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.