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The EBMT Handbook : Hematopoietic Stem Cell Transplantation and Cellular Therapies.

Bibliographic Details
Main Author: Carreras, Enric.
Other Authors: Dufour, Carlo., Mohty, Mohamad., Kröger, Nicolaus.
Format: eBook
Language:English
Published: Cham : Springer International Publishing AG, 2019.
Edition:7th ed.
Subjects:
Electronic books.
Online Access:Click to View
Table of Contents:
  • Intro
  • Preface
  • Acknowledgment
  • Contents
  • Contributors
  • About the Editors
  • Abbreviations
  • Part I: Introduction Topic leaders: Mohamad Mohty and Jane Apperley
  • 1: HSCT: Historical Perspective
  • 1.1 Introduction
  • 1.2 Early Enthusiasm and Disappointment
  • 1.3 Back to the Laboratory: Focus on Animal Studies
  • 1.4 Resuming Clinical Transplantation: 1968-1980s
  • 1.5 Moving Ahead: The 1990s and Beyond
  • References
  • 2: The EBMT: History, Present, and Future
  • 2.1 Introduction
  • 2.2 The Past: Development of HSCT and EBMT
  • 2.3 The Present
  • 2.4 The Future
  • References
  • 3: The Role of Unrelated Donor Registries in HSCT
  • 3.1 Introduction
  • 3.1.1 From Anthony Nolan to 32 Million Volunteer Donors Worldwide
  • 3.1.2 Registry: Structure and Duties
  • 3.2 Current Landscape
  • 3.2.1 Ethnic Diversity and Chance to Find a Donor
  • 3.2.2 Donor Profile
  • 3.2.3 Recruitment, Retention and Data Confidentiality
  • 3.3 Connections and Worldwide Collaboration
  • 3.3.1 WMDA
  • 3.3.2 Quality and Accreditation
  • 3.3.3 Network Formalities
  • 3.4 Challenges and Opportunities
  • 3.4.1 Donor Attrition
  • 3.4.2 Ethical Challenges
  • 3.4.3 Donor Pool HLA Diversity
  • 3.5 Future Developments
  • 3.5.1 New Level of HLA Matching
  • 3.5.2 Related Donors Provision and Follow-Up
  • 3.5.3 Advisory Services Provided by Registries
  • References
  • 4: The HSCT Unit
  • 4.1 Introduction
  • 4.2 Inpatient Unit
  • 4.3 Ancillary Medical Services
  • 4.4 Outpatient Unit
  • 4.5 Blood Bank
  • 4.6 Laboratory
  • 4.7 HLA Typing Lab
  • 4.8 Stem Cell Collection
  • 4.9 Stem Cell Processing Facility
  • 4.10 Radiology
  • 4.11 Pharmacy
  • 4.12 Staffing and Human Resources
  • 4.13 Institutional Database and Data Manager
  • 4.14 Quality Control
  • 4.15 Transplant Coordinator
  • Recommended References.
  • 5: JACIE Accreditation of HSCT Programs
  • 5.1 Introduction
  • 5.2 Background
  • 5.3 Impact of Accreditation in Clinical Practice
  • 5.4 JACIE-FACT Accreditation System
  • References
  • 6: Statistical Methods in HSCT and Cellular Therapies
  • 6.1 Introduction
  • 6.2 Endpoints
  • 6.3 Analysis of Time-to-Event Outcomes
  • 6.3.1 Kaplan-Meier Curves
  • 6.3.2 Cumulative Incidence Curves
  • 6.3.3 Comparison of Groups
  • 6.3.4 Proportional Hazards Regression Analysis
  • 6.4 Advanced Methods
  • 6.4.1 Multistate Models
  • 6.4.2 Random Effect Models
  • 6.4.3 Long-Term Outcomes: Relative Survival/Cure Models
  • 6.4.4 Propensity Scores
  • 6.4.5 Methods for Missing Values
  • References
  • Part II: Biological Aspects Topic leaders: Chiara Bonini and Jürgen Kuball
  • 7: Biological Properties of HSC: Scientific Basis for HSCT
  • 7.1 Introduction
  • 7.2 Self-Renewal
  • 7.3 Commitment and Differentiation: New Data Challenge the Historical View of Hematopoietic Hierarchy
  • 7.4 The Bone Marrow Niches and Maintenance of Stemness (Fig. 7.1)
  • 7.5 Preclinical Models of HSCT
  • 7.6 Gene Transfer/Gene Editing/Gene Therapy Targeting HSC (Fig. 7.2)
  • 7.7 Studying Dynamics of Hematopoietic Reconstitution upon HSCT (Fig. 7.3)
  • 7.8 From Experimental Hematology to Medical Practices and Hematopoietic Cellular Therapies
  • References
  • 8: Biological Properties of Cells Other Than HSCs
  • 8.1 Introduction
  • 8.2 Conventional or Alpha-Beta T Cells
  • 8.3 Unconventional T Cells
  • 8.4 NK Cells
  • 8.5 Mesenchymal Stromal Cells
  • References
  • 9: Histocompatibility
  • 9.1 Introduction
  • 9.2 The Biology of Histocompatibility
  • 9.2.1 Major Histocompatibility Antigens
  • 9.2.2 HLA Class I and II Structure and Function
  • 9.2.3 HLA Polymorphism and Tissue Typing
  • 9.2.4 T Cell Alloreactivity
  • 9.3 HLA Matching in Allogeneic HSCT.
