Open Access

Background: Radiotherapy (RT) involving craniospinal irradiation (CSI) is important in the initial treatment of medulloblastoma. At recurrence, the re-irradiation options are limited and associated with severe side-effects. Methods: For pre-irradiated patients, patients with re-irradiation (RT2) were matched by sex, histology, time to recurrence, disease status and treatment at recurrence to patients without RT2. Results: A total of 42 pre-irradiated patients with RT2 were matched to 42 pre-irradiated controls without RT2. RT2 improved the median PFS [21.0 (CI: 15.7–28.7) vs. 12.0 (CI: 8.1–21.0) months] and OS [31.5 (CI: 27.6–64.8) vs. 20.0 (CI: 14.0–36.7) months]. Concerning long-term survival after ten years, RT2 only lead to small improvements in OS [8% (CI: 1.4–45.3) vs. 0%]. RT2 improved survival most without (re)-resection [PFS: 17.5 (CI: 9.7–41.5) vs. 8.0 (CI: 6.6–12.2)/OS: 31.5 (CI: 27.6–NA) vs. 13.3 (CI: 8.1–20.1) months]. In the RT-naïve patients, CSI at recurrence improved their median PFS [25.0 (CI: 16.8–60.6) vs. 6.6 (CI: 1.5–NA) months] and OS [40.2 (CI: 18.7–NA) vs. 12.4 (CI: 4.4–NA) months]. Conclusions: RT2 could improve the median survival in a matched cohort but offered little benefit regarding long-term survival. In RT-naïve patients, CSI greatly improved their median and long-term survival.

Background Craniopharyngiomas (CPs) are rare embryonic tumors. Clinical presentation and outcome of patients perinatally diagnosed with congenital CP (cCP) are not clear and refer mainly to a few case reports in the literature. The aim of this study was to analyze clinical presentation and outcome in patients with cCP. Study design Three hundred and sixty-one patients diagnosed with adamantinomatous CP were recruited 2007–2022 in KRANIOPHARYNGEOM 2007/Registry 2019 and prospectively observed. In two cases, cCP was diagnosed prenatally and in one case on the second day of life. Pre- and perinatal diagnostic findings, postnatal evaluation, and therapeutic interventions and outcome in these three cases of cCP were analyzed. Results All patients survived. One patient developed psychomotor retardation and a mild hemiparesis. Prenatal routine ultrasound examination led to the diagnosis of cCP. Tumor resection was performed during the early postnatal period (range: 11–51 days of age). Functional capacity, measured by Fertigkeitenskala-Münster-Heidelberg (FMH) was reduced in three and behavioral parameters, measured by the Strength and Difficulties Questionnaire (SDQ) were abnormal in two cases. Conclusion cCP is a rare diagnosis with a prevalence of 0.83% in our study group. Compared to cases reported in the literature, the presented cases were treated immediately and had a better prognosis. Based on improvements of diagnostic and therapeutic techniques, prenatal diagnosis of cCP should lead to transfer prior to delivery of cCP patients to a specialized center for delivery and postnatal treatment of newborns with sellar masses by a multidisciplinary team to secure the improved prognosis of these patients. Significance statement We previously reported that lower event-free survival rates after craniopharyngioma are associated with younger age at diagnosis. Perinatally diagnosed congenital craniopharyngiomas are very rare. This article presents three unique cases with congenital craniopharyngioma, comparing their diagnostics, therapy, and development. All three cases had surgery during the early postnatal period with sparing of the posterior hypothalamus. In each case, endocrinopathy was present at follow-up. Low functional capacity was reported in all cases and an abnormal total difficulties score in two cases. Compared to the literature, the presented cases had better prognosis in morbidity and mortality. This report and the review of the literature confirm the importance of a multidisciplinary approach in the diagnostic and treatment of the very rare condition of congenital craniopharyngioma.

Aims Our objective was to analyse tumour- and treatment-related factors influencing endocrine morbidity and obesity pre- and post-proton beam therapy (PBT) in paediatric patients with craniopharyngioma. Materials and methods A total of 65 patients at the onset of PBT were included in the analysis within our prospective registry study. The data pertaining to endocrine deficits and BMI prior to PBT were retrieved from the medical records on a retrospective basis. Cumulative incidences (CI) of endocrinopathies, age- and sex-adjusted BMI standard deviation scores (BMI-SDS) were calculated. Results Before PBT, 90.8% had ≥1 neuroendocrine deficit. Diabetes insipidus (DI) was attributed to surgery in 96%. Patients with postoperative DI had a higher 3-year CI of adrenocorticotropic hormone and thyroid-stimulating hormone deficiency rates compared to those without DI (p < .001). At PBT start, 47.7% had already panhypopituitarism compared to 67.7% at the last follow-up (FU). Median FU post-PBT was 3.2 years (range, 1.0–9.6). Post-PBT, 38.2% remained free of additional hormone deficiencies. A trend towards lower endocrine morbidity scores for patients who received PBT during their primary treatment compared to irradiation at progression did not reach statistical significance (p = .068). The BMI-SDS increase from diagnosis to the start of radiotherapy was significantly greater than from the start of PBT to the end of FU (mean BMI-SDS increase: 0.61, ±1.16 vs. 0.13, ±0.84, p = 0.019), with a median time of 10.2 and 38.4 months, respectively. In the multivariate analysis, hypothalamic involvement (p = .042) and the BMI-SDS level at diagnosis (p = .006) were identified as clinical factors indicating severe obesity at FU (BMI-SDS ≥+2). Conclusions Panhypopituitarism is frequently observed in paediatric patients with craniopharyngioma prior to PBT. The potential benefits of early PBT on endocrine outcomes require further investigation through longer FU periods. The greatest increase in weight occurred before radiotherapy. Endocrine deficiencies and weight gain are multifactorial and require close monitoring.