Long-Term Outcome of Young Infants With Suspected Neuroblastoma following Observation as Primary Therapy: A Report From the Children’s Oncology Group

Dholaria H, Naranjo A, Zhang FF, London WB, Diller L, Shamberger RC, Barnewolt CE, Schmidt ML, Maris JM, Cohn SL, Bagatell R, Nuchtern JG. Long-Term Outcome of Young Infants With Suspected Neuroblastoma following Observation as Primary Therapy: A Report From the Children’s Oncology Group. Pediatr Blood Cancer. 2025 Aug;72(8):e31829. doi: 10.1002/pbc.31829. Epub 2025 Jun 1. PMID: 40452136; PMCID: PMC12442495.

Several studies have established that patients with localized perinatal neuroblastoma can be safely observed; however, long-term outcomes have not been previously reported. We evaluated long-term outcomes of infants with suspected perinatal neuroblastoma enrolled on the Children’s Oncology Group (COG) ANBL00P2, which included an expectant observation approach. Among 87 eligible patients, the 10-year event-free survival (EFS) was 93.7%, and overall survival (OS) was 98.0%. Most tumors regressed spontaneously; only three patients reported progression to higher stage disease. This report reinforces the long-term safety of non-interventional management in perinatal neuroblastoma, sparing infants unnecessary surgery and associated risks.