Contents

• Preface
• Abbreviations
• Editors and Contributors
• I. IntroductionTopic Leaders: Anna Sureda and Jane Apperley
  • 1. HCT: Historical Perspective
    Noa Granot and Rainer Storb.
    Published online: April 11, 2024.
    • 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
    Anna Sureda, Nicolaus Kröger, Jane Apperley, and Alois Gratwohl.
    Published online: April 11, 2024.
    • 2.1. Introduction
    • 2.2. The Past: Development of HCT and EBMT
    • 2.3. The Present
    • 2.4. The Future
    • References
  • 3. Registries: A Bridge Between Donor and Recipient
    Irina Evseeva, Lydia Foeken, and Juliana Villa.
    Published online: April 11, 2024.
    • 3.1. Introduction
    • 3.2. The Role of the Registry: Patient Perspective
    • 3.3. The Role of a Registry: Donor Perspective
    • 3.4. Working Together to Deliver Best Care to Patient and Donor
    • 3.5. Future of Registries
    • Acknowledgements
    • References
  • 4. The HCT Unit
    Walid Rasheed, Dietger W. Niederwieser, and Mahmoud Aljurf.
    Published online: April 11, 2024.
    • 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. Chimeric Antigen Receptor T-Cell (CAR-T Cell) Unit
    • 4.14. Institutional Database and Data Manager
    • 4.15. Quality Control
    • 4.16. Transplant Coordinator
    • Further Reading
  • 5. JACIE Accreditation of HCT Programs
    Riccardo Saccardi, Tuula Rintala, Eoin McGrath, and John A. Snowden.
    Published online: April 11, 2024.
    • 5.1. Introduction
    • 5.2. Background
    • 5.3. Impact of Accreditation in Clinical Practice
    • 5.4. JACIE-FACT Accreditation System
    • 5.5. Benchmarking
    • 5.6. Activity of JACIE, Including Impact of Pandemic
    • References
  • 6. Statistical Methods in HCT and Cellular Therapies
    Simona Iacobelli and Liesbeth C. de Wreede.
    Published online: April 11, 2024.
    • 6.1. Introduction
    • 6.2. Endpoints
    • 6.3. Analysis of Time-to-Event Outcomes
    • 6.4. Advanced Methods
    • Acknowledgements
    • References
• II. Biological AspectsTopic Leaders: Chiara Bonini, Raffaella Greco and Jürgen Kuball
  • 7. Biological Properties of Hematopoietic Stem Cells: Scientific Basis for Hematopoietic Cell Transplantation
    Alessandro Aiuti, Serena Scala, and Christian Chabannon.
    Published online: April 11, 2024.
    • 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
    • 7.5. Preclinical Models of HCT
    • 7.6. Gene Transfer/Gene Editing/Gene Therapy (GT) Targeting HSCs
    • 7.7. Studying the Dynamics of Hematopoietic Reconstitution Upon HCT
    • 7.8. From Experimental Hematology to Medical Practices and Hematopoietic Cellular Therapies
    • References
  • 8. Biological Properties of Cells Other Than HSCs
    Attilio Bondanza, Ulrike Koehl, Andrea Hoffmann, and Antoine Toubert.
    Published online: April 11, 2024.
    • 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
    Eric Spierings, Alejandro Madrigal, and Katharina Fleischhauer.
    Published online: April 11, 2024.
    • 9.1. Introduction
    • 9.2. The Biology of Histocompatibility
    • 9.3. HLA Matching in Allo-HCT
    • 9.4. Non-HLA Immunogenetic Factors
    • References
  • 10. Clinical and Biological Concepts for Mastering Immune Reconstitution After Hematopoietic Cell Transplantation: Toward Practical Guidelines and Greater Harmonization
    Jürgen Kuball, Raffaella Greco, Stefan Nierkens, and Jaap Jan Boelens.
    Published online: April 11, 2024.
    • 10.1. Introduction/Background
    • 10.2. Graft Composition as an Additional Predictor of Immune Reconstitution and Clinical Outcomes
    • 10.3. From Transplantation to Immune Monitoring of CAR T Cells, Harmonization Is Needed
    • 10.4. In Summary
    • References
• III. Methodology and Clinical AspectsTopic Leaders: Nicolaus Kröger, Arnon Nagler, Enric Carreras and Selim Corbacioglu
  • 11. Evaluation and Counseling of Candidates
    Pere Barba and Alessandro Rambaldi.
    Published online: April 11, 2024.
    • 11.1. Evaluation of Candidates and Risk Factors for HCT
    • 11.2. Counseling of Candidates
    • References
  • 12. Donor Selection for Adults and Pediatrics
    Francis Ayuk, Adriana Balduzzi, and Nina Worel.
    Published online: April 11, 2024.
    • 12.1. Introduction
    • 12.2. Donor Human Leukocyte Antigen (HLA) Compatibility (See Chap. 9)
    • 12.3. Donor Selection for Adult Patients
    • 12.4. Donor Selection for Pediatric Patients
    • References
  • 13. Conditioning
    Avichai Shimoni, Vera Radici, and Arnon Nagler.
    Published online: April 11, 2024.
    • 13.1. An Overview
    • 13.2. Total Body Irradiation
    • 13.3. Myeloablative Non-TBI-Containing Conditioning
    • 13.4. Non-Myeloablative, Reduced Intensity and Reduced Toxicity Conditioning
    • 13.5. Conditioning Regimens for Allo-HCT from Alternative Donors: Mismatched Unrelated Donor (MMUD), CB, and Haploidentical
    • 13.6. Preparative Conditioning for Autologous HCT
    • References
  • 14. Selection of Stem Cell Source
    Claire Horgan and David Valcárcel.
    Published online: April 11, 2024.
