Paroxysmal Nocturnal HemoglobinuriaUrgent

Young adult with dark morning urine, fatigue and abdominal pain; DAT-negative haemolysis, low counts; new hepatic vein thrombosis on ultrasound

• Abdominal pain with ascites or hepatomegaly (Budd-Chiari)
• Headache with focal signs (cerebral venous thrombosis)
• Fever on a complement inhibitor
• Rapidly falling Hb

• Autoimmune haemolysis (DAT positive)
• Aplastic anaemia / MDS
• TTP/HUS
• Antiphospholipid syndrome

• Admit: Haemolytic crisis with symptomatic anaemia, any thrombosis, fever on a complement inhibitor, severe cytopenias
• ICU: Budd-Chiari with liver failure, cerebral venous thrombosis with ↓GCS, sepsis
• Transfuse: Symptomatic anaemia

• Think PNH in haemolysis + thrombosis, haemolysis + cytopenias, or thrombosis in unusual sites (hepatic, portal, cerebral) in a young person.
• Complement inhibitors cut thrombosis and transfusion needs dramatically; access in India is limited — refer early to haematology.
• Iron may be needed (urinary loss) and can briefly increase haemolysis.

• Start eculizumab or ravulizumab without meningococcal vaccination or antibiotic cover
• Ignore a fever in a patient on a complement inhibitor
• Stop anticoagulation after a PNH thrombosis without haematology advice

• Not sending flow cytometry in DAT-negative haemolysis
• Missing thrombosis behind abdominal pain
• Not vaccinating before complement inhibition

Ward; ICU for major thrombosis. Long-term haematology/PNH clinic; anticoagulation after thrombosis.