Effectiveness and safety of perampanel as add-on treatment in pediatric patients with epilepsy: a single center, prospective, cohort study_Journal of Epilepsy

Authors：

MA Nan , LI Xia , LI Bei , YANG Le , LIANG Lili ,  WANG Yan

Department of Neurology, Xi'an Children's Hospital, Xi’an 710003, China;

Corresponding?author：

WANG Yan, Email: wy800709@163.com

Keywords：

Perampanel; Epilepsy; Pediatric patients; effectiveness; Safety

DOI：

10.7507/2096-0247.202508009

Video：

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Objective To investigate the effectiveness and safety of Perampanel (PER) add-on treatment in the pediatric patients with uncontrolled epilepsy. Methods Patients aged 0 ~ 18 years who visited the Xi'an Children's Hospital between June 2021 and December 2024 were recruited. They had uncontrolled seizures and were treated with PER as add-on therapy. The outcomes included response rate, seizure-free rate, and retention rate at 3, 6, and 12 months, and at the last visit. Tolerability was also assessed throughout follow-up. Results A total of 105 pediatric patients with epilepsy were followed up for at least 6 months. Eighty-three cases were aged ≥4 years while 22 cases were aged <4 years. Focal seizures occurred in 76 (72.4%) cases and 29 (27.6%) cases suffered other seizure types. The cause of epilepsy was clarified in 46 (43.8%) cases. The diagnosis of epileptic syndrome could be established in 34 (32.4%) cases at the time of PER add-on. The median seizure frequency per 28 days was 8 at baseline. The median number of prior antiseizure therapy before PER add-on was 3. The overall response rates at 3, 6 and 12 months were 61.9%, 63.8%, and 64.7%, respectively. The seizure-free rates were 39.0%, 42.9%, and 52.9%, respectively. There was no significant difference in the response rate between focal seizures and other seizure types at the last visit (69.7% vs. 51.7%, P=0.084). No significant difference was perceived in the response rate between patients aged ≥4 years and those aged <4 years as well (68.7% vs. 50%, P=0.103). Multivariate Logistic regression analysis showed that the effectiveness of PER was probably related to the maintenance dose and numbers of pervious treatments. At the last visit, the retention rate of PER was 80.0% and the adverse events (AEs) occurred in 30.0% patients. The most common AEs include somnolence/sleepy in 12 (11.4%) cases and dizziness in 8 (7.6%) cases. Conclusion PER add-on treatment has a good effectiveness, safety and tolerability in the pediatric patients with epilepsy. It is probably that the effectiveness is related to the maintenance dose and previous treatment numbers.

Citation： MA Nan, LI Xia, LI Bei, YANG Le, LIANG Lili, WANG Yan. Effectiveness and safety of perampanel as add-on treatment in pediatric patients with epilepsy: a single center, prospective, cohort study. Journal of Epilepsy, 2025, 11(6): 482-487. doi: 10.7507/2096-0247.202508009 Copy

