Global Hemophilia Report

Host Patrick James Lynch and co-host Dr. Donna D. Micheli frame hemophilia progress as the product of sustained collaboration and investment, not inevitability, amid an increasingly uncertain research environment. Joined by University of Michigan physician-scientist and HTRS president Jordan Shavit, University of Washington professor and bleeding disorders center director Rebecca Kruse-Jarres, and HTRS executive director Lorilyn Jacobsen-Tews, they discuss momentum in gene therapy, non-factor and longer-acting factor options, and emerging ideas like oral therapies, alongside a broader, more inclusive focus across bleeding disorders. The panel highlights shrinking federal funding, the growing importance of professional societies and foundation support, and mentorship pipelines that attract and retain investigators from student to mid-career stages. They emphasize patient partnership in defining meaningful outcomes and express optimism rooted in community, collaboration, and the long arc of scientific progress.   Guests: Lorilyn Jacobsen-Tews Executive Director, Hemostasis and Thrombosis Research Society (HTRS) Rebecca Kruse-Jarres, MD Professor of Medicine and Pediatrics, University of Washington Executive & Medical Director, Washington Center for Bleeding Disorders Jordan Shavit, MD, PhD Physician-Scientist & Professor of Pediatrics and Hematology, University of Michigan    Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes: Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on X/Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on X/Twitter   

At the World Federation of Hemophilia World Congress in Kuala Lumpur, thousands gathered to discuss the future of bleeding disorders care. Gene therapies. Rebalancing agents. Artificial intelligence. New possibilities seemed to be everywhere. But beneath the excitement, one question echoed throughout the meeting: who still gets left behind? In this episode of the Global Hemophilia Report, Patrick Lynch sits down with Believe Limited's Amy Board, WFH Humanitarian Aid Director Assad Haffar, and Sanofi's Bonnie Anderson to explore the promises of innovation, the realities of global inequity, and the humanitarian efforts working to close the gap. From breakthrough science to life-changing access programs, this conversation examines what "Treatment for All" really means in 2026. Guests: Amy Board – Director, Engagement and Programs, Believe Limited Assad Haffar – WFH Humanitarian Aid Director Bonnie Anderson –  Head of Humanitarian Aid, Rare Diseases at Sanofi   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report Show Notes:   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on X/Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on X/Twitter   

The hemophilia treatment landscape is evolving faster than ever. In this episode of Global Hemophilia Report, Patrick James Lynch and Dr. Donna DiMichele are joined by Drs. Hermans, Carpenter, and Hansen to break down the emerging class of rebalancing agents—therapies that don't replace clotting factor, but target new parts of the coagulation cascade. We explore what these treatments mean for patients, clinics, and shared decision-making around the world. Key takeaways include the urgent need for real-world data, better patient education, and the potential for new therapies to protect joint health in ways never seen before. Tune in to hear expert insights, practical considerations, and the future of hemophilia care. Guests: Dr. Cedric Hermans, MD, - Hemophilia Centre,  Saint-Luc University Hospital, Brussels  Dr. Shannon Carpenter, MD - Pediatric Hematology, Kansas City Hemophilia Center: CJ Hansen BSN, RN - Nurse Coordinator & Program Manager,  OSU Comprehensive Cancer Center, Patient Advocate   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes:   The Bigger Picture in Hemophilia B:  Hemophilia A and hemophilia B are different bleeding disorders with unique pathologies and clinical features.1 Due to the distinct behavior of factor IX, multiple PK parameters should be considered when assessing bleed prevention. Learn how a broader view of PK may influence evaluation of treatment and management for patients with hemophilia B.2,3 Learn more at thebiggerpictureinhemb.com  1. Castaman G, Matino D. Haematologica. 2019;104(9):1702-1709. 2. Dolan G, Benson G, Duffy A, et al. Blood Rev. 2018;32(1):52-60.  3. Mann DM, Stafford KA, Poon M-C, Matino D, Stafford DW. Haemophilia. 2021;27(3):332-339.   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on X/Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on X/Twitter   

This season opener explores comprehensive hemophilia care in 2025: what's working, what's strained, and what needs to evolve. Clinicians from the US, UK, and Ireland join host Patrick James Lynch and Dr. Donna DiMichele to discuss treatment personalization, shared decision-making, psychosocial burden, and global sustainability challenges. Guest Megan Iditarod from Nigeria offers perspective on delivering comprehensive care with limited resources, highlighting the universal need for multidisciplinary support and good data collection.   Guests: Megan Iditarod Kate Khair, PhD Amy Dunn, MD Prof Niamh O'Connell, MB PhD   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes:   #MyHemophiliaTruth. Real stories. Powered by Sanofi. It's time to consider the whole experience when it comes to managing hemophilia and change the conversation. Explore the full story at www.myhemophiliatruth.com   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on X/Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on X/Twitter   

In this special episode, host Patrick James Lynch reflects on turning forty…and on forty years of life with hemophilia. Joined by Dr. Donna DiMichele, Patrick traces the parallel evolution of his own experience and the hemophilia community's journey through crisis, discovery, and innovation. From the HIV epidemic of the 1980s to the arrival of gene therapy and digital health, they explore how science, data, and humanity have shaped the story of hemophilia. Together, they ask: what have we learned, what still can't be measured, and what might the next forty years hold? Guests: Donna DiMichele, MD   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes:   Join Kevin as he shares about his journey with hemophilia and the hurdles he faced in communicating with friends, family, and healthcare providers about his condition. He highlights how hemophilia affects far more than just physical health — and why honest, open conversations that focus on the full patient experience, not just symptoms, are so essential.   Click here to watch his story: https://www.youtube.com/watch?v=3v1cCTbhClA&list=PLmqBxf22n4lNK82h3QZ-9YlpIjYdzDOer&index=5   Rich's Story Join Rich as he discusses how hemophilia has impacted his ability to travel and live an active lifestyle. He highlights how hemophilia affects far more than just physical health and shares his journey of overcoming barriers to be able to safely travel internationally, fly fish, garden, and more.    Click here to watch his story: https://www.youtube.com/watch?v=ihEvFywE43E&list=PLmqBxf22n4lNK82h3QZ-9YlpIjYdzDOer&index=8   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on X/Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on X/Twitter   

In this episode, we take a deep dive into the state of the treatment pipeline for bleeding disorders. From the way clinical trials are structured to what's actually available for different conditions, we examine both the promise and the reality of innovation in this space. Recorded live at the NBDF Bleeding Disorders Conference, we join Mike Recht, MD, PhD, Chief Science and Medical Officer of NBDF, for a "research posters walk & talk" to explore what's happening right now — and why so many promising treatments never make it to market. Guests: Mike Recht, MD, PhD Maria Santaella, PhD(c), MSN, RN-BC, CPHON Samantha Carlson, LMSW   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch   Written by: Kay Vermeil   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes:   Join Kevin as he shares about his journey with hemophilia and the hurdles he faced in communicating with friends, family, and healthcare providers about his condition. He highlights how hemophilia affects far more than just physical health — and why honest, open conversations that focus on the full patient experience, not just symptoms, are so essential.   Click here to watch his story: https://www.youtube.com/watch?v=3v1cCTbhClA&list=PLmqBxf22n4lNK82h3QZ-9YlpIjYdzDOer&index=5   Sanofi's Global Hemophilia Survey uncovers significant care gaps and emotional challenges faced by patients and caregivers. Learn how improving health literacy and fostering better patient-provider communication are essential to addressing these inequities. Explore the findings and see how Sanofi is driving health equity for the hemophilia community. Explore the survey findings here: Global Hemophilia Survey Page. Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on X/Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on X/Twitter   

This episode of the Global Hemophilia Report explores the long-overdue conversation about women with hemophilia. Experts discuss the challenges women face in getting diagnosed, the persistent gaps in clinical research and care, and the impact of outdated language and systemic bias. The panel highlights the need for better data, more inclusive clinical trials, and a shift in medical culture to ensure women with hemophilia are recognized and prioritized.    Guests: Andra James, MD, MPH Michelle Sholzberg MDCM, FRCPC, MSc. Dawn Rotellini Len Valentino, MD Robert Sidonio, Jr., MD   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch   Written by: Kay Vermeil   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes:   Presenting Sponsor: Presented by Sanofi Join Shellye as she shares her inspiring journey as a woman living with hemophilia. After years of unanswered questions, her diagnosis highlights the critical role of health literacy in bridging care gaps. Through advocacy, Shellye raises awareness and empowers underserved communities. Watch her story and see how health literacy can break barriers and transform lives. Click here to watch her story: Sanofi – See Hemophilia Through My Eyes: Women & Girls For too long, women and girls who bleed have been dismissed. Left out of the narrative. Ignored by the system. But not anymore. In our new film, "Dismissed," meet Isabelle—a 15-year-old with hemophilia who's using her voice to uplift the unheard. Alongside her are four powerful stories of women challenging what's "normal" and demanding recognition, care, and justice. This is more than a film. It's a movement. 📽️ Watch and share the trailer now. Learn more at http://dismissedfilm.com/   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on X/Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on X/Twitter  

