Objective: To collect clinical and treatment outcome data in a large patient cohort, and specifically to report experience with temozolomide (TMZ).
Design: Cohort study based on an electronic survey open for participation to ESE members Dec 2015-Nov 2016.
Results: Reports on 167 patients, 40 pituitary carcinomas, and 127 aggressive pituitary tumours were obtained. Median age at diagnosis was 43 (range 479) years. 59% of tumours were clinically functioning at presentation, and the most frequent were corticotroph tumours. TMZ was the first line chemotherapy in 157 patients. At the end of TMZ treatment (mean 9.92 cycles) radiological evaluation showed complete response in 6%, partial response in 31%, stable disease in 33% and progressive disease in 30%. Progression occurred more often in pituitary carcinomas (40%) than in aggressive adenomas (26%), P=0.05. Clinically silent tumours showed less regression compared with secreting tumours, 17 vs 45%, P=0.01 (overall χ2 test). Median follow-up after TMZ treatment was 21 months. Of patients with complete response, partial response and stable disease 22, 34 and 40% respectively showed progression during further follow-up. Twenty five patients received a second course of TMZ, 3 had a partial response. Overall mortality was 33%, and highest in patients with progression after TMZ treatment (54%).
Conclusion: TMZ was accompanied by tumour regression in 37% of patients, documenting its value in the management of these aggressive tumours. The high recurrence rate following TMZ cessation highlights the need to identify additional effective therapies.
20 - 23 May 2017
European Society of Endocrinology