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Expert Review of Hematology

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https://www.readbyqxmd.com/read/29337599/pearson-syndrome
#1
Piero Farruggia, Floriana Di Marco, Carlo Dufour
Pearson syndrome (PS) is a sporadic and very rare syndrome classically associated with single large-scale deletions of mitochondrial DNA and characterized by refractory sideroblastic anemia during infancy. Areas covered: This review presents an analysis and interpretation of the published data that forms the basis for our understanding of PS. PubMed, Google Scholarand Thompson ISI Web of Knowledge were searched for relevant data. Expert Commentary: PS is a very rare mitochodrial disease that involves different organs and systems...
January 16, 2018: Expert Review of Hematology
https://www.readbyqxmd.com/read/29307226/new-concepts-in-the-treatment-and-diagnosis-of-amyloidosis
#2
Paolo Milani, Giovanni Palladini, Giampaolo Merlini
The most common form of systemic amyloidosis in Western countries is light chain amyloidosis. It is characterized by the deposition of a misfolded light chain in target organs. This amyloid precursor is produced by a usually small but dangerous B-cell clone. Areas covered: This review examines the diagnostic workup of this disease and current knowledge of biomarker-based staging systems. In addition, a risk-adapted treatment approach is presented, as well as an overview of the new treatment strategies. Expert commentary: The cornerstone of treatment is rapid and effective chemotherapy targeting the underlying plasma cell clone...
January 10, 2018: Expert Review of Hematology
https://www.readbyqxmd.com/read/29313725/prognostic-risk-model-for-patients-with-high-risk-polycythemia-vera-and-essential-thrombocythemia
#3
Abhishek A Mangaonkar, Katherine P Hoversten, Naseema Gangat
Polycythemia Vera (PV) and essential thrombocythemia (ET) are the most frequent Philadelphia chromosome-negative myeloproliferative neoplasms, the other entity being myelofibrosis. Management of patients with PV and ET is fraught with difficulties as they have an inherent tendency to cause thrombotic and hemorrhagic events. There are no curative treatment options therefore it is important that a risk-adapted treatment approach is applied. Areas covered: This review discusses existing literature about prognosis in PV and ET, and addresses critical aspects related to defining 'high-risk' disease...
January 9, 2018: Expert Review of Hematology
https://www.readbyqxmd.com/read/29300108/measuring-success-in-hemophilia-gene-therapy-using-a-factor-level-outcomes-yardstick
#4
Nicoletta Machin, Margaret V Ragni
No abstract text is available yet for this article.
January 4, 2018: Expert Review of Hematology
https://www.readbyqxmd.com/read/29285951/seifem-2017-from-real-life-to-an-agreement-on-the-use-of-granulocyte-transfusions-and-colony-stimulating-factors-for-prophylaxis-and-treatment-of-infectious-complications-in-patients-with-hematologic-malignant-disorders
#5
Alessandro Busca, Simone Cesaro, Luciana Teofili, Mario Delia, Chiara Cattaneo, Marianna Criscuolo, Francesco Marchesi, Nicola Stefano Fracchiolla, Caterina Giovanna Valentini, Francesca Farina, Roberta Di Blasi, Lucia Prezioso, Angelica Spolzino, Anna Candoni, Maria Ilaria Del Principe, Luisa Verga, Annamaria Nosari, Franco Aversa, Livio Pagano
The rapid spread of severe infections mainly due to resistant pathogens, justifies the search for therapies aiming to restore immune functions severely compromised in patients with hematologic malignancies. Areas covered: The present review summarizes the current knowledge on the role of granulocyte transfusions and colony-stimulating factors as treatment strategy for hematologic patients with serious infectious complications. In addition, a survey among 21 hematologic centers, to evaluate the clinical practice for the use of G-CSF originator and biosimilars was performed...
January 3, 2018: Expert Review of Hematology
https://www.readbyqxmd.com/read/29279013/novel-approaches-to-diagnosis-and-treatment-of-juvenile-myelomonocytic-leukemia
#6
Franco Locatelli, Mattia Algeri, Pietro Merli, Luisa Strocchio
Juvenile myelomonocytic leukemia (JMML) is a clonal hematopoietic disorder of infancy/early childhood, resulting from oncogenic mutations in genes involved in the Ras pathway. As JMML often exhibits an aggressive course, the timing of diagnosis and treatment is critical to outcome. Areas covered: This review summarizes current approaches to diagnosis and treatment of JMML, highlighting most recent insights into genetic and epigenetic mechanisms underlying the disease, and providing an overview of novel potential therapeutic strategies...
