New non-myeloablative conditioning regimen for multiple sclerosis patients undergoing autologous hematopoietic stem cell transplantation

• Published in: Multiple sclerosis 2008-09, Vol.14, p.S169-S169
• Main Authors: Novik, A A, Kuznetsov, AN, Melnichenko, VY, Fedorenko, DA, Ionova, TI, Kruglina, R V, Kartashov, A V, Gorodokin, G J
• Format: Article
• Language: English
• ISSN: 1352-4585

Background: During the last decade myeloablative autologous hematopoietic stem cell transplantation AHSCT has been used with increasing frequency as a therapeutic option for multiple sderosis(MS) patients. Taking into account the information about risk of transplant-related mortality and severe side effects of myeloablative conditioning regimens, the rationale to use non-myeloablative regimens is reasonable. Objective: The goal of our research was to study safety and efficacy of non-myeloablative AHSCT in MS patients. Methods: Thirty-six patients with MS (secondary progressive - 15 patients, primary progressive - 8, progressive-relapsing - 2, and relapsing-remitting - 11) were included in this study (mean age - 33.0, range: 17-51; male/female - 15/21). The non-myeloablative conditioning regimen included BCNU (300 mg/m2) alone or combined with melphalan (50 mg/m2) on day 1 before transplantation. In vivo T-cell depletion was performed through an infusion of 30 mg/kg of horse anti-thymocite globulin on day 1 and 2 after stem cell reinfusion. Median Expanded Disability Status Scale (EDSS) score at baseline was 4.0 (range 1.5 - 7.0). The mean follow-up duration was 6 months (range 3 - 12 months). Neurological and quality of life (QoL) evaluation was performed at baseline, at discharge, at 3, 6, 9, 12 months, and every 6 months following AHSCT; magnetic resonance imaging examinations - at baseline, at 6, 12 months, and at the end of follow-up. Results: Notably, no transplant-related deaths were observed. The transplantation procedure was well tolerated by the patients with no unpredictable severe adverse events. All of 28 patients included in the efficacy analysis experienced clinical stabilization (n=17) or improvement (n=11). Ten patients showed improvement by 0.5 points on EDSS and one patient by 1.0. All the patients included in QoL analysis (n=13) exhibited QoL response at 6 months post-transplant and improved QoL during the follow-up. Conclusions: In conclusion, the new non-myeloablative regimen of AHSCT appears to be safe and effective treatment for MS. The collection of further data on long-term follow-up is worthwhile to confirm the efficacy of the new treatment regimen.