Hazzaa Alzahrani

Nov 13, 2024 | onlinelibrary.wiley.com | Alexander Roth |Rong Fu |Guangsheng He |Hazzaa Alzahrani

1 Introduction Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, acquired, clonal haematological disease caused by a somatic mutation in the phosphatidylinositol glycan class A gene in haematopoietic stem cells [1-3]. In patients who do not receive adequate treatment, PNH is life-threatening; the 5-year mortality rate was approximately 35% between 1950 and 1995, prior to the development of modern targeted therapies [4].