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Hematopoietic Stem Cell Transplantation for Systemic Sclerosis: If You Are Confused, Remember: 'It Is a Matter of the Heart'

Authors :
Richard K. Burt
James W. Schroeder
Eric Ruderman
Sanjiv J. Shah
Mihai Gheorghiade
Source :
The Journal of Rheumatology. 39:206-209
Publication Year :
2012
Publisher :
The Journal of Rheumatology, 2012.

Abstract

For the past 20 years, the standard of care for systemic sclerosis (SSc) with lung involvement has been oral or intravenous (IV) cyclophosphamide (CYC). To date, there have been 2 randomized trials and 2 metaanalyses of prospective studies using oral or IV CYC in SSc-related interstitial pneumonitis (interstitial lung disease; ILD) and none have reported improvement in lung function1,2,3,4,5. The Scleroderma Lung Research Study Group’s study in The New England Journal of Medicine reported that oral CYC daily for 1 year is of “modest benefit” compared to placebo1. However, the term “modest benefit” does not mean that lung function improved. In fact, the forced vital capacity (FVC) and DLCO declined in both placebo and CYC-treated patients1. “Modest benefit” means that, after 1 year, the rate of decline in FVC was less in those receiving CYC compared to placebo, but the lung function still worsened on CYC1. Further, at 2-year followup there was no difference in loss of lung function between oral daily CYC and placebo2. Due to a lack of effective standard therapy, SSc — a lethal disease that involves vital organs — needs a new and effective approach. An approach that began in patients about 14 years ago, hematopoietic stem cell transplantation (HSCT), has been demonstrated to improve both skin and lung function as well as quality of life in patients with SSc6,7,8. Transplantation has been performed safely in some studies8, but results have been complicated by high treatment-related mortality in others9. Mortality of HSCT for SSc can be markedly reduced, however, if the reasons for mortality are properly recognized. The safety of HSCT is determined by 3 variables: (1) … Address correspondence to Dr. Burt; E-mail: rburt{at}northwestern.edu

Details

ISSN :
14992752 and 0315162X
Volume :
39
Database :
OpenAIRE
Journal :
The Journal of Rheumatology
Accession number :
edsair.doi.dedup.....01907cb3975905c32c4214e3a8651580
Full Text :
https://doi.org/10.3899/jrheum.111302