Hematopoietic Stem Cell Transplantation and Cellular Therapies for Autoimmune Diseases

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

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

Biography

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