Individualized Nonfactor Therapy for Hemophilia

Target Audience

The educational design of this activity addresses the needs of US-based pediatric and adult hematologists, hematologist-oncologists, hematology advanced practice providers, and other clinicians (pediatricians/primary care) who manage patients with hemophilia.

Program Overview

Although the standard of care for individuals with hemophilia involves prophylactic factor replacement therapy, the IV mode of administration, need for frequent injections, and potential for development of inhibitors not only negatively impacts quality of life (QoL) but may also significantly reduce the efficacy of prophylaxis. Fortunately, the factor VIII mimetic agent, emicizumab, has been available since 2017 for the treatment of patients with hemophilia A, and in 2024, 3 novel rebalancing agents, concizumab, marstacimab, and fitusiran, were US Food and Drug Administration (FDA)-approved for the treatment of patients with hemophilia. In this Hematology News: Hemophilia Edition activity, Individualized Nonfactor Therapy for Hemophilia, Drs. Guy Young and Stacy Croteau discuss the most recent data and their expert opinions regarding how to incorporate nonfactor therapy into treatment regimens for patients with hemophilia A and B with and without inhibitors.

Educational Objectives 

After completing this activity, the participant should be better able to:

1. Describe the mechanisms of action and therapeutic targets of novel and emerging nonfactor therapies for hemophilia.
2. Evaluate the clinical efficacy, safety, and durability of nonfactor therapy in the management of hemophilia.
3. Examine how nonfactor therapy advancements influence individualized treatment plans and long-term care strategies for patients with hemophilia.
4. Develop best practices for monitoring hemostatic status and managing breakthrough bleeding in patients with hemophilia.

Physician Accreditation Statement

Integritas Communications is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Physician Credit Designation

Integritas designates this enduring activity for a maximum of 0.75 AMA PRA Category 1 Credit™.  Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Disclosures of Relevant Financial Relationships

Integritas adheres to the policies and guidelines, including the Standards for Integrity and Independence in Accredited CE, set forth to providers by the Accreditation Council for Continuing Medical Education (ACCME) and all other professional organizations, as applicable, stating those activities where continuing education credits are awarded must be balanced, independent, objective, and scientifically rigorous. All persons in a position to control the content are required to disclose all financial relationships with any ineligible company within the past 24 months to Integritas.  All financial relationships reported are identified as relevant and mitigated by Integritas in accordance with the Standards for Integrity and Independence in Accredited CE in advance of delivery of the activity to learners. The content of this activity was vetted by Integritas to assure objectivity and that the activity is free of commercial bias.  

All relevant financial relationships have been mitigated. 

The faculty have the following relevant financial relationships with ineligible companies:

Stacy E. Croteau, MD, MMS:

• Consulting Fees: BioMarin, Pfizer Inc., Sanofi S.A.
• Contracted Research: Sanofi S.A., Spark Therapeutics, Inc. 

Guy Young, MD:

• Consulting Fees: Alnylam Pharmaceuticals, Inc., ASC Therapeutics, Inc., Bayer Corporation, BioMarin, CSL Behring, Genentech Inc./Hoffmann-La Roche Ltd, HEMA Biologics LLC/LFB SA, Novo Nordisk Inc., Octapharma USA, Inc., Pfizer Inc., Sanofi S.A., Spark Therapeutics, Inc., Takeda Pharmaceutical Company Limited 
• Contracted Research: HEMA Biologics LLC, Sanofi S.A.

The planners and managers at Integritas Communications have no relevant financial relationships to disclose.

Disclosure of Unlabeled Use

This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the US Food and Drug Administration. Integritas Communications does not recommend the use of any agent outside of the labeled indications.  

The opinions expressed in the educational activity are those of the faculty and do not necessarily represent the views of any organization associated with this activity. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.

Disclaimer

Participants have an implied responsibility to use the newly acquired information to enhance patient outcomes and their own professional development. The information presented in this activity is not meant to serve as a guideline for patient management. Any procedures, medications, or other courses of diagnosis or treatment discussed in this activity should not be used by clinicians without evaluation of patient conditions and possible contraindications or dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities.

Instructions to Receive Credit

In order to receive credit for this activity, the participant must score 70% on the posttest and complete the program evaluation.

Fee Information & Refund/Cancellation Policy

There is no fee for this educational activity.

Integritas Contact Information

For questions about this activity, please contact Integritas Communications at info@exchangecme.com.