Clinical efficacy analysis of selumetinib in treating children with plexiform neurofibromas
10.3760/cma.j.cn113694-20250425-00244
- VernacularTitle:司美替尼治疗丛状神经纤维瘤患儿的临床疗效分析
- Author:
Xuan YUE
1
;
Xiaoming LIU
1
;
Jiao CHEN
1
;
Shuowang WEN
1
;
Li LIU
1
;
Li QIU
1
Author Information
1. 徐州市儿童医院神经内科，徐州221000
- Publication Type:Journal Article
- Keywords:
Neurofibroma, plexiform;
Neurofibromatosis 1;
Child;
Selumetinib;
Clinical efficacy;
Adverse events
- From:
Chinese Journal of Neurology
2025;58(9):963-970
- CountryChina
- Language:Chinese
-
Abstract:
Objective:To evaluate the clinical efficacy, safety, and adverse effects of selumetinib in the treatment of children with plexiform neurofibromas (PNF).Methods:A retrospective analysis was conducted on the clinical data of 11 children with PNF who were treated with oral selumetinib for at least 9 months at Xuzhou Children′s Hospital from January 2024 to February 2025. The dosage was 25 mg/m2 twice daily. General patient information was collected, and clinical efficacy parameters and adverse events were compared before treatment, at 6 months, and at 9 months post-treatment. Tumor volume changes were assessed using magnetic resonance imaging (MRI) according to the Response Evaluation Criteria in Solid Tumours (RECIST). Pain severity and its impact on daily life were evaluated using the Wong-Baker Faces Pain Rating Scale Revision (FPS-R) and Pain Interference Index (PII). Safety and adverse events were monitored via regular clinical examinations, cardiac ultrasound, and ophthalmologic evaluations, with adverse events graded using Common Terminology Criteria for Adverse Events (CTCAE) v5.0.Results:All 11 patients completed follow-up [9.20-13.60 (10.65±1.64) months]. The cohort included 6 males and 5 females, aged 4.00-14.00 (9.35±3.00) years. At 6 months post-treatment, MRI revealed≥20% tumor volume reduction in 2 patients (2/11), meeting RECIST criteria for partial response (PR). The remaining 9 patients showed no significant tumor changes, though 2 exhibited lightening of café-au-lait macules (CALMs). By 9 months, 5 patients (5/11) achieved PR per RECIST, with residual tumors demonstrating volume reductions below PR thresholds. Nine patients showed further lightening of CALMs. Comparisons of FPS-R scores at 6 (5.27±1.01) and 9 months (3.64±0.81) post-treatment with baseline scores (6.72±1.01) showed statistically significant differences ( F=29.059, P<0.001). FPS-R scores at both 6 months post-treatment ( t=3.365, P=0.007) and 9 months post-treatment ( t=7.889, P<0.001) were significantly lower compared to baseline scores. FPS-R scores at 9 months post-treatment were significantly lower than those at 6 months post-treatment ( t=4.175, P=0.002). The PII at 9 months (15.64±2.86) differed significantly from baseline (19.64±2.66, t=3.396, P=0.003) and 6-month scores (18.27±2.45, t=2.316, P=0.031), whereas no significant difference was observed between 6-month and baseline PII scores ( t=1.256, P=0.225). Five of the 11 children experienced adverse reactions after taking the medicine, mainly manifested as acne, rash, paronychia, and vomiting. The CTCAE grades of all adverse reactions were≤2, and all could be relieved after symptomatic treatment. There were no significant changes in the cardiac ejection fraction and electrocardiogram of the 11 children compared with the baseline. Conclusions:Selumetinib effectively alleviated pain and improved daily function in children with PNF. Prolonged treatment significantly reduced tumor volumes, demonstrating durable efficacy. The drug exhibited a favorable safety profile, with manageable adverse events.
