Expert review of hematology
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Sickle cell anemia is one of the most common genetic blood disorders worldwide. Individuals with sickle cell disease (SCD) experience clinical manifestations such as chronic anemia, developmental delay, vaso-occlusive pain, acute chest syndrome, and neurological complications. ⋯ This review focuses on these challenges with particular emphasis on delayed menarche and vaso-occlusive pain episodes associated with menstruation, in addition to the evaluation and initial management of heavy menstrual bleeding for adolescents with SCD. We highlight research opportunities in this neglected area to help enhance the comprehensive care model for this population.
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Since affecting hemostasis, all the anticoagulant drugs carry a risk of bleeding. Minor bleeds may be managed without the need to reverse the anticoagulant effect, which is instead a key step to ensure efficacious hemostasis in major and life-threatening bleeding. Drug withdrawal applies to all anticoagulants. ⋯ For vitamin K antagonists-induced major bleeding, rapid reversal with prothrombin complex concentrates (PCC) or plasma and intravenous vitamin K to confer lasting correction are recommended. PCC, activated PCC and rFVIIa are suggested for major bleeding related to new direct oral anticoagulants (DOAC), despite proper studies are lacking. Premarketing studies are ongoing on new antidotes (idarucizumab, andexanet, aripazine), which appear to be suitable for the treatment of DOAC-induced life-threatening hemorrhage.
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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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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.