Safety and efficacy of autologous hematopoietic stem cell transplantation for multiple sclerosis in Norway.
Mult Scler
; 26(14): 1889-1897, 2020 12.
Article
em En
| MEDLINE
| ID: mdl-31833798
ABSTRACT
BACKGROUND:
Hematopoietic stem cell treatment (HSCT) is a promising treatment option for multiple sclerosis (MS), but detailed safety and efficacy measures are still scarce.OBJECTIVE:
To evaluate the efficacy and safety of HSCT in MS.METHODS:
Retrospective single-center observational study of all MS patients that underwent HSCT in Norway during January 2015 to January 2018. The primary outcome was no evidence of disease activity (NEDA-3) status.RESULTS:
A total of 30 patients with a median follow-up time of 26 months (range 11-48) were evaluated. In total, 25 (83%) achieved NEDA-3 status, and none received disease-modifying treatment after HSCT. For 13 (43%) of the patients, there were sustained improvement in Expanded Disability Status Scale (EDSS) score, and 10 (33%) were working full time after the treatment, compared to only 1 (3%) before treatment. There were no serious treatment-related complications and was no mortality. Five patients (17%) were diagnosed with an autoimmune thyroid disease after the procedure, and 10 (43%) of the women had amenorrhea lasting >12 months and symptoms of ovarian failure.CONCLUSION:
HSCT in MS is an effective and relatively safe treatment option, with few serious complications and no mortality in Norway, so far. However, long-term adverse event with amenorrhea is a common problem.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Transplante de Células-Tronco Hematopoéticas
/
Esclerose Múltipla Recidivante-Remitente
/
Esclerose Múltipla
Tipo de estudo:
Observational_studies
Limite:
Female
/
Humans
Idioma:
En
Revista:
Mult Scler
Assunto da revista:
NEUROLOGIA
Ano de publicação:
2020
Tipo de documento:
Article
País de afiliação:
Noruega