Towards the integration of patient-reported outcomes into the global clinical management of multiple myeloma.
Authors: Niscola P, Scaramucci L, Efficace F PMID: 31317793 [PubMed - as supplied by publisher] (Source: Expert Review of Hematology)
Source: Expert Review of Hematology - July 20, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Molecular genetics in allogeneic blood stem cell transplantation for myelodysplastic syndromes.
Authors: Kobbe G, Schroeder T, Rautenberg C, Kaivers J, Gattermann N, Haas R, Germing U Abstract Introduction: Myelodysplastic Syndromes (MDS) are a heterogeneous group of myeloid neoplasms arising in a multipotent hematopoietic stem cell. In about 50% of cases, chromosomal aberrations are detected, which can serve as clonal markers as well as important prognostic factors. In recent years, many somatic mutations have been recognized to be involved in the initiation and clonal evolution of MDS. They provide prognostic information, not only regarding the natural course of disease but also regarding the outcome of all...
Source: Expert Review of Hematology - July 20, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Management of aplastic anemia after failure of frontline immunosuppression.
Authors: Pierri F, Dufour C Abstract Introduction: About 60% of aplastic anemia (AA) patients are in need of further treatment after front line standard immunosuppressive therapy (IST). This along with the prolonged survival of AA subjects who do not respond to or relapse after this treatment makes management of these patients a rising and very challenging issue. Areas Covered: Literature research, carried out from the most commonly used data bases, included the following key words: aplastic anemia, immunosuppressive treatment, Anti Thymocyte Globuline, Ciclosporine A, refractory aplastic anemia, relapsing aplastic...
Source: Expert Review of Hematology - July 19, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Pharmacokinetic and safety considerations when switching from standard to extended half-life clotting factor concentrates in hemophilia.
Authors: Morfini M, Farrugia A Abstract Introduction: Plenty of new FVIII/IX concentrates have been developed and entered the market of hemophilia treatment. Others are going to end the long/demanding procedures for approval. Changes of the FVIII molecule (single chain), pegylation of B-domain deleted FVIII, and fusion with Fc succeeded to improve the FVIII half-life, about 4 hours. Pegylation and fusion with albumin or Fc of rFIX caused a substantial increase of half-life, approximately 3-4 times that of FIX standard concentrates. Area covered: Extended Half-life concentrates may allow a longer time interval betwe...
Source: Expert Review of Hematology - July 19, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Aggressive Non-Hodgkin Lymphomas: risk factors and treatment of central nervous system recurrence.
Authors: Santambrogio E, Nicolosi M, Vassallo F, Castellino A, Novo M, Chiappella A, Vitolo U Abstract Introduction: Secondary central nervous system lymphoma (SCNSL) is a potentially fatal event in the setting of aggressive Non-Hodgkin Lymphomas. Nowadays, despite of the very poor outcome of SCNSL, several studies are going to identify the "high risk" patients' subgroup that could early develop this detrimental event and in whom the central nervous system (CNS) prophylaxis could improve survival. Areas covered: Herein, the authors will review the prophylactic and treatment strategy for SCNSL, focusing on...
Source: Expert Review of Hematology - July 16, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

T-cell deplete versus T-cell replete haploidentical hematopoietic stem cell transplantation for sickle cell disease: where are we?
Authors: Patel DA, Akinsete AM, Connelly JA, Kassim AA Abstract Introduction: Severe sickle cell disease is associated with progressive end-organ damage and early mortality in adults. While allogeneic hematopoietic cell transplant from a matched related donor is curative, the vast majority of patients do not have a compatible sibling. Accordingly, platforms using haploidentical donors have been developed, which provide near universal availability. Areas covered: This review focuses on the two commonly used approaches for haploidentical hematopoietic transplants, namely T-cell deplete and T-cell replete, each of whi...
Source: Expert Review of Hematology - July 13, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Tranexamic acid for childbirth: why, when, and for whom.