    • 14.1. Introduction
    • 14.2. Factors Influencing the Selection of the Source
    • 14.3. Differences in the Composition of BM vs. PB
    • 14.4. Main Results of Studies Comparing BM and PB
    • 14.5. The Role of Cord Blood
    • References
  • 15. Bone Marrow Harvesting for HCT
    Norbert Claude Gorin, Enric Carreras, Jesús Fernández-Sojo, and Juliana Villa.
    Published online: April 11, 2024.
    • 15.1. Introduction
    • 15.2. Advantages and Disadvantages of BM
    • 15.3. The Technique of BM Collection
    • 15.4. In-House Collection Kits
    • 15.5. Major and Minor Complications According to the NMDP (Pulsipher et al.
    • 15.6. Other Therapeutic Applications of BM Harvesting
    • References
  • 16. Mobilization and Collection of HSC
    Nina Worel, Yavuz M. Bilgin, and Patrick Wuchter.
    Published online: April 11, 2024.
    • 16.1. Introduction
    • 16.2. Strategies of Autologous Stem Cell Mobilization
    • 16.3. CD34+ Cell Count and Timing of Leukapheresis
    • 16.4. Target HSC Collection Count
    • 16.5. Leukapheresis
    • 16.6. Management of Poor Mobilizers
    • 16.7. Future Directions
    • References
  • 17. Mobilization and Collection of HSCs in Children
    Volker Witt, Herbert Pichler, and Norbert Ahrens.
    Published online: April 11, 2024.
    • 17.1. Introduction
    • 17.2. Patient Selection and Pre-Procedure Preparation
    • 17.3. Bone Marrow Harvesting
    • 17.4. Peripheral Blood Stem Cell Harvesting
    • References
  • 18. Procurement and Management of Cord Blood Unit for Allogeneic Transplantation
    Sergio Querol, Vanderson Rocha, and Annalisa Ruggeri.
    Published online: April 11, 2024.
    • 18.1. Introduction
    • 18.2. Collection
    • 18.3. Processing and Banking
    • 18.4. Selecting CBU for Transplantation
    • References
  • 19. Graft Manipulation
    Peter Lang, Michael Schumm, Antonio Pierini, and Rupert Handgretinger.
    Published online: April 11, 2024.
    • 19.1. Introduction
    • 19.2. Graft Manipulation
    • 19.3. Regulatory Requirements
    • References
  • 20. Processing, Cryopreserving, and Controlling the Quality of HSC
    Patrick Wuchter.
    Published online: April 11, 2024.
    • 20.1. Assessment of HSC by Measuring CD34 and the Presence of Other Cell Subsets
    • 20.2. HSC Cryopreservation
    • 20.3. HSC Quality Assessment
    • 20.4. Collection of Reference Samples for Quality Control
    • References
  • 21. Documentation of Engraftment and Chimerism After HCT
    Peter Bader, Hermann Kreyenberg, and Andrea Bacigalupo.
    Published online: April 11, 2024.
    • 21.1. Definition
    • 21.2. Introduction
    • 21.3. Methods for Chimerism Analysis
    • 21.4. Chimerism Investigation in the Clinical Setting
    • References
  • 22. Short- and Long-Term Controls After HCT
    Maria Suárez-Lledó and Montserrat Rovira.
    Published online: April 11, 2024.
    • 22.1. Introduction
    • 22.2. Monitoring Depending on the Type of HCT
    • 22.3. Organ-Specific Long-Term Monitoring
    • 22.4. Fertility (See Chap. 56)
    • 22.5. Quality of Life (See Chap. 34)
    • References
• IV. General Management of the PatientTopic Leaders: Selim Corbacioglu, Jan Styczynski and John Murray
  • 23. Vascular Access
    Simone Cesaro and Giulia Caddeo.
    Published online: April 11, 2024.
    • 23.1. Introduction
    • 23.2. Type of CVC Materials
    • 23.3. Type of CVC
    • 23.4. Venous Access
    • 23.5. CVC Complications
    • 23.6. Catheters for Leukapheresis
    • References
  • 24. Transfusion Support
    Hubert Schrezenmeier, Sixten Körper, Britta Höchsmann, and Christof Weinstock.
    Published online: April 11, 2024.
    • 24.1. General Aspects
    • 24.2. Irradiation for Prevention of Transfusion-Associated GvHD (ta-GvHD)
    • 24.3. Prevention of CMV Transmission
    • 24.4. Red Blood Cell Concentrates (RBCs)
    • 24.5. Platelet Concentrates (PCs)
    • 24.6. Immunohematological Consequences of ABO-Mismatched Transplantation
    • 24.7. Transfusion in ABO- or RhD-Incompatible HCT
    • 24.8. Granulocyte Concentrates
    • References
  • 25. Nutritional Support
    Annic Baumgartner and Philipp Schuetz.
    Published online: April 11, 2024.
    • 25.1. Introduction
    • 25.2. Screening for Malnutrition
    • 25.3. Nutritional Recommendations (See General Recommendations in Table 25.1 and Fig. 25.1; Monitoring in Table 25.2 and Nutritional Strategies in Fig. 25.1)
    • 25.4. Immunonutrition
    • 25.5. Long-Term Follow-Up
    • References
  • 26. GVHD Prophylaxis
    Jaap Jan Boelens, Francesca Bonifazi, Lars Klingen Gjærde, David Michonneau, Annalisa Ruggeri, Laura Saavedra, and Gérard Socié.
    Published online: April 11, 2024.
    • 26.1. Introduction
    • 26.2. GVHD Prophylaxis After MAC: The “Gold” Standard Combination of CNI and MTX
    • 26.3. GVHD Prophylaxis After RIC: Is CNI Plus MMF Standard?
