Contents

• Preface
• Acknowledgment
• Abbreviations
• Contributors
• About the Editors
• I. Introduction Topic leaders: Mohamad Mohty and Jane Apperley
  • 1. HSCT: Historical Perspective
    Rainer Storb.
    • 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
    Alois Gratwohl, Mohamad Mohty, and Jane Apperley.
    • 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
    Irina Evseeva, Lydia Foeken, and Alejandro Madrigal.
    • 3.1. Introduction
    • 3.2. Current Landscape
    • 3.3. Connections and Worldwide Collaboration
    • 3.4. Challenges and Opportunities
    • 3.5. Future Developments
    • Acknowledgements
    • References
  • 4. The HSCT Unit
    Walid Rasheed, Dietger W. Niederwieser, and Mahmoud Aljurf.
    • 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
    Riccardo Saccardi, Eoin McGrath, and John A. Snowden.
    • 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
    Simona Iacobelli and Liesbeth C. de Wreede.
    • 6.1. Introduction
    • 6.2. Endpoints
    • 6.3. Analysis of Time-to-Event Outcomes
    • 6.4. Advanced Methods
    • Acknowledgements
    • References
• II. Biological Aspects Topic leaders: Chiara Bonini and Jürgen Kuball
  • 7. Biological Properties of HSC: Scientific Basis for HSCT
    Alessandro Aiuti, Serena Scala, and Christian Chabannon.
    • 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
    Attilio Bondanza, Ulrike Koehl, Andrea Hoffmann, and Antoine Toubert.
    • 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 and Katharina Fleischhauer.
    • 9.1. Introduction
    • 9.2. The Biology of Histocompatibility
    • 9.3. HLA Matching in Allogeneic HSCT
    • 9.4. Non-HLA Immunogenetic Factors
    • References
  • 10. Clinical and Biological Concepts for Mastering Immune Reconstitution After HSCT: Toward Practical Guidelines and Greater Harmonization
    Jürgen Kuball and Jaap Jan Boelens.
    • 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
    • 10.5. Summary
    • References
• III. Methodology and Clinical Aspects Topic leaders: Arnon Nagler and Nicolaus Kröger
  • 11. Evaluation and Counseling of Candidates
    Enric Carreras and Alessandro Rambaldi.
    • 11.1. Evaluation of Candidates and Risk Factors for HSCT
      Enric Carreras.
    • 11.2. Counseling of Candidates
      Alessandro Rambaldi.
    • References
  • 12. Donor Selection for Adults and Pediatrics
    Francis Ayuk and Adriana Balduzzi.
    • 12.1. Introduction
    • 12.2. Donor HLA Compatibility (See Chap. 9)
    • 12.3. Donor Selection for Adult Patients
    • 12.4. Donor Selection for Pediatric Patients
    • References
  • 13. Conditioning
    Arnon Nagler and Avichai Shimoni.
    • 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
    Norbert Claude Gorin.
    • 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
    Kai Hübel.
    • 15.1. Introduction
    • 15.2. Strategies of Mobilization
    • 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
    Volker Witt and Christina Peters.
    • 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
    Patrick Wuchter.
    • 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
    Sergio Querol and Vanderson Rocha.
    • 18.1. Introduction
    • 18.2. Collection
    • 18.3. Processing and Banking
    • 18.4. Selecting CBU for Transplantation
    • References
  • 19. Graft Manipulation
    Michael Schumm, Peter Lang, and Rupert Handgretinger.
    • 19.1. Introduction
    • 19.2. Graft Manipulation
    • 19.3. Regulatory Issues
    • References
  • 20. Documentation of Engraftment and Chimerism After HSCT
    Peter Bader.
    • 20.1. Introduction
    • 20.2. Methods for Chimerism Analysis
    • 20.3. Chimerism Investigation in the Clinical Setting
    • References
  • 21. Short- and Long-Term Controls After HSCT
    Montserrat Rovira and Maria Suárez-Lledó.
    • 21.1. Introduction
    • 21.2. Monitoring Depending on the Type of HSCT
    • 21.3. Organ-Specific Long-Term Monitoring
    • 21.4. Fertility (See Chap. 56)
    • 21.5. Quality of Life (See Chap. 34)
    • Recommended References
• IV. General Management of the Patient Topic leaders: Carlo Dufour, Silvia Montoto and John Murray
  • 22. Vascular Access
    Simone Cesaro and Federica Minniti.