Authors: Sentilhes L, Madar H, Mattuizzi A, Froeliger A, Merlot B, Elleboode B, Deneux-Tharaux C Abstract Introduction: Postpartum hemorrhage (PPH) is a major cause of maternal death and severe maternal morbidity after childbirth. Areas covered: Tranexamic acid, an antifibrinolytic agent, reduces bleeding-related mortality in women with PPH, especially when administered shortly after delivery, and is consequently recommended in this situation (1g intravenously with a second dose of 1 g if bleeding continues), even in high income countries where the magnitude of the effect of tranexamic is uncertain. Expert opinion:...
Source: Expert Review of Hematology - July 13, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Moxetumomab pasudotox for the treatment of relapsed and/or refractory hairy cell leukemia.
Authors: Abou Dalle I, Ravandi F Abstract Introduction: Hairy cell leukemia is a rare indolent B-cell malignancy, characterized by pancytopenia, recurrent infections and splenomegaly. After initial therapy with purine nucleoside analogs, up to 50% of patients relapse after several years of remission. The number of relapsed patients is increasing and, until recently, there was no approved therapy with durable responses for hairy cell leukemia patients in the relapsed setting, thus the need for new non-chemotherapy approach with significant efficacy and less myelosuppression. Areas covered: Moxetumomab pasudotox is a...
Source: Expert Review of Hematology - July 13, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Switching patients in the age of long-acting recombinant products?
Authors: Escobar M, Santagostino E, Mancuso ME, Coppens M, Balasa V, Taylor JA, Iorio A, Negrier C Abstract Introduction: Prophylaxis with factor replacement therapy is the gold standard for the treatment of hemophilia, but this often requires frequent infusions. A number of long-acting factor products have been developed to reduce the burden on patients. Areas covered: This is an overview of information presented at two symposia held at the World Federation of Hemophilia and International Society on Thrombosis and Haemostasis - Scientific and Standardization Committee annual meetings. The pharmacokinetic, safety a...
Source: Expert Review of Hematology - July 10, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Risk factors, management and prevention of transfusion-related acute lung injury: a comprehensive update.
Authors: Kuldanek SA, Kelher M, Silliman CC Abstract Introduction: Despite mitigation strategies that include the exclusion of females from plasma donation or the exclusion of females with a history of pregnancy or known anti-leukocyte antibody, transfusion-related acute lung injury (TRALI) remains a leading cause of transfusion-related morbidity and mortality. Areas covered: The definition of TRALI is discussed and re-aligned with the new Berlin Diagnostic Criteria for the acute respiratory distress syndrome (ARDS). The risk factors associated with TRALI are summarized as are the mitigation strategies to further r...
Source: Expert Review of Hematology - July 10, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Are triplet therapies really living up to their hype as the "standard of care'' for multiple myeloma and what else is needed?
Are triplet therapies really living up to their hype as the "standard of care'' for multiple myeloma and what else is needed? Expert Rev Hematol. 2019 Jul 09;: Authors: Schreder M, Knop S PMID: 31287740 [PubMed - as supplied by publisher] (Source: Expert Review of Hematology)
Source: Expert Review of Hematology - July 10, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

High-dose intravenous immunoglobulin for the treatment and prevention of heparin-induced thrombocytopenia: a review.
Authors: Warkentin TE Abstract Introduction: Heparin-induced thrombocytopenia (HIT) is known for its strong association with thrombosis and distinct pathogenesis involving anti-PF4/polyanion antibodies that activate platelets strongly through clustering of platelet FcγIIa receptors. Autoimmune HIT (aHIT) refers to a subgroup of patients whose HIT antibodies have both heparin-dependent and heparin-independent platelet-activating properties. aHIT patients have atypical clinical presentations including delayed-onset HIT, persisting (refractory) HIT, heparin 'flush' HIT, fondaparinux-associated HIT, severe thromb...
Source: Expert Review of Hematology - July 6, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Optimal management of mantle cell lymphoma in the primary setting.
