Kirgizov, K., Burman, J., Snowden, J.A. orcid.org/0000-0001-6819-3476 et al. (1 more author) (2024) Autologous hematopoietic stem cell transplantation for pediatric autoimmune neurologic disorders. In: Inglese, M. and Mancardi, G.L., (eds.) Hematopoietic Stem Cell Transplantation for Neurologic Diseases. Handbook of Clinical Neurology, 202 . Elsevier , pp. 249-258. ISBN 9780323902427
Abstract
Autologous hematopoietic stem cell transplantation (aHSCT) may be effective in carefully selected pediatric patients with multiple sclerosis (MS), neuromyelitis optica (NMO), and chronic inflammatory demyelinating polyneuropathy (CIDP). aHSCT for pediatric MS (same as for adults) is performed to eradicate inflammatory autoreactive cells with lympho-ablative regimens and restore immune tolerance. Its therapeutic effect in MS relies on various mechanisms: (1) the immunosuppressive conditioning regimen prior to aHSCT was able to eradicate the autoreactive cells and (2) the regeneration/renewal of the immune system to reset the aberrant immune response against self-antigens. The aHSCT procedure includes the following different steps, as described in this chapter: patient selection through careful pretransplant screening, “wash-out” period from previous treatments, mobilization of hematopoietic stem cells (HSC), conditioning regimen, HSC infusion, and posttransplant monitoring for early and late complications. Moreover, specific aspects of pediatric population undergoing aHSCT are described.
According to the available evidence, aHSCT appears to be safe in pediatric MS, obtaining disease control for a prolonged time after the procedure. A reasonable approach in this setting includes the application of less toxic treatments while reserving aHSCT procedure for patients with severe/refractory forms of the disease. The EBMT considers MS, NMO, and CIDP in pediatric patients within the category of the clinical option (CO), where candidates for aHSCT can be selected on the basis of careful consideration of individual case history in the multidisciplinary setting.
Metadata
Item Type: | Book Section |
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Authors/Creators: |
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Editors: |
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Copyright, Publisher and Additional Information: | © 2024 Elsevier B.V. |
Keywords: | Autoimmune disorders; Children; Hematopoietic stem cell transplantation; Multiple sclerosis; Humans; Hematopoietic Stem Cell Transplantation; Child; Transplantation, Autologous; Autoimmune Diseases of the Nervous System; Neuromyelitis Optica; Pediatrics |
Dates: |
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Institution: | The University of Sheffield |
Academic Units: | The University of Sheffield > Faculty of Medicine, Dentistry and Health (Sheffield) > School of Medicine and Population Health |
Depositing User: | Symplectic Sheffield |
Date Deposited: | 19 Sep 2024 16:18 |
Last Modified: | 19 Sep 2024 16:18 |
Status: | Published |
Publisher: | Elsevier |
Series Name: | Handbook of Clinical Neurology |
Refereed: | Yes |
Identification Number: | 10.1016/b978-0-323-90242-7.00004-3 |
Related URLs: | |
Open Archives Initiative ID (OAI ID): | oai:eprints.whiterose.ac.uk:217247 |