Autologous Stem Cell Transplantation for High-Risk Neuroblastoma - A Real-World Evidence from A Tertiary Cancer Care...

Management of high-risk neuroblastoma (HR-NB) in resource-limited settings is challenging with dismal outcomes. There are only limited published data from India on outcomes for children with HR-NB who underwent autologous stem cell transplantation (ASCT). We intended to report the outcomes of children with HR-NB who underwent ASCT at our center. Patients managed as HR-NB and underwent ASCT from January 2010 to December 2020 at the Cancer Institute (WIA), Chennai, India were retrospectively analysed. Twenty-five children with HR-NB underwent ASCT at our center during the study period. The median age at diagnosis was 5 years, with a male predominance (M: F ratio 3:1). Most patients had metastatic disease (92%). Most children received induction based on the Children's Cancer Group (CCG) 3891 study protocol (n = 22). Fourteen children underwent surgery before ASCT, with 10 achieving complete macroscopic resection. The median time to ASCT from diagnosis was 8 months. Stem cells were primarily harvested from peripheral blood, with a median CD34 + cell count of 4.5 × 10⁶ cells/kg body weight. Conditioning regimens utilised included: intravenous (IV) Melphalan alone (n = 4); IV Busulfan + Melphalan (n = 21). Neutrophil and platelet engraftment occurred at a median of 12- and 14-days post-transplantation, respectively. The median duration of hospital stay for ASCT was 28 days. The most common non-infectious toxicity was mucositis, and the only mortality during ASCT was due to dengue shock syndrome. The median follow-up for survivors was 97 months. The 3-year event-free survival (EFS1) and overall survival (OS1) rates were 28% [95% CI: 12 - 46%] and 36% [95% CI: 18 - 54%] respectively. The 3-year post-ASCT event-free survival (EFS2) and post-ASCT overall survival (OS2) rates of the study cohort were 19% [95% CI: 7 - 37%] and 28% [95% CI: 12 - 46%], respectively. Relapse occurred in 80% of the children after a median of 12 months post-ASCT. Multidisciplinary care for children HR-NB, including ASCT is feasible in resource-limited settings. Despite intensive multidisciplinary management, HR-NB remains difficult to treat, with a high relapse rate post-ASCT.

The online version contains supplementary material available at 10.1007/s12288-025-02012-z.