Cooley's Anemia

Ninth Symposium, Volume 1202
1. Auflage 2010
ISBN: 978-1-57331-782-5
Verlag: Wiley

Buch, Englisch, 300 Seiten, Format (B × H): 175 mm x 249 mm, Gewicht: 567 g

Thanks to scientific advances, individuals with thalassemia—a group of genetic blood disorders which includes Cooley's Anemia — are now living into their 40's and 50's. Not only are individuals living longer, but their quality of life has increased. Scientific and clinical advancements have resulted in new iron-chelating drugs, early detection of organ failure, an understanding of adult complications associated with living with thalassemia (osteoporosis, heart failure, growth hormone deficiency, pulmonary hypertension, and infertility) and promising progress towards the ultimate magic bullet—a cure in the form of bone marrow and cord blood transplants, or gene therapy.


This volume integrates basic science and clinical research so that both scientists and clinicians can develop a mutual understanding of recent progress in thalassemia.

NOTE: Annals volumes are available for sale as individual books or as a journal. For information on institutional journal subscriptions, please visit www.blackwellpublishing.com/nyas.

Autoren/Hrsg.

Vichinsky, Elliott P

Neufeld, Ellis

Fachgebiete

• Medizin | Veterinärmedizin Medizin | Public Health | Pharmazie | Zahnmedizin Klinische und Innere Medizin Hämatologie, Transfusionsmedizin

Inhaltsverzeichnis

Clinical trials in thalassemia: insights from the patient community
Pathogenesis and management of iron toxicity in thalassemia

The role of antioxidants and iron chelators in the treatment of oxidative stress in thalassemia

Thalassemia as a global health problem: recent progress toward its control in the developing countries

Iron metabolism and ineffective erythropoiesis in [beta]-thalassemia mouse models

Hepcidin in [beta]-thalassemia

Therapy for [beta]-globinopathies: a brief review and determinants for successful and safe correction

Humanized mouse models of Cooley's anemia: correct fetal-to-adult hemoglobin switching, disease onset, and disease pathology

Strategy for a multicenter phase I clinical trial to evaluate globin gene transfer in [beta]-thalassemia

Hematopoietic stem cell mobilization strategies for gene therapy of beta thalassemia and sickle cell disease

Transcriptional silencing of fetal hemoglobin by BCL11A

The challenge of obtaining therapeutic levels of genetically modified hematopoietic stem cells in [beta]-thalassemia patients

Deferiprone

Combined iron chelation therapy

Deferasirox: current knowledge and future challenges

Monitoring the efficiency of iron chelation therapy: the potential of nontransferrin-bound iron

Critical appraisal of growth retardation and pubertal disturbances in thalassemia

Magnetic resonance assessment of iron overload by separate measurement of tissue ferritin and hemosiderin iron Predicting pituitary iron and endocrine dysfunction

Better survival and less cardiac morbidity in female patients with thalassemia major: a review of the literature. A preclinical approach for gene therapy of [beta]-thalassemia

The role of reduced intensity preparative regimens in patients with thalassemia given hematopoietic transplantation

Progress in hematopoietic stem cell transplantation as allogeneic cellular gene therapy in thalassemia

Emerging insights in the management of hemoglobin E beta thalassemia

Fetal globin gene inducers: novel agents and new potential

Detection of the cardiovascular complications of thalassemia by echocardiography

Cardiovascular MRI in thalassemia major

Complexity of alpha thalassemia: growing health problem with new approaches to screening, diagnosis, and therapy

Nutritional deficiencies in patients with thalassemia

Ferritin iron minerals are chelator targets, antioxidants, and coated, dietary iron

Pulmonary hypertension in thalassemia

The natural history of thalassemia intermedia

Hepcidin and Hfe in iron overload in [beta]-thalassemia

Fertility potential in thalassemia major women: current findings and future diagnostic tools

Redefining thalassemia as a hypercoagulable state

Treatment options for thalassemia patients with osteoporosis

Health care transition in thalassemia: pediatric to adult-oriented care

Ninth Cooley's Anemia Symposium: summary and perspective.

Clinical trials in thalassemia: insights from the patient community

Pathogenesis and management of iron toxicity in thalassemia

The role of antioxidants and iron chelators in the treatment of oxidative stress in thalassemia

Thalassemia as a global health problem: recent progress toward its control in the developing countries

Iron metabolism and ineffective erythropoiesis in ß-thalassemia mouse models

Hepcidin in ß-thalassemia

Therapy for ß-globinopathies: a brief review and determinants for successful and safe correction

Humanized mouse models of Cooley's anemia: correct fetal-to-adult hemoglobin switching, disease onset, and disease pathology

Strategy for a multicenter phase I clinical trial to evaluate globin gene transfer in ß-thalassemia

Hematopoietic stem cell mobilization strategies for gene therapy of beta thalassemia and sickle cell disease

Transcriptional silencing of fetal hemoglobin by BCL11A

The challenge of obtaining therapeutic levels of genetically modified hematopoietic stem cells in ß-thalassemia patients

Deferiprone

Combined iron chelation therapy

Deferasirox: current knowledge and future challenges

Monitoring the efficiency of iron chelation therapy: the potential of nontransferrin-bound iron

Critical appraisal of growth retardation and pubertal disturbances in thalassemia

Magnetic resonance assessment of iron overload by separate measurement of tissue ferritin and hemosiderin iron

Predicting pituitary iron and endocrine dysfunction

Better survival and less cardiac morbidity in female patients with thalassemia major: a review of the literature.

A preclinical approach for gene therapy of ß-thalassemia

The role of reduced intensity preparative regimens in patients with thalassemia given hematopoietic transplantation

Progress in hematopoietic stem cell transplantation as allogeneic cellular gene therapy in thalassemia

Emerging insights in the management of hemoglobin E beta thalassemia

Fetal globin gene inducers: novel agents and new potential

Detection of the cardiovascular complications of thalassemia by echocardiography

Cardiovascular MRI in thalassemia major

Complexity of alpha thalassemia: growing health problem with new approaches to screening, diagnosis, and therapy

Nutritional deficiencies in patients with thalassemia

Ferritin iron minerals are chelator targets, antioxidants, and coated, dietary iron

Pulmonary hypertension in thalassemia

The natural history of thalassemia intermedia

Hepcidin and Hfe in iron overload in ß-thalassemia

Fertility potential in thalassemia major women: current findings and future diagnostic tools

Redefining thalassemia as a hypercoagulable state

Treatment options for thalassemia patients with osteoporosis

Health care transition in thalassemia: pediatric to adult-oriented care

Ninth Cooley's Anemia Symposium: summary and perspective

Über Autor(innen)

Elliott P. Vichinsky is the editor of Cooley's Anemia: Ninth Symposium, Volume 1202, published by Wiley. Ellis Neufeld is the editor of Cooley's Anemia: Ninth Symposium, Volume 1202, published by Wiley.