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

part Section I|57 pages

Biology of Immune Cells

chapter 1|8 pages

B Cells in Aging, Age-Associated Diseases and Stem Cell Transplantation

ByDaniela Frasca, Maria Romero

chapter 3|7 pages

Plasma Cells in Autoimmunity

ByFalk Hiepe, Alexander Tobias, Andreas Radbruch

chapter 4|7 pages

Car T Cells for Autoimmunity

ByMichael C. Milone

chapter 5|14 pages

Regulatory T Cells for the Treatment of Autoimmune Diseases

ByClaudia Selck, Margarita Dominguez-Villar

chapter 6|11 pages

Mesenchymal Stromal Cell-Derived Extracellular Vesicles

ByInés Colmegna, Janusz Rak

part Section II|44 pages

Biology of Stem Cells

chapter 7|10 pages

Induced Pluripotent Stem Cells: The Story of Genetic Reprogramming

ByRichard K. Burt

chapter 8|9 pages

Mesenchymal Stromal Cell Biology and Clinical Application

BySéverine Loisel, Karin Tarte

chapter 9|8 pages

Neural Stem Cell Biology

ByAngela Genchi, Beatrice Von Wunster, Paola Panina-Bordignon, Gianvito Martino

chapter 10|6 pages

Hematopoietic Stem Cells

Hierarchical versus Continuum
ByPeter Quesenberry, Sicheng Wen, Laura Goldberg, Mark Dooner

chapter 11|10 pages

Biology of Umbilical Cord Blood Cells

ByGesine Kögler, Stefanie Liedtke

part Section III|20 pages

Regulatory Requirements

chapter 12|7 pages

Quality, Accreditation, and Regulation in Hematopoietic Stem Cell Transplantation

ByEoin McGrath, Joseph Schwartz, Warkentin, Riccardo Saccardi, John A. Snowden

chapter 13|7 pages

European Perspective on Cellular Therapy

Good Tissue Practice (GTP) versus Good Manufacturing Practice (GMP)
ByElodie Kerjean, Bechara Mfarrej, Christian Chabannon

chapter 14|5 pages

Gmp Facility Requirements in the United States

ByCheryl Stratton, Ann LeFever

part Section IV|67 pages

Mesenchymal Stem Cells for Immune Diseases

chapter 16|8 pages

Adipose-Derived Stem Cells for Systemic Sclerosis Digital Ulcers

ByNicoletta Del Papa, Antonina Minniti, Francesca Pignataro, Wanda Maglione, Dominique Farge, Roberto F. Caporali

chapter 17|9 pages

Musculoskeletal Stromal/Progenitor Cell-Based Cell Therapy for Rheumatoid Arthritis (RA)

MSC for Cell Therapy in RA
ByYves-Marie Pers, Dominique Farge, Christian Jorgensen

chapter 18|7 pages

Mesenchymal Stromal Cells for Systemic Sclerosis

ByDominique Farge

chapter 19|9 pages

Mesenchymal Stem Cells (MSC) for Systemic Lupus Erythematosus (SLE)

ByLingyun Sun, Xinran Yuan

chapter 20|8 pages

Mesenchymal Stem/Stromal Cells for Multiple Sclerosis and Other Neurological Diseases

ByAntonio Uccelli, Tiziana Vigo, Alice Laroni

chapter 21|11 pages

Mesenchymal Stromal cells for Perianal Fistulizing Crohn's Disease

ByAntonio Giordano, Julián Panés

part Section V|103 pages

General Concepts — HSCT for Autoimmune Diseases

chapter 23|14 pages

Animal Models of Bone Marrow Transplantation for Autoimmune Diseases

ByYasushi Adachi, Ming Li, Richard K. Burt, Naoki Hosaka, Susumu Ikehara

chapter 24|9 pages

Autologous HSCT Conditioning Regimens for Autoimmune Diseases

ByRichard K. Burt, Dominique Farge, Riccardo Saccardi, Kathleen Quigley, Xiaoqiang Han, John A. Snowden

chapter 25|9 pages

Immune Reconstitution after Hematopoietic Stem Cell Transplantation in Autoimmune Disease

ByPaolo A. Muraro, Maria T. Cencioni

chapter 26|7 pages

Microbiota and Immune Response

ByErnst Holler, Hendrik Poeck, Daniela Weber

chapter 27|12 pages

Infectious Complications in Patients Undergoing Hsct for Autoimmune Diseases

ByRichard K. Burt, Dominique Farge

chapter 28|11 pages

Early Toxicity and Supportive Care of Hsct for Autoimmune Diseases

ByJohn A. Snowden, Azza Ismail, Richard K. Burt, Helen Jessop

chapter 29|12 pages

Late Toxicity and Supportive Care of Patients Undergoing Hematopoietic Cell Transplantation for Autoimmune Diseases

ByNina Salooja, Richard K. Burt, John A. Snowden

chapter 30|7 pages

Transfusion Medicine

ByKhaled El-Ghariani

chapter 31|11 pages

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

chapter 32|10 pages

Hematopoietic Stem Cell Mobilization, Collection, Apheresis, and Harvest Procedures

ByEmilian Snarski, Richard K. Burt

part Section VI|43 pages

Allogeneic HSCT for Autoimmune Disease

chapter 33|7 pages

Hematopoietic Stem Cell Donor Registries

Search Strategies and HLA Methodologies
ByC. Stavropoulos, V. Gkioka, D. Charron

chapter 34|10 pages

Allogeneic HSCT and Graft Versus Host Disease (GvHD)

