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EBSCO

https://doi.org/10.14300/mnnc.2022.17038

Long-term follow-up analysis after autologous hematopoietic stem cell transplantation for children with multiple sclerosis

[Original research] [Pediatrics]
Kirill Kirgizov; Gleb Valeryevich  Demyanov; Mikhail Valentinovich  Kiselevsky; Semen Valeryevich  Demyanov; Elena Skopobogatova; Anton Valeryevich  Ershov;

Autologous hematopoietic stem cell transplantation (aHSCT) is effective and safe treatment for multiple sclerosis in children. The purpose of this study was to improve treatment outcomes by analyzing long-term follow-up after transplantation and evaluating late effects, as well as studying the immune profile in patients after aHSCT. Sixteen patients were included in the study. All patients included in the study were under 18 years of age. All patients had severe refractory multiple sclerosis. Prior to the initiation of aHSCT, all patients received therapy with 1st and 2nd line drugs with no positive effect. Mean improvement on the Expanded Disability Status Scale (EDSS) was 3.1±1.3 during the first 60 days after aHSCT. Long-term remission after stem cell transplantation was achieved in the patients included in the study, with median follow-up period of 48 months (8–93 months). Restoration of neurological functions was confirmed by immunological data: increasing of immune regulation index (CD4+/CD8+) from 1.36±0.84 to 2.2±0.53 as well as CD4+CD25+FoxP3+ T-lymphocytes from 1.38±0.86 % to 3.56±1.53 % (in comparison with the baseline). Late effects were moderate in all patients.
Thus, autologous hematopoietic stem cell transplantation proved to be effective and safe treatment for pediatric patients with severe refractory multiple sclerosis, and the effectiveness of aHSCT was proved by immunological data.

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Keywords: autologous hematopoietic stem cell transplantation, multiple sclerosis, immune reconstitution, immunosuppression, children