J Clin Neurol.  2023 May;19(3):280-287. 10.3988/jcn.2022.0213.

Effect of Serum Perampanel Concentration on Sporadic Amyotrophic Lateral Sclerosis Progression

• Kato H
• Naito M
• Saito T
• Hideyama T
• Terashi H
• Kwak S
• Aizawa H

• KMID: 2542076
• DOI: http://doi.org/10.3988/jcn.2022.0213

Abstract

Background and Purpose
To clarify the effect of perampanel (PER) on sporadic amyotrophic lateral sclerosis (sALS) progression, the relationship between the changes in Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) scores and serum PER concentrations was investigated.
Methods
12 patients with sALS from our hospital who agreed to participate and completed the PER for sALS randomized phase 2 study were included. After completing the study, we retrospectively obtained serum PER concentration data from the patients. Based on their mean PER concentrations, we divided the patients who had been taking PER into two groups: four patients with a mean PER concentration of ≥400 ng/mL were assigned to the H group, and three with a mean PER concentration of <400 ng/mL were assigned to the L group. The control group consisted of five patients who had been taking a placebo. We obtained the ALSFRS-R scores of each patient at 36 and 48 weeks after randomization. The differences in ALSFRS-R scores at baseline (0 weeks) and each subsequent week were used in the analysis.
Results
At 48 weeks, there were no differences in the degree of deterioration of the bulbar, upper and lower limb, and respiratory ALSFRS-R subscores and total ALSFRS-R score. However, at 36 weeks, the bulbar subscore was significantly lower in the H group than in the control group (p=0.032).
Conclusions
Because high PER concentrations may exacerbate bulbar symptoms in patients with sALS, serum PER measurements may be beneficial when patients with sALS are taking PER.

Keyword

amyotrophic lateral sclerosis; perampanel; revised amyotrophic lateral sclerosis functional rating scale; serum concentration; sporadic amyotrophic lateral sclerosis