Szczepanik Elzbieta, Mierzewska Hanna, Antczak-Marach Dorota, Figiel-Dabrowska Anna, Terczynska Iwona, Tryfon Jolanta, Krzesniak Natalia, Noszczyk Bartlomiej Henryk, Sawicka Ewa, Domanska-Janik Krystyna, Sarnowska Anna
Clinic of Child and Adolescent Neurology, Institute of Mother and Child, Poland.
Mossakowski Medical Research Centre, Polish Academy of Sciences, Poland.
Stem Cells Int. 2020 Jan 3;2020:7104243. doi: 10.1155/2020/7104243. eCollection 2020.
. Evaluation of efficacy and safety of autologous adipose-derived regenerative cells (ADRCs) treatment in autoimmune refractory epilepsy. . Six patients with proven or probable autoimmune refractory epilepsy (2 with Rasmussen encephalitis, 2 with antineuronal autoantibodies in serum, and 2 with possible FIRES) were included in the project with approval of the Bioethics Committee.
Intrathecal injection of autologous ADRC acquired through liposuction followed by enzymatic isolation was performed. The procedure was repeated 3 times every 3 months with each patient. Neurological status, brain MRI, cognitive function, and antiepileptic effect were monitored during 12 months.
Immediately after the procedure, all patients were in good condition. In some cases, transient mildly elevated body temperature, pain in regions of liposuction, and slight increasing number of seizures during 24 hours were observed. During the next months, some improvements in school, social functioning, and manual performance were observed in all patients. One patient has been seizure free up to the end of trial. In other patients, frequency of seizures was different: from reduced number to the lack of improvement (3-year follow-up).
Autologous ADRC therapy may emerge as a promising option for some patients with autoimmune refractory epilepsy. Based on our trial and other clinical data, the therapy appears to be safe and feasible. Antiepileptic efficacy proved to be various; however, some abilities improved in all children. No signs of psychomotor regression were observed during the first year following the treatment.
自体脂肪源性再生细胞(ADRCs)治疗自身免疫性难治性癫痫的疗效和安全性评估。6例经证实或可能为自身免疫性难治性癫痫的患者(2例患有拉斯穆森脑炎,2例血清中存在抗神经元自身抗体,2例可能患有FIRES)在获得生物伦理委员会批准后被纳入该项目。
通过抽脂获取自体ADRC,经酶分离后进行鞘内注射。每位患者每3个月重复该操作3次。在12个月内监测神经状态、脑部MRI、认知功能和抗癫痫效果。
术后即刻,所有患者情况良好。在某些情况下,观察到短暂的轻度体温升高、抽脂部位疼痛以及24小时内癫痫发作次数略有增加。在接下来的几个月里,所有患者在学业、社交功能和手部操作方面均有一些改善。1例患者直至试验结束无癫痫发作。在其他患者中,癫痫发作频率各不相同:从发作次数减少到无改善(3年随访)。
自体ADRC治疗可能成为一些自身免疫性难治性癫痫患者的有前景的选择。基于我们的试验和其他临床数据,该治疗似乎是安全可行的。抗癫痫疗效各不相同;然而,所有儿童的一些能力都有所改善。治疗后的第一年未观察到精神运动发育倒退的迹象。