January 3, 2018: Expert Review of Hematology
https://www.readbyqxmd.com/read/29264938/a-concise-review-of-bcl-2-inhibition-in-acute-myeloid-leukemia
#7
Meera Yogarajah, Richard M Stone
Acute myeloid leukemia (AML) is a heterogeneous disease characterized by clonal proliferation of myeloid precursors with impaired ability to differentiate to mature cells causing accumulation of leukemic blasts in bone marrow, peripheral blood, and extramedullary tissue. Our understanding of the genomic landscape of AML has improved prognostic accuracy and lead to the development of targeted therapies. In 2017 the Food and Drug Administration (FDA) approved midostaurin, gemtuzumab ozogamicin, CPX-351 and enasidenib for the treatment of AML...
January 3, 2018: Expert Review of Hematology
https://www.readbyqxmd.com/read/29292655/diagnosis-management-and-response-criteria-of-iron-overload-in-myelodysplastic-syndromes-mds-updated-recommendations-of-the-austrian-mds-platform
#8
Peter Valent, Reinhard Stauder, Igor Theurl, Klaus Geissler, Thamer Sliwa, Wolfgang R Sperr, Peter Bettelheim, Heinz Sill, Michael Pfeilstöcker
Despite the availability of effective iron chelators, transfusion-related morbidity is still a challenge in chronically transfused patients with myelodysplastic syndromes (MDS). In these patients, transfusion-induced iron overload may lead to organ dysfunction or even organ failure. In addition, iron overload is associated with reduced overall survival in MDS. Areas covered: During the past 10 years, various guidelines for the management of MDS patients with iron overload have been proposed. In the present article, we provide our updated recommendations for the diagnosis, prevention and therapy of iron overload in MDS...
January 2, 2018: Expert Review of Hematology
https://www.readbyqxmd.com/read/29260588/upfront-treatment-of-elderly-myeloma-patients-an-overview-and-update
#9
Hussein G Elsayed, Amal S Alabdulwahab
Multiple myeloma is primarily a disease of older age, with a median age of 70 years at diagnosis. Management of the disease in this diverse population is challenging, in the face comorbidities and frailties. Areas covered: This review discusses the management challenges of elderly myeloma patients in view of the current evidence and propose for performing a formal objective assessment of the functional status to guide choice of treatment. Expert commentary: The approval of many antimyeloma medications with various mechanisms of action in the past two decades had sparked the debate about choosing the best combination, duration of therapy, role of transplant and the possibility to cure myeloma after being changed to a chronic disease...
December 20, 2017: Expert Review of Hematology
https://www.readbyqxmd.com/read/29258406/a-contemporary-look-at-fviii-inhibitor-development-still-a-great-influence-on-the-evolution-of-hemophilia-therapies
#10
Elena Santagostino, Guy Young, Manuel Carcao, Pier Mannuccio Mannucci, Susan Halimeh, Steve Austin
The development of inhibitors against factor VIII (FVIII) replacement therapy remains the most important challenge for clinicians in the treatment of hemophilia patients. This review focusses on risk factors and management of FVIII inhibitors, particularly in light of SIPPET study findings and subsequent analyses. Areas covered: A brief history and evolution of hemophilia therapies is provided, including an overview of conventional and new (including investigational) therapeutic approaches for the treatment of hemophilia...
December 19, 2017: Expert Review of Hematology
https://www.readbyqxmd.com/read/29231766/corrigendum
#11
(no author information available yet)
No abstract text is available yet for this article.
December 12, 2017: Expert Review of Hematology
https://www.readbyqxmd.com/read/29207881/sickle-cell-disease-a-malady-beyond-a-hemoglobin-defect-in-cerebrovascular-disease
#12
Junaid Ansari, Youmna E Moufarrej, Rafal Pawlinski, Felicity N E Gavins
Sickle cell disease (SCD) is a devastating monogenic disorder that presents as a multisystem illness and affects approximately 100,000 individuals in the United States alone. SCD management largely focuses on primary prevention, symptomatic treatment and targeting of hemoglobin polymerization and red blood cell sickling. Areas covered: This review will discuss the progress of SCD over the last few decades, highlighting some of the clinical (mainly cerebrovascular) and psychosocial challenges of SCD in the United States...
December 5, 2017: Expert Review of Hematology
https://www.readbyqxmd.com/read/29160119/a-review-of-the-infection-pathogenesis-and-prophylaxis-recommendations-in-patients-with-chronic-lymphocytic-leukemia
#13
Tamar Tadmor, Manfred Welslau, Iwoma Hus
The majority of patients with CLL will suffer from infections during their disease, accounting for approximately 60% of deaths in CLL. Patients are predisposed to infection due to immune defects related to the primary disease, and as a result of therapy. The range of infectious complications has evolved alongside therapeutic advances in the treatment of CLL. More recently several novel therapeutic compounds have been introduced in CLL, whose unique safety profiles will probably have an impact on the prophylaxis and management of infections in these patients...
