Background: Childhood pineal and/or suprasellar intracranial germ cell tumours (IGCT) are highly curable (>90%) with neuraxial radiation alone; international (SIOP) trials have aimed to decrease late radiation-induced neuroendocrine morbidity by substituting chemotherapy. However, without longitudinal study, disease and treatment contributions to long term outcomes remain unknown.
Objective: To define tumour and treatment factors implicated in neuroendocrine morbidity across 2 patient cohorts: Group A (n=20) treated with neuraxial radiation alone; and Group B (n=26) with adjuvant chemotherapy/reduced field radiation between 1.1.83 and 31.12.14.
Method: Retrospective longitudinal case note review of all confirmed IGCT cases. Nonparametric paired analyses and Kaplan Meier statistics were used to compare Endocrine Morbidity scores (EMS) and longitudinal evolution of pituitary endocrinopathies (EEFS), correlated with tumour location tumour 3D-volume (n=28) with TK-SNAPv3.2.0 software and treatment type.
Results: 46(26 Male) patients aged median(range) 10.4(5.117.6) years presented with symptom duration of 0.4(02.5) year and were followed for 6.5(0.724.4) years. The 19 with pineal (P) tumours tended to present quicker (0.4±0.7 vs 0.9±1.3 months; pNS) and had fewer pituitary deficits (P<0.0001) than the 16 suprasellar (S) tumours, despite smaller volume disease (PvsS:1.2 cm3 (0.211) vs 4.6 cm3 (1.013.9), pNS). 37 had surgery (10 only biopsy). 18/24 suprasellar cases (of whom 5 were bifocal (B)) presented with DI, and 2 others were pineal with hypothalamic involvement (P<0.0001). A further 6 (4 suprasellar) developed DI after surgery. Neither surgery nor treatment type (GpAvsGpB) reduced pituitary deficits, visual or educational outcomes, or BMIsds increase, though renal (n=2), hearing (n=2) and cardiac (n=1) toxicity was only observed in chemotherapy (GpB), despite shorter follow up (126.96.36.199) vs 7.4(2.812.7) years, P=0.013).
Conclusion: In patients with IGCT, tumour location dictates symptom duration and ultimate endocrinopathies. The latter are frequent (89%), multiple (69%), especially in suprasellar disease (P<0.0001) but are not influenced by treatment. Hence substituting chemotherapy for neuraxial radiation is unlikely to improve these outcomes, newer treatments may well add peripheral toxicity.
10 - 12 Sep 2016
European Society for Paediatric Endocrinology