Hematopoietic Stem Cell Transplantation and Cellular Therapies for Autoimmune Diseases

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 diseases
• Discusses the advantages of HSCT over conventional therapies
• Reviews the entire process of stem cell therapy from harvest and ethics to indications, efficacy, and regulatory oversight

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 crohn’s 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 Crohn’s Disease: Incidence, Diagnosis, Standard Treatment, Prognosis, Outcome Measures

Ch 53 Hematopoietic Stem cell transplantation an alternative to treat Crohn’s 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.