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.