Open Access

M. Bischoff, J. Beckhaus, D. A. Khalil, F. Sen, S. Frisch, B. Koska, C. Kiewert, Brigitte Bison, R.-D. Kortmann, C. Friedrich, H. L. Müller, B. Timmermann

• 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 FUAims 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.…