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Hematology/oncology Clinics of North America

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https://www.readbyqxmd.com/read/29458738/thalassemia
#1
EDITORIAL
Ali T Taher
No abstract text is available yet for this article.
April 2018: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/29458737/erratum
#2
(no author information available yet)
No abstract text is available yet for this article.
April 2018: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/29458736/emerging-therapies
#3
REVIEW
Amaliris Guerra, Khaled M Musallam, Ali T Taher, Stefano Rivella
At present, the only definitive cure for β-thalassemia is a bone marrow transplant (BMT); however, HLA-blood-matched donors are scarcely available. Current therapies undergoing clinical investigation with most potential for therapeutic benefit are the β-globin gene transfer of patient-specific hematopoietic stem cells followed by autologous BMT. Other emerging therapies deliver exogenous regulators of several key modulators of erythropoiesis or iron homeostasis. This review focuses on current approaches for the treatment of hemoglobinopathies caused by disruptions of β-globin...
April 2018: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/29458735/gene-therapy-and-genome-editing
#4
REVIEW
Farid Boulad, Jorge Mansilla-Soto, Annalisa Cabriolu, Isabelle Rivière, Michel Sadelain
The β-thalassemias are inherited blood disorders that result from insufficient production of the β-chain of hemoglobin. More than 200 different mutations have been identified. β-Thalassemia major requires life-long transfusions. The only cure for severe β-thalassemia is to provide patients with hematopoietic stem cells. Globin gene therapy promises a curative autologous stem cell transplantation without the immunologic complications of allogeneic transplantation. The future directions of gene therapy include enhancement of lentiviral vector-based approaches, fine tuning of the conditioning regimen, and the design of safer vectors...
April 2018: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/29458734/hematopoietic-stem-cell-transplantation-in-thalassemia
#5
REVIEW
Luisa Strocchio, Franco Locatelli
Although recent advances in gene therapy are expected to increase the chance of disease cure in thalassemia major, at present hematopoietic stem cell transplantation (HSCT) remains the only consolidated curative approach for this disorder. The widest experience has been obtained in the HLA-matched family donor (MFD) setting, with probabilities of overall and thalassemia-free survival exceeding 90% and 85%, respectively. As for most patients a suitable MFD is not available, alternative donors (HLA-matched unrelated donor, unrelated cord blood, HLA-haploidentical relative) have been increasingly explored, translating into the expansion of the number of patients treatable with HSCT...
April 2018: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/29458733/fertility-and-pregnancy-in-women-with-transfusion-dependent-thalassemia
#6
REVIEW
Katie T Carlberg, Sylvia T Singer, Elliott P Vichinsky
As more women with transfusion-dependent thalassemia are seeking pregnancy, ensuring the best outcomes for both the mother and baby requires concerted, collaborative efforts between practitioners and the family. Proactive counseling, early fertility evaluation, recent developments in reproductive technology, and optimal management of iron overload, have resulted in more successful pregnancies and the birth of healthy newborns. With advances in technology for prenatal screening and increased awareness to perform screening for hemoglobinopathies, healthy pregnancy outcomes have become the expectation...
April 2018: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/29458732/mri-for-iron-overload-in-thalassemia
#7
REVIEW
Juliano Lara Fernandes
MRI is a key tool in the current management of patients with thalassemia. Given its capability of assessing iron overload in different organs noninvasively and without contrast, it has significant advantages over other metrics, including serum ferritin. Liver iron concentration can be measured either with relaxometry methods T2*/T2 or signal intensity ratio techniques. Myocardial iron can be assessed in the same examination through T2* imaging. In this review, we focus on showing how MRI evaluates iron in both organs and the clinical applications as well as practical approaches to using this tool by clinicians taking care of patients with thalassemia...
