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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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 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

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Thalassemia, An Issue of Hematology/Oncology Clinics of North America

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Edward J. BenzJr

Vijay G. Sankaran