__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.

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zlib/Science (General)/Mahmoud Aljurf, Éliane Gluckman and Carlo Dufour (Eds.)/Congenital and Acquired Bone Marrow Failure_3307159.pdf

M. D Aljurf; Eliane Gluckman; C Dufour; European Group for Blood and Marrow Transplantation; European School of Hematology; Elsevier

Aljurf, Mahmoud Deeb; Gluckman, Eliane; Dufour, Carlo

Mahmoud Deeb Aljurf; Eliane Gluckman; Carlo Dufour

Eliane Gluckman; C Dufour; Mahmoud Deeb Aljurf

John E Cooper; Gordon Hull

Academic Press, Incorporated

Morgan Kaufmann Publishers

Elsevier, Academic Press

Brooks/Cole

United States, United States of America

Elsevier Ltd., London, 2017

Saint Louis, 2017

1, 2016

lg2065042

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Cover; Title Page; Copyright Page; Contents; List Of Contributors; Introduction; Chapter 1 -- Epidemiology Of Acquired Bone Marrow Failure; 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; References; Chapter 2 -- Pathophysiology Of Acquired Bone Marrow Failure. Introduction: Evidence And Inferences From The Clinicpathophysiology; Hematopoiesis In Aa; Immune Mechanisms In Aa; Clinical Data; T Cells And Cytokines; Hla And Cytokine Gene Polymorphisms; Immune Escape Clones (pnh, 6ploh); Stat3 Mutant Clones; Innate Immunity; Micrornas; Autoantibodies; Immune-mediated Bm Failure Mouse Models; Mouse Models Of Chemical And Drug Hematopoietic Toxicity; Genetic Risk Factors In Aa; Clonal Evolution In Aa; Treatments For Aa; Bmt With Matched Sibling Donors; Bmt From Alternative Donors; Experimental Hsct; Ist; Eltrombopag; Supportive Care; Conclusions. Aa And Hla-dr Typingaa And Lgl; Diagnostic Of Aa In The Elderly; Defining Severity Of Aa; Defining Response After Treatment; Future Challenges In The Diagnostics Of Aa; Acknowledgment; References; Chapter 4 -- Acquired Overlap Bone Marrow Failure Disorders; 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; References; Chapter 5 -- Supportive Care In Aplastic Anemia; Introduction. Prevention Of Infections By General Medical Management Of Aplastic Anemia Patientsprotective Environment; Protective Isolation; Special Low Bacterial Diet; Individual Hygiene Rules; Prevention Of Infections By Antibiotic/antimycotic/antiviral Prophylaxis; Antimycotic Prophylaxis; Antibiotic Prophylaxis; Antiviral Prophylaxis; Vaccination; Hematopoietic Growth Factors As Prophylaxis Of Infections Or In Combination With Immunosuppression To Improve Quality Of Re ... ; Treatment Of Infections; Granulocyte Transfusions; Transfusion Therapy; Iron Chelation Therapy; Physical Exercise. Edited By M.d. Aljurf, E. Gluckman, C. Dufour. Includes Bibliographical References And Index. Mode Of Access: World Wide Web.

