Clinical outcome of biomarker-guided therapies in adult patients with tumors of the nervous system
Abstract 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 tum...
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| Veröffentlicht in: | Neuro-oncology advances 2023-01, Vol.5 (1), p.vdad012-vdad012 |
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| creator | 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 |
| description | Abstract
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. |
| doi_str_mv | 10.1093/noajnl/vdad012 |
| format | Article |
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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.</description><identifier>ISSN: 2632-2498</identifier><identifier>EISSN: 2632-2498</identifier><identifier>DOI: 10.1093/noajnl/vdad012</identifier><identifier>PMID: 36915613</identifier><language>eng</language><publisher>US: Oxford University Press</publisher><subject>Clinical Investigations</subject><ispartof>Neuro-oncology advances, 2023-01, Vol.5 (1), p.vdad012-vdad012</ispartof><rights>The Author(s) 2023. Published by Oxford University Press, the Society for Neuro-Oncology and the European Association of Neuro-Oncology. 2023</rights><rights>The Author(s) 2023. Published by Oxford University Press, the Society for Neuro-Oncology and the European Association of Neuro-Oncology.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c425t-a4f0ee510fe46a19bbfb31fd442e4e32e54cac9e6b06532ee53cefa8a141d0363</citedby><cites>FETCH-LOGICAL-c425t-a4f0ee510fe46a19bbfb31fd442e4e32e54cac9e6b06532ee53cefa8a141d0363</cites><orcidid>0000-0002-4375-0691 ; 0000-0002-9168-6209 ; 0000-0003-1739-4598 ; 0000-0001-5162-1542 ; 0000-0002-5496-293X ; 0000-0002-3542-8782</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC10007909/pdf/$$EPDF$$P50$$Gpubmedcentral$$Hfree_for_read</linktopdf><linktohtml>$$Uhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC10007909/$$EHTML$$P50$$Gpubmedcentral$$Hfree_for_read</linktohtml><link.rule.ids>230,314,723,776,780,860,881,1598,27901,27902,53766,53768</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/36915613$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Renovanz, Mirjam</creatorcontrib><creatorcontrib>Kurz, Sylvia C</creatorcontrib><creatorcontrib>Rieger, Johannes</creatorcontrib><creatorcontrib>Walter, Bianca</creatorcontrib><creatorcontrib>Becker, Hannes</creatorcontrib><creatorcontrib>Hille, Hanni</creatorcontrib><creatorcontrib>Bombach, Paula</creatorcontrib><creatorcontrib>Rieger, David</creatorcontrib><creatorcontrib>Grosse, Lucia</creatorcontrib><creatorcontrib>Häusser, Lara</creatorcontrib><creatorcontrib>Skardelly, Marco</creatorcontrib><creatorcontrib>Merk, Daniel J</creatorcontrib><creatorcontrib>Paulsen, Frank</creatorcontrib><creatorcontrib>Hoffmann, Elgin</creatorcontrib><creatorcontrib>Gani, Cihan</creatorcontrib><creatorcontrib>Neumann, Manuela</creatorcontrib><creatorcontrib>Beschorner, Rudi</creatorcontrib><creatorcontrib>Rieß, Olaf</creatorcontrib><creatorcontrib>Roggia, Cristiana</creatorcontrib><creatorcontrib>Schroeder, Christopher</creatorcontrib><creatorcontrib>Ossowski, Stephan</creatorcontrib><creatorcontrib>Armeanu-Ebinger, Sorin</creatorcontrib><creatorcontrib>Gschwind, Axel</creatorcontrib><creatorcontrib>Biskup, Saskia</creatorcontrib><creatorcontrib>Schulze, Martin</creatorcontrib><creatorcontrib>Fend, Falko</creatorcontrib><creatorcontrib>Singer, Stephan</creatorcontrib><creatorcontrib>Zender, Lars</creatorcontrib><creatorcontrib>Lengerke, Claudia</creatorcontrib><creatorcontrib>Brucker, Sara Yvonne</creatorcontrib><creatorcontrib>Engler, Tobias</creatorcontrib><creatorcontrib>Forschner, Andrea</creatorcontrib><creatorcontrib>Stenzl, Arnulf</creatorcontrib><creatorcontrib>Kohlbacher, Oliver</creatorcontrib><creatorcontrib>Nahnsen, Sven</creatorcontrib><creatorcontrib>Gabernet, Gisela</creatorcontrib><creatorcontrib>Fillinger, Sven</creatorcontrib><creatorcontrib>Bender, Benjamin</creatorcontrib><creatorcontrib>Ernemann, Ulrike</creatorcontrib><creatorcontrib>Öner, Öznur</creatorcontrib><creatorcontrib>Beha, Janina</creatorcontrib><creatorcontrib>Malek, Holly