TY - JOUR
T1 - Hematopoietic stem cell transplantation for systemic sclerosis
T2 - History and current status
AU - Burt, Richard K
AU - Milanettib, Francesca
PY - 2011/11/1
Y1 - 2011/11/1
N2 - 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.
AB - 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.
KW - Hematopoietic stem cell
KW - Scleroderma
KW - Systemic sclerosis
KW - Transplantation
UR - http://www.scopus.com/inward/record.url?scp=82955187639&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=82955187639&partnerID=8YFLogxK
U2 - 10.1097/BOR.0b013e32834aa45f
DO - 10.1097/BOR.0b013e32834aa45f
M3 - Review article
C2 - 21857226
AN - SCOPUS:82955187639
SN - 1040-8711
VL - 23
SP - 519
EP - 529
JO - Current Opinion in Rheumatology
JF - Current Opinion in Rheumatology
IS - 6
ER -