Currently, the treatment of refractory epilepsy (RE) in children is still a difficult point in epilepsy treatment. In China, pirenzapine (PER) is still a new drug for treating RE in children, and there is currently a lack of recommendations for adding PER to the treatment of RE in children. And in Chinese reports, the sample size of RE patients treated with PER is relatively small. Therefore, the efficacy of PER for pediatric RE, especially for young children with RE, still needs to be further studied with a large sample size.

To explore the efficacy, possible indications, adverse reactions, and tolerability of PER addition therapy for RE in children.

A self-control and retrospective analysis was conducted on children with RE aged 0-18 who were treated at the Women and Children's Hospital, Qingdao University from January 2022 to January 2023. The frequency of seizures at different observation points before and after the addition of PER treatment was compared, and the effective rate of PER was evaluated. Adverse drug reactions and drug retention rates were recorded, and the clinical characteristics of the effective and ineffective groups of PER were analyzed.

A total of 192 study subjects were included. After adding PER treatment, the effective rates at 12, 24, and 36 weeks were 56.3% (108/192), 62.1% (113/182), and 69.7% (122/175), respectively, and the seizure free rates were 19.3% (37/192), 21.4% (39/182), and 24.6% (43/175). The incidence of adverse reactions was 16.1% (31/192), mainly including dizziness, irritability, weakness, and drowsiness. The last follow-up drug retention rate was 91.1% (175/192). There was a statistically significant difference in the onset age, duration of anti-epileptic treatment, type of origin, seizure form, frequency of seizures before the addition of PER, number of combined anti-epileptic drugs (ASMs), and ketogenic diet/surgical treatment between patients with RE who received continuous medication for 12 weeks (P<0.05). In addition, 178 children underwent EEG examination, and 167 children underwent cranial magnetic resonance imaging examination. There was a statistically significant difference in the electroencephalogram (EEG) and head magnetic resonance imaging (MRI) results between patients who received effective and ineffective treatment. In the results of electroencephalogram examination, the effective rate of discharge in the anterior (anterior, middle, temporal anterior, and middle) part of the brain was higher (P<0.05) ; In the results of cranial imaging examination, the normal group had a higher effective rate, followed by children with mainly white matter damage.

The overall effective rate and retention rate of PER addition therapy for RE in children are high, with mild adverse reactions and good drug tolerance. It is more effective for children with RE who have a late onset age, seizures in the form of motor seizures, focal origin, short course of anti-epileptic treatment, fewer combination medications, and less frequency of seizures. In electroencephalography, patients with normal discharge in the anterior (anterior, middle, anterior temporal, and middle temporal) of the brain and normal results in cranial magnetic resonance imaging have a higher effective rate.

目前儿童难治性癫痫（RE）仍是癫痫的治疗难点，国内吡仑帕奈（PER）治疗儿童RE尚属新药，缺少PER对儿童RE添加治疗的建议，且国内报道中使用PER治疗的RE患儿样本量较少。因此PER对于儿童RE，尤其低年龄RE患儿的疗效仍需大样本、进一步研究。

探究PER添加治疗儿童RE的疗效、可能的适应证、不良反应及耐受性。

对2022年1月—2023年1月在青岛大学附属妇女儿童医院诊治的0~18岁RE患儿进行自身对照、回顾性分析，对比PER添加治疗前后不同观察时点的癫痫发作频率的变化，评估PER的治疗效果，记录药物不良反应及药物保留率，并分析PER有效组与无效组的临床特征。

共纳入192例研究对象，PER添加治疗后，第12、24、36周的有效率分别为56.3%（108/192）、62.1%（113/182）、69.7%（122/175），无发作率分别为19.3%（37/192）、21.4%（39/182）、24.6%（43/175）。不良反应发生率为16.1%（31/192），主要为头晕、暴躁易怒、无力嗜睡等，末次随访药物保留率为91.1%（175/192）。PER添加治疗的RE患儿连续用药12周时，治疗总有效与无效/恶化患儿起病年龄、抗癫痫治疗病程、起源类型、发作形式、添加PER前发作频率、联合其他抗癫痫发作药物（ASMs）数量、生酮饮食/外科治疗情况，差异有统计学意义（P<0.05）。此外，178例患儿进行了脑电图检查，167例患儿进行了头颅磁共振检查；治疗总有效与无效/恶化患儿脑电图检查结果、头颅磁共振检查结果比较，差异有统计学意义（P<0.05）。脑电图检查结果中以脑前部（前、中、颞前、中部）放电总有效占比较高；头颅影像学检查结果中以正常者总有效占比较高，其次为以脑白质损伤为主的患儿。

PER添加治疗儿童RE整体有效率及保留率较高，不良反应轻微，药物耐受情况好。对于起病年龄晚、发作形式为运动性发作、局灶性起源、抗癫痫治疗病程短、联合用药数量及发作频率少的RE患儿更为有效。脑电图中以脑前部（前、中、颞前、颞中部）放电、头颅磁共振结果正常者总有效占比较高。