Efficacy of Arimoclomol for Amyotrophic Lateral Sclerosis: A Systematic Review and Meta-analysis of Randomized Control Trials
Warda Alrubasy1, Mohammad Jehad Taha1, Shams Sameer1, Fatema Alhnidi1, Husam Abu Daoud2, Hamidullah Sahibzada1, Mohammad Abuawwad1, mohamed salim3, Hend Abo Helow4, Ekram Hasanin5
1kasr Alainy Faculty of Medicine, Cairo University, 2Palestine Polytechnic University, 3Faculty of medicine modern university for information and technology, 4Faculty of medicine-Alazhar university, 5University of Tripoli
Objective:
This study aims to assess the efficacy of Arimoclomol in patients with amyotrophic lateral sclerosis (ALS)
Background:
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease marked by muscle weakness and eventual respiratory failure. Arimoclomol ,heat shock protein enhancer, prevents this misfolded protein aggregation
Design/Methods:
A systematic search was performed across 5 databases( PubMed, Scopus, Google Scholar, Cochrane Central Register of Controlled Trials (CENTRAL), and ClinicalTrials.gov.) to identify relevant RCTs comparing the efficacy of Arimoclomol to placebo in patients with ALS. The protocol was submitted to PROSPERO ( CRD42024586655)
Results:
Three RCTs were included in this study, with 359 participants total (239 received arimoclomol, 120 a placebo). Participants were predominantly middle-aged ALS patients, with a male-to-female ratio of 1.6:1, and the average time from symptom onset to enrollment was 445 days. At enrollment, 85.8% were on riluzole. Follow-up duration ranged from 6 to 19 months, with a median of 12 months. Arimoclomol dosage varied across the studies. The results show that arimoclomol was associated with a slightly slower decline in ALSFRS-R scores compared to placebo, with a mean difference of 2.64 (95% CI: -0.95 to 6.23, P = 0.15), however the results were not statistically significant. A secondary analysis focusing on the total change in ALSFRS-R scores at 6 months also showed non-significant results, with a mean difference of 1.23 (95% CI: -0.95 to 3.41, P = 0.27). However, statistical significance was achieved when the study by Benatar et al. (2024) was excluded from the secondary analysis. Furthermore, Patients treated with arimoclomol experienced a smaller decline in SVC% and FEV6% and higher average CAFS scores, though none of these findings reached statistical significance.
Conclusions:
Efficacy of Arimoclomol in patients with ALS is still doubtful. Further studies on larger patient cohorts are needed to establish more definitive conclusions.
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