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CME: Beta thalassemia: best practices and novel approaches in its recognition and treatment

Activity Description / Statement of Need:

In this online, self-learning activity:

Beta-thalassemia (BT) is a progressive, hereditary, microcytic, hypochromic form of anemia characterized by the reduced synthesis of hemoglobin subunit beta and the underproduction of hemoglobin A (HbA). Although there are limited data regarding the incidence of BT, historical estimates of BT have been placed at about 1 in 100,000 individuals. BT is most commonly found in patients with Mediterranean, the Middle Eastern, Central Asian, Indian, East Asian, and the North African heritage. BT is caused by a point mutation in the encoding gene for hemoglobin subunit beta (HBB) on chromosome 11, which either results in lower beta-globin production (termed beta-plus [B+] or prevent cells from producing any beta-globin at all (termed beta-zero [B0]). The clinical severity the disease depends on the extent of β and γ chain imbalance; more than 350 genetic alterations that can cause BT have been identified.

There is a demonstrated variation in treatment between providers who practice at a beta thalassemia center of excellence (CoE) and those who do not, and those practitioners at CoEs demonstrate greater familiarity with beta thalassemia and its therapies, including butyrates, gene therapy, and luspatercept. Additional areas of educational need include transitioning patients from pediatric care, management of complications, and clinical trial updates.

Target Audience:

The following healthcare professionals: hematologists; physician assistants, nurse practitioners, and pharmacists who practice in hematology; and any other healthcare professionals with an interest in or who may clinically encounter patients with BT.

Commercial Support Disclosure: This program is supported by educational grants from Agios and Bluebird Pharmaceuticals.

This activity is free of charge.

Release Date: November 06, 2021 -- Expiration Date: November 06, 2023

Faculty: Cheryl Mensah, MD


Introduction, faculty disclosures

Review of BT

·       Epidemiology of and risk factors for BT

·       Review of pathophysiology

·       Challenges in recognition and diagnosis

Treatment of patients with BT

  • Non-transfusion-dependent vs transfusion-dependent
  • Supportive care and present therapy
  • Addressing the need for iron chelation therapy
  • Recently approved treatment options, including TGF-beta superfamily protein inhibitors
  • Management of complications
  • Special populations: pregnancy, peri-operative care, pediatric-to-adult transitions in care
  • Challenges in care: identifying and addressing adherence issues, transitioning from pediatric to adult care

Investigational and emerging treatments

  • Treatment options in development: gene therapy, pyruvate kinase stimulation, JAK2 regulation

Patient case(s)

Summary, conclusions, and best practice recap

Learning Objectives

By the end of the session the participant will be able to:

  • Recall the challenges associated with the recognition, diagnosis, and treatment of BT
  • Summarize the safety and effectiveness of conventional and emerging treatment options for BT
  • Formulate a treatment plan for a patient with BT and related complications


ACCME Activity #201800389

This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education through ScientiaCME. ScientiaCME is accredited by the ACCME to provide continuing medical education for physicians.

Credit Designation: ScientiaCME designates this educational activity for a maximum of 1.00 AMA PRA Category 1 Credit(s)™ toward the AMA Physician's Recognition Award. Physicians should only claim credit commensurate with the extent of their participation in the activity.


ABIM MOC Recognition Statement: Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 1.0 MOC points in the American Board of Internal Medicine's (ABIM) Maintenance of Certification (MOC) program. Participants will earn MOC points equivalent to the amount of CME credits claimed for the activity. It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting ABIM MOC credit.

PhysiciansFor maintenance of certification (MOC) credit, you must enter your board certification ID # and birth date correctly. It is the learner's responsibility to provide this information completely and accurately at the completion of the activity. Without providing it, the learner will NOT receive MOC credit for this activity. Please note: Not all activities on this site provide MOC credit. If this activity does not specify that it provides MOC credit in this section, then it does NOT provide MOC credit.


ScientiaCME is accredited by the Accreditation Council for Pharmacy Education (ACPE) as a provider of continuing pharmacy education. This activity is approved for 1.00 contact hours (0.1 CEUs) of continuing pharmacy education credit. Proof of participation will be posted to your NABP CPE profile within 4 to 6 weeks to participants who have successfully completed the post-test. Participants must participate in the entire presentation and complete the course evaluation to receive continuing pharmacy education credit. ACPE #0574-0000-21-048-H01-P.  This is an Application (A)-type activity. 

PharmacistsYou must enter your NABP # and birth date correctly so that proof of participation can be posted to your NABP CPE profile. It is the learner's responsibility to provide this information completely and accurately at the completion of the activity. Without providing it, the learner will NOT receive CPE credit for this activity.

Nurse Practitioners (NPs): The American Academy of Nurse Practitioners accepts AMA PRA Category 1 Credit(s)™ from organizations accredited by the ACCME.  ScientiaCME will provide NPs who successfully complete each activity with a certificate of participation indicating that the activity was designated for AMA PRA Category 1 Credit(s)™.

Physician Assistants: The American Academy of Physician Assistants accepts AMA PRA Category 1 Credit(s)™ from organizations accredited by the ACCME.

Faculty Disclosure and Resolution of COI

As a provider of continuing medical education, it is the policy of ScientiaCME to ensure balance, independence, objectivity, and scientific rigor in all of its educational activities. In accordance with this policy, faculty and educational planners must disclose any significant relationships with commercial interests whose products or devices may be mentioned in faculty presentations, and any relationships with the commercial supporter of the activity. The intent of this disclosure is to provide the intended audience with information on which they can make their own judgments. Additionally, in the event a conflict of interest (COI) does exist, it is the policy of ScientiaCME to ensure that the COI is resolved in order to ensure the integrity of the CME activity. For this CME activity, any COI has been resolved thru content review ScientiaCME.

Faculty Disclosure:

Cheryl Mensah, M.D.., Ph.D., Assistant Professor, Division of Hematology and Oncology,        Department of Medicine, Weil Cornell School of Medicine, has received financial compensation from Chiesi for consulting work, and is an Adjudication Committee Member for Vertex Pharmaceuticals.

Disclosures of Educational Planners: Charles Turck, PharmD, BCPS, BCCCP, President of ScientiaCME, has no relevant financial disclosures.

Faculty will not discuss off-label uses.

All relevant financial relationships have been mitigated.

ScientiaCME adheres to the ACCME’s Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of a CE activity, including faculty, planners, reviewers or others are required to disclose all relevant financial relationships with ineligible entities (commercial interests). All relevant conflicts of interest have been mitigated prior to the commencement of the activity.

Commercial Support Disclosure: This program is supported by educational grants from Agios and Bluebird Pharmaceuticals.


  • Read the learning objectives above
  • Take the Pre-Test (optional). Completion of the pre-test will help us evaluate the knowledge gained by participating in this CME activity.
  • View the online activity. You may view this is in more than one session, and may pause or repeat any portion of the presentation if you need to.
  • Minimum participation threshold: Take the post-test. A score of 70% or higher is required to pass and proceed to the activity evaluation.
  • Complete the activity evaluation and CME registration. A CE certificate will be emailed to you immediately.

Cultural/Linguistic Competence & Health Disparities

System Requirements

Windows 7 or above
Internet Explorer 8
*Adobe Acrobat Reader
Mac OS 10.2.8
Safari or Chrome or Firefox
*Adobe Acrobat Reader
Internet Explorer is not supported on the Macintosh

*Required to view Printable PDF Version

Perform Pre-Test (optional)

Please take a few minutes to participate in the optional pre-test. It will help us measure the knowledge gained by participating in this activity.