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Exploring curative therapies for sickle cell disease

When to consider HCT or gene therapy

Release date: Sept. 6, 2023

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Interested in claiming CME credit?

Be sure to complete the pre-test, then complete the post-test after watching the webinar. The pre- and post-test can be found at the on-demand webinar link. For more information, see the instructions for participation and requesting credit below.

Target Audience

This educational activity is intended for physicians, nurses and other members of the health care team who care for patients with sickle cell disease (SCD) who may benefit from hematopoietic cell transplantation (HCT) or gene therapy. 

Program Overview

This webinar focuses on recent research in the use of hematopoietic cell transplant (HCT) and gene therapy as curative therapies for SCD. A panel of experts explore patient eligibility, perceptions and overcoming barriers to care. Many patients and providers alike are unfamiliar with HCT as a potential curative option for SCD patients, and there are many misperceptions as gene therapy gains more public attention. Demystifying transplant among SCD providers and elucidating inclusion and exclusion criteria for curative therapies can help physicians and families make informed treatment decisions. Encouraging discussion about HCT and gene therapy’s role in the treatment of SCD can help ensure that patients and providers can appropriately weigh their options, increasing access to curative care when appropriate. 

During this webinar, our expert HCT, gene therapy, and SCD panelists discuss some of the latest research and advances in HCT and gene therapy for SCD, as well as compare eligibility criteria and barriers to care through an example case. The panelists further explore prominent physician and patient perceptions of curative therapies for SCD and how to ensure access to these therapies for patients and their families. This webinar is relevant for both transplant centers and community hematologists/oncologists and SCD program providers who refer out for transplant and cell therapies, discussing the role that all practitioners can play in improving access to curative treatment options.

Please review this information, and proceed below

Learning Objectives

Upon completion of this course, the learners should be able to:

Define current trends and advances in HCT and gene therapy as curative therapies for SCD 

  • Demystify HCT and gene therapy in the treatment of SCD
  • Identify eligibility criteria for curative SCD therapies and barriers to care
  • Describe patient resources for SCD through the Sickle Cell Warrior Program and HLA Today
  • Recognize how transplant centers and hematology/oncology or SCD providers can work together to improve access to HCT


Heather Stefanski, MD, PhD (moderator)
Vice President, Medical Services
National Marrow Donor Program® (NMDP)

Elizabeth Stenger, MD, MSc
Assistant Professor
Aflac Cancer & Blood Disorders Center
Children’s Healthcare of Atlanta/Emory University

Haydar Frangoul, MD, MS
Program Director
Sarah Cannon Pediatric Hematology/Oncology & Cellular Therapy
TriStar Centennial

Shalu Narang, MD, MPH
Associate Clinical Professor, Rutgers & Robert Wood Johnson Medical School
Section Chief, Benign Hematology
Associate Director, Pediatric Hematology/Oncology Fellowship
Newark Beth Israel Medical Center & Children’s Hospital of New Jersey

Planning Committee

Anna Cincotta, FNP
Senior Clinical Operations Partner 

Karla Dawson, MPH
Health Equity Professional 

Olivia Gunn
Membership and Operations Senior Associate
American Society for Transplantation and Cellular Therapy 

Amber Ruffin, MPH
Associate Program Manager, Health Equity

Samantha Watters, MPH
Research and Education Lead, Community Physician Engagement

Instructions For Participation And Requesting Credit

There are no fees for participating and receiving credit for this activity. Successful completion is achieved by reviewing the CE information, completing the pre-test, completing the activity, and completing the post-test and evaluation form. A score of at least 75% on the posttest is required to successfully complete this activity. The participant may take the test until successfully passed.

Credit fulfillment 

  • If you are requesting AMA credits or a certificate of participation, your certificate will be available for download.

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Accredited Provider

This activity is jointly provided by The France Foundation, the National Marrow Donor Program (NMDP), and the American Society for Transplantation and Cellular Therapy (ASTCT).

Accreditation Statement

In support of improving patient care, this activity has been planned and implemented by The France Foundation and the National Marrow Donor Program (NMDP), and the American Society for Transplantation and Cellular Therapy (ASTCT). The France Foundation 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.

Credit Designation


The France Foundation designates this enduring activity for a maximum of 1.0 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

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

Disclosure Policy

In accordance with the ACCME Standards for Integrity and Independence in Accredited Continuing Education, The France Foundation (TFF), the National Marrow Donor Program (NMDP), and the American Society for Transplantation and Cellular Therapy (ASTCT) require that individuals in a position to control the content of an educational activity disclose all relevant financial relationships with any commercial entity. TFF, NMDP, and ASTCT resolve all conflicts of interest to ensure independence, objectivity, balance, and scientific rigor in all their educational programs. Furthermore, TFF, NMDP, ASTCT verify that all scientific research referred to, reported, or used in a CME/CE activity conforms to the generally accepted standards of experimental design, data collection, and analysis. TFF, NMDP, and ASTCT committed to providing learners with high-quality CME/CE activities that promote improvements in health care and not those of a commercial interest.

