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CONGENITAL AND ACQUIRED BONE MARROW FAILURE (hb) 2017
Author: MAHMOUD DEEP ALJURF
ISBN: 9780128041529
Year: 2017
Publisher: ELSEVIER INC. USA
Category: MEDICINE
Edition: 1
Format: Hard Cover
Language: English
Pages: 274
 ABOUT THE TITLE
Congenital and Acquired Bone Marrow Failure is a comprehensive guide to congenital and acquired bone marrow failure in adult and pediatric patients. Chapters are divided into two sections, acquired aplastic anemia and inherited bone marrow failure syndromes. Content ranges from the basic, to the translational, and from the epidemiology of acquired aplastic anemia and telomere biology, to the management, treatment, and supportive care of pediatric, adult, and geriatric patients.

Contributors are world leading experts in the field of bone marrow failure. The book is required reading for residents, fellows, clinicians, and researchers across hematology, oncology, pathology, bone marrow transplantation, pediatrics, and internal medicine.

Key Features
Provides an overview of all congenital and acquired bone marrow failure syndromes
Focuses on the molecular pathogenesis, clinical manifestation and diagnosis, laboratory features, and treatment of each disease within the syndromes
Features the area of supportive care which is a topic of great interest to infectious disease physicians and those involved in transfusion services

Table of Contents
List of Contributors
Introduction
Chapter 1: Epidemiology of Acquired Bone Marrow Failure
Abstract
Introduction
Incidence of AA in different geographical regions and race
Age and gender related demographics of AA
Posthepatitis AA and AA occurring after viral infections
AA and association with toxins/drugs
AA and association with HLA genes
AA and autoimmune disorders
AA during pregnancy
AA postvaccination
Problems with epidemiological studies in AA and future strategies
Chapter 2: Pathophysiology of Acquired Bone Marrow Failure
Abstract
Introduction: Evidence and inferences from the clinic
Pathophysiology
Treatments for AA
Conclusions
Chapter 3: Diagnosis of Acquired Aplastic Anemia
Abstract
Introduction
Approach to diagnosis of aplastic anemia
Diagnosis confirmation
Characterization of aplastic anemia
Future challenges in the diagnostics of AA
Acknowledgment
Chapter 4: Acquired Overlap Bone Marrow Failure Disorders
Abstract
Introduction
Hypoplastic MDS
Single lineage cytopenias (pure red cell aplasia or immune thrombocytopenia)
T cell large granular lymphocytes
Paroxysmal nocturnal hemoglobinuria
Congenital marrow failure undiagnosed
Conclusions
Chapter 5: Supportive Care in Aplastic Anemia
Abstract
Introduction
Prevention of infections by general medical management of aplastic anemia patients
Prevention of infections by antibiotic/antimycotic/antiviral prophylaxis
Hematopoietic growth factors as prophylaxis of infections or in combination with immunosuppression to improve quality of response
Treatment of infections
Transfusion therapy
Iron chelation therapy
Physical exercise
Gender-specific issues/sex life
Psychological support
Chapter 6: Immunosuppressive Therapy for Aplastic Anemia
Abstract
The immune defect in aplastic anemia and the rationale for immunosuppressive therapy
Treatment options and indications for IST
ATG: possible mechanisms of action and administration
Historical development of the current standard ATG protocol (horse ATG combined with cyclosporine)
Alternative strategies used in an attempt to improve response to standard IST with ATG + CSA (Fig. 6.2)
The use of alemtuzumab in AA
Treatment of NSAA
Predictive factors for response to ATG
Repeat courses of ATG for nonresponse and relapse
Clonal transformation to MDS/AML after IST
Future directions
Chapter 7: Identical Sibling Donor Transplantation
Abstract
Introduction
Indication for identical sibling donor transplantation
Conditioning regimen
Syngeneic stem cell transplantation in aplastic anemia
The source of the stem cells
Posttransplantation immunosuppression
Posttransplant care
Chapter 8: Unrelated Donor Transplants for Acquired Aplastic Anemia
Abstract
Eligibility in acquired SAA for UD transplantation
Upper age limit for UD transplants
Outcome of patients activating a UD search
HLA matched or mismatched donors
Graft rejection and stem cell source
Cyclophosphamide and the conditioning regimen for UD transplants
An update of EBMT data on UD transplants
Alemtuzumab instead of ATG
Graft versus host disease prophylaxis
Improvement of UD transplants with time and supportive care
Conclusions
Chapter 9: Umbilical Cord Blood Transplantation for Patients With Acquired and Inherited Bone Marrow Failure Syndromes on Behalf of Eurocord
Abstract
Introduction
Candidates for cord blood transplantation for aplastic anemia
HLA-identical sibling cord blood transplant: Eurocord results
Unrelated cord blood transplantation for BMFS
Recommendations for cord blood transplantation in BMF
Future directions
Chapter 10: Haploidentical Transplantation
Abstract
Ex vivo T-cell depletion
Unmanipulated graft haplo-SCT
Conclusions
Chapter 11: Management of Acquired Aplastic Anemia in Children
Abstract
Diagnosis and clinical characteristics
Supportive treatment
General concepts for specific treatment
Options for first-line treatment
Options for second line treatments
Options for third line treatments
Chapter 12: Treatment of Elderly Patients With Aplastic Anemia
Abstract
Aging and its consequences on the approach to treatment
Comprehensive geriatric assessment
Treatment of aplastic anemia in the elderly
Open questions in the treatment of elderly patients with aplastic anemia
Conclusions
Acknowledgments
Chapter 13: Emerging New Therapies for Acquired Bone Marrow Failure Disorders
Abstract
Introduction
Alternative strategies of immunosuppression
Nonimmunosuppressive strategies
Combination strategies
Conclusions
Chapter 14: Bone Marrow Failure in Paroxysmal Nocturnal Hemoglobinuria
Abstract
Introduction
Pathophysiology of BMF in PNH
PNH clone in patients with BMF
Treatment
Conclusions
Chapter 15: Telomere Biology and Disease
Abstract
Introduction
Molecular biology of telomeres and telomerase
Genotype and phenotype in telomere disease
Bone marrow, organ failure, and malignancy in telomeropathies
Diagnosis of telomere disease
Conclusions
Chapter 16: Fanconi Anemia
Abstract
Introduction
Diagnosis and staging
Hematopoietic stem cell transplantation (HSCT)
Post-HCT monitoring in FA
Conclusions
Chapter 17: Ribosomopathies and the Quality Control of Ribosome Assembly
Abstract
Introduction
Diamond–Blackfan anemia
Shwachman–Diamond syndrome
Additional ribosomopathies
Conclusions
Acknowledgments
Chapter 18: Dyskeratosis Congenita
Abstract
Background
Pathobiology
Clinical features
Diagnosis
Management
Conclusions
Chapter 19: Amegakaryocytic Thrombocytopenia
Abstract
Introduction
Chapter 20: Severe Congenital Neutropenias and Other Rare Inherited Disorders With Marrow Failure
Abstract
Severe congenital neutropenia
Other rare diseases
Chapter 21: Bone Marrow Failure Syndromes in Children
Abstract
Introduction
Next generation sequencing for inherited BMFs
Childhood aplastic anemia and refractory cytopenia of childhood
The treatment algorithm for severe AA in children
Index

Author: Mahmoud Deeb Aljurf Eliane Gluckman Carlo Dufour
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