Community Practice Connections™: New Horizons in Hemophilia: Preparing for the Advent of Gene Therapy

Released On
February 4, 2022

Expires On
February 4, 2023

Media Type

Completion Time
60 minutes

Emergency Medicine, Genetics, Hematology-Oncology, Pediatrics

Bleeding Disorders, Hemophilia, Pharmacology

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This activity is provided by Physicians’ Education Resource®, LLC.


This activity is supported by an educational grants from BioMarin Pharmaceutical Inc. and CSL Behring LLC.

Credit Available

  • Physicians — maximum of 1.0 AMA PRA Category 1 Credit(s)
  • Nurses — 1.0 Contact Hours

All other healthcare professionals completing this course will be issued a statement of participation.

Target Audience

This educational program is directed toward hematologists, specialty NPs, PAs, and RNs. Pediatricians, emergency medicine clinicians, and other HCPs involved in the management of patients with hemophilia are invited to participate.

Program Overview

Hemophilia A is caused by a mutation in the gene coding for coagulation factor VIII (FVIII), resulting in the loss of functional FVIII protein. FVIII is a critical cofactor for factor IX (FIX), which is deficient in patients with hemophilia B. Treatment of hemophilia requires lifelong use of clotting factor replacement therapy. Recent and ongoing clinical trials are currently investigating novel therapeutic approaches such as gene therapy to improve management of this condition.

This Community Practice Connections™ program provides an in-depth review of some of the key highlights from the live program New Horizons in Hemophilia: Preparing for the Advent of Gene Therapy held in December 2021. This unique and engaging multimedia activity is ideal for the community-based clinician and focuses on the practical aspects of managing patients with hemophilia, putting recent trial data into a clinical context. The program is designed for those who did not attend the live meeting and to help reinforce learnings for those who did.

Learning Objectives

Upon completion of this activity, participants should be better able to:

  • Describe the role of gene therapy for the treatment of hemophilia A and B
  • Evaluate clinical trial findings investigating the role of gene therapy in patients with hemophilia A and B
  • Assess current evidence regarding the relationship between cost-effectiveness data and access barriers for gene therapy
  • Select appropriate therapies for patients with hemophilia based on patient-centered considerations



Steven W. Pipe, MD

Laurence A. Boxer Research Professor of Pediatrics
Director, Pediatric Hemophilia and Coagulation Disorders Program
Medical Director, Special Coagulation Laboratory
University of Michigan
Ann Arbor, MI


Stacy E. Croteau, MD, MMS

Medical Director
Boston Hemophilia Center
Assistant Professor of Pediatrics
Harvard Medical School
Boston, MA


Andrew D. Leavitt, MD

Director, Noncancerous Blood Disorders
Co-Director, Hemophilia Treatment Center
University of California San Francisco
San Francisco, CA

Accreditation/Credit Designation

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 1.0 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 1.0 Contact Hours.

Faculty, Staff, and Planners’ Disclosures

The staff of Physicians’ Education Resource®, LLC have no relevant financial relationships with ineligible entities.

Steven W. Pipe, MD
  • Grant/Research Support: Siemens, YewSavin
  • Consultant: ApcinteX, ASC Therapeutics, Bayer Healthcare, BioMarin Pharmaceutical, Catalyst Biosciences, CSL Behring, GeneVentiv Therapeutics, HEMA Biologics, Freeline Therapeutics, Novo Nordisk, Pfizer, F. Hoffmann-La Roche/Genentech, Sangamo Therapeutics, Sanofi, Takeda Pharmaceutical Company, Spark Therapeutics, uniQure
Stacy E. Croteau, MD, MMS
  • Grant/Research Support: Spark Therapeutics
  • Consultant: BioMarin Pharmaceutical, CSL Behring, Pfizer, Sanofi
Andrew D. Leavitt, MD
  • Grant/Research Support: Sangamo Therapeutics, Pfizer, BioMarin Pharmaceutical, Catalyst Biosciences
  • Consultant: Genentech, BioMarin Pharmaceutical, Catalyst Biosciences, Merck, HEMA Biologics
  • Shareholder: Johnson & Johnson

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

  1. Complete the activity (including pre- and post-activity assessments).
  2. Answer the evaluation questions.
  3. Request credit using the drop-down menu.
You may immediately download your certificate.

Course Viewing Requirements

Supported Browsers:
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.