Pediatric High-Risk Neuroblastoma Treated With Autologous Stem Cell Transplant-Experience From a Tertiary Cancer Center in India.

Source: PubMed, NCBI / U.S. National Library of Medicine

High-risk neuroblastoma (HR-NB) remains a therapeutic challenge in low- and middle-income countries. We retrospectively analyzed the clinical characteristics, treatment response, transplant outcomes, event-free survival, and overall survival of pediatric patients with HR-NB treated with intensive multimodality therapy, including autologous stem cell transplant (ASCT) at a resource-limited tertiary cancer center between January 2013 and January 2024. Children aged 18 years or younger received rapid-COJEC induction chemotherapy, followed by surgical resection when feasible, ASCT with busulfan-melphalan conditioning, radiotherapy, and differentiation therapy with 13-cis-retinoic acid. Eighteen patients were included, with a male-to-female ratio of 2:1 and a median age of 4 years. The adrenal gland was the most common primary site in 55.55% of patients. Bone marrow and bone metastases were present in 72.2% and 44.4% of patients, respectively. Complete response after induction was achieved in 44.4% of patients. Median neutrophil engraftment occurred at 13 days. At a median follow-up of 17 months, 55.5% of patients were alive in remission. Relapse occurred in 50%, with most succumbing to the disease. There was no treatment-related mortality. Two-year event-free and overall survival were 55.3% and 65.6%, respectively. Outcomes were comparable to other resource-limited settings, and the lack of complete response after induction did not adversely affect event-free survival.