A systematic review of long-term outcomes (>10-year follow-up) of paediatric craniopharyngioma

Clinical Snapshot

Key Findings

• P Value: Not reported

• Effect Size: Mean overall survival: 91.6% at 5 years, 84.4% at 10 years, 77.5% at 15 years, 68.0% at 20 years. GTR alone and radiotherapy alone associated with higher survival than combined treatment. Endocrine dysfunction in 75% of patients; hypothyroidism most prevalent (81.9% of those with endocrinopathy). Late radiotherapy complications in approximately 10% of patients, predominantly vasculopathy.

• Primary Outcome: Overall survival at 5, 10, 15, and 20 years post-diagnosis in children with ACP

• Nnt Or Sensitivity: Not calculable from reported data; no NNT, hazard ratios, or relative risk estimates provided. Survival figures are descriptive means without variance measures.

• Confidence Interval: Not reported

Clinical Application

The review's clinical conclusions — favouring hypothalamus-sparing GTR where safely achievable, and caution regarding combined surgery plus radiotherapy — are feasible to implement in centres with experienced paediatric neurosurgeons. However, GTR without hypothalamic injury is technically demanding and not universally achievable. Endocrine surveillance and QoL monitoring are standard of care and feasible in Australian tertiary centres. Australia has a small number of specialist paediatric neurosurgical centres (Sydney Children's Hospital Network, Royal Children's Hospital Melbourne, Queensland Children's Hospital) where ACP management is concentrated. The findings support existing RACGP and paediatric endocrinology guidelines emphasising lifelong endocrine surveillance post-treatment. Proton beam therapy — relevant given the late vasculopathy risk from conventional radiotherapy — is not yet widely available in Australia, though the Australian Bragg Centre in Adelaide (opening anticipated 2025) will change this landscape. TGA-approved growth hormone replacement and thyroid hormone replacement are PBS-listed and central to long-term management. The rarity of ACP in Australia (~5–10 paediatric cases per year nationally) means participation in international registries such as KRANIOPHARYNGEOM 2007 or PFKPOH is important for evidence generation. Children under 18 years diagnosed with adamantinomatous craniopharyngioma managed at tertiary paediatric neurosurgical centres. Most applicable to patients with newly diagnosed ACP where treatment strategy decisions (extent of resection vs. radiotherapy) are being made by multidisciplinary teams.

Abstract

PURPOSE: Adamantinomatous craniopharyngiomas (ACP) in children are rare benign tumours. Relatively few publications have addressed the long-term impact of this tumour and its treatment. We undertake a systemic review of the existing literature aiming to define the outcome of ACP beyond 10 years' follow-up. METHODS: Ovid MEDLINE, Embase and Google Scholar were used to search the literature. The search criteria were children (aged under 18 at diagnosis), any intervention for and outcomes of ACP. Publications with less than 10 years' follow-up and single case reports were excluded. RESULTS: Twenty-one studies were included reporting on 1152 children, with a mean age of 8.8 years at diagnosis. Mean follow-up was 14 years. The most common presenting symptom was headache. The mean overall survival at 5, 10, 15 and 20 years post-diagnosis was 91.6%, 84.4%, 77.5% and 68.0%, respectively. In children undergoing gross total resection only or radiotherapy only, survival was higher than with combined treatments. Seventy-five percent of children had endocrine dysfunction after intervention; the most prevalent was hypothyroidism (81.9%). The prevalence of endocrinopathies was similar across all treatment groups. Lower QoL was reported than the general population, predominantly related to the effects of hypothalamic injury. One in ten patients suffered a late complication related to radiotherapy with vasculopathy being the most prevalent. CONCLUSIONS: The long-term morbidity and mortality for paediatric ACP remain high. The impact from hypothalamic injury cannot be understated and is likely the cause for this finding. As surgical treatment advances, less hypothalamic injury may arise and improved outcomes may follow. Gross total resection, if felt achievable without hypothalamic injury, should form the mainstay of treatment due to the late radiotherapy-related complications that arise.

References

1. 1.Lawrence, A. J., & Aquilina, K. (2026). A systematic review of long-term outcomes (>10-year follow-up) of paediatric craniopharyngioma. Child's Nervous System. Advance online publication. PubMed ID: 42414633