Authors: Tang C, Kuruvilla J Abstract Introduction: The management of mantle cell lymphoma (MCL) has significantly improved since the use of intensified induction and autologous stem cell transplant consolidation. Evolving developments in minimal residual disease detection and novel agent therapy are now challenging this frontline treatment paradigm. Areas covered: This review discusses both the established role of induction and transplant consolidation in MCL, followed by evolving concepts in the use of novel agents in the frontline setting, as well as use of minimal residual disease as a driver for MCL management...
Source: Expert Review of Hematology - July 5, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Monoclonal antibodies against RANKL and sclerostin for myeloma-related bone disease: can they change the standard of care?
Authors: Kleber M, Ntanasis-Stathopoulos I, Dimopoulos MA, Terpos E Abstract Introduction: Over 80% of patients with multiple myeloma (MM) develop myeloma bone disease (MBD) during the disease course. The clinical consequences include serious skeletal related events (SRE) that impact survival and quality of life. Bisphosphonates are the mainstay in the treatment of MBD. Currently, new therapeutic strategies are being introduced and broaden the therapeutic options in MBD. Areas covered: The purpose of this review is to summarize the current clinical management of MBD, and present novel data regarding monoclonal anti...
Source: Expert Review of Hematology - July 5, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Quality of life outcomes in multiple myeloma patients: a summary of recent clinical trials.
Authors: Martino M, Rossi M, Ferreri A, Loteta B, Morabito A, Moscato T, Console G, Innao V, Naso V, Provenzano PF, Recchia AG, Gentile M Abstract Introduction: Management of multiple myeloma (MM) has improved over recent years. Health-related quality of life (HRQoL) data is becoming increasingly important, owing to improved survival outcomes. Areas covered: The authors performed an expert review of the literature to identify evidence-based data available on HRQoL in frontline and relapsed/refractory MM (RRMM) patients. Expert opinion: De-novo patients should be informed that the HRQoL is expected to improve during...
Source: Expert Review of Hematology - June 30, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Current and emerging treatments for immune thrombocytopenia.
Authors: Dou X, Yang R Abstract Introduction Immune thrombocytopenia (ITP) is an autoimmune disease. Even though there are many treatments available, some patients remain resistant to multiple treatments. Therefore, it is very important to develop new treatment options. Areas covered Here, the authors summarize several current and emerging treatments developed for ITP in recent years. They include a summary of their mechanisms of action and clinical trial results. Expert opinion At present, the first-line treatment of ITP is glucocorticoid and intravenous immunoglobulin (IVIg). Other traditional therapies include s...
Source: Expert Review of Hematology - June 28, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Novel treatments for relapsed/refractory acute myeloid leukemia with FLT3 mutations.
Authors: Tallis E, Borthakur G Abstract Introduction Mutations in the gene encoding for the FMS-like tyrosine kinase 3 (FLT3) are present in about 30% of adults with AML and are associated with shorter disease-free and overall survival after initial therapy. Prognosis of relapsed/refractory AML with FLT3 mutations is even more dismal with median overall survival of a few months only. Areas covered This review will cover current and emerging treatments for relapsed/refractory AML with FLT3 mutations, preclinical rationale and clinical trials with new encouraging data for this particularly challenging population. The...
Source: Expert Review of Hematology - June 26, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

One plus one does not always equal two, especially with regard to hypomethylating agents: The question of synergy of azacitidine and lenalidomide for treatment of relapsed acute myeloid leukemia and myelodysplastic syndromes post allogeneic hematopoietic stem cell transplant.
Authors: Bewersdorf JP, Stahl M, Zeidan AM Abstract Key paper evaluation: Craddock C, et al. Combination Lenalidomide and Azacitidine: A Novel Salvage Therapy in Patients Who Relapse After Allogeneic Stem-Cell Transplantation for Acute Myeloid Leukemia. J Clin Oncol. 2019; 37: 580-8. Allogeneic hematopoietic stem cell transplant (allo-HSCT) is the only potentially curative treatment for acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). However, most patients relapse after allo-HSCT and treatment options are limited. Craddock et al. reported recently in the Journal of Clinical Oncology results from a ...
Source: Expert Review of Hematology - June 23, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Bariatric venous thromboembolism prophylaxis: an update on the literature.
