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Clinical characteristics and outcome of central nervous system tumors harboring NTRK gene fusions
Clinical Cancer Research ( IF 10.0 ) Pub Date : 2024-12-03 , DOI: 10.1158/1078-0432.ccr-24-0581 Audrey-Anne Lamoureux, Michael J. Fisher, Lauriane Lemelle, Elke Pfaff, Pouneh Amir-Yazdani, Christof Kramm, Bram De Wilde, Bernarda Kazanowska, Caroline Hutter, Stefan M. Pfister, Dominik Sturm, David T.W. Jones, Daniel Orbach, Gaëlle Pierron, Scott Raskin, Alexander Drilon, Eli L. Diamond, Guilherme Harada, Michal Zapotocky, Josef Zamecnik, Lenka Krskova, Benjamin Ellezam, Alexander G. Weil, Dominic Venne, Marc Barritault, Pierre Leblond, Hallie Coltin, Rawan Hammad, Uri Tabori, Cynthia Hawkins, Jordan R. Hansford, Deborah Meyran, Craig Erker, Kathryn McFadden, Mariko Sato, Nicholas G. Gottardo, Hetal Dholaria, Dorte Schou. Nørøxe, Hiroaki Goto, David S. Ziegler, Frank Y. Lin, Donald Williams. Parsons, Holly Lindsay, Tai-Tong Wong, Yen-Lin Liu, Kuo-Sheng Wu, Andrea T. Franson, Eugene Hwang, Ana Aguilar-Bonilla, Sylvia Cheng, Chantel Cacciotti, Maura Massimino, Elisabetta Schiavello, Paul Wood, Lindsey M. Hoffman, Andréa Cappellano, Alvaro Lassaletta, An Van Damme, Anna Llort, Nicolas U. Gerber, Mariella Spalato Ceruso, Anne E. Bendel, Maggie Skrypek, Dima Hamideh, Naureen Mushtaq, Andrew Walter, Nada Jabado, Aysha Alsahlawi, Jean-Pierre Farmer, Christina Coleman, Sabine Mueller, Claire Mazewski, Dolly Aguilera, Nathan J. Robison, Katrina O’Halloran, Samuel Abbou, Pablo Berlanga, Birgit Geoerger, Ingrid Øra, Christopher L. Moertel, Evangelia D. Razis, Anastasia Vernadou, François Ducray, Charlotte Bronnimann, Romuald Seizeur, Matthew Clarke, Adam C. Resnick, Mélanie Alves, Chris Jones, François Doz, Theodore W. Laetsch, Sébastien Perreault
Clinical Cancer Research ( IF 10.0 ) Pub Date : 2024-12-03 , DOI: 10.1158/1078-0432.ccr-24-0581 Audrey-Anne Lamoureux, Michael J. Fisher, Lauriane Lemelle, Elke Pfaff, Pouneh Amir-Yazdani, Christof Kramm, Bram De Wilde, Bernarda Kazanowska, Caroline Hutter, Stefan M. Pfister, Dominik Sturm, David T.W. Jones, Daniel Orbach, Gaëlle Pierron, Scott Raskin, Alexander Drilon, Eli L. Diamond, Guilherme Harada, Michal Zapotocky, Josef Zamecnik, Lenka Krskova, Benjamin Ellezam, Alexander G. Weil, Dominic Venne, Marc Barritault, Pierre Leblond, Hallie Coltin, Rawan Hammad, Uri Tabori, Cynthia Hawkins, Jordan R. Hansford, Deborah Meyran, Craig Erker, Kathryn McFadden, Mariko Sato, Nicholas G. Gottardo, Hetal Dholaria, Dorte Schou. Nørøxe, Hiroaki Goto, David S. Ziegler, Frank Y. Lin, Donald Williams. Parsons, Holly Lindsay, Tai-Tong Wong, Yen-Lin