    • 22.1. Introduction
    • 22.2. Type of CVC Materials
    • 22.3. Type of CVC
    • 22.4. Venous Access
    • 22.5. CVC Complications
    • 22.6. Catheters for Leukapheresis
    • References
  • 23. Transfusion Support
    Hubert Schrezenmeier, Sixten Körper, Britta Höchsmann, and Christof Weinstock.
    • 23.1. General Aspects
    • 23.2. Irradiation for Prevention of Transfusion-Associated GvHD (ta-GvHD)
    • 23.3. Prevention of CMV Transmission
    • 23.4. Red Blood Cell Concentrates (RBCs)
    • 23.5. Platelet Concentrates (PCs)
    • 23.6. Immunohematological Consequences of ABO-Mismatched Transplantation
    • 23.7. Transfusion in ABO- or RhD-Incompatible HSCT
    • 23.8. Granulocyte Concentrates
    • References
  • 24. Nutritional Support
    Annic Baumgartner and Philipp Schuetz.
    • 24.1. Introduction
    • 24.2. Screening for Malnutrition
    • 24.3. Nutritional Recommendations (See General Recommendations in Table 24.1 and Fig. 24.1; Monitoring in Table 24.2 and Nutritional Strategies in Fig. 24.1)
    • 24.4. Immunonutrition
    • 24.5. Long-Term Follow-Up
    • References
  • 25. GVHD Prophylaxis (Immunosuppression)
    David Michonneau and Gérard Socié.
    • 25.1. Introduction
    • 25.2. GVHD Prophylaxis After MAC; The “Gold” Standard; CNI in Combination with MTX
    • 25.3. GVHD Prophylaxis After RIC; Is CNI Plus MMF Standard?
    • 25.4. Can PT-CY Be Considered as Standard GVHD Prophylaxis in Transplantation from Haploidentical Donors and Beyond?
    • 25.5. ATG or Alemtuzumab for GVHD Prophylaxis in HSCT
    • 25.6. New Immunosuppressive Regimens for GVHD Prophylaxis
    • 25.7. Conclusion and Perspective
    • References
  • 26. Management ATG (SIRS)
    Francesca Bonifazi.
    • 26.1. Introduction
    • 26.2. ATLG/ATG Infusion Protocol (See Table 26.1)
    • 26.3. ATLG/ATG Dose
    • 26.4. Infusion Reactions
    • 26.5. SIRS
    • References
  • 27. Infection Control and Isolation Procedures
    Malgorzata Mikulska.
    • 27.1. Introduction
    • 27.2. Standard Precautions
    • 27.3. Transmission-Based Precautions
    • 27.4. Management of the Threat of MDR Bacteria
    • 27.5. HSCT Environment
    • 27.6. Food Safety in Transplant Recipients
    • References
  • 28. General Management of the Patient: Specific Aspects of Children
    Francesca Riccardi and Elio Castagnola.
    • 28.1. Introduction
    • 28.2. Psychological Aspects (Table 28.1)
    • 28.3. Infectious Diseases
    • References
  • 29. Vaccinations
    Rafael de la Cámara.
    • 29.1. General Concepts
    • 29.2. General Principles of Vaccination in HSCT Patients
    • 29.3. Benefits and Risks of Vaccination in HSCT Patients
    • 29.4. Vaccination Recommendations
    • 29.5. Specific Vaccines
    • 29.6. Vaccinations Before Travel to Areas Endemic for Infections (See Table 29.3) (Ljungman et al.
    • 29.7. Serological Testing
    • 29.8. Vaccinations for Donors, Close Contacts/Family, and HCWs of HSCT Recipients (See Table 29.4) (Ljungman et al. ; Cordonnier et al. ; Rubin et al.
    • References
  • 30. Psychological Morbidity and Support
    Alice Polomeni, Enrique Moreno, and Frank Schulz-Kindermann.
    • 30.1. Introduction
    • 30.2. The Period Preceding HSCT
    • 30.3. Hospitalisation for HSCT
    • 30.4. Post-HSCT
    • 30.5. Close Relatives
    • 30.6. Related Donors
    • 30.7. Adolescents and Young Adults (AYA)
    • 30.8. Paediatric Patients
    • References
  • 31. Clinically Relevant Drug Interactions in HSCT
    Tiene Bauters.
