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Feasibility of Proton Beam Therapy for Infants with Brain Tumours: Experiences from the Prospective KiProReg Registry
D Jazmati1, T Steinmeier1, D Ahamd Khalil1
1Department of Particle Therapy, University Hospital Essen, West German Proton Therapy Centre Essen, West German Cancer Centre, Essen, Germany.
Insights
Proton beam therapy (PBT) is feasible for infants with central nervous system tumors, showing promising short-term outcomes. However, PBT requires intensive care and further research is needed to understand long-term effects.
Area of Science:
- Pediatric Oncology
- Radiation Oncology
- Neuro-oncology
Background:
- Proton beam therapy (PBT) is increasingly used for pediatric radiotherapy.
- Treating infants with central nervous system (CNS) tumors presents unique challenges.
- PBT requires intensive multimodality care and significant logistical planning.
Purpose of the Study:
- To evaluate the feasibility and outcomes of PBT in infants with CNS tumors.
- To analyze treatment characteristics, toxicities, and survival rates.
- To assess short-term adverse events associated with PBT in this population.
Main Methods:
- Retrospective analysis of infants (<2 years) with CNS tumors treated with PBT.
- Data collected from the prospective registry study KiProReg.
- Adverse events classified using CTCAE V4.0 criteria.
Main Results:
- 51 infants received PBT for tumors including ependymoma, atypical teratoid rhabdoid tumors, and gliomas.
- Estimated 3-year survival rates: local control 62.7%, progression-free survival 47.1%, overall survival 76.5%.
- Higher-grade toxicities occurred in 24 events; late effects included endocrinopathy and hearing/visual impairment.
Conclusions:
- PBT is a feasible short-term option for infants with CNS tumors.
- Treatment necessitates complex interdisciplinary care and logistics.
- Longer follow-up is crucial to evaluate late effects and neurocognitive outcomes.
Aims:
Proton beam therapy (PBT) has increasingly been applied for the treatment of young children when radiotherapy is needed. The treatment requires intensive multimodality care and is logistically demanding. In this analysis, we evaluated our experiences in treating infants with tumours of the central nervous system with PBT.
Materials And Methods:
Children younger than 2 years of age treated with PBT for central nervous system tumours enrolled in the prospective registry study KiProReg were retrospectively analysed. Information on patient characteristics, treatment, toxicities and outcome were evaluated. Adverse events were classified according to the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE V4.0) before, during and after PBT.
Results:
Between September 2013 and June 2018, 51 infants were eligible. The median age was 19 months (range 11-23 months) at the time of PBT. Tumour entities were ependymoma (51.0%), atypical teratoid rhabdoid tumour (39.0%), high-grade glioma (6.0%), pineoblastoma (2.0%) and medulloblastoma (2.0%). The prescribed median total dose was 54.0 Gy (range 45.0-59.4 Gy). Most received local radiotherapy. In four patients, craniospinal irradiation followed by a boost to the local tumour bed was applied. The median follow-up time was 42.0 months (range 7.3-86.2 months). The estimated 3-year local control, progression-free survival and overall survival rates for all patients were 62.7, 47.1 and 76.5%, respectively. During radiotherapy, 24 events of higher-grade (CTCAE ≥ °III) toxicities were reported. Interruption of radiotherapy for more than 2 days was due to infection (n = 3) or shunt complication (n = 2). Unexpected hospitalisation during radiotherapy affected 12 patients. Late adverse events attributable to radiotherapy included endocrinopathy (CTCAE °II; 7.8%), new onset of hearing loss (CTCAE °III; 5.8%) and visual impairment (CTCAE °IV; 1.9%). Transient radiation-induced imaging changes occurred in five patients (9.8%).
Conclusions:
Our study indicates that PBT is feasible for very young children with central nervous system tumours, at least in the short term. However, it requires challenging interdisciplinary medical care and high logistical effort. For evaluation of late effects, longer follow-up and evaluation of neurocognitive outcome are desirable. More data have to be gathered to further define the role of radiotherapy in infants over time.
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