Exploring curative therapies for sickle cell disease
When to consider HCT or gene therapy
Release date: Sept. 6, 2023
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
Faculty
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
NMDP
Karla Dawson, MPH
Health Equity Professional
NMDP
Olivia Gunn
Membership and Operations Senior Associate
American Society for Transplantation and Cellular Therapy
Amber Ruffin, MPH
Associate Program Manager, Health Equity
NMDP
Samantha Watters, MPH
Research and Education Lead, Community Physician Engagement
NMDP
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
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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
Physicians
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 |
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The following relationship was mitigated:
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| Haydar Frangoul, MD | Faculty |
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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 |
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The following financial relationships have been mitigated:
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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.
Disclaimer
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RESOURCES
CME Webinars: ASTCT & NMDP Sickle Cell Disease Webinar Series