    • 31.1. Introduction
    • 31.2. Drug Interactions in HSCT Practice
    • 31.3. Interactions with Herbal Drugs and Food
    • 31.4. Resources for Drug Interactions
    • 31.5. Conclusion
    • References
  • 32. Role of Nursing in HSCT
    Aleksandra Babic and John Murray.
    • 32.1. Introduction: HSCT Nursing
    • 32.2. Role of Nursing Throughout HSCT Patient Pathway
    • 32.3. Specific Aspects with a Prominent Role for Nurses
    • References
  • 33. Ethical Issues in HSCT
    Khaled El-Ghariani and Jean-Hugues Dalle.
    • 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 HSCT for Pediatric and Adults
    Anna Barata and Heather Jim.
    • 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. HSCT Complications and Management Topic leaders: Jan Styczynski, Enric Carreras and Per Ljungman
  • 35. Neutropenic Fever
    Malgorzata Mikulska.
    • 35.1. Introduction
    • 35.2. Initial Management of Fever During Neutropenia
    • 35.3. Main Changes in the Last Decade and Empirical Therapy Modalities
    • 35.4. Fever Persistent Despite Empirical Antibiotic Therapy
    • References
  • 36. Bacterial Infections
    Diana Averbuch.
    • 36.1. Introduction
    • 36.2. Epidemiology of Bacteremia
    • 36.3. Gram-Positive Infections
    • 36.4. Gram-Negative Infections (Averbuch et al. ; Mikulska et al. ; Trecarichi et al. ; Girmenia et al.
    • 36.5. Bacterial Infection Syndromes
    • 36.6. Improving Management of Bacterial Infection (Averbuch et al. ; Satlin and Walsh
    • 36.7. Prevention of Bacterial Infection (Tomblyn et al. ; Tacconelli et al. ; Cordonnier et al. ; ECIL-meeting
    • References
  • 37. Invasive Fungal Infections
    Johan A. Maertens.
    • 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, Jan Styczynski, and Hermann Einsele.
    • 38.1. Herpes Viruses
    • 38.2. Adenovirus (ADV)
    • 38.3. Respiratory Viruses
    • 38.4. Polyomaviruses
    • 38.5. Hepatotropic Viruses
    • 38.6. Norovirus
    • 38.7. Zika Virus (ZIKV)
    • Recommended References
  • 39. Other Life-Threatening Infections
    Rodrigo Martino.
    • 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
    Shruti Chaturvedi, Binsah George, and Bipin N. Savani.
    • 40.1. Introduction
    • 40.2. Thrombotic Complications
    • 40.3. Bleeding Complications
    • References
  • 41. Graft Failure
    David Valcárcel and Anna Sureda.
    • 41.1. Introduction
    • 41.2. Definitions
    • 41.3. Causes and Risk Factors
    • 41.4. Management of GF
    • References
  • 42. Early Complications of Endothelial Origin
    Enric Carreras and Maribel Diaz-Ricart.
    • 42.1. Introduction
    • 42.2. Sinusoidal Obstruction Syndrome (See Chap. 49)
    • 42.3. Engraftment Syndrome
    • 42.4. Pre-engraftment Syndrome
    • 42.5. Capillary Leak Syndrome
    • 42.6. Thrombotic Microangiopathy Associated with HSCT (TA-TMA)
    • References
  • 43. Acute Graft-Versus-Host Disease
    Ernst Holler, Hildegard Greinix, and Robert Zeiser.
    • 43.1. Introduction
    • 43.2. Definition
    • 43.3. Risk Factors
    • 43.4. Diagnosis and Scoring
    • 43.5. Epidemiology
    • 43.6. Prevention (Also See Chap. 25)
    • 43.7. Treatment
    • 43.8. Future Perspectives: Biomarkers and Risk-Adapted Treatment
    • References
  • 44. Chronic Graft-Versus-Host Disease
    Daniel Wolff and Anita Lawitschka.
    • 44.1. Introduction
    • 44.2. Clinical Manifestations
    • 44.3. Diagnosis
    • 44.4. Treatment
    • Appendix 1
    • Appendix 2
    • Appendix 3
    • References
  • 45. Post transplant Lymphoproliferative Syndromes
    Jan Styczynski and Sebastian Giebel.
    • 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
    • Recommended References
  • 46. Iron Overload
    Emanuele Angelucci.
