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

CME: Beta thalassemia: best practices and novel approaches in its recognition and treatment is organized by ScientiaCME.

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

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, Middle Eastern, Central Asian, Indian, East Asian, and 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 prevents cells from producing any beta-globin at all (termed beta-zero [B0]). The clinical severity of the disease depends on the extent of and 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.

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