Authors: Hamadi R, Marlow CF, Nassereddine S, Taher A, Finianos A Abstract Introduction: Rates of obesity have been increasing worldwide and with the current situation obesity now represents an epidemic. Bariatric surgery is one the most effective ways to help reduce weight and sustain weight loss. Venous thromboembolism is a major cause of morbidity and mortality among bariatric surgery patients with no clearly established guidelines on prophylaxis. Areas covered: In this review the authors summarize clinical studies evaluating unfractionated heparin (UFH) and low molecular weight heparins (LMWH) in bariatric surg...
Source: Expert Review of Hematology - June 22, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Prevalence of human T-cell lymphotropic virus type-1 infection among blood bonors in mainland China: a systematic review and meta-analysis of the last 20 years.
Conclusion: A disproportionate distribution of HTLV-1 infection among blood donors in different provinces/municipalities regarding its prevalence has been reported. This study recommends developing appropriate HTLV-1 screening strategies of blood donors in various regions of China considering regional prevalence, and cost-effectiveness. PMID: 31220418 [PubMed - as supplied by publisher] (Source: Expert Review of Hematology)
Source: Expert Review of Hematology - June 22, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Thyroid hemodynamic alterations in Egyptian patients with sickle cell disease: relation to disease severity, total body iron and thyroid function.
Conclusion: The authors demonstrated an increased intra-thyroidal RI & PI and a decreased thyroid volume among SCD patients which might be related to impaired thyroidal microcirculation and vasculopathy rather than iron overload. PMID: 31206301 [PubMed - as supplied by publisher] (Source: Expert Review of Hematology)
Source: Expert Review of Hematology - June 19, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Considering the spleen in sickle cell disease.
Authors: El Hoss S, Brousse V Abstract Introduction: In human physiology, the spleen is generally neglected, and its role is considered anecdotal. In sickle cell disease, splenic dysfunction is the main cause of life-threatening complications, particularly in early childhood with the risk of pneumococcal overwhelming sepsis and acute splenic sequestration crisis, notably. During the course of the disease, the spleen functionally declines and anatomically disappears, albeit with great individual variability depending on modulating genetic and environmental factors. Areas covered: The present review aims to provide a...
Source: Expert Review of Hematology - June 16, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Current perspectives of Sickle Cell Disease in Nigeria: Changing the narratives.
Authors: Ojewunmi OO, Adeyemo TA, Ayinde OC, Iwalokun B, Adekile A Abstract Introduction: Sickle cell disease (SCD) is an inherited blood disorder characterized by clinical heterogeneity that may be influenced by environmental factors, ethnicity, race, social and economic factors as well as genetic and epigenetic factors. Areas covered: The present review was carried out to provide a comprehensive assessment of the current burden of SCD and treatments available for persons with SCD in Nigeria with the aim of identifying surveillance and treatment gaps, informing to guide the planning and implementation of better cr...
Source: Expert Review of Hematology - June 16, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

A review of chimeric antigen receptor T-cells in lymphoma.
Authors: Anderson JK, Mehta A Abstract Introduction: Immunotherapy has revolutionized the treatment of cancer. Antibodies, antibody drug conjugates, and bispecific antibodies have improved outcomes in various cancers especially lymphomas. Chimeric antigen receptor T cell (CAR-T) is a step forward in the immunotherapy paradigm for the treatment of Lymphomas. Recently two CAR-T products, Tisagenlecleucel and Axicabtagene ciloleucel, were approved by the US FDA. While it is exciting to have such novel treatment available, the challenges of production, administration, related toxicity, and cost remain. Specific toxicit...
Source: Expert Review of Hematology - June 12, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Health care expenses for treatment of acute myeloid leukemia.
Authors: Bewersdorf JP, Shallis R, Wang R, Huntington S, Perreault S, Ma X, Zeidan AM Abstract Introduction: The cost of acute myeloid leukemia (AML) treatment is substantial and increasing. Inpatient treatment costs for allogeneic hematopoietic stem cell transplant (HSCT) and intensive chemotherapy are the main cost drivers in AML, however this pattern may change as new, expensive oral therapies enter the market. Areas covered: The authors provide an overview of the healthcare costs in patients with AML treated with various modalities (intensive chemotherapy, allogeneic HSCT, low-intensity treatment and supportive...
