PODCAST
Advances in Hemophilia: From Joint Health to FVIII Guidelines and the Clinical Integration of Rebalancing Agents
Guy Young, MD; Annette von Drygalski, MD, PharmD, RMSK; Steven Pipe, MD; Robert F. Sidonio Jr., MD, MSc
First published: xx April 2026
Chapter 1. Optimizing Joint Health
Director, Hemostasis and Thrombosis Center
Director, Clinical Coagulation Laboratory
Cancer and Blood Disease Institute
Children's Hospital Los Angeles
Professor of Pediatrics
Division of Hematology/Oncology
Department of Pediatrics
University of Southern California Keck School of Medicine
Los Angeles, California
​Annette von Drygalski, MD,
PharmD, RMSK
Professor of Clinical Medicine
Director, Center for Bleeding and Clotting Disorders
Associate Director, Center of Excellence for Hereditary Hemorrhagic Telangiectasia
Program Director, Coagulation Medicine Fellowship
Associate Program Director, Hematology Fellowship
University of California, San Diego
San Diego, California
Chapter 2. Guidelines for FVIII Levels
Director, Hemostasis and Thrombosis Center
Director, Clinical Coagulation Laboratory
Cancer and Blood Disease Institute
Children's Hospital Los Angeles
Professor of Pediatrics
Division of Hematology/Oncology
Department of Pediatrics
University of Southern California Keck School of Medicine
Los Angeles, California
Professor of Pediatrics and Pathology
Laurence A. Boxer Research Professor of Pediatrics
University of Michigan
Ann Arbor, Michigan
Chapter 3. Rebalancing Agents: Current Evidence and Clinical Integration
Director, Hemostasis and Thrombosis Center
Director, Clinical Coagulation Laboratory
Cancer and Blood Disease Institute
Children's Hospital Los Angeles
Professor of Pediatrics
Division of Hematology/Oncology
Department of Pediatrics
University of Southern California Keck School of Medicine
Los Angeles, California
Professor of Pediatrics
Emory University School of Medicine
Medical Director
Hemophilia of Georgia Pediatric Center for
Bleeding and Clotting Disorders of Emory
Atlanta, Georgia
This activity is jointly provided by Partners for Advancing Clinical Education (PACE) and CMEology.
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This activity is supported by an independent educational grant from Sanofi.
Abstract
Despite major advances in hemophilia care, many patients continue to experience breakthrough bleeding, progressive joint morbidity, inhibitor development, and substantial treatment burden. To help more individuals achieve active, unrestricted lives, health care professionals must move beyond traditional prophylaxis targets by applying evolving evidence on factor VIII (FVIII) levels, optimizing prophylaxis to provide sustained bleed protection, and individualizing therapy to meet patients' needs, preferences, and real-world considerations. This 3-segment podcast features expert discussions on optimizing joint health across the lifespan, including subclinical bleeding and imaging-based monitoring; applying current and emerging guidance on FVIII targets and factor-based strategies; and integrating rebalancing agents into practice, with a focus on clinical outcomes, safety and risk mitigation, treatment burden, patient selection, and individualized hemophilia management.
Plain Language Abstract
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Despite major advances in hemophilia care, many people with hemophilia still experience breakthrough bleeding, long-term joint complications, and significant treatment burden. This 3-part podcast series focuses on central aspects of hemophilia management, including joint health, factor VIII (FVIII) treatment targets, and newer therapeutic approaches.
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Joint damage remains a major concern, even with preventive treatment. Bleeding that may not be noticeable can still occur and contribute to long-term joint damage, emphasizing the need for regular monitoring and treatment adjustments.
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Factor VIII levels play a central role in treatment planning for hemophilia A. Higher and more sustained FVIII levels are increasingly recognized as important for reducing bleeding risk and protecting joint health. Different factor-based treatment strategies can help clinicians achieve these targets while balancing treatment frequency, effectiveness, and patient needs.
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Rebalancing agents provide additional treatment options for people with hemophilia A and B, including those with inhibitors. These therapies work differently from traditional approaches and may reduce bleeding, ease treatment burden, and improve quality of life; they require careful patient selection and monitoring for safety.