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Expert Review of Hematology

Elena Campello, Luca Spiezia, Angelo Adamo, Paolo Simioni
Fifty-three years after the first description of an inherited prothrombotic condition (antithrombin deficiency), our knowledge on hereditary and acquired causes of hypercoagulability that can predispose carriers to venous thromboembolism (VTE) has greatly improved. Areas covered: Main causes of hereditary thrombophilia are summarized alongside new prothrombotic mutations recently discovered. The main causes of acquired thrombophilia, and namely, antiphospholipid antibody syndrome and hyperhomocysteinemia, are also discussed together with other common acquired prothrombotic states characterized by an increase of procoagulant factors and/or a decrease of natural anticoagulants...
February 18, 2019: Expert Review of Hematology
Foluso Joy Ogunsile, Rakhi Naik, Sophie Lanzkron
Venous thromboembolism (VTE) is a common comorbid condition found in sickle cell disease (SCD) and is associated with increased mortality for adults with SCD. The pathophysiology that leads to the thrombophilic state in SCD has been previously reviewed; however, evidence-based guidelines to aid in diagnosis, prevention and management of VTE are lacking. Areas covered: This review article will cover the pathophysiology underlying the hypercoagulable state, the epidemiology of VTE, and management strategies of VTE in SCD...
February 16, 2019: Expert Review of Hematology
Iuliana Vaxman, Morie Gertz
The survival of multiple myeloma patients is increasing due to new medications, the widespread implementation of autologous stem cell transplantation and better supportive treatments. The controversy surrounding post-transplant treatment is debated due to a lack of large randomized trials comparing the different treatment modalities. The questions for each proposed treatment are whether it improves outcomes, has low cumulative toxicities and is easy to administer. Areas covered: In this review, we have summarized the current data on maintenance therapy in newly diagnosed MM patients undergoing ASCT, focusing on bortezomib, thalidomide and lenalidomide as well as newer agents Expert opinion: Maintenance treatment has been shown to deepen and prolong responses and increase PFS and OS...
January 29, 2019: Expert Review of Hematology
Richard F Pollock, Gordon Muduma
Objectives The efficacy of oral iron in treating iron deficiency anemia (IDA) can be limited by poor gastrointestinal (GI) absorption and adverse GI symptoms; intravenous (IV) iron is a well-established alternative. The present study compared the efficacy of two IV iron formulations in patients with IDA: iron isomaltoside (IIM) and ferric carboxymaltose (FCM). Methods A systematic literature review (SLR) was conducted to identify randomized controlled trials (RCTs) of IIM and FCM in patients with IDA. An adjusted indirect treatment comparison (ITC) of IIM and FCM was then conducted via iron sucrose to evaluate differences in change from baseline hemoglobin and the proportion of patients achieving a clinically-relevant response...
January 28, 2019: Expert Review of Hematology
Hanne Skaadel, Øystein Bruserud
Even though thrombotic events are rare in patients with coagulation deficiencies, several cases of both arterial and venous thromboses have been reported in patients with single coagulation factor defects and platelet defects. Thromboses have been described both in hemophilia A and B, von Willebrand disease as well as in many other rare congenital coagulation factor and platelet defects. Thromboses may also occur in patients with acquired hemophilia and in patients with severe thrombocytopenia due to hematological malignancies or intensive chemotherapy...
January 28, 2019: Expert Review of Hematology
Adalgisa Condoluci, Davide Rossi
Several recurrently deregulated pathways implicated in the development of chronic lymphocytic leukemia (CLL) have been described over the last decades. Knowledge of the CLL genetic heterogeneity led to the definition of molecular biomarkers informing about prognosis and treatment outcome. Areas covered: English literature published from January 2008 through December 2018 was searched in PubMed, Cochrane Central Register of Controlled Trials, and hematology meeting abstracts to obtain literature on clinical predictive factors for CLL...