    • 26.4. New Immunosuppressive Regimens for GVHD Prophylaxis
    • 26.5. ATG
    • 26.6. Alemtuzumab
    • 26.7. Naïve T-Cell Depletion
    • 26.8. PTCy
    • 26.9. Conclusion and Perspective
    • References
  • 27. Infection Control and Isolation Procedures
    Malgorzata Mikulska.
    Published online: April 11, 2024.
    • 27.1. Introduction
    • 27.2. Standard Precautions
    • 27.3. Transmission-Based Precautions
    • 27.4. Management of the Threat of MDR Bacteria
    • 27.5. HCT Environment
    • 27.6. Food Safety in Transplant Recipients
    • References
  • 28. General Management of the Patient: Specific Aspects of Infectious Disease Supportive Care in Children
    Elio Castagnola and Andreas H. Groll.
    Published online: April 11, 2024.
    • 28.1. Management of Bacterial Infections
    • 28.2. Management of Invasive Fungal Diseases
    • 28.3. Management of Viral Infection and Disease
    • 28.4. Nonpharmacological Prevention
    • References
  • 29. Vaccinations
    Rafael de la Cámara and Roland Meisel.
    Published online: April 11, 2024.
    • 29.1. General Concepts
    • 29.2. General Principles of Vaccination in HCT Patients
    • 29.3. Benefits and Risks of Vaccination in HCT Recipients
    • 29.4. Vaccination Recommendations
    • 29.5. Specific Vaccines
    • 29.6. Vaccinations in Children Post-allogeneic Hematopoietic Cell Transplantation
    • 29.7. Vaccinations Before Travel to Areas Endemic for Infections (See Table 29.2) (Ljungman et al.
    • 29.8. Serological Testing
    • 29.9. Vaccinations for Donors, Close Contacts/Family, and HCWs of HCT Recipients (See Table 29.3) (Ljungman et al. ; Cordonnier et al. ; Rubin et al.
    • References
  • 30. Psychological Morbidity and Care
    Alice Polomeni and Angela Scherwath.
    Published online: April 11, 2024.
    • 30.1. Introduction
    • 30.2. The Period Preceding HCT
    • 30.3. Hospitalisation for HCT
    • 30.4. Post-HCT
    • 30.5. Family Caregivers and Related Donors
    • 30.6. Adolescents and Young Adults (AYA)
    • 30.7. Paediatric Patients
    • 30.8. Psychological Interventions
    • References
  • 31. Clinically Relevant Drug Interactions in HCT
    Tiene Bauters and Adrin Dadkhah.
    Published online: April 11, 2024.
    • 31.1. Introduction
    • 31.2. Drug Interactions in HCT Practice
    • 31.3. Interactions with Herbal Drugs and Food
    • 31.4. Resources for Drug Interactions
    • 31.5. Conclusion
    • References
  • 32. The Roles of the HCT Nurse
    Michelle Kenyon and John Murray.
    Published online: April 11, 2024.
    • 32.1. Introduction: HCT Nursing
    • 32.2. The Roles of Nursing Throughout the Patient Transplant and Cell Therapy Pathway
    • 32.3. The Transplant Coordinator
    • 32.4. The Apheresis Nurse
    • 32.5. The HCT Ward Nurse
    • 32.6. Advanced Clinical Practice Nursing in HCT
    • 32.7. Post-HCT Nursing
    • 32.8. Ambulatory and Day Unit Nursing
    • 32.9. Summary
    • References
  • 33. Ethical Issues in HCT
    Khaled El-Ghariani and Jean-Hugues Dalle.
    Published online: April 11, 2024.
    • 33.1. Introduction
    • 33.2. Ethical Challenges of High-Risk Treatment
    • 33.3. Engagement with Funding Issues as a Professional Moral Obligation
    • 33.4. The Ethical Issues in Donor Management
    • References
  • 34. Quality of Life Assessment After HCT for Pediatric and Adults
    Anna Barata and Heather Jim.
    Published online: April 11, 2024.
    • 34.1. Introduction
    • 34.2. QoL Assessment
    • 34.3. Measures to Assess QoL in Adults and Pediatric Patients Undergoing HCT
    • 34.4. Challenges when Implementing QoL Assessment
    • References
• V. HCT Complications and ManagementTopic Leaders: Enric Carreras, Jan Styczynski and Per Ljungman
  • 35. Neutropenic Fever
    Malgorzata Mikulska.
    Published online: April 11, 2024.
    • 35.1. Introduction
    • 35.2. Initial Management of Fever During Neutropenia
    • 35.3. Main Changes in the Management of Neutropenic Fever
    • 35.4. Fever Persistent despite Empirical Antibiotic Therapy
    • References
  • 36. Bacterial Infections
    Dina Averbuch.
    Published online: April 11, 2024.
    • 36.1. Introduction (Tomblyn et al. ; Averbuch et al. ; Misch and Andes ; Dandoy et al. ; Martinez-Nadal et al. ; Vu et al.
    • 36.2. Epidemiology and Treatment of Bacterial Infections (Averbuch et al. ; Misch and Andes ; Dandoy et al. ; Martinez-Nadal et al. ; Vu et al. ; Trecarichi et al.
    • 36.3. Gram-Positive Infections
    • 36.4. Gram-Negative Infections (Paul et al. ; Tamma et al.
    • 36.5. Bacterial Infection Syndromes (Freifeld et al. ; Misch and Andes ; McDonald et al. ; Schmidt-Hieber et al. ; van de Beek et al.
    • 36.6. General Principles of Management of Bacterial Infections (Freifeld et al. ; Averbuch et al.
    • 36.7. Prevention of Bacterial Infections (Tomblyn et al. ; Mikulska et al. ; Egan et al. ; Buetti et al.
