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Cases and Conversations™: The Rationale for Utilizing Non-Factor Replacement Therapies in Hemophilia Care

Sponsor: Novo Nordisk, Inc.
Program: Friday Satellite Symposia
Hematology Disease Topics & Pathways:
Bleeding and Clotting, Hemophilia, Education, Diseases
Friday, December 6, 2024: 3:00 PM-6:00 PM
Room 6A (San Diego Convention Center)
Chair:
Steven R. Lentz, MD, PhD, University of Iowa
Disclosures:
Lentz: Sanofi: Consultancy; Novo Nordisk: Consultancy.
Speakers:
Lynn Malec, MD, MSc, Versiti Blood Research Institute and Alice Ma, MD, University of North Carolina
Disclosures:
Malec: Sobi: Consultancy; Spark Therapeutics: Consultancy; Novo Nordisk: Consultancy; Sanofi: Consultancy, Speakers Bureau; CSL Behring: Consultancy, Speakers Bureau; Takeda: Consultancy; Pfizer: Consultancy. Ma: Takeda: Honoraria, Research Funding.

Hemophilia is an inherited bleeding disorder resulting from a deficiency in plasma coagulation proteins. The WFH recommends prophylactic treatment with replacement clotting factors starting before age 3 for all patients with a severe hemophilia. Prophylaxis has dramatically improved treatment, leading to reduced hospitalizations and absence from school or work and improving health related quality of life (HRQOL). Nevertheless, even with the progress achieved with replacement factors, therapy is costly and burdensome. New approaches to hemophilia treatment are therefore being developed to try and address these concerns. Through engaging case-based discussion and video animations, this accredited symposium will provide learners with insights regarding the pathophysiological pathways underlying hemophilia and new non-factor therapeutic strategies, the limitations in the current standard of care for hemophilia, and the role of non-factor replacement therapy in improving patient outcomes and quality of life.