Thalassemia, An Issue of Hematology/Oncology Clinics of North America
- 1st Edition, Volume 37-2 - March 16, 2023
- Editors: Edward J. BenzJr, Vijay G. Sankaran
- Language: English
- Hardback ISBN:9 7 8 - 0 - 4 4 3 - 1 8 3 2 0 - 1
- eBook ISBN:9 7 8 - 0 - 4 4 3 - 1 8 3 2 1 - 8
In this issue of Hematology/Oncology Clinics, guest editors Drs. Edward J. Benz, Jr. and Vijay G. Sankaran bring their considerable expertise to the topic of Thalassemia. Top… Read more
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Request a sales quoteIn this issue of Hematology/Oncology Clinics, guest editors Drs. Edward J. Benz, Jr. and Vijay G. Sankaran bring their considerable expertise to the topic of Thalassemia. Top experts in the field provide an overview of the history, epidemiology, pathogenic mechanisms, and clinical management of the thalassemia syndromes.
- Cover image
- Title page
- Table of Contents
- Copyright
- Contributors
- Forthcoming Issues
- Preface
- Dedication
- Introduction to the Thalassemia Syndromes: Molecular Medicine’s Index Case
- Key points
- Introduction
- The Thalassemia Syndromes: the Consequences of Unbalanced Expression of the Individual Globin Genes
- The Thalassemias as Molecular Medicine’s Index Case
- Clinics care point
- The Need for Translational Epidemiology in Beta Thalassemia Syndromes: A Thalassemia International Federation Perspective
- Introduction
- Epidemiology-based planning of services
- Methodology and sources of data collected by Thalassaemia International Federation
- Epidemiology of beta thalassemia
- Discussion
- Clinics care points
- Molecular Basis and Genetic Modifiers of Thalassemia
- Key points
- Introduction
- α-thalassemia
- β-thalassemia
- Molecular techniques for the diagnosis of thalassemia syndromes
- Cell and gene therapy strategies for the cure of thalassemia
- Summary
- Clinics care points
- Fetal Hemoglobin Regulation in Beta-Thalassemia
- Key points
- Introduction
- Hemoglobin switching and the β-hemoglobinopathies
- The clinical benefit of fetal hemoglobin
- Molecular regulation of fetal hemoglobin
- New regulators of fetal hemoglobin
- Concluding remarks
- Clinics care points
- Disclosure
- Clinical Classification, Screening, and Diagnosis in Beta-Thalassemia and Hemoglobin E/Beta-Thalassemia
- Key points
- Introduction
- Clinical classification
- Summary
- Clinics care points
- The Clinical Phenotypes of Alpha Thalassemia
- Key points
- Introduction
- Diagnosis
- Summary
- Clinics care points
- Pathogenic Mechanisms in Thalassemia I: Ineffective Erythropoiesis and Hypercoagulability
- Key points
- Introduction
- The process of erythropoiesis and its regulation
- Ineffective erythropoiesis in β-thalassemia
- Hypercoagulability and vascular disease in beta-thalassemia
- Summary
- Clinics care points
- Disclosure
- Pathogenic Mechanisms in Thalassemia II: Iron Overload
- Key points
- Introduction/history/definition/background
- Summary
- Clinics care points
- Clinical Complications and Their Management
- Key points
- Introduction
- Autoimmune hemolytic anemia
- Cardiac complications
- Cholelithiasis
- Endocrine complications
- Extramedullary hematopoietic pseudotumors
- Hypercoagulability and vascular disease
- Leg ulcers
- Liver complications
- Malignancy
- Osteoporosis and bone disease
- Pain
- Pulmonary hypertension
- Renal dysfunction
- Summary
- Clinics care points
- Disclosure
- Clinical Challenges with Iron Chelation in Beta Thalassemia
- Key points
- Introduction
- Adherence
- Variable pharmacokinetics
- Adverse effects of iron chelators
- Challenges with assessing chelation response using serum ferritin levels
- COST AND ACCESS
- Clinics care points
- Disclosure
- Fertility and Pregnancy in Women with Transfusion-Dependent Thalassemia
- Key points
- Introduction
- Pathophysiology of subfertility
- Pregnancies in women with transfusion-dependent thalassemia
- Pregnancies in women with non–transfusion-dependent thalassemia and alpha-thalassemia
- Planning for pregnancy
- Fertility
- Management during pregnancy
- Options to Attaining a Family
- Summary
- Hematopoietic Stem Cell Transplantation in Thalassemia
- Key points
- Introduction
- Pretransplant risk stratification
- Advances in conditioning regimen
- Donor selection and stem cell source
- Conclusions and future perspectives
- Clinics care points
- Disclosures
- Gene Therapy and Gene Editing for β-Thalassemia
- Key points
- Introduction
- Summary
- Clinics care points
- Emerging Therapies in β-Thalassemia
- Key points
- Introduction
- Correction of the α/β-globin chain imbalance
- Targeting ineffective erythropoiesis
- Targeting iron dysregulation
- Summary
- Disclosure
- Clinics care points
- No. of pages: 240
- Language: English
- Edition: 1
- Volume: 37-2
- Published: March 16, 2023
- Imprint: Elsevier
- Hardback ISBN: 9780443183201
- eBook ISBN: 9780443183218
EB
Edward J. BenzJr
Affiliations and expertise
Professor, Pediatrics, Richard and Susan Smith Professor, Medicine, Professor, Genetics,Harvard Medical School ,President and CEO, Emeritus, Office of the President, Dana-Farber Cancer InstituteVS
Vijay G. Sankaran
Affiliations and expertise
Jan Ellen Paradise, M.D. Associate Professor of Pediatrics, Harvard Medical School Lodish Family Chair, Division of Hematology/Oncology, Boston Children's Hospital Robertson Investigator, New York Stem Cell , Foundation, Principal Faculty, Harvard Stem Cell Institute, Attending Physician, Dana Farber/Boston Children's Cancer and Blood Disorders Center, Associate Member, Broad Institute of MIT and Harvard