Clinical efficacy of combined therapy in children with stage 4 neuroblastoma
LIANG Wei-Ling, YE Xiao-Fan, ZHONG Gong, CHEN Jian-Jun, DAI Kang-Lin, CHEUK Ka Leung Daniel, MO Shu, WANG Bo-Shen, LI Chun-Yu, JIANG Xuan-Zhu, XU Zhi-Yuan, ZHOU Li, CHAN Irene, CHEN Jian-Liang, CHU Patrick., LEE Pui Wah Pamela, CHAN Chi Fung Godfrey
Department of Pediatrics, Hong Kong University-Shenzhen Hospital, Shenzhen, Guangdong 518000, China
Abstract：Objective To study the early clinical efficacy of combined therapy of stage 4 neuroblastoma. Methods A retrospective analysis was performed on the medical data and follow-up data of 14 children with stage 4 neuroblastoma who were diagnosed in Hong Kong University-Shenzhen Hospital from January 2016 to June 2021. Results The median age of onset was 3 years and 7.5 months in these 14 children. Among these children, 9 had positive results of bone marrow biopsy, 4 had N-Myc gene amplification, 13 had an increase in neuron-specific enolase, and 7 had an increase in vanilmandelic acid in urine. Based on the results of pathological examination, differentiated type was observed in 6 children, undifferentiated type in one child, mixed type, in one child and poorly differentiated type in 6 children. Of all the children, 10 received chemotherapy with the N7 regimen (including 2 children receiving arsenic trioxide in addition) and 4 received chemotherapy with the Rapid COJEC regimen. Thirteen children underwent surgery, 14 received hematopoietic stem cell transplantation, and 10 received radiotherapy. A total of 8 children received Ch14.18/CHO immunotherapy, among whom 1 child discontinued due to anaphylactic shock during immunotherapy, and the other 7 children completed Ch14.18/CHO treatment without serious adverse events, among whom 1 child was treated with Lu177 Dotatate 3 times after recurrence and is still undergoing chemotherapy at present. The median follow-up time was 45 months for all the 14 children. Four children experienced recurrence within 2 years, and the 2-year overall survival rate was 100%; 4 children experienced recurrence within 3 years, and 7 achieved disease-free survival within 3 years. Conclusions Multidisciplinary combined therapy is recommended for children with stage 4 neuroblastoma and can help them achieve better survival and prognosis.
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