1.	Faulkner MA. Spotlight on perampanel in the management of seizures: design, development and an update on place in therapy. Drug design, development and therapy, 2017, 11: 2921-2930.
2.	Renfroe JB, Mintz M, Davis R, et al. Adjunctive perampanel oral suspension in pediatric patients from ≥2 to <12 years of age with epilepsy: pharmacokinetics, safety, tolerability, and efficacy. Journal of Child Neurology, 2019, 34(5): 284-294.
3.	Krauss GL, Perucca E, Kwan P, et al. Final safety, tolerability, and seizure outcomes in patients with focal epilepsy treated with adjunctive perampanel for up to 4 years in an open-label extension of phase III randomized trials: Study 307. Epilepsia, 2018, 59(4): 866-876.
4.	French JA, Krauss GL, Steinhoff BJ, et al. Evaluation of adjunctive perampanel in patients with refractory partial-onset seizures: results of randomized global phase III study 305. Epilepsia, 2013, 54(1): 117-125.
5.	Scheffer IE, Berkovic S, Capovilla G, et al. ILAE classification of the epilepsies: Position paper of the ILAE Commission for Classification and Terminology. Epilepsia, 2017, 58(4): 512-521.
6.	Fisher RS, Cross J H, French JA, et al. Operational classification of seizure types by the International League Against Epilepsy: Position Paper of the ILAE Commission for Classification and Terminology. Epilepsia, 2017, 58(4): 522-530.
7.	Wirrell EC, Nabbout R, ScheffeR IE, et al. Methodology for classification and definition of epilepsy syndromes with list of syndromes: Report of the ILAE Task Force on Nosology and Definitions. Epilepsia, 2022, 63(6): 1333-1348.
8.	Lagae L, Villanueva V, Meador KJ, et al. Adjunctive perampanel in adolescents with inadequately controlled partial-onset seizures: a randomized study evaluating behavior, efficacy, and safety. Epilepsia, 2016, 57(7): 1120-1129.
9.	Yun Y, Kim D, Lee YJ, et al. Efficacy and tolerability of adjunctive perampanel treatment in children under 12 years of age with refractory epilepsy. Korean Journal of Pediatrics, 2019, 62(7): 269-273.
10.	Hwang SK, Lee YJ, Nam SO, et al. Real-life effectiveness and tolerability of perampanel in pediatric patients aged 4 years or older with epilepsy: a korean national multicenter study. Journal of Clinical Neurology (Seoul, Korea), 2020, 16(1): 53-59.
11.	Swiderska N, Tan HJ, Rajai A, et al. Effectiveness and tolerability of Perampanel in children, adolescents and young adults with refractory epilepsy: A UK national multicentre study. Seizure, 2017, 52: 63-70.
12.	French JA, Krauss GL, Wechsler RT, et al. Perampanel for tonic-clonic seizures in idiopathic generalized epilepsy a randomized trial. Neurology, 2015, 85(11): 950-957.
13.	Steinhoff BJ, Hamer H, Trinka E, et al. A multicenter survey of clinical experiences with perampanel in real life in Germany and Austria. Epilepsy Research, 2014, 108(5): 986-988.
14.	Villanueva V, Montoya J, Castillo A, et al. Perampanel in routine clinical use in idiopathic generalized epilepsy: the 12-month GENERAL study. Epilepsia, 2018, 59(9): 1740-1752.
15.	Chang FM, Fan PC, Weng WC, et al. The efficacy of perampanel in young children with drug-resistant epilepsy. Seizure, 2020, 75: 82-86.
16.	Biró A, Stephani U, Tarallo T, et al. Effectiveness and tolerability of perampanel in children and adolescents with refractory epilepsies: first experiences. Neuropediatrics, 2015, 46(2): 110-116.
17.	張捷, 謝涵, 鄧泂, 等. 基于隨訪6個月療效結局的加用吡侖帕奈治療兒童藥物難治性癲癇的單中心前瞻性隊列研究. 中國循證兒科雜志, 2021(04): 269-274.
18.	De Liso P, Vigevano F, Specchio N, et al. Effectiveness and tolerability of perampanel in children and adolescents with refractory epilepsies-An Italian observational multicenter study. Epilepsy Research, 2016, 127: 93-100.
19.	Rosenfeld W, Conry J, Lagae L, et al. Efficacy and safety of perampanel in adolescent patients with drug-resistant partial seizures in three double-blind, placebo-controlled, phase III randomized clinical studies and a combined extension study. European Journal of Paediatric Neurology, 2015, 19(4): 435-445.
20.	褚思嘉, 湯繼宏, 李巖, 等. 吡侖帕奈治療兒童難治性癲癇的療效和安全性研究. 癲癇雜志, 2021(05): 385-391.
21.	Ikemoto S, Hamano SI, Hirata Y, et al. Efficacy and serum concentrations of perampanel for treatment of drug-resistant epilepsy in children, adolescents, and young adults: comparison of patients younger and older than 12 years. Seizure, 2019, 73: 75-78.
22.	Trinka E, Steinhoff BJ, Nikanorova M, et al. Perampanel for focal epilepsy: insights from early clinical experience. Acta Neurologica Scandinavica, 2016, 133(3): 160-172.
23.	Klein P, Devinsky O, French J, et al. Suicidality Risk of Newer Antiseizure Medications: A Meta-analysis. JAMA Neurology, 2021, 78(9): 1118-1127.
24.	Chinvarun Y, Huang CW, Wu Y, et al. Optimal use of perampanel in asian patients with epilepsy: expert opinion. Therapeutics and Clinical Risk Management, 2021, 17: 739-746.

