Outcomes of Proton Beam Therapy in Infants and Very Young Children.
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PURPOSE: Radiation has historically been deferred or avoided in infants and very young children because of concern about late toxicity. Although proton beam therapy (PBT) is increasingly used in this age group, outcome data remain limited. We evaluated disease control, patterns of failure, and late toxicity in children aged ≤3 years treated through the UK national proton pathway. METHODS AND MATERIALS: We retrospectively reviewed 76 consecutive children aged ≤3 years at PBT initiation who were treated at XXXX (anonymized) between December 2018 and January 2025. Diagnoses included central nervous system (CNS) tumors (n=33; ependymoma, 20; embryonal tumors, 12; other, 1) and non-CNS tumors (n=43; sarcoma, 20; neuroblastoma, 12; renal tumors, 11). All patients received daily image-guided, optimized pencil-beam-scanning PBT under general anesthesia. Overall survival (OS), progression-free survival (PFS), local control (LC), and Common Terminology Criteria for Adverse Events (CTCAE) grade ≥3 late toxicity-free survival were estimated by the Kaplan-Meier method. RESULTS: Median age at PBT start was 28.1 months (range, 9.4-36.0 months). Treatment was completed as planned in 72 of 76 patients (94.7%); 23.7% were hospitalized and 38.2% required enteral feeding support during PBT. With a median follow-up of 2.9 years, 1-, 2-, and 3-year OS were 87.9%, 85.1%, and 81.1%, respectively; 2-year PFS and LC were 65.5% and 85.2%. All 9 local failures (12.0%) occurred within the high-dose target volume. High-grade late toxicity occurred in 11 patients (14.9%); 2-year high-grade toxicity-free survival was 90.4%. One treatment-related death occurred from brainstem radionecrosis despite meeting accepted brainstem constraints. CONCLUSIONS: PBT is deliverable in children aged ≤3 years within a national pathway. However, all local failures occurred within the high-dose target volume, high-grade late toxicity affected 14.9% over a short follow-up, and one treatment-related death occurred despite adherence to accepted constraints. These findings support biology-guided patient selection, age-adapted organ-at-risk objectives and structured long-term surveillance.