  • 9.3.1 Donor Types
  • 9.3.2 Clinical Impact of HLA Mismatches
  • 9.3.3 Models of High-Risk/Nonpermissive HLA Mismatches
  • 9.3.4 Guidelines for UD Selection by Histocompatibility
  • 9.4 Non-HLA Immunogenetic Factors
  • 9.4.1 Overview
  • 9.4.2 Clinical Impact of Non-HLA Immunogenetic Factors
  • References
  • 10: Clinical and Biological Concepts for Mastering Immune Reconstitution After HSCT: Toward Practical Guidelines and Greater Harmonization
  • 10.1 Introduction/Background
  • 10.2 Impact of Conditioning Regimens on Immune Reconstitution and Outcomes: Pharmacokinetics-Pharmacodynamics (PK-PD), Individualized Dosing
  • 10.3 Graft Composition as an Additional Predictor for Immune Reconstitution and Clinical Outcomes
  • 10.4 Immune Monitoring
  • 10.4.1 Immune Cell Phenotyping
  • 10.4.2 Immune Monitoring: Secretome Analyses
  • 10.5 Summary
  • References
  • Part III: Methodology and Clinical Aspects Topic leaders: Arnon Nagler and Nicolaus Kröger
  • 11: Evaluation and Counseling of Candidates
  • 11.1 Evaluation of Candidates and Risk Factors for HSCT
  • 11.1.1 Introduction
  • 11.1.2 Candidates' Evaluation Work Flow
  • 11.1.2.1 First Visit
  • 11.1.2.2 Visit Preharvesting (Auto-HSCT)
  • 11.1.2.3 Last Visit Before Admission
  • 11.1.2.4 Medical History
  • 11.1.2.5 Information to Provide (See Detailed Information in Counseling Section)
  • 11.1.3 Complementary Explorations
  • 11.1.4 Risk Assessment
  • 11.1.4.1 Individual Risk Factors
  • 11.1.4.2 Predictive Models
  • Disease Risk Index (DRI) (Armand et al. 2012, 2014)
  • EBMT Risk Score (Gratwohl et al. 1998, 2009)
  • HCT-Comorbidity Index (HCT-CI) (Sorror et al. 2005)
  • Pretransplantation Assessment of Mortality (PAM) Score (Parimon et al. 2006
  • Au et al. 2015)
  • EBMT Machine Learning Algorithm (Shouval et al. 2015)
  • 11.1.4.3 Predictive Capacity of These Models.
