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CME: From alpha to optimal: contemporary strategies for managing alpha thalassemia and its complications

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 (e.g., up to 23% in Southeast Asia). AT is typically caused by deletions of one or more α-globin genes, leading to reduced or abolished α-globin production; non-deletional 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. Patients with both deletional and non-deletional types of AT can develop various clinical complications, such as iron overload, gallstones, impaired liver function, osteoporosis, and elevated uric acid levels. Cardiopulmonary and skeletal deformities are common in patients from countries in the Western hemisphere who have elevated ferritin, while infections are the leading complication and cause of death in patients who live in countries in the Eastern hemisphere and have transfusion-dependent thalassemia.

Target Audience:

HCPs including but not limited to: hematologists; physician assistants, nurse practitioners, and nurses who practice in hematology; and any other healthcare professionals with an interest in or who may clinically encounter patients with Alpha thalassemia.


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

This activity is free of charge.


Release Date: February 10, 2026 -- Expiration Date: February 10, 2028

Faculty: Ashutosh Lal, MD

Agenda

Introduction, faculty disclosures

Overview of Alpha thalassemia (AT) 

  • Epidemiology and burden of AT for patients and caregivers

o   Transfusion dependence: economic and clinical implications

o   Morbidity and mortality with anemia

  • Pathophysiology of AT
  • AT classification

o   Genotype and phenotype

o   Severity

o   Transfusion dependence

  • Diagnosis and screening

o   Techniques to accurately diagnose AT

o   Exclusion of other potential diagnoses

·  Overall health burden

Management of AT 

  • Non-transfusion-dependent AT: Supportive care
  • Transfusion-dependent AT
      • Standard-of-care options to safely reduce transfusion burden
      • Indications for transfusion, selection of patients
      • Preventing iron overload using iron chelation therapy
  • Novel and investigational therapies
      • Allogenic hematopoietic cell transplant, candidates for therapy
      • Pyruvate kinase activators
      • Gene therapy
  • AT monitoring
      • Hemoglobin levels
      • Patient-reported outcomes
      • Complications and comorbidities
  • Patient case(s)

Summary, conclusions, and best practice recap

Learning Objectives

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

  • Review the burden of Alpha thalassemia (AT), including the burden of both frequent and intermittent transfusions as well as the economic and clinical constraints on patients and families
  • Evaluate screening recommendations and procedures useful in the differential diagnosis of AT in children and adults
  • Review standard-of-care treatment options for AT, and describe the unmet needs in this patient population, including the morbidity and mortality associated with untreated anemia
  • Describe the latest safety and efficacy data supporting the use of novel therapeutic treatment strategies, and select treatment options on the basis of disease subtype and severity and patient characteristics

Accreditation

ACCME Activity #203381497

ScientiaCME is accredited by the Accreditation Council for Continuing Medical Education (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.

CME-MOC_badge

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.

Nurses: This activity is designated for up to 1.0 AMA PRA Category 1 Credit(s)™, and the American Nurses Credentialing Center (ANCC) accepts AMA PRA Category 1 Credit(s)™ to meet the continuing education renewal requirements of nursing re-certification for APRNs and RN specialty. Some state nursing boards accept AMA PRA Category 1 Credit(s)™ for re-licensure requirements, some do not. Check your state board of nursing's CE requirements before applying credit from this course to your re-licensure.

Physician Assistants: The American Academy of Physician Assistants (AAPA) accepts AMA PRA Category 1 Credit™ assigned by organizations accredited by the ACCME as satisfying Category 1 CME for National Commission on Certification of Physician Assistants (NCCPA) national certification maintenance. This activity is designated for up to 1.0 AMA PRA Category 1 Credit(s)™.

Nurse Practitioners: The American Academy of Nurse Practitioners Certification Board (AANPCB) states that continuing education providers accredited by the ACCME may provide acceptable, accredited Advanced Practice Provider content. This activity is designated for up to 1.0 AMA PRA Category 1 Credit(s)™.


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.

Disclosures of Faculty: Ashutosh Lal, MD, Professor, Pediatrics, UCSF School of Medicine, has received financial compensation from Hemex Health as a stockholder and consulting services; in addition to grant/research support from Agios Pharmaceuticals, Novo Nordisk, Bristol Myers Squibb, and Pharmacosmos.

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

Faculty WILL discuss off-label uses of a commercial product. 

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. 

Instructions

  • 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

PC
Windows 7 or above
Internet Explorer 8
*Adobe Acrobat Reader
MAC
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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