Source: Expert Review of Hematology - June 6, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Prothrombin complex concentrate for vitamin K antagonist reversal in acute bleeding settings: efficacy and safety.
Authors: Ostermann H, von Heymann C Abstract INTRODUCTION: Current guidelines recommend the administration of prothrombin complex concentrate in combination with vitamin K for normalization of coagulation in patients presenting with vitamin K antagonist-associated major bleeding, but until recently no adequately powered comparative trials had been conducted to support these recommendations. In this article, the authors review the evidence from studies assessing prothrombin complex concentrate treatment in these patients. Areas covered: A PubMed search (spanning January 1900 to September 2018) was conducted using th...
Source: Expert Review of Hematology - June 6, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

The gut microbiota - a modulator of endothelial cell function and a contributing environmental factor to arterial thrombosis.
Authors: Formes H, Reinhardt C Abstract INTRODUCTION: There is emerging evidence linking the commensal gut microbiota with the development of cardiovascular disease and arterial thrombosis. In immunothrombosis, the host clotting system protects against the dissemination of invading microbes, not considering the huge number of microbes that interact with host physiology in a mutualistic fashion. Areas covered: Interestingly, recent research revealed that colonizing gut microbes profoundly influence host innate immune pathways that support arterial thrombus growth. The gut microbiota promotes arterial thrombus format...
Source: Expert Review of Hematology - June 6, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Improving fibrinolysis in venous thromboembolism: impact of fibrin structure.
Authors: Undas A Abstract Introduction. Fibrinolysis is of key importance in maintaining vessel patency. Impaired fibrinolysis associated with more compact fibrin structure has been shown in patients with venous thromboembolism (VTE), including deep-vein thrombosis and pulmonary embolism (PE). Currently recombinant or modified plasminogen activators are the only commonly available thrombolytic agents. However, they are fraught with side effects and suboptimal effectiveness. Areas covered. Based on the available literature, the current evidence linking fibrinolysis with VTE and potential therapeutic targets among fi...
Source: Expert Review of Hematology - June 6, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

How much does 2016 WHO classification of myeloproliferative neoplasms affect the clinic?
Authors: Cassinat B, Giraudier S, Kiladjian JJ PMID: 31161817 [PubMed - as supplied by publisher] (Source: Expert Review of Hematology)
Source: Expert Review of Hematology - June 6, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

An alternative way - tyrosine kinase inhibitor (TKI) de-escalation - to discontinue TKIs in order to achieve treatment-free remission.
Authors: Kunbaz A, Eskazan AE PMID: 31166812 [PubMed - as supplied by publisher] (Source: Expert Review of Hematology)
Source: Expert Review of Hematology - June 6, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

High-grade B-cell lymphoma: how to diagnose and treat.
Authors: Novo M, Castellino A, Nicolosi M, Santambrogio E, Vassallo F, Chiappella A, Vitolo U Abstract Introduction: High Grade B-cell Lymphomas (HGBL) have been defined as a new separate entity in the 2016 revised WHO classification of lymphoid neoplasms. The previously well-known Double- and Triple-Hit Lymphomas (DHL/THL) are included in this umbrella category under the name of HGBL with MYC and BCL2 and/or BCL6 rearrangements (HGBL,R). A comprehensive diagnosis of HGBL is laborious, the diagnostic analyses required are expensive and time-consuming; moreover, a uniform consensus on which patients should be invest...
Source: Expert Review of Hematology - June 3, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Novel targets for the treatment of relapsing multiple myeloma.
Authors: Giuliani N, Accardi F, Marchica V, Dalla Palma B, Storti P, Toscani D, Vicario E, Malavasi F Abstract Introduction: Multiple myeloma (MM) is characterized by the high tendency to relapse and develop drug resistance. Areas covered: This review focused on the main novel targets identified to design drugs for the treatment of relapsing MM patients. CD38 and SLAMF7 are the main surface molecules leading to the development of monoclonal antibodies (mAbs) recently approved for the treatment of relapsing MM patients. B cell maturation antigen (BCMA) is a suitable target for antibody-drug conjugates, bispecific T ...