    • 46.1. Introduction
    • 46.2. Iron Overload Before HSCT (Before the Start of Conditioning)
    • 46.3. Iron Overload During HSCT (From the Start of Conditioning Up to Sustained Engraftment)
    • 46.4. Iron Overload After HSCT (After Sustained Engraftment Has Been Achieved)
    • References
  • 47. Secondary Neoplasia (Other than PTLPS)
    André Tichelli.
    • 47.1. Definitions
    • 47.2. Types of Secondary Neoplasia After HSCT
    • 47.3. Pathophysiology
    • 47.4. Frequency and Risk Factors (See Table 47.1)
    • 47.5. Screening (Majhail et al. (See Also Chap. 21)
    • 47.6. Treatment
    • 47.7. Outcome
    • References
• VI. Specific Organ Complications Topic leaders: Grzegorz Basak and John Snowden
  • 48. Ocular and Oral Complications
    Shahrukh K. Hashmi.
    • 48.1. Ocular Complications
    • 48.2. Oral Complications
    • References
  • 49. Hepatic Complications
    Tapani Ruutu and Enric Carreras.
    • 49.1. Introduction
    • 49.2. Sinusoidal Obstruction Syndrome
    • 49.3. Hepatitis After HSCT
    • 49.4. Cirrhosis and Heparocellular Carcinoma
    • 49.5. Other Less Frequent Hepatic Complications
    • References
  • 50. Gastrointestinal Complications
    Grzegorz W. Basak.
    • 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.
    • 51.1. Haemorrhagic Cystitis
    • 51.2. Renal Dysfunction
    • References
  • 52. Noninfectious Pulmonary Complications
    Enric Carreras and Kenneth R. Cooke.
    • 52.1. Introduction
    • 52.2. Diagnostic Methodology of Pulmonary Complications
    • 52.3. Pulmonary Edema Due to Fluid Overload
    • 52.4. Idiopathic Pneumonia Syndrome
    • 52.5. Diffuse Alveolar Hemorrhage (DAH)
    • 52.6. Late-Onset Noninfectious Pulmonary Complications (LONIPC)
    • References
  • 53. Neurological Complications
    Rémy Duléry.
    • 53.1. Definitions and Epidemiology
    • 53.2. Causative Agents 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.
    • 54.1. Complications Involving the Skin and Hair
    • 54.2. Musculoskeletal Complications
    • References
  • 55. Cardiovascular Diseases and Metabolic Syndrome
    Diana M. Greenfield and John A. Snowden.
    • 55.1. Introduction
    • 55.2. Risk Factors
    • 55.3. Metabolic Syndrome Definition
    • 55.4. Preventative Practices in the HSCT and Late Effects Clinic: A Practical Approach
    • 55.5. Future Directions: Implementation, Education and Research
    • References
  • 56. Endocrine Disorders, Fertility and Sexual Health
    Nina Salooja, Zeev Shoham, and Jean-Hugues Dalle.
    • 56.1. Endocrine Function
    • 56.2. Gonadal Dysfunction and Infertility
    • 56.3. Sexual Function
    • References
• VII. Prevention and Management of Relapse Topic leaders: Peter Bader and Nicolaus Kröger
  • 57. Monitoring Minimal Residual Disease in ALL and AML
    Peter Bader, Hermann Kreyenberg, and Gert Ossenkoppele.
    • 57.1. Monitoring MRD in ALL
      Peter Bader and Hermann Kreyenberg.
    • 57.2. Monitoring MRD in AML
      Gert Ossenkoppele.
    • References
  • 58. Prevention and Treatment of Relapse by Drugs
    Nicolaus Kröger.
    • 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)
    • References
  • 59. Delayed Transfer of Immune Cells or the Art of Donor Lymphocyte Infusion
    J. H. Frederik Falkenburg, Christoph Schmid, Hans Joachim Kolb, Franco Locatelli, and Jürgen Kuball.
    • 59.1. Biology of Donor Lymphocyte Infusion (DLI)
    • 59.2. Guidelines for Prophylactic and Preemptive DLI as Well as DLI After Relapse
    • References
  • 60. Cellular Therapy with Engineered T Cells, Efficacy and Side Effects
    Attilio Bondanza, Chiara Bonini, Boris Fehse, and Michael Hudecek.
    • 60.1. Introduction
    • 60.2. Suicide Gene Therapy
    • 60.3. CAR-T Cells
    • 60.4. TCR Gene Transfer and Future Perspectives
    • References
  • 61. Mechanisms of Immune Resistance
    Luca Vago and Francesco Dazzi.
