Center for Rare Neurological Diseases, Atlanta, GA, USA.
Le Bonheur Children's Hospital, University of Tennessee Health Science Center, Memphis, TN, USA.
J Child Neurol. 2023 May;38(6-7):389-393. doi: 10.1177/08830738231185424. Epub 2023 Jul 16.
Pediatric developmental epileptic encephalopathies are often refractory to treatment despite stable antiseizure therapy. The safety profile of diazepam nasal spray (Valtoco) as rescue therapy for seizure clusters was described in a long-term safety study. This post hoc analysis assessed safety and effectiveness within a subpopulation of patients with developmental epileptic encephalopathies. Of 163 treated patients, 64 were diagnosed with ≥1 pediatric developmental epileptic encephalopathy. Among the most common developmental epileptic encephalopathies were Rett syndrome (n = 16), Lennox-Gastaut syndrome (n = 9), and Dravet syndrome (n = 7). In the broad pediatric developmental epileptic encephalopathy group, 10.6% of seizure clusters were treated with a second dose, with similar proportions in the 3 individual encephalopathies. Across groups, treatment-emergent adverse event rates ranged from 66.7% to 100%. Only epistaxis (n = 2) was treatment-related and reported in >1 patient. In this long-term safety analysis in patients with developmental epileptic encephalopathies, diazepam nasal spray demonstrated a consistent safety profile, supporting its use in these hard-to-treat patients (ClinicalTrials.gov NCT02721069).
儿科发育性癫痫性脑病尽管采用了稳定的抗癫痫治疗，但往往仍难以治疗。在一项长期安全性研究中，描述了 diazepam 鼻喷剂（Valtoco）作为癫痫发作群的抢救治疗的安全性概况。本事后分析评估了发育性癫痫性脑病亚人群中的安全性和有效性。在 163 例接受治疗的患者中，有 64 例被诊断患有≥1 种儿科发育性癫痫性脑病。最常见的发育性癫痫性脑病包括雷特综合征（n=16）、 Lennox-Gastaut 综合征（n=9）和 Dravet 综合征（n=7）。在广泛的儿科发育性癫痫性脑病组中，10.6%的癫痫发作群接受了第二剂治疗，在这 3 种个别脑病中也有类似的比例。在所有组中，治疗中出现的不良事件发生率从 66.7%到 100%不等。只有鼻出血（n=2）是治疗相关的，且有>1 例患者报告。在这项针对发育性癫痫性脑病患者的长期安全性分析中，diazepam 鼻喷剂表现出一致的安全性特征，支持将其用于这些难以治疗的患者（ClinicalTrials.gov NCT02721069）。