    • Acknowledgement
    • References
  • 37. Invasive Fungal Diseases
    Johan A. Maertens.
    Published online: April 11, 2024.
    • 37.1. Epidemiology
    • 37.2. Diagnosis of Fungal Disease
    • 37.3. Prevention and Prophylaxis
    • 37.4. Treatment of Fungal Disease
    • References
  • 38. Viral Infections
    Per Ljungman, Jose Luis Piñana, Simone Cesaro, and Rafael de la Cámara.
    Published online: April 11, 2024.
    • 38.1. Herpes Viruses
    • 38.2. Community-Acquired Respiratory Viruses (CARVs) (Excluding SARS-CoV-2)
    • 38.3. Severe Acute Respiratory Syndrome Coronavirus-2 (SARS-CoV-2) Infection and Coronavirus 2019 Disease (COVID-19)
    • 38.4. Adenovirus (ADV)
    • 38.5. Polyomaviruses
    • 38.6. Norovirus
    • 38.7. Flaviviruses
    • 38.8. West Nile Virus (WNV)
    • 38.9. Human T-Cell Lymphotropic Virus (HTLV)
    • 38.10. Viruses Covered in Other Chapters
    • References
  • 39. Other Life-Threatening Infections
    Rodrigo Martino.
    Published online: April 11, 2024.
    • 39.1. Toxoplasmosis
    • 39.2. Tuberculosis (TBC)
    • 39.3. Nontuberculous (or Atypical) Mycobacterial (NTM) Infections
    • 39.4. Listeriosis
    • 39.5. Nocardiosis
    • References
  • 40. Bleeding and Thrombotic Complications
    Rahul Shah, Bipin N. Savani, and Shruti Chaturvedi.
    Published online: April 11, 2024.
    • 40.1. Introduction
    • 40.2. Thrombotic Complications in HCT
    • 40.3. Bleeding Complications
    • 40.4. Thrombosis and Bleeding in CAR-T
    • References
  • 41. Graft Failure
    David Valcárcel, Isabel Sánchez-Ortega, and Anna Sureda.
    Published online: April 11, 2024.
    • 41.1. Introduction
    • 41.2. Definitions (Kharfan-Dabaja et al.
    • 41.3. Causes and Risk Factors
    • 41.4. Management of GF
    • References
  • 42. Early Complications of Endothelial Origin
    Enric Carreras, M. Diaz-Ricart, S. Jodele, O. Penack, and S. Vasu.
    Published online: April 11, 2024.
    • 42.1. Early Complications of Endothelial Origin
    • 42.2. Fluid Overload Syndrome (FOS)
    • 42.3. Capillary Leak Syndrome (CLS)
    • 42.4. Engraftment Syndrome (ES)
    • 42.5. Pre-engraftment Syndrome (pES)
    • 42.6. Thrombotic Microangiopathy Associated with HCT (TA-TMA)
    • 42.7. Posterior Reversible Encephalopathy Syndrome (PRES)
    • References
  • 43. Acute Graft-Versus-Host Disease
    Ernst Holler, Hildegard Greinix, and Robert Zeiser.
    Published online: April 11, 2024.
    • 43.1. Introduction
    • 43.2. Definition
    • 43.3. Risk Factors
    • 43.4. Diagnosis and Scoring
    • 43.5. Epidemiology
    • 43.6. Prevention (also See Chap. 26)
    • 43.7. Treatment
    • 43.8. Future Perspectives: Biomarkers, Risk-Adapted Treatment, and Tissue Regeneration
    • References
  • 44. Chronic Graft-Versus-Host Disease
    Daniel Wolff, Zinaida Peric, and Anita Lawitschka.
    Published online: April 11, 2024.
    • 44.1. Introduction
    • 44.2. Clinical Manifestations
    • 44.3. Diagnosis
    • 44.4. Treatment
    • Appendix A
    • Appendix B
    • Appendix C
    • References
  • 45. Posttransplant Lymphoproliferative Syndromes
    Jan Styczynski and Sebastian Giebel.
    Published online: April 11, 2024.
    • 45.1. Definitions
    • 45.2. Types of PTLD
    • 45.3. Pathogenesis
    • 45.4. Clinical Manifestations
    • 45.5. Diagnosis
    • 45.6. Risk Factors
    • 45.7. Grading
    • 45.8. Treatment
    • References
  • 46. Iron Overload
    Emanuele Angelucci and Anna Maria Raiola.
    Published online: April 11, 2024.
    • 46.1. Introduction
    • 46.2. Iron Overload Before HCT (Before the Start of Conditioning)
    • 46.3. Iron Overload During HCT (from the Start of Conditioning up to Sustained Engraftment)
    • 46.4. Iron Overload After HCT (After Sustained Engraftment Has Been Achieved)
    • References
  • 47. Secondary Neoplasia (Other Than PTLPS)
    André Tichelli and Alicia Rovó.
    Published online: April 11, 2024.
    • 47.1. Definitions
    • 47.2. Types of Secondary Neoplasia After HCT
    • 47.3. Pathophysiology
    • 47.4. Frequency and Risk Factors (See Table 47.1)
    • 47.5. Screening (Majhail et al. ; Inamoto et al. (See Also Chap. 21)
    • 47.6. Treatment
    • 47.7. Outcome
    • References
• VI. Specific Organ ComplicationsTopic Leaders: Raffaella Greco and Enric Carreras
  • 48. Ocular and Oral Complications
    Philipp Steven, Jacqueline W. Mays, and Shahrukh K. Hashmi.
    Published online: April 11, 2024.
    • 48.1. Ocular Complications
    • 48.2. Oral Complications
    • References
  • 49. Hepatic Complications
    Enric Carreras, Tapani Ruutu, Mohamad Mohty, Selim Corbacioglu, and Rafael de la Cámara.
