This site is intended for healthcare professionals

Go to /sign-in page

You can view 5 more pages before signing in

Paroxysmal nocturnal haemoglobinuria

Last reviewed dd mmm yyyy. Last edited dd mmm yyyy

Authoring team

Paroxysmal nocturnal haemoglobinuria is an acquired clonal, benign, haematopoietic stem cell disorder which results in the formation of defective red cells, white cells and platelets. It is characterised by intravascular haemolysis and frequent venous thrombosis or bleeding.

It is caused by a mutation in an X-linked gene involved in the formation of membrane phosphatidyl inositol anchors. The membranes of these cells are deficient in those proteins normally anchored to the cell through a phosphatidyl inositol linkage, including complement deactivating factors.

PNH is a very rare disorder and should be suspected in confusing cases of haemolytic anaemia.

About 4 decades ago 10-year survival for this condition was only 50 percent

  • however, in the last 15 years, advances in treatment such as the development of eculizumab have improved survival to more than 75 percent (1)


  • Schrezenmeier H, Muus P, Socié G, Szer J, Urbano-Ispizua A, Maciejewski JP, Brodsky RA, Bessler M, Kanakura Y, Rosse W, Khursigara G, Bedrosian C, Hillmen P. Baseline characteristics and disease burden in patients in the International Paroxysmal Nocturnal Hemoglobinuria Registry. Haematologica. 2014 May;99(5):922-9.

Related pages

Create an account to add page annotations

Add information to this page that would be handy to have on hand during a consultation, such as a web address or phone number. This information will always be displayed when you visit this page