High-precision radiotherapy achieves excellent long-term control and preserves function in pediatric craniopharyngioma-Subset analysis of a randomized trial.

Background: The evolving treatment paradigm in children and adolescents with craniopharyngioma (CP) aims at minimizing late functional sequelae. Advanced radiotherapeutic techniques offer theoretical advantages of preserving neurological functions; however, clinical evidence of such is limited. The current study constitutes a secondary analysis of CP patients in a Randomized Control Trial testing Conventional RT (ConvRT) versus Stereotactic Conformal Radiotherapy (SCRT; NCT00517959).

Methods: Eighty-two patients of CP (SCRT: 39, ConvRT: 43, Dose: 54 Gy in 30 fractions) were analyzed, assessing the clinical impact of dosimetric sparing on neurocognitive function, endocrine function, overall survival (OS) and local control (LC). Patients were longitudinally assessed from baseline through 5 years post-treatment using the Weschler Intelligence Score Chart/WAIS scales.

Results: The median age was 13 years (IQR = 9-17 years). The 10-year OS and LC rates were 86.4% and 92.7%, respectively, with no significant difference between the arms. SCRT patients showed significant improvement in mean full-scale IQ (difference in slope = 3.3 points per year, P = .01) and performance quotient (difference in slope = 3.6, P = .04) compared to those treated with ConvRT. Freedom from cognitive decline (a 5-point drop) at 5 years was higher with SCRT (66.6% vs. 38.2%; HR = 0.41, P = .03). Younger age (<15 years) was a significant negative predictor of neurocognitive outcomes (P = .002). SCRT patients also experienced a lower cumulative incidence of new neuroendocrine dysfunction (25.7% vs. 48.8%, P = .029).

Conclusions: SCRT offers excellent tumor control and similar survival with superior long-term preservation of neurocognitive and endocrine functions in CP patients compared to conventional RT. High-precision RT should constitute the standard of care in CP.