This review summarises and discusses the methodology and findings of a retrospective French registry study conducted by Loschi et al.1 that compared paroxysmal nocturnal haemoglobinuria (PNH) patients treated with eculizumab with historical control patients not treated with eculizumab. The study is notable for being the first comparative investigation to support a significant association of eculizumab with improved overall survival and fewer thrombotic events in patients with PNH, without increasing the risk of clonal evolution.
Independent commentary has been provided by Dr D’Rozario who is currently Senior Staff Specialist and Clinical Director of Haematology at the Canberra Region Cancer Service, Canberra Hospital, Australia. He is also Clinical Lead for the hospital’s Stem Cell Transplant program and Director of the Clinical Trials Unit.
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