Background and purpose: The prognosis of brain stem glioma in children is poor

which is a serious threat to children’s lives. Most gliomas are overexpressed with epidermal growth factor receptor (EGFR)

and nimotuzumab is a therapeutic drug specifically binding to EGFR. This study was to investigate the efficacy and toxicity of nimotuzumab in combination with chemoradiotherapy in patients with brain stem gliomas in children. Methods: From January 2010 to December 2014

20 children with brain stem glioma confirmed by imaging or pathology in Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine were randomly divided into 3 groups. Seven patients with brain stem glioma in group 1 were treated with nimotuzumab combined with chemoradiotherapy. Seven patients in group 2 were treated with radiotherapy and chemotherapy

and 6 patients in group 3 were treated with radiotherapy alone. Results: The hematological toxicity occurred in only 4 cases

nausea in 2 cases and fever in 1 case

and all of which were grade Ⅰ/Ⅱ. The median overall survival (OS) for group 1

2 and 3 was 11.6

9.7 and 7.1 months

respectively. There was a significant difference in survival curve among the three groups (Log-rank P=0.008). The median OS of high grade gliomas for group 1

2 and 3 was 12.1

8.1 and 5.8 months

respectively. There was a significant difference in survival curve among the three groups (Log-rank P=0.015 2). The median OS was 12.1 months in the patients with high-grade glioma treated with nimotuzumab and 7.1 months without nimotuzumab. The difference between the two survival curves was statistically significant (P=0.011 9). Partial remission (PR) rates showed no statistical significant difference in this study. Conclusion: Nimotuzumab combined with chemoradiotherapy can significantly improve the prognosis and prolong overall survival of children with brain stem glioma. However

the results need to be verified in a study with larger sample size.