1st Edition

Hematopoietic Stem Cell Transplantation and Cellular Therapies for Autoimmune Diseases

    686 Pages 172 Color & 29 B/W Illustrations
    by CRC Press

    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

    able of Contents

    Section 1- Biology of Immune Cells
    Ch 1 B cells in aging, age-associated diseases and stem cell transplantation
    Daniela Frasca, Maria Romero

    Ch 2 T cell biology: changes with disease, aging, and hematopoietic stem cell transplantation
    Valquiria Bueno

    Ch 3 Plasma cells in autoimmunity
    Falk Hiepe, Tobias Alexander, and Andreas Radbruch

    Ch 4 CAR T cells for Autoimmunity
    Michael C. Milone

    Ch 5 Regulatory T cells for the treatment of autoimmune diseases
    Claudia Selck and Margarita Dominguez-Villar

    Ch 6 Mesenchymal Stromal Cells Derived Extracellular Vesicles
    Inés Colmegna and Janusz Rak

    Section 2 - Biology of Stem Cells

    Ch 7 Induced Pluripotent Stem Cells: the story of genetic reprogramming
    Richard K Burt

    Ch 8 Mesenchymal stromal cell biology and clinical application
    Séverine Loisel & Karin Tarte

    Ch 9 Neural Stem Cell Biology
    Angela Genchi, Beatrice von Wunster, Paola Panina-Bordignon and Gianvito Martino

    Ch 10 Hematopoietic stem cells: Hierarchical versus Continuum
    Peter Quesenberry, Sicheng Wen, Laura Goldberg and Mark Dooner

    Ch 11 Biology of Umbilical Cord Blood Cells
    Gesine Kogler and Stefanie Liedtke

    Section 3 – Regulatory requirements
    Ch 12 Quality, Accreditation, and Regulation in HSCT and Cellular Therapy for Autoimmune Diseases
    Eoin McGrath, Joseph Schwartz, Phyllis I. Warkentin, Riccardo Saccardi, John A. Snowden

    Elodie Kerjean, Bechara Mfarrej, & Christian Chabannon

    Ch 14 GMP Facility Requirements in the United States of America
    Cheryl Stratton and Ann LeFever

    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
    Erik Boberg and Katarina Le Blanc

    Ch 16 Adipose-Derived Stem Cells for Systemic Sclerosis Digital Ulcers
    Nicoletta Del Papa, Antonina Minniti, Francesca Pignataro, Wanda Maglione and Roberto F. Caporali

    Ch 17 Musculoskeletal stromal/progenitor cells-based cell therapy for Rheumatoid Arthritis (RA)
    Yves-Marie PERS, Dominique Farge and Christian Jorgensen

    Ch 18 Mesenchymal Stromal cells for Systemic Sclerosis
    Dominique Farge

    Ch 19 Mesenchymal stem cells (MSC) for systemic lupus erythematosus (SLE)
    Lingyun Sun and Xinran Yuan

    Ch 20 Mesenchymal stem/stromal cells for multiple sclerosis and other neurological diseases
    Antonio Uccelli, Tiziana Vigo and Alice Laroni

    Ch 21 Mesenchymal stromal cells for perianal fistulizing crohn’s disease
    Antonio Giordano and Julián Panés

    Ch 22 Umbilical Cord Blood or Mesenchymal Stromal Cells as Potential Therapies for Autism Spectrum Disorder
    Jessica M. Sun and Joanne Kurtzberg

    Section 5 – General concepts: HSCT for Autoimmune Diseases

    Ch 23 Animal models of bone marrow transplantation for autoimmune diseases
    Yasushi Adachi, Ming Li, Richard K. Burt, Naoki Hosaka, Susumu Ikehara

    Ch 24 Autologous HSCT conditioning regimens for autoimmune diseases
    Richard K Burt, Dominique Farge, Riccardo Saccardi, Kathleen Quigley, Xiaoqiang Han and John A. Snowden

    Ch 25 Immune reconstitution after hematopoietic stem cell transplantation in autoimmune disease
    Paolo A. Muraro and Maria T. Cencion

    Ch 26 Microbiota and immune response
    Ernst Holler, Hendrik Poeck, and Daniela Weber

    Ch 27 Infectious complications in patients undergoing HSCT for autoimmune diseases.
    Richard K Burt and Dominique Farge

    Ch 28 Early Toxicity and Supportive Care of HSCT for Autoimmune Diseases
    John A Snowden, Azza Ismail, Richard K Burt, Helen Jessop

    Ch 29 Late toxicity and supportive care of patients undergoing haematopoietic cell transplantation for autoimmune diseases
    Nina Salooja, Richard K Burt and John A. Snowden

    Ch 30 Transfusion Medicine
    Khaled El-Ghariani

    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)
    John A. Snowden, Raffaella Greco, Manuela Badoglio, Myriam Labopin, Omar F. Dávila Alvelo and Marcelo C. Pasquini

    Ch 32 Hematopoietic stem cell mobilization, collection, apheresis, and harvest procedures
    Emilian Snarski and Richard K Burt

    Section 6 – Allogeneic HSCT for Autoimmune disease
    Ch 33 Hematopoietic Stem Cell Donor Registries: Search Strategies and HLA Methodologies
    Catherine Stavropoulos-Giokas, Vasiliki Gkioka and Dominique Charron

    Ch 34 Allogeneic HSCT and Graft-versus-Host Disease (GvHD): general principles with reference to autoimmune diseases
    Jose Ros-Soto, Richard K Burt and John A. Snowden

    Ch 35 Haematopoietic cell transplantation in monogenic autoimmune diseases
    Su Han Lum, Emilie P Buddingh, Mary A Slatter and Arjan C Lankester