    • 61.1. Introduction
    • 61.2. Mechanisms of Immune Evasion
    • References
  • 62. Regulatory Aspects of ATMP Versus Minimally Manipulated Immune Cells
    Eoin McGrath and Christian Chabannon.
    • References
• VIII. Specific Modalities of HSCT and Management Topic leaders: Andrew Gennery, Anna Sureda and Arjan Lankester
  • 63. At-Home HSCT
    Francesc Fernandez-Avilés and Gonzalo Gutiérrez-García.
    • 63.1. Autologous HSCT
    • 63.2. Allogeneic HSCT
    • References
  • 64. Umbilical Cord Blood Transplantation in Children and Adults
    Jaime Sanz, Paul Veys, and Vanderson Rocha.
    • 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 (See Table 64.3)
    • References
  • 65. Haploidentical HSCT
    Fabio Ciceri, Andrea Bacigalupo, Arjan Lankester, and Alice Bertaina.
    • 65.1. Introduction
    • 65.2. Ex Vivo TCD Platforms
    • 65.3. Unmanipulated Haploidentical HSCT
    • 65.4. Other Relevant Aspects of Haplo-HSCT
    • References
  • 66. Photopheresis in Adults and Pediatrics
    Hildegard Greinix.
    • 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.
    • 67.1. Introduction
    • 67.2. Influence of Overweight and Obesity on the Pharmacokinetics of Drugs
    • 67.3. Recommendations for Drug Dosing
    • References
  • 68. HSCT in Elderly Patients
    Rafael F. Duarte and Isabel Sánchez-Ortega.
    • 68.1. Introduction
    • 68.2. HSCT Activity in Elderly Patients
    • 68.3. HSCT Outcomes in Elderly Patients
    • 68.4. Assessment of Elderly Candidates for HSCT
    • References
• IX. Indications and Results Topic leaders: Rafael Duarte and Nicolaus Kröger
  • 69. Acute Myeloid Leukemia in Adults
    Jurjen Versluis, Jan J. Cornelissen, Charles Craddock, Miguel Á. Sanz, Jonathan Canaani, and Arnon Nagler.
    • 69.1. AML in CR1
      Jurjen Versluis and Jan J. Cornelissen.
    • 69.2. Allo-HSCT in Advanced AML
      Charles Craddock.
    • 69.3. Practical Issues in Allo-HSCT for AML
      Jonathan Canaani and Arnon Nagler.
    • 69.4. Acute Promyelocytic Leukemia
      Miguel Á. Sanz.
    • References
  • 70. Acute Myeloid Leukemia in Children
    Brenda E. S. Gibson, Martin G. Sauer, and Persis Amrolia.
    • 70.1. Introduction
    • 70.2. Prognostic Factors and Indications
    • 70.3. Conditioning Regimens
    • 70.4. Donor Selection Hierarchy and Stem Cell Source
    • 70.5. GVHD Prophylaxis
    • 70.6. Donor Lymphocyte Infusions (DLI)
    • 70.7. Management of Relapse Post transplant
    • References
  • 71. Acute Lymphoblastic Leukemia in Adults
    Matthias Stelljes and David I. Marks.
    • 71.1. Definition and Epidemiology
    • 71.2. Diagnosis
    • 71.3. Classification
    • 71.4. Risk Factors
    • 71.5. Prognostic Factors Used to Indicate Allo-HSCT in CR1
    • 71.6. First-Line Treatment
    • 71.7. Second-Line Treatment
    • 71.8. Autologous HSCT
    • 71.9. Allogeneic HSCT
    • References
  • 72. Acute Lymphoblastic Leukemia in Children and Adolescents
    Christina Peters, Franco Locatelli, and Peter Bader.
    • 72.1. Introduction
    • 72.2. Prognostic Factors and Indications for HSCT
    • 72.3. Donor Selection and Stem Cell Source
    • 72.4. Conditioning Regimen
    • 72.5. GVHD Prophylaxis
    • 72.6. Post-transplant Follow-Up and Interventions
    • 72.7. Results
    • References
  • 73. Myelodysplastic Syndromes
    Marie Robin and Theo de Witte.
