Abstract Background Pediatric low-grade gliomas (pLGG) are the most common childhood CNS tumors. Recent advances in molecular profiling have reshaped treatment paradigms, enabling increasing use of targeted therapies (TT). We conducted a retrospective, single-center observational study to systematically evaluate the impact of molecularly TT compared with conventional chemotherapy (CT) in children with pLGG. Methods We reviewed all pLGG diagnoses managed at Bambino Gesù Children’s Hospital (Rome) between 2011 and 2025. Among 311 diagnosed patients, 95 who received at least one systemic treatment line (CT and/or TT) and had adequate clinical, radiological, and therapeutic documentation were included. Patients were stratified by systemic treatment received: CT only (n = 18), TT only (n = 38), or combined/sequential CT+TT (n = 39). Survival outcomes were estimated with Kaplan–Meier methods and compared using log-rank tests. A multivariable Cox model assessed factors associated with progression-free survival (PFS). Results Median age at diagnosis was 4.6 years; 52% were male. Five-year OS for the whole cohort was 97.4%. PFS at 1, 2, and 5 years was 87.4%, 73.7%, and 43.6%, respectively. OS did not differ significantly across treatment groups. Median PFS was longer with TT (5.53 vs 2.95 years). In multivariable Cox regression, the only factor independently associated with a longer PFS was first-line TT (HR for CT compared with TT: 2.63; 95% CI 1.50–4.62; p 0.001). Tumor site, histology, molecular class, and extent of surgery were not significantly associated with PFS in our experience. Targeted therapy showed a favorable safety profile: treatment-related toxicity was generally low and clinically manageable, with most adverse events being grade 1–2. Conclusions In this real-world pLGG cohort, survival was excellent. Targeted therapy was associated with a significantly lower risk of progression in multivariable analysis, supporting its growing role as an effective systemic strategy in molecularly characterized pLGG.
Baldo et al. (Tue,) studied this question.
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