In this episode of the Global Hemophilia Report, host Patrick James Lynch and a panel of experts discuss the importance of real world data and patient engagement in hemophilia care. The conversation explores how data collected outside of clinical trials provides deeper insights into treatment outcomes, challenges, and lived experiences. Guests share strategies for improving data reliability, motivating patient participation, and balancing privacy with research needs. Tune in for key takeaways on how both numbers and personal stories shape better care for the hemophilia community.   Guests: Mike Recht, MD, PhD, MBA Samantha Gouw, MD, PhD Maria Santaella, RN-BC, MSN, PhD(c)   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch   Written by: Kay Vermeil   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes:   Presenting Sponsor: Presented by Sanofi   Sanofi's Global Hemophilia Survey uncovers significant care gaps and emotional challenges faced by patients and caregivers. Learn how improving health literacy and fostering better patient-provider communication are essential to addressing these inequities. Explore the findings and see how Sanofi is driving health equity for the hemophilia community. Explore the survey findings here: Global Hemophilia Survey Page. For too long, women and girls who bleed have been dismissed. Left out of the narrative. Ignored by the system. But not anymore. In our new film, "Dismissed," meet Isabelle—a 15-year-old with hemophilia who's using her voice to uplift the unheard. Alongside her are four powerful stories of women challenging what's "normal" and demanding recognition, care, and justice. This is more than a film. It's a movement. 📽️ Watch and share the trailer now. Learn more at http://dismissedfilm.com/   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on X/Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on X/Twitter   

On this episode, we explore the concept of shared decision making (SDM) in modern hemophilia care. With insights from experts Brendan Hayes and Dr. Cedric Hermans, the discussion explores the evolution of treatment options, patient education, and the collaborative decision-making process. The conversation also highlights the development and impact of the SDM tool by the World Federation of Hemophilia, emphasizing the importance of patient empowerment and ongoing education.   Guests: Kate Bazinsky Randy Curtis Debbie de la Riva Jackie Bottacari   Senior Advisor: Donna DiMichele, MD   Hosted & Written by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes:   #MyHemophiliaTruth. Real stories. Powered by Sanofi. It's time to consider the whole experience when it comes to managing hemophilia and change the conversation. Explore the full story at www.myhemophiliatruth.com   Emil's Story: Join Emil as he opens up about his journey living with hemophilia and the profound impact it has had on his mental health. He emphasizes how the effects of hemophilia extend beyond physical symptoms and underscores the importance of recognizing its emotional and psychological challenges. Emil also highlights the need for comprehensive care that supports both the mental and physical health of children and adults living with hemophilia.   Click here to watch his story: https://www.youtube.com/watch?v=9ljWxS57-ZA&list=PLmqBxf22n4lPMpAfLe4yv8haC6Ejcp1XL&index=2   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on X/Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on X/Twitter

On this episode, we delve into the concept of shared decision making (SDM) in modern hemophilia care. With insights from experts Brendan Hayes and Dr. Cedric Hermans, the discussion explores the evolution of treatment options, patient education, and the collaborative decision-making process. The conversation also highlights the development and impact of the SDM tool by the World Federation of Hemophilia, emphasizing the importance of patient empowerment and ongoing education.   Guests: Brendan Hayes, MPH, CPH Cedric Hermans MD PhD FRCP   Senior Advisor: Donna DiMichele, MD   Hosted & Written by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes:   #MyHemophiliaTruth. Real stories. Powered by Sanofi. It's time to consider the whole experience when it comes to managing hemophilia and change the conversation. Explore the full story at www.myhemophiliatruth.com   Mark's Story Join Mark as he shares about his experience living with hemophilia, including the impact it had on building and maintaining relationships throughout grade school, college, dating, and raising his family. Mark also highlights how shared decision making with his healthcare providers, and strong family and caregiver support in those decisions, can make all the difference when it comes to managing the daily challenges of hemophilia.   Click here to watch his story: https://www.youtube.com/watch?v=tQ8oeM0zL24&list=PLmqBxf22n4lPMpAfLe4yv8haC6Ejcp1XL&index=8   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on X/Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on X/Twitter   

On this episode of the Global Hemophilia Report, we discuss the limitations of current hemophilia care metrics. They explore the importance of measuring the impact on quality of life, the role of nurse-patient relationships, and the challenges faced by underrepresented patient groups, such as those with moderate hemophilia and women.    Guests: Brittany Savage, NP Cathy Harrison, RN, Adv Dip, BMedSci, MSc   Senior Advisor: Donna DiMichele, MD   Hosted & Written by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes:   #MyHemophiliaTruth. Real stories. Powered by Sanofi. It's time to consider the whole experience when it comes to managing hemophilia and change the conversation. Explore the full story at www.myhemophiliatruth.com   The Bigger Picture in Hemophilia B:  Hemophilia A and hemophilia B are different bleeding disorders with unique pathologies and clinical features.1 Due to the distinct behavior of factor IX, multiple PK parameters should be considered when assessing bleed prevention. Learn how a broader view of PK may influence evaluation of treatment and management for patients with hemophilia B.2,3 Learn more at thebiggerpictureinhemb.com  1. Castaman G, Matino D. Haematologica. 2019;104(9):1702-1709. 2. Dolan G, Benson G, Duffy A, et al. Blood Rev. 2018;32(1):52-60.  3. Mann DM, Stafford KA, Poon M-C, Matino D, Stafford DW. Haemophilia. 2021;27(3):332-339. Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

On this episode of the Global Hemophilia Report, we explore the critical topic of health literacy and its direct impact on health equity within the bleeding disorders community. Featuring insights from experts, the discussion highlights the urgent need to address gaps in health literacy to ensure better medical outcomes. The episode also examines the challenges posed by recent rollbacks of diversity, equity, and inclusion policies and presents innovative solutions being utilized to bridge these gaps.   Guests: Kerry Funkhouser, EdD Brendan Hayes, MPH, CPH Paula James, MD Magdalena Lewandowska, MD, CACP Keri Norris, PhD, JM, MPH, MCHES   Senior Advisor: Donna DiMichele, MD   Hosted & Written by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes: Join Shellye as she shares her inspiring journey as a woman living with hemophilia. After years of unanswered questions, her diagnosis highlights the critical role of health literacy in bridging care gaps. Through advocacy, Shellye raises awareness and empowers underserved communities. Watch her story and see how health literacy can break barriers and transform lives. Click here to watch her story: Sanofi – See Hemophilia Through My Eyes: Women & Girls   Sanofi's Global Hemophilia Survey uncovers significant care gaps and emotional challenges faced by patients and caregivers. Learn how improving health literacy and fostering better patient-provider communication are essential to addressing these inequities. Explore the findings and see how Sanofi is driving health equity for the hemophilia community. Explore the survey findings here: Global Hemophilia Survey Page.   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

On this episode of the Global Hemophilia Report, we speak with guests who are each pioneering hemophilia advocacy in their respective regions. The discussion highlights the pivotal role of data and patient storytelling in driving systemic change and policy advancements. The episode emphasizes how global collaboration and technological innovations are paving the way for more equitable hemophilia care worldwide.    Guests: Harshal Kale, World Federation of Hemophilia Matthew Delaney, National Bleeding Disorders Foundation Megan Adediran, Hemophilia of Nigeria   Senior Advisor: Donna DiMichele, MD   Hosted & Written by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes: Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

On this episode of the Global Hemophilia Report, host Patrick James Lynch and Senior Advisor Dr. Donna DiMichele review significant discussions from the season on therapeutic advancements, patient advocacy, and critical hematological research. They highlight key topics explored, such as the evolving therapeutic landscape, the role of data and patient involvement in advancing hemophilia care, and the persisting challenges in treatment, particularly for hemophilia B patients. The episode also addresses the impact of artificial intelligence in research and the importance of securing funding for post-licensure studies.   Senior Advisor: Donna DiMichele, MD   Hosted & Written by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes: Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

The comprehensive care model for hemophiliathe is experiencing transformative evolution. Our panel of esteemed experts highlight how data and innovation are driving change across the globe, and share their insights on the challenges and advancements in advancing specialized care for individuals with hemophilia.   Contributors: Amy Dunn, M.D. Sanjay Ahuja, M.D.  Cedric Hermans, M.D.,  Ph.D., FRCP Jan Blatný, M.D., Ph.D Suely Rezende, M.D., Ph.D   Senior Advisor: Donna DiMichele, MD   Hosted & Written by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes: Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter

This episode of the Global Hemophilia Report podcast explores the significance of grassroots advocacy within hemophilia associations and chapters, emphasizing the critical role of data in advancing advocacy efforts. Experts from various U.S. organizations discuss the historical impact of the 1980s blood contamination crisis and the evolution of advocacy through its challenges and triumphs. The conversation highlights the importance of data collection, transparency, and community engagement in sustaining and enhancing treatment and support for those affected by hemophilia. The episode also addresses future advocacy strategies and the importance of continuous collaboration at the national and local levels. Contributors: Rigo Garcia, Hemophilia Federation of Southern California Sue Lerch, Great Lakes VE HTC Sue Martin, Bleeding Disorders Association of South Carolina Linda Mugford, Hemophilia Association of New York, Inc. Ray Stanhope, Lone Star Bleeding Disorders Foundation Senior Advisor: Donna DiMichele, MD   Hosted & Written by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes: Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

On this episode we talk with experts Dr. Amy Shapiro, Dr. Maria Elisa Mancuso, Dr. Steve Pipe, Dr. Johnny Mahlangu, and Dr. Lynn Malec to delve into the ongoing evolution of hemophilia therapies. The discussion highlights recent advancements in treatments such as emicizumab, extended half-life factor VIII therapies, and investigates medications in clinical trials like Concizumab, Marstacimab, and Fitusiran. The episode also reflects on the role of treatment individualization and the need for more inclusive research data.   Contributors: Johnny Mahlangu, MBBCh, MMed, FCPath Lynn Malec, MD, MSc Elisa Mancuso, MD Steven Pipe, MD Amy Shapiro, MD   Senior Advisor: Donna DiMichele, MD   Hosted & Written by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes: Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

On this episode of the Global Hemophilia Report, Senior Advisor Dr. Donna DiMichele leads a comprehensive discussion on FVIII inhibitors—a critical complication in Hemophilia A. The episode features experts such as Dr. Kathleen Pratt, Dr. David Lillicrap, Dr. Bhavya Doshi, Dr. Carol Miao, and Dr. Radek Kazmarek who explore the intricacies of immune responses, gene therapy, and novel therapies like emicizumab. They delve into factors like antigen-presenting cells, B cells, T cells, microbiome effects, and glycans that influence inhibitor development and tolerance. Despite advancements in treatment, the experts underscore the enduring importance of ongoing research in understanding and preventing FVIII inhibitors.   Contributors: Glaivy Batsuli, MD Bhavya Doshi, MD David Lillicrap, MD Carol Miao, PhD Kathleen Pratt, PhD Radek Kaczmarek, PhD   Senior Advisor: Donna DiMichele, MD   Hosted & Written by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes: Listen to the Global Hemophilia Report Episode 1: Inhibitors: Prevention, Eradication, and Lived Experiences   Conflicts of Interest: Dr. Kathleen Pratt is an inventor on patents related to factor VIII immunogenicity, and she has received research funding in the past from pharmaceutical companies that manufacture human FVIII. She has no other relevant conflicts to report.   Disclaimer: The contents of Dr. Pratt's presentation are the sole responsibility of the speaker and do not necessarily reflect the views, opinions or policies of Uniformed Services University of the Health Sciences (USUHS), The Henry M. Jackson Foundation for the Advancement of Military Medicine, Inc., the Department of Defense (DoD) or the Departments of the Army, Navy, or Air Force. Mention of trade names, commercial products, or organizations does not imply endorsement by the U.S. Government.    Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter  

In this episode of the Global Hemophilia Report, we delve into the crucial role of Lived Experience Experts (LEEs) in hemophilia research. Featuring a distinguished panel, the discussion covers the evolution, importance, and future of LEE involvement in research. The episode emphasizes the pivotal contributions of LEEs in making research more relevant and impactful, driven by personal and community experiences.   Contributors: Len Valentino MD Samantha Carlson MSW, LMSW Randy Curtis Ray Stanhope Kyle Davis MD   Senior Advisor: Donna DiMichele, MD   Special Episode Advisor: Maria Santaella PhD(c), MSN, RN-BC, CPHON   Hosted & Written by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

Our panel of subject matter and lived experience experts engage in a data-driven discussion about caregiver burden in hemophilia, historically, and how the rapidly evolving treatment landscapes impacts that burden. Contributors: Michelle Witkop, DNP, FNP-BC Kate Khair, PhD Beatriz Caceres, MD Kasha Lumsden, BSN, RN, RAC-CT Yasmin Pavri    Senior Advisor: Donna DiMichele, MD   Special Episode Advisor: Michelle Witkop, DNP, FNP-BC   Hosted & Written by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

On this episode of the Global Hemophilia Report, host Patrick James Lynch is on location in Madrid, Spain for the WFH 2024 World Congress. Patrick talks about the history of World Hemophilia Day and talks with some of the attendees at the congress. Contributors: Hazri Aris Johnny Mahlangu Rebecca, Hematology Resident   Senior Advisor: Donna DiMichele, MD   Hosted & Written by: Patrick James Lynch Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter

In this episode, delve into the transformative journey of patient-reported outcomes measures (PROMs) within hemophilia care, exploring their evolution from broad assessments of health-related quality of life to nuanced evaluations of physical ability, functionality, and psychological impact. Learn about pioneering instruments like the Hemo-TEM and the PROBE questionnaire, designed to capture the multifaceted burdens of hemophilia treatment and the patient experience. However, as the landscape of PROMs continues to evolve, discover lingering debates around their sufficiency compared to standardized measures like PROMIS, and the ongoing quest to ensure their responsiveness to change in evaluating novel therapeutics and treatment paradigms. Join us as we unravel the complexities of translating patient-reported clinical outcomes into patient-informed clinical research outcomes.   Contributors: Mark Skinner, JD Brian O'Mahony Bradley Rayner Dawn Rotellini Mosi Williams, LCSW, MSW, PsyD   Senior Advisor: Donna DiMichele, MD   Hosted & Written by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

The topic of discussion for this episode leans more toward the scientific side but has huge clinical relevance. For today, we will be asking our expert panel to lend their experience and expertise to the topic of FVIII and FIX, which we will be discussing from several related perspectives.    Contributors: Radek Kaczmarek, PhD, MSc; University of Indiana, USA Peter Lenting, PhD; INSERM, Paris, France Ben Samuelson -Jones, M.D., PhD; CHOP Research Institute, USA Courtney Thornburg, M.D., MS; Rady Children's Hospital, UCSD, USA   Senior Advisor: Donna DiMichele, MD   Hosted & Written by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter

Gene therapy for hemophilia A and B now exists as a licensed, prescribable treatment option for patients in certain countries; however, many questions and challenges remain. Senior advisor Dr. Donna DiMichele and patient-host Patrick James Lynch speak to global KOLs about the current state of hemophilia A and B gene therapies, both commercially and investigationally.   Contributors: Lindsey A. George, MD Margareth Ozelo, MD Steven Pipe, MD   Senior Advisor: Donna DiMichele, MD   Hosted & Written by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Notes and References:   Nathwani: Hematology Am Soc Hematol Educ Program . 2022 Dec 9;2022(1):569-578.   The current state of this exciting and rapidly evolving field, as well as the challenges that need to be overcome for the widespread adaptation of this new treatment paradigm, is the subject of this review.   Pipe: N Engl J Med. 2023 Feb 23;388(8):706-718   The annualized bleeding rate decreased from 4.19 (95% confidence interval [CI], 3.22 to 5.45) during the lead-in period to 1.51 (95% CI, 0.81 to 2.82) during months 7 through 18 after treatment, for a rate ratio of 0.36 (95% Wald CI, 0.20 to 0.64; P   Long-Term Effects of Hemophilia B Gene Therapy. Makris M.N Engl J Med. 2023 May 18;388(20):1918.    Long-Term Effects of Hemophilia B Gene Therapy. Reply. Pipe SW, Monahan PE.N Engl J Med. 2023 May 18;388(20):1918-1919   Mahlangu: N Engl J Med. 2023 Feb 23;388(8):694-705   Results: At week 104, a total of 132 participants, including 112 with data that were prospectively collected at baseline, remained in the study. The mean annualized treated bleeding rate decreased by 84.5% from baseline (P   Miesbach : Dtsch Arztebl Int. 2022 Dec 27;119(51-52):887-894.   Data from non-randomized phase 1 to phase 3 trials reveal an adequate expression of factors VIII and IX in patients with mostly severe hemophilia A or B. Even though they were no longer receiving prophylactic treatment, most patients experienced a considerable reduction, by 53% to 96%, in the number of bleedings compared to previous therapy. Persistently elevated factor levels have been described for up to six years in hemophilia A and up to eight years in hemophilia B. The most common side effect of gene therapy is an inflammatory response with elevated alanine aminotransferase levels (17% to 89%, depending on the study), which may be associated with a reduced clotting factor level and requires treatment with transient immunosuppression. Gene therapy for hemophilia holds out the prospect of freedom from hemorrhage without the need for regular treatment with drugs. The various steps that need to be carried out in gene therapy should be coordinated in a graded and partly overlapping integrated care model (a so-called hub-and-spoke model). Electronic platforms should be used for data acquisition and transmission.   Samuelson Jones & George: Annu Rev Med. 2023 Jan 27:74:231-247.   Importantly, despite repeated proof-of-concept success in current hemophilia gene therapy, stable, durable FVIII or FIX expression able to ameliorate bleeding in all patients is an unrealized hope. This defines the development goals of the next generation of gene-based therapies for hemophilia.   *Valentino et al: J Thromb Haemost. 2023 Sep;21(9):2354-2361   In 2022, the National Hemophilia Foundation submitted a citizen petition (docket number FDA-2022-P-1444-0001) to the FDA requesting that a risk evaluation and mitigation strategy (REMS) be required as a condition of approval for both valoctocogene roxaparvovec and etranacogene dezaparvovec. Other organizations including the World Federation of Hemophilia, Hemophilia Federation of America, and the European Haemophilia Consortium joined this effort submitting separate but supportive statements to the FDA. However, the FDA reasoned that the requirements for the REMS for etranacogene dezaparvovec were not met, and it has received marketing authorization from the FDA without the requirement for the REMS. While this may be the case for people living with hemophilia B, for whom the benefit to risk equation for gene therapy is generally favorable [[58]], it may not be the same for people with hemophilia A when considering gene therapy. Standard and extended half-life products for the prevention of bleeding for people living with either hemophilia A and B may provide very good outcomes but at a substantial burden of treatment and also fail to prevent all bleeding.   Another approach is a restrictive introduction, such as a conditional marketing authorization in which a medicine fulfills an unmet medical need but there is still a need for comprehensive safety, efficacy, and quality data generation after approval [[62]]. Such approvals are granted for 1 year but can be renewed annually. Compliance with specific obligations with defined timelines is a necessary prerequisite for continued authorization [   [63]]. This mechanism was utilized in the case of onasemnogene abeparvovec, an AAV9 gene therapy for spinal muscular atrophy [[64]].   Here, we recommend that the following steps be taken by the hemophilia community to ensure the safety and optimal outcomes for PwH who choose to receive a gene therapy product:   1.Training and education must be provided for physicians and HCPs on gene therapy and the management of PwH who receive a gene therapy product 2.Training and education on shared decision making must be provided for physicians and HCPs who will evaluate, administer, and follow PwH who are candidates to receive a gene therapy product [ 3.Facilities administering valoctocogene roxaparvovec and etranacogene dezaparvovec must receive adequate training and instruction on all aspects of gene therapy [ 4.Valoctocogene roxaparvovec and etranacogene dezaparvovec must only be administered at or in conjunction with a hemophilia treatment center with knowledge and expertise in evaluating, administering, and managing PwH who have received investigational gene therapy products [ 5.Individuals receiving valoctocogene roxaparvovec and etranacogene dezaparvovec must be enrolled in the global gene therapy registry initiated by the World Federation of Hemophilia in order to collect robust data, including adverse events of special interest [ 6.Educational support should be developed in a transparent and unbiased way to facilitate learning by PwH so that they may participate in shared decision making  7.Formal collaboration between the relevant national hemophilia patient organizations and the centers administering gene therapy on the provision of education and information should be ensured so that the PwH is ready to fully participate in a shared and informed decision-making process. 8.Work to ensure that postmarket studies, registries, and future registrational studies take into consideration principles of health equity in their design.   *Baas: J Thromb Haemost. 2023 Mar;21(3):413-420. (Ethics)   Based on our analysis of the literature, we identified 3 ethical themes. The theme Living up to expectations describes the existing hopes for gene therapy and the unlikelihood of the currently approved product becoming a permanent cure. In the theme Psychosocial impacts, we discuss the fear that gene therapy will impact the identity of people with hemophilia and their need for psychosocial support. The theme Costs and access discusses the expected cost-effectiveness of gene therapy and its implications on accessibility worldwide. We conclude that it may be necessary to change the narratives surrounding gene therapy, from describing it as a cure to describing it as one of the many treatments that temporarily relieve symptoms and that there is a need to reevaluate the desirability of gene therapy for hemophilia, given the availability of other treatments. *Kumar: J Thromb Haemost. 2024 Jan;22(1):23-34    Of note, multiple preclinical studies using platelet-specific lentiviral gene delivery to hematopoietic stem cells in hemophilia have demonstrated promising results with therapeutic levels of neo-protein that rescue the hemorrhagic bleeding phenotype and induce antigen-specific immune tolerance. Further studies using ovalbumin as a surrogate protein for platelet gene therapy have shown robust antigen-specific immune tolerance induced via peripheral clonal deletions of antigen-specific CD4- and CD8-T effector cells and induction of antigen-specific regulatory T (Treg) cells. This review discusses platelet-targeted gene therapy, focusing on immune tolerance induction.   Shah: Curr Med Res Opin. 2023 Feb;39(2):227-237   Bayesian and Frequentist linear mixed models predicted no more than 6/55 (10.91%) observed participants would have FIX activity levels 80% would be free from prophylactic FIX replacement products 25.5 years post-infusion. Both models predicted FIX activity levels were not significantly influenced by pre-treatment AAV5 NAb status.   Miesbach: J Thromb Haemost. 2023 Feb;21(2):200-203   It is therefore important that hemophilia treaters and hepatologists collaborate at all stages of gene therapy to assess potential safety issues and ensure the long-term success of gene therapy. Special attention should be given to patients with not well-defined conditions, e.g. patients with some degree of liver fibrosis or fatty liver disease, patients with a history of hepatitis C and hepatitis B infection, patients with HIV infection, and patients taking medications that may affect liver function.   Rasul: J Patient Exp. 2023; 10: 23743735231193573.   Hemophilia is a bleeding disorder caused by a single absent/defective gene and characterized by a lack of functional clotting factors. People with hemophilia may experience joint damage, pain, and psychological impairments, all of which could contribute to reduced health-related quality of life (HRQoL). The current standard of care is clotting factor replacement, which is associated with regular infusions; therefore, alternative treatments such as gene therapy (GT) are in development. GT involves the delivery of a functional copy of the clotting factor 8/9 gene by a single infusion into the patient's cells, enabling them to produce their own clotting factor VIII/IX. The impact of treatment on patients' HRQoL can be assessed using hemophilia-specific patient-reported outcome (PRO) measures. Since these measures were designed before the advent of GT, there is a need for updated individualized PRO measures. Patient groups and regulatory authorities emphasize the need for increased patient engagement when considering clinical trial design. Here, we provide patients' perspective on undergoing GT and discuss how to capture the patient voice when measuring the therapy's transformative impact   Valentino: J Thromb Haemost. 