Source: Expert Review of Hematology - May 29, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Emicizumab for hemophilia A without inhibitors.
Authors: Cafuir L, Kruse-Jarres R, Mancuso ME, Kempton CL Abstract Introduction: Hemophilia A (HA) is an inherited bleeding disorder that, if not properly treated, is associated with debilitating joint damage due to recurrent hemarthroses as well as life-threatening bleeds including intracranial hemorrhage. For decades, the only method to prevent bleeding events was to infuse factor (F) VIII concentrates intravenously two to three times weekly. Although successful in reducing bleeding frequency, preventing a high proportion of joint disease, and extending life expectancy, standard continuous prophylaxis with FVIII ...
Source: Expert Review of Hematology - May 29, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Management of antithrombin deficiency: an update for clinicians.
Authors: Bravo-Pérez C, Vicente V, Corral J Abstract Introduction. Antithrombin is a serpin that inhibits multiple procoagulant serine proteases and acts as an endogenous anticoagulant. Thus, congenital antithrombin deficiency constitutes a major thrombophilic state, the most severe so far. Areas covered. In the present work, we globally review the biology, genetics, diagnosis, and management of congenital antithrombin deficiency, and also discuss puzzling questions and future perspectives regarding this severe inherited thrombophilia. Expert opinion. Although this disorder exerts high clinical heterogeneity...
Source: Expert Review of Hematology - May 24, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Current Perspectives on the Treatment of Double Hit Lymphoma.
Authors: Li LR, Wang L, He YZ, Young KH Abstract Introduction: Double hit lymphoma (DHL) represents a new diagnostic category with genetic, immunohistochemical and clinical characteristics intermediate between diffuse large B-cell lymphoma and Burkitt lymphoma. Patients with DHL usually experience poor survival after frontline R-CHOP treatment and require alternative therapies. However, the ideal therapeutic options remain undefined. Areas covered: Traditional therapies for the treatment of DHL are discussed, including intensive induction, hematopoietic stem cell transplantation (HSCT), methotrexate CNS-directed pr...
Source: Expert Review of Hematology - May 24, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

An update on pathogenesis and diagnosis of thrombotic thrombocytopenic purpura.
Authors: Joly BS, Coppo P, Veyradier A Abstract INTRODUCTION: Thrombotic thrombocytopenic purpura (TTP) is a rare and life-threatening thrombotic microangiopathy characterized by microangiopathic hemolytic anemia, consumption thrombocytopenia, and organ injury. TTP pathophysiology is based on a severe ADAMTS13 deficiency, the specific von Willebrand factor (VWF)-cleaving protease. Areas covered: VWF/ADAMTS13 interaction is specific, and ADAMTS13 conformation has been elucidated recently. ADAMTS13 deficiency is congenital or acquired. Several targets are used in TTP treatment. Expert opinion: Differential diagnosis ...
Source: Expert Review of Hematology - May 22, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Quality of life in multiple myeloma: considerations and recommendations.
Authors: Seitzler S, Finley-Oliver E, Simonelli C, Baz R Abstract INTRODUCTION: Advances in the management of multiple myeloma have culminated in the availability of novel agents which have resulted in improvement in patient outcomes. Nevertheless, the disease remains largely incurable and attention to long-term toxicity and quality of life is of importance. Limited data have addressed quality-of-life consideration in myeloma and most studies have assessed a finite time period during the course of the disease. Quality-of-life measures that have been used have largely focused on symptom reporting. In general, qualit...
Source: Expert Review of Hematology - May 17, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

The role of the thrombopoietin receptor MPL in myeloproliferative neoplasms: recent findings and potential therapeutic applications.