Content:
Front matter,Copyright,List of Contributors,IntroductionEntitled to full textChapter 1 - Epidemiology of Acquired Bone Marrow Failure, Pages 1-11, F. Grimaldi, S. Gandhi, A.M. Risitano
Chapter 2 - Pathophysiology of Acquired Bone Marrow Failure, Pages 13-33, K. Hosokawa, P. Scheinberg, N.S. Young
Chapter 3 - Diagnosis of Acquired Aplastic Anemiaa, Pages 35-50, A. Rovó, C. Dufour, A. Tichelli
Chapter 4 - Acquired Overlap Bone Marrow Failure Disorders, Pages 51-56, J.R. Passweg
Chapter 5 - Supportive Care in Aplastic Anemia, Pages 57-71, B. Höchsmann, H. Schrezenmeier
Chapter 6 - Immunosuppressive Therapy for Aplastic Anemia, Pages 73-89, J.C.W. Marsh, S. Gandhi, G.J. Mufti
Chapter 7 - Identical Sibling Donor Transplantation, Pages 91-99, G. Aldawsari, H. Alzahrani, M.D. Aljurf
Chapter 8 - Unrelated Donor Transplants for Acquired Aplastic Anemia, Pages 101-110, A. Bacigalupo, S. Sica, M.T. Van Lint, C. Dufour
Chapter 9 - Umbilical Cord Blood Transplantation for Patients With Acquired and Inherited Bone Marrow Failure Syndromes on Behalf of Eurocord, Pages 111-119, E. Gluckman, A. Ruggeri, R. Peffault de Latour
Chapter 10 - Haploidentical Transplantation, Pages 121-125, F. Ciceri, M.T.L. Stanghellini
Chapter 11 - Management of Acquired Aplastic Anemia in Children, Pages 127-139, C. Dufour, S. Samarasinghe, M. Miano
Chapter 12 - Treatment of Elderly Patients With Aplastic Anemia, Pages 141-151, A. Tichelli, A. Rovó, J.C.W. Marsh
Chapter 13 - Emerging New Therapies for Acquired Bone Marrow Failure Disorders, Pages 153-167, A.M. Risitano, R. Peffault de Latour
Chapter 14 - Bone Marrow Failure in Paroxysmal Nocturnal Hemoglobinuria, Pages 169-180, R. Peffault de Latour, A.M. Risitano
Chapter 15 - Telomere Biology and Disease, Pages 181-194, J.N. Cooper, N.S. Young
Chapter 16 - Fanconi Anemia, Pages 195-201, R. Peffault de Latour, A.M. Risitano, E. Gluckman
Chapter 17 - Ribosomopathies and the Quality Control of Ribosome Assembly, Pages 203-224, A.J. Warren
Chapter 18 - Dyskeratosis Congenita, Pages 225-233, M. Ayas, S.O. Ahmed
Chapter 19 - Amegakaryocytic Thrombocytopenia, Pages 235-240, D. Meyran, T. Leblanc, S. Giraudier, J.H. Dalle
Chapter 20 - Severe Congenital Neutropenias and Other Rare Inherited Disorders With Marrow Failure, Pages 241-253, F. Fioredda, P. Farruggia, M. Miano, C. Dufour
Chapter 21 - Bone Marrow Failure Syndromes in Children, Pages 255-267, S. Elmahdi, S. Kojima
Index, Pages 269-276

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 -- Future directions -- References -- Chapter 10 - Haploidentical Transplantation -- Ex vivo T-cell depletion -- Unmanipulated graft haplo-SCT -- Conclusions -- References -- Chapter 11 - Management of Acquired Aplastic Anemia in Children -- Diagnosis and clinical characteristics -- Supportive treatment -- General concepts for specific treatment -- Options for first-line treatment -- HSCT from MSD -- Immunosuppressive Therapy -- HSCT from MUD -- Options for second line treatments -- Options for third line treatments -- Haploidentical HSCT -- Cord Blood Transplant -- Other Nontransplant Treatments -- References -- 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 -- Conclusions -- Acknowledgments -- References -- Chapter 13 - Emerging New Therapies for Acquired Bone Marrow Failure Disorders -- Introduction -- Alternative Strategies of Immunosuppression -- Adding a Third Immunosuppressive Agent -- Mycophenolate Mofetil -- Rapamycin/Sirolimus -- Replacing h-ATG With Other Lymphocyte-Depleting Agents -- Rabbit ATG -- Cyclophosphamide -- Alemtuzumab -- The Lesson From Alternative Lymphocyte-Depleting Agents -- Novel Strategies Without Conventional Lymphocyte Depletion -- Nonimmunosuppressive Strategies -- Androgens -- Eltrombopag -- Combination Strategies -- Conclusions -- References

Chapter 14 - Bone Marrow Failure in Paroxysmal Nocturnal Hemoglobinuria -- Introduction -- 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 -- Pathophysiology -- Risk of Thrombosis -- Evolution to Classic PNH -- Treatment -- Immunosuppressive Therapy -- Hematopoietic Stem Cell Transplantation -- Eculizumab Treatment -- Conclusions -- References -- Chapter 15 - Telomere Biology and Disease -- Introduction -- Molecular biology of telomeres and telomerase -- Genotype and phenotype in telomere disease -- Telomerase Complex Mutations -- Shelterin Complex Mutations -- Additional Mutations -- Bone marrow, organ failure, and malignancy in telomeropathies -- Bone Marrow Failure -- Pulmonary Disease -- Liver Disease -- Telomeres and Malignancy Risk -- Diagnosis of telomere disease -- Conclusions -- References -- Chapter 16 - Fanconi Anemia -- Introduction -- Diagnosis and staging -- Clinical Presentation -- Evaluating New Onset Cytopenia in Children -- Diagnosis and Staging -- Hematopoietic stem cell transplantation (HSCT) -- HLA Identical Sibling Transplants -- Alternative Donor Transplants -- Matched Unrelated Donors -- Cord Blood Transplantation -- FA Patients With MDS and AML -- Post-HCT monitoring in FA -- Conclusions -- References -- Chapter 17 - Ribosomopathies and the Quality Control of Ribosome Assembly -- Introduction -- Diamond-Blackfan anemia -- Clinical Features -- Diagnostic Criteria -- Genetics -- Molecular Pathophysiology -- Management -- Shwachman-Diamond syndrome -- Clinical Features -- Hematology -- Solid Tumors -- Gastrointestinal Tract -- Skeletal -- Neurocognitive -- Cardiac -- Miscellaneous -- Diagnosis -- Clinical Management