2023 Nov;21(11):3033-3044   The therapeutic landscape for people living with hemophilia A (PwHA) has changed dramatically in recent years, but many clinical challenges remain, including the development of inhibitory antibodies directed against factor VIII (FVIII) that occur in approximately 30% of people with severe hemophilia A. Emicizumab, an FVIII mimetic bispecific monoclonal antibody, provides safe and effective bleeding prophylaxis for many PwHA, but clinicians still explore therapeutic strategies that result in immunologic tolerance to FVIII to enable effective treatment with FVIII for problematic bleeding events. This immune tolerance induction (ITI) to FVIII is typically accomplished through repeated long-term exposure to FVIII using a variety of protocols. Meanwhile, gene therapy has recently emerged as a novel ITI option that provides an intrinsic, consistent source of FVIII. As gene therapy and other therapies now expand therapeutic options for PwHA, we review the persistent unmet medical needs with respect to FVIII inhibitors and effective ITI in PwHA, the immunology of FVIII tolerization, the latest research on tolerization strategies, and the role of liver-directed gene therapy to mediate FVIII ITI.   Limjoco: Patient Prefer Adherence. 2023 Apr 19:17:1093-1105   These data highlight the utility of a SDM tool for hemophilia gene therapy and key information needs. Data including comparison to other treatments should be provided along with patient testimonials in a transparent format. Patients will engage the Hemophilia Treatment Center, family, and community members in the decision-making process.   Miesbach: Hamostaseologie. 2023 Jun;43(3):196-207   Gene therapy has recently become a realistic treatment perspective for patients with hemophilia. Reviewing the literature and our personal experience from clinical trials, we discuss key aspects of hemophilia A and B gene therapy with vectors derived from adeno-associated virus, including predictable results, risks, adverse events, and patient-reported outcomes. Patient selection, informed consent, administration, and monitoring of gene therapy as well as data collection are explained. We also discuss the need for interdisciplinary cooperation with hepatology and other specialties. We emphasize structural and organizational requirements for treatment centers according to the hub-and-spoke model and recommend the use of electronic diaries to ensure safe and timely collection and exchange of data. Electronic diaries will play a key role as a primary source of data for pharmacovigilance, post-marketing clinical studies, national and international registries, as well as health technology and benefit assessment. Reimbursement aspects and the future of gene therapy in adolescents and children are also considered. In a rapidly evolving scientific environment, these recommendations aim to support treatment providers and payers to prepare for the implementation of gene therapy following marketing authorization   Butterfield: Cell Immunol. 2023 Sep-Oct:391-392:104742   Oral immunotherapies are being developed for various autoimmune diseases and allergies to suppress immune responses in an antigen-specific manner. Previous studies have shown that anti-drug antibody (inhibitor) formation in protein replacement therapy for the inherited bleeding disorder hemophilia can be prevented by repeated oral delivery of coagulation factor antigens bioencapsulated in transplastomic lettuce cells. Here, we find that this approach substantially reduces antibody development against factor VIII in hemophilia A mice treated with adeno-associated viral gene transfer. We propose that the concept of oral tolerance can be applied to prevent immune responses against therapeutic transgene products expressed in gene therapy   Nguyen: J Thromb Haemost. 2023 Aug;21(8):2101-2113.   The novel hFVIII-Δ3-SP/DE variant of the furin and a3 cleavage sites significantly improved secretion compared with hFVIII-BDD. This key feature of the Δ3-SP/DE variant provides a unique strategy that can be combined with other approaches to further improve factor VIII expression to achieve superior efficacy in AAV-based gene therapy for hemophilia A.   La Mura: Blood Adv. 2023 Oct 10;7(19):5817-5824   Overall, risk factors of chronic liver damage are frequent after HCV clearance, but changes in LSM and NITs after clearance may be inaccurate to rule out advanced fibrosis/cirrhosis. A specific diagnostic workup is warranted to evaluate liver health in PWH in the era of gene therapy.   Kashiwakura: Mol Ther Methods Clin Dev. 2023 Aug 22:30:502-514   Gene therapy using adeno-associated virus (AAV)-based vectors has become a realistic therapeutic option for hemophilia. We examined the potential of a novel engineered liver-tropic AAV3B-based vector, AAV.GT5, for hemophilia B gene therapy. In vitro transduction with AAV.GT5 in human hepatocytes was more than 100 times higher than with AAV-Spark100, another bioengineered vector used in a clinical trial. However, liver transduction following intravenous injection of these vectors was similar in mice with a humanized liver and in macaques. This discrepancy was due to the low recovery and short half-life of AAV.GT5 in blood, depending on the positive charge of the heparin-binding site in the capsid. Bypassing systemic clearance with the intra-hepatic vascular administration of AAV.GT5, but not AAV-Spark100, enhanced liver transduction in pigs and macaques. AAV.GT5 did not develop neutralizing antibodies (NAbs) in two of four animals, while AAV-Spark100 induced serotype-specific NAbs in all macaques tested (4 of 4). The NAbs produced after AAV-Spark100 administration were relatively serotype specific, and challenge with AAV.GT5 through the hepatic artery successfully boosted liver transduction in one animal previously administered AAV-Spark100. In summary, AAV.GT5 showed different vector kinetics and NAb induction compared with AAV-Spark100, and intra-hepatic vascular administration may minimize the vector dose required and vector dissemination.   Hermans: Ther Adv Hematol. 2023 Jan 12:14:20406207221145627.   First, awareness, communication, and education about the therapeutic potential and modalities of gene therapy must be further strengthened. To this end, objective, unbiased, transparent, and regularly updated information must be shared, in an appropriate way and understandable language with the support of patients' organizations. Second, healthcare providers should adopt a patient-centred approach, as the 'one size fits all' approach is inappropriate when considering gene therapy. Instead, a holistic patient view taking into account their physical and mental dimensions, along with unexpressed expectations and preferences, is mandatory. Third, the consent procedure must be improved, ensuring that patients' interests are maximally protected. Finally, gene therapy is likely to be first delivered in a few centres, with the highest expertise and experience in this domain. Thus, patients should be managed based on a hub-and-spoke model, taking into account that the key to gene therapy's success lies in an optimal communication and collaboration both within and between haemophilia centres sharing their experiences in the frame of international registries. This review describes recent progress and explains outstanding hurdles that must be tackled to ease the implementation of this paradigm-changing new therapy.   Castaman: Haemophilia. 2023 Mar;29(2):435-444   Use of the hub-and-spoke organisational model and multidisciplinary teams are expected to optimize patient selection for gene therapy, as well as the management of dosing and patient follow-up, patient engagement, laboratory surveillance, and patient expectations regarding outcomes. This approach should allow the benefits of AAV-based gene therapy for haemophilia A to be maximized.   Pipe: Haemophilia. 2023 Nov;29(6):1430-1441   The patient journey includes: information gathering; decision making; comprehensive patient assessment; preparation for the infusion itself; short- and long-term monitoring; lifestyle modifications; and the possible need for immunosuppressive treatment. Informed decision-making may require patient education with extensive discussions and an understanding that not all people with haemophilia will choose or be eligible for gene therapy, although eligibility criteria continue to evolve. The institutional journey includes: consideration of biosafety procedures; planning for product procurement, handling, storage, and administration; development of detailed protocols and guidance documents; contingency planning for immunosuppressive and haemostatic management; consideration of clinical capabilities and staff training needs; coordination of efforts by the full multidisciplinary team; and collaboration between referring, dosing, and follow-up treatment centres. Documented protocols and guidance documents are pivotal for this complex therapy to ensure safe handling, optimal delivery, and post-infusion management and follow-up.   Fletcher: Haemophilia. 2023 May;29(3):776-783   PwH have many expectations about the difference gene therapy can make to their lives. Studies show that these expectations may not be fully realised. For those who have either withdrawn or been withdrawn from gene therapy, any expectations they had may now be unachievable. The nature of these expectations and the loss expressed by the participants indicate that support needs to be provided to help them and their families manage it.   Chen: Mol Ther Nucleic Acids. 2023 Oct 5:34:102043   Development of factor VIII (FVIII) inhibitors is a serious complication in the treatment of hemophilia A (HemA) patients. In clinical trials, anti-CD3 antibody therapy effectively modulates the immune response of allograft rejection or autoimmune diseases without eliciting major adverse effects. In this study, we delivered mRNA-encapsulated lipid nanoparticles (LNPs) encoding therapeutic anti-CD3 antibody (αCD3 LNPs) to overcome the anti-FVIII immune responses in HemA mice. It was found that αCD3 LNPs encoding the single-chain antibodies (Fc-scFv) can efficiently deplete CD3+ and CD4+ effector T cells, whereas αCD3 LNPs encoding double-chain antibodies cannot. Concomitantly, mice treated with αCD3 (Fc-scFv) LNPs showed an increase in the CD4+CD25+Foxp3+ regulatory T cell percentages, which modulated the anti-FVIII immune responses. All T cells returned to normal levels within 2 months. HemA mice treated with αCD3 LNPs prior to hydrodynamic injection of liver-specific FVIII plasmids achieved persistent FVIII gene expression without formation of FVIII inhibitors. Furthermore, transgene expression was increased and persistent following secondary plasmid challenge, indicating induction of long-term tolerance to FVIII. Moreover, the treated mice maintained their immune competence against other antigens. In conclusion, our study established a potential new strategy to induce long-term antigen-specific tolerance using an αCD3 LNP formulation.   Ramamurthy: Front Immunol. 2022 Dec 15:13:954984   Although we did not achieve our primary objective, our results validate the utility of both PLCs and hLSECs as cell-based delivery vehicles for a fVIII transgene, and they highlight the hurdles that remain to be overcome before primary human cells can be gene-edited with sufficient efficiency for use in cell-based gene therapy to treat HA.     Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter

