This Infographic activity uses a self-directed, graphic approach to introducing emerging hemophilia A and hemophilia B therapies. In this activity, Steven Pipe, MD and Guy Young, MD introduce the unmet medical needs experienced by patients with hemophilia. They review the mechanisms of action and most recent clinical trial data for the most advanced substitution therapies (emicizumab), hemostatic rebalancing agents (concizumab, marstacimab, and fitusiran), and gene therapies. The faculty also discuss how approval of these treatments could affect patient care, and the questions that need to be addressed in ongoing phase 3 clinical trials for them to enter clinical practice.
Course Credit:
1 AMA PRA Category 1 CreditTM
1 ANCC Contact Hour
Dates:
Opens: 2022-07-29
Closes: 2023-07-29
Target Audience:
This activity was developed for pediatric and adult hematologists and nurse practitioners who specialize in treating patients with hemophilia, especially those practicing in hemophilia treatment centers (HTCs) or who treat complex cases (eg, patients with inhibitors).
This activity is supported by an independent medical education grant from Novo Nordisk, Inc.
Accreditation
The Annenberg Center for Health Sciences at Eisenhower designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Annenberg Center for Health Sciences at Eisenhower designates this enduring activity for a maximum of 1 contact hour.
Additional Content Planners
Chris Fischer, MSc (Medical Writer)No significant relationships to disclose.
Amber Lea Lambert, MSN, FNP-C, DNP (Nurse Reviewer)
No significant relationships to disclose.
Kam A. Newman, MD (Peer Reviewer)
No significant relationships to disclose.
Annenberg Center for Health Sciences
Staff at the Annenberg Center for Health Sciences at Eisenhower have no relevant financial relationships to disclose.
All of the financial relationships listed for these individuals have been mitigated.
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Steven Pipe, MD
Professor of Pediatrics and Pathology
Laurence A. Boxer Research Professor of Pediatrics and Communicable Diseases
Pediatric Medical Director, Hemophilia and Coagulation Disorders Program
Director, Special Coagulation Laboratory
University of Michigan
Ann Arbor, Michigan -
Guy Young, MD
Director, Hemostasis and Thrombosis Center
Director, Clinical Coagulation Laboratory
Cancer and Blood Disorders 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
Presenting Faculty
Downloads
Learning Objectives
- Describe the mechanism of action for emerging treatments for hemophilia
- Recall the clinical trial data demonstrating the efficacy of approved and emerging hemophilia treatments
- Recall the clinical trial data demonstrating the safety of approved and emerging hemophilia treatments
- Identify clinical applications for approved and emerging therapies by applying clinical trial results
Faculty Disclosures
Steven Pipe, MD
Consultant: Apcintex, ASC Therapeutics, Bayer, BioMarin, Catalyst Biosciences, CSL Behring, GeneVentiv, HEMA Biologics, Freeline, LFB, Novo Nordisk, Pfizer, Regeneron/Intellia, Roche/Genentech, Sangamo Therapeutics, Sanofi, Spark Therapeutics, Takeda, uniQure
Guy Young, MD
Consultant: Apcintex, BioMarin, Genentech/Roche, Pfizer, Sanofi Genzyme, Spark, Takeda
Speakers Bureau: BioMarin, Genentech/Roche, Grifols, Sanofi Genzyme, Spark
Research Support: Genentech/Roche, Grifols, Takeda