Abstract

During the last decade high-dose immunosuppressive therapy with autologous haematopoietic stem cell transplantation (HDIT+ASCT) has been used with increasing frequency as a therapeutic option for MS patients. We aimed to study clinical outcomes in patients with different MS types after HDIT+ASCT. 156 MS patients (secondary progressive, 54; primary progressive, 28; progressive relapsing, 5; relapsing-remitting, 69) were included in this study (mean age, 33.0; male/female, 63/93). Median EDSS at base-line, 4.0. Median follow-up duration, 20.6 months (range 6–126). Neurological evaluation was performed at baseline, at discharge, at 3, 6, 9, 12 months, and every 6 months thereafter. Transplantation procedure was well tolerated by the patients with no transplant-related deaths. The efficacy analysis was performed in 101 patients at 6 months posttransplant: 48 patients (48%) achieved an objective improvement of neurological symptoms; 52 (51%) disease stabilisation; 1 patient (1%) progressed. At long-term follow-up (median, 27.5 months) clinical response was classified as improvement in 41 (62%); stabilization in 20 (30%); progression in 5 (8%) patients. No active, new or enlarging lesions were registered in patients without disease progression. HDIT+ASCT appears to be a safe and effective treatment for MS. Further studies should be done to establish the best timing for transplantation and to validate conditioning regimens.