Gene Therapy for Hemophilia B—The Next Step Forward in Disease Management
April 20, 2023
April 20, 2024
This activity is provided by Physicians’ Education Resource®, LLC.
This activity is supported by an educational grant from CSL Behring LLC.
- Physicians — maximum of 1.0 AMA PRA Category 1 Credit(s)™
All other healthcare professionals completing this course will be issued a statement of participation.
This educational 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 attend.
Gene therapy for hemophilia B has the potential to reduce the treatment burden for patients and their care partners by eliminating the need for regular prophylaxis through long-term expression of endogenous factor IX at levels sufficient to provide protection from bleeding. Challenges associated with gene therapy still need to be addressed to ensure widespread therapeutic use, but it offers a promising option for this patient population. In this program, expert faculty will assess clinical trial data for gene therapy in patients with hemophilia B, review strategies regarding patient selection for gene therapy, and discuss the role of the multidisciplinary team in the ongoing management of patients with hemophilia B.
Upon completion of this activity, participants should be better able to:
- Outline the current standard of care for patients with hemophilia B
- Assess clinical trial data for gene therapy for hemophilia B
- 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
Michael Recht, MD, PhD, MBA
Professor of Clinical Pediatrics, Hematology/Oncology
Chief Science Officer, Research
Yale University School of Medicine
New Haven, CT
Rajiv K. Pruthi, MBBS
Codirector, Special Coagulation Laboratory
Director, Hemophilia Treatment Center
Codirector, Molecular Hematopathology Laboratory
Annette von Drygalski, MD, PharmD, RMSK
Professor of Clinical Medicine
Director, Hemophilia and Thrombosis Treatment Center
University of California
San Diego, CA
Physician Accreditation Statement
Physicians’ Education Resource®, LLC, is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.
Physician Credit Designation
Physicians’ Education Resource®, LLC, designates this enduring material for a maximum of 1.0 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Disclosures of Conflicts of Interest
The staff of Physicians’ Education Resource®, LLC have no relevant financial relationships with ineligible entities.
- Michael Recht, MD, PhD, MBA
- Grant/Research Support: Bayer, BioMarin Pharmaceutical, CSL Behring, Genentech, Grifols, HEMA Biologics, LFB, Novo Nordisk A/S, Octapharma USA, Pfizer, Spark Therapeutics, Takeda, uniQure NV
- Consultant: Catalyst Pharmaceuticals, CSL Behring, Genentech, HEMA Biologics, Kedrion, Novo Nordisk A/S, Pfizer, Sanofi, Takeda, uniQure NV
Rajiv K. Pruthi, MBBS, has no relevant financial relationships with ineligible companies.
- Annette von Drygalski, MD, PharmD, RMSK
- Other: Honoraria for participating in scientific advisory board panels, consulting, and speaking engagements for Biomarin Pharmaceutical, Bioverativ/ Sanofi, CSL Behring, Genentech, Novo Nordisk A/S, Pfizer, Regeneron Pharmaceuticals, Spark Therapeutics, uniQure NV; cofounder and member of the board of directors of Hematherix, a biotech company that is developing SuperFVa therapy for bleeding complications
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
There are no fees for participating and receiving CME credit for this enduring activity. To receive CME credit participants must:
- Read the CME/CE information and faculty disclosures.
- Participate in the online activity.
- Submit the evaluation form.
Certificates will be emailed to the participant.
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 healthcare 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.