Is cerebrospinal fluid amyloid-ß42 a promising biomarker of response to nusinersen in adult spinal muscular atrophy patients?
Muscle Nerve
; 63(6): 905-909, 2021 06.
Article
em En
| MEDLINE
| ID: mdl-33660868
ABSTRACT
INTRODUCTION:
Nusinersen was approved as the first treatment for all types of spinal muscular atrophy (SMA), including adults with SMA types 2 and 3. Robust biomarkers of treatment response in SMA adults are lacking. Our aim was to examine cerebrospinal fluid (CSF) amyloid-ß40 (Aß40) and amyloid-ß42 (Aß42) peptides as biomarkers of treatment response.METHODS:
Eight patients with SMA types 2 and 3 were recruited consecutively in a single-center study. CSF was sampled at baseline, after a loading dose, and after three maintenance doses. Levels of Aß42 and Aß40 were evaluated for each CSF sampling. Wilcoxon matched-pairs signed-rank test was used to detect longitudinal changes.RESULTS:
CSF levels of Aß42 increased from baseline to day 420 (95% confidence interval, P = .018), with a significant increase at days 180 and 420 compared with days 0 and 300, respectively (95% confidence interval, P = .012 and P = .018).DISCUSSION:
The maintenance and promotion of wellness of residual motor neurons mediated by the restored level of SMN protein due to nusinersen could result in an increased level of amyloid peptides.Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Oligonucleotídeos
/
Fragmentos de Peptídeos
/
Atrofia Muscular Espinal
/
Peptídeos beta-Amiloides
Limite:
Adolescent
/
Adult
/
Aged
/
Female
/
Humans
/
Male
/
Middle aged
Idioma:
En
Ano de publicação:
2021
Tipo de documento:
Article