Describe the manegement in all patients.
Patient education and genetic counseling.
Screening tests for relatives
Describe the treatment of thalassemia minor.
Usually no treatment required
Episodic folic acid supplementation may be indicated (e.g., during pregnancy, acute infections)
Describe the treatment for thalassemia major and intermedia.
Transfusion therapy (erythrocyte concentrates)
Indication (for transfusion-dependent thalassemias): Hb < 7 g/dL or marked clinical symptoms
Target: Hb > 9–10 g/dL
Surveillance and treatment of complications
Iron overload diseases: chelating agents, e.g., deferasirox, indicated when iron accumulation reaches toxic levels
Other complications: e.g., gallstones, asplenia, extramedullary hematopoietic pseudotumors
What can be performed in selected patients?
Splenectomy
Potentially curative treatment
Stem cell transplantation: allogenic HSCT
Gene therapy
Zuletzt geändertvor 2 Jahren