Efficacy of perampanel in pediatric epilepsy with known and presumed genetic etiology

Miao, Pu; Zhu, Xueying; Jin, Wenqin; Yu, Lingyan; Li, Yanfang; Wang, Ye; Su, Qunyan; Xu, Sha; Wang, Shuang; Feng, Jianhua

doi:10.1002/acn3.51828

Cited by 6 publications

(1 citation statement)

References 30 publications

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“…The patients’ medical records, dosage of PER, age, sex, body weight, and comedications (other AEDs). The dosing strategy was set by the physician according to its label, which was detailly described in our previous publication ( Miao et al, 2023 ). In our hospital, PER TDM was performed 3 weeks after initiation of PER treatment, as PER would achieve steady states after 19 days of dosing.…”

Section: Methodsmentioning

confidence: 99%

Initial therapeutic target attainment of perampanel in pediatric patients with epilepsy

Yu,

Chen,

Liu

et al. 2023

Front. Pharmacol.

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Perampanel is a promising option for the treatment of pediatric epilepsy, but its plasma concentration varies among patients. This retrospective study aimed to investigate the initial target attainment of perampanel plasma concentration in pediatric patients with epilepsy in China. Inpatients admitted from January 2020 to December 2021 in a tertiary hospital were retrospectively included according to pre-set criteria. Demographic characteristics of patients and dosing strategies and therapeutic drug monitoring results were collected. A total of 137 pediatric patients (84 females and 53 males, aged from 0.6 to 16.4 years) were include for analysis. The perampanel concentrations varied greatly from 60 to 1,560 mg/L among patients, but 89.8% had suitable perampanel concentrations (100–1,000 ng/mL). The concomitant use of enzyme-inductive antiepileptic drugs (AEDs) was the only identified risk factor associated with target nonattainment (OR = 5.92, 95% confidence interval 1.68–20.9). Initial perampanel target attainment in pediatric patients is satisfactory. Routine therapeutic drug monitoring to achieved the suggested concentration range for these patients may be unnecessary, except for those receiving combined enzyme inductive AEDs.

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Section: Methodsmentioning

confidence: 99%

Initial therapeutic target attainment of perampanel in pediatric patients with epilepsy

Yu,

Chen,

Liu

et al. 2023

Front. Pharmacol.

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Response to amoxicillin and perampanel in infantile Alexander disease

Boronat,

Turon‐Viñas,

Mac Manus

et al. 2024

Epilepsia Open

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Type I Alexander disease (AxD) presents with paroxysmal neurodegeneration, refractory epilepsy, and encephalopathy in the first years of life and is associated with a poor prognosis. Although there is no treatment, mild symptomatic improvement has been reported in one case of adult Alexander treated with ceftriaxone, given its interaction with the mutant glial fibrillary acid protein (GFAP) responsible for the disease's pathogenesis. We describe a patient presenting with irritability starting at 2 months of age, initially attributed to gastroesophageal reflux. A ventriculoperitoneal shunt was placed at 3 months of age due to hydrocephalus secondary to aqueduct stenosis detected through an MRI scan, but the irritability persisted. At 5 months, a new brain MRI was performed due to irritability worsening, onset of abnormal ocular movements and seizures. In addition genetic testing was performed. AxD was diagnosed due to the mutation c.716G>A (p.Arg239His) in GFAP. Since irritability had worsened and had not responded to levomepromazine, treatment with amoxicillin (80 mg/kg/day) was attempted to modulate glutamate levels. The patient showed a striking improvement of irritability in 48 h that persisted over the next months. The patient had frequent daily seizures which did not respond to valproate, clonazepam, or phenobarbital. Perampanel, a postsynaptic AMPA receptor antagonist, was added to phenobarbital and he was seizure free for more than 3 months. Drugs modulating glutamate levels in the central nervous system, including β‐lactam antibiotics and perampanel, may have an important role in the symptomatic treatment of AxD and other neurodegenerative diseases where glutamatergic excitotoxicity is a pathogenic determinant.Plain Language SummaryAlexander disease is a rare and serious condition that affects the brain, often leading to neurodegeneration (brain damage), seizures, and other problems in early childhood. The disease is caused by a mutation in a gene called GFAP. There is no cure, and current treatments mainly focus on relieving symptoms. This article discusses the case of a baby who showed signs of irritability and seizures from a young age. The baby was diagnosed with Alexander disease after brain scans and genetic testing. Despite treatment with various drugs, the baby continued to experience seizures and irritability. The doctors decided to try amoxicillin, a common antibiotic, because of its potential to help control the disease by affecting a brain chemical called glutamate. Surprisingly, the baby's irritability improved within 2 days of starting amoxicillin, and the improvement lasted for several months. However, the seizures persisted until another medication, perampanel, was added. This combination controlled the baby's seizures for over 3 months. Unfortunately, the baby passed away at 13 months due to complications from the disease. However, doctors believe that drugs like amoxicillin and perampanel could be promising treatments for managing symptoms of Alexander disease and other similar brain conditions in the future, especially where excess glutamate plays a role in the damage. This case suggests that these treatments may help control irritability and seizures, offering hope for better management of this challenging disease.

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Perampanel for the treatment of epilepsy with genetic aetiology: Real-world evidence from the PERMIT Extension study

Delanty,

Mohanraj,

Shankar

et al. 2024

Epilepsy Research

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Efficacy of perampanel in pediatric epilepsy with known and presumed genetic etiology

Cited by 6 publications

References 30 publications

Initial therapeutic target attainment of perampanel in pediatric patients with epilepsy

Initial therapeutic target attainment of perampanel in pediatric patients with epilepsy

Response to amoxicillin and perampanel in infantile Alexander disease

Perampanel for the treatment of epilepsy with genetic aetiology: Real-world evidence from the PERMIT Extension study

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