Current and future prospects for hemophilia gene therapy.

abstract：INTRODUCTION:The discovery of the factor V Leiden (FVL) missense mutation (Arg506Gln) causing factor V resistance to the anticoagulant action of activated protein C was a landmark that allowed a better understanding of the basis of inherited thrombotic risk. FVL mutation is currently the most common known hereditary de...

journal_title：Expert review of hematology

authors： Campello E,Spiezia L,Simioni P

更新日期：2016-12-01 00:00:00

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 an...

journal_title：Expert review of hematology

authors： Santambrogio E,Nicolosi M,Vassallo F,Castellino A,Novo M,Chiappella A,Vitolo U

更新日期：2019-09-01 00:00:00

abstract：INTRODUCTION:Immune surveillance is the dynamic process whereby the immune system identifies and kills tumor cells based on their aberrant expression of stress-related surface molecules or presentation of tumor neoantigens. It plays a crucial role in controlling the initiation and progression of hematologic cancers suc...

journal_title：Expert review of hematology

authors： Nesic M,El-Galaly TC,Bøgsted M,Pedersen IS,Dybkær K

更新日期：2020-06-01 00:00:00

abstract：:Iron overload is frequently observed in patients with hematologic diseases before and after allogeneic stem-cell transplantation because they usually receive multiple red blood cell transfusions. Elevated pretransplant serum ferritin levels, which are widely used as indicators of body iron status, are significantly as...

journal_title：Expert review of hematology

authors： Kanda J,Kawabata H,Chao NJ

更新日期：2011-02-01 00:00:00

abstract：:The fms-like tyrosine kinase 3 (FLT3) plays an important role in both normal and malignant hematopoiesis. Activating mutations in the FLT3 receptor can be detected in approximately 30% of acute myeloid leukemias (AMLs) and are associated with a distinctly poor clinical outcome for patients. There are now several class...

journal_title：Expert review of hematology

authors： el-Shami K,Stone RM,Smith BD

更新日期：2008-12-01 00:00:00

abstract：:Indolent non-Hodgkin's lymphoma (iNHL) describes a group of B-cell lymphomas with a long median survival and a relapsing-remitting clinical course. Although existing treatments are initially effective, patients often relapse, demonstrating decreasing efficacy with successive treatment courses. Alternative treatments a...

journal_title：Expert review of hematology

authors： Davies A

更新日期：2015-10-01 00:00:00

abstract：INTRODUCTION:Iliopsoas muscle hemorrhage in patients with hemophilia is a serious complication with relevant related morbidity. AREAS COVERED:A Cochrane Library and PubMed (MEDLINE) search of studies related to iliopsoas hematomas in hemophilia was analyzed. Pain in the hip joint area and femoral nerve involvement in ...

journal_title：Expert review of hematology

authors： Rodriguez-Merchan EC,De la Corte-Rodriguez H

更新日期：2020-08-01 00:00:00

abstract：INTRODUCTION:Most patients with Hodgkin lymphoma (HL) enjoy durable remissions following front-line treatment but 30% of patients are refractory or relapse after first line therapy. Salvage chemotherapy followed by autologous stem cell transplantation (ASCT) can cure an additional 50-55% of relapsing patients but new t...

journal_title：Expert review of hematology

authors： Donato EM,Fernández-Zarzoso M,De La Rubia J

更新日期：2017-05-01 00:00:00

abstract：INTRODUCTION:In the last decade, the availability of new drugs for the treatment of Multiple Myeloma (MM) significantly improved patients' outcomes, but also raised attention towards a new spectrum of adverse events. Recently, four novel agents with different mechanisms of action (carfilzomib, elotuzumab, daratumumab a...

journal_title：Expert review of hematology

authors： Brioli A,Mügge LO,Hochhaus A,Von Lilienfeld-Toal M

更新日期：2017-03-01 00:00:00

abstract：INTRODUCTION:First and second generation tyrosine kinase inhibitors, represent a new, fully biologic and targeted approach to chronic lymphocytic leukemia and allowed to obtain high responses and acceptable tolerability even in elderly and high risk patients. On the other hand, prolonged experience with these agents ha...

