Aims: Although rare, craniopharyngiomas are the commonest suprasellar tumour in childhood. Despite high overall survival, children and young people <19 years with craniopharyngiomas are at risk of multiple relapses and long-term tumour- and treatment-related neuroendocrine, cognitive and visual morbidity. A recent international survey highlighted the considerable variation in management strategies employed for these tumours, and the lack of any evidence- and/ or consensus-based guidelines. We sought to provide, for the first time, a national standard for best practice based on currently available evidence for the assessment, treatment and follow-up of paediatric craniopharyngiomas under the auspices of the Royal College of Paediatrics & Child Health (RCPCH), UK Children's Cancer & Leukaemia Group (CCLG) and the British Society for Paediatric Endocrinology & Diabetes (BSPED).
Methods: Clinical questions were formulated based on a PICO (Population, Intervention, Comparison,Outcome) format by a multidisciplinary Guideline Development Group. Systematic searches were conducted via the Ovid MEDLINE (1946-February 2017) and Cochrane Library (2016, Issue 12) databases, identifying 2023 separate research articles. Publications underwent a three-tier filtering process and 300 were reviewed using the GRADE approach. Where recommendations could not be made, a two-stage international Delphi consensus process was conducted. The guideline was developed using AGREE II criteria.
Results: 44 clinical questions were identified, leading to 35 recommendations largely based on low to very low quality evidence. 30 further recommendations achieved >70% agreement via the Delphi consensus process. Important highlights include the recommendation that craniopharyngiomas are managed in tertiary paediatric centres with sufficient neuro-oncology, neurosurgery, endocrinology, radiology, pathology and neuropsychology multidisciplinary experience. At diagnosis, tumours should be graded using the "Paris" grading system (Puget et al., J Neurosurg 2007; 106(Suppl 1):3-12) and subsequent surgical treatment tailored to avoid hypothalamic damage, with adjuvant upfront radiotherapy being offered where tumour resection is incomplete. Detailed recommendations on the neuroendocrine, ophthalmological and psychological pre-treatment assessment of patients and long-term follow-up of survivors are also made, with a review on the safety of growth hormone replacement therapy in this cohort.
Conclusions: These guidelines provide the first evidence- and consensus-based national recommendations for the management of paediatric craniopharyngioma, and highlight the need for further research in areas such as the efficacy of proton beam therapy, radiosurgery and intracystic therapies in children, and the management of late effects such as hypothalamic obesity. Through their implementation, we hope to achieve better consistency in the quality of care of such patients and improve long-term quality of survival.