Oral etoposide as a single agent in childhood and young adult cancer in England: Still a poorly evaluated palliative treatment

Jess Fraser, Lorna Wills, Fahmina Fardus-Reid, Lucy Irvine, Lucy Elliss-Brookes, Lorna Fern, Alison L. Cameron, Kathy Pritchard-Jones, Richard G. Feltbower, Jon Shelton, Charles Stiller, Martin G. McCabe*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

3 Downloads (Pure)


Background: Oral etoposide is commonly used in palliative treatment of childhood and young adult cancer without robust evidence. We describe a national, unselected cohort of young people in England treated with oral etoposide using routinely collected, population-level data. 

Methods: Patients aged under 25 years at cancer diagnosis (1995–2017) with a treatment record of single-agent oral etoposide in the Systemic AntiCancer Dataset (SACT, 2012–2018) were identified, linked to national cancer registry data using NHS number and followed to 5 January 2019. Overall survival (OS) was estimated for all tumours combined and by tumour group. A Cox model was applied accounting for age, sex, tumour type, prior and subsequent chemotherapy. 

Results: Total 115 patients were identified during the study period. Mean age was 11.8 years at cancer diagnosis and 15.5 years at treatment with oral etoposide. Median OS was 5.5 months from the start of etoposide; 13 patients survived beyond 2 years. Survival was shortest in patients with osteosarcoma (median survival 3.6 months) and longest in CNS embryonal tumours (15.5 months). Across the cohort, a median of one cycle (range one to nine) of etoposide was delivered. OS correlated significantly with tumour type and prior chemotherapy, but not with other variables. 

Conclusions: This report is the largest series to date of oral etoposide use in childhood and young adult cancer. Most patients treated in this real world setting died quickly. Despite decades of use, there are still no robust data demonstrating a clear benefit of oral etoposide for survival.

Original languageEnglish
Article number29204
Number of pages11
JournalPediatric Blood and Cancer
Issue number11
Early online date6 Jul 2021
Publication statusPublished - Nov 2021

Bibliographical note

Funding Information: This work uses data that have been provided by patients and collected by the NHS as part of their care and support. The data are collated, maintained and quality assured by the National Cancer Registration and Analysis Service, which is part of Public Health England (PHE). The work was undertaken as part of the Cancer Research UK – Public Health England partnership. The study was exempt from gaining individual consent having obtained Section 251 approval from the UK Patient Information Advisory Group (PIAG; now the Confidentiality Advisory Group, CAG), under Section 251 of the NHS Act 2006 (PIAG 03(a)/2001). Lorna Fern is funded by Teenage Cancer Trust.

Open Access: This is an open access article under the terms of the Creative Commons Attribution-NonCommercial License, which permits use, distribution and reproduction in any
medium, provided the original work is properly cited and is not used for commercial purposes.

Publisher Copyright: © 2021 The Authors. Pediatric Blood & Cancer published by Wiley Periodicals LLC.

Citation: Fraser, J, Fardus-Reid, F, Irvine, L, et al. Oral etoposide as a single agent in childhood and young adult cancer in England: Still a poorly evaluated palliative treatment. Pediatr Blood Cancer. 2021; 68:e29204.

DOI: https://doi.org/10.1002/pbc.29204


  • AYA
  • cancer
  • children
  • etoposide
  • population
  • survival


Dive into the research topics of 'Oral etoposide as a single agent in childhood and young adult cancer in England: Still a poorly evaluated palliative treatment'. Together they form a unique fingerprint.

Cite this