April 2018: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/29458731/iron-chelation-therapy-as-a-modality-of-management
#8
REVIEW
Yesim Aydinok
Introduction of MRI techniques for identifying and monitoring tissue iron overload and the current understanding of iron homeostasis in transfusion-dependent (TDT) and non-transfusion-dependent thalassemia have allowed for a more robust administration of iron chelation therapies. The development of safe and efficient oral iron chelators and the insights gained from large-scale prospective studies using these agents have improved iron overload management. A significant reduction in iron toxicity-induced morbidity and mortality and improvements in quality of life were observed in TDT...
April 2018: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/29458730/interaction-of-transfusion-and-iron-chelation-in-thalassemias
#9
REVIEW
John B Porter, Maciej W Garbowski
The relationship between blood transfusion intensity, chelatable iron pools, and extrahepatic iron distribution is described in thalassemia. Risk factors for cardiosiderosis are discussed with particular reference to the balance of transfusional iron loading rate and transferrin-iron utilization rate as marked by plasma levels of soluble transferrin receptors. Low transfusion regimens increase residual erythropoiesis allowing for apotransferrin-dependent clearance of non-transferrin-bound iron species otherwise destined for myocardium...
April 2018: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/29458729/hypercoagulability-and-vascular-disease
#10
REVIEW
Ali T Taher, Maria Domenica Cappellini, Rayan Bou-Fakhredin, Daniel Coriu, Khaled M Musallam
The presence of a high incidence of thrombotic events, mainly in nontransfusion-dependent β-thalassemia syndromes, has led to the identification of a hypercoagulable state in thalassemia patients. This article highlights the mechanisms leading to hypercoagulability in thalassemia. It also discusses the clinical experience and available evidence on prevention and management approaches.
April 2018: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/29458728/clinical-complications-and-their-management
#11
REVIEW
Alessia Marcon, Irene Motta, Ali T Taher, Maria Domenica Cappellini
The hallmarks of thalassemias are ineffective erythropoiesis and peripheral hemolysis leading to a cascade of events responsible for several clinical complications. This pathophysiologic mechanism can be partially controlled by blood transfusions or by correction of the severity of ineffective erythropoiesis. Thalassemias include a spectrum of phenotypes. Two main groups can be clinically distinguished: transfusion-dependent (TDT) and non-transfusion-dependent (NTDT) thalassemia. Both conditions are characterized by several clinical complications along life; some are shared, whereas some have higher prevalence in one group over the other...
April 2018: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/29458727/ineffective-erythropoiesis-anemia-and-iron-overload
#12
REVIEW
Ritama Gupta, Khaled M Musallam, Ali T Taher, Stefano Rivella
Stress erythropoiesis (SE) is characterized by an imbalance in erythroid proliferation and differentiation under increased demands of erythrocyte generation and tissue oxygenation. β-thalassemia represents a chronic state of SE, called ineffective erythropoiesis (IE), exhibiting an expansion of erythroid-progenitor pool and deposition of alpha chains on erythrocyte membranes, causing cell death and anemia. Concurrently, there is a decrease in hepcidin expression and a subsequent state of iron overload. There are substantial investigative efforts to target increased iron absorption under IE...
April 2018: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/29458726/clinical-classification-screening-and-diagnosis-for-thalassemia
#13
REVIEW
Vip Viprakasit, Supachai Ekwattanakit
At present, thalassemia diseases are classified into transfusion-dependent thalassemia and non-transfusion-dependent thalassemia. This classification is based on the clinical severity of patients determining whether they do require regular blood transfusions to survive (transfusion-dependent thalassemia) or not (non-transfusion-dependent thalassemia). In addition to the previous terminology of "thalassemia major" or "thalassemia intermedia," this classification has embraced all other forms of thalassemia syndromes such as α-thalassemia, hemoglobin E/β-thalassemia and combined α- and β-thalassemias...
