International journal of hematology
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The genetic modification of autologous T cells with chimeric antigen receptors (CARs) represents a breakthrough for gene engineering as a cancer therapy for hematologic malignancies. By targeting the CD19 antigen, we have demonstrated robust and rapid anti-leukemia activity in patients with heavily pre-treated and chemotherapy-refractory B cell acute lymphoblastic leukemia (B-ALL). We demonstrated rapid induction of deep molecular remissions in adults, which has been recently confirmed in a case report involving a child with B-ALL. ⋯ We review the clinical trial experience targeting B-ALL and CLL and speculate on the possible reasons for the different outcomes and propose potential optimization to CAR T cell therapy when targeting CLL or other indolent NHL. Lastly, we discuss the pre-clinical development and potential for clinical translation for using CAR T cells against multiple myeloma and acute myeloid leukemia. We highlight the potential risks and benefits by targeting these poor outcome hematologic malignancies.
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Beta (β)-thalassemia is characterized by a hypercoagulable state and an increased risk of thrombosis, which can result in significant morbidity and mortality. The coagulation pattern and determinants of thrombosis in patients with β-thalassemia remain largely unknown. The aim of this study was to evaluate the whole blood thromboelastometry (TEM) profile of β-thalassemic children by ROTEM(®). ⋯ Of the patients with β-thalassemia, MCF was higher and clot formation time was shorter in splenectomized subjects than in non-splenectomized subjects on EXTEM and INTEM (p = 0.026, p = 0.002, p < 0.001, p < 0.001, respectively). TEM profiles in β-thalassemic children were more hypercoagulable compared with controls. Larger prospective studies are needed to evaluate the relevance of the association between ROTEM(®) profile and thromboembolic events in patients with β-thalassemia.