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Efficacy and safety of adjunctive perampanel treatment in pediatric patients with epilepsy aged 4–12 years: a real-world study

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Abstract

Objective

To determine the efficacy and safety of perampanel (PER) as an adjunctive therapy in children aged 4–12 years with epilepsy.

Methods

We performed a non-randomized, open-label, placebo-uncontrolled, real-world self-controlled study that included 216 young children (aged 4–12 years) with epilepsy who received PER as adjunctive therapy at the children’s hospital affiliated with Chongqing Medical University from July 4, 2020, to September 20, 2023.

Results

(1) The efficacy rates of adjunctive PER therapy at 3, 6, 9, and 12 months were 62.8%, 67.8%, 65.3%, and 61.2%, respectively. PER showed efficacy in alleviating focal seizures, generalized tonic–clonic seizures, myoclonic seizures, and absence seizures. The efficacy rates for variants of self-limited epilepsy with centrotemporal spikes (SeLECTS) and Lennox-Gastaut syndrome (LGS) were 89.5% and 66.7%, respectively. (2) Focal non-motor onset seizures with or without impaired awareness, focal to bilateral tonic–clonic seizures (FBTCS), LGS, variants of SeLECTS, the number of concomitant antiseizure medications (ASMs), a family history of epilepsy, and focal lesions on cranial magnetic resonance imaging were independent factors affecting efficacy. The order of PER addition did not affect efficacy. The retention rates at 3, 6, 9, and 12 months were 90.7%, 84.7%, 74.7%, 64.9%, respectively. (3) Adverse reactions occurred in 45 patients (45/216, 20.8%), with irritability/aggressive behavior (18/216, 8.3%) and somnolence (14/216, 6.5%) being the most common. Twelve patients (12/216, 5.6%) withdrew from the study because of adverse reactions.

Conclusion

In young Chinese children with epilepsy, PER is effective, safe, and well-tolerated as an adjunctive therapy, making it a viable option for use with broad-spectrum ASMs.

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Data availability

The data that support the findings are available from the corresponding author upon reasonable request.

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Funding

This research did not receive any specific grant from any funding agency in the public, commercial or not-for-profit sector.

Author information

Authors and Affiliations

  1. Department of Neurology, Children’s Hospital of Chongqing Medical University, No. 136 Zhongshan 2nd Road, Yu Zhong District, Chongqing, 400014, China

    Qiao Zeng, Xueqian Xia, Li Jiang, Jin Chen, Yuhang Liu & Yue Hu

  2. Ministry of Education Key Laboratory of Child Development and Disorders, Chongqing, China

    Qiao Zeng, Xueqian Xia, Li Jiang, Jin Chen, Yuhang Liu & Yue Hu

  3. National Clinical Research Center for Child Health and Disorders (Chongqing), Chongqing, China

    Qiao Zeng, Xueqian Xia, Li Jiang, Jin Chen, Yuhang Liu & Yue Hu

  4. China International Science and Technology Cooperation Base of Child Development and Critical Disorders, Chongqing, China

    Qiao Zeng, Xueqian Xia, Li Jiang, Jin Chen, Yuhang Liu & Yue Hu

  5. Chongqing Key Laboratory of Child Neurodevelopment and Cognitive Disorders, Chongqing, China

    Qiao Zeng, Xueqian Xia, Li Jiang, Jin Chen, Yuhang Liu & Yue Hu

  6. Big Data Engineering Center, Children’s Hospital of Chongqing Medical University, Chongqing, China

    Qiao Zeng & Yue Hu

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  1. Qiao Zeng
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Contributions

Qiao Zeng and Yue Hu contributed to the conceptualization and design of the study, data acquisition and analysis, and drafting of the manuscript. All authors contributed to the data acquisition, review, and editing.

Corresponding author

Correspondence to Yue Hu.

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Conflicts of interest

None of the authors has any conflict of interest to disclose.

Ethical approval

Ethics approval was granted by the Ethics Committee of the Children’s Hospital affiliated with Chongqing Medical University [Ethical Approval Number: (2023) LunShen (Yan) No. 473]. All steps performed in our study (data collection and interviews) were in accordance with the ethical standards of the national research committee, with the 1964 Helsinki declaration and its later amendments and with the national data protection office requirements.

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Zeng, Q., Xia, X., Jiang, L. et al. Efficacy and safety of adjunctive perampanel treatment in pediatric patients with epilepsy aged 4–12 years: a real-world study. J Neurol (2024). https://doi.org/10.1007/s00415-024-12416-y

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  • DOI: https://doi.org/10.1007/s00415-024-12416-y

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