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.