Authors: Vainchenker W, Plo I, Marty C, Varghese LN, Constantinescu SN Abstract INTRODUCTION: Classical Myeloproliferative Neoplasms (MPNs) include three disorders: Polycythemia Vera (PV), Essential Thrombocythemia (ET) and Primary Myelofibrosis (PMF). MPNs are associated with constitutive activation of JAK2 leading to persistent cell signaling downstream of the dimeric myeloid cytokine receptors due to mutations in three genes encoding JAK2, calreticulin (CALR) and the thrombopoietin (TPO) receptor (MPL or TPOR). CALR and MPL mutants induce JAK2 activation that depends on MPL expression, thus explaining why they i...
Source: Expert Review of Hematology - May 17, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Developments in the use of plasma exchange and adjunctive therapies to treat immune-mediated thrombotic thrombocytopenic purpura.
Authors: Picod A, Coppo P Abstract INTRODUCTION: Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a life-threatening disease characterized by a severe functional deficit in the von-Willebrand cleaving protease ADAMTS13, due to autoantibody production. The once-dismal prognosis of the disease has been changed by the discovery of the dramatic efficiency of therapeutic plasma exchange (TPE). Areas covered: This review focuses on the history and recent developments in the use of TPE for iTTP with a special emphasis on the consequences for TPE practice of the recent introduction of new highly effective immu...
Source: Expert Review of Hematology - May 17, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Potential of NK Cells in Multiple Myeloma Therapy.
Authors: Khan AM, Devarakonda S, Bumma N, Chaudhry M, Benson DM Abstract INTRODUCTION: Despite rapid advances in myeloma treatment with the development of new drugs, curative therapies remain elusive. Relapsed/refractory disease related to progressive dysregulation of immune system and acquired genetic abnormalities continues to be a major obstacle in achieving cure. Immune-based therapy harnessing the host defense mechanism of natural killer (NK) cells is a promising avenue in the treatment of myeloma. Areas covered: Here, we discuss the biology and cytotoxic activity of NK cells and the potential role of these in...
Source: Expert Review of Hematology - May 10, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

What role can next-generation sequencing play in myelodysplastic syndrome care?
Authors: Bonadies N, Bacher VU PMID: 31063400 [PubMed - as supplied by publisher] (Source: Expert Review of Hematology)
Source: Expert Review of Hematology - May 9, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Current Treatment of Lymphoma in Pregnancy.
Authors: Gurevich-Shapiro A, Avivi I Abstract INTRODUCTION: Lymphoproliferative diseases occurring during pregnancy present the treating physician with unique diagnostic and therapeutic challenges, aiming to achieve maternal cure without impairing fetal health, growth and survival. Due to the rarity of this complication, there is limited data to guide clinical decision making, especially regarding the safety of novel emerging therapies. . Areas covered: The current review presents the current practice of treatment of Hodgkin's (HL) and Non-Hodgkin's (NHL) lymphoma in the pregnant patient, according to disease stage...
Source: Expert Review of Hematology - May 7, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Preserving fertility in female patients with hematological malignancies: the key points.
Authors: Salama M, Anazodo A, Woodruff TK PMID: 31058548 [PubMed - as supplied by publisher] (Source: Expert Review of Hematology)
Source: Expert Review of Hematology - May 7, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Molecular features, prognosis, and novel treatment options for pediatric acute megakaryoblastic leukemia.
Authors: De Marchi F, Araki M, Komatsu N Abstract INTRODUCTION: Acute megakaryoblastic leukemia (AMegL) is a rare hematological neoplasm most often diagnosed in children and is commonly associated with Down syndrome (DS). Although AMegLs are specifically characterized and typically diagnosed by megakaryoblastic expansion, recent advancements in molecular analysis have highlighted the heterogeneity of this disease, with specific cytogenic and genetic alterations characterizing different disease sub-types. Areas covered: This review will focus on describing recurrent molecular variations in both DS and non-DS pediatr...
Source: Expert Review of Hematology - April 19, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Getting personal with myelodysplastic syndromes: is now the right time?
