Evaluating the efficacy and safety of single-agent etoposide intra-CSF chemotherapy in children and young people with relapsed/refractory central nervous system tumours

Butler, Anna; Meijer, Lisethe; Liu, Jo-Fen; Chohan, Manjit; Jalloh, Ibrahim; Macarthur, Donald; Parr, Margaret; Wilne, Sophie; Wilson, Shaun; Walker, David; Grundy, Richard; Dandapani, Madhumita

Abstract

Purpose
The aim of the project was to evaluate intra-CSF etoposide administration in a palliative setting for children and young people with relapsed/refractory central nervous system (CNS) tumours, with the primary endpoints being overall survival and progression-free survival time. A safety endpoint was to assess the side effect profile and complications of intra-CSF etoposide.

Methods
Thirty-five patients under the age of 30 years (median age: 5.33 years) were enrolled onto the project. The cross-centre study was a service evaluation, with a data collection spreadsheet designed in Nottingham and completed by both Nottingham and Oxford centres. Data was analysed using SPSS, assessing the overall survival and progression-free survival times, as well as the 6-month and 1-year survival rates.

Results
The median overall survival and progression-free survival times were 10.97 and 5.91 months, respectively. The 6-month and 1-year overall survival rates were 67% and 48%, and the progression-free survival rates were 50% and 22%. Age at the start of intra-CSF therapy was significantly associated with overall survival (P = 0.046), with the 6 + age group having improved overall survival. Treatment type was significantly associated with overall survival (P = 0.012), with etoposide intra-CSF treatment being associated with improved overall survival. Treatment duration was significantly associated with both overall survival (P < 0.001) and progression-free survival (P < 0.001).

Conclusion
Intra-CSF etoposide treatment has shown to increase both overall and progression-free survival significantly, whilst having few side effects and maintaining a good quality of life for patients, reflecting it as a beneficial therapy in the palliative setting.

Citation

Butler, A., Meijer, L., Liu, J., Chohan, M., Jalloh, I., Macarthur, D., …Dandapani, M. (2023). Evaluating the efficacy and safety of single-agent etoposide intra-CSF chemotherapy in children and young people with relapsed/refractory central nervous system tumours. Child's Nervous System, 39, 1537-1544. https://doi.org/10.1007/s00381-023-05872-w

• Journal Article Type: Article
• Acceptance Date: Jan 31, 2023
• Online Publication Date: Mar 23, 2023
• Publication Date: 2023-06
• Deposit Date: Mar 23, 2023
• Publicly Available Date: Mar 23, 2023
• Journal: Child's Nervous System
• Print ISSN: 0256-7040
• Electronic ISSN: 1433-0350
• Publisher: Springer Verlag
• Peer Reviewed: Peer Reviewed
• Volume: 39
• Pages: 1537-1544
• DOI: https://doi.org/10.1007/s00381-023-05872-w
• Keywords: Neurology (clinical); General Medicine; Pediatrics, Perinatology and Child Health
• Public URL: https://nottingham-repository.worktribe.com/output/18812679
• Publisher URL: https://link.springer.com/article/10.1007/s00381-023-05872-w