    • 73.1. Introduction
    • 73.2. Indication of HSCT in MDS and Timing to Transplant
    • 73.3. Post-HSCT Outcomes
    • 73.4. Alternative Donors and Donor Choice
    • 73.5. Treatment Prior to HSCT
    • 73.6. Preparative Regimen
    • 73.7. Post-HSCT Treatment
    • References
  • 74. Pediatric MDS Including Refractory Cytopenia and Juvenile Myelomonocytic Leukemia
    Charlotte M. Niemeyer.
    • 74.1. Introduction
    • 74.2. Refractory Cytopenia of Childhood (RCC)
    • 74.3. Juvenile Myelomonocytic Leukemia (JMML)
    • References
  • 75. Myelodysplastic/Myeloproliferative Neoplasms
    Francesco Onida and Yves Chalandon.
    • 75.1. Definition, Epidemiology, Diagnosis, and Classification
    • 75.2. Risk Factors and Prognostic Index
    • 75.3. Pretransplantation Treatment
    • 75.4. Autologous HSCT
    • 75.5. Allogeneic HSCT
    • 75.6. Source of HSC
    • 75.7. Conditioning and GvHD Prophylaxis
    • 75.8. Maintenance/Post transplant Strategies
    • References
  • 76. Myeloproliferative Neoplasms
    Nicolaus Kröger and Yves Chalandon.
    • 76.1. Primary and Post ET/PV Myelofibrosis
      Nicolaus Kröger.
    • 76.2. Chronic Myeloid Leukemia
      Yves Chalandon.
    • References
  • 77. Severe Aplastic Anemia and PNH
    Régis Peffault de Latour, Antonio Risitano, and Carlo Dufour.
    • 77.1. Definition and Epidemiology
    • 77.2. Diagnosis and Indication for Treatment for SAA
    • 77.3. Treatment of SAA
    • 77.4. Treatment of PNH
    • References
  • 78. Fanconi’s Anemia and Other Hereditary Bone Marrow Failure Syndromes
    Cristina Díaz de Heredia, Marc Bierings, Jean-Hugues Dalle, Francesca Fioredda, and Brigitte Strahm.
    • 78.1. Introduction
    • 78.2. Fanconi’s Anemia
    • 78.3. Dyskeratosis Congenita
    • 78.4. Severe Congenital Neutropenia and Shwachman-Diamond Syndrome
    • 78.5. Diamond-Blackfan Anemia
    • 78.6. Congenital Amegakaryocytic Thrombocytopenia
    • Recommended References
  • 79. Hemoglobinopathies (Sickle Cell Disease and Thalassemia)
    Barbara Cappelli, Eliane Gluckman, Khaled Ghanem, and Miguel R. Abboud.
    • 79.1. HSCT for Sickle Cell Disease
      Barbara Cappelli and Eliane Gluckman.
    • 79.2. Thalassemia
      Khaled Ghanem and Miguel R. Abboud.
    • References
  • 80. Multiple Myeloma
    Joan Bladé, Benedetto Bruno, and Mohamad Mohty.
    • 80.1. Definition, Epidemiology, and Diagnosis
    • 80.2. Risk Stratification
    • 80.3. First-Line Treatment (Induction Prior to Auto-HSCT)
    • 80.4. Criteria of Response and Progression
    • 80.5. High-Dose Therapy (HDT), Consolidation, and Maintenance
    • 80.6. Treatment at Relapse After Auto-HSCT
    • 80.7. Allogenic HSCT in MM
    • 80.8. Allogenic HSCT and New Agents
    • 80.9. Indications of allo-HSCT in MM
    • Recommended References
  • 81. Systemic Light Chain Amyloidosis
    Monique Minnema and Stefan Schönland.
    • 81.1. Definition and Epidemiology
    • 81.2. Diagnosis
    • 81.3. Classification
    • 81.4. Risk Factors and Prognostic Scores
    • 81.5. First-Line Treatment
    • 81.6. Second-Line Treatment
    • 81.7. Autologous HSCT
    • 81.8. Allogeneic HCT
    • References
  • 82. POEMS Syndrome and Disease Produced by Other Monoclonal Immunoglobulins
    Gordon Cook and Montserrat Rovira.
    • 82.1. POEMS Syndrome
    • 82.2. Monoclonal Ig Deposition Disease
    • Recommended References
  • 83. Follicular Lymphoma
    Stephen Robinson.
