Ten Challenges in the Management of Neuroblastoma

Abstract

Neuroblastoma is a complex disease with many contradictions and challenges. It is, by and large, a cancer of babies and preschool children, but it does occur, albeit increasingly rarely, in older children, adolescents and young adults. The prognosis is very variable, with outcome related to age, stage and molecular pathology. Neuroblastoma may behave in an almost benign way, with spontaneous regression in some infants, but the majority of older patients have high-risk disease, which is usually fatal, despite best current treatments. As a rare disease, international collaboration is essential to run clinical trials of adequate statistical power to answer important questions in a reasonable time frame. High-risk disease requires multimodality therapy including chemotherapy, surgery and radiotherapy as well as biological and immunological treatments for optimal outcomes. Innovative treatment approaches, sometimes associated with appreciable toxicity, offer hope for the future but, despite parental wishes, cannot be generally implemented without adequate assessment in clinical trials.

Keywords:

• high-dose chemotherapy
• immunotherapy
• international collaboration
• molecular pathology
• molecular radiotherapy
• neuroblastoma
• risk stratification
• staging

Financial & competing interests disclosure

This work was undertaken at UCLH/UCL, which received a proportion of funding from the Department of Health’s NIHR Biomedical Research Centres funding scheme and The Neuroblastoma Children’s Cancer Alliance. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.

Acknowledgements

The authors would like to thank Professor NJ Sebire for providing the photomicrographs (Figures 2 & 3).

Additional information

Funding

This work was undertaken at UCLH/UCL, which received a proportion of funding from the Department of Health’s NIHR Biomedical Research Centres funding scheme and The Neuroblastoma Children’s Cancer Alliance. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.