HGG-11. Clinical characteristics and clinical evolution of a large cohort of pediatric patients with primary central nervous system (CNS) tumors and tropomyosin receptor kinase (TRK) fusion. (3rd June 2022)

• Record Type: Journal Article
• Authors: Lamoureux, Audrey-Anne; Fisher, Michael; Lemelle, Lauriane; Pfaff, Elke; Kramm, Christof; De Wilde, Bram; Kazanowska, Bernarda; Hutter, Caroline; Pfister, Stefan M; Sturm, Dominik; Jones, David; Orbach, Daniel; Pierron, Gaëlle; Raskin, Scott; Drilon, Alexander; Diamond, Eli; Harada, Guilherme; Zapotocky, Michal; Ellezam, Benjamin; Weil, Alexander G; Venne, Dominic; Barritault, Marc; Leblond, Pierre; Coltin, Hallie; Hammad, Rawan; Tabori, Uri; Hawkins, Cynthia; Hansford, Jordan R; Meyran, Deborah; Erker, Craig; McFadden, Kathryn; Sato, Mariko; Gottardo, Nicholas G; Dholaria, Hetal; Nørøxe, Dorte Schou; Goto, Hiroaki; Ziegler, David S; Lin, Frank Y; Parsons, Donald Williams; Lindsay, Holly; Wong, Tai-Tong; Liu, Yen-Lin; Wu, Kuo-Sheng; Franson, Andrea Flynn; Hwang, Eugene; Aguilar-Bonilla, Ana; Cheng, Sylvia; Cacciotti, Chantel; Massimino, Maura; Schiavello, Elisabetta; Wood, Paul; Hoffman, Lindsey M; Cappellano, Andréa; Lassaletta, Alvaro; Van Damme, An; Llort, Anna; Gerber, Nicolas U; Ceruso, Mariella Spalato; Bendel, Anne E; Skrypek, Maggie; Hamideh, Dima; Mushtaq, Naureen; Walter, Andrew; Jabado, Nada; Alsahlawi, Aysha; Farmer, Jean-Pierre; Abadi, Christina Coleman; Mueller, Sabine; Mazewski, Claire; Aguilera, Dolly; Robison, Nathan; O'Halloran, Katrina; Abbou, Samuel; Berlanga, Pablo; Geoerger, Birgit; Øra, Ingrid; Moertel, Christopher L; Razis, Evangelia D; Vernadou, Anastasia; Doz, François; Laetsch, Theodore W; Perreault, Sébastien;
• Is Part Of: Neuro-oncology. Volume 24(2022)Supplement 1
• Page Start: i61
• Page End: i62
• Publication Date: 2022-06-03
• Subjects: Brain Neoplasms -- Periodicals; Brain -- Tumors -- Periodicals; Brain -- Cancer -- Periodicals; Nervous system -- Cancer -- Periodicals; 616.99481
• DOI: 10.1093/neuonc/noac079.226
• Languages: English
• ISSNs: 1522-8517

Abstract

Abstract: BACKGROUND: TRK fusions are detected in less than 3% of CNS tumors. Given their rarity, there are limited data on the clinical course of these patients. METHODS: We contacted 166 oncology centers worldwide to retrieve data on patients with TRK fusion-driven CNS tumors. Data extracted included demographics, histopathology, NTRK gene fusion, treatment modalities and outcomes. Patients less than 18 years of age at diagnosis were included in this analysis. RESULTS: Seventy-three pediatric patients with TRK fusion-driven primary CNS tumors were identified. Median age at diagnosis was 2.4 years (range 0.0–17.8) and 60.2 % were male. NTRK2 gene fusions were found in 37 patients (50.7%), NTRK1 and NTRK3 aberrations were detected in 19 (26.0%) and 17 (23.3%), respectively. Tumor types included 38 high-grade gliomas (HGG; 52.1%), 20 low-grade gliomas (LGG; 27.4%), 4 embryonal tumors (5.5%) and 11 others (15.1%). Median follow-up was 46.5 months (range 3-226). During the course of their disease, a total of 62 (84.9%) patients underwent surgery with a treatment intent, 50 (68.5%) patients received chemotherapy, 35 (47.9%) patients received radiation therapy, while 34 (46.6%) patients received NTRK inhibitors (3 as first line treatment). Twenty-four (32.9%) had no progression including 9 LGG (45%) and 9 HGG (23.6%). At last follow-up, only one (5.6%-18 evaluable) patient with LGG died compared to 11 with HGG (35.5%-31 evaluable). For LGG the median progression-free survival (PFS) after the first line of treatment was 17 months (95% CI: 0.0-35.5) and median overall survival (OS) was not reached. For patients with HGG the median PFS was 30 months (95% CI: 11.9-48.1) and median OS was 182 months (95% CI 20.2-343.8). CONCLUSIONS: We report the largest cohort of pediatric patients with TRK fusion-driven primary CNS tumors. These results will help us to better understand clinical evolution and compare outcomes with ongoing clinical trials.