Sundberg</creatorcontrib><creatorcontrib>Möller, Yvonne</creatorcontrib><creatorcontrib>Ruhm, Kristina</creatorcontrib><creatorcontrib>Tatagiba, Marcos</creatorcontrib><creatorcontrib>Schittenhelm, Jens</creatorcontrib><creatorcontrib>Bitzer, Michael</creatorcontrib><creatorcontrib>Malek, Nisar</creatorcontrib><creatorcontrib>Zips, Daniel</creatorcontrib><creatorcontrib>Tabatabai, Ghazaleh</creatorcontrib><title>Clinical outcome of biomarker-guided therapies in adult patients with tumors of the nervous system</title><title>Neuro-oncology advances</title><addtitle>Neurooncol Adv</addtitle><description>Abstract
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.</description><subject>Clinical Investigations</subject><issn>2632-2498</issn><issn>2632-2498</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2023</creationdate><recordtype>article</recordtype><sourceid>TOX</sourceid><recordid>eNqFkbtPwzAQxi0Eoqh0ZUQeYQj1K24zIVTxkiqxwGw5zqU1JHGwnSL-e1y1IJiYzif_7rvHh9AZJVeUFHzaOf3aNdNNpStC2QE6YZKzjIlifvjrPUKTEF4JISwXuSDsGI24LGguKT9B5aKxnTW6wW6IxrWAXY1L61rt38Bnq8FWUOG4Bq97CwHbDutqaCLudbTQxYA_bFzjOLTOh21tQnEHfuOGgMNniNCeoqNaNwEm-zhGL3e3z4uHbPl0_7i4WWZGsDxmWtQEIKekBiE1LcqyLjmtKyEYCOAMcmG0KUCWROYphZwbqPVcU0ErwiUfo-udbj-ULVQmTed1o3pv0zKfymmr_v50dq1WbqNous2sSAcdo4u9gnfvA4SoWhsMNI3uIO2j2Gwu51QwuUWvdqjxLgQP9U8fStTWHLUzR-3NSQXnv6f7wb-tSMDlDnBD_5_YF0mGnss</recordid><startdate>20230101</startdate><enddate>20230101</enddate><creator>Renovanz, Mirjam</creator><creator>Kurz, Sylvia C</creator><creator>Rieger, Johannes</creator><creator>Walter, Bianca</creator><creator>Becker, Hannes</creator><creator>Hille, Hanni</creator><creator>Bombach, Paula</creator><creator>Rieger, David</creator><creator>Grosse, Lucia</creator><creator>Häusser, Lara</creator><creator>Skardelly, Marco</creator><creator>Merk, Daniel J</creator><creator>Paulsen, Frank</creator><creator>Hoffmann, Elgin</creator><creator>Gani, Cihan</creator><creator>Neumann, Manuela</creator><creator>Beschorner, Rudi</creator><creator>Rieß, Olaf</creator><creator>Roggia, Cristiana</creator><creator>Schroeder, Christopher</creator><creator>Ossowski, Stephan</creator><creator>Armeanu-Ebinger, Sorin</creator><creator>Gschwind, Axel</creator><creator>Biskup, Saskia</creator><creator>Schulze, Martin</creator><creator>Fend, Falko</creator><creator>Singer, Stephan</creator><creator>Zender, Lars</creator><creator>Lengerke, Claudia</creator><creator>Brucker, Sara Yvonne</creator><creator>Engler, Tobias</creator><creator>Forschner, Andrea</creator><creator>Stenzl, Arnulf</creator><creator>Kohlbacher, Oliver</creator><creator>Nahnsen, Sven</creator><creator>Gabernet, Gisela</creator><creator>Fillinger, Sven</creator><creator>Bender, Benjamin</creator><creator>Ernemann, Ulrike</creator><creator>Öner, Öznur</creator><creator>Beha, Janina</creator><creator>Malek, Holly Sundberg</creator><creator>Möller, Yvonne</creator><creator>Ruhm, Kristina</creator><creator>Tatagiba, Marcos</creator><creator>Schittenhelm, Jens</creator><creator>Bitzer, Michael</creator><creator>Malek, Nisar</creator><creator>Zips, Daniel</creator><creator>Tabatabai, Ghazaleh</creator><general>Oxford University Press</general><scope>TOX</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7X8</scope><scope>5PM</scope><orcidid>https://orcid.org/0000-0002-4375-0691</orcidid><orcidid>https://orcid.org/0000-0002-9168-6209</orcidid><orcidid>https://orcid.org/0000-0003-1739-4598</orcidid><orcidid>https://orcid.org/0000-0001-5162-1542</orcidid><orcidid>https://orcid.org/0000-0002-5496-293X</orcidid><orcidid>https://orcid.org/0000-0002-3542-8782</orcidid></search><sort><creationdate>20230101</creationdate><title>Clinical outcome of biomarker-guided therapies in adult patients with tumors of the nervous