干细胞研究与治疗杂志

抽象的

Non-myeloablative Autologous Haematopoietic Stem Cell Transplantation with Consolidation Therapy using Mitoxantrone as a Treatment Option in Multiple Sclerosis Patients

Novik AA, Kuznetsov AN, Melnichenko VY, Fedorenko DA, Ionova TI and Gorodokin GV

High-dose immunosuppressive therapy with autologous haematopoietic stem cell transplantation (AHSCT) is a new and promising approach to multiple sclerosis (MS) treatment. Recently, the rationale of evolution from myeloablative to non-myeloablative (NM) transplant regimens has been discussed. We aimed to study clinical outcomes in MS patients after NM -AHSCT with consolidation therapy using Mitoxantrone. 55 MS patients were included in this study (mean age - 29.1; male/female - 23/32). Median EDSS at base-line - 4.0 (1.5-8.0), the mean follow-up duration - 26 months (range 9.0 - 50). No transplant related deaths were reported. There were no deaths in the study throughout the follow-up period. The mobilization and transplantation procedures were well tolerated. All the patients responded to the treatment. At long-term follow-up in the group with relapsingremitting MS improvement was demonstrated in 15 patients (58%) and stabilization in 11 (42%). No relapses throughout the whole follow-up period were found. In the group with progressive MS improvement was achieved in 15 patients (82%) and stabilization in 3 (18%). No active new or enlarging lesions were found according to MRI data. Thus, NM-AHSCT with consolidation therapy by Mitoxantrone appears to be a safe and effective treatment for MS. The results of our study support the feasibility of NM-AHSCT with consolidation therapy in this patient population.