Congenital and Acquired Bone Marrow Failure 1st edition by Mahmoud Aljurf,Eliane Gluckman,Carlo Dufour 9780128041529 0128041528

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ISBN 10: 0128041528

ISBN 13:9780128041529 

Author: Mahmoud Aljurf,Eliane Gluckman,Carlo Dufour

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.

• 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

 

Congenital and Acquired Bone Marrow Failure 1st Table of contents: 

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

 

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