Long-term efficacy of docosahexaenoic acid (DHA) for Spinocerebellar Ataxia 38 (SCA38) treatment: An open label extension study.
Ataxia
Cerebellum
Clinical trial
Docosahexaenoic acid (DHA)
Spinocerebellar ataxia 38 (SCA38)
Journal
Parkinsonism & related disorders
ISSN: 1873-5126
Titre abrégé: Parkinsonism Relat Disord
Pays: England
ID NLM: 9513583
Informations de publication
Date de publication:
06 2019
06 2019
Historique:
received:
14
11
2018
revised:
06
02
2019
accepted:
23
02
2019
pubmed:
14
3
2019
medline:
6
5
2020
entrez:
14
3
2019
Statut:
ppublish
Résumé
Spinocerebellar Ataxia 38 (SCA38) is caused by ELOVL5 gene mutation, with significant reduction of serum docosahexaenoic acid (DHA) levels. DHA supplementation has been proven effective at short-term follow-up. In the present paper, we evaluated long-term safety and efficacy of 600 mg/day oral DHA in SCA38 by a 2-year open label extension study. Nine SCA38 patients underwent standardised clinical assessment at 62 (T1), 82 (T2) and 104 (T3) weeks, and compared to pre-treatment scores (T0). Brain 18-Fluorodeoxyglucose Positron Emission Tomography and electroneurography were performed at T0 and T3. We found a significant maintenance of clinical symptom improvement at each follow-up time-point (p < 0.001) as compared to T0, a sustained increase of cerebellar metabolism at T3 as compared to T0 (p = 0.013), and no worsening of neurophysiological parameters. No side effect was recorded. Long-term DHA supplementation is an eligible treatment for SCA38.
Identifiants
pubmed: 30862453
pii: S1353-8020(19)30085-9
doi: 10.1016/j.parkreldis.2019.02.040
pii:
doi:
Substances chimiques
ELOVL5 protein, human
0
Radiopharmaceuticals
0
Fluorodeoxyglucose F18
0Z5B2CJX4D
Docosahexaenoic Acids
25167-62-8
Fatty Acid Elongases
EC 2.3.1.-
Types de publication
Clinical Trial
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
191-194Informations de copyright
Copyright © 2019. Published by Elsevier Ltd.