AA and Association With Autoimmune Diseases -- AA and HLA-DR Typing -- AA and LGL -- Diagnostic of AA in the Elderly -- Defining Severity of AA -- Defining Response After Treatment -- Future challenges in the diagnostics of AA -- Acknowledgment -- References -- Chapter 4 - Acquired Overlap Bone Marrow Failure Disorders -- 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 -- References -- Chapter 5 - Supportive Care in Aplastic Anemia -- Introduction -- Prevention of infections by general medical management of aplastic anemia patients -- Protective Environment -- Protective Isolation -- Special Low Bacterial Diet -- Individual Hygiene Rules -- Prevention of infections by antibiotic/antimycotic/antiviral prophylaxis -- Antimycotic Prophylaxis -- Antibiotic Prophylaxis -- Antiviral Prophylaxis -- Vaccination -- Hematopoietic growth factors as prophylaxis of infections or in combination with immunosuppression to improve quality of re ... -- Treatment of infections -- Granulocyte Transfusions -- Transfusion therapy -- Iron chelation therapy -- Physical exercise -- Gender-specific issues/sex life -- Menstrual Cycle -- Fertility -- Pregnancy -- Pregnancy Prevention -- Psychological support -- References -- 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

Cover -- Title page -- Copyright page -- Contents -- List of Contributors -- Introduction -- Chapter 1 - Epidemiology of Acquired Bone Marrow Failure -- 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 -- References -- Chapter 2 - Pathophysiology of Acquired Bone Marrow Failure -- Introduction: Evidence and Inferences from the Clinic -- Pathophysiology -- Hematopoiesis in AA -- Immune Mechanisms in AA -- Clinical Data -- T Cells and Cytokines -- HLA and Cytokine Gene Polymorphisms -- Immune Escape Clones (PNH, 6pLOH) -- STAT3 Mutant Clones -- Innate Immunity -- microRNAs -- Autoantibodies -- Immune-Mediated BM Failure Mouse Models -- Mouse Models of Chemical and Drug Hematopoietic Toxicity -- Genetic Risk Factors in AA -- Clonal Evolution in AA -- Treatments for AA -- BMT With Matched Sibling Donors -- BMT From Alternative Donors -- Experimental HSCT -- IST -- Eltrombopag -- Supportive Care -- Conclusions -- References -- Chapter 3 - Diagnosis of Acquired Aplastic Anemiaa -- Introduction -- Approach to diagnosis of aplastic anemia -- Diagnosis confirmation -- Clinical Examination -- Complete Blood Count -- Bone Marrow Examination -- Cytogenetic -- Molecular Analysis -- Telomere Length Measurement -- HLA-Typing -- Characterization of aplastic anemia -- Exclusion of Congenital Bone Marrow Failures -- Differential Diagnosis of AA From the Hypocellular Variant of MDS -- AA and PNH -- AA and Viral Infections -- AA and Hepatitis -- Drug-Induced AA -- AA Associated With Chemicals

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 -- Conclusions -- 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 -- References -- Chapter 7 - Identical Sibling Donor Transplantation -- 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 -- Chimerism -- Survival -- Fertility -- Secondary Malignancy -- References -- 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 -- Conclusions -- References -- Chapter 9 - Umbilical Cord Blood Transplantation for Patients With Acquired and Inherited Bone Marrow Failure Syndromes on Beha ... -- Introduction -- 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

<p><i>Congenital and Acquired Bone Marrow Failure</i> 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. </p> <p>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.</p><ul> <li>Provides an overview of all congenital and acquired bone marrow failure syndromes</li> <li>Focuses on the molecular pathogenesis, clinical manifestation and diagnosis, laboratory features, and treatment of each disease within the syndromes</li> <li>Features the area of supportive care which is a topic of great interest to infectious disease physicians and those involved in transfusion services</li></ul>

2017-08-08