About Alpha-Thalassemia
Alpha-thalassemia results from deletions or mutations affecting one to four alpha-globin genes on chromosome 16, disrupting hemoglobin alpha-chain synthesis and causing a spectrum of disease from silent carrier state to hydrops fetalis. Hemoglobin H disease (three-gene deletion) causes moderate to severe hemolytic anemia, while four-gene deletion (Hb Bart hydrops fetalis) is typically fatal without in utero intervention. The condition is most prevalent in Southeast Asia, sub-Saharan Africa, and the Mediterranean basin.
Common Clinical Features
Clinical Trial Eligibility Tips
What to know before applying to Alpha-Thalassemia trials.
Specify your genotype precisely (number of deleted alpha-globin genes and any non-deletion mutations) as trials distinguish between HbH disease, Hb Bart, and carrier states.
Transfusion dependency status and splenectomy history are key eligibility criteria for emerging gene therapy trials targeting alpha-thalassemia.
Newborn screening results and hemoglobin electrophoresis or HPLC records help confirm diagnosis for registry enrollment and trial screening.
Patient Resources
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