    • 83.1. Introduction
    • 83.2. Autologous HSCT
    • 83.3. Allogeneic HSCT
    • 83.4. Patient Selection for HSCT in FL
    • 83.5. Auto-HSCT or Allo-HSCT as a First Transplant Procedure
    • 83.6. Allo-HSCT in Patients Relapsing After Auto-HSCT
    • 83.7. Conclusions
    • References
  • 84. Chronic Lymphocytic Leukemia
    Johannes Schetelig and Peter Dreger.
    • 84.1. Introduction
    • 84.2. Principles of Treatment for CLL
    • 84.3. Results of CLL Treatment with Pathway Inhibitors
    • 84.4. Allogeneic HSCT
    • 84.5. Summary and Perspectives
    • References
  • 85. Diffuse Large B-Cell Lymphoma
    Norbert Schmitz, Matthias Stelljes, and Ali Bazarbachi.
    • 85.1. Definition and Epidemiology
    • 85.2. Diagnosis
    • 85.3. Classification
    • 85.4. Risk Factors
    • 85.5. First-Line Treatment
    • 85.6. Second-Line Treatment
    • 85.7. Autologous HSCT
    • 85.8. Allogeneic HSCT
    • 85.9. Therapeutic Algorithm Recommended by the Authors (See Fig. 85.1)
    • 85.10. Long-Term Outcomes of Auto- and Allo-HSCT in Patients with R/R DLBCL (See Fig. 85.2)
    • References
  • 86. Mantle Cell Lymphoma
    Sascha Dietrich.
    • 86.1. Introduction
    • 86.2. Autologous HSCT
    • 86.3. Allogeneic HSCT
    • References
  • 87. Other T- and B-Aggressive Lymphomas and Lymphomas Associated with HIV
    Kai Hübel and Silvia Montoto.
    • 87.1. Burkitt Lymphoma (BL)
    • 87.2. Lymphoblastic Lymphoma (LBL)
    • 87.3. Peripheral T-Cell Lymphomas (PTCLs)
    • 87.4. HIV-Associated Lymphomas
    • References
  • 88. Classical Hodgkin’s Lymphoma
    Anna Sureda and Carmen Martínez.
    • 88.1. Definition and Epidemiology
    • 88.2. Diagnosis
    • 88.3. Classification
    • 88.4. Risk Factors
    • 88.5. First-Line Treatment
    • 88.6. Second-Line Treatment Before Auto-HSCT
    • 88.7. Autologous HSCT
    • 88.8. Tandem Auto-HSCT
    • 88.9. Disease Relapse After Auto-HSCT
    • 88.10. Allogeneic HSCT
    • 88.11. Therapeutic Algorithm Recommended by the Authors (Modified from Yethava et al.)
    • 88.12. Long-term outcomes of Auto-HSCT and Allo-HSCT in Patients with Relapsed/Refractory cHL (EBMT Database, with Permission)
    • References
  • 89. Primary Immunodeficiencies
    Michael Albert, Arjan Lankester, and Andrew Gennery.
    • 89.1. Introduction
    • 89.2. Diseases
    • 89.3. SCID
    • 89.4. Non-SCID Immunodeficiency
    • 89.5. Alternative Therapies
    • References
  • 90. Inborn Errors of Metabolism and Osteopetrosis
    Robert Wynn and Ansgar Schulz.
    • 90.1. Inborn Errors of Metabolism
    • 90.2. Osteopetrosis
    • Recommended References
  • 91. Autoimmune Disease
    Tobias Alexander, Basil Sharrack, Montserrat Rovira, Dominique Farge, and John A. Snowden.
    • 91.1. Introduction
    • 91.2. HSCT for Multiple Sclerosis (MS)
    • 91.3. HSCT for Systemic Sclerosis (SSc)
    • 91.4. HSCT for Systemic Lupus Erythematosus (SLE)
    • 91.5. HSCT for Crohn’s Disease (CD)
    • 91.6. Allogeneic HSCT for Autoimmune Diseases
    • 91.7. Other Indications
    • 91.8. Mechanisms of Action
    • 91.9. Conclusions and Future Directions
    • Acknowledgement
    • References
  • 92. Solid Tumours
    Ruth Ladenstein, Evgenia Glogova, and Francesco Lanza.
    • 92.1. Introduction
    • 92.2. Solid Tumours in Children and Adolescents
      Ruth Ladenstein and Evgenia Glogova.
    • 92.3. Solid Tumours in Adults
      Francesco Lanza.
    • References
• Appendix: EBMT Registry
  Carmen Ruiz de Elvira and Shelley Hewerdine.