Childhood Craniopharyngioma – Changes of treatment strategies in the multinational prospective trials KRANIOPHARYNGEOM 2000/2007

Background: Despite high survival rates in childhood craniopharyngioma, prognosis is frequently impaired due to sequelae. Radical surgery was the treatment of choice for several decades. However, even at experienced surgical facilities radical surgery can result in hypothalamic disorders such as severe obesity. Materials and methods: We compared the grade of pre-surgical hypothalamic involvement, intention-to-treat, degree of resection and grade of surgical hypothalamic lesions between patients recruited in KRANIOPHARYNGEOM 2000 (n = 120; 2001 – 2007) and KRANIOPHARYNGEOM 2007 (n = 107; 2007 – 2013). Results: The grade of initial hypothalamic involvement was similar in patients treated 2001 – 2007 and 2007 – 2013. The intention-to-treat was more radical (p = 0.01) in patients recruited 2001 – 2007 (38%) when compared with patients treated 2007 – 2013 (18%). In patients with pre-surgical involvement of anterior/posterior hypothalamic areas, the rate of hypothalamus-sparing operations resulting in no (further) hypothalamic lesions was higher (p = 0.005) in patients treated 2007 – 2013 (35%) in comparison with the 2001 – 2007 cohort (13%). Event-free-survival rates were similar in both cohorts. Conclusions: A trend towards less radical surgical approaches is observed, which resulted in a reduced rate of severe hypothalamic lesions. Event-free survival was not compromised by this development. Radical surgery is not an appropriate treatment strategy in patients with hypothalamic involvement. Treatment should be confined to experienced multidisciplinary teams.