science Research Summit: the foundation of a national research blueprint for inherited bleeding disorders

Year: 2023
Grants:
N/A
Author(s):
Leonard A. Valentino, Michelle L. Witkop, Maria E. Santaella, Donna DiMichele & Michael Recht
Lived experience experts: a name created by us for us

Lived experience experts: a name created by us for us

Year: 2023
Grants:
N/A
Author(s):
Esmeralda Vázquez, Michelle Kim & Maria E. Santaella
Soliciting international perspectives on an American national research agenda for inherited bleeding disorders

Soliciting international perspectives on an American national research agenda for inherited bleeding disorders

Year: 2023
Grants:
N/A
Author(s):
Michelle L. Witkop, Fiona Robinson & Donna DiMichele
Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities to transform the care of people with hemophilia

Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities to transform the care of people with hemophilia

Year: 2023
Grants:
N/A
Author(s):
Duc Q. Tran, Craig C. Benson, Judith A. Boice, Meera Chitlur, Amy L. Dunn, Miguel A. Escobar, Kalpna Gupta, Jill M. Johnsen, James Jorgenson, Scott D. Martin, Suzanne Martin, Shannon L. Meeks, Alfredo A. Narvaez Jr, Doris V. Quon, Mark T. Reding, Ulrike M. Reiss, Brittany Savage, Kim Schafer, Bruno Steiner, Courtney Thornburg, Lena M. Volland & Annette von Drygalski
Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities for mucocutaneous bleeding disorders

Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities for mucocutaneous bleeding disorders

Year: 2023
Grants:
N/A
Author(s):
Robert F. Sidonio, Jr, Paulette C. Bryant, Jorge Di Paola, Sarah Hale, Meadow Heiman, G Shellye Horowitz, Christi Humphrey, Julie Jaffray, Lora C. Joyner, Raj Kasthuri, Barbara A. Konkle, Peter A. Kouides, Robert Montgomery, Keith Neeves, Anna M. Randi, Nikole Scappe, Cristina Tarango, Kelly Tickle, Pamela Trapane, Michael Wang, Brittany Waters & Veronica H. Flood
Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities for ultra-rare inherited bleeding disorders

Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities for ultra-rare inherited bleeding disorders

Year: 2023
Grants:
N/A
Author(s):
Diane Nugent, Suchitra S. Acharya, Kimberly J. Baumann, Camille Bedrosian, Rebecca Bialas, Kai Brown, Deya Corzo, Amar Haidar, Catherine P. M. Hayward, Peter Marks, Marzia Menegatti, Margaret E. Miller, Kate Nammacher, Roberta Palla, Skye Peltier, Rajiv K. Pruthi, Michael Recht, Benny Sørensen, Michael Tarantino, Alisa S. Wolberg & Amy D. Shapiro
Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research to advance the health of people with inherited bleeding disorders with the potential to menstruate

Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research to advance the health of people with inherited bleeding disorders with the potential to menstruate

Year: 2023
Grants:
N/A
Author(s):
Maureen K. Baldwin, Homa K. Ahmadzia, Diane L. Bartlett, Debbie Bensen-Kennedy, Vidhi Desai, Kristina M. Haley, Sherry L. Herman-Hilker, Amanda M. Kilgore, Roshni Kulkarni, Michelle Lavin, Shari Luckey, Kristen A. Matteson, Kristin Paulyson-Nuñez, Claire S. Philipp, Sachiko Ragosta, Kimberly Rosen, Dawn Rotellini & Angela C. Weyand
Institution:
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Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities in health services; diversity, equity, and inclusion; and implementation science

Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities in health services; diversity, equity, and inclusion; and implementation science

Year: 2023
Grants:
N/A
Author(s):
Vanessa R. Byams, Judith R. Baker, Cindy Bailey, Nathan T. Connell, Melissa S. Creary, Randall G. Curtis, Alexis Dinno, Christine J. Guelcher, Michelle Kim, Roshni Kulkarni, Susan Lattimore, Keri L. Norris, Lucy Ramirez, Mark W. Skinner, Susan Symington, Patricia Tobase, Esmeralda Vázquez, Beth B. Warren, Emily Wheat & Tyler W. Buckner
Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: facilitating research through infrastructure, workforce, resources and funding

Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: facilitating research through infrastructure, workforce, resources and funding

Year: 2023
Grants:
N/A
Author(s):
Margaret V. Ragni, Guy Young, Glaivy Batsuli, Emily Bisson, Shannon L. Carpenter, Stacy E. Croteau, Adam Cuker, Randall G. Curtis, Michael Denne, Bruce Ewenstein, Amber Federizo, Neil Frick, Kerry Funkhouser, Lindsey A. George, W. Keith Hoots, Shawn M. Jobe, Emily Krava, Christopher James Langmead, Roger J. Lewis, José López, Lynn Malec, Ziva Mann, Moses E. Miles III, Emma Neely, Ellis J. Neufeld, Glenn F. Pierce, Steven W. Pipe, Lisa R. Pitler, Leslie Raffini, Kathaleen M. Schnur & Jordan A. Shavit
The National Hemophilia Foundation State of the Science Research Summit initiative: executive summary

The National Hemophilia Foundation State of the Science Research Summit initiative: executive summary

Year: 2023
Grants:
N/A
Author(s):
Leonard A. Valentino, Michelle L. Witkop, Maria E. Santaella, Donna DiMichele & Michael Recht
Relationship between Perceived Social Support, Mental Health, Activity, and Chronic Pain in Persons with Inheritable Bleeding Disorders

Relationship between Perceived Social Support, Mental Health, Activity, and Chronic Pain in Persons with Inheritable Bleeding Disorders

Year: 2022
Grants:
N/A
CVR
Author(s):
Maria E Santaella, Cynthia Nichols, Samantha Carlson, Michelle Witkop

The aim of this analysis was to describe the relationship between self-reported mental health diagnosis, history of mental health treatment, participation in physical activity, chronic pain, and social support. Persons with inheritable bleeding disorders with self-reported mental health conditions had at least 1 mental health comorbidity, report a lack of perceived social support and were less physically active than Persons with inheritable bleeding disorders without a self-reported mental health condition

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National Hemophilia Foundation Convenes Diverse Community Voices to Define an Actionable National Research Blueprint for Inherited Bleeding Disorders

National Hemophilia Foundation Convenes Diverse Community Voices to Define an Actionable National Research Blueprint for Inherited Bleeding Disorders

Year: 2021
Grants:
N/A
Author(s):
Michelle L Witkop, Michael Recht, Donna DiMichele, Kevin Mills, Leonard A. Valentino, Maria E Santaella; National Hemophilia Foundation Convenes Diverse Community Voices to Define an Actionable National Research Blueprint for Inherited Bleeding Disorders. Blood 2021; 138 (Supplement 1): 4952. doi: https://doi.org/10.1182/blood-2021-149518
National Hemophilia Foundation Enlists Diverse Patient Voices to Inform a National Research Blueprint for Inherited Bleeding Disorders

National Hemophilia Foundation Enlists Diverse Patient Voices to Inform a National Research Blueprint for Inherited Bleeding Disorders

Year: 2021
Grants:
N/A
Author(s):
Maria E Santaella, Michelle L Witkop, Kevin Mills, Michael Recht, Donna DiMichele, Leonard A. Valentino; National Hemophilia Foundation Enlists Diverse Patient Voices to Inform a National Research Blueprint for Inherited Bleeding Disorders. Blood 2021; 138 (Supplement 1): 1904. doi: https://doi.org/10.1182/blood-2021-147857
Bleeding Disorder Data Registry Reveals Racial/Ethnic Disparities That Could Significantly Impact Patient Journey

Bleeding Disorder Data Registry Reveals Racial/Ethnic Disparities That Could Significantly Impact Patient Journey

Year: 2021
Grants:
N/A
CVR
Author(s):
Maria E Santaella, Michelle L Witkop, Cynthia Nichols, Rosaura Vidal, Leonard A. Valentino; Bleeding Disorder Data Registry Reveals Racial/Ethnic Disparities That Could Significantly Impact Patient Journey. Blood 2021; 138 (Supplement 1): 5004. doi: https://doi.org/10.1182/blood-2021-148025

The demographic disparities between WCNHs and other participants in the CVR are critical and emphasize the need to focus on correlations between known social determinants of health and self-reported health outcomes and quality-of-life information.

