Neuroblastoma (Peripheral neuroblastic tumours)

Area of Science:

• Pediatric Oncology
• Cancer Genetics
• Sympathetic Nervous System Tumors

Background:

• Peripheral neuroblastic tumours (PNTs) constitute 7-10% of pediatric cancers, originating from the sympathetic nervous system.
• PNTs frequently arise in adrenal glands or retroperitoneal ganglia, with over a third presenting with metastases, primarily to bone and bone marrow.
• Prognostic factors include disease extent, MYCN oncogene status, and patient age, influencing treatment strategies.

Purpose of the Study:

• To review the current understanding and treatment outcomes of peripheral neuroblastic tumours in children.
• To highlight the challenges in treating metastatic PNTs and those with MYCN gene amplification.
• To discuss ongoing research exploring novel therapeutic approaches for PNTs.

Main Methods:

• Review of existing literature on PNTs, treatment protocols, and survival data.
• Analysis of prognostic factors and their impact on patient outcomes.
• Summary of current clinical trials and emerging treatment strategies.

Main Results:

• Surgical resection alone achieves >85% 3-year event-free survival (EFS) for localized, operable PNTs.
• Locally advanced PNTs treated with chemotherapy, surgery, and/or radiotherapy have an EFS of approximately 75%.
• Outcomes remain poor for children with metastatic PNTs or MYCN gene amplification despite intensive treatments.

Conclusions:

• Treatment advancements have significantly improved cure rates for PNTs over the past two decades.
• Localized disease is highly curable, but metastatic and high-risk PNTs require further therapeutic innovation.
• Novel drugs and immunotherapies are under investigation to improve survival for high-risk pediatric neuroblastic tumours.