Renovanz Mirjam, Kurz Sylvia C, Rieger Johannes, Walter Bianca, Becker Hannes, Hille Hanni, Bombach Paula, Rieger David, Grosse Lucia, Häusser Lara, Skardelly Marco, Merk Daniel J, Paulsen Frank, Hoffmann Elgin, Gani Cihan, Neumann Manuela, Beschorner Rudi, Rieß Olaf, Roggia Cristiana, Schroeder Christopher, Ossowski Stephan, Armeanu-Ebinger Sorin, Gschwind Axel, Biskup Saskia, Schulze Martin, Fend Falko, Singer Stephan, Zender Lars, Lengerke Claudia, Brucker Sara Yvonne, Engler Tobias, Forschner Andrea, Stenzl Arnulf, Kohlbacher Oliver, Nahnsen Sven, Gabernet Gisela, Fillinger Sven, Bender Benjamin, Ernemann Ulrike, Öner Öznur, Beha Janina, Malek Holly Sundberg, Möller Yvonne, Ruhm Kristina, Tatagiba Marcos, Schittenhelm Jens, Bitzer Michael, Malek Nisar, Zips Daniel, Tabatabai Ghazaleh
Department of Neurology and Interdisciplinary Neuro-Oncology, Hertie Institute for Clinical Brain Research, Eberhard Karls University Tübingen, Germany.
Center for Neuro-Oncology, Comprehensive Cancer Center Tübingen-Stuttgart, University Hospital Tübingen, Germany.
Neurooncol Adv. 2023 Feb 14;5(1):vdad012. doi: 10.1093/noajnl/vdad012. eCollection 2023 Jan-Dec.
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.
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.
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.
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.
在临床试验之外，分子图谱分析和靶向治疗对神经肿瘤患者的临床效用尚未确立。我们旨在通过一项前瞻性观察性研究，调查分子图谱分析和靶向治疗在成年晚期神经系统肿瘤患者中的可行性和临床效用。
分子肿瘤委员会（MTB）@ZPM（NCT03503149）是一项针对晚期肿瘤患者的前瞻性观察性精准医学研究。纳入患者后，我们进行了全面的分子图谱分析，根据分子肿瘤委员会的共识制定了按优先级排序的生物标志物指导治疗建议，并收集了前瞻性临床结局数据。
在此，我们展示了截至2021年12月31日入组的661例成年神经系统肿瘤患者的初始数据。其中，408例患者在分子肿瘤委员会进行了讨论。在380/408（93.1%）的病例中可以做出分子指导的治疗建议，并根据证据水平进行优先级排序。截至数据截止时，86/380（22.6%）的病例开始了治疗。我们观察到31.3%的患者无进展生存率>1.3。
我们的研究支持生物标志物指导的治疗对神经肿瘤患者的临床效用，并表明在一部分患者中具有临床益处。我们的数据可能为未来的临床试验、转化研究乃至临床护理提供参考。
