[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.
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Unmet needs of current hemophilia A treatment include the requirement for frequent intravenous infusions, inhibitor development, and containment of high medical costs. In order to overcome these issues, we produced FVIII which mimics a bispecific antibody against FIXa/FX. ACE910 demonstrated hemostatic effects on both ongoing and spontaneous joint bleeding in the primate acquired hemophilia A model. ⋯ There were no severe ACE910 related adverse events. Furthermore, bleeding was remarkably decreased by weekly subcutaneous administration in patients with severe hemophilia A, regardless of whether an inhibitor was used. ACE910 has the remarkable advantages of prophylactic efficacy which can be achieved by convenient subcutaneous administrations at a markedly reduced frequency.
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Case Reports
Successful switching from eltrombopag to romiplostim in a pediatric patient with refractory chronic ITP.
Herein, we report a successful treatment experience with romiplostim in a child with immune thrombocytopenia (ITP) refractory to eltrombopag. A 9-year-old female developed chronic ITP, which was refractory to standard treatments, including intravenous immunoglobulin, cepharanthine and splenectomy, and she thus became dependent on prednisolone (PSL). ⋯ Platelet counts increased and PSL could thus be terminated. Our case suggests TPO-RA alteration to potentially be effective for chronic refractory ITP.
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Recombinant human thrombomodulin (rTM) improves the blood coagulation disorder characteristic of disseminated intravascular coagulation (DIC) as well as, or even better than, other anti-DIC drugs. On post-marketing surveillance, its effectiveness has been recognized for hematologic disorders, sepsis and solid tumor subgroups. However, the effect on hemophagocytic syndrome (HPS) complicated by DIC remains unclear. ⋯ Although being refractory to medical management was suspected in our cases, clinical status rapidly came under control including not only amelioration of the blood coagulation disorder but also inflammatory reactions, such as serum ferritin and lactic acid dehydrogenase abnormalities, which represent HPS activity. These observations suggest that rTM might exert marked synergistic effects on HPS with DIC. Given the results obtained in these three cases, administration of rTM appears to offer a promising method of treating HPS complicated by DIC.