Table 1.

Current limitations of thalassemia care

Limitation	Potential solutions
Regular transfusion appointments	Reduce transfusion requirement Stem cell transplantation
Iron overload	Reduce transfusion requirement Block intestinal iron absorption Escalate chelation
Iron chelator toxicity	Reduce transfusion requirement Block intestinal iron absorption Additional chelation options
Lack of allogeneic stem cell donor	Genetically modified autologous transplantation Nontransplant therapies that preclude desirability of transplant