Final results from the Autologous Stem Cell Transplantation
International Scleroderma (ASTIS) trial published in the Journal of the American
Medical Association (JAMA) on June 25th 2014 evidence that autologous
haematopoietic stem cell transplantation (HSCT) confers better long term
survival than 12 monthly intravenous pulses of cyclophosphamide treatment for
diffuse cutaneous sclerosis.
From March 2001 to October 2009, 156 patients from 10 countries with
early diffuse cutaneous systemic sclerosis were recruited and followed up until
the end of October 2013. After registration, patients were randomly assigned to
receive HSCT or monthly intravenous pulses of cyclophosphamide with the aim to
compare survival rates of both treatments. The primary end point of ASTIS was
event-free survival, defined as time from randomization until the occurrence of
death or persistent major organ failure.
ASTIS trial is an international, multi-centre, investigator-based,
open label, phase III trial that involved 29 centers. It is a unique
groundbreaking academic collaborative project coordinated by two leading
entities in the fields of the study, the European Society for Blood and Marrow
Transplantation (EBMT) and the European League Against Rheumatism
(EULAR).
Even though HSCT therapy shows more treatment-related mortality in
early stages of the follow up, long term event-free and overall survival rates
are higher in the HSCT treatment group than in the control group. In a post-hoc
analysis, the difference was greater in non-smoking patients. “These results are
very encouraging and shed light on new research on the type of patient that will
benefit from immunosuppressive therapy” said Professor Jaap van Laar (UMC
Utrecht) and Professor Dominique Farge Bancel (Assistance Publique Hôpitaux de Paris) with Alan Tyndall (Basel University) and other
co-authors.
Systemic sclerosis is a rare heterogeneous autoimmune connective
disease tissue disease characterized by vasculopathy, autoantibody formation,
low-grade inflammation, and fibrosis in skin and internal organs. It affects
2-22 people per one million every year and the diffuse cutaneous form of the
condition – which is a more severe form of the disease - makes up to 30% of the
total cases. This severe autoimmune disease can lead to premature death by lung
fibrosis and pulmonary hypertension with no curative treatment yet
discovered.
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