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Systemic Sclerosis as an Indication for Autologous Hematopoietic Cell Transplantation: Position Statement from the American Society for Blood and Marrow Transplantation. Biol Blood Marrow Transplant 2018 10;24(10):1961-1964

Date

06/29/2018

Pubmed ID

29953945

Pubmed Central ID

PMC6239926

DOI

10.1016/j.bbmt.2018.06.025

Scopus ID

2-s2.0-85054357023   19 Citations

Abstract

Systemic sclerosis is a progressive inflammatory disease that is frequently fatal and has limited treatment options. High-dose chemotherapy with autologous hematopoietic cell transplantation (AHCT) has been evaluated as treatment for this disease in observational studies, multicenter randomized controlled clinical trials, and meta-analyses. On behalf of the American Society for Blood and Marrow Transplantation (ASBMT), a panel of experts in transplantation and rheumatology was convened to review available evidence and make a recommendation on AHCT as an indication for systemic sclerosis. Three randomized trials have compared the efficacy of AHCT with cyclophosphamide only, and all demonstrated benefit for the AHCT arm for their primary endpoint (improvement in the American Scleroderma Stem Cell versus Immune Suppression Trial, event-free survival in Autologous Stem Cell Transplantation International Scleroderma trial, and change in global rank composite score in Scleroderma: Cyclophosphamide or Transplantation trial). AHCT recipients also had better overall survival and a lower rate of disease progression. These findings have been confirmed in subsequent meta-analyses. Based on this high-quality evidence, the ASBMT recommends systemic sclerosis should be considered as a "standard of care" indication for AHCT. Close collaboration between rheumatologists and transplant clinicians is critical for optimizing patient selection and patient outcomes. Transplant centers in the United States are strongly encouraged to report patient and outcomes data to the Center for International Blood and Marrow Transplant Research on their patients receiving AHCT for this indication.

Author List

Sullivan KM, Majhail NS, Bredeson C, Carpenter PA, Chatterjee S, Crofford LJ, Georges GE, Nash RA, Pasquini MC, Sarantopoulos S, Storek J, Savani B, St Clair EW

Author

Marcelo C. Pasquini MD, MS Professor in the Medicine department at Medical College of Wisconsin




MESH terms used to index this publication - Major topics in bold

Autografts
Bone Marrow Transplantation
Cyclophosphamide
Hematopoietic Stem Cell Transplantation
Humans
Randomized Controlled Trials as Topic
Scleroderma, Systemic
Societies, Medical
United States
jenkins-FCD Prod-482 91ad8a360b6da540234915ea01ff80e38bfdb40a