Overcoming Treatment Challenges in Beta Thalassemia: Novel Therapeutic Targets and Transition Preparedness

Overcoming Treatment Challenges in Beta Thalassemia: Novel Therapeutic Targets and Transition Preparedness

On-Demand Webcast

James B. Bussel, MD
Professor Emeritus of Pediatrics
Weill Cornell Medicine
New York, New York

Sujit Sheth, MD
Weill Cornell Medicine
New York, New York

Alexis A. Thompson, MD, MPH
Hematology Section Head
Ann & Robert H. Lurie Children’s Hospital of Chicago
Northwestern University Feinberg School of Medicine
Chicago, Illinois


Provided by North American Center for Continuing Medical Education, LLC (NACCME), an HMP Company.

For questions regarding this educational activity, please call 609-371-1137 or email info@naccme.com.


This educational activity is specifically designed for hematologists/oncologists, physician assistants, nurse practitioners, nurses, and pharmacists who specialize in hematology, as well as other healthcare providers who care for patients with hematologic malignancies.


Beta thalassemia is an inherited blood disorder that causes mild to severe anemia. Genetic mutations of the beta-globin gene are responsible for the disorder and are very heterogeneous at the molecular level. Traditional treatments for beta thalassemia have major limitations and hematopoietic stem cell transplant (HSCT) is the only cure available to patients with a matched donor. A better understanding of molecular geneticin beta thalassemia has led to the development of novel treatments. These treatments include novel therapeutic targets and molecular therapies that have the potential to optimize outcomes for beta thalassemia patients. With significant improvements in treatment options for beta thalassemia, previously considered a fatal pediatric disease, patient lifespan now extends well into adulthood. Hematologists and the multidisciplinary care team may not be aware of the clinical and psychosocial challenges associated with transitioning pediatric patients to adult care. The beta thalassemia care team should be educated on effective transition and disease monitoring practices as well as the availability of novel treatments that may increase quality of life for patients transitioning to adult care.

This webcast aims to fill the gaps surrounding the treatment of beta-thalassemia using novel therapeutics.


After participating in this activity, learners should be better able to:

  • Demonstrate the application of molecular genetics in the development of novel therapeutic targets for beta thalassemia patients
  • Identify strategies for transitioning pediatric patients to adult care considering clinical and psychosocial challenges as well as the introduction of adult therapies unavailable to pediatric patients

To be eligible for credit, participants must complete the online activity and the evaluation. Upon completing the activity, there will be instructions on how to complete the evaluation and print a certificate or other documentation of credit.

Release Date: November 16, 2020
Expiration Date: November 16, 2021
Estimated Time to Complete: 1.50 hours

There is no fee associated with this activity.


Supported by an educational grant from: Bristol Myers Squibb


 In support of improving patient care, NACCME 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.


NACCME designates this internet enduring activity for a maximum of 1.50 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.


American Academy of Nurse Practitioners National Certification Program accepts AMA PRA Category 1 Credit from organizations accredited by the ACCME.


This continuing nursing education activity awards 1.50 contact hours.

Provider approved by the California Board of Registered Nursing, Provider #13255 for 1.50 contact hours. 


This internet enduring, knowledge-based activity (Universal Activity Number: JA0006201-000-20-201-H01-P) has been approved for a maximum of 1.50 contact hours (.150 CEUs).

The certificate is not the official record of your participation in the activity. The official record of credit will be the information in the CPE Monitor system. Following ACPE Policy, NACCME must transmit your claim to CPE Monitor within 60 days from the date you complete this CPE activity. NACCME is not able to report your claimed credit after this 60-day period. Eligibility for pharmacy credit is contingent upon the successful completion of a post-test and/or evaluation for each activity or session attended.


NACCME has been authorized by the American Academy of PAs (AAPA) to award AAPA Category 1 CME credit for activities planned in accordance with the AAPA CME Criteria. This activity is designated for 1.50 AAPA Category 1 credit. PAs should only claim credit commensurate with the extent of their participation.


This continuing medical education activity may include device or medicine brand names for participant clarity purposes only, due to the presence of different branded versions of the same product. No product promotion or recommendation should be inferred.


This activity may contain information about experimental and other uses of drugs or devices that are not currently approved by the European Medicines Agency (EMA) of the European Union or the Food and Drug Administration (FDA) of the United States. Participants are strongly encouraged to consult approved product labeling for any drug or device mentioned in this activity before use. The opinions expressed during this activity are the opinions of the respective authors, presenters or moderators and do not necessarily reflect the opinions of NACCME.


