Abstract
OBJECTIVES: To investigate the correlation between induction therapy response and prognosis in children with high-risk neuroblastoma, and to analyze factors associated with the induction therapy response. METHODS: Data of 55 children with high-risk neuroblastoma diagnosed and treated at Shanghai Children's Hospital from January 2019 to December 2023 were retrospectively reviewed. Induction response was assessed according to the International Neuroblastoma Response Criteria and patients were categorized into a good-response group (complete response or very good partial response) and a poor-response group (partial response, progressive disease, mixed response, or no response). Clinical and biological characteristics, treatments, and prognostic factors were analyzed. RESULTS: Among the 55 children, 29 were male and 26 were female; the median age at onset was 39 months. Follow-up was performed until December 31, 2024. The 3-year overall survival (OS) and event-free survival (EFS) rates were (83.8±5.3)% and (47.0±10.3)%, respectively. Neuron-specific enolase level at initial diagnosis, induction therapy response, radiotherapy, and recurrence were prognostic factors for EFS and OS (P<0.05). The 3-year OS was (83.5±7.4)% in the good-response group and (66.7±13.6)% in the poor-response group (P=0.012), while the 3-year EFS was (62.8±10.4)% and (27.8±14.8)%, respectively (P<0.001). Intracranial metastasis at initial diagnosis was associated with a poor induction response (P=0.033). A platelet count ≥400×10(9)/L was associated with a better induction response (P=0.002). CONCLUSIONS: Induction therapy response is a significant prognostic factor in high-risk neuroblastoma. Absence of intracranial metastasis and a platelet count ≥400×10(9)/L at initial diagnosis are associated with a favorable induction therapy response.