Join us as we shed light on the often-overlooked intersection of Post Traumatic Stress Disorder (PTSD) and Hemophilia, discussing innovative treatments, personal stories, and the journey towards understanding and managing these complex disorders.   Contributors: Stephen W. Porges, PhD Debbie de la Riva Amanda Stahl, MSW, LICSW Justin Levesque   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch Written by: Simantini Karve, PhD   Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook  

The US National Research Blueprint (NRB). Thriving in the Face of an Inheritable Bleeding Disorder Begins with Community-Inspired Research.    Contributors: Len Valentino, MD Keri Norris, MD Maria Santaella, MD Michael Recht, MD Sammie Valadez Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch Written by: Simantini Karve, PhD   Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

In this episode, we address the critical intersections of LGBTQ+ health and hemophilia care. Delving into the historical links with the HIV crisis, we scrutinize the disparities in care access, the mental health implications, and the unique challenges faced during transition to independent care. We discuss the intricacies of prophylactic drug regimens, vaccines, and the hemophilic bleeding risk related to LGBTQ+ sexual health. With insights into gender-affirming care, reproductive counseling, and knowledge gaps, we provide a comprehensive perspective tailored for physicians and clinicians aiming for holistic patient care.   Contributors: Nathan Connell, MD Nancy Sokkary, MD Robert Sidonio, MD Greig Blamey, Physiotherapist Dakota J. Rosenfelt, PharmD, RPh, MBA    Senior Advisor: Donna DiMichele, MD Hosted by: Patrick James Lynch Written by: Simantini Karve, PhD  

On this episode, we will address defining sexual health and its importance to hemophilia care, best practices and standards for addressing sexual health, implementing a practice of addressing sexual health into a comprehensive care model, envisioning progress in care models for addressing sexual health in both global and personalized ways and more.    Contributors: Greig Blamey, Physiotherapist Maureen Baldwin, MD William McKeown, MD   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch Written by: Simantini Karve, PhD   Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

Persons with mild hemophilia have been shown to experience more bleeding - related morbidity than previously recognized while achieving lifespans that parallel the general population. Consequently, persons living with mild hemophilia are more likely to experience the comorbidities associated with aging, often complicating their management in their later years.    Contributors: Prof Karin (C. J.) Fijnvandraat MD PhD Maria Elisa Mancuso, MD PhD Michael Recht, MD PhD MBA Vaughn Ripley   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch Written by: Simantini Karve, PhD  

Telehealth options are assumed to provide added benefit to providers and patients alike, but how much do we really know and understand about telemedicine? Listen in to the latest episode of Global Hemophilia Report as our contributors share how telemedicine has potentially benefited and detracted from hemophilia care across different populations as well as what research must still be done on the rise of telehealth in hemophilia care   Contributors: Sativa Rangarajan, Associate Professor of Clinical Haematology  Michelle Witkop, DNP, FNP-BC  Dr. Roshni Kulkarni, Professor & Former Director Michigan State University Centers For Bleeding And Clotting Disorders Annette von Drygalski, MD, PharmD   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch Written by: Simantini Karve, PhD   Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

The social determinants of health (SDH) are the non-medical factors that influence health outcomes. They are the conditions in which people are born, grow, work, live, and age, and the wider set of forces and systems shaping the conditions of daily life. These forces and systems include economic policies and systems, development agendas, social norms, social policies and political systems.   Contributors: Keri Norris, PhD, JM, MPH, MCHES Karina Lopez, MPH Adrian Palau-Tejeda Marissa Melton, MPH Chris Bombardier Mosi Williams   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch   Written by: Simantini Karve, PhD     Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

Historically, camps for kids with hemophilia have played a specific and valuable role, enabling a young person with hemophilia to experience all the benefits of a traditional, summer camp experience. On this episode, we dig into the camp experience with some of the leading voices within the space.  Contributors: Tracey Gaslin PhD, CPNP, FNP-BC, CRNI, RN-BC Michael Wang, MD Pat Torrey Sarah Shinkman Chris Bombardier   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch   Written by: Simantini Karve, PhD   Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

Historically, people with hemophilia and especially children, have lived with considerable limitations when it comes to engagement in sports and athletics, thereby missing out on the numerous biopsychosocial and general health. However, both advancements in hemophilia care/treatment and advancements in understanding the benefits of engaging in sports have significantly changed the risk/benefit equation– but exactly, how?   Contributors: Marilyn Manco Johnson, M.D. Olav Versloot, PhD Paul Mclaughlin Chris Bombardier   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch   Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report   Watch the film Bombardier Blood Listen to The Final Summit podcast   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

On this episode we're in conversation with research leaders to learn more about the current state of hemophilia research, the barriers and opportunities we're facing today, and what the future of hemophilia research might look like.     Contributors: Flora Peyvandi, MD, PhD Keith Hoots, MD Len Valentino, MD Michelle Witkop, DNP, FNP-BC Glenn Pierce, MD, PhD   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch   Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

There has been a remarkable increase in the life expectancy of persons living with hemophilia (PLWH) over the past century, largely due to access to safe factor replacement products.  However, the course of progress has been uneven, and rocky, for many. This is part two of our series on Aging with Hemophilia. Contributors: William McKeown, MD Randall Curtis Gerry Dolan, MD, MBChB, FRCP, FRCPath Barbara Konkle, MD Sara L. Schwartz, PhD  Tam Perry, PhD   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch   Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

There has been a remarkable increase in the life expectancy of persons living with hemophilia (PLWH) over the past century, largely due to access to safe factor replacement products.  However, the course of progress has been uneven, and rocky, for many.   Contributors: William McKeown, MD Randall Curtis Gerry Dolan, MD, MBChB, FRCP, FRCPath Barbara Konkle, MD   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch   Listen to Blood of the Paladin Podcast   Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report Listen to BloodStream's Journey to Joint Health episode.   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter 

Renowned hematologist, researcher, and senior advisor to the Global Hemophilia Report Dr. Donna DiMichele shares some of her Season 1 takeaways and thoughts about the future of hemophilia research. Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch   Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report Listen to BloodStream's Journey to Joint Health episode.   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter 

On this episode, we continue our discussion of chronic pain in hemophilia. We'll discuss exercise, medication and investigations.   Contributors: Nathalie Anne Roussel, PhD, MSc, PT Paul Mc Laughlin,  BSc, MSc, MMACP   Senior Advisor: Donna DiMichele, MD   Episode Advisors (also contributors): Michelle Witkop, DNP, FNP-BC Tyler Buckner, MD   Hosted by: Patrick James Lynch   Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report Listen to BloodStream's Journey to Joint Health episode.   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter 

According to the Centers for Disease Control, pain is one of the most common reasons people seek medical care. However, for persons affected by hemophilia A and B, the experience of pain has provided the subtext for many issues related to health and wellbeing across the lifespan, and irrespective of gender, age, and, potentially, hemophilia severity levels. Although less-well characterized in children, pain is already embedded in the life experience of a PWH, and a substantial contributor to poor health-related quality of life in adolescence and young adulthood, further increasing in negative impact throughout adult life. In this episode, we explore pain's prevalence, mechanisms, and more.    Contributors: Nathalie Anne Roussel, PhD, MSc, PT Paul Mc Laughlin,  BSc, MSc, MMACP Michelle Rice    Senior Advisor: Donna DiMichele, MD   Episode Advisors (also contributors): Michelle Witkop, DNP, FNP-BC Tyler Buckner, MD   Hosted by: Patrick James Lynch   Show Notes: Presenting Sponsor: Sanofi Subscribe to the Global Hemophilia Report   Listen to BloodStream's Pain Podcast   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter  

In Part 2 of our Hemophilia B episode, we spotlight research priorities into women with hemophilia B, novel therapies to treat people with hemophilia B, and we conclude the story of Alexei Nikolaevich, the last Tsesarevich of Russia.   Contributors: Bethany Samuelson Bannow, MD Brian O'Mahony Kathaleen M. Schnur, MSW, LCSW   Senior Advisor: Donna DiMichele, MD   Episode Advisors (also contributors): Dr. Amy Shapiro Professor Jan Astermark   Hosted by: Laurence Woollard   Links to learn more:   Brown A. "The Royal Disease and The Royal Collapse: Political Effects of Hemophilia in the Royal Houses of Europe." Honor Scholar Theses. 2017;63. [Online]. Available at: https://scholarship.depauw.edu/cgi/viewcontent.cgi?article=1063&context=studentresearch [Accessed 10 October 2022]   Gualtierotti R, et al. Updates on novel non-replacement drugs for hemophilia. Pharmaceuticals 2022;15(10):1183. Doi: 10.3390/ph15101183   Hart DP, et al. International consensus recommendations on the management of people living with haemophilia B. Ther Adv Hematol 2022;13:1-22. Doi: 20406207221085202   Hoffman TA. "Bad Blood: Hemophilia and Its Detriment to the Russian Imperial Family." Young Historians Conference. 2022;8. [Online]. Available at: https://pdxscholar.library.pdx.edu/cgi/viewcontent.cgi?article=1244&context=younghistorians [Accessed 10 October 2022]   Lannoy N, Hermans C. The 'royal disease' – haemophilia A or B? A haematological mystery is finally solved. Haemophilia 2010;16:843-47. Doi: 10.1111/j.1365-2516.2010.02327.x   Nathwani A, et al. Liver gene therapy. Hum Gene Thur 2022;33:17-18. Doi: 10.1089/hum.2022.169   O'Donovan M, et al. Recombinant factor IX-Fc fusion protein in severe hemophilia B: Patient-reported outcomes and health-related quality of life. Res Pract Thromb Haemost 2021;5(7):e12602. Doi: 10.1002/rth2.12602   Peeling S. Russo-Japanese War. International Encyclopaedia of the First World War. 1914-1918. [Online]. Available at: https://encyclopedia.1914-1918-online.net/article/russo-japanese_war [Accessed 10 October 2022]   Potts WTW. Royal haemophilia. J Biol Educ 1996;30(3):207-17. DOI: 10.1080/00219266.1996.9655504   Price KD. "Diary of Nicholas II, 1917-1918, an annotated translation." Graduate Student Theses, Dissertations, & Professional Papers. 1966;2065. [Online]. Available at: https://scholarworks.umt.edu/cgi/viewcontent.cgi?article=3084&context=etd [Accessed 10 October 2022]   Radcliffe J. "Rasputin and the Fragmentation of Imperial Russia." Young Historians Conference. 2017;14. [Online]. Available at: https://pdxscholar.library.pdx.edu/younghistorians/2017/oralpres/14/ [Accessed 10 October 2022]   Show Notes: Presenting Sponsor: Sanofi Subscribe to the Global Hemophilia Report   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter 

