Intracystic interferon-alpha in pediatric craniopharyngioma patients: an international multicenter assessment on behalf of SIOPE and ISPN

John-Paul Kilday, Massimo Caldarelli, Luca Massimi, Robert Hsin-Hung Chen, Yi Yen Lee, Muh-Lii Liang, Jeanette Parkes, Thuran Naiker, Marie-Lise van Veelen, Erna Michiels, Conor Mallucci, Benedetta Pettorini, Lisethe Meijer, Christian Dorfer, Thomas Czech, Manuel Diezi, Antoinette Y. N. Schouten-van Meeteren, Stefan Holm, Bengt Gustavsson, Martin BeneschHermann L. Müller, Anika Hoffmann, Stefan Rutkowski, Joerg Flitsch, Gabriele Escherich, Michael Grotzer, Helen A. Spoudeas, Kristian Azquikina, Michael Capra, Rolando Jiménez-Guerra, Patrick MacDonald, Donna L. Johnston, Rina Dvir, Shlomi Constantini, Meng-Fai Kuo, Shih-Hung Yang, Ute Bartels

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43 Citations (Scopus)

Abstract

Background: Craniopharyngiomas are frequent hypothalamo-pituitary tumors in children, presenting predominantly as cystic lesions. Morbidity from conventional treatment has focused attention on intracystic drug delivery, hypothesized to cause fewer clinical consequences. However, the efficacy of intracystic therapy remains unclear. We report the retrospective experiences of several global centers using intracystic interferon-alpha. Methods: European Societe Internationale d'Oncologie Pediatrique and International Society for Pediatric Neurosurgery centers were contacted to submit a datasheet capturing pediatric patients with cystic craniopharyngiomas who had received intracystic interferon-alpha. Patient demographics, administration schedules, adverse events, and outcomes were obtained. Progression was clinical or radiological (cyst reaccumulation, novel cysts, or solid growth). Results: Fifty-six children (median age, 6.3 y) from 21 international centers were identified. Median follow-up from diagnosis was 5.1 years (0.3-17.7 y). Lesions were cystic (n = 22; 39%) or cystic/solid (n = 34; 61%). Previous progression was treated in 43 (77%) patients before interferon use. In such cases, further progression was delayed by intracystic interferon compared with the preceding therapy for cystic lesions (P = 0.0005). Few significant attributable side effects were reported. Progression post interferon occurred in 42 patients (median 14 mo; 0-8 y), while the estimated median time to definitive therapy post interferon was 5.8 (1.8-9.7) years. Conclusions: Intracystic interferon-alpha can delay disease progression and potentially offer a protracted time to definitive surgery or radiotherapy in pediatric cystic craniopharyngioma, yet demonstrates a favorable toxicity profile compared with other therapeutic modalities-important factors for this developing age group. A prospective, randomized international clinical trial assessment is warranted
Original languageEnglish
Pages (from-to)1398-1407
JournalNeuro-Oncology
Volume19
Issue number10
Early online date2017
DOIs
Publication statusPublished - 2017

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