Hematopoietic Stem Cell Transplantation and Cellular Therapies for Autoimmune Diseases

Richard K. Burt, MD (www.astemcelljourney.com) is Fulbright Scholar and Professor of Medicine at Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA, and the first Autoimmune Committee Chairperson for the International Bone Marrow Transplant Registry (IBMTR). Dominique Farge, MD, PhD is Professor of Internal Medicine at Saint-Louis Hospital, Assistance Publique-Hôpitaux de Paris, (AP-HP), University of Paris, Paris, France; Adjunct Professor at McGill University, Montreal, Canada; and Chairperson of the European Society for Blood and Marrow Transplantation Autoimmune Disease Working Party from 2010 to 2016.Milton A. Ruiz, MD, PhD is former Professor at the University of São Paulo and the head coordinator of the Hematopoietic Stem Cell Transplant Service of the Beneficência Portuguesa Hospital of São José do Rio Preto, São Paulo, Brazil.Riccardo Saccardi, MD is Director of Cellular Therapies and Transfusion Medicine Department at Careggi University Hospital in Florence, Italy and Chairperson of the European Society for Blood and Marrow Transplantation Autoimmune Disease Working Party from 2004 to 2010.John A. Snowden, MD is Consultant Haematologist & Director of Blood and Marrow Transplantation, Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK; Honorary Professor, Department of Oncology & Metabolism, The University of Sheffield, Sheffield, UK; Honorary Clinical Professor, The Cancer Institute, Department of Haematology, University College London, UK; and Chairperson of the European Society for Blood and Marrow Transplantation Autoimmune Disease Working Party from 2016 to 2020.

"Over the past 25 years, the utilization of hematopoietic stem cell transplantation (HSCT) specifically for the treatment of autoimmune diseases has gradually evolved from an initial state of ‘salvage therapy’ for severely affected patients towards a ‘standard procedure’ for selected patients in the major indications multiple sclerosis (MS) and systemic sclerosis (SSc). The recently published book entitled ‘Hematopoietic Stem Cell Transplantation and Cellular Therapies for Autoimmune Diseases’ now summarizes all recent developments and achievements in the area and provides the evidence and state-of-the-art of HSCT and emerging cellular therapies. The selected topics comprehensively highlight all relevant aspects of the procedures. The book was developed under the direction of Richard Burt from the Northwestern University, Chicago, one of pioneers and most eminent leaders in the field, in collaboration with the co-editors Dominique Farge, Milton A Ruiz, Riccardo Saccardi and John A Snowden. The book will attract a broad readership, particularly clinical scientists, clinicians and fellows dedicated to HSCT, both from a hematologic or autoimmune disease perspective.Overall, this book summarizes all relevant milestones in the field, extensively covering HSCT and cellular therapies in ADs, a field characterized by ongoing progress. It reflects on the dramatic developments and achievements of cellular therapies in recent years, along the dynamics of modern biologic and targeted therapies for ADs. The book is a valuable reading, serving as an ideal manual of good practices for hematologists, disease specialists, as well as researchers and young physicians. Finally, the book acknowledges the contributions and efforts of individual experts and personalities, first and foremost Richard Burt, all the co-editors and the authors of the book, to whom we bow for this work."- Tobias Alexander and Raffaella Greco and on behalf of the European Society for Blood and Marrow Transplantation (EBMT) Autoimmune Diseases Working Party (ADWP)- Tobias Alexander, Charité - Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Department of Rheumatology and Clinical Immunology, Berlin, Germany.- Raffaella Greco, Unit of Hematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University, Milan, Italy."This is a comprehensive collection of current knowledge on hematopoietic stem cell transplantation therapy for immune diseases. With 140 international experts in the field, this book serves as a valuable resource for those whopractice and research in the field of transplantation and those who use such knowledge to care for their patients."- Massoud Mahmoudi, DO, PhD, University of California San Francisco

