# Outcomes of Infants and Young Children With CNS Embryonal Tumors Using Pre‐Irradiation Chemotherapy: A Decade Long Experience

## TL;DR

This study examines the outcomes of infants and young children with CNS embryonal tumors treated with surgery and pre-irradiation chemotherapy, finding moderate effectiveness and safety in resource-limited settings.

## Contribution

The study provides a decade-long analysis of pre-irradiation chemotherapy outcomes in young children with CNS embryonal tumors, emphasizing its safety and applicability in resource-limited areas.

## Key findings

- 3-year event-free survival and overall survival rates were 39.8% and 44.8% respectively for the entire cohort.
- Medulloblastoma patients had 57.4% progression-free survival and 62% overall survival at 3 years.
- Group 3 metastatic medulloblastoma and postoperative residual tumor were significant prognostic factors.

## Abstract

Management of infants and young children with embryonal tumors of the central nervous system remains a challenge. We report the outcomes in these children with the strategy of surgery, pre‐irradiation chemotherapy, and delayed radiation.

Children less than 3 years and diagnosed with medulloblastoma(MB), atypical teratoid rhabdoid tumor(ATRT), embryonal tumor with multilayered rosettes(ETMR), embryonal tumor tumors NOS(ET NOS) and pineoblastoma(PB), underwent standard evaluation and staging. They were treated with maximal safe surgical excision followed by pre‐irradiation chemotherapy (cyclophosphamide, etoposide and carboplatin) up to 36 months of age or till disease progression, followed by either craniospinal (standard 35Gy or reduced‐dose 23.4 Gy) with boost to a total of 54.8 Gy or focal irradiation depending on attained age, residual tumor, and metastasis.

72 children between January 2011 and December 2022 were eligible for inclusion; medulloblastoma‐43 (59.7%), ATRT‐9 (12.9%), ET NOS‐8 (11.1%), ETMR‐8 (11.1%) and PB‐4 (5.6%). The median chemotherapy cycles were 6 (range 2–15); 41 children went on to receive RT.

For the entire cohort, 3‐year event‐free survival (EFS) and overall survival (OS) were 39.8% ± 5.8% and 44.8% ± 5.9%. For medulloblastoma, 3‐year PFS and OS were 57.4% ± 7.6% and 62% ± 7.5%. Multivariable analysis for survival in medulloblastoma found group 3 metastatic (HR 3.5, 95% CI 1.16–10.99, p = 0.026) and postoperative residual tumor (HR 1.9%, 95% CI 0.52–5.31, p = 0.24) to be significant prognostic factors. Among long‐term survivors (n = 27), over 80% have late toxicities requiring intervention.

The use of moderate‐intensity pre‐irradiation chemotherapy is safe and effective in infants and young children with CNS ETs. Despite inferior outcomes compared to high‐dose chemotherapy and intraventricular chemotherapy, this strategy could be used in resource‐limited settings.