  • 11.1.5 Practical Applications of Risk Assessment
  • 11.2 Counseling of Candidates
  • 11.2.1 Introduction
  • 11.2.2 Understanding the Benefit and Risk of Allogeneic Transplant
  • 11.2.3 Understanding the Transplant Procedure: The Donor, the Conditioning Regimen, and the Clinical Complications
  • 11.2.4 Logistics
  • References
  • 12: Donor Selection for Adults and Pediatrics
  • 12.1 Introduction
  • 12.2 Donor HLA Compatibility (See Chap. 9)
  • 12.3 Donor Selection for Adult Patients
  • 12.3.1 Donor Type (Summarized in Fig. 12.1)
  • 12.3.1.1 Matched Related Siblings and Unrelated Donors
  • 12.3.1.2 Haploidentical Related Donors
  • 12.3.2 Role of Non-HLA Donor Characteristics
  • 12.3.3 Donor Choice According to Stem Cell Source
  • 12.3.4 Anti-HLA Antibodies
  • 12.4 Donor Selection for Pediatric Patients
  • 12.4.1 Pediatric Recipient Size
  • 12.4.2 Indications
  • 12.4.3 Donor Type
  • 12.4.4 Haploidentical Donors in Pediatrics
  • 12.4.5 Stem Cell Source
  • 12.4.6 Other Donor-Recipient-Related Factors
  • References
  • 13: Conditioning
  • 13.1 Overview
  • 13.2 Total Body Irradiation
  • 13.3 Myeloablative Non-TBI-Containing Conditioning
  • 13.4 Nonmyeloablative, Reduced Intensity and Reduced Toxicity Conditioning
  • 13.5 Conditioning Regimens for Allo-HSCT from Alternative Donors: MMUD, CB, and Haploidentical
  • 13.6 Preparative Conditioning for Autologous HSCT
  • References
  • 14: Bone Marrow Harvesting for HSCT
  • 14.1 Introduction
  • 14.2 Indications for Considering and Possibly Selecting BM as a Preferred Source of HSC
  • 14.3 Mobilized or Primed Marrow
  • 14.4 Technique of BM Collection and Impact of the Dose of Nucleated Cells Infused
  • 14.5 Complications of Bone Marrow Collections
  • 14.6 Bone Marrow Cryopreservation
  • 14.7 Quality Control for BM Harvesting and Cryopreservation
  • 14.8 Conclusions
  • References.
  • 15: Mobilization and Collection of HSC
  • 15.1 Introduction
  • 15.2 Strategies of Mobilization
  • 15.2.1 Mobilization Without Chemotherapy ("Steady State")
  • 15.2.2 Mobilization with Chemotherapy
  • 15.3 CD34+ Cell Count and Timing of Leukapheresis
  • 15.4 Target HSC Collection Count
  • 15.5 Leukapheresis
  • 15.6 Poor Mobilizer
  • 15.7 Future Directions
  • References
  • 16: Collection of HSC in Children
  • 16.1 Introduction
  • 16.2 Bone Marrow Harvest (See Chap. 14)
  • 16.3 Peripheral Blood Stem Cell Harvest
  • 16.4 Risk Analysis BM Versus PBMNC
  • 16.5 Pediatrics as Allogeneic Donors
  • References
  • 17: Processing, Cryopreserving and Controlling the Quality of HSCs
  • 17.1 Assessment of HSCs by Measuring CD34 and the Presence of Other Cell Subsets
  • 17.2 HSCs Cryopreservation
  • 17.3 HSCs Quality Assessment
  • 17.4 Collection of Reference (Retention) Samples for Quality Control
  • References
  • 18: Procurement and Management of Cord Blood
  • 18.1 Introduction
  • 18.2 Collection
  • 18.3 Processing and Banking
  • 18.3.1 UCB Cell Processing
  • 18.3.2 Testing and Quality Assessment
  • 18.4 Selecting CBU for Transplantation
  • References
  • 19: Graft Manipulation
  • 19.1 Introduction
  • 19.2 Graft Manipulation
  • 19.2.1 Physical Manipulations
  • 19.2.1.1 Volume Reduction
  • 19.2.1.2 Washing to Reduce Plasma Antibodies or Anticoagulants
  • 19.2.1.3 Depletion of Erythrocytes
  • 19.2.2 Immunomagnetic Procedures
  • 19.2.2.1 CD34 Enrichment
  • 19.2.2.2 CD133 Enrichment
  • 19.2.2.3 T-Cell Depletion
  • CD3 Depletion
  • TcRαβ Depletion
  • CD19 Depletion
  • Stem Cell Boosts
  • 19.2.3 DLI and T Cells
  • 19.2.3.1 CD45RA Depletion
  • 19.2.3.2 DLI in Relapse
  • 19.2.3.3 DLI in Mixed Chimerism
  • 19.2.3.4 Virus-Specific T Cells
  • 19.3 Regulatory Issues
  • References.
  • 20: Documentation of Engraftment and Chimerism After HSCT.

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