November 27, 2017: Expert Review of Hematology
https://www.readbyqxmd.com/read/29168399/vacuolar-atpase-as-a-possible-therapeutic-target-in-human-acute-myeloid-leukemia
#14
Elise Aasebø, Sushma Bartaula-Brevik, Maria Hernandez-Valladares, Øystein Bruserud
V-ATPase is a proton pump expressed both in the membrane of intracellular organelles (e.g. endosomes, lysosomes, Golgi structures) and the plasma membrane. It is an important regulator of organellar functions, intracellular molecular trafficking, intercellular communication and intracellular signaling. It is therefore considered as a possible therapeutic target in the treatment of human malignancies. Areas covered: Relevant publications were identified through literature searches in the PubMed database. We searched for original articles and reviews describing the possible importance of V-ATPase for leukemogenesis and chemosensitivity in human myeloid cells, especially acute myeloid leukemia (AML) cells...
November 23, 2017: Expert Review of Hematology
https://www.readbyqxmd.com/read/29149573/the-intrinsic-genetic-and-epigenetic-regulator-factors-as-therapeutic-targets-and-the-effect-on-fetal-globin-gene-expression
#15
Pegah Adelvand, Mohammed Hamid, Soroush Sardari
Introduction The effort to induce fetal globin or Hb F gene expression as an alternative therapy for blood transfusion has been ongoing for few decades, with promising results evident in patients with hemoglobinopathies. Although the clinical outcomes have been satisfactory and significant, there are still concerns about the safety of Hb F inducers in the long-term. There are potent inducers which lose their potency and safety over the course of therapy. Area Covered: In this work, efforts have been made to review the latest findings on intrinsic genetic and epigenetic factors which are able to induce the gene expression of fetal globin in adult patients with beta (β)-thalassemia Major, Intermedia and sickle cell disease (SCD)...
November 17, 2017: Expert Review of Hematology
https://www.readbyqxmd.com/read/29091481/the-quest-for-safer-antithrombotic-treatment-regimens-in-patients-with-coronary-artery-disease-new-strategies-and-paradigm-shifts
#16
Jae Youn Moon, Francesco Franchi, Fabiana Rollini, Dominick J Angiolillo
Despite the undeniable benefits on reducing ischemic adverse events, antiplatelet regimens including dual antiplatelet therapy (DAPT) with aspirin and P2Y12 receptor inhibitors are associated with an increased risk of bleeding. The awareness of the unfavorable prognostic implications associated with bleeding complications have somewhat hampered the enthusiasm towards the use of more potent antiplatelet treatment regimens or prolonged use of DAPT. This awareness also has prompted a series of investigations geared towards the identification of antithrombotic treatment regimens which are efficacious at reducing ischemic recurrences while also safe in terms of bleeding risk profile...
November 14, 2017: Expert Review of Hematology
https://www.readbyqxmd.com/read/29103337/missing-angiogenic-factors-in-hemophilic-arthropathy
#17
Amir Hossein Norooznezhad
No abstract text is available yet for this article.
November 13, 2017: Expert Review of Hematology
https://www.readbyqxmd.com/read/29092644/response-to-missing-angiogenic-factors-in-hemophilic-arthropathy
#18
E Carlos Rodriguez-Merchan
No abstract text is available yet for this article.
November 7, 2017: Expert Review of Hematology
https://www.readbyqxmd.com/read/29072100/gonadal-dysfunction-in-adult-male-patients-with-thalassemia-major-an-update-for-clinicians-caring-for-thalassemia
#19
Vincenzo De Sanctis, Ashraf T Soliman, Heba Elsedfy, Salvatore Di Maio, Duran Canatan, Nada Soliman, Mehran Karimi, Christos Kattamis
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. Areas covered: The diagnosis requires careful clinical assessment and appropriate laboratory testing. Its management is more complex compared to other 'classical' causes of hypogonadism because of multiple associated disorders (cardiac, hepatic and endocrine) and other contributing factors basically iron overload and iron toxicity...
November 2, 2017: Expert Review of Hematology
https://www.readbyqxmd.com/read/29082835/efficacy-and-safety-of-bispecific-t-cell-engager-blinatumomab-and-the-potential-to-improve-leukemia-free-survival-in-b-cell-acute-lymphoblastic-leukemia
#20
Josep-Maria Ribera
Immunotherapy is a promising modality of treatment of neoplastic diseases, including acute lymphoblastic leukemia (ALL). The CD19/CD3-bispecific T cell-engaging (BiTE®) monoclonal antibody blinatumomab can transiently bind cytotoxic T cells to CD19(+) target B cells of ALL inducing their serial lysis. Areas covered: This review focuses on the efficacy and safety of blinatumomab used for the treatment of relapsed/refractory (R/R) ALL and minimal residual disease (MRD)-positive B-cell precursor (BCP) ALL in adults and children, as well as the future prospects of this drug in the treatment of ALL...
October 30, 2017: Expert Review of Hematology
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