Álvaro Lassaletta1, Eric Bouffet2, Palma Solano-Páez3, Adriana Fonseca2, Lorena V Baroni4, Nisreen Amayiri5, Marta P Somarriba6, Candela Freytes4, Eduardo Quiroga7, Mónica Rivero8, Javier Márquez8. 1. Department of Pediatric Hematology and Oncology, Hospital Universitario Niño Jesús, Madrid, Spain. 2. Division of Neuro-Oncology, The Hospital for Sick Children, Toronto, ON, Canada. 3. Department of Pediatric Oncology, Hospital Infantil Virgen del Rocío, Avda/Manuel Siurot S/N CP, 41003, Seville, Spain. palma.solano.sspa@juntadeandalucia.es. 4. Department of Hematology/Oncology, Hospital de Pediatría SAMIC Prof. Dr. Juan P. Garrahan, Buenos Aires, Argentina. 5. Department of Pediatrics, Hematology/Oncology, King Hussein Cancer Center, Amman, Jordan. 6. Department of Pediatric Oncology, Hospital Sant Joan de Déu, Barcelona, Spain. 7. Department of Pediatric Oncology, Hospital Infantil Virgen del Rocío, Avda/Manuel Siurot S/N CP, 41003, Seville, Spain. 8. Department of Pediatric Neurosurgery, Hospital Infantil Virgen del Rocío, Seville, Spain.
Abstract
INTRODUCTION: Ventriculo-peritoneal shunt (VPS) related ascites is a rare complication of pediatric low grade gliomas (pLGG). Physiopathology of this complication is not fully understood and there is paucity of data regarding the molecular profile of pLGG gliomas complicating with ascites and the optimal management of this unusual event. METHODS: International multi-institutional retrospective analysis of patients diagnosed with BRAF altered pLGG and ascites arising as a complication of VPS. Demographics, tumor characteristics, therapeutic approaches and outcomes were recorded. RESULTS: Nineteen patients were identified. Median age at diagnosis was 14 months (R: 2-144). Most patients (17; 89.4%) presented with lesions involving the optic pathway. Mean tumor standard volume was 34.8 cm2 (R: 12.5-85.4). Pilocytic Astrocytoma was the most frequent histological diagnosis (14;7 3.7%). Eight (42.1%) tumors harbored BRAF V600-E mutation and seven (36.8%) KIAA1549 fusion. The onset of ascites was documented at a median time of 5 months following VPS insertion. Four (21%) patients were managed with paracentesis only, 7(36.8%) required both paracentesis and shunt diversion, 7(36.8%) required only a shunt diversion and 1 (5.2%) patient was managed conservatively. Chemotherapy regimen was changed in 10 patients following ascites. Eight patients received targeted therapy (4 dabrafenib/4 trametinib) and 5 were radiated. There were eleven survivors with a median OS of 69 months (R: 3-144). CONCLUSIONS: Ascites is an early feature in the clinical course of young patients with midline BRAF altered pLGG, with high mortality rate observed in our cohort. The hypothesis of ascites as an adverse prognostic factor in pLGG warrants further prospective research.
INTRODUCTION: Ventriculo-peritoneal shunt (VPS) related ascites is a rare complication of pediatric low grade gliomas (pLGG). Physiopathology of this complication is not fully understood and there is paucity of data regarding the molecular profile of pLGG gliomas complicating with ascites and the optimal management of this unusual event. METHODS: International multi-institutional retrospective analysis of patients diagnosed with BRAF altered pLGG and ascites arising as a complication of VPS. Demographics, tumor characteristics, therapeutic approaches and outcomes were recorded. RESULTS: Nineteen patients were identified. Median age at diagnosis was 14 months (R: 2-144). Most patients (17; 89.4%) presented with lesions involving the optic pathway. Mean tumor standard volume was 34.8 cm2 (R: 12.5-85.4). Pilocytic Astrocytoma was the most frequent histological diagnosis (14;7 3.7%). Eight (42.1%) tumors harbored BRAF V600-E mutation and seven (36.8%) KIAA1549 fusion. The onset of ascites was documented at a median time of 5 months following VPS insertion. Four (21%) patients were managed with paracentesis only, 7(36.8%) required both paracentesis and shunt diversion, 7(36.8%) required only a shunt diversion and 1 (5.2%) patient was managed conservatively. Chemotherapy regimen was changed in 10 patients following ascites. Eight patients received targeted therapy (4 dabrafenib/4 trametinib) and 5 were radiated. There were eleven survivors with a median OS of 69 months (R: 3-144). CONCLUSIONS: Ascites is an early feature in the clinical course of young patients with midline BRAF altered pLGG, with high mortality rate observed in our cohort. The hypothesis of ascites as an adverse prognostic factor in pLGG warrants further prospective research.
Authors: Roger J Packer; Stephan Pfister; Eric Bouffet; Robert Avery; Pratiti Bandopadhayay; Miriam Bornhorst; Daniel C Bowers; David Ellison; Jason Fangusaro; Nicholas Foreman; Maryam Fouladi; Amar Gajjar; Daphne Haas-Kogan; Cynthia Hawkins; Cheng-Ying Ho; Eugene Hwang; Nada Jabado; Lindsay B Kilburn; Alvaro Lassaletta; Keith L Ligon; Maura Massimino; Schouten-van Meeteren; Sabine Mueller; Theo Nicolaides; Giorgio Perilongo; Uri Tabori; Gilbert Vezina; Katherine Warren; Olaf Witt; Yuan Zhu; David T Jones; Mark Kieran Journal: Neuro Oncol Date: 2017-06-01 Impact factor: 12.300