Clinical studies

• Guidelines for management of the thrombophilia of PNH based on prospective randomized studies

  • Treatment

    • Does treatment of hemolysis with complement inhibitors modify the thrombophilia of PNH?

  • Prophylaxis

• Guidelines for bone marrow and stem cell transplantation in the era of therapeutic complement inhibitors such as eculizumab

• Pregnancy and PNH

  • Maternal and fetal risks

  • Management

• Ethnic and geographic differences

  • Are different management guidelines necessary?

• Natural history of subclinical PNH (PNH-sc)

Basic studies

• What is the mechanism of the thrombophilia of PNH?

  • What is the basis of the disproportionate involvement of hepatic, mesenteric, cerebral, and dermal veins?

• What is the basis of clonal selection?

  • What is the relationship between PNH and aplastic anemia and PNH refractory anemia-MDS?

  • How is PNH-sc different from PNH/aplastic anemia and classic PNH?

• What is the basis of clonal dominance?