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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:9780128041529
9 7 8 - 0 - 1 2 - 8 0 4 1 5 2 - 9
eBook ISBN:9780128041758
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.
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
Residents, fellows, professional clinicians and researchers in hematology, oncology, pathology, bone marrow transplantation, pediatrics, 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)
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
No. of pages: 274
Language: English
Published: December 23, 2016
Imprint: Elsevier
Hardback ISBN: 9780128041529
eBook ISBN: 9780128041758
MA
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
EG
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
CD
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