April 2018: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/29458725/molecular-basis-and-genetic-modifiers-of-thalassemia
#14
REVIEW
Sachith Mettananda, Douglas R Higgs
Thalassemia is a disorder of hemoglobin characterized by reduced or absent production of one of the globin chains in human red blood cells with relative excess of the other. Impaired synthesis of β-globin results in β-thalassemia, whereas defective synthesis of α-globin leads to α-thalassemia. Despite being a monogenic disorder, thalassemia exhibits remarkable clinical heterogeneity that is directly related to the intracellular imbalance between α- and β-like globin chains. Novel insights into the genetic modifiers have contributed to the understanding of the correlation between genotype and phenotype and are being explored as therapeutic pathways to cure this life-limiting disease...
April 2018: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/29458724/the-evolving-spectrum-of-the-epidemiology-of-thalassemia
#15
REVIEW
David J Weatherall
The thalassemias and other inherited disorders of hemoglobin are likely to remain a serious global health problem for the foreseeable future. Currently, they are most frequent in the tropical belt; an assessment of their true frequency and the likely cost of management for the governments of these countries will require a form of micromapping. Over recent years, there has been major progress toward better prevention and management of the thalassemias in richer countries; it is likely that, using the tools of molecular genetics, they will eventually be completely curable, although this is probably a long time in the future...
April 2018: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/29157623/castleman-disease
#16
EDITORIAL
Frits van Rhee, Nikhil C Munshi
No abstract text is available yet for this article.
February 2018: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/29157622/treatment-of-idiopathic-castleman-disease
#17
REVIEW
Frits van Rhee, Amy Greenway, Katie Stone
Important progress has been made in the treatment of idiopathic multicentric Castleman disease (iMCD) with the introduction of interleukin-6 targeting monoclonal antibodies. This article describes the clinical results obtained with different treatment modalities and uses this evidence to provide treatment guidelines for the practicing clinician. Much is still to be learned about the pathophysiology of iMCD and further research is urgently needed to develop novel and curative treatment approaches for all patients...
February 2018: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/29157621/treatment-of-kaposi-sarcoma-herpesvirus-associated-multicentric-castleman-disease
#18
REVIEW
Kathryn Lurain, Robert Yarchoan, Thomas S Uldrick
Kaposi sarcoma herpesvirus (KSHV)-associated multicentric Castleman disease (MCD) is a rare, polyclonal lymphoproliferative disorder characterized by flares of inflammatory symptoms, edema, cytopenias, lymphadenopathy, and splenomegaly. Diagnosis requires a lymph node biopsy. Pathogenesis is related to dysregulated inflammatory cytokines, including human and viral interleukin-6. Rituximab alone or in combination with chemotherapy, such as liposomal doxorubicin, has led to an overall survival of over 90% at 5 years...
February 2018: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/29157620/unicentric-castleman-disease
#19
REVIEW
Raymond S M Wong
Unicentric Castleman disease (UCD) is a rare lymphoproliferative disorder that manifests typically as proliferation of a single lymph node or region of lymph nodes. Histologically, hyaline vascular variant is found in a majority of UCDs. UCD commonly presents in younger patient populations. Patients with UCD may be asymptomatic or present with symptoms related to mass effects on surrounding structures. It is difficult to achieve a definitive diagnosis by imaging alone. Histologic examination of the lesion remains the gold standard for diagnosis...
February 2018: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/29157619/diagnosis-of-castleman-disease
#20
REVIEW
Raphaël Szalat, Nikhil C Munshi
Castleman disease (CD) is a rare and heterogenous group of disorders sharing in common an abnormal lymph node pathology. CD comprises distinct subtypes with different prognoses. Unicentric CD and multicentric CD are featured by specific systemic manifestations and may be associated with Kaposi sarcoma, non-Hodgkin and Hodgkin lymphoma, and POEMS syndrome. Multicentric CD is classically associated with systemic symptoms and poorer prognosis. In this article, the authors review how to diagnose the disease, keeping in context the clinical findings, biochemical changes and complications associated with CD...
February 2018: Hematology/oncology Clinics of North America
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