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Poor outcomes in people with hemophilia: Physician and subject matter expert perspectives

Poor outcomes in people with hemophilia: Physician and subject matter expert perspectives

Year: 2021
Grants:
N/A
CVR
Author(s):
Jarren Santos, Martin Chandler, Christine Gerber, Moses Miles, Maria Santaella, Michelle Witjop, Michael Recht
Integrated Hemophilia Patient Care via a National Network of Care Centers in the United States: A Model for Rare Coagulation Disorders

Integrated Hemophilia Patient Care via a National Network of Care Centers in the United States: A Model for Rare Coagulation Disorders

Year: 2021
Grants:
N/A
Collaboration/Team Models
Author(s):
Leonard A Valentino, Judith R Baker, Regina Butler, Miguel Escobar, Neil Frick, Susan Karp, Kollet Koulianos, Susan Lattimore, Diane Nugent, Joseph N Pugliese, Michael Recht, Mark T Reding, Michelle Rice, Constance B Thibodeaux, Mark Skinner

Rare, chronic diseases such as hemophilia and other congenital coagulation disorders require coordinated delivery of services for optimal outcomes.  Hemophilia Treatment Centers (HTCs) are specialized, multidisciplinary healthcare centers providing team-based care to meet the physical, psychosocial, and emotional needs of people with hemophilia (PWH) and may serve as a model for other rare coagulation disorders. Health-care purchasers, as well as the general medical community, may not appreciate the breadth and quality of services provided by HTCs. They exemplify the acculturalization and actualization of integrated care by providing comprehensive diagnostic and treatment services that reduce morbidity, mortality, avoidable emergency room visits, hospitalizations, and overall costs while promoting a longer lifespan and improved patient functioning and outcomes.

This is accomplished by a  team-based approach relying upon a shared decision-making model to effectively prevent complications and manage symptoms in PWH, who are dependent on high-cost treatments. This article provides a concise yet comprehensive description of the core components of an HTC and the regional and national networks in the United States, which together achieve their incomparable value for all stakeholders.

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Preferences of people with hemophilia A and B for treatments including gene therapies in the US: A discrete choice experiment

Preferences of people with hemophilia A and B for treatments including gene therapies in the US: A discrete choice experiment

Year: 2021
Grants:
N/A
Author(s):
Witkop M, Morgan G, O’Hara J, et al. Patient preferences and priorities for haemophilia gene therapy in the US: a discrete choice experiment. Haemophilia. 2021;1-14. https://doi.org/10.1111/hae.14383
The Relationship Between Self-Reported Physical Activity, Treatment Regimen, Mental Health and Pain In Persons with Hemophilia

The Relationship Between Self-Reported Physical Activity, Treatment Regimen, Mental Health and Pain In Persons with Hemophilia

Year: 2021
Grants:
N/A
CVR
Author(s):
Michelle Witkop; Maria Santaella; Rosaura Vidal; Cynthia Nichols.

The objective of this study was to assess the relationship between self-reported physical activity, treatment regimen, mental health, and pain in persons with hemophilia (PWH) enrolled in CVR. Despite education to the contrary, PWSH continue to engage in high-risk, aggressive physical activities and would like to be even more physically active. As treatment options progress, offering more opportunity for physical activity, research is required to understand an acceptable balance between benefit and harm in PWH.

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(2021). ABSTRACTS. Haemophilia, 27: 3-20. https://doi.org/10.1111/hae.14385