Pediatric Neurology Division, Department of Pediatrics, All India Institute of Medical Sciences, Rishikesh, Uttarakhand 249203, India.
Department of Neurology, IQRAA International Hospital and Research Centre, Kozhikode, Kerala 673009, India.
Seizure. 2022 Dec;103:11-17. doi: 10.1016/j.seizure.2022.10.005. Epub 2022 Oct 4.
In adult patients with epilepsy, predictive models have been developed and validated for anticipating a favorable response to immunotherapy. However, no such model has been evaluated in children.
This retrospective cohort study intended to assess the performance of a pediatric adaptation of the Response to Immunotherapy in Epilepsy (RITE) score: P-RITE score and Antibody Prevalence in Epilepsy (APE) score: P-APE score in patients aged 1-18 years. We included data of those patients who had epilepsy duration of not more than 12 months, no other known etiology (e.g., genetic, metabolic, neoplastic, or structural causes), and tested for neural-specific antibody in cerebrospinal fluid or serum for P-APE score and only those who received immunotherapy for P-RITE score. We added cognitive dysfunction, speech dysfunction, sleep disturbance, and movement disorder to the original scores to increase specificity for pediatric autoimmune epilepsy. We assumed at least a 50% reduction in seizure frequency at 6 months as a favorable response to immunotherapy. Cut-offs were chosen for both scores to maximize true positives and minimize false negatives using ROC curves.
We included data from a total of 237 patients with epilepsy (10.4 ± 2.5 years, 129 boys, 54%), out of which, 25 (10.5%, 13 girls, 52%) tested positive for autoantibodies. The median P-APE score in the subgroup with and without antibody positivity were 7 (IQR: 5-11) and 2 (IQR: 1-5), respectively (p<0.0001). ROC analysis of the P-APE score determined an AUC of 0.96. The sensitivity and specificity values of the P-APE score ≥6 were 94% and 92%, respectively. A total of 162 patients (10.3 ± 2.5 years, 88 boys, 54%) received immunotherapy, out of which, 101 had a favorable response at 6 months. The median P-RITE score in the subgroup with and without favorable response following a trial of immunotherapy were 10 (IQR: 6-17) and 3 (IQR: 1-6), respectively (p<0.0001). ROC analysis of the P-RITE score determined an AUC of 0.96. The sensitivity and specificity values of P-RITE score ≥8 were 95% and 93%, respectively. The AUC of both these ROCs was significantly higher than the AUC of ROCs for original scores in our cohort.
The P-RITE and P-APE scores can be used to predict the response to immunotherapy and predict autoantibody positivity in children with epilepsy with/without encephalopathy or cognitive dysfunction.
在患有癫痫的成年患者中，已经开发和验证了预测模型，以预测免疫治疗的良好反应。然而，在儿童中尚未评估此类模型。
本回顾性队列研究旨在评估儿科版反应性免疫疗法评分（RITE）的表现：P-RITE 评分和抗体在癫痫中的流行率（APE）评分：P-APE 评分在 1-18 岁患者中的表现。我们纳入了癫痫持续时间不超过 12 个月、无其他已知病因（例如遗传、代谢、肿瘤或结构原因）的患者的数据，并为 P-APE 评分检测脑脊液或血清中的神经特异性抗体，仅对 P-RITE 评分进行免疫治疗的患者进行检测。我们将认知功能障碍、言语功能障碍、睡眠障碍和运动障碍添加到原始评分中，以提高儿科自身免疫性癫痫的特异性。我们假设免疫治疗后 6 个月内至少减少 50%的癫痫发作频率为免疫治疗的良好反应。使用 ROC 曲线为两个评分选择截断值，以最大化真阳性并最小化假阴性。
我们共纳入了 237 名癫痫患者的数据（10.4±2.5 岁，129 名男孩，54%），其中 25 名（10.5%，13 名女孩，52%）抗体检测阳性。抗体阳性亚组和无抗体阳性亚组的中位 P-APE 评分分别为 7（IQR：5-11）和 2（IQR：1-5）（p<0.0001）。P-APE 评分的 ROC 分析确定 AUC 为 0.96。P-APE 评分≥6 的灵敏度和特异性分别为 94%和 92%。共 162 名患者（10.3±2.5 岁，88 名男孩，54%）接受了免疫治疗，其中 101 名在 6 个月时有良好反应。免疫治疗试验后有良好反应和无良好反应的亚组的中位 P-RITE 评分分别为 10（IQR：6-17）和 3（IQR：1-6）（p<0.0001）。P-RITE 评分的 ROC 分析确定 AUC 为 0.96。P-RITE 评分≥8 的灵敏度和特异性分别为 95%和 93%。这两个 ROC 的 AUC 均显著高于我们队列中原评分的 AUC。
P-RITE 和 P-APE 评分可用于预测免疫治疗反应，并预测伴有或不伴有脑病或认知功能障碍的癫痫儿童的自身抗体阳性。
