The Craniopharyngioma Working Group consists of a multidisciplinary team with members (>150) coming from a wide range of disciplines, including neurosurgery, paediatric neuro-oncology, radiation oncology, endocrinology, neurology, pathology, psychology and neuropsychology with the collaborative aim of optimising high-quality, long-term survival for children and young people with this rare tumour. In line with this, co-chairs of this working group always come from two different disciplines. The group meets annually at the SIOPE BTG Annual Meeting, and quarterly by Teams to discuss complex cases.

For many years the mainstay of treatment for craniopharyngiomas has been neurosurgery and radiotherapy (more recently proton beam therapy), neither of which are usually curative. Therefore, unlike many other brain tumours, craniopharyngiomas are essentially a chronic disease, and hypothalamo-pituitary dysfunction remains a significant, debilitating morbidity leading to poor quality of survival, despite overall survival being high. This group therefore has two primary objectives – (1) to help develop novel therapeutic approaches to lead to better quality long-term survival and (2) to optimise management of long-term co-morbidities faced by survivors (such as hypothalamo-pituitary dysfunction). Of note, the group will shortly be publishing the first European Standards of Clinical Practice Recommendations for Paediatric Craniopharyngioma.

Newer molecular therapies are in trials via the CONNECT and PNOC consortia, including tocilizumab (IL-6 receptor antagonist), binimetinib (MEK inhibitor) and tovorafenib (pan-RAF inhibitor) which will hopefully improve survival and quality of survival for these patients. Unfortunately none of these trials are open within Europe and this group therefore aims to increase the rates of participation of member countries in future international studies.

Until recently, there have been no efficacious treatments for the management of many aspects of hypothalamo-pituitary dysfunction, particularly hypothalamic obesity. Recently, setmelanotide, an MC4R agonist already licensed for treatment of other genetic forms of obesity, has been found to be effective in phase III trials for the treatment of acquired hypothalamic obesity. Oral MC4R agonists (eg bivamelagon) are currently also in phase II trials, which will pave the way for similar drugs targeting the appetite- and weight-regulating networks controlled by the hypothalamus. There are plans with some member countries to set up a multi-centre trial for the use of the GLP-1 agonist semaglutide for the same purpose. However, other aspects of hypothalamo-pituitary dysfunction (sleep, temperature dysregulation, autonomic dysfunction, behavioural disturbance) all remain poorly managed and more research is needed in these areas.

A pan-European registry for paediatric craniopharygiomas also does not exist and is one of the major objectives for the group in the coming years.

The group additionally provides an educational function through the organisation of the biennial Postgraduate Childhood Craniopharyngioma Course.