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Among the topics our agreed speakers will address are:

  • Pathophysiology of PNH, focusing on biologic aspects of the GPI-defect in PNH and on the mechanism expansion of PNH hematopoiesis, as well as on complement biology

  • Current thoughts on the treatment of PNH, including anti-complement therapies for hemolytic/thrombotic PNH, immunosuppressive treatment for bone marrow failure, as well as bone marrow transplantation

  • Hot Topics in PNH (defining response and endpoints, breakthrough hemolysis: terminal vs proximal inhibitors, proximal inhibitors: monotherapy vs combination treatment)

  • A special session entirely dedicated to recent data coming from clinical trials investigating complement inhibitors in PNH (see below)

  • World-wide impact of PNH, focusing on geographic area-specific unmet clinical needs

  • Ongoing collaborative projects of IPIG: the IPIG PNH International Registry

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