Authors: Chokr N, Pine AB, Bewersdorf JP, Shallis RM, Stahl M, Zeidan AM Abstract INTRODUCTION: Commonly used scoring systems rely on blood counts, histological and cytological examination of bone marrow and peripheral blood as well as cytogenetic assessments to estimate prognosis of patients with myelodysplastic syndromes (MDS) and guide therapy decisions. Next-generation sequencing (NGS) has identified recurrent genetic abnormalities in up to 90% of patients with MDS and may provide important information regarding the pathogenesis of the disease, diagnostic and prognostic evaluation, and therapy selection. Areas ...
Source: Expert Review of Hematology - April 13, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

A systematic literature review of incidence, mortality and relapse of patients diagnosed with chronic graft versus host disease.
This study aims to provide a systematic literature review on incidence, mortality and relapse of chronic GVHD patients. Areas covered: The authors searched for English-language articles published between 2007 and 2017 using PubMed. Studies that applied the 2005 or 2015 NIH Consensus Criteria for the diagnosis and staging of chronic GVHD, and had a cohort size of at least 100 patients were included. Expert opinion: The authors found a wide variation of incidence rates, which can be explained by heterogeneity in the characteristics of study samples and applied transplantation protocols. Chronic GVHD was associated with highe...
Source: Expert Review of Hematology - April 10, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Updates on thrombotic events associated with multiple myeloma.
Authors: Fotiou D, Gavriatopoulou M, Ntanasis-Stathopoulos I, Migkou M, Dimopoulos MA, Terpos E Abstract INTRODUCTION: Despite the implementation of thromboprophylaxis guidelines, the risk of venous thromboembolism (VTE) in patients with Multiple Myeloma (MM) remains significant. There is therefore a need for more sensitive risk stratification tools and optimization of our thromboprophylaxis approach. Areas covered: Since 2015 there have been no breakthrough data in this field. This review aims to cover recent data that have evaluated the effectiveness and extent to which IMWG 2014 guidelines are being applied. In ...
Source: Expert Review of Hematology - April 10, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

An update on the 'danger theory' in inhibitor development in haemophilia A.
Authors: Schep SJ, Boes ML, Schutgens REG, van Vulpen LFD Abstract INTRODUCTION: Nowadays, one of the most serious treatment complications in haemophilia A is the formation of neutralizing antibodies against coagulation factor VIII (FVIII). These so-called inhibitors develop in about 30% of all patients with severe haemophilia A. Once formed, inhibitors reduce FVIII efficacy in blood coagulation, which has a negative impact on patients' health and quality of life and significantly increases haemophilia A treatment costs. The pathophysiology of inhibitor development is a complex and multi-causal process, in which bo...
Source: Expert Review of Hematology - April 7, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Risks and patient outcomes of surgical intervention for hemophilic arthropathy.
Authors: Rodriguez-Merchan EC Abstract INTRODUCTION: Surgical interventions usually needed for hemophilic arthropathy include arthroscopic synovectomy, alignment osteotomy, arthroscopic joint debridement, radial head removal, arthrodesis of the ankle, and total joint arthroplasty (elbow, hip, knee, ankle). Areas covered: Whatever the surgical technique, appropriate surgical hemostasis must be achieved through infusion of concentrate of the deficient factor (factor VIII or factor IX), either in recombinant or plasma-derived form, at the correct dosage (ideally for 10-14 days). In patients with inhibitors, there are ...
Source: Expert Review of Hematology - April 3, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Contemporary management of essential thrombocythemia in children.
Authors: Randi ML, Bertozzi I, Putti MC Abstract INTRODUCTION: Essential thrombocythemia (ET) is a disease which is extremely rare in children. Only recently, data on pediatric ET have become available Areas covered: In children with sustained platelet count over 450 x 109/L, secondary thrombocytosis must be ruled out. ET workup comprehends research of JAK2V617F, CALR and MPL mutations and bone marrow biopsy (BM). In asymptomatic children wait and watch is the best option. Aspirin controls headache and other microvascular disturbances. Patients with venous thrombosis need anticoagulation. Cytoreductive drugs in chi...
Source: Expert Review of Hematology - March 31, 2019 Category: Hematology Tags: Expert Rev Hematol Source Type: research