Comparative effectiveness of treatment options for pediatric craniopharyngiomas

Abstract

Object: No clear treatment guidelines for pediatric craniopharyngiomas exist. The authors developed a decision analytical model to evaluate outcomes of 4 surgical approaches for craniopharyngiomas in children, including attempted gross-total resection (GTR), planned subtotal removal plus radiotherapy, biopsy plus radiotherapy, and endoscopic resections of all kinds.

Methods: Pooled data, including the authors' own experience, were used to create evidence tables, from which incidence, relative risks, and summary outcomes in quality-adjusted life years (QALYs) were calculated for the 4 management strategies.

Results: Quality-adjusted life years at the 5-year follow-up were 2.3 ± 0.1 for attempted GTR, 2.9 ± 0.2 for planned subtotal removal plus radiotherapy, 3.9 ± 0.2 for biopsy plus radiotherapy, and 3.7 ± 0.2 for endoscopic resection (F = 17,150, p < 0.001). Similarly, QALYs at 10-year follow-up were 4.5 ± 0.2 for attempted GTR, 5.7 ± 0.5 for planned subtotal removal plus radiotherapy, and 7.8 ± 0.5 for biopsy plus radiotherapy (F = 6,173, p < 0.001). On post hoc pairwise comparisons, the differences between all pairs compared were also highly significant (p < 0.001). Since follow-up data at 10 years are lacking for endoscopic cases, this category was excluded from 10-year comparisons.

Conclusions: Biopsy with subsequent radiotherapy is the preferred approach with respect to improved overall quality of life. While endoscopic approaches also show promise in preserving quality of life at five-year follow-up, there are not sufficient data to draw conclusions about this comparison at 10 years.