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 J�urgen 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 Kr�oger -- 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 -- TcR(Sab(B 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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Electronic reproduction. Ann Arbor, Michigan : ProQuest Ebook Central, 2023. Available via World Wide Web. Access may be limited to ProQuest Ebook Central affiliated libraries.