Autologous hematopoietic stem cell transplantation with reduced-intensity conditioning in multiple sclerosis

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• 作者：Jury L. Shevchenko ; Alexey N. Kuznetsov ; Tatyana I. Ionova ; Vladimir Y. Melnichenko ; Denis A. Fedorenko ; Andrei V. Kartashov ; Kira A. Kurbatova ; Gary I. Gorodokin ; Andrei A. Novik
• 刊名：Experimental Hematology
• 出版年：2012
• 出版时间：November, 2012
• 年：2012
• 卷：40
• 期：11
• 页码：892-898
• 全文大小：313 K

文摘

High-dose immunosuppressive therapy with autologous hematopoietic stem cell transplantation (AHSCT) is a new and promising approach to multiple sclerosis (MS) treatment. In this article, we present the results of a prospective phase II open-label single-center study with the analysis of the safety and efficacy of high-dose immunosuppressive therapy+AHSCT with reduced-intensity conditioning regimen in 95 patients with different types of MS. The patients underwent early, conventional, and salvage/late transplantation. Efficacy was evaluated based on clinical and quality of life outcomes. No transplantation-related deaths were observed. The mobilization and transplantation procedures were well tolerated. All the patients, except one, responded to the treatment. At long-term follow-up (mean 46 months), the overall clinical response in terms of disease improvement or stabilization was 80 % . The estimated progression-free survival at 5?years was 92 % in the group after early AHSCT vs 73 % in the group after conventional/salvage AHSCT. Statistically significant difference between the survival probabilities of two groups was determined (p = 0.01). No active, new, or enlarging lesions in magnetic resonance imaging were registered in patients without disease progression. All patients who did not have disease progression were off therapy throughout the post-transplantation period. AHSCT was?accompanied by a significant improvement in patient¡¯s quality of life with statistically significant changes in the majority of quality of life parameters (p < 0.05).The results of our study support the feasibility of AHSCT with reduced-intensity conditioning in MS patients. Multicenter cooperative studies are needed for better assessment of treatment results and optimization of the treatment protocol of AHSCT with reduced-intensity conditioning regimens in MS.