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Stem cell therapies for systemic sclerosis
Author(s) -
Cipriani Paola,
Ruscitti Piero,
Giacomelli Roberto
Publication year - 2015
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.13201
Subject(s) - medicine , stem cell , transplantation , immune system , clinical trial , multiple sclerosis , immunology , hematopoietic stem cell transplantation , cell therapy , oncology , biology , genetics
Summary The presence of autoimmune diseases, including Systemic Sclerosis ( SS c), suggest failure of the normal immune regulatory processes leading to activation and expansion of autoreactive effector immune cells. Recently, stem cell transplantation emerged as a novel rescue therapy for a variety of refractory autoimmune diseases. The therapeutic strategy involves the ablation of the aberrant self‐reactive immune cells by chemotherapy and the regeneration of a new self‐tolerant immune system formed by the transplanted stem cells. In the last few years, thousands of patients worldwide have received haematopoietic stem cell transplantation ( HSCT ), mostly autologous, as treatment for severe irreversible autoimmune diseases, with promising results. Here we review the results of published small series of SS c patients treated with allogeneic and autologous HSCT , as well as three randomized trials, exploring the safety and efficacy of autologous HSCT in SS c. Although the results are encouraging, nonetheless, the correct application of stem cell transplantation remains an area of active investigation. Results of larger randomized, double blind clinical trials, will certainly improve our knowledge of the appropriate clinical use of stem cell therapy in SS c patients.