[Rinshō ketsueki] The Japanese journal of clinical hematology
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Immune thrombocytopenia (ITP) may occur following a viral infection. We report the case of a 30-year-old woman with thrombocytopenia who was subsequently diagnosed with ITP. ⋯ She responded well to 400 mg/kg of intravenous immunoglobulin therapy. Coronavirus disease of 2019 or COVID-19 should be considered as a cause of ITP during the pandemic, and chest CT scans and RT-PCR tests should be performed in patients suspected of ITP.
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Thrombotic thrombocytopenic purpura (TTP) is a life-threatening disease in which platelets are consumed and thrombotic microangiopathy develops in multiple organs due to a severe deficiency of the metalloproteinase, ADAMTS13. TTP should be suspected in any case associated with thrombocytopenia and hemolytic anemia; TTP can be diagnosed in cases of profound reduction in ADAMTS13 activity (to <10% of the normal level). ⋯ Rituximab was recently approved in Japan for use in refractory or relapsing TTP. Likewise, caplacizumab, an anti-von Willebrand factor, may contribute to disease control and overall survival by preventing ongoing thrombosis and acute end-organ damage.
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Hepcidin is a key molecule that regulates iron metabolism in the body. Iron refractory iron deficiency anemia (IRIDA) is a genetic disorder caused by a defect in the TMPRSS6 gene encoding matriptase-2, a transmembrane serine protease that physiologically inhibits hepcidin production. In patients with IRIDA, the iron uptake in the intestine is remarkably reduced, and iron deficiency anemia (IDA) develops. ⋯ Due to the malabsorption of iron in the intestine, IRIDA is refractory to oral iron supplementation, but partially responds to parenteral iron administration. A high hepcidin level gives IRIDA a lot of similarities with anemia of chronic disease, and a differential diagnosis between the two disorders needs careful inspection. Diagnosis of IRIDA needs genetic testing that is hardly available in most facilities, and therefore its clinical features are not fully understood.
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Graft-versus-host-disease (GvHD) is a major complication and leading cause of non-relapse mortality after allogeneic hematopoietic stem cell transplantation. Corticosteroids remain the standard initial therapy for GvHD; however, patients frequently become steroid-refractory (SR) or remain steroid dependent. Cytokine inhibition appears to be a potential option; however, blockade of any single cytokine may not be sufficient probably because of the redundant effects of multiple cytokines. ⋯ A prompt and sustained ruxolitinib response contributes to the steroid-sparing effect; however, accumulating evidence showed that ruxolitinib exerts substantial myelosuppression and immunosuppressive activity in patients with myelofibrosis (MF). Additionally, serious adverse events following discontinuation of ruxolitinib treatment, characterized by acute relapse of the disease and/or GvHD, have been recognized. Herein we discuss the advantages and disadvantages of ruxolitinib as treatment for GvHD in patients with MF.
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Although immune thrombocytopenia (ITP) and thrombotic thrombocytopenic purpura (TTP) appear similar, their symptoms differ. The number of domestic patients diagnosed with ITP and TTP annually has been estimated to be around 24,000 and 400, respectively. Moreover, no major differences in the incidence rate, age of onset, and prognosis have been observed between Europe, the United States (US), and Japan. ⋯ Meanwhile, TTP was designated as an intractable disease in Japan in 2015, and the first clinical practice guidelines were published in 2017. A single-arm study involving rituximab was conducted on high-risk patients in whom treatment with five plasma exchanges was ineffective or ADAMTS13 inhibitor was >2 BU/ml. Approval for the new indication of rituximab for acquired TTP is expected in 2019.