John-Paul Kilday1, Massimo Caldarelli1, Luca Massimi1, Robert Hsin-Hung Chen1, Yi Yen Lee1, Muh-Lii Liang1, Jeanette Parkes1, Thuran Naiker1, Marie-Lise van Veelen1, Erna Michiels1, Conor Mallucci1, Benedetta Pettorini1, Lisethe Meijer1, Christian Dorfer1, Thomas Czech1, Manuel Diezi1, Antoinette Y N Schouten-van Meeteren1, Stefan Holm1, Bengt Gustavsson1, Martin Benesch1, Hermann L Müller1, Anika Hoffmann1, Stefan Rutkowski1, Joerg Flitsch1, Gabriele Escherich1, Michael Grotzer1, Helen A Spoudeas1, Kristian Azquikina1, Michael Capra1, Rolando Jiménez-Guerra1, Patrick MacDonald1, Donna L Johnston1, Rina Dvir1, Shlomi Constantini1, Meng-Fai Kuo1, Shih-Hung Yang1, Ute Bartels1. 1. Children's Brain Tumour Research Network (CBTRN), Royal Manchester Children's Hospital, Oxford Road, Manchester, England, UK; The Centre for Paediatric, Teenage and Young Adult Cancer, Institute of Cancer Sciences, The University of Manchester, England, UK; Department of Pediatric Neurosurgery, A Gemelli Hospital, Rome, Italy; Division of Pediatric Neurosurgery, Neurological Institute, Taipei Veterans General Hospital, Taipei, Taiwan; Groote Schuur Hospital, University of Cape Town, South Africa; Sophia Children's Hospital, Erasmus MC, Rotterdam, Netherlands; Department of Neurosurgery, Alder Hey Children's Hospital NHS Foundation Trust, Liverpool, England, UK ; Beatrix Childrens' Hospital, University MC, Groningen, Netherlands; Department of Neurosurgery, Medical University of Vienna, Vienna, Austria; Pediatric Haemato-Oncology Unit, University Hospital Lausanne, Switzerland; Pediatric Oncology Department, Emma Children's Hospital of the Academic Medical Center, Amsterdam, Netherlands; Department of Pediatric Neurosurgery, Karolinska University Hospital, Stockholm, Sweden; Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Medical University of Graz, Graz, Austria; Department of Pediatrics, Klinikum Oldenburg AöR, Medical Campus University Oldenburg, Oldenburg, Germany; Departments of Neurosurgery and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany; Department of Neurosurgery, University Children's Hospital Zurich, Switzerland; Departments of Paediatric Neuroendocrinology and Neurosurgery, Great Ormond Street Hospital, London, England, UK; Department of Paediatrics, Our Lady's Children's Hospital, Crumlin, Ireland; Department of Pediatric Neurosurgery, Hospital Angeles Mexico, Mexico City,Mexico; Department of Neurosurgery, Winnipeg Children's Hospital, Canada; Department of Paediatrics, Children's Hospital of Eastern Ontario, Ottawa, Canada; Department of Neurosurgery, Tel-Aviv Sourasky Medical Center, Israel; Department of Neurosurgery, National Taiwan University Hospital, Taiwan; Department of Paediatric Oncology, The Hospital for Sick Children, Toronto, Canada.
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 Société Internationale d'Oncologie Pédiatrique 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.
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 Société Internationale d'Oncologie Pédiatrique 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.
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