Retrospective observational study on the use of acetyl-l-carnitine in ALS
-
Published:2023-06-28
Issue:11
Volume:270
Page:5344-5357
-
ISSN:0340-5354
-
Container-title:Journal of Neurology
-
language:en
-
Short-container-title:J Neurol
Author:
Sassi Serena, Bianchi Elisa, Diamanti Luca, Tornabene Danilo, Sette Elisabetta, Medici Doriana, Matà Sabrina, Leccese Deborah, Sperti Martina, Martinelli Ilaria, Ghezzi Andrea, Mandrioli Jessica, Iuzzolino Valentina Virginia, Dubbioso Raffaele, Trojsi Francesca, Passaniti Carla, D’Alvano Giulia, Filosto Massimiliano, Padovani Alessandro, Mazzini Letizia, De Marchi Fabiola, Zinno Lucia, Nuredini Andi, Bongioanni Paolo, Dolciotti Cristina, Canali Elena, Toschi Giulia, Petrucci Antonio, Perna Alessia, Riso Vittorio, Inghilleri Maurizio, Libonati LauraORCID, Cambieri Chiara, Pupillo Elisabetta
Abstract
AbstractALCAR (Acetyl-L-carnitine) is a donor of acetyl groups and increases the intracellular levels of carnitine, the primary transporter of fatty acids across the mitochondrial membranes. In vivo studies showed that ALCAR decrease oxidative stress markers and pro-inflammatory cytokines. In a previous double-blind placebo-controlled phase II trial showed positive effects on self-sufficiency (defined as a score of 3+ on the ALSFRS-R items for swallowing, cutting food and handling utensils, and walking) ALSFRS-R total score and FVC. We conducted an observational, retrospective, multicentre, case–control study to provide additional data on the effects of ALCAR in subjects with ALS in Italy. Subjects treated with ALCAR 1.5 g/day or 3 g/day were included and matched with not treated subjects by sex, age at diagnosis, site of onset, and time from diagnosis to baseline, (45 subjects per group). ALCAR 3 g/day vs not treated: 22 not treated subjects (48.9%) were still alive at 24 months after baseline, compared to 23 (51.1%) treated subjects (adj. OR 1.18, 95% CI 0.46–3.02). No statistically significant differences were detected in ALSFRS nor FVC nor self-sufficiency. ALCAR 1.5 g/day vs not treated: 22 not treated subjects (48.9%) were still alive at 24 months after baseline, compared to 32 (71.1%) treated subjects (adj. OR 0.27, 95% CI 0.10–0.71). For ALSFRS-R, a mean slope of − 1.0 was observed in treated subjects compared to − 1.4 in those not treated (p = 0.0575). No statistically significant difference was detected in the FVC nor self-sufficiency. Additional evidence should be provided to confirm the efficacy of the drug and provide a rationale for the dosage.
Funder
Università degli Studi di Roma La Sapienza
Publisher
Springer Science and Business Media LLC
Subject
Neurology (clinical),Neurology
Reference14 articles.
1. Feldman EL, Goutman SA, Petri S, Mazzini L, Savelieff MG, Shaw PJ, Sobue G (2022) Amyotrophic lateral sclerosis. Lancet 400(10360):1363–1380. https://doi.org/10.1016/S0140-6736(22)01272-7 2. Virmani A, Binienda Z (2004) Role of carnitine esters in brain neuropathology. Mol Aspects Med 25(5–6):533–549. https://doi.org/10.1016/j.mam.2004.06.003 3. Bigini P, Larini S, Pasquali C, Muzio V, Mennini T (2002) Acetyl-l-carnitine shows neuroprotective and neurotrophic activity in primary culture of rat embryo motoneurons. Neurosci Lett 329(3):334–338. https://doi.org/10.1016/s0304-3940(02)00667-5 4. Bigini P, Muzio V, de Angelis C, Mennini T (1999) In vitro and in vivo effect of acetyl-l-carnitine on motor neuron degeneration. Neurosci Lett 52:S18 5. Li X, Zhang C, Zhang X et al (2015) An acetyl-l-carnitine switch on mitochondrial dysfunction and rescue in the metabolomics study on aluminium oxide nanoparticles. Part Fibre Toxicol 13:4. https://doi.org/10.1186/s12989-016-0115-y
Cited by
4 articles.
订阅此论文施引文献
订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献
|
|