This book summarizes the global progress in medical and scientific research toward converting traditionally chronic autoimmune diseases into a drug-free reversible illness using hematopoietic stem cell transplantation (HSCT) and other cellular therapies such as T regulatory cells (Treg), mesenchymal stromal/stem cells, and chimeric antigen receptor T (CAR T) cells in order to reintroduce sustained immune tolerance. This title provides information on different types of stem cells and immune cells; post-transplant immune regeneration; cellular regulatory requirements; ethical and economic considerations; and the advantages and disadvantages of HSCT in the treatment of a variety of autoimmune diseases versus current conventional treatments. Arranged by disease, the text provides a comprehensive guide to HSCT for all types of autoimmune/immune disorders including monogenetic autoimmune diseases; autoimmune aplastic anemia; neurologic immune diseases including multiple sclerosis, chronic inflammatory demyelinating polyneuropathy, neuromyelitis optica, and stiff person syndrome; rheumatologic diseases such as systemic sclerosis and systemic lupus erythematosus; dermatologic diseases such as pemphigus; gastrointestinal disorders such as Crohn’s disease and celiac disease; and immune-mediated endocrinologic disease type I diabetes mellitus. Guidance is provided on the transplantation technique, cell collection and processing, conditioning regimens, infections, and early and late complications.
Key Features
Outlines therapies and techniques for HSCT for autoimmune diseasesDiscusses the advantages of HSCT over conventional therapiesReviews the entire process of stem cell therapy from harvest and ethics to indications, efficacy, and regulatory oversight
This book summarizes the progress made across the international medical and scientific research communities towards the goal of converting a chronic autoimmune disease into a drug-free reversible illness using hematopoietic stem cell transplantation (HSCT) and other cellular therapies to generate sustained immune tolerance.
Table of Contents
Section 1- Biology of Immune Cells
Ch 1 B cells in aging, age-associated diseases and stem cell transplantation
Ch 2 T cell biology: changes with disease, aging, and hematopoietic stem cell
transplantation
Ch 3 Plasma cells in autoimmunity
Ch 4 CAR T cells for Autoimmunity
Ch 5 Regulatory T cells for the treatment of autoimmune diseases
Ch 6 Mesenchymal Stromal Cells Derived Extracellular Vesicles
Section 2 - Biology of Stem Cells
Ch 7 Induced Pluripotent Stem Cells: the story of genetic reprogramming
Ch 8 Mesenchymal stromal cell biology and clinical application
Ch 9 Neural Stem Cell Biology
Ch 10 Hematopoietic stem cells: Hierarchical versus Continuum
Ch 11 Biology of Umbilical Cord Blood Cells
Section 3 Regulatory requirements
Ch 12 Quality, Accreditation, and Regulation in HSCT and Cellular Therapy for
Autoimmune Diseases
Ch 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 America
Section 4 Mesenchymal Stem Cells for Immune Diseases
Ch 15 Mesenchymal stromal cell therapy for acute graft versus host disease
from bench to clinical approval
Ch 16 Adipose-Derived Stem Cells for Systemic Sclerosis Digital Ulcers
Ch 17 Musculoskeletal stromal/progenitor cells-based cell therapy for
Rheumatoid Arthritis (RA)
Ch 18 Mesenchymal Stromal cells for Systemic Sclerosis
Ch 19 Mesenchymal stem cells (MSC) for systemic lupus erythematosus (SLE)
Ch 20 Mesenchymal stem/stromal cells for multiple sclerosis and other
neurological diseases
Ch 21 Mesenchymal stromal cells for perianal fistulizing crohns disease
Ch 22 Umbilical Cord Blood or Mesenchymal Stromal Cells as Potential
Therapies for Autism Spectrum Disorder
Section 5 General concepts: HSCT for Autoimmune Diseases
Ch 23 Animal models of bone marrow transplantation for autoimmune diseases
Ch 24 Autologous HSCT conditioning regimens for autoimmune diseases
Ch 25 Immune reconstitution after hematopoietic stem cell transplantation in
autoimmune disease
Ch 26 Microbiota and immune response
Ch 27 Infectious complications in patients undergoing HSCT for autoimmune
diseases.
