An increased understanding of the molecular and pathogenic factors that govern the thalassemia pathophysiology support the development of new therapeutic approaches to thalassemia care.
Dr. Ali Taher will summarize recent advances in iron overload and iron chelation. Several modalities are currently available for the diagnosis and monitoring of iron overload in patients with transfusion-dependent thalassemia (TDT) and non-transfusion-dependent thalassemia (NTDT), allowing a better understanding of the timing of accumulation in different organs and at different rates. Dr. Taher will provide recommendations on how to tailor the chelation regimen to a single patient. New oral iron chelator formulation will be discussed in addition to new modalities to remove the excess of body iron.
Dr. Antonio Piga will focus on the impact of bone disease and pain on the quality of life of thalassemia patients, and he will provide recommendations for prevention and treatment of early bone loss. Considering the long-term survival of thalassemia patients, all the new strategies for treating osteopenia and osteoporosis, as well as the underlying causes, must be evaluated.
Dr. Maria Domenica Cappellini will discuss the new therapeutic targets in TDT and NTDT that are in pre-clinical and clinical experimental phases and that mainly address ineffective erythropoiesis. These therapeutic targets include the activin receptor ligand trap molecules and JAK2 inhibitors. Gene therapy and genome editing will also be discussed.
Maria Domenica Cappellini
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Associate, International Associate, Student, and Resident Members