About Beta-Thalassemia
Beta-thalassemia is an inherited blood disorder caused by mutations in the HBB gene that reduce or eliminate production of the beta-globin chain of hemoglobin, leading to chronic hemolytic anemia. The severity ranges from thalassemia minor (trait, typically asymptomatic) to thalassemia intermedia and thalassemia major (Cooley anemia), which requires lifelong transfusions. Without treatment, thalassemia major causes progressive organ damage from iron overload and severe anemia.
Common Clinical Features
Clinical Trial Eligibility Tips
What to know before applying to Beta-Thalassemia trials.
Transfusion-dependent patients (TDT) and non-transfusion-dependent patients (NTDT) are often enrolled in separate trial arms — clarify your transfusion history and frequency before screening.
Many gene therapy and gene editing trials require patients to have no matched sibling donor and adequate organ function; obtain recent ferritin, liver iron concentration (LIC), and cardiac T2* MRI results.
Prior splenectomy status and alloantibody burden from transfusions may affect eligibility — gather your full transfusion and surgical history.
Patient Resources
Find recruiting Beta-Thalassemia trials
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