General Principles with Reference to Autoimmune Diseases
ByJose Ros-Soto, Richard K. Burt, John A. Snowden

chapter 35|16 pages

Hematopoietic Cell Transplantation in Monogenic Autoimmune Diseases

ByS.H. Lum, E.P. Buddingh, M.A. Slatter, A.C. Lankester

chapter 36|9 pages

Advances in the Pathogenesis and Treatment of Immune-Mediated Aplastic Anemia

ByShinji Nakao, Sabrina Giammarco, Andrea Bacigalupo

part Section VII|191 pages

HSCT for Autoimmune Disease

chapter 37|8 pages

Magnetic Resonance Imaging in Multiple Sclerosis

ByFlavia Nelson

chapter 38|11 pages

Multiple Sclerosis

ByAzza Ismail, Richard K. Burt, Joachim Burman, Basil Sharrack

chapter 39|14 pages

Disease-Modifying Therapies and Supportive Care of Multiple Sclerosis

ByEdith Graham, Eileah Loda, Roumen Balabanov

chapter 40|6 pages

Autologous Hematopoietic Stem Cell Transplantation for Secondary Progressive Multiple Sclerosis

ByRichard K. Burt, Basil Sharrack, Roumen Balabanov, John Rose, Xiaoqiang Han, Kathleen Quigley, Riccardo Saccardi

chapter 41|11 pages

Autologous Hematopoietic Stem Cell Transplantation for Relapsing-Remitting Multiple Sclerosis

ByRichard K. Burt, Basil Sharrack, Joachim Burman, Flavia Nelson, John Rose, Xiaoqiang Han, Kathleen Quigley, Riccardo Saccardi

chapter 43|8 pages

Chronic Inflammatory Demyelinating Polyradiculoneuropathy

Basics and Interpretation of Electrodiagnostic Testing
ByHamza Malek

chapter 44|7 pages

Diagnosis and Differentiation of Cidp from Other Peripheral Polyneuropathies

ByCéline Labeyrie, Fanny Urbain, Cristina Castilla Llorente

chapter 45|11 pages

Hematopoietic Stem Cell Transplantation for Chronic Inflammatory Demyelinating Polyradiculoneuropathy

ByGavin Brittain, Richard K. Burt, Basil Sharrack

chapter 46|10 pages

Neuromyelitis Optica

Autologous and Allogeneic HSCT for AQP4-IgG-Positive Neuromyelitis Optica Spectrum Disorders
ByRaffaella Greco, Shailee Shah, Sean J. Pittock, Richard K. Burt

chapter 47|15 pages

Systemic Sclerosis

ByKatja Lakota, Katja Perdan Pirkmajor, John Varga

chapter 48|14 pages

The Systemic Sclerosis Heart

BySanjiv J. Shah

chapter 49|7 pages

Scleroderma Renal Crisis with Special Considerations for Autologous Hematopoietic Stem Cell Transplant

ByMarie Hudson, Harold L. Atkins, Cybele Ghossein

chapter 50|16 pages

Systemic Sclerosis and the Lung

ByDavid G. Kiely, Robin Condliffe

chapter 51|10 pages

Autologous Hematopoietic Stem Cell Transplantation in Systemic Sclerosis

ByDominique Farge, Zora Marjanovic, Richard K. Burt

chapter 52|9 pages

Crohn's Disease

Incidence, Diagnosis, Standard Treatment, Prognosis, Outcome Measures
ByGabriel Marcellier, Xavier Treton

chapter 53|11 pages

Hematopoietic Stem Cell Transplantation an Alternative to Treat Crohn's Disease

ByMilton Artur Ruiz, Montserrat Rovira, Elena Ricart, Roberto Luiz Kaiser, Richard K. Burt

chapter 54|9 pages

Type 1 Diabetes Mellitus

Hematopoietic Stem Cell Transplantation for Type 1 Diabetes Mellitus
ByCarlos Eduardo Couri, Maria Carolina Oliveira

chapter 55|8 pages

Systemic Lupus Erythematosus

Hematopoietic Stem Cell Transplantation for Systemic Lupus Erythematosus (SLE)
ByTobias Alexander, Renate Arnold, Falk Hiepe, Richard K. Burt

part Section VIII|73 pages

HSCT for Autoimmune Disease — Developing Indications

chapter 56|8 pages

Refractory Celiac Disease

Hematopoietic Stem Cell Transplantation for Refractory Celiac Disease Type II
ByAbdulbaqi Al-Toma

chapter 57|12 pages

Hematopoietic Stem Cell Transplantation for Juvenile Idiopathic Arthritis

ByMario Abinun, Su Han Lum, Arina Lazareva, Paul Veys, Mary A. Slatter

chapter 58|10 pages

Hematopoietic Stem Cell Transplantation for Rheumatoid Arthritis

ByRoss Penglase, Robert D. Sandler, John Moore, John A. Snowden

chapter 59|12 pages

Hematopoietic Stem Cell Transplantation for BehÇet's Syndrome

ByLeandro Lara do Prado, Lilian Piron-Ruiz, Milton Ruiz, Célio Roberto Gonçalves

chapter 60|11 pages

Hematopoietic Stem Cell Transplantation and Myasthenia Gravis

ByOsman Radhwi, Harold Atkins

chapter 61|9 pages

Autologous Hematopoietic Stem Cell Transplantation in Stiff Person Spectrum Disorder

ByZeid Yasiry, Richard K. Burt, Basil Sharrack

chapter 62|5 pages

Hematopoietic Stem Cell Transplant-Based Treatment for Psychiatric Disorders

BySophia Chaudry, Sander Markx

chapter 63|5 pages

Hematopoietic Stem Cell Transplantation for Psoriasis and Pemphigus Vulgaris

ByFloyd Silva, John Durkin, Leslie Andritsos

part Section IX|13 pages

Ethics and Economics

chapter 65|4 pages

Is Health Care a Human Right?

ByLawrence J. Metzger