Global Hemophilia Report

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:

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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

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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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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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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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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

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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

Featured Advertiser:

Sanofi

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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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