Thalassaemia
Thalassaemia is an inherited disorder of reduced alpha- or beta-globin synthesis causing ineffective erythropoiesis, haemolysis and microcytic anaemia; severity ranges from an asymptomatic carrier state to transfusion-dependent disease with lifelong risk from iron overload.
Definition
Thalassaemia is a group of inherited haemoglobin disorders in which alpha- or beta-globin synthesis is reduced or absent. The resulting globin-chain imbalance causes ineffective erythropoiesis, haemolysis and microcytic hypochromic anaemia, with severity ranging from an asymptomatic carrier state to transfusion-dependent disease and iron-mediated organ injury.
First principles
Globin-chain imbalance causes both ineffective erythropoiesis and haemolysis
Reduced production of one globin chain leaves its partner in relative excess. Unpaired chains damage developing erythroid cells in the marrow and mature red cells in the circulation, so the anaemia reflects both ineffective erythropoiesis and haemolysis rather than simple iron deficiency.
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