Abstract 7348: Long-term outcomes of pediatric craniopharyngiomas: multi-center comparative study

Abstract Background: Craniopharyngioma (CP) account for 80% of childhood hypothalamo-pituitary tumors and, though benign, their proximity to the visual pathways, hypothalamus, and pituitary incurs significant long-term neurologic and endocrine morbidity. Informed surgical decision-making has the potential to reduce morbidity and improve outcomes. There remains a high degree of variability in how these lesions continue to be managed. Methods: This multicenter retrospective observational study aimed to compare long-term outcomes of CP at two pediatric neurosurgery units (GOSH, UK, and USP, Brazil), delineating management paradigms associated with better prognosis. CP treated over a 20-year period were identified and standardized clinical outcomes recorded for comparative analysis. Results: 123 patients were identified (USP=52; GOSH=71). No demographic differences were identified between series (mean age: USP=9.2±4.6; GOSH=8.2±4.1 years). Headaches (USP=75%; GOSH=62%) and visual deficits (USP=46%; GOSH=38%) were the most common presenting symptoms. Growth hormone deficiency at presentation was more common in the USP cohort (USP=62.5%; GOSH=35%; p=0.009). There was significantly more hypothalamic invasion in the GOSH cohort (Paris grade 2: GOSH=67.0%; USP=37.5%; p=0.026), but significantly larger cystic volumes in the USP cohort (USP=27.5±37cm3; GOSH=10.5±8.9cm3; p=0.004). These differences led to different surgical approaches, with more subtotal resections at USP (78%) and more cyst aspirations at GOSH (62%). 80% of patients at GOSH received RT compared to 32% at USP. Mean follow-up at GOSH was 7.5±4.8 years compared to 10.1±5.6 years at USP. Cognitive outcome did not vary. GOSH cohort presented higher rates of levothyroxine replacement (USP=67%; GOSH=86%; p=0.017). GOSH exhibited better hypothalamic outcomes (p=0.08) and better visual outcomes (field deficits: GOSH=44%; USP=66%; p=0.01). In multivariate analysis (n=123), subtotal/gross-total resection (OR=0.17; 95%CI:0.05-0.55, p<0.01) and older age at diagnosis (OR=0.91, 95%CI:0.82-0.99, p=0.04) were associated with longer progression-free survival. Conclusions: Comparative analysis of two large craniopharyngioma series identified different paradigms of surgical management driven by clinical-radiological presentation with similar long-term outcomes. Subtotal/gross-total resection was not related to worse clinical outcome or better progression-free survival. Citation Format: Vitor Nagai Yamaki, Jai Sidpra, Bruno Santana Peres, Catuto Domingos Quianga, Guilherme Jose Borsatto, Joao Paulo Telles, Ines Nobrega Silva, Asthik Biswas, Kshitij Mankad, Hani Marcus, Noor ul Owase Jeelani, Sueli Mieko Oba-Shinjo, Hamilton Matushita, Suely Kazue Marie, Kristian Aquilina. Long-term outcomes of pediatric craniopharyngiomas: multi-center comparative study [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2025; Part 1 (Regular Abstracts); 2025 Apr 25-30; Chicago, IL. Philadelphia (PA): AACR; Cancer Res 2025;85(8_Suppl_1):Abstract nr 7348.