    Published online: April 11, 2024.
    • 49.1. Introduction
    • 49.2. Veno-Occlusive Disease in Adults
      Mohamad Mohty.
    • 49.3. SOS/VOD in Children
      Selim Corbacioglu.
    • 49.4. Hepatotropic Viruses
      Rafael de la Cámara.
    • 49.5. Other Hepatic Complications
      Enric Carreras and Tapani Ruutu.
    • References
  • 50. Gastrointestinal Complications
    Grzegorz W. Basak.
    Published online: April 11, 2024.
    • 50.1. Introduction
    • 50.2. Nausea/Vomiting
    • 50.3. Diarrhea
    • 50.4. Esophagitis/Gastritis
    • 50.5. GI Bleeding
    • 50.6. Typhlitis
    • 50.7. Pancreatic Disease
    • 50.8. Chronic Esophageal GVHD
    • References
  • 51. Haemorrhagic Cystitis and Renal Dysfunction
    Simone Cesaro.
    Published online: April 11, 2024.
    • 51.1. Haemorrhagic Cystitis
    • 51.2. Renal Dysfunction
    • References
  • 52. Noninfectious Pulmonary Complications
    Anne Bergeron and Kenneth R. Cooke.
    Published online: April 11, 2024.
    • 52.1. Introduction
    • 52.2. Approach to Diagnostic Workup for Pulmonary Complications After HCT
    • 52.3. Acute Noninfectious Pulmonary Complications After HCT
    • 52.4. Late-Onset Noninfectious Pulmonary Complications (LONIPC)
    • References
  • 53. Neurological Complications
    Rémy Duléry, Martin Schmidt-Hieber, and Basil Sharrack.
    Published online: April 11, 2024.
    • 53.1. Definitions and Epidemiology
    • 53.2. Causes and Types of Neurological Complications
    • 53.3. Diagnostic Algorithm
    • 53.4. Conclusions
    • References
  • 54. Skin, Hair, and Musculoskeletal Complications
    Francis Ayuk and Bipin N. Savani.
    Published online: April 11, 2024.
    • 54.1. Complications Involving the Skin and Hair
    • 54.2. Musculoskeletal Complications
    • References
  • 55. Cardiovascular Diseases and Metabolic Syndrome
    Elisa Roldan Galvan, John A. Snowden, and Diana M. Greenfield.
    Published online: April 11, 2024.
    • 55.1. Introduction
    • 55.2. Cardiovascular Disease in HCT
    • 55.3. Risk Factors for CVD in HCT Recipients
    • 55.4. Metabolic Syndrome Definition
    • 55.5. Preventative Practices in the HCT and Late-Effect Clinic: A Practical Approach
    • 55.6. Future Directions: Implementation, Education, and Research
    • References
  • 56. Endocrine Disorders, Fertility, and Sexual Health
    Nina Salooja, Alicia Rovó, and Jean-Hugues Dalle.
    Published online: April 11, 2024.
    • 56.1. Endocrine Function
    • 56.2. Gonadal Dysfunction and Infertility
    • 56.3. Sexual Function
    • References
• VII. Prevention and Management of RelapseTopic Leaders: Nicolaus Kröger and Peter Bader
  • 57. Monitoring Measurable Residual Disease in ALL and AML
    Peter Bader, Hermann Kreyenberg, and Gert Ossenkoppele.
    Published online: April 11, 2024.
    • 57.1. Monitoring MRD in ALL
      Peter Bader and Hermann Kreyenberg.
    • 57.2. MRD in AML
      Gert Ossenkoppele.
    • References
  • 58. Prevention and Treatment of Relapse by Drugs
    Nicolaus Kröger and Nico Gagelmann.
    Published online: April 11, 2024.
    • 58.1. Introduction
    • 58.2. Tyrosine Kinase Inhibitors (TKI) Targeting BCR/ABL
    • 58.3. TKI Targeting FLT3-ITD
    • 58.4. Checkpoint Inhibitors
    • 58.5. Hypomethylating Agents
    • 58.6. Immunomodulating Drugs (IMiDs)
    • 58.7. Proteasome Inhibitors
    • 58.8. Monoclonal Antibodies
    • 58.9. Histone Deacetylase Inhibition (HDACI)
    • 58.10. BCL-2 Inhibitors
    • 58.11. IDH1/IDH2 Inhibitors
    • References
  • 59. Delayed Transfer of Immune Cells or the Art of Donor Lymphocyte Infusion (DLI) 2.0
    J. H. Frederik Falkenburg, Christoph Schmid, Hans Joachim Kolb, and Jürgen Kuball.
    Published online: April 11, 2024.
    • 59.1. Biology of Donor Lymphocyte Infusion (DLI)
    • 59.2. Recommendations for Prophylactic and Preemptive DLI as well as DLI After Relapse
    • 59.3. Conclusion
    • References
  • 60. Cellular Therapy with Engineered T Cells, Efficacy, and Side Effects: Gene Editing/Gene Therapy
    Chiara Bonini, Marina Cavazzana, Fabio Ciceri, Boris Fehse, and Michael Hudecek.
    Published online: April 11, 2024.
    • 60.1. Introduction
    • 60.2. Suicide Gene Therapy
    • 60.3. CAR-T Cells
    • 60.4. TCR Gene Transfer, TCR Gene Editing, and Future Perspectives
    • References
  • 61. Mechanisms of Immune Resistance
    Luca Vago and Francesco Dazzi.
    Published online: April 11, 2024.
    • 61.1. Introduction
    • 61.2. Mechanisms of Immune Evasion
    • References
  • 62. Regulatory Aspects of ATMP Versus Minimally Manipulated Immune Cells
    Christian Chabannon and Harry Dolstra.