Planning committee members, faculty, reviewers, and activity staff have disclosed the following relevant financial relationships. All relevant financial relationships listed have been mitigated.

Name of individual Individual's Rose in Activity Name of Commercial Entity Nature of Relationship(s) Mechanism(s) implemented to resolve conflict of interest
Amber Ruffin, MPH Planning Committee Member, NMDP Staff No relevant financial disclosures N/A N/A
Anna Cincotta, FNP Planning Committee Member, Content Reviewer No relevant financial disclosures N/A N/A
Ashley Smeaton, MHA TFF Staff No relevant financial disclosures N/A N/A
Elizabeth Stenger, MD, MSc Faculty
  1. Editas Medicine, Bluebird Bio Inc.
  1. Contract Research
The following relationship was mitigated:
  • Bluebird Bio Inc ended in February 2023
Content was reviewed by a non-conflicted member of the planning committee to ensure that it is not commercially biased, was fair-balanced, and is based on scientific evidence and/or clinical reasoning.
Haydar Frangoul, MD Faculty
  1. Vertex Pharmaceuticals, Editas Medicine
  2. Jazz Pharmaceutical
  1. Non-CE Consulting 
  2. Non-CE Speakers Bureau
Content was reviewed by a non-conflicted member of the planning committee to ensure that it is not commercially biased, was fair-balanced, and is based on scientific evidence and/or clinical reasoning.
Heather Stefanski, MD, PhD Faculty Moderator No relevant financial disclosures N/A N/A
Heather Tarbox, MPH TFF Staff  No relevant financial disclosures N/A N/A
Karla Dawson, MPH Planning Committee Member, NMDP Staff No relevant financial disclosures N/A N/A
Olivia Gunn Planning Committee Member, ASTCT Staff No relevant financial disclosures N/A N/A
Samantha Watters, MPH Planning Committee Member, NMDP Staff No relevant financial disclosures N/A N/A
Shalu Narang, MD Faculty
  1. GBT, Genentech
  2. Genentech
  1. Non-CE Consulting
  2. Contract Research
The following financial relationships have been mitigated:
  • GBT and Genentech non-CE consulting relationships ended in May 2023
Genentech contract research relationship ended in November 2022

Disclosure of Unlabeled Use:

TFF, NMDP, and ASTCT require CME faculty (speakers) to disclose when products or procedures being discussed are off label, unlabeled, experimental, and/or investigational, and any limitations on the information that is presented, such as data that are preliminary, or that represent ongoing research, interim analyses, and/or unsupported opinion. Faculty in this activity may discuss information about pharmaceutical agents that is outside of US Food and Drug Administration approved labeling. This information is intended solely for continuing medical education and is not intended to promote off-label use of these medications. TFF, NMDP, and ASTCT do not recommend the use of any agent outside of the labeled indications. If you have questions, contact the Medical Affairs Department of the manufacturer for the most recent prescribing information.


TFF, NMDP, and ASTCT present this information for educational purposes only. The content is provided solely by faculty who have been selected because of recognized expertise in their field. Participants have the professional responsibility to ensure that products are prescribed and used appropriately on the basis of their own clinical judgment and accepted standards of care. The France Foundation, NMDP, ASTCT and the commercial supporter(s) assume no liability for the information herein.

Copyright Information

Copyright © 2023 The France Foundation. Any unauthorized use of any materials on the site may violate copyright, trademark, and other laws. You may view, copy, and download information or software ("Materials") found on the Site subject to the following terms, conditions, and exceptions:

  • The materials are to be used solely for personal, noncommercial, informational and educational purposes. The materials are not to be modified. They are to be distributed in the format provided with the source clearly identified. The copyright information or other proprietary notices may not be removed, changed, or altered.
  • Materials may not be published, uploaded, posted, transmitted (other than as set forth herein), without The France Foundation's prior written permission.

Privacy Policy

The France Foundation protects the privacy of personal and other information regarding participants and educational collaborators. The France Foundation will not release personally identifiable information to a third party without the individual's consent, except such information as is required for reporting purposes to the ACCME.
The France Foundation maintains physical, electronic, and procedural safeguards that comply with federal regulations to protect against the loss, misuse or alteration of information that we have collected from you.

Additional information regarding The France Foundation’s Privacy Policy can be viewed at http://www.francefoundation.com/privacy.

If you have questions about this CME activity, please contact The France Foundation at (860) 434-1650 or info@francefoundation.com.

Access on-demand webinar


CME Webinar: Increasing Access to HCT for Sickle Cell Disease: Connecting Transplant Centers and Sickle Cell Programs

CME Webinars: ASTCT & NMDP Sickle Cell Disease Webinar Series