High-dose immunosuppressive therapy with autologous hematopoietic stem cell transplantation as a treatment option in multiple sclerosis.
High-dose immunosuppressive therapy (HDIT) with autologous hematopoietic stem cell transplantation (auto-HSCT) is a new and promising approach to the treatment of multiple sclerosis (MS) patients because currently there are no effective treatment methods for this disease. In this article, we present results of a prospective clinical study of efficacy of HDIT + auto-HSCT in MS patients. The following treatment strategies were employed in the study: "early," "conventional," and "salvage/late" transplantation. Fifty patients with various types of MS were included in this study. No toxic deaths were reported among 50 MS patients; transplantation procedure was well-tolerated by the patients. The efficacy analysis was performed in 45 patients. Twenty-eight patients achieved an objective improvement of neurological symptoms, defined as at least 0.5-point decrease in the Expanded Disability Status Scale (EDSS) score as compared to the baseline and confirmed during 6 months, and 17 patients had disease stabilization (steady EDSS level as compared to the baseline and confirmed during 6 months). The progression-free survival at 6 years after HDIT + auto-HSCT was 72%. Magnetic resonance imaging data were available in 37 patients before transplantation showing disease activity in 43.3%. No active, new, or enlarging lesions were registered in patients without disease progression. In conclusion, HDIT + auto-HSCT suggests positive results in management of patients with different types of MS. Identification of treatment strategies based on the level of disability, namely "early," "conventional," and "salvage/late" transplantation, appears to be feasible to improve treatment outcomes.
Shevchenko
Yury L
YL
Pirogov National Medical Surgical Center, the Department of Haematology and Cellular Therapy, Pirogov National Medical Surgical Center, Moscow, Russia.
Novik
Andrei A
AA
Kuznetsov
Aleksey N
AN
Afanasiev
Boris V
BV
Lisukov
Igor A
IA
Kozlov
Vladimir A
VA
Rykavicin
Oleg A
OA
Ionova
Tatyana I
TI
Melnichenko
Vladimir Y
VY
Fedorenko
Denis A
DA
Kulagin
Alexander D
AD
Shamanski
Sergei V
SV
Ivanov
Roman A
RA
Gorodokin
Gary
G
D000293:Adolescent; D000328:Adult; D003131:Combined Modality Therapy; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D009103:Multiple Sclerosis / Q000628:therapy*; D012426:Russia; D014182:Transplantation, Autologous; D016896:Treatment Outcome
D007166:Immunosuppressive Agents