Global Hemophilia Report: Recent Episodes
BloodStream Media
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
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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
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Show Notes:
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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
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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
-
Castaman G, Matino D. Haematologica. 2019;104(9):1702-1709.
-
Dolan G, Benson G, Duffy A, et al. Blood Rev. 2018;32(1):52-60.
-
Mann DM, Stafford KA, Poon M-C, Matino D, Stafford DW. Haemophilia. 2021;27(3):332-339.
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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
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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
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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
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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
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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.
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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
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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/
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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
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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
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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
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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
-
Castaman G, Matino D. Haematologica. 2019;104(9):1702-1709.
-
Dolan G, Benson G, Duffy A, et al. Blood Rev. 2018;32(1):52-60.
-
Mann DM, Stafford KA, Poon M-C, Matino D, Stafford DW. Haemophilia. 2021;27(3):332-339.
Connect with the Global Hemophilia Report
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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.
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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
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Show Notes:
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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
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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
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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
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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
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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.
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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
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Show Notes:
Presenting Sponsor: Sanofi
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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
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Show Notes:
Presenting Sponsor: Sanofi
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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
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Show Notes:
Presenting Sponsor: Sanofi
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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
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Show Notes:
Presenting Sponsor: Sanofi
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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
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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
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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
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
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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
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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
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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
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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, MPH, MCHES
Karina Lopez, MPH
Adrian Palau-Tejeda
Marissa Melton
Chris Bombardier
Mosi Williams
Senior Advisor:
Donna DiMichele, MD
Hosted by:
Patrick James Lynch
Written by:
Simantini Karve, PhD
Show Notes:
Presenting Sponsor: Sanofi
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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
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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
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Watch the film Bombardier Blood
Listen to The Final Summit podcast
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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
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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
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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
Sara L. Schwartz, PhD
Tam Perry, PhD
Senior Advisor:
Donna DiMichele, MD
Hosted by:
Patrick James Lynch
Listen to Blood of the Paladin Podcast
Show Notes:
Presenting Sponsor: Sanofi
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Listen to BloodStream’s Journey to Joint Health episode.
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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
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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
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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
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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:
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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]
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Gualtierotti R, et al. Updates on novel non-replacement drugs for hemophilia. Pharmaceuticals 2022;15(10):1183. Doi: 10.3390/ph15101183
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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
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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]
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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
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Nathwani A, et al. Liver gene therapy. Hum Gene Thur 2022;33:17-18. Doi: 10.1089/hum.2022.169
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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
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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]
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Potts WTW. Royal haemophilia. J Biol Educ 1996;30(3):207-17. DOI: 10.1080/00219266.1996.9655504
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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]
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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
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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
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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
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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
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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.
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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
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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
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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
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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
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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
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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
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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
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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
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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
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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
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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
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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
Senior Advisor:
Donna DiMichele, MD
Episode Advisors:
Angela Weyand, MD
Robert Sidonio, MD
Hosted by:
Laurence Woollard
Links to learn more:
Show Notes:
Presenting Sponsor: Sanofi
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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
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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.
Contributors:
Dr. Marilyn Manco-Johnson
Dr. Kathelijn Fischer
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
Episode Advisors:
Dr. Marilyn Manco Johnson
Dr. Kathelijn Fischer
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
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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: Dr. Marilyn Manco Johnson
Dr. Kathelijn Fischer
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
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Global Hemophilia Report on LinkedIn
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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
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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
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