[Rinshō ketsueki] The Japanese journal of clinical hematology
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Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hemolytic anemia, which generally affects women of childbearing age. PNH hemolysis increases the risk of complications such as thrombosis during pregnancy and the postpartum period. ⋯ Eculizumab, a C5 complement inhibitor, is a potential therapeutic option for such patients. This review presents the current strategies for the management of pregnant women with PNH, the cases experienced in our hospital, and the recently proposed remedy guide regarding the management of pregnancy in cases with PNH by the Pregnancy Working Group of The Japan PNH Interest group.
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This review summarizes the presentation entitled "Genetic variants in C5 and poor response to eculizumab" (N Engl J Med. 2014; 370: 632-639), given at Symposium 3 entitled "Basic and clinical topics on red blood cell membrane", during the 76th Annual Meeting of the Japanese Society of Hematology. The molecular basis for the poor response to eculizumab in Japanese patients is unclear. Of 345 Japanese patients with PNH who received eculizumab, 11 showed a poor response. ⋯ In vitro hemolysis due to non-mutant and mutant C5 was completely blocked by N19-8, a monoclonal antibody that binds to a different site on C5 than does eculizumab. The functional capacity of the C5 polymorphism p. Arg885His, together with its failure to undergo blockade by eculizumab, accounts for the poor response to this agent of patients who carry this mutation.