Intratumoral tocilizumab in children with cystic craniopharyngiomas: targeted therapy.
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PURPOSE: The craniopharyngioma, although benign, exhibits aggressive behavior due to its capacity to invade the surrounding neurovascular structures. Conventional treatment with surgery and radiotherapy is related to significant morbidity and impact on quality of life. Recent findings prove the increase of interleukin-6 (IL-6) and its receptor (IL-6R) in those tumors, making tocilizumab, an anti-IL-6R antibody used for rheumatological diseases, a possible targeted therapy. The present study investigated the safety and effectiveness of the intracystic application of tocilizumab in children with cystic craniopharyngiomas. METHODS: Longitudinal prospective analysis of the use of the maximum dose of 3,6 mg/kg of intracystic tocilizumab in seven individuals under 20 years old who have a confirmed diagnosis of cystic adamantinomatous craniopharyngioma. Following an initial dose of 0.8-1.2 mg/kg, patients underwent follow-up imaging. If this revealed cyst progression (defined as any measurable increase or no reduction), a second dose, tripled from the initial, was administered. The follow-up took two years. Tumor cystic volume, IL-6 quantification, tumor fluid color, endocrinological and visual dysfunctions, and patient/main care quality of life before and after treatment were evaluated RESULTS: Out of nine treated cysts in seven patients, only three of them (33,3%) showed cystic control during the monitoring period. Six of the seven patients (85.7%) exhibited cyst progression following the initial dose, requiring a second, tripled dose as per the study protocol. This repeat administration was performed within a median of 9 months after the initial dose. There was no systemic or local toxicity. The color of the tumor fluid was correlated to the levels of IL-6 (p = 0.05), but not to the clinical outcome. There were no significant changes in the levels of life quality or visual/endocrine functionality. CT and MRI of the brain showed a strong correlation to the evaluation of the cystic volume (p < 0,01). CONCLUSION: The use of intratumoral tocilizumab was shown to be safe; however, not effective at the frequency and dosage used. The tumor fluid color and IL-6 levels might be related to the prognosis in larger series. Further studies should explore higher doses and frequency when applying it in order to reach a therapeutic optimization.