Autologous haematopoietic stem cell transplantation (AHSCT) in autoimmune disease adult patients in France: analysis of the long-term outcome from the French Society for Bone Marrow Transplantation and Cellular Therapy (SFGM-TC)

摘要

IntroductionThe use of autologous haematopoietic stem cell transplantation (AHSCT) in autoimmune disease (AD) patients has increased progressively worldwide. We retrospectively analysed the long-term outcome of AHSCT for AD reported to the French Society for Bone Marrow Transplantation and Cellular Therapy (SFGM-TC).MethodAll French AD patients (18years at transplant) with a first AHSCT between 1997 and 2013 were included. Primary data were derived from the European Society for Blood and Marrow Transplantation (EBMT) registry, and additional data were obtained through a specific questionnaire designed for the study. Primary end-point was overall survival (OS). Secondary end points were progression-free survival (PFS) and non-relapse mortality (NRM).ResultsNinety-four AD patients were included, of whom 71% suffered from rheumatologic diseases (n=67, including 56 systemic sclerosis (SSc)), 16% from neurological disease (n=15, including 14 multiple sclerosis (MS)) and 13% from various other AD (n=12). After a median (interquartile range, IQR) follow-up of 83months (38-130), OS at 5 and 10years were 77% (95% CI 68.5-86.2) and 64% (95% CI 51.7-76.3), and for PFS 51% (95% CI 40.4-61.6) and 44% (95% CI 32.8-55.3), respectively. Overall, NRM was 8.7% (95% CI 4.0-15.5) at day 100, 9.8% (95% CI 4.8-16.9) at 5years and 13.6% (95% CI 6.9-22.5) at 10years.ConclusionsThis first SFGM-TC retrospective report shows long-term benefit of AHSCT in AD patients with acceptable toxicity.