Liu, Kuo-Sheng Wu, Andrea T. Franson, Eugene Hwang, Ana Aguilar-Bonilla, Sylvia Cheng, Chantel Cacciotti, Maura Massimino, Elisabetta Schiavello, Paul Wood, Lindsey M. Hoffman, Andréa Cappellano, Alvaro Lassaletta, An Van Damme, Anna Llort, Nicolas U. Gerber, Mariella Spalato Ceruso, Anne E. Bendel, Maggie Skrypek, Dima Hamideh, Naureen Mushtaq, Andrew Walter, Nada Jabado, Aysha Alsahlawi, Jean-Pierre Farmer, Christina Coleman, Sabine Mueller, Claire Mazewski, Dolly Aguilera, Nathan J. Robison, Katrina O’Halloran, Samuel Abbou, Pablo Berlanga, Birgit Geoerger, Ingrid Øra, Christopher L. Moertel, Evangelia D. Razis, Anastasia Vernadou, François Ducray, Charlotte Bronnimann, Romuald Seizeur, Matthew Clarke, Adam C. Resnick, Mélanie Alves, Chris Jones, François Doz, Theodore W. Laetsch, Sébastien Perreault
Purpose: TRK fusions are detected in less than 2% of central nervous system tumors. There are limited data on the clinical course of affected patients. Experimental design: We conducted an international retrospective cohort study of patients with TRK fusion-driven CNS tumors. Results: 119 patients were identified. The median age at time of diagnosis was 4.5 years. The majority were reported to have a histology consistent with a diagnosis of high-grade glioma (HGG) (57.1%) followed by low-grade glioma (LGG) (27.7%). Pediatric patients had a better prognosis with a median overall survival of 185.5 months compared to 24.8 months in adults (p<.0001). Patients with LGG also had a better outcome when compared to HGG (p=0.0012). The objective response was 68.8% with larotrectinib compared to 38.1% for non-targeted treatment. Conclusions: Children with LGG glioma had a favorable outcome compared to adult and HGG. TRK inhibitors appear to improve tumor control.
中文翻译：
携带 NTRK 基因融合的中枢神经系统肿瘤的临床特征和结局
目的：在不到 2% 的中枢神经系统肿瘤中检测到 TRK 融合。关于受影响患者临床病程的数据有限。实验设计： 我们对 TRK 融合驱动的 CNS 肿瘤患者进行了一项国际回顾性队列研究。结果： 确定了 119 例患者。诊断时的中位年龄为 4.5 岁。据报道，大多数患者的组织学与高级别胶质瘤 （HGG） 的诊断一致 （57.1%），其次是低级别胶质瘤 （LGG） （27.7%）。儿科患者的预后较好，中位总生存期为 185.5 个月，而成人为 24.8 个月 （p<.0001）。与 HGG 相比，LGG 患者的预后也更好 （p=0.0012）。larotrectinib 的客观缓解率为 68.8%，而非靶向治疗的客观缓解率为 38.1%。结论： 与成人和 HGG 相比，LGG 胶质瘤患儿预后良好。TRK 抑制剂似乎可以改善肿瘤控制。
更新日期：2024-12-03
中文翻译：
携带 NTRK 基因融合的中枢神经系统肿瘤的临床特征和结局
目的：在不到 2% 的中枢神经系统肿瘤中检测到 TRK 融合。关于受影响患者临床病程的数据有限。实验设计： 我们对 TRK 融合驱动的 CNS 肿瘤患者进行了一项国际回顾性队列研究。结果： 确定了 119 例患者。诊断时的中位年龄为 4.5 岁。据报道，大多数患者的组织学与高级别胶质瘤 （HGG） 的诊断一致 （57.1%），其次是低级别胶质瘤 （LGG） （27.7%）。儿科患者的预后较好，中位总生存期为 185.5 个月，而成人为 24.8 个月 （p<.0001）。与 HGG 相比，LGG 患者的预后也更好 （p=0.0012）。larotrectinib 的客观缓解率为 68.8%，而非靶向治疗的客观缓解率为 38.1%。结论： 与成人和 HGG 相比，LGG 胶质瘤患儿预后良好。TRK 抑制剂似乎可以改善肿瘤控制。
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