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. This book succinctly covers the entire field of hematopoietic stem cell therapy for the treatment of autoimmune diseases. Utilizing the authors' extensive experience, this title provides advice on best practice and evaluates the advantages and disadvantages of HSCT in the treatment of a variety of autoimmune diseases versus current conventional treatments. Arranged by disease, it provides the ideal guide to HSCT for all conditions, from multiple sclerosis (MS) to diabetes, and provides general guidance on the transplantation process.
Key Features
Outlines therapies and techniques for HSCT for autoimmune diseases, and d
Discusses its the advantages of HSCT over conventional therapies
Reviews the entire process of stem cell therapy from harvest and ethics to indications of efficacy.
Table 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
Ch 13 EUROPEAN PERSPECTIVE ON CELLULAR THERAPY: GOOD TISSUE PRACTICE (GTP) VERSUS GOOD MANUFACTURING PRACTICE (GMP).
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
Ch 51 AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION IN SYSTEMIC SCLEROSIS
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
