Community Practice Connections™: Exploring the Potential Role for Gene Therapy in Hemophilia B
January 31, 2023
January 31, 2024
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This activity is provided by Physicians’ Education Resource®, LLC.
This activity is supported by an educational grant from CSL Behring LLC.
- Physicians — maximum of 1.5 AMA PRA Category 1 Credits™
- Nurses — 1.5 Contact Hours
All other healthcare professionals completing this course will be issued a statement of participation.
This activity is directed toward hematologists and hematologist-oncologists. Pediatricians, nurses, nurse practitioners, physician assistants, and other clinicians involved in the management of patients with hemophilia B are invited to participate.
In hemophilia B, an absence of clotting factor IX leads to a failure to facilitate and stabilize the primary platelet and fibrin plug that initially forms a clot. As a result, patients experience prolonged bleeding. Despite recent improvements in its treatment options, hemophilia B continues to have a significant negative impact on patients’ health-related quality of life. Gene therapy for hemophilia B has the potential to reduce the treatment burden for patients and their care providers by eliminating the need for regular prophylaxis.
This Community Practice Connection™ program provides an in-depth review of some of the key highlights from the 2022 American Society of Hematology Annual Meeting and Exposition held in December 2022. This unique and engaging multimedia activity is ideal for the community-based clinician, and it focuses on the practical aspects the emerging gene therapy for patients with hemophilia B, putting recent clinical trial data into clinical context. The program is designed for those who did not attend the live meeting and to help reinforce learnings for those who did.
- Upon successful completion of this activity, you should be better prepared to:
- Discuss the burden of disease for patients with hemophilia B
- Outline the current standard of care for patients with hemophilia B
- Assess clinical trial data for gene therapy for hemophilia B
- Describe models for implementation of gene therapy for hemophilia B into clinical practice
- Apply strategies to select appropriate patients for gene therapy
- Review the role of the multidisciplinary team in the management of patients receiving gene therapy for hemophilia B
Christine L. Kempton, MD, MSc
Professor, Department of Hematology and Medical Oncology and Department of Pediatrics
HoG Inc Director's Chair in Hemostasis
Director, Benign Hematology Program
Director, Hemophilia of Georgia Center for Bleeding & Clotting Disorders of Emory
Vice Chair for Faculty Development, Department of Hematology and Medical Oncology
Emory University School of Medicine
Andrew D. Leavitt, MD
Director, Program for Noncancerous Blood Disorders
Co-Director, Hemophilia Treatment Center
University of California, San Francisco
San Francisco, CA
Steven Pipe, MD
Professor of Pediatrics and Pathology
Director, Special Coagulation Laboratory
Laurence A. Boxer, MD Research Professor of Pediatrics and Communicable Diseases
Medical Director, Pediatric Hemophilia and Coagulation Disorders Program
University of Michigan
Ann Arbor, MI
Annette Von Drygalski, MD, PharmD
Professor of Medicine
Director, Hemophilia and Thrombosis Treatment Center
UC San Diego Health
San Diego, CA
Physicians’ Education Resource®, LLC, is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.
Physicians’ Education Resource®, LLC, designates this enduring material for a maximum of 1.5 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Physicians’ Education Resource®, LLC, is approved by the California Board of Registered Nursing, Provider #16669, for 1.5 Contact Hours.
Faculty, Staff, and Planners’ Disclosures
The staff of Physicians’ Education Resource®, LLC, have no relevant financial relationships with ineligible entities.
- Christine L. Kempton, MD, MSc
- Consultant: BioMarin Pharmaceutical, Genentech, Pfizer, Sanofi US, Spark Therapeutics, Takeda
- Andrew D. Leavitt, MD
- Grant/Research Support: BioMarin Pharmaceutical, Pfizer, Sangamo Therapeutics
- Consultant: BioMarin Pharmaceutical, Pfizer
- Steven Pipe, MD
- Grant/Research Support: Siemens Corporation, YewSavin
- Consultant: Apotex Pharmaceutical Holdings, ASC Therapeutics, Bayer, BioMarin Pharmaceutical, CSL Behring, Freeline Therapeutics Holdings, Genentech, GeneVentiv Therapeutics, HEMA Biologics, LFB, Novo Nordisk, Pfizer, Regeneron Pharmaceuticals, Roche, Sanofi, Spark Therapeutics, Takeda
- Annette Von Drygalski, MD, PharmD
- Grant/Research Support: Pfizer, Sanofi
- Consultant: ASC, Biomarin, CSL-Behring, Genentech, Novo Nordisk, Regeneron, Sanofi, Takeda, uniQure
PER® mitigated all COI for faculty, staff, and planners prior to the start of this activity by using a multistep process.
Instructions for Participation and Credit
- Complete the activity (including pre- and post-activity assessments).
- Answer the evaluation questions.
- Request credit using the drop-down menu.
Course Viewing Requirements
Internet Explorer 8.0+ for Windows 2003, Vista, XP, Windows 7, Windows 8.1 and above
Google Chrome 28.0+ for Windows, Mac OS, or Linux
Mozilla Firefox 23.0+ for Windows, Mac OS, or Linux
Safari 6.0+ for Mac OSX 10.7 and above
Supported Phones & Tablets:
Android 4.0.3 and above
iPhone/iPad with iOS 6.1 or above
Off-Label Disclosure and Disclaimer
This activity may or may not discuss investigational, unapproved, or off-label use of drugs. Learners are advised to consult prescribing information for any products discussed. The information provided in this accredited activity is for continuing education purposes only and is not meant to substitute for the independent clinical judgment of a health care professional relative to diagnostic, treatment, or management options for a specific patient’s medical condition. The opinions expressed in the content are solely those of the individual faculty members and do not reflect those of PER® or any company that provided commercial support for this activity.