Type: Hematologic
Paroxysmal nocturnal hemoglobinuria
An acquired clonal disorder of complement regulation; terminal complement and proximal C3 inhibitors control haemolysis.
Polycythemia vera
Myeloproliferative neoplasm with erythrocytosis and thrombosis risk.
Thrombotic thrombocytopenic purpura
A thrombotic microangiopathy from ADAMTS13 deficiency; urgent plasma exchange with immunosuppression is the mainstay, with a targeted adjunct.
Sickle Cell Disease
An inherited haemoglobin disorder; disease-modifying drugs and, increasingly, curative gene therapies are available.
Systemic Mastocytosis
A clonal mast-cell disorder spanning indolent to advanced forms; KIT-targeted therapy is available.
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