Hemophilia B is a rare inherited X-linked disorder characterized by a congenital anomaly in the factor IX gene leading to a variable deficiency in clotting factor IX. In this episode, we explore the known differences between Hemophilia B and Hemophilia A and discuss priority areas for further research into Hemophilia B. The episode also guides listeners on an entertaining journey through 19th and 20th century European monarchy and the unlikely role that hemophilia played in forever shaping Europe's governance!    Contributors: Bethany Samuelson Bannow, MD Brian O'Mahony Kathaleen M. Schnur, MSW, LCSW   Senior Advisor: Donna DiMichele, MD   Episode Advisors (also contributors): Dr. Amy Shapiro Professor Jan Astermark   Hosted by: Laurence Woollard   Links to learn more:   Brown A. "The Royal Disease and The Royal Collapse: Political Effects of Hemophilia in the Royal Houses of Europe." Honor Scholar Theses. 2017;63. [Online]. Available at: https://scholarship.depauw.edu/cgi/viewcontent.cgi?article=1063&context=studentresearch [Accessed 10 October 2022]   Hoffman TA. "Bad Blood: Hemophilia and Its Detriment to the Russian Imperial Family." Young Historians Conference. 2022;8. [Online]. Available at: https://pdxscholar.library.pdx.edu/cgi/viewcontent.cgi?article=1244&context=younghistorians [Accessed 10 October 2022]   Lannoy N, Hermans C. The 'royal disease' – haemophilia A or B? A haematological mystery is finally solved. Haemophilia 2010;16:843-47. Doi: 10.1111/j.1365-2516.2010.02327.x   Potts WTW. Royal haemophilia. J Biol Educ 1996;30(3):207-17. DOI: 10.1080/00219266.1996.9655504   Price KD. "Diary of Nicholas II, 1917-1918, an annotated translation." Graduate Student Theses, Dissertations, & Professional Papers. 1966;2065. [Online]. Available at: https://scholarworks.umt.edu/cgi/viewcontent.cgi?article=3084&context=etd [Accessed 10 October 2022]   Radcliffe J. "Rasputin and the Fragmentation of Imperial Russia." Young Historians Conference. 2017;14. [Online]. Available at: https://pdxscholar.library.pdx.edu/younghistorians/2017/oralpres/14/ [Accessed 10 October 2022]   Funnell APW, Crossley M. Hemophilia B Leyden and once mysterious cis-regulatory mutations. Trends Genet 2014;30(1):18-23. Doi: 10.1016/j.tig.2013.09.007   Simioni P, et al. X-linked thrombophilia with a mutant factor IX (factor IX Padua). N Engl J Med 2009;361(17):1671-5. Doi: 10.1056/NEJMoa0904377   Nogami K, et al. Clinical conditions and risk factors for inhibitor-development in patients with haemophilia: A decade-long prospective cohort study in Japan, J-HIS2 (Japan Hemophilia Inhibitor Study 2). Haemophilia 2022;28(5):745-59. Doi: 10.1111/hae.14602   Thorland EC, et al. Anaphylactic response to FIX replacement therapy in haemophilia B patients: complete gene deletions confer the highest risk. Haemophilia 1999;5(2):101-5.    Chitlur M, et al. Inhibitors in factor IX deficiency a report of the ISTH-SSC international FIX inhibitor registry (1997-2006). Haemophilia 2009;15(5):1027-31. Doi: 10.1111/j.1365-2516.2009.02039.x   DiMichele D. The North American Immune Tolerance Registry: contributions to the thirty-year experience with immune tolerance therapy. Haemophilia 2009;15(1):320-8. Doi: 10.1111/j.1365-2516.2008.01880.x   Astermark J, et al. The B-Natural Study – The outcome of immune tolerance induction therapy in patients with severe haemophilia B. Haemophilia 2021;27(5):802-13. Doi: 10.1111/hae.14357   Iorio A, et al. Establishing the prevalence and prevalence at birth of hemophilia in males: a meta-analytic approach using national registries. Ann Intern Med 2009;171(8):540-46. Doi: 10.7326/M19-1208   Soucie JM, et al. Occurance rates of haemophilia among males in the United States based on surveillance conducted in specialized haemophilia treatment centres. Haemophilia 2020;26(3):487-93. Doi: 10.1111/hae.13998   Berntorp E, et al. Quality of life in a large multinational haemophilia B cohort (The B-Natural Study – Unmet needs remain. Haemophilia 2022;28(3):453-61. Doi: 10.1111/hae.14525   Kihlberg K, et al. Treatment outcomes in persons with severe haemophilia B in the Nordic region: The B-NORD study. Haemophilia 2021;27(3):366-74. Doi: 10.1111/hae.14299   Feng D, et al. Evidence of clinically significant extravascular stores of factor IX. Thromb Haemost 2013;11(12):2176-2178. Doi: 10.1111/jth.12421   DiMichele DM, et al. Severe and moderate haemophilia A and B in US females. Haemophilia 2014;20(2):e136-43. Doi: 10.1111/hae.12364   Buckner TW, et al. Management of US men, women, and children with hemophilia and methods and demographics of the Bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B-HERO-S) study. Eur J Haematol 2017;98:5-17. Doi: 10.1111/ejh.12854     Show Notes: Presenting Sponsor: Sanofi Subscribe to the Global Hemophilia Report   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter 

On this episode, Executive Producer of the Global Hemophilia Report, Patrick James Lynch, is joined in person by Senior Advisor, Dr. Donna DiMichele, and Writer/Host, Laurence Woollard, to reflect on Season 1 so far as well as debate existing and emerging research priorities from each episode. Contributors: Patrick James Lynch Laurence Woollard Donna DiMichele, MD   Senior Advisor: Donna DiMichele, MD   Links to Learn More: Episode 1 - Inhibitors: Prevention, Eradication & Lived Experiences Episode 2 - Novel Therapies: Entering Uncharted Territory Episode 3 - Prophylaxis: Protection, Achievements & Shortcomings Episode 4 - Bone & Joint Health: Monitoring & Detection Episode 5 - Mental Health & Hemophilia in Adolescence & Young Adulthood Episode 6 - Women with Hemophilia: Research Priorities   Show Notes: Recorded in person at the National Hemophilia Foundation's Bleeding Disorders Conference on August 26th, 2022 in Houston, Texas, US.  Presenting Sponsor: Sanofi Subscribe to the Global Hemophilia Report   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter 

