TY - JOUR
T1 - Myeloablation followed by autologous stem cell transplantation normalises systemic sclerosis molecular signatures
AU - Assassi, Shervin
AU - Wang, Xuan
AU - Chen, Guocai
AU - Goldmuntz, Ellen
AU - Keyes-Elstein, Lynette
AU - Ying, Jun
AU - Wallace, Paul K.
AU - Turner, Jacob
AU - Zheng, W. Jim
AU - Pascual, Virginia
AU - Varga, John
AU - Hinchcliff, Monique E.
AU - Bellocchi, Chiara
AU - McSweeney, Peter
AU - Furst, Daniel E.
AU - Nash, Richard A.
AU - Crofford, Leslie J.
AU - Welch, Beverly
AU - Pinckney, Ashley
AU - Mayes, Maureen D.
AU - Sullivan, Keith M.
N1 - Funding Information:
Funding The scoT study was supported by awards from the niaiD, niH to Duke University, the study contract holder (n01-ai05419 and HHsn 272201100025c). The study was also supported by grants from Karen Brown scleroderma Foundation, niH niaMs (P30-ar061271), niH r01ar073284, niH Ul1-Tr000371 and Department of Defense (W81XWH-16-1-0296).
Publisher Copyright:
© © Author(s) (or their employer(s)) 2019. No commercial re-use. See rights and permissions. Published by BMJ.
PY - 2019/10/1
Y1 - 2019/10/1
N2 - In the SCOT trial, the effectiveness of haematopoietic stem cell transplantation (HSCT) was compared to cyclophosphamide (CYC) treatment in systemic sclerosis (SSc) patients. The objective was to observe global molecular changes in whole blood transcripts and serum protein levels following treatments. Pre and post-treatment (at 8 and 26 months) analyses revealed that at baseline, interferon (IFN) and neutrophil modules were heightened, while the cytotoxic/NK module was lowered in SSc patients compared to unaffected controls. Post-treatment, a significant decrease in IFN and neutrophil modules and an increase in the cytotoxic/NK module were observed in the HSCT group, with no notable change in the CYC group. This molecular 'correction' associated with HSCT correlated with clinical improvements in pulmonary and skin scores, implying that HSCT could significantly amend disease-related molecular signatures in systemic sclerosis, unlike conventional CYC treatment.
AB - In the SCOT trial, the effectiveness of haematopoietic stem cell transplantation (HSCT) was compared to cyclophosphamide (CYC) treatment in systemic sclerosis (SSc) patients. The objective was to observe global molecular changes in whole blood transcripts and serum protein levels following treatments. Pre and post-treatment (at 8 and 26 months) analyses revealed that at baseline, interferon (IFN) and neutrophil modules were heightened, while the cytotoxic/NK module was lowered in SSc patients compared to unaffected controls. Post-treatment, a significant decrease in IFN and neutrophil modules and an increase in the cytotoxic/NK module were observed in the HSCT group, with no notable change in the CYC group. This molecular 'correction' associated with HSCT correlated with clinical improvements in pulmonary and skin scores, implying that HSCT could significantly amend disease-related molecular signatures in systemic sclerosis, unlike conventional CYC treatment.
KW - cyclophosphamide
KW - hematopoietic stem cell transplantation
KW - interferon signature
KW - neutrophil
KW - systemic sclerosis
UR - http://www.scopus.com/inward/record.url?scp=85070614837&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85070614837&partnerID=8YFLogxK
U2 - 10.1136/annrheumdis-2019-215770
DO - 10.1136/annrheumdis-2019-215770
M3 - Article
C2 - 31391177
AN - SCOPUS:85070614837
SN - 0003-4967
VL - 78
SP - 1371
EP - 1378
JO - Annals of the Rheumatic Diseases
JF - Annals of the Rheumatic Diseases
IS - 10
ER -