    Published online: April 11, 2024.
    • References
• VIII. Specific Modalities of HCT and ManagementTopic Leaders: Anna Sureda, Nicolaus Kröger and Andrew Gennery
  • 63. At-Home HCT
    Francesc Fernández-Avilés.
    Published online: April 11, 2024.
    • 63.1. Introduction
    • 63.2. Ambulatory AutoHCT Models (Martino et al.
    • 63.3. Suggested Inclusion Criteria for Ambulatory AutoHCT
    • 63.4. General Recommendations for At-Home AutoHCT
    • 63.5. Most Frequent Reasons for Readmission (Ordered by Frequency)
    • 63.6. Treatment of Fever in At-Home Setting
    • 63.7. Incidence of Readmission in Outpatient and At-Home AutoHCT
    • 63.8. Quality of Life
    • 63.9. Cost Data
    • 63.10. At-Home AlloHCT
    • References
  • 64. Role of Umbilical Cord Blood Transplantation
    Jaime Sanz and Vanderson Rocha.
    Published online: April 11, 2024.
    • 64.1. Introduction
    • 64.2. Potential Advantages and Disadvantages of UCBT
    • 64.3. Indications
    • 64.4. Approaches to Improve Outcomes After UCBT
    • 64.5. Conditioning Regimens
    • 64.6. GVHD Prophylaxis
    • 64.7. Supportive Care
    • 64.8. Results
    • 64.9. Adults
    • 64.10. Children
    • 64.11. Conclusions
    • References
  • 65. Haploidentical HCT
    Andrea Bacigalupo, Arjan Lankester, Fabio Ciceri, and Alice Bertaina.
    Published online: April 11, 2024.
    • 65.1. Ex Vivo TCD Platforms
    • 65.2. Unmanipulated Haploidentical HCT
    • 65.3. Other Relevant Aspects of Haplo-HCT
    • References
  • 66. Photopheresis in Adults and Pediatrics
    Hildegard Greinix.
    Published online: April 11, 2024.
    • 66.1. Introduction
    • 66.2. Technical Aspects
    • 66.3. Results of ECP in Acute GVHD
    • 66.4. Results of ECP in Chronic GVHD
    • 66.5. Conclusions
    • References
  • 67. Overweight and Obese Patients
    Claudia Langebrake.
    Published online: April 11, 2024.
    • 67.1. Introduction
    • 67.2. Influence of Excess Weight and Obesity on the Pharmacokinetics of Drugs
    • 67.3. Recommendations for Drug Dosing
    • References
  • 68. HCT in Elderly Patients
    Rafael F. Duarte and Isabel Sánchez-Ortega.
    Published online: April 11, 2024.
    • 68.1. Introduction
    • 68.2. HCT Activity in Elderly Patients
    • 68.3. HCT Outcomes in Elderly Patients
    • 68.4. Assessment of Elderly Candidates for HCT
    • References
  • 69. Feasibility and Experiences of HCT in Resource-Constrained Settings
    Alok Srivastava, Fernando Barroso Duarte, and Lawrence Faulkner.
    Published online: April 11, 2024.
    • 69.1. Introduction
    • 69.2. Establishing HCT Centers: Personnel/Infrastructure/Types of HCTs
    • 69.3. Patient Selection and Indications for HCT
    • 69.4. Donor Selection: Alternative Donors—Graft Manipulations
    • 69.5. Harvest of Graft: CD34+ HSC Dose Calculation and Cryopreservation Issues
    • 69.6. Conditioning Regimen
    • 69.7. Supportive Care
    • 69.8. Graft Versus Host Disease (GVHD) Management
    • 69.9. Long-term Follow-Up
    • 69.10. Socioeconomic Issues
    • 69.11. Conclusion
    • References
• IX. Indications and ResultsTopic Leaders: Selim Corbacioglu, Nicolaus Kröger, Anna Sureda, Raffaella Greco and Arnon Nagler
  • 70. AML in Adults
    Jurjen Versluis, Jan J. Cornelissen, Charles Craddock, Miguel Á. Sanz, and Arnon Nagler.
    Published online: April 11, 2024.
    • 70.1. AML in CR1
      Jurjen Versluis and Jan J. Cornelissen.
    • 70.2. Allo-HCT in Advanced AML
      Charles Craddock.
    • 70.3. Practical Issues for Allo-HCT in AML
      Arnon Nagler.
    • 70.4. Acute Promyelocytic Leukemia
      Miguel Á. Sanz.
    • References
  • 71. Acute Myeloid Leukaemia in Children
    Brenda E. S. Gibson, Martin G. Sauer, Subramaniam Ramanathan, and Persis J. Amrolia.
    Published online: April 11, 2024.
    • 71.1. Introduction
    • 71.2. Prognostic Factors and Indications
    • 71.3. Conditioning Regimens
    • 71.4. Donor Selection Hierarchy and Stem Cell Source
    • 71.5. GVHD Prophylaxis
    • 71.6. Prevention of Relapse Post-Transplant
    • 71.7. Role of the Second HCT
    • Abbreviations
    • References
  • 72. Acute Lymphoblastic Leukemia in Adults
    Matthias Stelljes, David I. Marks, and Sebastian Giebel.
    Published online: April 11, 2024.
    • 72.1. Definition and Epidemiology
    • 72.2. Diagnosis
    • 72.3. Classification
    • 72.4. Risk Factors
    • 72.5. Prognostic Factors Used to Indicate Allo-HCT in CR1
    • 72.6. First-Line Treatment
    • 72.7. Second-Line Treatment
    • 72.8. Autologous HCT
    • 72.9. Allogeneic HCT
    • 72.10. Therapeutic Algorithm Recommended by the Authors
    • References
  • 73. Acute Lymphoblastic Leukaemia in Children and Adolescents
    Christina Peters, Franco Locatelli, and Peter Bader.
