Balancing intervention timing and spontaneous regression for optimal prognosis in neonatal neuroblastoma

Abstract Background Neonatal neuroblastoma is defined as neuroblastoma diagnosed via prenatal screening or within the first 28 days of life, comprising approximately 5% of all cases. Most belong to low- or intermediate-risk groups and have favorable prognoses. However, their rarity and limited case numbers contribute to ongoing debate regarding the optimal treatment strategy. Methods This retrospective study analyzed 53 neonatal neuroblastoma patients diagnosed and treated at Beijing Children's Hospital, Capital Medical University, between July 2015 and January 2020. Results Of the 53 patients, 13 (24.53%) were classified as stage L1, 18 (33.96%) as stage L2, 6 (11.32%) as stage M, and 16 (30.19%) as stage MS. The timing of treatment initiation was identified as a prognostic risk factor. Spontaneous regression occurred in eight patients without further treatment; of these, seven later received additional therapy, while one remained under observation, all achieving favorable outcomes. The 2-year overall survival (OS) and event-free survival (EFS) rates were 90.4% and 81.1%, respectively. Among 38 patients who underwent nuclear medicine imaging (positron emission tomography—computed tomography or bone scan), three had bone metastases. However, statistical analysis showed no significant impact of nuclear imaging on prognosis ( P = 0.650). Conclusion Neonatal neuroblastoma has a generally favorable prognosis. Limited observation in asymptomatic cases does not compromise outcomes. For non-high-risk, asymptomatic patients eligible for complete surgical resection, surgery alone is a viable option with close follow-up. Routine nuclear medicine imaging is not recommended for non-high-risk patients without radiological evidence of metastases.