Expert review of hematology
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Sticky platelet syndrome (SPS) is a prothrombotic thrombocytopathy with familial occurrence, characterized by hyperaggregability of platelets in response to adenosine diphosphate (ADP), epinephrine (EPI) or both. The syndrome has been identified in approximately 21% of unexplained arterial thrombotic episodes, regarded to be the most common thrombophilia in arterial thrombosis and 13.2% of unexplained venous thromboembolism (VTE). ⋯ As the various localizations of the thrombosis in SPS have been reported, its management often requires a multidisciplinary approach. This review deals with the clinical aspects of thrombophilia, its etiopathogenesis, diagnosis as well as novel advances in the treatment and outlines the challenges for the further research.
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Checkpoint inhibitors with monoclonal antibodies targeting the CTLA-4 or PD-1 axis have revolutionized treatment in some solid tumors, especially melanoma and lung. The role of the CTLA-4 and PD-1 pathways and their inhibition in lymphoma may be different compared to solid tumors. In heavily pretreated Hodgkin lymphoma, PD-1-directed treatment has led to high remission rates. ⋯ Clinical evidence of CAR T-cell treatment in B-cell malignancies is limited to small series, because of the dedicated resources needed. However, impressive response rates have been observed, but toxicities associated with cytokine release can be very severe and fatal. We herein review the background, early clinical evidence, and future perspectives of T-cell-directed immune manipulation for lymphomas including checkpoint inhibitors and CAR T-cell therapies.
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Andexanet alfa is a specific reversal agent for Factor Xa inhibitors. The molecule is a recombinant protein analog of factor Xa that binds to Factor Xa inhibitors and antithrombin:LMWH complex but does not trigger prothrombotic activity. In ex vivo, animal, and volunteer human studies, andexanet alfa (AnXa) was able to dose-dependently reverse Factor Xa inhibition and restore thrombin generation for the duration of drug administration. Further trials are underway to examine its safety and efficacy in the population of patients experiencing FXa inhibitor-related bleeding.
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Iron chelating agents - deferoxamine (DFO), deferiprone (DFP), and deferasirox (DFX) - are used to treat chronic iron overload in patients with β-thalassemia in an attempt to reduce morbidity and mortality related to siderosis. Each of the approved iron chelating agents has its own advantages over the others and also has its own risks, whether related to over-chelation or not. In this review, we briefly discuss the methods to monitor the efficacy of iron chelation therapy (ICT) and the evidence behind the use of each iron chelating agent. We also portray the risks and complications associated with each iron chelating agent and recommend strategies to manage adverse events.
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Optimal trauma care is the neglected disease of the modern era. Hemorrhage is the largest cause of potentially preventable death, and most of these patients present to the hospital with abnormal coagulation profiles. ⋯ Several encouraging studies have been recently completed that are helping to highlight these issues. High-quality studies, combining mechanistic approaches and clinical outcomes will improve our understanding of trauma-induced coagulopathy and decrease trauma related mortality.