Clinical Consults™: Hemophilia Perspectives: Current Insights on the Evolution of Gene Therapy

This activity has expired.
Release Date: January 31, 2019
Expiration Date: January 31, 2020
Estimated Time to Complete Activity: 30 minutes
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The following activity features highlights and faculty commentary from a satellite CME Dinner Symposium preceding the 60th American Society of Hematology Annual Meeting & Exposition (ASH) that address issues on the evolution of gene therapy in hemophilia.

Target Audience
This online activity is designed to meet the educational needs of physicians, advanced practice registered nurses, registered nurses, as well as other clinicians who diagnose, treat, and counsel persons with hemophilia.

Statement of Need/Program Overview
Because the advancements in gene therapy indicate that hemophilia may be curable, it is essential that healthcare providers understand the complex nature of the genetics of hemophilia, how gene therapies work in general, and the evidence to date regarding their effectiveness in persons with hemophilia.

Educational Objectives
After participating in this activity, the participant should be better able to:

  • Describe the genetic complexity underlying hemophilia
  • Discuss potential benefits of undergoing genetic diagnostic testing for hemophilia
  • Relate the essential aspects of hemophilia gene therapy and gene editing
  • Review emerging gene therapy‐based options available for people with hemophilia (PWH)

Faculty Chair
Michael Recht, MD, PhD
Michael Recht, MD, PhD
Professor of Pediatrics and Medicine
Director, The Hemophilia Center
Oregon Health & Science University
Portland, OR

Faculty Presenters
Tammuella  Chrisentery Singleton, MD
Tammuella "Tami" Chrisentery Singleton, MD
Assistant Professor of Medicine and Pediatrics Section Chief, Pediatric
Hematology/Oncology Louisiana Center for Bleeding and Clotting Disorders
Tulane University School of Medicine
New Orleans, LA

Gerald Dolan, MD
Gerald Dolan, MD
Director, St Thomas' Haemophilia Centre and Head, Haemostasis & Thrombosis Service
Guy’s and St Thomas' Hospital
London, UK

Joint Accreditation Statement
In support of improving patient care, this activity has been planned and implemented by the Postgraduate Institute for Medicine and Educational Concepts in Medicine. Postgraduate Institute for Medicine is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.

Physician Continuing Medical Education
The Postgraduate Institute for Medicine designates this enduring material for a maximum of 0.5 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Continuing Nursing Education
The maximum number of hours awarded for this Continuing Nursing Education activity is 0.5 contact hour. 

ASCLS P.A.C.E.® Credits
Postgraduate Institute for Medicine is approved as a provider of continuing education programs in the clinical laboratory sciences by the ASCLS P.A.C.E. ® Program. This program is approved for up to 0.50 ASCLS P.A.C.E.® contact hours.  

Method of Participation
During the period of January 31, 2019, through January 31, 2020, participants must:

  • Read the learning objectives and faculty disclosures
  • Study the educational activity
  • Complete the posttest by recording the best answer to each question
  • Complete the evaluation form

A statement of credit will be issued automatically upon successful completion of the post-activity survey with a score of 75% or better and submitting the activity evaluation.

Fee Information
There is no fee for this educational activity.

Disclosure of Conflicts of Interest
The faculty reported the following financial relationships or relationships to products or devices they or their spouse/life partner have with commercial interests related to the content of this CME activity:

Name of Faculty or Presenter Reported Financial Relationship
Michael Recht, MD, PhD Consulting Fees: Genentech, Kedrion, Pfizer, and uniQure

Contracted Research: Bioverativ, Genentech, Novo Nordisk, and Shire
Tami Singleton, MD Consulting Fees: Novo Nordisk, Shire, Bayer, Grifols, Genentech, Bioverativ, Biomarin and CSL Behring

Fees for NON-CME/CE Services Received Directly from a Commercial Interest or their Agents: Novo Nordisk, Shire, Genentech, Grifols and CSL Behring
Gerald Dolan, MD Consulting Fees: Shire, Novo Nordisk, Pfizer, Bayer, Roche, and Spark Therapeutics, Inc.

Fees for Non-CME/CE Services Received Directly from a Commercial Interest or their Agents: Shire, Pfizer, Roche and Novo Nordisk

Contracted Research: Pfizer, Bayer, Novo Nordisk. Biomarin, Spark Therapeutics, Freeline, and Roche

The planners and managers reported the following financial relationships or relationships to products or devices they or their spouse/life partner have with commercial interests related to the content of this CME activity:

Name of Planner or Manager Reported Financial Relationship
Postgraduate Institute for Medicine Clinical Reviewers Have no real or apparent conflicts of interest to report.
Educational Concepts in Medicine Reviewers Have no real or apparent conflicts of interest to report.

Disclosure of Unlabeled Use
This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. The planners of this activity do 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 the planners. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.

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 or suggested in this activity should not be used by clinicians without evaluation of their patients’ conditions and possible contraindications and/or dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities.


Commercial Support
This educational activity is supported by an independent medical education grant from Spark Therapeutics, Inc.