Clinical outcome of biomarker-guided therapies in adult patients with tumors of the nervous system

被引:14
作者
Renovanz, Mirjam [1 ,2 ,4 ]
Kurz, Sylvia C. [1 ,2 ]
Rieger, Johannes [1 ,2 ]
Walter, Bianca [1 ]
Becker, Hannes [1 ,2 ,4 ]
Hille, Hanni [1 ]
Bombach, Paula [1 ,2 ,3 ]
Rieger, David [1 ,2 ]
Grosse, Lucia [1 ,2 ]
Haeusser, Lara [1 ,21 ]
Skardelly, Marco [1 ,2 ]
Merk, Daniel J. [1 ]
Paulsen, Frank [2 ,3 ,6 ]
Hoffmann, Elgin [2 ,3 ,6 ]
Gani, Cihan [2 ,3 ,6 ,21 ]
Neumann, Manuela [2 ,3 ,7 ,21 ]
Beschorner, Rudi [2 ,3 ,7 ,21 ]
Riess, Olaf [3 ,9 ,20 ,21 ]
Roggia, Cristiana [3 ,9 ]
Schroeder, Christopher [3 ,9 ]
Ossowski, Stephan [3 ,9 ]
Armeanu-Ebinger, Sorin [3 ,9 ]
Gschwind, Axel [3 ,9 ]
Biskup, Saskia [10 ,11 ,20 ]
Schulze, Martin [10 ,11 ]
Fend, Falko [3 ,8 ,20 ,21 ]
Singer, Stephan [3 ,8 ,20 ,21 ]
Zender, Lars [3 ,15 ,20 ,21 ]
Lengerke, Claudia [16 ,21 ]
Brucker, Sara Yvonne [19 ,21 ]
Engler, Tobias [19 ,21 ]
Forschner, Andrea [17 ,21 ]
Stenzl, Arnulf [18 ,21 ]
Kohlbacher, Oliver [3 ,14 ,21 ]
Nahnsen, Sven [3 ,12 ,15 ,20 ,21 ]
Gabernet, Gisela [12 ]
Fillinger, Sven [12 ]
Bender, Benjamin [2 ,3 ,13 ]
Ernemann, Ulrike [2 ,3 ,13 ]
Oener, Oeznur [3 ]
Beha, Janina [3 ,21 ]
Malek, Holly Sundberg [3 ]
Moeller, Yvonne [3 ]
Ruhm, Kristina [3 ]
Tatagiba, Marcos [2 ,3 ,4 ]
Schittenhelm, Jens [2 ,3 ,7 ]
Bitzer, Michael [3 ,5 ,20 ]
Malek, Nisar [3 ,5 ,20 ,21 ]
Zips, Daniel [2 ,3 ,6 ,21 ]
Tabatabai, Ghazaleh [1 ,2 ,3 ,20 ,21 ]
机构
[1] Eberhard Karls Univ Tubingen, Hertie Inst Clin Brain Res, Dept Neurol & Interdisciplinary Neurooncol, Tubingen, Germany
[2] Univ Hosp Tubingen, Ctr Neurooncol, Comprehens Canc Ctr Tubingen Stuttgart, Tubingen, Germany
[3] Eberhard Karls Univ Tubingen, Ctr Personalized Med Tubingen, Tubingen, Germany
[4] Eberhard Karls Univ Tubingen, Dept Neurosurg, Tubingen, Germany
[5] Eberhard Karls Univ Tubingen, Dept Internal Med 1, Tubingen, Germany
[6] Eberhard Karls Univ Tubingen, Dept Radiat Oncol, Tubingen, Germany
[7] Eberhard Karls Univ Tubingen, Inst Pathol & Neuropathol, Dept Neuropathol, Tubingen, Germany
[8] Eberhard Karls Univ Tubingen, Inst Pathol & Mol Pathol, Dept Pathol & Neuropathol, Tubingen, Germany
[9] Eberhard Karls Univ Tubingen, Inst Med Genet & Appl Genom, Tubingen, Germany
[10] Ctr Genom & Transcript CeGaT, Tubingen, Germany
[11] Ctr Human Genet Tubingen, Tubingen, Germany
[12] Eberhard Karls Univ Tubingen, Quantitat Biol Ctr QBiC, Tubingen, Germany
[13] Eberhard Karls Univ Tubingen, Dept Diagnost & Intervent Neuroradiol, Dept Radiol, Tubingen, Germany
[14] Eberhard Karls Univ Tubingen, Inst Translat Bioinformat, Tubingen, Germany
[15] Eberhard Karls Univ Tubingen, Dept Med Oncol & Pneumol Internal Med 8, Tubingen, Germany
[16] Eberhard Karls Univ Tubingen, Dept Internal Med 2, Tubingen, Germany
[17] Eberhard Karls Univ Tubingen, Dept Dermatol Oncol, Tubingen, Germany
[18] Eberhard Karls Univ Tubingen, Dept Urol, Tubingen, Germany
[19] Eberhard Karls Univ Tubingen, Dept Gynecol & Obstet, Tubingen, Germany
[20] Eberhard Karls Univ Tubingen, Cluster Excellence EXC 2180 Image Guided & Func, Tubingen, Germany
[21] Eberhard Karls Univ Tubingen, DKFZ Partner Site Tubingen, Tubingen, Germany
关键词
Molecular tumor board; MTB@ZPM-001 (NCT03503149); precision medicine; real-world data; targeted therapy; RESPONSE ASSESSMENT; TARGETED THERAPY; OPEN-LABEL; CANCER; NEUROONCOLOGY; GLIOBLASTOMA; GUIDELINES; MEDICINE; ONCOLOGY; CRITERIA;
D O I
10.1093/noajnl/vdad012
中图分类号
R73 [肿瘤学];
学科分类号
100214 ;
摘要
Background The clinical utility of molecular profiling and targeted therapies for neuro-oncology patients outside of clinical trials is not established. We aimed at investigating feasibility and clinical utility of molecular profiling and targeted therapy in adult patients with advanced tumors in the nervous system within a prospective observational study. Methods molecular tumor board (MTB)@ZPM (NCT03503149) is a prospective observational precision medicine study for patients with advanced tumors. After inclusion of patients, we performed comprehensive molecular profiling, formulated ranked biomarker-guided therapy recommendations based on consensus by the MTB, and collected prospective clinical outcome data. Results Here, we present initial data of 661 adult patients with tumors of the nervous system enrolled by December 31, 2021. Of these, 408 patients were presented at the MTB. Molecular-instructed therapy recommendations could be made in 380/408 (93.1%) cases and were prioritized by evidence levels. Therapies were initiated in 86/380 (22.6%) cases until data cutoff. We observed a progression-free survival ratio >1.3 in 31.3% of patients. Conclusions Our study supports the clinical utility of biomarker-guided therapies for neuro-oncology patients and indicates clinical benefit in a subset of patients. Our data might inform future clinical trials, translational studies, and even clinical care.
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页数:14
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