    Ch 36 Advances In The Pathogenesis And Treatment Of Immune Mediated Aplastic Anemia
    Shinji Nakao, Sabrina Giammarco and Andrea Bacigalupo

    Section 7- HSCT for autoimmune disease Multiple Sclerosis

    Ch 37 Magnetic Resonance Imaging in Multiple Sclerosis
    Flavia Nelson

    Ch 38 Multiple Sclerosis
    Azza Ismail, Richard K Burt, Joachim Burman, Basil Sharrack

    Ch 39 Disease-modifying therapies and supportive care of multiple sclerosis
    Edith Graham, Eileah Loda, and Roumen Balabano

    Ch 40 Autologous hematopoietic stem cell transplantation for Secondary Progressive Multiple Sclerosis
    Richard K Burt, Basil Sharrack, Roumen Balabanov, John Rose, Xiaoqiang Han, Kathleen Quigley and Riccardo Saccardi

    Ch 41 Autologous hematopoietic stem cell transplantation for Relapsing Remitting Multiple Sclerosis
    Richard K Burt, Basil Sharack, Joachim Burman, Flavia Nelson, John Rose, Xiaoqiang Han, Kathleen Quigley and Riccardo Saccardi

    Ch 42 Immune mechanisms of autologous hematopoietic stem cell transplantation for multiple sclerosis
    Joachim Burman

    Chronic Inflammatory Demyelinating Polyradiculoneuropathy

    Ch 43 Basics and interpretation of electrodiagnostic testing
    Hamza Malek

    Ch 44 Diagnosis and Differentiation of CIDP from other peripheral polyneuropathies
    Céline Labeyrie Fanny Urbain and  Cristina Castilla Llorente

    Ch 45 Hematopoietic stem cell transplantation for chronic inflammatory demyelinating polyradiculoneuropathy
    Gavin Brittain, Richard K Burt, Basil Sharrack

    Neuromyelitis Optica

    Ch 46 Autologous and Allogeneic HSCT for AQP4-IgG positive Neuromyelitis Optica Spectrum Disorders
    Raffaella Greco, Shailee Shah, Sean J Pittock and Richard K Burt

    Systemic Sclerosis

    Ch 47 Systemic Sclerosis
    Katja Lakota, Katja Perdan Pirkmajer and John Varga

    Ch 48 The Systemic Sclerosis Heart
    Sanjiv J. Shah

    Ch 49 Scleroderma Renal Crisis with special considerations for Autologous Hematopoietic Stem Cell Transplant
    Marie Hudson, Harold L. Atkins and Cybele Ghossein

    Ch 50 Systemic Sclerosis and the Lung
    David G Kiely and Robin Condliffe

    Dominique Farge, Zora Marjanovic, Richard K Burt

    Crohn Disease
    Ch 52 Crohn’s Disease: Incidence, Diagnosis, Standard Treatment, Prognosis, Outcome Measures
    Gabriel Marcellier MD, Xavier Treton

    Ch 53 Hematopoietic Stem cell transplantation an alternative to treat Crohn’s disease
    Milton Artur Ruiz, Montserrat Rovira, Elena Ricart, Roberto Luiz Kaiser Junior, Richard K Burt

    Type I Diabetes Mellitus

    Ch 54 Hematopoietic Stem Cell Transplantation for Type I Diabetes Mellitus
    Carlos Eduardo Couri and Maria Carolina Oliveira

    Systemic Lupus Erythematosus

    Ch 55 Hematopoietic stem cell transplantation for systemic lupus erythematosus (SLE)
    Tobias Alexander, Renate Arnold, Falk Hiepe, and Richard K Burt

    Celiac Disease

    Ch 56 Hematopoietic Stem Cell Transplantation for Refractory Celiac Disease type II
    Abdulbaqi Al-Toma

    Section 8 – Developing indications for HSCT

    Ch 57 Haematopoietic stem cell transplantation for Juvenile Idiopathic Arthritis
    Mario Abinun, Su Han Lum, Arina Lazareva, Paul Veys, Mary A Slatter

    Ch 58 Haematopoietic Stem Cell Transplantation for Rheumatoid Arthritis
    Ross Penglase, Robert D Sandler, John Moore and John A. Snowden

    Ch 59 Hematopoietic Stem Cell Transplantation for Behçet's Syndrome
    Leandro Lara do Prado, Lilian Piron-Ruiz, Milton A. Ruiz, and Célio Roberto Gonçalves

    Ch 60 Hematopoietic Stem Cell Transplantation and Myasthenia Gravis
    Osman Radhwi and Harold Atkins

    Ch 61 Autologous Haematopoietic Stem Cell Transplantation in Stiff Person Spectrum Disorder
    Zeid Yasiry, Richard K Burt, Basil Sharrack

    Ch 62 Hematopoietic Stem Cell Transplant-Based Treatment for Psychiatric Disorders
    Sophia Chaudry and Sander Markx

    Ch 63 Hematopoietic Stem Cell Transplantation for Psoriasis and Pemphigus Vulgaris

    Floyd Silva, John Durkin and Leslie Andritsos

    Section 9 – Ethics and economics

    Ch 64 Health Economics of Multiple Sclerosis
    Daniel M. Hartung

    Ch 65 Ethics of Stem Cells and Stem Cell Transplantation for Autoimmune diseases
    Kevin FitzGerald SJ

    Ch 66 Is Healthcare a Human Right?
    Lawrence J. Metzger

    Ch 67 Appendices
    Indira Arnautovic and Kathleen Quigley


    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 who
    practice 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