Xanthogranuloma, Rathke's cyst, and childhood craniopharyngioma: results of prospective multinational studies of children and adolescents with rare sellar malformations

Abstract

Context: Craniopharyngioma (CP), Rathke's cyst (RC), and xanthogranuloma (XG) are closely related rare sellar masses. Treatment strategies in children lack consensus.

Objective: Our objective was to study clinical manifestations and treatment-related outcome in RC, XG, and CP patients.

Design: We conducted a multicenter surveillance trial.

Patients: Inclusion criteria were 1) histological diagnosis of CP, XG, or RC and 2) diagnosis at age of 18 yr or less. A total of 117 CP, 14 XG (2001-2006), and 14 RC (1996-2006) were recruited.

Main outcome: Overall survival (OS), event-free survival (EFS), and quality of life (QoL) were evaluated.

Results: The 5-yr OS rates were 1.00 ± 0.00 in RC and XG and 0.97 ± 0.02 in CP. The 5-yr EFS rates were 0.85 ± 0.10 in RC, 1.00 ± 0.00 in XG, and 0.50 ± 0.05 in CP. Surgical resection of XG results in complete remission without recurrence. Recurrences occur in RC (14%) and CR (59%) but can be efficiently treated by irradiation, reoperation, and/or intracystic treatment. Severe hypothalamic sequelae such as obesity and others affecting QoL are predominant in CP due to presurgical involvement (59%) and postsurgical lesions (44%) of posterior hypothalamic structures. Centers with lower neurosurgery patient load use more radical surgical approaches to treat CP, resulting in higher rates of obesity and reduced QoL. Despite 46% anterior hypothalamic involvement, severe obesity is not encountered in XG.

Conclusions: Treatment of choice in XG and RC is radical surgery. In CP involving hypothalamic structures, less radical surgical approaches preserving hypothalamic integrity are recommended. Due to frequent relapses, regular imaging during follow-up is recommended for CP and RC. Treatment of patients with sellar masses should be confined to experienced multidisciplinary teams.