Exploring Curative Therapies for Sickle Cell Disease: When to Consider HCT or Gene Therapy
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Background:
This webinar will focus on recent research in the use of hematopoietic cell transplant (HCT) and gene therapy as curative therapies for sickle cell disease (SCD), convening a panel of experts to 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.
Description:
During this interactive roundtable-style webinar, our expert HCT, gene therapy, and SCD panelists will 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. Panelists will 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 will be relevant for both transplant centers and community hematologists/oncologists and SCD program providers who refer out for transplant and cellular therapies, discussing the role that all practitioners can play in improving access to curative treatment options.
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
Accreditation Statement: In support of improving patient care, this activity has been planned and implemented by The France Foundation, National Marrow Donor Program, and 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.
TARGET AUDIENCE
This educational activity is intended for physicians, nurses, and other members of the health care team who care for patients who may benefit from hematopoietic stem cell transplantation.
PROGRAM OVERVIEW
This webinar will focus on recent research in the use of hematopoietic cell transplant (HCT) and gene therapy as curative therapies for sickle cell disease (SCD), convening a panel of experts to 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 interactive roundtable-style webinar, our expert HCT, gene therapy, and SCD panelists will 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. Panelists will 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 will be relevant for both transplant centers and community hematologists/oncologists and SCD program providers who refer out for transplant and cellular therapies, discussing the role that all practitioners can play in improving access to curative treatment options.
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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
NMDP/Be The Match
Elizabeth Stenger, MD, MSc
Blood and Marrow Transplant Physician
Aflac Cancer & Blood Disorders Center
Children’s Healthcare of Atlanta/Emory University
Haydar Frangoul, MD Pediatric Hematology/Oncology and Transplant Physician Program Director Sarah Cannon Pediatric Hematology/Oncology and Cellular Therapy
TriStar Centennial
Shalu Narang, MD
Pediatric Hematologist/Oncologist Associate Clinical Professor, Rutgers and Robert Wood Johnson Medical School
Section Chief, Benign Hematology
Associate Director, Pediatric Hematology/Oncology Fellowship, Newark Beth Israel Medical Center and Children’s Hospital of New Jersey
Anna Cincotta, FNP
Senior Clinical Operations Partner
National Marrow Donor Program (NMDP)/ Be The Match
Karla Dawson, MPH
Health Equity Professional
National Marrow Donor Program (NMDP)/ Be The Match
Olivia Gunn
Membership and Operations Senior Associate
American Society for Transplantation and Cellular Therapy
Amber Ruffin, MPH
Associate Program Manager, Health Equity
National Marrow Donor Program (NMDP)/ Be The Match
Samantha Watters, MPH
Research and Education Lead
Community Physician Engagement
National Marrow Donor Program (NMDP)/ Be The Match
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, attending the activity, and completing the evaluation form.
Credit fulfillment
- If you are requesting AMA credits or a certificate of participation, your certificate will be available for download.
TECHNICAL REQUIREMENTS
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ACCREDITED PROVIDER This activity is jointly provided by The France Foundation, National Marrow Donor Program, and American Society for Transplantation and Cellular Therapy (ASTCT).
In support of improving patient care, this activity has been planned and implemented by The France Foundation, National Marrow Donor Program, and American Society for Transplantation and Cellular Therapy. 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 Commercial Support, 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 interest. 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, and ASTCT seek to 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 are 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 Role 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/ Be The Match Staff | No relevant financial disclosures | NA | NA |
Anna Cincotta, FNP | Planning Committee Member, Content Reviewer | No relevant financial disclosures | NA | NA |
Ashley Smeaton, MHA | TFF Staff | No relevant financial disclosures | NA | NA |
Elizabeth Stenger, MD, MSc | Faculty | 1. Editas Medicine, Bluebird Bio Inc.
| 1. Contract Research | The following relationship was mitigated:
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 Pharmaceuticals | 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 | NA | NA |
Heather Tarbox, MPH | TFF Staff | No relevant financial disclosures | NA | NA |
Karla Dawson, MPH | Planning Committee Member, NMDP/ Be The Match Staff | No relevant financial disclosures | NA | NA |
Olivia Gunn | Planning Committee Member, ASTCT Staff | No relevant financial disclosures | NA | NA |
Samantha Watters, MPH | Planning Committee Member, NMDP/ Be The Match Staff | No relevant financial disclosures | NA | NA |
Shalu Narang, MD | Faculty | 1. GBT, Genentech
2. Genentech | 1. Non-CE Consulting
2. Contract Research | The following financial relationships have been mitigated:
|
Disclosure of Unlabeled Use:
TFF, NMDP/ Be The Match, 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/ Be The Match, 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.
MEDIA DISCLOSURE
Your registration, attendance at, and/or participation in this meeting constitutes consent to being photographed, videotaped, or recorded during the meeting by The France Foundation, its educational collaborators, or anyone authorized on behalf of these organizations. You further authorize, without any compensation paid to you, the use of any photographs, videos, or recordings that contain your likeness, image, or voice in any educational, informational, commercial, or promotional materials produced and/or distributed by The France Foundation, its educational collaborators, or anyone authorized by these organizations, as well as on any internet sites maintained by any of these entities. Images/recordings used for these purposes will not be sold, and no personal information about you or the subject (other than event location and date) will be included in the production of any material.
Disclaimer
TFF, NMDP/ Be The Match, 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/ Be The Match, ASTCT and the commercial supporter(s) assume no liability for the information herein.
CONTACT INFORMATION
If you have questions about this CME activity, please contact The France Foundation at 860-434-1650 or info@francefoundation.com.
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