Détail du document
Identifiant
doi:10.1007/s00415-023-11844-6...
Auteur
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 Marchi, Fabiola Zinno, Lucia Nuredini, Andi Bongioanni, Paolo Dolciotti, Cristina Canali, Elena Toschi, Giulia Petrucci, Antonio Perna, Alessia Riso, Vittorio Inghilleri, Maurizio Libonati, Laura Cambieri, Chiara Pupillo, ElisabettaLangue
enEditeur
Springer
Catégorie
Medicine & Public Health
Année
2023
Date de référencement
05/07/2023
Mots clés
observational study treatment amyotrophic lateral sclerosis neurological disease case–control efficacy nor baseline compared treated subjects 0Résumé
ALCAR (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.
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,Marchi, Fabiola,Zinno, Lucia,Nuredini, Andi,Bongioanni, Paolo,Dolciotti, Cristina,Canali, Elena,Toschi, Giulia,Petrucci, Antonio,Perna, Alessia,Riso, Vittorio,Inghilleri, Maurizio,Libonati, Laura,Cambieri, Chiara,Pupillo, Elisabetta, 2023, Retrospective observational study on the use of acetyl-l-carnitine in ALS, Springer
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