January 28, 2019: Expert Review of Hematology
Vanessa E Kennedy, Lori S Muffly
Allogeneic hematopoietic cell transplantation (allo-HCT) has seen marked growth among older adults, where chronological age is no longer a barrier to transplant. As allo-HCT expands to older and potentially less fit individuals, prognosticating transplant outcomes in this population remains an ongoing need. Areas Covered: This review summarizes pre-transplant assessment tools in optimizing patient selection and predicting transplant outcomes in older adults, including comorbidity indices, psychosocial assessment, geriatric assessment, serum biomarkers, and disease risk...
January 11, 2019: Expert Review of Hematology
Leland Metheny, Marcos de Lima
Allogeneic hematopoietic cell transplantation is frequently used to treat malignant and non-malignant conditions, and many patients lack a human leukocyte antigen (HLA) matched related or unrelated donor. For those patients, available alternative graft sources include HLA mismatched unrelated donors, cord blood, or haplo-identical donors. These graft sources have unique characteristics and associated outcomes requiring graft-specific variations to conditioning regimens, graft-versus-host disease prophylaxis, and post-transplant care...
December 24, 2018: Expert Review of Hematology
Christine Biron-Andreani, Jean-François Schved
Hemophilia A and B are X-linked recessive disorders caused by the deficiency of factor VIII or factor IX, respectively. Bleeding episodes are treated with factor replacement therapy. The most serious complication of this treatment is the development of inhibitors. In such patients, bypassing agents, such as activated recombinant human factor VII (rhFVIIa) or plasma-derived activated prothrombin complex concentrates, are administered to prevent or treat bleeding episodes. The high cost of the current bypassing agents limits their availability in emerging countries...
December 21, 2018: Expert Review of Hematology
Massimo Breccia, Robin Foà
The outcome of acute promyelocytic leukemia (APL) has drastically improved following the identification of the PML-RARA oncogene as a key player in the pathogenesis of APL, and the subsequent introduction of all-trans retinoic acid (ATRA) as a therapeutic agent. Areas covered: Randomized trials have recently demonstrated the efficacy of arsenic trioxide (ATO) in combination with ATRA for the front-line treatment of standard and medium risk APL patients. This chemotherapy-free combination is associated with a decreased cumulative rate of relapse, prolonged overall survival and reduced early death rate...
December 20, 2018: Expert Review of Hematology
Carrie van der Weyden, Michael Dickinson, James Whisstock, H Miles Prince
Brentuximab vedotin is an antibody-drug conjugate, which combines a CD30 monoclonal antibody with the microtubule disrupting agent monomethylauristatin E. The utility of brentuximab vedotin has been explored in a number of diseases, with a recent focus on T-cell lymphoma, particularly systemic anaplastic large-cell lymphoma (sALCL) and cutaneous T-cell lymphoma (CTCL), as well as other peripheral T-cell lymphoma (PTCL) histologies. Areas covered: This review surveys current data on the efficacy of brentuximab vedotin in T-cell lymphoma, as well as embedding it in a therapeutic context by reviewing potential competitor agents in the clinic...
December 10, 2018: Expert Review of Hematology
Parameswaran Hari, Marlo Blazer, Aditya Raju, Eileen Farrelly, Richard Labotka, Tomas Skacel, Dorothy Romanus
BACKGROUND: Triplets with immunomodulators (IMIDs) and proteasome inhibitors (PIs) improve overall survival (OS) in trials of newly diagnosed multiple myeloma (NDMM) patients although reported outcomes in routine practice are lacking. Authors compared outcomes in NDMM patients in the USA by use of triplet vs doublet frontline therapy (FT). METHODS: This is a retrospective electronic health record database study of NDMM patients without transplant in FT between 1/1/2008 and 6/30/2017...
December 4, 2018: Expert Review of Hematology
Paolo Gresele, Loredana Bury, Anna Maria Mezzasoma, Emanuela Falcinelli
Hemorrhagic diseases associated with platelet dysfunction include inherited platelet function disorders (IPFD) and a large number of non-hereditary conditions, defined as acquired platelet function disorders (APFD). Their identification requires a careful clinical evaluation and a rational use of diagnostic laboratory assays. Areas covered: Here we describe the laboratory techniques currently available for the assessment of platelet function, including new and experimental laboratory assays, and their alterations in platelet function disorders...
