Long-term outcomes of paediatric craniopharyngiomas: a comparison of two large international series

PURPOSE: Adamantinomatous craniopharyngioma (ACP) is an uncommon and anatomically variable tumor in children. The balance between critical treatment objectives, including the pursuit of gross total surgical resection and the reduction of hypothalamic injury and tumor recurrence, remains difficult and controversial. In this retrospective observational study, we compare the management and outcome of ACP in two large paediatric neurosurgical centres to determine how variations in tumor characteristics and management influence long-term outcomes. METHODS: In this retrospective observational study, consecutive children (aged ≤ 18 years) diagnosed with primary ACP between 1997 and 2023 at two tertiary paediatric neurosurgical centres (Great Ormond Street Hospital (GOSH), United Kingdom, and Universidade de São Paulo (USP), Brazil) were evaluated. Functional outcomes related to pituitary function, hypothalamic injury, cognition, and vision were analysed. Scans were independently reviewed for tumor size, characteristics, and relationship to the optic apparatus and hypothalamus. Extent of surgery was documented. RESULTS: 123 patients (USP = 52; GOSH = 71) were included. Mean follow-up was 10.1 ± 5.6 years at USP and 7.5 ± 4.8 years at GOSH. There were no demographic differences. Rates of growth hormone deficiency (USP = 62.5%; GOSH = 35%; p = 0.009) and visual deficits (p < 0.05) were higher in the USP cohort at initial presentation. Patients at GOSH presented with predominantly cystic lesions (n = 46/60; 76.7%) while at USP solid tumors (n = 24/32; 75%) (p = 0.05) were more prevalent. Paris grade for hypothalamic involvement was higher in the GOSH cohort (p = 0.01). Although gross total resection (GTR) was similar in the two groups, cyst aspiration plus radiotherapy was commoner at GOSH, whereas debulking was commoner at USP (p < 0.001). Post-treatment hypothalamic scores were better in the GOSH cohort (p < 0.04). Patients at GOSH had earlier recurrences (25.5 ± 32 months) compared to patients treated at USP (37.4 ± 59.5 months) (p = 0.01). In multivariate analysis, STR/GTR (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. CONCLUSION: Comparative analysis of two large series of ACP in children identified different paradigms of surgical management driven by distinct clinical-radiological presentations. While cyst aspiration plus radiotherapy protects against hypothalamic injury, this may occur at the cost of earlier tumour recurrence. Further studies to define this balance are urgently needed. PREVIOUS PRESENTATIONS: 21st International Symposium on Pediatric Neuro-Oncology. June 29-July 2, 2024.