Complications

Complication
Timeframe
Likelihood
short term
low

Develops in 2% to 5% of patients with PNH, usually the acute myelogenous type.[18] The diagnosis is made by inspection of the blood or bone marrow biopsy. This normally does not respond well to usual treatment.

long term
medium

About 10% of patients with PNH have hepatic vein thrombosis. It is especially likely during pregnancy or with severe haemoglobinuric episodes. It is best diagnosed by computed tomography or flow Doppler of the hepatic veins.[61] It is treated with thrombolysis (if recent), anticoagulation (low-molecular-weight heparin by preference), and eculizumab, which seems to prevent its occurrence.[61]

Budd-Chiari syndrome

long term
medium

Other thromboses occur with high frequency in PNH. They are diagnosed and treated in much the same way as the Budd-Chiari syndrome: thrombolysis (if recent), anticoagulation (low-molecular-weight heparin by preference), and eculizumab, which seems to prevent its occurrence.[61]

long term
medium

Probably the second most common type of thrombosis (after abdominal events).[20]

Venous sinus thrombosis is more common. Arterial events also occur. Magnetic resonance imaging and magnetic resonance angiography are the best methods for diagnosis. Treatment is mainly supportive. There is no demonstrated benefit for anti-oedema and anticoagulation therapy.[62]

Cavernous sinus thrombosis

long term
medium

Probably 40% of patients have significant renal dysfunction, and 5% to 15% die of renal failure.[18] The cause of death is presumably damage from haemoglobinuria. Treatment, once renal failure has occurred, is as for any other renal failure (dietary changes, phosphate-lowering measures, renal dialysis, etc). Eculizumab may prevent renal failure.

Chronic kidney disease

long term
low

The bone marrow may ultimately fail in PNH, becoming like that in aplastic anaemia. It is diagnosed by biopsy. Treatment is with antithymocyte globulin and ciclosporin (cyclosporine) with eltrombopag, or with stem cell transplantation.[55][56][57][58]

Aplastic anaemia

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