Systemic Sclerosis as an Indication for Autologous Hematopoietic Cell Transplantation: Position Statement from the American Society for Blood and Marrow Transplantation.
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.
Type
Journal articlePermalink
https://hdl.handle.net/10161/17670Published Version (Please cite this version)
10.1016/j.bbmt.2018.06.025Publication Info
Sullivan, Keith M; Majhail, Navneet S; Bredeson, Christopher; Carpenter, Paul A; Chatterjee,
Soumya; Crofford, Leslie J; ... St Clair, E William (2018). Systemic Sclerosis as an Indication for Autologous Hematopoietic Cell Transplantation:
Position Statement from the American Society for Blood and Marrow Transplantation.
Biology of blood and marrow transplantation : journal of the American Society for
Blood and Marrow Transplantation. 10.1016/j.bbmt.2018.06.025. Retrieved from https://hdl.handle.net/10161/17670.This is constructed from limited available data and may be imprecise. To cite this
article, please review & use the official citation provided by the journal.
Collections
More Info
Show full item recordScholars@Duke
Stefanie Sarantopoulos
Professor of Medicine
Keith Michael Sullivan
James B. Wyngaarden Distinguished Professor Emeritus of Medicine
Research areas
Late effects of cancer treatment and stem cell transplantation
Chronic graft-versus-host disease
Transplantation for sickle cell and autoimmune diseases
Knowledge engineering
OverviewEarly on, Dr. Sullivan and the team at Fred Hutchinson Cancer Research Center
developed a systematic investigative approach for the diagnosis and treatment of chronic
graft-versus-host
Alphabetical list of authors with Scholars@Duke profiles.

Articles written by Duke faculty are made available through the campus open access policy. For more information see: Duke Open Access Policy
Rights for Collection: Scholarly Articles
Works are deposited here by their authors, and represent their research and opinions, not that of Duke University. Some materials and descriptions may include offensive content. More info