Effect of Serum Perampanel Concentration on Sporadic Amyotrophic Lateral Sclerosis Progression
0
조회
0
다운로드
0
인용
0
참고문헌
키워드
초록
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.
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.
참고문헌 (30)
- Talbott EO, "The epidemiology of amyotrophic lateral sclerosis", Handb Clin Neurol, 138, pp.225~238 (2016)
- Arai T, "TDP-43 is a component of ubiquitin-positive tau-negative inclusions in frontotemporal lobar degeneration and amyotrophic lateral sclerosis", Biochem Biophys Res Commun, 351, pp.602~611 (2006)
- Neumann M, "Ubiquitinated TDP-43 in frontotemporal lobar degeneration and amyotrophic lateral sclerosis", Science, 314, pp.130~133 (2006)
- Aizawa H, "TDP-43 pathology in sporadic ALS occurs in motor neurons lacking the RNA editing enzyme ADAR2", Acta Neuropathol, 120, pp.75~84 (2010)
- Melcher T, "A mammalian RNA editing enzyme", Nature, 379, pp.460~464 (1996)
- Hideyama T, "Induced loss of ADAR2 engenders slow death of motor neurons from Q", R site-unedited GluR2, 2010, pp.30, 11917~11925
- Rogawski MA, "Preclinical pharmacology of perampanel, a selective non-competitive AMPA receptor antagonist", Acta Neurol Scand Suppl, 197, pp.19~24 (2013)
- French JA, "Adjunctive perampanel for refractory partial-onset seizures : randomized phase III study 304", Neurology, 79, pp.589~596 (2012)
- French JA, "Evaluation of adjunctive perampanel in patients with refractory partial-onset seizures : results of randomized global phase III study 305", Epilepsia, 54, pp.117~125 (2013)
- Krauss GL, "Randomized phase III study 306 : adjunctive perampanel for refractory partial-onset seizures", Neurology, 78, pp.1408~1415 (2012)
- Gil-Nagel A, "A retrospective, multicentre study of perampanel given as monotherapy in routine clinical care in people with epilepsy", Seizure, 54, pp.61~66 (2018)
- Toledano Delgado R, "Effectiveness and safety of perampanel monotherapy for focal and generalized tonic-clonic seizures : experience from a national multicenter registry", Epilepsia, 61, pp.1109~1119 (2020)
- Hanada T, "The discovery and development of perampanel for the treatment of epilepsy", Expert Opin Drug Discov, 9, pp.449~458 (2014)
- Akamatsu M, "The AMPA receptor antagonist perampanel robustly rescues amyotrophic lateral sclerosis(ALS)pathology in sporadic ALS model mice", Sci Rep, 6, pp.28649~ (2016)
- Aizawa H, "Randomized phase 2 study of perampanel for sporadic amyotrophic lateral sclerosis", J Neurol, 269, pp.885~896 (2022)
- Ludolph A, "A revision of the El Escorial criteria-2015", Amyotroph Lateral Scler Frontotemporal Degener, 16, pp.291~292 (2015)
- Posner K, "The Columbia-suicide severity rating scale : initial validity and internal consistency findings from three multisite studies with adolescents and adults", Am J Psychiatry, 168, pp.1266~1277 (2011)
- Clinical Practice Guidelines for Epilepsy Development Committee, "Clinical Practice Guidelines for Epilepsy 2018", pp.123~125 (2018), Igaku-Shoin Ltd
- Steinhoff BJ, "Efficacy and safety of adjunctive perampanel for the treatment of refractory partial seizures : a pooled analysis of three phase III studies", Epilepsia, 54, pp.1481~1489 (2013)
- Ishikawa N, "Clinical profiles associated with serum perampanel concentrations in children with refractory epilepsy", Epilepsy Behav, 94, pp.82~86 (2019)
- Inoue Y, "Evaluation of real-world effectiveness of perampanel in Japanese adults and older adults with epilepsy", Epileptic Disord, 24, pp.123~132 (2022)
- Yamamoto Y, "Therapeutic drug monitoring for perampanel in Japanese epilepsy patients : influence of concomitant antiepileptic drugs", Ther Drug Monit, 39, pp.446~449 (2017)
- Patsalos PN, "The clinical pharmacology profile of the new antiepileptic drug perampanel : a novel noncompetitive AMPA receptor antagonist", Epilepsia, 56, pp.12~27 (2015)
- Ohkubo S, "Quantification of the plasma concentrations of perampanel using high-performance liquid chromatography and effects of the CYP3A4*1G polymorphism in Japanese patients", J Chromatogr Sci, 58, pp.915~921 (2020)
- Eggert K, "Safety and efficacy of perampanel in advanced Parkinson’s disease : a randomized, placebo-controlled study", Mov Disord, 25, pp.896~905 (2010)
- Lees A, "Perampanel, an AMPA antagonist, found to have no benefit in reducing"off"time in Parkinson’s disease", Mov Disord, 27, pp.284~288 (2012)
- Lattanzi S, "Efficacy and safety of perampanel in Parkinson’s disease. A systematic review with meta-analysis", J Neurol, 265, pp.733~740 (2018)
- Ueda J, "Perampanel inhibits α-synuclein transmission in Parkinson’s disease models", Mov Disord, 36, pp.1554~1564 (2021)
- Chiò A, "Phenotypic heterogeneity of amyotrophic lateral sclerosis : a population based study", J Neurol Neurosurg Psychiatry, 82, pp.740~746 (2011)
- Patsalos PN, "Serum protein binding of 25 antiepileptic drugs in a routine clinical setting : a comparison of free non-protein-bound concentrations", Epilepsia, 58, pp.1234~1243 (2017)