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

Subscribe to the Global Hemophilia Report

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

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