1. Faulkner MA. Spotlight on perampanel in the management of seizures: design, development and an update on place in therapy. Drug design, development and therapy, 2017, 11: 2921-2930.
2. Renfroe JB, Mintz M, Davis R, et al. Adjunctive perampanel oral suspension in pediatric patients from ≥2 to <12 years of age with epilepsy: pharmacokinetics, safety, tolerability, and efficacy. Journal of Child Neurology, 2019, 34(5): 284-294.
3. Krauss GL, Perucca E, Kwan P, et al. Final safety, tolerability, and seizure outcomes in patients with focal epilepsy treated with adjunctive perampanel for up to 4 years in an open-label extension of phase III randomized trials: Study 307. Epilepsia, 2018, 59(4): 866-876.
4. French JA, Krauss GL, Steinhoff BJ, et al. Evaluation of adjunctive perampanel in patients with refractory partial-onset seizures: results of randomized global phase III study 305. Epilepsia, 2013, 54(1): 117-125.
5. Scheffer IE, Berkovic S, Capovilla G, et al. ILAE classification of the epilepsies: Position paper of the ILAE Commission for Classification and Terminology. Epilepsia, 2017, 58(4): 512-521.
6. Fisher RS, Cross J H, French JA, et al. Operational classification of seizure types by the International League Against Epilepsy: Position Paper of the ILAE Commission for Classification and Terminology. Epilepsia, 2017, 58(4): 522-530.
7. Wirrell EC, Nabbout R, ScheffeR IE, et al. Methodology for classification and definition of epilepsy syndromes with list of syndromes: Report of the ILAE Task Force on Nosology and Definitions. Epilepsia, 2022, 63(6): 1333-1348.
8. Lagae L, Villanueva V, Meador KJ, et al. Adjunctive perampanel in adolescents with inadequately controlled partial-onset seizures: a randomized study evaluating behavior, efficacy, and safety. Epilepsia, 2016, 57(7): 1120-1129.
9. Yun Y, Kim D, Lee YJ, et al. Efficacy and tolerability of adjunctive perampanel treatment in children under 12 years of age with refractory epilepsy. Korean Journal of Pediatrics, 2019, 62(7): 269-273.
10. Hwang SK, Lee YJ, Nam SO, et al. Real-life effectiveness and tolerability of perampanel in pediatric patients aged 4 years or older with epilepsy: a korean national multicenter study. Journal of Clinical Neurology (Seoul, Korea), 2020, 16(1): 53-59.
11. Swiderska N, Tan HJ, Rajai A, et al. Effectiveness and tolerability of Perampanel in children, adolescents and young adults with refractory epilepsy: A UK national multicentre study. Seizure, 2017, 52: 63-70.
12. French JA, Krauss GL, Wechsler RT, et al. Perampanel for tonic-clonic seizures in idiopathic generalized epilepsy a randomized trial. Neurology, 2015, 85(11): 950-957.
13. Steinhoff BJ, Hamer H, Trinka E, et al. A multicenter survey of clinical experiences with perampanel in real life in Germany and Austria. Epilepsy Research, 2014, 108(5): 986-988.
14. Villanueva V, Montoya J, Castillo A, et al. Perampanel in routine clinical use in idiopathic generalized epilepsy: the 12-month GENERAL study. Epilepsia, 2018, 59(9): 1740-1752.
15. Chang FM, Fan PC, Weng WC, et al. The efficacy of perampanel in young children with drug-resistant epilepsy. Seizure, 2020, 75: 82-86.
16. Biró A, Stephani U, Tarallo T, et al. Effectiveness and tolerability of perampanel in children and adolescents with refractory epilepsies: first experiences. Neuropediatrics, 2015, 46(2): 110-116.
17. 張捷, 謝涵, 鄧泂, 等. 基于隨訪6個月療效結局的加用吡侖帕奈治療兒童藥物難治性癲癇的單中心前瞻性隊列研究. 中國循證兒科雜志, 2021(04): 269-274.
18. De Liso P, Vigevano F, Specchio N, et al. Effectiveness and tolerability of perampanel in children and adolescents with refractory epilepsies-An Italian observational multicenter study. Epilepsy Research, 2016, 127: 93-100.
19. Rosenfeld W, Conry J, Lagae L, et al. Efficacy and safety of perampanel in adolescent patients with drug-resistant partial seizures in three double-blind, placebo-controlled, phase III randomized clinical studies and a combined extension study. European Journal of Paediatric Neurology, 2015, 19(4): 435-445.
20. 褚思嘉, 湯繼宏, 李巖, 等. 吡侖帕奈治療兒童難治性癲癇的療效和安全性研究. 癲癇雜志, 2021(05): 385-391.
21. Ikemoto S, Hamano SI, Hirata Y, et al. Efficacy and serum concentrations of perampanel for treatment of drug-resistant epilepsy in children, adolescents, and young adults: comparison of patients younger and older than 12 years. Seizure, 2019, 73: 75-78.
22. Trinka E, Steinhoff BJ, Nikanorova M, et al. Perampanel for focal epilepsy: insights from early clinical experience. Acta Neurologica Scandinavica, 2016, 133(3): 160-172.
23. Klein P, Devinsky O, French J, et al. Suicidality Risk of Newer Antiseizure Medications: A Meta-analysis. JAMA Neurology, 2021, 78(9): 1118-1127.
24. Chinvarun Y, Huang CW, Wu Y, et al. Optimal use of perampanel in asian patients with epilepsy: expert opinion. Therapeutics and Clinical Risk Management, 2021, 17: 739-746.

Journal of Epilepsy

Effectiveness and safety of perampanel as add-on treatment in pediatric patients with epilepsy: a single center, prospective, cohort study

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