Seminars in hematology
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Seminars in hematology · Jul 2018
ReviewC3-mediated extravascular hemolysis in PNH on eculizumab: Mechanism and clinical implications.
The introduction of eculizumab, a human monoclonal antibody against the C5 component of complement, has changed radically the management of paroxysmal nocturnal hemoglobinuria (PNH). The blockade of the terminal complement pathway by eculizumab abrogates intravascular hemolysis, reduces the transfusion requirement and the risk of thrombosis in most of hemolytic PNH patients. ⋯ The treatment of the few PNH patients in which this de novo extravascular hemolysis become clinically relevant is still unsatisfactory. Nevertheless, the investigations of the mechanisms responsible of the extravascular hemolysis on eculizumab have resulted in the development of novel strategies for complement blockade that could overcome this condition.
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Seminars in hematology · Oct 2017
ReviewThe use and impact of Twitter at medical conferences: Best practices and Twitter etiquette.
The use of social media, and in particular, Twitter, for professional use among healthcare providers is rapidly increasing across the world. One medical subspecialty that is leading the integration of this new platform for communication into daily practice and for information dissemination to the general public is the field of hematology/oncology. ⋯ Hematologists/oncologists are engaging regularly in one of the most common forms of social media, Twitter, during major medical conferences, for purposes of debate, discussion, and real-time evaluation of the data being presented. As interest has grown in this area, this article aims to review the new norms, practices, and impact of using Twitter at the time of medical conferences, and also explores some of the barriers and pitfalls that users are encountering in this emerging field.
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Seminars in hematology · Oct 2017
ReviewDisease-specific hashtags and the creation of Twitter medical communities in hematology and oncology.
Twitter is being increasingly used for information gathering and dissemination of ideas in both medical practice and scientific research. A major limitation to its use has been the surplus of available information and difficulty in categorizing that information into topics of individual interest. ⋯ As new disease-specific hashtags are created for hematologic and oncologic diseases, more users can connect across the world, even for the rarest of cancer subtypes. A major challenge for this emerging application will be the development of specific and easily identifiable hashtags over time to add more clarity, while still trying to grow Twitter users and expand its reach.
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Seminars in hematology · Apr 2017
ReviewClassical inherited bone marrow failure syndromes with high risk for myelodysplastic syndrome and acute myelogenous leukemia.
The inherited marrow failure syndromes (IBMFS) are a heterogeneous group of diseases characterized by failure in the production of one or more blood lineage. The clinical manifestations of the IBMFS vary according to the type and number of blood cell lines involved, including different combinations of anemia, leukopenia, and thrombocytopenia. In some IBMFS, systemic non-hematologic manifestations, including congenital malformations, mucocutaneous abnormalities, developmental delay, and other medical complications, may be present. ⋯ Patients with SDS have pancreatic insufficiency, neutropenia, as well as MDS and AML risks. Patients with severe congenital neutropenia (SCN), caused by pathogenic variants in genes essential in myeloid development, have profound neutropenia and high risk of MDS and AML. Herein we review the genetic causes, clinical features, diagnostic modalities, predisposition to malignancies with focus on leukemogenic markers whenever available, and approaches to treatments of the classical IBMFS: FA, DC, SDS, DBA, and SCN.
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Seminars in hematology · Jul 2015
ReviewEpigenetic Therapy in Acute Myeloid Leukemia: Current and Future Directions.
Epigenetic modifications affect gene expression without changes in the actual DNA sequence. Two of the most important mechanisms include DNA methylation and histone tail modifications (especially acetylation and methylation). Epigenetic modulation is a part of normal physiologic development; its dysregulation is an important mechanism of pathogenesis of some cancers, including acute myeloid leukemia (AML). ⋯ Currently, no epigenetic therapy is approved for the treatment of AML, although two DNA methyltransferase inhibitors (azacitidine and decitabine) are commonly used in clinical practice. Among the other epigenetic modifiers undergoing research in AML, the histone deacetylase inhibitors are the most studied. Other promising drugs, such as inhibitors of histone methylation (eg, EZH2 and DOT1L inhibitors), inhibitors of histone demethylases (eg, LSD1 inhibitors), inhibitors of bromodomain-containing epigenetic "reader" BET proteins, and inhibitors of mutant isocitrate dehydrogenases, are at early stages of clinical evaluation.