
Management
The variable clinical expression of the PNH defect makes general guidelines impossible.
- Anaemia - Correct any iron deficiency and give folic acid 5 mg daily. Check vitamin B12 level. Erythropoeitin may improve the bone marrow erythroid response. Blood transfusion may be required and blood should be leucodepleted. Washed red cells are only required if haemolytic transfusions occur despite leucodepletion.
- Thrombosis - High index of suspicion is required. For established thrombosis, heparin and warfarin should be given in the usual way and warfarin may need to be given life long.
- Bone Marrow Failure - This is managed in the same way as severe Aplastic Anaemia, with stem cell transplantation or ATG/Cyclospirin in the first instance. See Guidelines for the diagnosis and management of aplastic anaemia. May 2010.
- Other Treatments - The complement inhibitor, eculizumab has recently shown benefit in PNH with severe haemolysis.This is not currently available in New Zealand.
In view of the rarity of PNH, a global register has been established to study the natural history of this disorder. See www.PNHregistry.org.
Topic Code: 4938