Objective: Some patients with paediatric craniopharyngiomas (PCs) showed normal growth despite growth hormone deficiency, which is known as growth without GH (GWGH); however, its mechanism remains unclear. We aimed to develop a novel clinical score to predict the probability of GWGH in PCs.
Methods: A total of 708 PC patients were prospectively enrolled from six hospitals, among which 431 patients were finally included. Data from four of the six hospitals (n = 325) were used to develop the innovative clinical score (ICS), which was further validated using the data from the other two hospitals (n = 106). To establish and validate the ICS, sequential logistic regression was used to analyse the clinical characteristics including tumour growth pattern and tumour size and so on. Furthermore, C-statistic was employed to calibrate the discriminatory ability of the established clinical score, while a calibration plot was adopted for further assessment.
Results: The overall incidence of GWGH was 16.9% (73/431). The ICS ranged from 2 to 23, with an optimism-corrected C-statistic of 0.820, Furthermore, the optimism-corrected C-statistic of external validation was 0.835, indicating good discriminatory power and robustness of the clinical score. Additionally, no apparent overestimation or underestimation was observed in the calibration plots, which showed excellent calibration power of the clinical score.
Conclusions: Based on tumour growth patterns and PC patients' clinical characteristics, individualized surgical strategies were promising to achieve long-term effective management of PC patients. The ICS is valuable for the evaluation of probability of developing postoperative GWGH.
Clinical trial registration: ClinicalTrials.gov ID: NCT00949156.
Keywords: Growth without growth hormone; Paediatric craniopharyngiomas; Tumour growth pattern.