    Published online: April 11, 2024.
    • 73.1. Introduction
    • 73.2. Prognostic Factors and Indications for HCT
    • 73.3. Donor Selection and Stem Cell Source
    • 73.4. Conditioning Regimen
    • 73.5. GVHD Prophylaxis
    • 73.6. Children below 4 Years of Age
    • References
  • 74. Myelodysplastic Neoplasms/Syndromes (MDS)
    Marie Robin and Carmelo Gurnari.
    Published online: April 11, 2024.
    • 74.1. Introduction
    • 74.2. Indication of HCT in MDS and Timing to Transplant
    • 74.3. Treatment Prior to HCT
    • 74.4. Preparative Regimen
    • 74.5. Post-HCT Outcomes
    • 74.6. Alternative Donors and Donor Choice
    • 74.7. Post-HCT Treatment
    • References
  • 75. Allogeneic Hematopoietic Cell Transplantation in Pediatric MDS Including Refractory Cytopenia of Childhood and in Juvenile Myelomonocytic Leukemia
    Charlotte M. Niemeyer and Brigitte Strahm.
    Published online: April 11, 2024.
    • 75.1. Introduction
    • 75.2. Pediatric MDS Including Refractory Cytopenia
    • 75.3. Juvenile Myelomonocytic Leukemia
    • References
  • 76. Myelodysplastic/Myeloproliferative Neoplasms
    Francesco Onida and Yves Chalandon.
    Published online: April 11, 2024.
    • 76.1. Definition, Epidemiology, Diagnosis, and Classification
    • 76.2. Risk Factors and Prognostic Index
    • 76.3. Pretransplantation Treatment
    • 76.4. Autologous HCT
    • 76.5. Allogeneic HCT
    • 76.6. Source of HSC
    • 76.7. Conditioning and GvHD Prophylaxis
    • 76.8. Maintenance/Post Transplant Strategies
    • References
  • 77. Myeloproliferative Neoplasms
    Nicolaus Kröger, Donal P. McLornan, and Yves Chalandon.
    Published online: April 11, 2024.
    • 77.1. Primary and Post ET/PV Myelofibrosis
    • 77.2. Chronic Myeloid Leukemia
      Yves Chalandon.
    • References
  • 78. Acquired Bone Marrow Failure: Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
    Régis Peffault de Latour, Antonio Risitano, Austin Kulasekararaj, and Carlo Dufour.
    Published online: April 11, 2024.
    • 78.1. Definition and Epidemiology
    • 78.2. Diagnosis and Indication for Treatment for SAA
    • 78.3. Treatment of SAA
    • 78.4. Treatment of PNH
    • References
  • 79. Fanconi Anemia and Other Hereditary Bone Marrow Failure Syndromes
    Cristina Diaz-de-Heredia, Marc Bierings, Jean-Hugues Dalle, Francesca Fioredda, and Brigitte Strahm.
    Published online: April 11, 2024.
    • 79.1. Introduction
    • 79.2. Fanconi Anemia
    • 79.3. Telomere Disorders
    • 79.4. Severe Congenital Neutropenia and Shwachman-Diamond Syndrome
    • 79.5. Diamond-Blackfan Anemia
    • 79.6. Congenital Amegakaryocytic Thrombocytopenia
    • Further Reading
  • 80. Hemoglobinopathies (Sickle Cell Disease and Thalassemia)
    Barbara Cappelli, Eliane Gluckman, Selim Corbacioglu, Josu de la Fuente, and Miguel R. Abboud.
    Published online: April 11, 2024.
    • 80.1. HCT for Sickle Cell Disease
      Barbara Cappelli, Eliane Gluckman, and Selim Corbacioglu.
    • 80.2. Thalassemia
      Miguel R. Abboud and Josu de la Fuente.
    • References
  • 81. Multiple Myeloma
    Mohamad Mohty, Benedetto Bruno, Nico Gagelmann, and Maria-Victoria Mateos.
    Published online: April 11, 2024.
    • 81.1. Which Patients Are Candidates for Autologous Hematopoietic Cell Transplantation?
    • 81.2. What Is the Optimal Induction Treatment Prior to AHCT?
    • 81.3. What Is the Optimal Conditioning Regimen Prior to AHCT?
    • 81.4. What Is the Impact of Consolidation Therapy After AHCT?
    • 81.5. What Is the Impact of Maintenance Therapy After AHCT?
    • 81.6. What Is the Value of Single Versus Tandem AHCT?
    • 81.7. What Is the Role of AHCT as Salvage Therapy?
    • 81.8. What Is the Role of Allogeneic Hematopoietic Cell Transplantation in Multiple Myeloma?
    • 81.9. The Emerging Role CAR-T Cells in Multiple Myeloma
    • 81.10. Conclusion and Future Perspectives
    • Further Reading
  • 82. Systemic Light Chain Amyloidosis
    Monique Minnema and Stefan Schönland.
    Published online: April 11, 2024.
    • 82.1. Definition and Epidemiology
    • 82.2. Diagnosis
    • 82.3. Classification
    • 82.4. Risk Factors and Prognostic Scores
    • 82.5. First-Line Treatment
    • 82.6. Second-Line Treatment
    • 82.7. Autologous HCT
    • 82.8. Cellular Therapies
    • References
  • 83. Poems Syndrome and Disease Produced by Other Monoclonal IGs
    Gordon Cook and Montserrat Rovira.
    Published online: April 11, 2024.
    • 83.1. POEMS Syndrome
    • 83.2. Monoclonal Ig Deposition Disease
    • Further Reading
  • 84. Indolent Lymphoma
    Yasmina Serroukh and Silvia Montoto.