January 2019: Expert Review of Hematology
Gerrit J Schuurhuis, Gert J Ossenkoppele, Angѐle Kelder, Jacqueline Cloos
Measurable Residual Disease (MRD) in acute myeloid leukemia (AML) is a rapidly evolving area with many institutes embarking on it, both in academical and pharmaceutical settings. However, there is a multitude of approaches to design, perform and report flow cytometric MRD. Together with the long-term experience needed, this makes flow cytometric MRD in AML non-standardized and time-consuming. Areas covered: This paper briefly summarizes critical issues, like sample preparation and transport, markers and fluorochromes of choice, but in particular focuses on the main issues, which includes specificity and sensitivity, hereby providing a new model that may circumvent the main disadvantages of the present approaches...
November 23, 2018: Expert Review of Hematology
Johnny Mahlangu
rFVIIIFC was the first extended half-life product to complete the phase 3 development program and be registered. It was developed to reduce the high treatment burden imposed by prophylaxis. It is now one of four extended half-life products available for a variety of indications in hemophilia A. This article focus on the efficacy use of rFVIIIFC in the prevention of bleeds in hemophilia A. Areas covered: This article provides an update on efficacy data from three clinical studies describing the use of rFVIIIFC in the treatment and prevention of bleeds in hemophilia A...
November 19, 2018: Expert Review of Hematology
Hanna Gran Sestøl, Sine Munch Trangbæk, James B Bussel, Henrik Frederiksen
Immune thrombocytopenia (ITP) and the associated biologic and psychological effects can lead to reduced health-related quality of life (HRQoL). Areas covered: This review focuses on the impact of ITP itself and its treatment on patients' HRQoL. The findings show that the HRQoL is uniformly reduced amongst ITP patients, both with and without interventions. ITP significantly affects many aspects of patient HRQoL, which is comparable to that of patients with other chronic diseases. However, there is a lack of longitudinal studies among unselected ITP populations and the majority of studies focus on statistical differences between groups or between measures at different time points rather than quality of life (QoL) changes that are perceived as clinically relevant in patients...
November 16, 2018: Expert Review of Hematology
Michael Caspers, Marc Maegele, Matthias Fröhlich
Despite advances in the treatment of severely injured patients that have resulted in overall improved outcomes, uncontrolled hemorrhage still represents the most common cause of preventable death following major injury. While addressing both endo- and exogenous factors that lead to an acute trauma-induced coagulopathy, massive transfusion plays a key role in managing bleeding trauma patients. However, the best practice for hemostatic control including massive transfusion in these patients is still under debate...
November 15, 2018: Expert Review of Hematology
Iuliana Vaxman, Hasib Sidiqi, Morie Gertz
Treatment of multiple myeloma in the relapsed setting remains challenging, despite recent impressive advances in the management of these patients. Venetoclax (ABT-199) is a BCL-2 inhibitor recently approved by the US food and drug administration (FDA) for treatment of chronic lymphocytic leukemia but the drug has shown activity in a number of hematological malignancies. Venetoclax has broadened the treatment options for patients with relapsed or refractory multiple myeloma. Approximately 20% of myeloma patients will exhibit t (11;14) associated with high Bcl-2 expression making venetoclax an attractive therapeutic option...
November 14, 2018: Expert Review of Hematology
Fabio Efficace, Francesco Cottone
No abstract text is available yet for this article.
November 9, 2018: Expert Review of Hematology
Dimitrios C Ziogas, Meletios A Dimopoulos, Efstathios Kastritis
Multiple myeloma (MM) is characterized by notable inter-patient and intra-clonal heterogeneity that is gradually decoded over the last decade. Despite the deeper and better understanding of its biology and the development of novel therapeutic strategies that have prolonged overall survival, MM still retains a poor prognosis in patient subgroups with certain high-risk features. Areas covered: This article summarizes currently identified features that stratified patients in high-risk myeloma with impaired prognosis and discusses available therapeutic options that may partially overcome the impact of these adverse factors in patients' outcome...
November 2018: Expert Review of Hematology
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