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Autologous hematopoietic stem cell transplantation in pediatric multiple sclerosis: Long-time follow-up
K.I. Kirgizov, E.Y. Volkova, E.V. Skorobogatova, S.V. Piliya, A.A. Bologov, R.C. Bembeeva, N.N. Zavadenko
Department of Bone Marrow Transplantation, The Russian Children's Research Hospital, Moscow, Russia
DOI: http://dx.doi.org/10.1016/S1090-3798(15)30072-6
Abstract
Objective
Autologous hematopoietic stem cell transplantation (Auto-HSCT) is the method of choice for patients with refractory multiple sclerosis (MS). We have studied the effectiveness and long-time results of this method in pediatric patients.
Methods
10 patients were included into the study: 8 females, 2 males. Median age was 16.7±1.7. Median MS duration prior to Auto-HSCT was 15.5±4.1. Age of MS onset: 4–14 years of age (12.3±1.7). Initial median score on the Expanded Disability Status Scale (EDSS) was 6.16±0.2. All patients had severe refractory MS treated with corticosteroids, interferons, plasmapheresis and mitoxantron with negative results. All patients had inflammation signs. Procedures included: stem cells mobilization with Cyclophosphomide, conditioning with Cyclophosphomide 200 mg/kg and ATGAM 160 mg/kg. Median follow-up was 21.1±0.9 (4–60 months).
Results
All patients demonstrated positive results with EDSS improvement: from 2.5±0.21 in the first 60 days to 0.2±0.02 after 60 days. Maximal individual EDSS improvement was 5.5. EDSS at 2 patients improved to 1. Five patients had severe refractory secondary-progressive MS with the long duration of ineffective treatment. These patients showed the minor improvement and required the additional immunomodulation after Auto-HSCT. 2 patients relapsed (on clinical and MRI data). No severe complications were registered.
Conclusion
Auto-HSCT is an effective approach to autoimmune inflammation reduction and treatment of severe refractory pediatric MS. In-time Auto-HSCT can significantly minimize the disability level and improve the outcome of MS. Most patients remained in remission during the long period.
K.I. Kirgizov, E.Y. Volkova, E.V. Skorobogatova, S.V. Piliya, A.A. Bologov, R.C. Bembeeva, N.N. Zavadenko
Department of Bone Marrow Transplantation, The Russian Children's Research Hospital, Moscow, Russia
DOI: http://dx.doi.org/10.1016/S1090-3798(15)30072-6
Abstract
Objective
Autologous hematopoietic stem cell transplantation (Auto-HSCT) is the method of choice for patients with refractory multiple sclerosis (MS). We have studied the effectiveness and long-time results of this method in pediatric patients.
Methods
10 patients were included into the study: 8 females, 2 males. Median age was 16.7±1.7. Median MS duration prior to Auto-HSCT was 15.5±4.1. Age of MS onset: 4–14 years of age (12.3±1.7). Initial median score on the Expanded Disability Status Scale (EDSS) was 6.16±0.2. All patients had severe refractory MS treated with corticosteroids, interferons, plasmapheresis and mitoxantron with negative results. All patients had inflammation signs. Procedures included: stem cells mobilization with Cyclophosphomide, conditioning with Cyclophosphomide 200 mg/kg and ATGAM 160 mg/kg. Median follow-up was 21.1±0.9 (4–60 months).
Results
All patients demonstrated positive results with EDSS improvement: from 2.5±0.21 in the first 60 days to 0.2±0.02 after 60 days. Maximal individual EDSS improvement was 5.5. EDSS at 2 patients improved to 1. Five patients had severe refractory secondary-progressive MS with the long duration of ineffective treatment. These patients showed the minor improvement and required the additional immunomodulation after Auto-HSCT. 2 patients relapsed (on clinical and MRI data). No severe complications were registered.
Conclusion
Auto-HSCT is an effective approach to autoimmune inflammation reduction and treatment of severe refractory pediatric MS. In-time Auto-HSCT can significantly minimize the disability level and improve the outcome of MS. Most patients remained in remission during the long period.