journal_title：Expert review of hematology

authors： Frustaci AM,Montillo M,Picardi P,Mazzucchelli M,Cairoli R,Tedeschi A

更新日期：2016-07-01 00:00:00

abstract：INTRODUCTION:Purine analogs made dramatic improvements for patients with hairy cell leukemia (HCL), but patients often relapse, require multiple treatments, and may become refractory. Major developments in treatment of relapsed/refractory HCL occurred with discovery of disease biology. New agents increase the complexit...

journal_title：Expert review of hematology

authors： Chihara D,Kreitman RJ

更新日期：2020-10-01 00:00:00

abstract：:Akt is a Ser-Thr kinase with pleiotropic effects on cell survival, growth and metabolism. Recent evidence from gene-deletion studies in mice, and analysis of human platelets treated with Akt inhibitors, suggest that Akt regulates platelet activation, with potential consequences for thrombosis. Akt activation is regula...

journal_title：Expert review of hematology

authors： Woulfe DS

更新日期：2010-02-01 00:00:00

abstract：:Despite significant improvements in treatment modalities over the 10 years, the clinical course of patients with follicular lymphoma (FL) remains heterogeneous. Thus, prognostic indexes are still required to direct treatment choices and for the design of clinical trials. Investigators have conducted a variety of studi...

journal_title：Expert review of hematology

authors： Luminari S,Cox MC,Montanini A,Federico M

更新日期：2009-10-01 00:00:00

abstract：:Asparaginase has been a mainstay of therapy in the treatment of acute lymphoblastic leukemia since the 1970s. There are two major preparations available and FDA approved in the United States today, one derived from Escherichia coli and the other from Erwinia chrysanthemi. Erwinia asparaginase is antigenically distinct...

journal_title：Expert review of hematology

authors： Figueiredo L,Cole PD,Drachtman RA

更新日期：2016-03-01 00:00:00

abstract：:Although guidelines exist to deal with some aspects of platelet transfusion practice, many important clinical issues have not been addressed in large randomized controlled trials (RCTs). Slichter et al. conducted a RCT of prophylactic platelet transfusions to determine the effects of the dose of platelets on clinical ...

journal_title：Expert review of hematology

authors： Cid J,Lozano M

更新日期：2010-08-01 00:00:00

abstract：:Tumor lysis syndrome (TLS) is a rare but potentially life-threatening complication of neoplasms, preferentially hematological malignancies. Well known since at least ninety years ago, this condition can be misdiagnosed and incorrectly managed due to rapid onset of symptoms, sometimes overlapping with cancer-derived cl...

journal_title：Expert review of hematology

authors： Criscuolo M,Fianchi L,Dragonetti G,Pagano L

更新日期：2016-01-01 00:00:00

abstract：INTRODUCTION:Chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) are considered indolent lymphocytic malignancies, more often requiring active surveillance rather than intervention. Despite the indolent nature of CLL/SLL, treatment is likely indicated in a patients' lifetime. Recent changes in the t...

journal_title：Expert review of hematology

authors： Tees MT,Flinn IW

更新日期：2017-02-01 00:00:00

abstract：INTRODUCTION:Primary plasma cell leukemia (PPCL) is one of the most aggressive hematological malignancies. The prognosis of PPCL patients remains poor, although some improvements have been made in recent years. Areas covered: In this review recent clinical and biological advances in PPCL are reported. Some recommendati...

journal_title：Expert review of hematology

authors： Musto P,Statuto T,Valvano L,Grieco V,Nozza F,Vona G,Bochicchio GB,La Rocca F,D'Auria F

更新日期：2019-04-01 00:00:00

abstract：:In 2008, we reviewed the practical interface between transfusion medicine and the surgery and critical care of severely injured patients. Reviewed topics ranged from epidemiology of trauma to patterns of resuscitation to the problems of transfusion reactions. In the interim, trauma specialists have adopted damage cont...

journal_title：Expert review of hematology

authors： Murthi SB,Stansbury LG,Dutton RP,Edelman BB,Scalea TM,Hess JR

更新日期：2011-10-01 00:00:00

abstract：:Internal tandem duplication mutations of FMS-like tyrosine kinase-3 (FLT3) have been associated with poor outcomes in acute myelogenous leukemia. Over the course of the last several years, multiple agents have been developed and studied as potential inhibitors of FLT3 with the hope of providing clinical benefit for th...

journal_title：Expert review of hematology

authors： Fathi AT,Levis M

更新日期：2009-02-01 00:00:00

abstract：INTRODUCTION:Mitochondria are cellular organelles that perform numerous bioenergetic, biosynthetic, and regulatory functions and play a central role in iron metabolism. Extracellular iron is taken up by cells and transported to the mitochondria, where it is utilized for synthesis of cofactors essential to the function ...

