For the brain tumor committee of SFCE (Société Française des Cancers de lEnfant).
Objective: Therapeutic approach favors chemotherapy as the first-line-treatment in progressing OPG. There are few data on long term endocrine outcomes of aggressive OPG treated by upfront chemotherapy. Our main objective was to describe the long-term endocrine sequelae in these patients and to identify potential early predictors of the endocrine involvement.
Subjects and methods: Children diagnosed with OPG at an age younger than 16 years from the French multicentric BBSFOP registry were included. They were treated with upfront chemotherapy according to the BB-SFOP protocol in France between June 1990 and December 2004, and subsequent treatment (second-line chemotherapy, surgery, radiotherapy) was used depending on tumor progression. They underwent a late evaluation with clinical and biological assessment between January 2011 and March 2016.
Results: One hundred and two patients were included in our study. The mean age at tumor diagnosis was 3.3±0.3 years. The mean time of follow-up was 13.9±3.7 years. A history of precocious puberty was present in 36% of the subjects. At least one endocrine deficiency was present in 93% of the subjects (GHD 74%, TSH deficiency 57%, ACTH deficiency 36%, hypogonadotropism 33%, gonadic deficiency 30%, diabetes insipidus 15%; inappropriate AVP secretion 7%). 37% of males and 39% of females were overweight or obese. Mean adult height, reached in 51 subjects, was −1.2±1.3 SDS in males, and −0.7±1.4 SDS in females. Chemotherapy only was protective from pituitary deficiencies (odds ratio 0.19 to 0.37, P < 0.05). NF1 was protective from TSH and ACTH deficiencies (odds ratio 0.25 to 0.35, P < 0.05). Tumor volume on diagnostic MRI was not predictive of pituitary deficiencies. Gonadic deficiency was significantly more frequent in males than females (46,5% vs 12.2%, P < 0.05), and associated with chemotherapy only (OR 3.2, P < 0.05) and NF1 (OR 4,8, P < 0.05). Overweight/Obesity was associated with ACTH deficiency (OR 5, P < 0.05).
Conclusion: Obesity and late endocrine dysfunction were frequent in subjects treated by upfront chemotherapy for aggressive OPG during childhood. However, chemotherapy only, when possible, was protective from pituitary involvement.
27 - 29 Sep 2018
European Society for Paediatric Endocrinology