    Published online: April 11, 2024.
    • 84.1. Definition and Epidemiology
    • 84.2. Diagnosis
    • 84.3. Prognosis
    • 84.4. First Line Treatment
    • 84.5. Treatment at First Relapse and Beyond
    • 84.6. Autologous HCT
    • 84.7. Allogeneic HCT
    • 84.8. The Place of Auto- and Allo-HCT in the Therapeutic Algorithm of iNHL
    • 84.9. Latest (Selected) EBMT Results
    • 84.10. Conclusions
    • References
  • 85. Chronic Lymphocytic Leukemia
    Johannes Schetelig and Peter Dreger.
    Published online: April 11, 2024.
    • 85.1. Introduction
    • 85.2. Principles of Treatment for CLL
    • 85.3. Allogeneic HCT for CLL
    • 85.4. Remission Induction Prior to Start of the Conditioning Regimen
    • 85.5. Conditioning Regimens
    • 85.6. Outcome After Allogeneic Transplantation for CLL
    • 85.7. Post-Transplant Minimal Residual Disease Monitoring and Immune Intervention in CLL
    • 85.8. Autologous HCT and CAR-T Cell Therapy
    • 85.9. Summary and Perspectives
    • References
  • 86. Large B-Cell Lymphoma
    Leyre Bento, Bertram Glass, and Norbert Schmitz.
    Published online: April 11, 2024.
    • 86.1. Definition and Epidemiology
    • 86.2. Diagnosis
    • 86.3. Classification
    • 86.4. Risk Factors
    • 86.5. First-Line Treatment
    • 86.6. Second-Line Treatment
    • 86.7. Autologous HCT
    • 86.8. CAR T Cells
    • 86.9. Allogeneic HCT
    • References
  • 87. Mantle Cell Lymphoma
    Ben-Niklas Baermann and Sascha Dietrich.
    Published online: April 11, 2024.
    • 87.1. Introduction
    • 87.2. Autologous HCT
    • 87.3. Chimeric Antigen Receptor T-Cell Therapy
    • 87.4. Allogeneic HCT
    • References
  • 88. Other B- and T-Aggressive Lymphomas and Lymphomas Associated with HIV
    Kai Hübel, Silvia Montoto, Mustafa Güven, and Rafael F. Duarte.
    Published online: April 11, 2024.
    • 88.1. Burkitt Lymphoma
    • 88.2. Lymphoblastic Lymphoma
    • 88.3. Peripheral T Cell Lymphomas
    • 88.4. Cutaneous T Cell Lymphomas
    • 88.5. HIV-Associated Lymphomas
    • References
  • 89. Classical Hodgkin’s Lymphoma
    Carmen Martínez, Ali Bazarbachi, and Anna Sureda.
    Published online: April 11, 2024.
    • 89.1. Definition and Epidemiology
    • 89.2. Diagnosis
    • 89.3. Classification
    • 89.4. Risk Factors
    • 89.5. First-Line Treatment
    • 89.6. Second-Line Treatment Before Auto-HCT
    • 89.7. Autologous HCT
    • 89.8. Tandem Auto-HCT
    • 89.9. Disease Relapse After Auto-HCT
    • 89.10. Allogeneic HCT
    • 89.11. Therapeutic Algorithm Recommended by the Authors (Modified from Yethava et al.)
    • 89.12. Long-Term Outcomes of Auto-HCT and Allo-HCT in Patients with Relapsed/Refractory cHL (EBMT Database, with Permission)
    • References
  • 90. Inborn Errors of Immunity
    Michael H. Albert, Arjan Lankester, Andrew Gennery, and Bénédicte Neven.
    Published online: April 11, 2024.
    • 90.1. Introduction
    • 90.2. Diseases
    • 90.3. SCID
    • 90.4. Non-SCID IEI
    • 90.5. HCT for Radiosensitive IEI
    • 90.6. Alternative Therapies
    • References
  • 91. Inborn Errors of Metabolism and Osteopetrosis
    Robert Wynn and Ansgar Schulz.
    Published online: April 11, 2024.
    • 91.1. Inborn Errors of Metabolism
    • 91.2. Osteopetrosis
    • Further Reading
  • 92. Autoimmune Disease
    Tobias Alexander, Basil Sharrack, Montserrat Rovira, Riccardo Saccardi, Dominique Farge, John A. Snowden, and Raffaella Greco.
    Published online: April 11, 2024.
    • 92.1. Introduction
    • 92.2. Mechanisms of Action
    • 92.3. HCT for Multiple Sclerosis (MS)
    • 92.4. HCT for Systemic Sclerosis (SSc)
    • 92.5. HCT for Systemic Lupus Erythematosus (SLE)
    • 92.6. HCT for Crohn’s Disease (CD)
    • 92.7. Allogeneic HCT for Autoimmune Diseases
    • 92.8. Other Indications
    • 92.9. Conclusions and Future Directions
    • Acknowledgments
    • References
  • 93. CART Cells and Other Cell Therapies (ie MSC, Tregs) in Autoimmune Diseases
    Raffaella Greco and Dominique Farge.
    Published online: April 11, 2024.
    • 93.1. Introduction
    • 93.2. Mesenchymal Stem Cells (MSC)
    • 93.3. Regulatory T Cells (Tregs)
    • 93.4. Chimeric Antigen Receptor T Cells (CART)
    • 93.5. Conclusions and Future Directions
    • Acknowledgments
    • References
  • 94. Solid Tumours
    Ruth Ladenstein, Paolo Pedrazzoli, and Giovanni Rosti.
    Published online: April 11, 2024.
    • 94.1. Introduction
    • 94.2. Solid Tumours in Children and Adolescents
    • 94.3. Solid Tumours in Adults
    • References