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Outcomes of Infants and Young Children With CNS Embryonal Tumors Using Pre-Irradiation Chemotherapy: A Decade Long
Maya Prasad1,2, Duhita Sengupta1, Venkata Ramamohan Gollamudi1,2
1Division of Paediatric Oncology, Tata Memorial Centre, Mumbai, India.
Insights
Moderate-intensity pre-irradiation chemotherapy is a safe and effective strategy for treating infants and young children with central nervous system embryonal tumors (CNS ETs), offering a viable option in resource-limited settings.
Area of Science:
- Pediatric Oncology
- Neuro-oncology
- Radiation Oncology
Background:
- Management of central nervous system embryonal tumors (CNS ETs) in young children presents significant challenges.
- This study investigates a treatment strategy involving surgery, pre-irradiation chemotherapy, and delayed radiation for these patients.
Purpose of the Study:
- To evaluate the safety and efficacy of a specific treatment protocol for infants and young children diagnosed with CNS ETs.
- To determine survival outcomes and prognostic factors in this patient population.
Main Methods:
- A cohort of 72 children under 3 years old with various CNS ETs (medulloblastoma, ATRT, ETMR, ET NOS, pineoblastoma) were treated.
- Treatment included maximal safe surgical excision, pre-irradiation chemotherapy (cyclophosphamide, etoposide, carboplatin), and delayed radiation (craniospinal or focal).
Main Results:
- The 3-year event-free survival (EFS) and overall survival (OS) for the entire cohort were 39.8% and 44.8%, respectively.
- For medulloblastoma patients, 3-year progression-free survival (PFS) and OS were 57.4% and 62%.
- Metastatic disease and postoperative residual tumor were significant prognostic factors for medulloblastoma survival. Over 80% of long-term survivors experienced late toxicities.
Conclusions:
- Moderate-intensity pre-irradiation chemotherapy is a safe and effective approach for CNS ETs in young children.
- While outcomes may be inferior to high-dose or intraventricular chemotherapy, this strategy is suitable for resource-limited settings.
Background:
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.
Procedure:
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.
Results:
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.
Conclusion:
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.
