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CME: The spectrum of alpha-thalassemia: comprehensive care of an orphan disease

Activity Description / Statement of Need:

In this online, self-learning activity:

Thalassemias belong to a group of recessively inherited blood disorders characterized by little or no hemoglobin production and chronic anemia of varying severity. Alpha-thalassemia (AT) is most commonly found in people of Mediterranean, Middle Eastern, Asian, and North African descent. Worldwide, 5% of people are AT carriers, with a much higher prevalence in certain regions (eg, up to 23% in Southeast Asia). AT is typically caused by deletions of one or more α-globin genes, of which there are 4 in total, leading to reduced or abolished α-globin production; nondeletional forms of AT can also occur and are generally more severe. The loss of functional α-globin disrupts the globin chain equilibrium, leading to excess γ- and β-globin chain formation and causing ineffective erythropoiesis.

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 AT.

Commercial Support Disclosure: This program is supported by an educational grant from Agios Pharmaceuticals.

This activity is free of charge.

Release Date: June 01, 2023 -- Expiration Date: June 01, 2025

Faculty: Zahra Pakbaz, MD


Introduction, faculty disclosures

Review of alpha-thalassemia (AT)

  • Epidemiology of AT

o    Risk factors

o    Geographic and heritage influences

  • Genetics and pathophysiology of AT

o    ɑ-globin genes

o    Ineffective erythropoiesis

o    Differences between alpha- and beta-thalassemias

  • Recognition and diagnosis of AT

o    Screening programs and recommendations

o    Clinical presentations of AT

-  Genotype-phenotype correlations

-  Hemolytic anemia

-  Splenomegaly

-  Growth delays

o    Recommended testing

-  Genetic testing

-  Laboratory evaluation

Treatment of patients with AT

  • Conventional treatment approaches

    o   Supportive care
    o   Transfusion
    o   Splenectomy
    o   Iron chelation
  • Non-transfusion-dependent vs transfusion-dependent AT
  • Emerging treatment options

    o   Pyruvate kinase activators
    o   Phase 2 trial data
    o   Ongoing phase 2 and 3 trials
  • Special populations: pregnancy, perioperative care, and pediatric-to-adult transitions in care
  • Challenges in care: identifying and addressing adherence issues, transfusion burden, and care transitions

Patient case(s)

Summary, conclusions, and best practices recap

Learning Objectives

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

  • Evaluate the genetic and pathophysiologic causes of alpha-thalassemia, with a focus on targets of emerging therapies.
  • Describe the challenges clinicians are faced with regarding the recognition, diagnosis, and management of patients with alpha-thalassemia.
  • Discuss the latest safety and efficacy data regarding both conventional and emerging treatment options for patients with alpha-thalassemia, including the implications for ongoing untreated low hemoglobin levels.
  • Develop a plan for treating patients with non-transfusion-dependent and transfusion-dependent alpha-thalassemia.


ACCME Activity #202527762

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 1.0 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. It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting ABIM MOC credit.

ABIM MOC Credit Type: Medical Knowledge

Physicians: For maintenance of certification (MOC) points, 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 points for this activity. By providing this data, you acknowledge that it will be shared with ACCME and the applicable certifying board. Please note: Not all activities on this site provide MOC points. If this activity does not specify that it provides MOC points in this section, then it does NOT provide MOC points. This activity provides MOC points only for ABIM.


ScientiaCME is accredited by the Accreditation Council for Pharmacy Education (ACPE) as a provider of continuing pharmacy education. This activity is approved for 1.0 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 #0574-0000-23-023-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 by ScientiaCME.

Disclosure of Faculty: Zahra Pakbaz, MD, HS Associate Clinical Professor, Division of Hematology/Oncology, Department of Medicine, UCI Health, has received financial compensation from consulting and/or speakers bureau and/or research grants from Novartis, Amgen, Sanofi, Global Blood Therapeutics, Pfizer, Dova, Sobi, TerumoBCT, Vertex, Agios, Forma, and NovoNordisk.

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 an educational grant from Agios 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.

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