Women, girls, and those with the potential to menstruate (WGPPM) who live with hemophilia, have more difficulty getting a diagnosis and accessing adequate care than their biological male counterparts. But why? What research is underway to support the needs of these individuals? This episode takes a wide-lens approach to exploring the socio-cultural barriers and gender-related challenges facing WGPPM in the global hemophilia community.   Contributors: Dawn Rotellini Andra James, MD, MPH Connie Miller, PhD Tyler Buckner, MD Maureen Baldwin, MD, MPH Kristin Paulyson Nunez, M.S., C.G.C Yannick Cole   Senior Advisor: Donna DiMichele, MD   Episode Advisors: Angela Weyand, MD Robert Sidonio, MD   Hosted by: Laurence Woollard   Links to learn more: Bercovitz RS. "A new hemophilia carrier nomenclature to define hemophilia in women and girls: Communication from the SSC of the ISTH": Comment. J  Thromb Haemost 2022;20(7):1744-45. DOI: 10.1111/jth.15727   Chaudhury A, et al. Women and girls with haemophilia and bleeding tendencies: Outcomes related to menstruation, pregnancy, surgery and other bleeding episodes from a retrospective chart review. Haemophilia 2021;27(2):293-304. DOI: 10.1111/hae.14232   d'Oiron R, et al. Women and girls with haemophilia: Lessons learned. Haemophilia 2021;27(S3):75-81. DOI: 10.1111/hae.14094   DiMichele DM, et al. Severe and moderate haemophilia A and B in US females. Haemophilia 2014;20(2):e136-43. DOI: 10.1111/hae.12364   Fox L. Women and girls with hemophilia: Gender-based differences in comprehensive care. NHF Bleeding Disorders Conference 2019. [Online]. Available at: https://www.hemophilia.org/research/research-projects/women-and-girls-with-hemophilia-gender-based-differences-in-comprehensive-care   Kirtava A, et al. Trends in clinical management of women with von Willebrand disease: A survey of 75 women enrolled in haemophilia treatment centres in the United States. Haemophilia 2004;10:158-161. DOI: 10.1046/j.1351-8216.2003.00832.x    Malouin RA, et al. Practice Characteristics of Genetic Counselors Serving the North American Bleeding Disorders Community. [Poster]. World Federation of Hemophilia 2016 World Congress , May 2016.    Miller CH, et al. Women and girls with haemophilia receiving care at specialized haemophilia treatment centres in the United States. Haemophilia 2021;27(6):1037-44. DOI: 10.1111/hae.14403   Rajpurkar M, et al. Current challenges for men and women with mild-to-moderate haemophilia. Haemophilia 2021;27(S1):5-7. DOI: 10.1111/hae.14193   van Galen K, et al. European principles of care for women and girls with inherited bleeding disorders. Haemophilia 2021;27(5):837-47. DOI: 10.1111/hae.14379   van Galen KPM, et al. A new hemophilia carrier nomenclature to define hemophilia in women and girls: Communication from the SSC of the ISTH. J Thromb Haemost 2021;19(8):1883-87. DOI: 10.1111/jth.15397   Weyand AC, James PD. Sexism in the management of bleeding disorders. Res Pract Thromb Haemost 2020;5(1):51-4. DOI: 10.1002/rth2.12468   Show Notes: Presenting Sponsor: Sanofi Subscribe to the Global Hemophilia Report   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter  Listen to the FLOW podcast

In March 2022, a Washington Post article highlighted children and adolescents in the US experiencing a mental health crisis, but how does a hemophilia diagnosis further complicate a young person's mental wellbeing? What data exists to support interventions? This episode explores the state of research and understanding of hemophilia's impact on a young person's mental health.   Episode Advisors: Dr. Michelle Witkop Samantha Carlson, LMSW   Contributors: Randall G. Curtis, MBA Dr. Gráinne O'Brien Dr. Duc "Bobby" Tran Amanda Stahl, LCSW   Senior Advisor: Dr. Donna DiMichele   Show Notes: Presenting Sponsor: Sanofi Subscribe to the Global Hemophilia Report   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter 

Even with the introduction of prophylaxis, the goal of achieving perfect bone and joint health for patients with hemophilia through normalizing homeostasis remains elusive. In this episode, we cover the current monitoring and detection strategies and explore the investigational work that's aimed at enhancing existing tools, validating suspected tools, and discovering entirely new ones.     Episode Advisors: Dr. Marilyn Manco-Johnson Dr. Kathelijn Fischer   Contributors: Dr. Annette Von Drygalski Dr. Tyler W. Buckner Dr. Amy Dunn Dr. Suchitra S Acharya Dr. Andrea Doria Dr. Beth Warren Tom Russomano   Senior Advisor: Dr. Donna DiMichele   Links to learn more: Young adult outcomes of childhood prophylaxis for severe hemophilia A: results of the Joint Outcome Continuation Study Measurement of joint health in persons with haemophilia: A systematic review of the measurement properties of haemophilia-specific instruments Vascular remodeling underlies rebleeding in hemophilic arthropathy Quantitative measurements of haemophilic joint tissues by point‐of‐care musculoskeletal ultrasound: Associations with clinical and functional joint outcome parameters How Do PROMIS Scores Correspond to Common Physical Abilities? Evaluating international Haemophilia Joint Health Score (HJHS) results combined with expert opinion: Options for a shorter HJHS   Show Notes: Presenting Sponsor: Sanofi Subscribe to the Global Hemophilia Report   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter

Prophylaxis in hemophilia has led to extraordinary progress, however, recent studies suggest that although the overall musculoskeletal health of persons with hemophilia has significantly improved, the goal of attaining a level of musculoskeletal health comparable to that of the general population without hemophilia has still not been achieved. Furthermore, the impact of prophylaxis has been uneven across hemophilia populations and the promise of greater physical activity free of hemorrhage has not been entirely fulfilled. This episode explores the history, evolution, and current state of prophylaxis, and highlights the current and future areas of further investigation.   Contributors: Episode Advisors: Dr. Marilyn Manco Johnson Dr. Kathelijn Fischer   Contributors: Dr. Robert Sidonio Dr. Manuel Carcao  Dr. Maria Elisa Mancuso Susan Lynch   Senior Advisor: Dr. Donna DiMichele   Links to learn more: A history of prophylaxis in haemophilia Hemophilia A and B: Routine management including prophylaxis Young adult outcomes of childhood prophylaxis for severe hemophilia A: results of the Joint Outcome Continuation Study Tailored frequency-escalated primary prophylaxis for severe haemophilia A: results of the 16-year Canadian Hemophilia Prophylaxis Study longitudinal cohort Factor VIII replacement prophylaxis in patients with hemophilia A transitioning to adults: a systematic literature review Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens Optimising prophylaxis in haemophilia A: The ups and downs of treatment Show Notes: Presenting Sponsor: Sanofi Subscribe to the Global Hemophilia Report Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter 

Gene therapy, hemostatic rebalancing therapies, memetic therapies, and even factor replacement therapy are all currently being explored in clinical trials, but while these therapies are paving the way to historical treatment ease and efficacy, they are also sweeping the hemophilia community into uncharted territory of potentially new challenges that must be addressed. This episode explores the current state of novel therapy investigation in hemophilia. Contributors: Dr. Maria Elisa Mancuso Dr. Glenn Pierce Dr. Steven Pipe Dr. Lindsey George Dr. Margareth Ozelo Dr. Guy Young Dr. Michael Recht Dr. Rich Gorman Mark Skinner Luke Pembroke   Senior Advisor: Dr. Donna DiMichele Links to learn more: Future Therapies - National Hemophilia Foundation 2021 clinical trials update: Innovations in hemophilia therapy Advances in the management of haemophilia: emerging treatments and their mechanisms Emerging drugs for hemophilia A: insights into phase II and III clinical trials Discussing investigational AAV gene therapy with hemophilia patients: A guide Hemophilia Clinical Trials - ClinicalTrials.gov Show Notes: Featured Advertiser: Sanofi Subscribe to BloodStream Media's Global Hemophilia Report Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter 

Inhibitor development has long been considered the most significant complication of severe hemophilia, but with the licensure of the first non-factor therapy and a robust pipeline of novel and gene therapeutics being investigated, is it still of critical importance that we better understand inhibitors? If so, what research is currently taking place, and what can he hope to learn from it? This episode of the Global Hemophilia Report from BloodStream Media features contributions and citations from investigators, clinicians, and research funders dedicated to this topic, as well as from patients and caregivers who can speak to their lived experience of inhibitors.   For more on inhibitor research: Inhibitors and Hemophilia - CDC ATHN 8: PUPs Matter Study - ATHN The Hemophilia Inhibitor Eradication Trial - ClinicalTrials.gov A New Plan for Tackling Inhibitors - NHF ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders - ASH The prospective Hemophilia Inhibitor PUP Study reveals distinct antibody signatures prior to FVIII inhibitor development - ASH   Show Notes: Featured Advertiser: Sanofi Genzyme Subscribe to BloodStream Media's Global Hemophilia Report Senior Adviser: Dr. Donna DiMichele   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter 

Led by science, curiosity, and storytelling, the Global Hemophilia Report from BloodStream Media is an entertaining monthly journey through the investigations and science driving hemophilia research around the world. Produced by: Believe Limited & BloodStream Media Hosted by: Patrick James Lynch Senior Advisor: Dr. Donna DiMichele Featured Advertiser: Sanofi Genzyme #GHRpod #hemophilia #clinicalresearch #bloodstreammedia