Analyses of Treatment Variables for Patients with Childhood Craniopharyngioma – Results of the Multicenter Prospective Trial KRANIOPHARYNGEOM 2000 after Three Years of Follow-Up

Abstract

Background: Controversies surround various treatment variables for patients with childhood craniopharyngioma such as growth hormone (GH) replacement, which some believe can exacerbate recurrence/progression. We prospectively assessed the risk of tumor recurrence/progression in survivors of childhood craniopharyngioma. Methods: Multivariable analyses of risk factors (age at diagnosis, degree of resection, irradiation, GH treatment and gender) and descriptive analyses of overall survival (OS) and event-free survival (EFS) rates were performed in 117 patients, recruited prospectively and evaluated after 3 years of follow-up in the German, Austrian and Swiss multicenter trial KRANIOPHARYNGEOM 2000. Results: We observed a 3-year OS of 0.97 and a 3-year EFS of 0.46, indicating high recurrence rates after complete resection (CR) (n = 47; 3-year-EFS: 0.64) and high progression rates after incomplete resection (IR) (n = 64; 3-year EFS: 0.31). The risk of an event decreased by 80% after CR compared to IR (hazard ratio = 0.20; p < 0.001). Irradiation had protective effects on EFS: irradiated patients had an 88% lower risk of recurrence/progression compared to patients without/before irradiation (hazard ratio = 0.12; p < 0.001). GH treatment had no impact on 3-year EFS rates. Conclusions: Tumor recurrences/progressions are frequent and occur early after initial treatment of childhood craniopharyngioma. A radical resection preserving the integrity of hypothalamic structures appears optimal at original diagnosis. Irradiation was efficient in preventing recurrences/progressions. GH treatment had no impact on the low 3-year EFS observed in our study. However, further conclusions on the influence of GH on recurrence rates have to be refined to long-term follow-up studies of patients with childhood craniopharyngioma.

Tumour recurrence and enlargement in patients with craniopharyngioma with and without GH replacement therapy during more than 10 years of follow-up

“…The study had, however, a large difference in the frequency of initial RT between the GH-treated and non-GH-treated patients. In a recent study from Mü ller et al (36), there was no difference in tumour progression between 54 CP patients treated with GHRT and 60 CP patients without GHRT after a mean follow-up of 2.8 years. In the current study, after adjustment for initial RT, residual tumour after primary treatment and gender, the 10-year PFSRs were 85% for the GHRT-treated patients and 65% for patients not treated with GHRT.…”

Post-operative hypothalamic lesions and obesity in childhood craniopharyngioma: results of the multinational prospective trial KRANIOPHARYNGEOM 2000 after 3-year follow-up

“…Between 2001 and 2007, 120 German, Austrian and Swiss patients (58f/62m) with an original diagnosis of craniopharyngioma were recruited from 76 centres at a median age of 10.0 years (range: 1.2-18.0 years) (19). With a high degree of completeness (O90%), data on neurosurgery and imaging were collected.…”

“…Based on the protocol of the prospective surveillance trial KRANIOPHARYNGEOM 2000 (19), there was no stratified or randomised question in terms of neurosurgical strategies. A surgical strategy preserving the integrity of optic and hypothalamic structures was recommended.…”

“…Cranial magnetic resonance imaging (MRI) was prospectively performed according to the recommendations as part of the trial protocol KRANIOPHARYNGEOM 2000 (19,22) at diagnosis and at 3-month intervals during follow-up. The assessment of progression or recurrence was based on MRI and prospectively confirmed by reference panel in all cases.…”

“…This study presents the results of the first prospective multinational trial on childhood craniopharyngioma patients (KRANIOPHARYNGEOM 2000) (19), analysing the impact of tumour localisation, neurosurgical treatment strategies and extent of post-operative lesions on changes in QoL and body mass index (BMI) as a measure of obesity. A novel grading system for hypothalamic involvement/lesions was established and used to identify patients at risk for severe hypothalamic obesity and long-term impairment of QoL.…”