Ch 28 Early Toxicity and Supportive Care of HSCT for Autoimmune Diseases
Ch 29 Late toxicity and supportive care of patients undergoing haematopoietic
cell transplantation for autoimmune diseases
Ch 30 Transfusion Medicine
Ch 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 procedures
Section 6 Allogeneic HSCT for Autoimmune disease
Ch 33 Hematopoietic Stem Cell Donor Registries: Search Strategies and HLA
Methodologies
Ch 34 Allogeneic HSCT and Graft-versus-Host Disease (GvHD): general
principles with reference to autoimmune diseases
Ch 35 Haematopoietic cell transplantation in monogenic autoimmune diseases
Ch 36 Advances In The Pathogenesis And Treatment Of Immune Mediated Aplastic
Anemia
Section 7- HSCT for autoimmune disease Multiple Sclerosis
Ch 37 Magnetic Resonance Imaging in Multiple Sclerosis
Ch 38 Multiple Sclerosis
Ch 39 Disease-modifying therapies and supportive care of multiple sclerosis
Ch 40 Autologous hematopoietic stem cell transplantation for Secondary
Progressive Multiple Sclerosis
Ch 41 Autologous hematopoietic stem cell transplantation for Relapsing
Remitting Multiple Sclerosis
Ch 42 Immune mechanisms of autologous hematopoietic stem cell transplantation
for multiple sclerosis
Chronic Inflammatory Demyelinating Polyradiculoneuropathy
Ch 43 Basics and interpretation of electrodiagnostic testing
Ch 44 Diagnosis and Differentiation of CIDP from other peripheral
polyneuropathies
Ch 45 Hematopoietic stem cell transplantation for chronic inflammatory
demyelinating polyradiculoneuropathy
Neuromyelitis Optica
Ch 46 Autologous and Allogeneic HSCT for AQP4-IgG positive Neuromyelitis
Optica Spectrum Disorders
Systemic Sclerosis
Ch 47 Systemic Sclerosis
Ch 48 The Systemic Sclerosis Heart
Ch 49 Scleroderma Renal Crisis with special considerations for Autologous
Hematopoietic Stem Cell Transplant
Ch 50 Systemic Sclerosis and the Lung
Ch 51 AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION IN SYSTEMIC
SCLEROSIS
Crohn Disease
Ch 52 Crohns Disease: Incidence, Diagnosis, Standard Treatment, Prognosis,
Outcome Measures
Ch 53 Hematopoietic Stem cell transplantation an alternative to treat Crohns
disease
Type I Diabetes Mellitus
Ch 54 Hematopoietic Stem Cell Transplantation for Type I Diabetes Mellitus
Systemic Lupus Erythematosus
Ch 55 Hematopoietic stem cell transplantation for systemic lupus
erythematosus (SLE)
Celiac Disease
Ch 56 Hematopoietic Stem Cell Transplantation for Refractory Celiac Disease
type II
Section 8 Developing indications for HSCT
Ch 57 Haematopoietic stem cell transplantation for Juvenile Idiopathic
Arthritis
Ch 58 Haematopoietic Stem Cell Transplantation for Rheumatoid Arthritis
Ch 59 Hematopoietic Stem Cell Transplantation for Behçet's Syndrome
Ch 60 Hematopoietic Stem Cell Transplantation and Myasthenia Gravis
Ch 61 Autologous Haematopoietic Stem Cell Transplantation in Stiff Person
Spectrum Disorder
Ch 62 Hematopoietic Stem Cell Transplant-Based Treatment for Psychiatric
Disorders
Ch 63 Hematopoietic Stem Cell Transplantation for Psoriasis and Pemphigus
Vulgaris
Section 9 Ethics and economics
Ch 64 Health Economics of Multiple Sclerosis
Ch 65 Ethics of Stem Cells and Stem Cell Transplantation for Autoimmune
diseases
Ch 66 Is Healthcare a Human Right?
Ch 67 Appendices
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.
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