Background: Paediatric HPAT, though generally benign, incurs significant neuro-endocrine morbidity. Their management is unclear and the paediatric neuro-oncology or adult pituitary forum at which they are discussed lack pituitary or age-specific expertise respectively. The UK National HPAT Interest Group has pioneered a monthly, multi-site, interdisciplinary, video conferencing decision-making forum, to garner necessary experience and evidence of outcomes to assist worldwide referrers in management of childhood neuro-endocrine tumours. It delivers educational lectures, promotes research, and publishes evidence based (AGREE11) national guidance on idiopathic thickened pituitary stalk (TPS), craniopharyngioma, pituitary adenomas, and continuously audits evidence since 2010.
Patients: 182 patients were discussed on 256 occasions (46 repeated reviews) over 7 years from UK, Europe, Hong Kong, Canada and Australia. It receives referrals from eight London hospitals, seven7 UK paediatric oncology centres and occasionally six international centres. Paediatric and adult pituitary, surgical, neurooncology and neuroradiological specialists contribute.
Results: Craniopharyngiomas (47), pituitary adenomas (45) and TPS (36) constituted 70% of the cases. In craniopharyngiomas the commonest presenting features were growth failure, visual and intracranial pressure symptoms, while panhypopituitarism occurred in 78% of cases post treatment. Prolactinomas (23) accounted for 51% of the adenomas- often macro/giant/or resistant (56.5%)-presenting with pubertal delay, amenorrhoea, galactorrhoea, and neurological symptoms in one third of the patients. ACTH (3), GH (1), TSH (1), and dual producing adenomas (3.8%) occurred less frequently. Idiopathic TPS was most usually associated with cranial diabetes insipidus (72%). Referring institutions requested investigation and management guidance (44.5%) for surgical (e.g. biopsy in idiopathic TPS) and medical (e.g. radiation or cabergoline) treatment indications (22.5%) and initiation of GH replacement (6.0%). In 19.2% the MDT discussed complex surgical options (e.g. infant hypothalamic craniopharyngioma) and facilitated regional transfer for specialist endoscopic transsphenoidal surgery unavailable at referring centre (1 Cushing disease, 2 craniopharyngioma). Finally, clinical surveillance plans were agreed in 35% and discharge facilitated in 4% cases.
Conclusion: A supra-regional childhood HPAT discussion forum is clearly welcomed and continues to expand. It facilitated centralised clinical decision-making, patient referral and outcome audit, and raised awareness of rare disabling diseases. Our evidence regarding benefit suggests there is a demand for this expert advisory body to be formally recognised as a model of care, which might be adopted in a wider European research network (ERN) thereby sharing knowledge and facilitating research in these rare and often aggressive pituitary tumours.
27 - 29 Sep 2018
European Society for Paediatric Endocrinology