| Literature DB >> 28319075 |
J Burman1, K Kirgizov2, K Carlson3, M Badoglio4, G L Mancardi5, G De Luca6, B Casanova7, J Ouyang8, R Bembeeva9, J Haas10, P Bader11, J Snowden12, D Farge13,14.
Abstract
Autologous hematopoietic stem cell transplantation (aHSCT) is a promising therapy for multiple sclerosis (MS), which has mainly been used in adults. The purpose of this study was to investigate efficacy and adverse events of aHSCT in the treatment of children with MS using data from the European Society for Blood and Marrow Transplantation registry. Twenty-one patients with a median follow-up time of 2.8 years could be identified. PFS at 3 years was 100%, 16 patients improved in expanded disability status scale score and only 2 patients experienced a clinical relapse. The procedure was generally well tolerated and only two instances of severe transplant-related toxicity were recorded. There was no treatment-related mortality, although one patient needed intensive care. aHSCT may be a therapeutic option for children with disease that does not respond to standard care.Entities:
Mesh：
Year: 2017 PMID： 28319075 DOI： 10.1038/bmt.2017.40
Source DB: PubMed Journal: Bone Marrow Transplant ISSN： 0268-3369 Impact factor: 5.483