system</title><author>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</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c425t-a4f0ee510fe46a19bbfb31fd442e4e32e54cac9e6b06532ee53cefa8a141d0363</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2023</creationdate><topic>Clinical Investigations</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Renovanz, Mirjam</creatorcontrib><creatorcontrib>Kurz, 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Lucia</au><au>Häusser, Lara</au><au>Skardelly, Marco</au><au>Merk, Daniel J</au><au>Paulsen, Frank</au><au>Hoffmann, Elgin</au><au>Gani, Cihan</au><au>Neumann, Manuela</au><au>Beschorner, Rudi</au><au>Rieß, Olaf</au><au>Roggia, Cristiana</au><au>Schroeder, Christopher</au><au>Ossowski, Stephan</au><au>Armeanu-Ebinger, Sorin</au><au>Gschwind, Axel</au><au>Biskup, Saskia</au><au>Schulze, Martin</au><au>Fend, Falko</au><au>Singer, Stephan</au><au>Zender, Lars</au><au>Lengerke, Claudia</au><au>Brucker, Sara Yvonne</au><au>Engler, Tobias</au><au>Forschner, Andrea</au><au>Stenzl, Arnulf</au><au>Kohlbacher, Oliver</au><au>Nahnsen, Sven</au><au>Gabernet, Gisela</au><au>Fillinger, Sven</au><au>Bender, Benjamin</au><au>Ernemann, Ulrike</au><au>Öner, Öznur</au><au>Beha, Janina</au><au>Malek, Holly Sundberg</au><au>Möller, Yvonne</au><au>Ruhm, Kristina</au><au>Tatagiba, Marcos</au><au>Schittenhelm, Jens</au><au>Bitzer, Michael</au><au>Malek, Nisar</au><au>Zips, Daniel</au><au>Tabatabai, Ghazaleh</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Clinical outcome of biomarker-guided therapies in adult patients with tumors of the nervous system</atitle><jtitle>Neuro-oncology advances</jtitle><addtitle>Neurooncol Adv</addtitle><date>2023-01-01</date><risdate>2023</risdate><volume>5</volume><issue>1</issue><spage>vdad012</spage><epage>vdad012</epage><pages>vdad012-vdad012</pages><issn>2632-2498</issn><eissn>2632-2498</eissn><abstract>Abstract
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.</abstract><cop>US</cop><pub>Oxford University Press</pub><pmid>36915613</pmid><doi>10.1093/noajnl/vdad012</doi><orcidid>https://orcid.org/0000-0002-4375-0691</orcidid><orcidid>https://orcid.org/0000-0002-9168-6209</orcidid><orcidid>https://orcid.org/0000-0003-1739-4598</orcidid><orcidid>https://orcid.org/0000-0001-5162-1542</orcidid><orcidid>https://orcid.org/0000-0002-5496-293X</orcidid><orcidid>https://orcid.org/0000-0002-3542-8782</orcidid><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 2632-2498 |
| ispartof | Neuro-oncology advances, 2023-01, Vol.5 (1), p.vdad012-vdad012 |
| issn | 2632-2498 2632-2498 |
| language | eng |
| recordid | cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_10007909 |
| source | Oxford Journals Open Access Collection; DOAJ Directory of Open Access Journals; Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals; PubMed Central |
| subjects | Clinical Investigations |
| title | Clinical outcome of biomarker-guided therapies in adult patients with tumors of the nervous system |
| url | https://sfx.bib-bvb.de/sfx_tum?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2025-02-03T21%3A22%3A19IST&url_ver=Z39.88-2004&url_ctx_fmt=infofi/fmt:kev:mtx:ctx&rfr_id=info:sid/primo.exlibrisgroup.com:primo3-Article-proquest_pubme&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.genre=article&rft.atitle=Clinical%20outcome%20of%20biomarker-guided%20therapies%20in%20adult%20patients%20with%20tumors%20of%20the%20nervous%20system&rft.jtitle=Neuro-oncology%20advances&rft.au=Renovanz,%20Mirjam&rft.date=2023-01-01&rft.volume=5&rft.issue=1&rft.spage=vdad012&rft.epage=vdad012&rft.pages=vdad012-vdad012&rft.issn=2632-2498&rft.eissn=2632-2498&rft_id=info:doi/10.1093/noajnl/vdad012&rft_dat=%3Cproquest_pubme%3E2786814269%3C/proquest_pubme%3E%3Curl%3E%3C/url%3E&disable_directlink=true&sfx.directlink=off&sfx.report_link=0&rft_id=info:oai/&rft_pqid=2786814269&rft_id=info:pmid/36915613&rft_oup_id=10.1093/noajnl/vdad012&rfr_iscdi=true |