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