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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.

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Date
2018-06-25
Authors
Sullivan, Keith M
Majhail, Navneet S
Bredeson, Christopher
Carpenter, Paul A
Chatterjee, Soumya
Crofford, Leslie J
Georges, George E
Nash, Richard A
Pasquini, Marcelo C
Sarantopoulos, Stefanie
Storek, Jan
Savani, Bipin
St Clair, E William
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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 article
Subject
Autologous hematopoietic cell transplantation
Coverage
Indication
Scleroderma
Systemic sclerosis
Permalink
https://hdl.handle.net/10161/17670
Published Version (Please cite this version)
10.1016/j.bbmt.2018.06.025
Publication 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.
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Scholars@Duke

Sarantopoulos

Stefanie Sarantopoulos

Professor of Medicine
Sullivan

Keith Michael Sullivan

James B. Wyngaarden Distinguished Professor 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.
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