Sue-Ann Ng1, Keith M Sullivan2. 1. Department of Rheumatology and Immunology, Singapore General Hospital, Singapore. 2. Department of Medicine, Duke University Medical Center, Durham, North Carolina, USA.
Abstract
PURPOSE OF REVIEW: Autologous hematopoietic stem cell transplantation (HSCT) is a promising therapeutic modality for severe autoimmune diseases. In this review, we will outline the immunological mechanisms and the clinical evidence and experiences for therapeutic HSCT in autoimmune diseases, with particular focus on systemic sclerosis and multiple sclerosis. RECENT FINDINGS: Approximately 3000 patients with autoimmune diseases worldwide have been treated with HSCT. HSCT in systemic sclerosis has been shown in three randomized controlled trials to be associated with significant long-term event-free survival despite some transplant-related mortality in the first year. A recent controlled trial in multiple sclerosis has also show benefit with transplant. SUMMARY: The aim of HSCT is to 'reset' one's immune system into a naïve and self-tolerant state through immune depletion and regulation. HSCT requires careful patient selection, close collaboration between physicians and expertise of transplant team to ensure optimal outcome.
PURPOSE OF REVIEW: Autologous hematopoietic stem cell transplantation (HSCT) is a promising therapeutic modality for severe autoimmune diseases. In this review, we will outline the immunological mechanisms and the clinical evidence and experiences for therapeutic HSCT in autoimmune diseases, with particular focus on systemic sclerosis and multiple sclerosis. RECENT FINDINGS: Approximately 3000 patients with autoimmune diseases worldwide have been treated with HSCT. HSCT in systemic sclerosis has been shown in three randomized controlled trials to be associated with significant long-term event-free survival despite some transplant-related mortality in the first year. A recent controlled trial in multiple sclerosis has also show benefit with transplant. SUMMARY: The aim of HSCT is to 'reset' one's immune system into a naïve and self-tolerant state through immune depletion and regulation. HSCT requires careful patient selection, close collaboration between physicians and expertise of transplant team to ensure optimal outcome.
Authors: John J Moore; Jennifer C Massey; Carole D Ford; Melissa L Khoo; John J Zaunders; Kevin Hendrawan; Yael Barnett; Michael H Barnett; Kain A Kyle; Robert Zivadinov; Kris C Ma; Sam T Milliken; Ian J Sutton; David D F Ma Journal: J Neurol Neurosurg Psychiatry Date: 2018-12-11 Impact factor: 10.154
Authors: Marcelo C Pasquini; Julio Voltarelli; Harold L Atkins; Nelson Hamerschlak; Xiaobo Zhong; Kwang Woo Ahn; Keith M Sullivan; George Carrum; Jeffrey Andrey; Christopher N Bredeson; Mitchell Cairo; Robert Peter Gale; Theresa Hahn; Jan Storek; Mary M Horowitz; Peter A McSweeney; Linda M Griffith; Paolo A Muraro; Steven Z Pavletic; Richard A Nash Journal: Biol Blood Marrow Transplant Date: 2012-06-13 Impact factor: 5.742
Authors: G L Mancardi; M P Sormani; M Di Gioia; L Vuolo; F Gualandi; M P Amato; E Capello; D Currò; A Uccelli; A Bertolotto; C Gasperini; A Lugaresi; E Merelli; G Meucci; L Motti; M R Tola; E Scarpini; A M Repice; L Massacesi; R Saccardi Journal: Mult Scler Date: 2011-11-29 Impact factor: 6.312
Authors: Dominique Farge; Myriam Labopin; Alan Tyndall; Athanasios Fassas; Gian Luigi Mancardi; Jaap Van Laar; Jian Ouyang; Tomas Kozak; John Moore; Ina Kötter; Virginie Chesnel; Alberto Marmont; Alois Gratwohl; Riccardo Saccardi Journal: Haematologica Date: 2009-09-22 Impact factor: 9.941
Authors: Otylia Kowal-Bielecka; Jaap Fransen; Jerome Avouac; Mike Becker; Agnieszka Kulak; Yannick Allanore; Oliver Distler; Philip Clements; Maurizio Cutolo; Laszlo Czirjak; Nemanja Damjanov; Francesco Del Galdo; Christopher P Denton; Jörg H W Distler; Ivan Foeldvari; Kim Figelstone; Marc Frerix; Daniel E Furst; Serena Guiducci; Nicolas Hunzelmann; Dinesh Khanna; Marco Matucci-Cerinic; Ariane L Herrick; Frank van den Hoogen; Jacob M van Laar; Gabriela Riemekasten; Richard Silver; Vanessa Smith; Alberto Sulli; Ingo Tarner; Alan Tyndall; Joep Welling; Frederic Wigley; Gabriele Valentini; Ulrich A Walker; Francesco Zulian; Ulf Müller-Ladner Journal: Ann Rheum Dis Date: 2016-11-09 Impact factor: 19.103
Authors: Harold L Atkins; Marjorie Bowman; David Allan; Grizel Anstee; Douglas L Arnold; Amit Bar-Or; Isabelle Bence-Bruckler; Paul Birch; Christopher Bredeson; Jacqueline Chen; Dean Fergusson; Mike Halpenny; Linda Hamelin; Lothar Huebsch; Brian Hutton; Pierre Laneuville; Yves Lapierre; Hyunwoo Lee; Lisa Martin; Sheryl McDiarmid; Paul O'Connor; Timothy Ramsay; Mitchell Sabloff; Lisa Walker; Mark S Freedman Journal: Lancet Date: 2016-06-09 Impact factor: 79.321
Authors: Paolo A Muraro; Roland Martin; Giovanni Luigi Mancardi; Richard Nicholas; Maria Pia Sormani; Riccardo Saccardi Journal: Nat Rev Neurol Date: 2017-06-16 Impact factor: 42.937
Authors: Jacob M van Laar; Dominique Farge; Jacob K Sont; Kamran Naraghi; Zora Marjanovic; Jérôme Larghero; Annemie J Schuerwegh; Erik W A Marijt; Madelon C Vonk; Anton V Schattenberg; Marco Matucci-Cerinic; Alexandre E Voskuyl; Arjan A van de Loosdrecht; Thomas Daikeler; Ina Kötter; Marc Schmalzing; Thierry Martin; Bruno Lioure; Stefan M Weiner; Alexander Kreuter; Christophe Deligny; Jean-Marc Durand; Paul Emery; Klaus P Machold; Francoise Sarrot-Reynauld; Klaus Warnatz; Daniel F P Adoue; Joël Constans; Hans-Peter Tony; Nicoletta Del Papa; Athanasios Fassas; Andrea Himsel; David Launay; Andrea Lo Monaco; Pierre Philippe; Isabelle Quéré; Éric Rich; Rene Westhovens; Bridget Griffiths; Riccardo Saccardi; Frank H van den Hoogen; Willem E Fibbe; Gérard Socié; Alois Gratwohl; Alan Tyndall Journal: JAMA Date: 2014-06-25 Impact factor: 56.272
Authors: Kevin Hendrawan; Melissa L M Khoo; Malini Visweswaran; Jennifer C Massey; Barbara Withers; Ian Sutton; David D F Ma; John J Moore Journal: Front Immunol Date: 2022-01-19 Impact factor: 7.561
Authors: Ahmed Lotfy; Aya Elgamal; Anna Burdzinska; Ayman A Swelum; Reham Soliman; Ayman A Hassan; Gamal Shiha Journal: Stem Cell Res Ther Date: 2021-07-07 Impact factor: 6.832