OP71 – 2706: Autologous hematopoietic stem cell transplantation in pediatric multiple sclerosis: Long-time follow-up

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.