The material presented and related discussions are not intended to be medical advice, and the presentation or discussion of such material is not intended to create and does not establish a physician-patient relationship. Medical advice of any nature should be sought from an individual’s own physician.

Neither NACCME nor any of its subsidiaries or affiliates is affiliated with, or formally endorsed by a medical society.

The opinions expressed in this educational activity are those of the faculty and are not attributable to NACCME or HMP. Clinical judgment must guide each professional in weighing the benefits of treatment against the risk of toxicity. Dosages, indications, and methods of use for products referred to in this activity are not necessarily the same as indicated in the package insert for each product, may reflect the clinical experience of the presenters, and may be derived from the professional literature or other clinical sources. Consult complete prescribing information before administering.


NACCME, LLC is an independent provider of continuing medical education. NACCME, LLC has no proprietary or financial interest in medical or healthcare products over which the FDA (USA) or EMA (EU) has regulatory authority.

NACCME, LLC assures that all educational materials are reviewed to ensure for the absence of commercial bias, fair balance, scientific objectivity, and levels of evidence. The educational activity will not be influenced by commercial organizations and will not conflict with any other scheduled educational activities.

According to the disclosure policy of NACCME, faculty, editors, managers, and other individuals who are in a position to control content are required to disclose any relevant financial relationships with relevant commercial companies related to this activity. All relevant conflicts of interest that are identified are reviewed for potential conflicts of interest. If a conflict is identified, it is the responsibility of NACCME to initiate a mechanism to resolve the conflict. The existence of these interests or relationships is not viewed as implying bias or decreasing the value of the presentation.

Planning Committee

In addition to the faculty listed below, NACCME planners and staff include Samantha Conforti, Mary Johnson, Randy Robbin, Greaton Sellers, Shannon Stevenson, and Griselda Zuccarino-Catania. 

All planners have disclosed no relevant financial relationships with any commercial interest.


The faculty has reported the following:

James Bussel: Advisory Board-Amgen, Atara, Novartis, Dova, Rigel, UCB, Momenta, Argenx, Rally Bio, Regeneron, Pharming, Principia, ArchOncology, Pfizer, Grifols; Consultant- Amgen, Atara, Novartis, Dova, Rigel, UCB, Momenta, Argenx, Rally Bio, Regeneron, Pharming, Principia, ArchOncology, Pfizer, Grifols; Honoraria-Novartis, 3S Bio, Amgen, Rigel, Grifols

Sujit Sheth: Advisory Board-CRISPR/Vertex; Consultant-Agios, Bluebird Bio, Celgene/Bristol Myers Squibb; Other-Celgene/Bristol Myers Squibb, Dispersol DST

Alexis Thompson: Consultant-Agios Pharm, Beam, Bluebird Bio, Celgene BMS, Global Blood Therapeutics; Grant/Research Support-Baxalta, BioMarin, Bluebird Bio, Celgene BMS, Novartis Pharmaceuticals


North American Center for Continuing Medical Education (NACCME) complies with the legal requirements of the Americans with Disabilities Act and the rules and regulations thereof. If any participant in this educational activity is in need of accommodations, please call 609-371-1137.


NACCME protects the privacy of personal and other information regarding participants, educational partners, and joint providers. NACCME and our joint providers 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 appropriate accrediting agency. NACCME maintains physical, electronic, and procedural safeguards that comply with federal regulations to guard your nonpublic personal information.

Copyright © 2020 by North American Center for Continuing Medical Education, LLC. All rights reserved. No part of this accredited continuing education activity may be reproduced or transmitted in any form or by any means, electronic or mechanical, without first obtaining permission from North American Center for Continuing Medical Education.


Any participant wanting to file a grievance with respect to any aspect of a continuing education activity accredited by NACCME, LLC may contact Samantha Conforti, Manager, Accreditation and Compliance, by phone at 609-371-1137, by email at sconforti@naccme.com or in writing at 104 Windsor Center Drive, East Windsor, NJ 08520. The Manager, Accreditation and Compliance will review the grievance and respond within 30 days of receiving the complaint. If the participant is unsatisfied with the response, an appeal to the Senior Manager/Director or the Executive Vice President, Chris Bolwell, Accreditation and Compliance, can be requested for a second level of review. Mr. Bolwell can be contacted via phone at 678-242-0801, by email at c.bolwell@imedex.com or in writing at 11675 Rainwater Drive, Suite 600 Alpharetta, GA 30009.