Treatment and Outcome of Children With Neuroblastoma in India: Report From the InPOG-NB-18-01 India Neuroblastoma Registration Study.
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AIM: To describe the clinical, management, and outcome profile of childhood neuroblastoma (NB) in India and identify barriers to optimal management. METHODS: Retrospective (InPOG-NB-18-01) analysis of NB in children under 15 diagnosed between June 2016 and August 2021 across 14 centers, with prospective follow-up for survival outcomes. RESULTS: A total of 320 children with NB were included, with a median age of 29 months (interquartile range [IQR]: 14-55 months) and a male:female ratio of 1.36:1. Nuclear imaging for staging and MYCN amplification testing were performed in 60.3% and 72.5% of cases, respectively. International Neuroblastoma Risk Group (INRG) staging was L1, L2, M, and MS in 39 (12.2%), 89 (27.8%), 175 (54.7%), and 17 (5.3%), respectively. The INRG risk groups were Very low risk, Low risk, Intermediate risk, and High risk in 48 (15%), 29 (9%), 47 (14.7%), and 168 (52.5%) children, respectively, with 28 (8.8%) unknown due to diagnostic limitations. Over half of patients had high-risk neuroblastoma (HR-NB), and of those, only 55 (32.4%) underwent high-dose chemotherapy with autologous stem cell rescue (HDCT/ASCR), and one (0.6%) received anti-GD2 immunotherapy. The 3-year overall survival (OS) and event-free survival (EFS) for the entire cohort was 60% (54.1%-66.5%) and 50.2% (44.4%-56.7%), respectively. Risk factors for poor EFS included age (p < 0.001), INRG risk group (p < 0.001), MYCN amplification (p < 0.001), ferritin levels (p = 0.04), and lactate dehydrogenase levels (p < 0.001). CONCLUSIONS: Lack of accurate diagnostic risk stratification may contribute to poor outcomes of patients with NB in India. Sub-optimal outcomes are primarily seen in HR-NB, with limited access to HDCT/ASCR and anti-GD2 immunotherapy.