Evaluation of Long-Term Late Side Effects in High-Risk Neuroblastoma Patients Undergoing Autologous Bone Marrow Transplantation

Abstract

Survival in high-risk neuroblastoma has improved with multimodal treatment approaches and numerous studies have focused on treatment-related side effects. This study examines the prevalence, severity and risks of late effects following high-dose chemotherapy and autologous stem cell transplantation in high risk neuroblastoma patients.

This study included high-risk neuroblastoma patients who underwent autologous stem cell transplantation and survived 5 years without relapse in a single center.

Of the 41 patients who received high-dose chemotherapy and autologous stem cell transplantation, 20 patients survived without relapse for at least 5 years and were included in the analysis. Amid 20 patients, 12 patients (60%) were men. The median age of the patients was 13.5 years at the last follow up. After six cycles of induction chemotherapy, 11 patients (55%) received busulfan–melphalan as the consolidation regimen, while 9 patients (45%) received carboplatin–etoposide–melphalan. The median follow-up after transplantation was 9 years. At least one complication occurred in 19 out of 20 patients (95%). Severe late complications were observed in seven patients. Two patients developed treatment-related secondary neoplasms. In contrast to the literature, the most common adverse event was focal nodular hyperplasia (40%) in the liver. The second most common adverse event was ovarian failure (37.5%) and the third most common was hearing loss (35%). Endocrine pathologies were more common in patients receiving busulfan–melphalan as the consolidation regimen.

This study highlights the future risks of side effects in high-risk neuroblastoma survivors and emphasizes the need for a long-term follow-up.