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Congenital and Acquired Bone Marrow Failure

1st Edition - December 23, 2016

Editors: Mahmoud Deeb Aljurf, Eliane Gluckman, Carlo Dufour

Hardback ISBN:

9 7 8 - 0 - 1 2 - 8 0 4 1 5 2 - 9

eBook ISBN:

9 7 8 - 0 - 1 2 - 8 0 4 1 7 5 - 8

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… Read more

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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.

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

Mahmoud Deeb Aljurf

Dr. Mahmoud Aljurf is Professor of Medicine at Alfaisal University and the Director of Adult Stem Cell Transplant Program at King Faisal Specialist Hospital & Research Center in Riyadh, Saudi Arabia.

He received his M.D. degree in 1985. He completed residency training in Internal Medicine at Brigham and Women's Hospital, Harvard Medical School, and combined Hematology and Oncology Fellowships at Stanford University Medical Center. He also received Masters in Public Health (MPH) form Johns Hopkins University and FRCPath from UK. Dr. Aljurf is American Board Certified in Internal Medicine, Medical Oncology, Hematology, Medical Management and Quality Assurance. Dr. Aljurf has published over 150 scientific papers and several book chapters. He is the Scientific Director of the Eastern Mediterranean Blood and Marrow Transplantation Group (EMBMT), also the Editor in Chief for the journal Hematology/Oncology and Stem Cell Therapy. He was a recipient of the American College of Physician (ACP) Mastership Award for the year 2012. The King Faisal Specialist Hospital & Research Center in Riyadh, Saudi Arabia has one of the world’s largest units for treatment and transplantation of bone marrow failure patients.

Affiliations and expertise

Director, Adult HSCT Program; Deputy Director, Oncology Center, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia

Eliane Gluckman

Eliane Gluckman graduated from the medical school university in Paris. She specialized in clinical and research hematology. She was head of the Hematology Department and Bone Marrow Transplant unit in the Hospital Saint Louis, Paris from 1976 to 2005. She is a founding member and past president of EBMT. Currently, she is Professor Emeritus of the university Paris-Diderot, President of Eurocord, of the European School of Hematology, and past president of the World Marrow Association and Member of the Board of the World Blood and Marrow Transplantation Association and of Netcord. She has received many prizes and honours in France and abroad. She has published more than 800 peer reviewed articles in international journals. Her main research interests are: Allogeneic hematopoietic stem cell transplantation in children and adults, bone marrow transplants in children with inherited disorders including Fanconi anemia and sickle cell anemia. She was the first to perform cord blood transplantation in 1988. This first success led to the development of worldwide cord blood banks and helped to save the life of many patients who could not receive a transplant because of the lack of a donor. More recently she led the International observatory on sickle cell disease Monacord located at the Centre Scientifique de Monaco. The objective of the group is to develop and coordinate actions for establishing diagnosis, treatment tools and promote education for sickle cell disease all over the world including developing countries.

Affiliations and expertise

Professor and Head Eurocord-Monacord, University Paris-Diderot , APHP, President European School of Hematology, Paris, France

Carlo Dufour

Carlo Dufour graduated from the medical school university in Genova. He specialized in pediatrics and afterwards in clinical and research hematology. He attended the Department of Hematology of the Hammersmith Hospital, London, UK, (nowadays part of the Imperial College Heatltcare NHS) where he achieved the MsC degree in Haematology by the Royal Postgraduate Medical School, University of London. Within the G.Gaslini Children’s Hospital, Genova, Italy, the largest, multi specialist pediatric hospital of the country, he chairs the Hematology Unit where have seat the national Registry of Neutropenia, the national Registry of Alps and related disorders and the national Data Base of Fanconi Anemia. He founded the Marrow Failure Syndromes Study Group within the Italian Pediatric Hemato- Oncology Association. He is author or co-author of about 150 international publications and of various hematology textbook chapters. He acts as reviewer for top ranking international journals including New Englanf Journal of Medicine, Journal of Clinical Oncology, Clinical Immunology and Blood. He serves as expert evaluator for different institutions including the French Registry for Rare Diseases, the Fanconi Anemia Research Fund INC (US), the Leukemia and Lymphoma Research Foundation (UK), the American Society of Hematology Education Program. He is currently the chairman of the Working Party of the Severe Aplastic Anemia (WPSAA) and a Board member of the European Society for Blood and Marrow Transplantation (EBMT). He also serves as the chairman of the Scientific Working Group on Granulocyte and Monocyte Disorders of the European Hematology Association (EHA). He was recently part of the commission for the attribution of professorship position in molecular medicine by the University of London. In 2015 he received by the Fanconi Anemia Research Fund. INC the discovery award for participating to the identification of gene FANCT.

Affiliations and expertise

Chair, Clinical and Experimental Haematology Unit, Giannina Gaslini Children's Hospital, Genova, Italy