Chapter
Posthepatitis AA and AA occurring after viral infections
AA and association with toxins/drugs
AA and association with HLA genes
AA and autoimmune disorders
Problems with epidemiological studies in AA and future strategies
Chapter 2 - Pathophysiology of Acquired Bone Marrow Failure
Introduction: Evidence and Inferences from the Clinic
HLA and Cytokine Gene Polymorphisms
Immune Escape Clones (PNH, 6pLOH)
Immune-Mediated BM Failure Mouse Models
Mouse Models of Chemical and Drug Hematopoietic Toxicity
Genetic Risk Factors in AA
BMT With Matched Sibling Donors
BMT From Alternative Donors
Chapter 3 - Diagnosis of Acquired Aplastic Anemiaa
Approach to diagnosis of aplastic anemia
Telomere Length Measurement
Characterization of aplastic anemia
Exclusion of Congenital Bone Marrow Failures
Differential Diagnosis of AA From the Hypocellular Variant of MDS
AA Associated With Chemicals
AA and Association With Autoimmune Diseases
Diagnostic of AA in the Elderly
Defining Response After Treatment
Future challenges in the diagnostics of AA
Chapter 4 - Acquired Overlap Bone Marrow Failure Disorders
Single lineage cytopenias (pure red cell aplasia or immune thrombocytopenia)
T cell large granular lymphocytes
Paroxysmal nocturnal hemoglobinuria
Congenital marrow failure undiagnosed
Chapter 5 - Supportive Care in Aplastic Anemia
Prevention of infections by general medical management of aplastic anemia patients
Special Low Bacterial Diet
Prevention of infections by antibiotic/antimycotic/antiviral prophylaxis
Hematopoietic growth factors as prophylaxis of infections or in combination with immunosuppression to improve quality of re...
Gender-specific issues/sex life
Chapter 6 - Immunosuppressive Therapy for Aplastic Anemia
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
ATG and CSA Combined With GCSF
ATG and CSA Combined With Additional Immunosuppressive Agents
Use of Rabbit ATG Instead of Horse ATG in First Line IST
Variations in CSA Dose and Duration
Addition of Eltrombopag to ATG and CSA
The use of alemtuzumab in AA
Predictive factors for response to ATG
Repeat courses of ATG for nonresponse and relapse
Clonal transformation to MDS/AML after IST
Chapter 7 - Identical Sibling Donor Transplantation
Indication for identical sibling donor transplantation
Syngeneic stem cell transplantation in aplastic anemia
The source of the stem cells
Posttransplantation immunosuppression
Chapter 8 - Unrelated Donor Transplants for Acquired Aplastic Anemia
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
Chapter 9 - Umbilical Cord Blood Transplantation for Patients With Acquired and Inherited Bone Marrow Failure Syndromes on Beha...
Candidates for cord blood transplantation for aplastic anemia
HLA-identical sibling cord blood transplant: Eurocord results
Unrelated cord blood transplantation for BMFS
Results of Unrelated Cord Blood Transplants for Idiopathic Aplastic Anemia
Results of Unrelated Cord Blood Transplants for Fanconi Anemia
Results of Cord Blood Transplant for Hereditary BMF Other than Fanconi Anemia
Recommendations for cord blood transplantation in BMF
Chapter 10 - Haploidentical Transplantation
Unmanipulated graft haplo-SCT
Chapter 11 - Management of Acquired Aplastic Anemia in Children
Diagnosis and clinical characteristics
General concepts for specific treatment
Options for first-line treatment
Immunosuppressive Therapy
Options for second line treatments
Options for third line treatments
Other Nontransplant Treatments
Chapter 12 - Treatment of Elderly Patients With Aplastic Anemia
Aging and Its Consequences on the Approach to Treatment
Comprehensive Geriatric Assessment
Treatment of Aplastic Anemia in the Elderly
Immunosuppression in the Elderly
Algorithm for First-Line Treatment of Elderly Aplastic Anemia Patients
Transplantation in the Elderly
Open Questions in the Treatment of Elderly Patients With Aplastic Anemia
Chapter 13 - Emerging New Therapies for Acquired Bone Marrow Failure Disorders
Alternative Strategies of Immunosuppression
Adding a Third Immunosuppressive Agent
Replacing h-ATG With Other Lymphocyte-Depleting Agents
The Lesson From Alternative Lymphocyte-Depleting Agents
Novel Strategies Without Conventional Lymphocyte Depletion
Nonimmunosuppressive Strategies
Chapter 14 - Bone Marrow Failure in Paroxysmal Nocturnal Hemoglobinuria
Pathophysiology of BMF in PNH
Derangement of the Immune System
The Dual Pathophysiology Theory
The Role of Somatic Mutations
PNH clone in patients with BMF
PNH Disease Subcategories
The Clinical Relevance of a PNH Clone in the Context of BMF
Immunosuppressive Therapy
Hematopoietic Stem Cell Transplantation
Chapter 15 - Telomere Biology and Disease
Molecular biology of telomeres and telomerase
Genotype and phenotype in telomere disease
Telomerase Complex Mutations
Shelterin Complex Mutations
Bone marrow, organ failure, and malignancy in telomeropathies
Telomeres and Malignancy Risk
Diagnosis of telomere disease
Chapter 16 - Fanconi Anemia
Evaluating New Onset Cytopenia in Children
Hematopoietic stem cell transplantation (HSCT)
HLA Identical Sibling Transplants
Alternative Donor Transplants
Cord Blood Transplantation
FA Patients With MDS and AML
Post-HCT monitoring in FA
Chapter 17 - Ribosomopathies and the Quality Control of Ribosome Assembly
Molecular Pathophysiology
Shwachman–Diamond syndrome
Function of SBDS in Large Ribosomal Subunit Maturation
Alternative Models for eIF6 Release
Putative Extraribosomal Functions of the SBDS Protein
Visualizing SBDS and EFL1 Bound to the Ribosome
Interpreting Pathologic SBDS Missense Variants
Interpreting uL16 (RPL10) Mutations in Pediatric T-ALL
Proposed Mechanism of eIF6 Release by SBDS and the GTPase EFL1
SBDS and the Quality Control of Ribosome Assembly
How Do We Explain the SDS Phenotype?
Additional ribosomopathies
Chapter 18 - Dyskeratosis Congenita
Immune Suppressive Therapy
Hematopoietic Cell Transplantation
Choice of a Suitable Conditioning Regimen
Choice of a Suitable Donor
Unrelated cord blood transplantation
Haploidentical transplantation
Does Transplantation Accelerate Pulmonary Complications?
Conditioning and Cancer Risk
Chapter 19 - Amegakaryocytic Thrombocytopenia
Clinical Manifestation and Diagnosis
Treatment and Supportive Care
Graft Versus Host Disease Prophylaxis
Chapter 20 - Severe Congenital Neutropenias and Other Rare Inherited Disorders With Marrow Failure
Severe congenital neutropenia
Definition, Epidemiology, and Genetics
Physiopathologic Mechanism
Diagnosis and Clinical Phenotype
Definition, Epidemiology, Genetics, and Physiopathologic Mechanism
Clinical Picture, Diagnosis, and Differential Diagnosis
Definition, Epidemiology, and Pathogenic Mechanism
Congenital Dyserythropoietic Anemia
Chapter 21 - Bone Marrow Failure Syndromes in Children
Next generation sequencing for inherited BMFs
Childhood aplastic anemia and refractory cytopenia of childhood
The treatment algorithm for severe AA in children
Congenital and Acquired Bone Marrow Failure
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