Paroxysmal Nocturnal Hemoglobinuria

21 March 2019

Paroxysmal Nocturnal Hemoglobinuria (PNH) hit the news at the end of 2018 when the US Food and Drug Administration approved Ultomiris (ravulizumab-cwvz; Alexion) as a treatment for this very rare blood condition.1 This is ground-breaking news for a condition that is known to affect only one to nine people per million worldwide.2

What is paroxysmal nocturnal hemoglobinuria?

PNH is an acquired disorder that is not inherited but appears after birth.2 It is a blood disorder caused by specific mutations in a small number of body cells (somatic mutation).3 More specifically, the disorder has been traced back to a mutation in the PIG-A gene in ‘hematopoietic stem cells,’ which are stem cells in the bone marrow that give rise to red blood cells. This mutation results in the production of red blood cells that lack important proteins like CD55 and CD59. Cells that are produced without these essential proteins eventually lyse (die) and this lysis occurs in patients with PNH at such a rate that it leads to anaemia.3

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