journal_title：Expert review of hematology

authors： Paul BT,Manz DH,Torti FM,Torti SV

更新日期：2017-01-01 00:00:00

abstract：INTRODUCTION:Chronic Myeloid Leukemia (CML) is a myeloproliferative disorder that has become the neoplastic poster child for understanding the disease biology of a malignant disease and targeting effective therapy. The targeted therapy of BCR-ABL inhibition by tyrosine kinase inhibitors (TKI) has provided the epitome f...

journal_title：Expert review of hematology

authors： Poch Martell M,Sibai H,Deotare U,Lipton JH

更新日期：2016-10-01 00:00:00

abstract：INTRODUCTION:Hypogonadism is the most frequently reported endocrine complication, affecting 40%-80% of thalassemia major (TM) patients. The prevalence and severity of hypogonadism in TM varies among studies, depending on patients' age, genotype, transfusion frequency and starting age and efficiency of iron chelation. A...

journal_title：Expert review of hematology

authors： De Sanctis V,Soliman AT,Elsedfy H,Di Maio S,Canatan D,Soliman N,Karimi M,Kattamis C

更新日期：2017-12-01 00:00:00

abstract：INTRODUCTION:Hodgkin's lymphoma (HL) is largely a curable disease with excellent prognosis. The standard of care in patients with relapsed disease has been to try salvage chemotherapy followed by an autologous stem cell transplantation (ASCT). Managing the patients who relapse after ASCT, is challenging. With the appro...

journal_title：Expert review of hematology

authors： Kallam A,Armitage JO

更新日期：2018-04-01 00:00:00

abstract：INTRODUCTION:Ferumoxytol is a superparamagnetic molecule originally developed as a contrast agent for magnetic resonance imaging. Elemental iron is contained within the carbohydrate core and is released slowly after infusion allowing a large dose of iron to be administered in a short period of time. Ferumoxytol, origin...

journal_title：Expert review of hematology

authors： Auerbach M,Chertow GM,Rosner M

更新日期：2018-10-01 00:00:00

abstract：:The introduction of bortezomib, a first-generation proteasome inhibitor, changed the standard-of-care for newly diagnosed and relapsed multiple myeloma patients. The next generation of proteasome inhibitors, such as carfilzomib, provides a novel pharmacokinetic and pharmacodynamic profile. In vitro data suggest a more...

journal_title：Expert review of hematology

authors： Khan RZ,Badros A

更新日期：2012-08-01 00:00:00

abstract：INTRODUCTION:Peripheral T cell lymphomas (PTCL) are a heterogenous group of lymphoproliferative disorders which are generally not curable with conventional chemotherapy and associated with inferior outcomes. Pralatrexate is a novel folate analog, the first FDA approved drug) for the treatment of relapsed/refractory (R/...

journal_title：Expert review of hematology

authors： Jennifer C Z,Sara Mohamed J,Salma A,Francine F

更新日期：2020-06-01 00:00:00

abstract：:Relapsed or refractory classical Hodgkin lymphoma (HL) remains a therapeutic challenge. Patients with relapsed HL should be identified according to their prognostic factors at relapse (duration of remission and extranodal disease or stage). Patients with refractory disease, defined as progression during induction trea...

journal_title：Expert review of hematology

authors： Sibon D,Brice P

更新日期：2009-06-01 00:00:00

abstract：INTRODUCTION:Ruxolitinib is a potent and selective JAK1/JAK2 inhibitor that has shown superiority as compared to available conventional chemotherapies, in terms of reduction in splenomegaly and improvement of symptoms and quality of life. Areas covered: Data published about overall survival in the major randomized spon...

journal_title：Expert review of hematology

authors： Massaro F,Molica M,Breccia M

更新日期：2017-02-01 00:00:00

abstract：:Major advances have significantly improved the outcome of mantle cell lymphoma (MCL). Incorporation of rituximab to CHOP regimen, the adoption of high dose cytarabine with frontline autologous stem cell transplantation in young patients, maintenance rituximab or bortezomib based chemotherapy in elderly patients, impro...

journal_title：Expert review of hematology

authors： El Halabi L,Ghez D,Ribrag V

更新日期：2016-03-01 00:00:00