Richard J Kelly, Matthew Holt, Jenniner Vidler, Louise M Arnold, Joanna Large, Briony Forrest, Catherine Barnfield, Alexandra Pike, Morag Griffin, Talha Munir, Petra Muus, Sateesh Nagumantry, Abraham Mullasseril Varghese, John R Davies, Roochi Trikha, Austin G Kulasekararaj, Lindsay Mitchell, Shreyans A Gandhi
Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal hematopoietic disorder which occurs on a background of bone marrow failure (BMF). In PNH, chronic intravascular hemolysis causes an increase in morbidity and mortality, mainly due to thromboses. Over the last 20 years treatment of PNH has focused on the complement protein C5 to prevent intravascular hemolysis using the monoclonal antibody eculizumab and more recently ravulizumab. In the UK, all patients are under review at one of two reference centers...
December 24, 2023: Blood