Richard K Burt1, Francesca Milanetti. 1. Division of Immunotherapy, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA. rburt@northwestern.edu
Abstract
PURPOSE OF REVIEW: Systemic sclerosis (SSc) remains one of the last severe autoimmune disease with a poor prognosis and modest response to immunosuppressive therapy. Mortality in severe diffuse disease with internal organ involvement is elevated. Autologous hematopoietic transplantation (HSCT) has emerged in the last decade as a promising disease-modifying treatment. RECENT FINDINGS: In phase I/II trials, HSCT has demonstrated to induce impressive reversal of skin fibrosis, neoangiogenesis, improved functionality and quality of life, and stabilization of internal organ function. Treatment-related mortality was reduced over time by better pretransplant evaluation and by treating patients earlier in disease. SUMMARY: Two out of three randomized trials of autologous HSCT for SSc have been concluded: the nonmyeloablative American Systemic Sclerosis Immune Suppression versus Transplant, and Autologous Stem cell Transplantation International Scleroderma. The myeloablative Scleroderma Cyclophosphamide versus Transplant instead is still recruiting patients. The soon expected results from these trials should clarify the role of autologous HSCT in the challenging management of severe SSc.
PURPOSE OF REVIEW: Systemic sclerosis (SSc) remains one of the last severe autoimmune disease with a poor prognosis and modest response to immunosuppressive therapy. Mortality in severe diffuse disease with internal organ involvement is elevated. Autologous hematopoietic transplantation (HSCT) has emerged in the last decade as a promising disease-modifying treatment. RECENT FINDINGS: In phase I/II trials, HSCT has demonstrated to induce impressive reversal of skin fibrosis, neoangiogenesis, improved functionality and quality of life, and stabilization of internal organ function. Treatment-related mortality was reduced over time by better pretransplant evaluation and by treating patients earlier in disease. SUMMARY: Two out of three randomized trials of autologous HSCT for SSc have been concluded: the nonmyeloablative American Systemic Sclerosis Immune Suppression versus Transplant, and Autologous Stem cell Transplantation International Scleroderma. The myeloablative SclerodermaCyclophosphamide versus Transplant instead is still recruiting patients. The soon expected results from these trials should clarify the role of autologous HSCT in the challenging management of severe SSc.
Authors: Katarzyna Pawlak-Buś; Wiktor Schmidt; Michał Olejarz; Anna Czyż; Mieczysław Komarnicki; Piotr Leszczyński Journal: Reumatologia Date: 2019-02-28
Authors: Julia Spierings; Anna van Rhenen; Paco Mw Welsing; Anne Ca Marijnissen; Ellen De Langhe; Nicoletta Del Papa; Daan Dierickx; Karina R Gheorghe; Joerg Henes; Roger Hesselstrand; Tessa Kerre; Per Ljungman; Arjan A van de Loosdrecht; Erik Waf Marijt; Miro Mayer; Marc Schmalzing; Roland Schroers; Vanessa Smith; Reinhard E Voll; Madelon C Vonk; Alexandre E Voskuyl; Jeska K de Vries-Bouwstra; Ulrich A Walker; Dirk M Wuttge; Jacob M van Laar Journal: BMJ Open Date: 2021-03-18 Impact factor: 2.692