• Table of ContentsSection 1- Biology of Immune CellsCh 1 B cells in aging, age-associated diseases and stem cell transplantationCh 2 T cell biology: changes with disease, aging, and hematopoietic stem cell transplantationCh 3 Plasma cells in autoimmunityCh 4 CAR T cells for AutoimmunityCh 5 Regulatory T cells for the treatment of autoimmune diseasesCh 6 Mesenchymal Stromal Cells Derived Extracellular VesiclesSection 2 - Biology of Stem CellsCh 7 Induced Pluripotent Stem Cells: the story of genetic reprogrammingCh 8 Mesenchymal stromal cell biology and clinical applicationCh 9 Neural Stem Cell BiologyCh 10 Hematopoietic stem cells: Hierarchical versus ContinuumCh 11 Biology of Umbilical Cord Blood CellsSection 3 – Regulatory requirementsCh 12 Quality, Accreditation, and Regulation in HSCT and Cellular Therapy for Autoimmune DiseasesCh 13 EUROPEAN PERSPECTIVE ON CELLULAR THERAPY: GOOD TISSUE PRACTICE (GTP) VERSUS GOOD MANUFACTURING PRACTICE (GMP).Ch 14 GMP Facility Requirements in the United States of AmericaSection 4 – Mesenchymal Stem Cells for Immune DiseasesCh 15 Mesenchymal stromal cell therapy for acute graft versus host disease – from bench to clinical approvalCh 16 Adipose-Derived Stem Cells for Systemic Sclerosis Digital UlcersCh 17 Musculoskeletal stromal/progenitor cells-based cell therapy for Rheumatoid Arthritis (RA)Ch 18 Mesenchymal Stromal cells for Systemic SclerosisCh 19 Mesenchymal stem cells (MSC) for systemic lupus erythematosus (SLE)Ch 20 Mesenchymal stem/stromal cells for multiple sclerosis and other neurological diseasesCh 21 Mesenchymal stromal cells for perianal fistulizing crohn’s diseaseCh 22 Umbilical Cord Blood or Mesenchymal Stromal Cells as Potential Therapies for Autism Spectrum DisorderSection 5 – General concepts: HSCT for Autoimmune DiseasesCh 23 Animal models of bone marrow transplantation for autoimmune diseasesCh 24 Autologous HSCT conditioning regimens for autoimmune diseasesCh 25 Immune reconstitution after hematopoietic stem cell transplantation in autoimmune diseaseCh 26 Microbiota and immune responseCh 27 Infectious complications in patients undergoing HSCT for autoimmune diseases.Ch 28 Early Toxicity and Supportive Care of HSCT for Autoimmune DiseasesCh 29 Late toxicity and supportive care of patients undergoing haematopoietic cell transplantation for autoimmune diseasesCh 30 Transfusion MedicineCh 31 International Registries Sites / Indications / Activity for HSCT in Autoimmune Diseases: the European Society for Blood and Marrow Transplantation (EBMT) and Center for International Blood and Marrow Transplant Research (CIBMTR)Ch 32 Hematopoietic stem cell mobilization, collection, apheresis, and harvest proceduresSection 6 – Allogeneic HSCT for Autoimmune diseaseCh 33 Hematopoietic Stem Cell Donor Registries: Search Strategies and HLA MethodologiesCh 34 Allogeneic HSCT and Graft-versus-Host Disease (GvHD): general principles with reference to autoimmune diseasesCh 35 Haematopoietic cell transplantation in monogenic autoimmune diseasesCh 36 Advances In The Pathogenesis And Treatment Of Immune Mediated Aplastic AnemiaSection 7- HSCT for autoimmune disease Multiple SclerosisCh 37 Magnetic Resonance Imaging in Multiple SclerosisCh 38 Multiple Sclerosis Ch 39 Disease-modifying therapies and supportive care of multiple sclerosisCh 40 Autologous hematopoietic stem cell transplantation for Secondary Progressive Multiple SclerosisCh 41 Autologous hematopoietic stem cell transplantation for Relapsing Remitting Multiple SclerosisCh 42 Immune mechanisms of autologous hematopoietic stem cell transplantation for multiple sclerosisChronic Inflammatory Demyelinating Polyradiculoneuropathy Ch 43 Basics and interpretation of electrodiagnostic testingCh 44 Diagnosis and Differentiation of CIDP from other peripheral polyneuropathiesCh 45 Hematopoietic stem cell transplantation for chronic inflammatory demyelinating polyradiculoneuropathyNeuromyelitis OpticaCh 46 Autologous and Allogeneic HSCT for AQP4-IgG positive Neuromyelitis Optica Spectrum DisordersSystemic SclerosisCh 47 Systemic SclerosisCh 48 The Systemic Sclerosis HeartCh 49 Scleroderma Renal Crisis with special considerations for Autologous Hematopoietic Stem Cell TransplantCh 50 Systemic Sclerosis and the LungCh 51 AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION IN SYSTEMIC SCLEROSISCrohn DiseaseCh 52 Crohn’s Disease: Incidence, Diagnosis, Standard Treatment, Prognosis, Outcome MeasuresCh 53 Hematopoietic Stem cell transplantation an alternative to treat Crohn’s diseaseType I Diabetes MellitusCh 54 Hematopoietic Stem Cell Transplantation for Type I Diabetes MellitusSystemic Lupus ErythematosusCh 55 Hematopoietic stem cell transplantation for systemic lupus erythematosus (SLE)Celiac DiseaseCh 56 Hematopoietic Stem Cell Transplantation for Refractory Celiac Disease type IISection 8 – Developing indications for HSCTCh 57 Haematopoietic stem cell transplantation for Juvenile Idiopathic ArthritisCh 58 Haematopoietic Stem Cell Transplantation for Rheumatoid ArthritisCh 59 Hematopoietic Stem Cell Transplantation for Behçet's SyndromeCh 60 Hematopoietic Stem Cell Transplantation and Myasthenia GravisCh 61 Autologous Haematopoietic Stem Cell Transplantation in Stiff Person Spectrum DisorderCh 62 Hematopoietic Stem Cell Transplant-Based Treatment for Psychiatric DisordersCh 63 Hematopoietic Stem Cell Transplantation for Psoriasis and Pemphigus VulgarisSection 9 – Ethics and economicsCh 64 Health Economics of Multiple SclerosisCh 65 Ethics of Stem Cells and Stem Cell Transplantation for Autoimmune diseasesCh 66 Is Healthcare a Human Right?Ch 67 Appendices