Treatment and Outcome of Children With Neuroblastoma in India: Report From the InPOG‐NB‐18‐01 India Neuroblastoma Registration Study

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.