Retrospective observational study on the use of acetyl-L-carnitine in ALS.
- 2023-06-28
- Journal of neurology 270(11)
- Serena Sassi
- Elisa Bianchi
- Luca Diamanti
- Danilo Tornabene
- Elisabetta Sette
- Doriana Medici
- Sabrina Matà
- Deborah Leccese
- Martina Sperti
- Ilaria Martinelli
- Andrea Ghezzi
- Jessica Mandrioli
- Valentina Virginia Iuzzolino
- Raffaele Dubbioso
- Francesca Trojsi
- Carla Passaniti
- Giulia D'Alvano
- Massimiliano Filosto
- Alessandro Padovani
- Letizia Mazzini
- Fabiola De Marchi
- Lucia Zinno
- Andi Nuredini
- Paolo Bongioanni
- Cristina Dolciotti
- Elena Canali
- Giulia Toschi
- Antonio Petrucci
- Alessia Perna
- Vittorio Riso
- Maurizio Inghilleri
- Laura Libonati
- Chiara Cambieri
- Elisabetta Pupillo
- PubMed: 37378756
- DOI: 10.1007/s00415-023-11844-6
Study Design
- Type
- Observational
- Sample size
- n = 45
- Population
- 45 subjects per group (ALCAR-treated and matched untreated) with ALS in Italy
- Methods
- Observational, retrospective, multicentre, case-control study; ALCAR 1.5 g/day or 3 g/day; matched by sex, age at diagnosis, site of onset, time from diagnosis to baseline
- Duration
- 24 months
- Funding
- Unclear
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.
Research Insights
No statistically significant differences were detected in ALSFRS nor FVC nor self-sufficiency.
- Effect
- Neutral
- Effect size
- Small
- Dose
- 3 g/day
No statistically significant differences were detected in ALSFRS nor FVC nor self-sufficiency.
- Effect
- Neutral
- Effect size
- Small
- Dose
- 3 g/day
No statistically significant differences were detected in ALSFRS nor FVC nor self-sufficiency.
- Effect
- Neutral
- Effect size
- Small
- Dose
- 3 g/day
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).
- Effect
- Beneficial
- Effect size
- Large
- Dose
- 1.5 g/day
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).
- Effect
- Beneficial
- Effect size
- Moderate
- Dose
- 1.5 g/day