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

Abstract

Purpose

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.

Methods

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

Results

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.

Conclusions

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

Zusammenfassung

ZielDurch multimodale Therapien hat sich das Überleben von Patienten mit Hochrisiko-Neuroblastom verbessert, wodurch therapiebedingte Spätfolgen zunehmend an Bedeutung gewinnen. Ziel dieser Studie war es, Häufigkeit, Schweregrad und Risikofaktoren von Spätfolgen nach Hochdosis-Chemotherapie (HDC) und autologer Stammzelltransplantation (ASCT) zu untersuchen.

MethodenIn diese retrospektive Einzelzentrumsstudie wurden Hochrisiko-Neuroblastom-Patienten eingeschlossen, die nach ASCT mindestens fünf Jahre rezidivfrei überlebt hatten.

ErgebnisseVon 41 Patienten, die HDC und ASCT erhielten, wurden 20 Patienten analysiert. Das mediane Alter bei der letzten Nachsorge betrug 13,5 Jahre, die mediane Nachbeobachtungszeit 9 Jahre. Mindestens eine Spätkomplikation trat bei 95 % der Patienten auf; schwere Spätfolgen wurden bei 7 Patienten beobachtet. Zwei Patienten entwickelten therapieassoziierte sekundäre Neoplasien. Entgegen der Literatur war die häufigste Spätfolge eine fokale noduläre Hyperplasie der Leber (40%), gefolgt von Ovarialversagen (37,5%) und Hörverlust (35%). Endokrine Erkrankungen traten häufiger bei Patienten auf, die Busulfan–Melphalan als Konsolidierungstherapie erhalten hatten.

SchlussfolgerungSpätfolgen sind bei Überlebenden eines Hochrisiko-Neuroblastoms häufig und unterstreichen die Notwendigkeit einer strukturierten Langzeitnachsorge.

Schlüsselwörter

pädiatrische Onkologie - Stammzelltransplantation - Hochdosis-Chemotherapie - Neuroblastom - Spätfolgen

Keywords

pediatric oncology - stem cell transplantation - high-dose chemotherapy - neuroblastoma - late effects

Publication History

Received: 17 September 2025

Accepted after revision: 16 February 2026

Accepted Manuscript online:
18 February 2026

Article published online:
06 March 2026

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