Ex vivo drug response profiling for response and outcome prediction in hematologic malignancies: the prospective non-interventional SMARTrial
Ex vivo drug response profiling is a powerful tool to study genotype-drug response associations and is being explored as a tool set for precision medicine in cancer. Here we conducted a prospective non-interventional trial to investigate feasibility of ex vivo drug response profiling for treatment g...
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| Hauptverfasser: | , , , , , , , , , , , , , , , , , , , , , , , |
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| Dokumenttyp: | Article (Journal) |
| Sprache: | Englisch |
| Veröffentlicht: |
02 October 2023
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| In: |
Nature cancer
Year: 2023, Jahrgang: 4, Heft: 12, Pages: 1648-1659, [1-15], 1-4 |
| ISSN: | 2662-1347 |
| DOI: | 10.1038/s43018-023-00645-5 |
| Online-Zugang: | Verlag, kostenfrei, Volltext: https://doi.org/10.1038/s43018-023-00645-5 Verlag, kostenfrei, Volltext: https://www.nature.com/articles/s43018-023-00645-5 
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| Verfasserangaben: | Nora Liebers, Peter-Martin Bruch, Tobias Terzer, Miguel Hernandez-Hernandez, Nagarajan Paramasivam, Donnacha Fitzgerald, Heidi Altmann, Tobias Roider, Carolin Kolb, Mareike Knoll, Angela Lenze, Uwe Platzbecker, Christoph Röllig, Claudia Baldus, Hubert Serve, Martin Bornhäuser, Daniel Hübschmann, Carsten Müller-Tidow, Friedrich Stölzel, Wolfgang Huber, Axel Benner, Thorsten Zenz, Junyan Lu & Sascha Dietrich |
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| 245 | 1 | 0 | |a Ex vivo drug response profiling for response and outcome prediction in hematologic malignancies |b the prospective non-interventional SMARTrial |c Nora Liebers, Peter-Martin Bruch, Tobias Terzer, Miguel Hernandez-Hernandez, Nagarajan Paramasivam, Donnacha Fitzgerald, Heidi Altmann, Tobias Roider, Carolin Kolb, Mareike Knoll, Angela Lenze, Uwe Platzbecker, Christoph Röllig, Claudia Baldus, Hubert Serve, Martin Bornhäuser, Daniel Hübschmann, Carsten Müller-Tidow, Friedrich Stölzel, Wolfgang Huber, Axel Benner, Thorsten Zenz, Junyan Lu & Sascha Dietrich |
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| 520 | |a Ex vivo drug response profiling is a powerful tool to study genotype-drug response associations and is being explored as a tool set for precision medicine in cancer. Here we conducted a prospective non-interventional trial to investigate feasibility of ex vivo drug response profiling for treatment guidance in hematologic malignancies (SMARTrial, NCT03488641). The primary endpoint to provide drug response profiling reports within 7 d was met in 91% of all study participants (N = 80). Secondary endpoint analysis revealed that ex vivo resistance to chemotherapeutic drugs predicted chemotherapy treatment failure in vivo. We confirmed the predictive value of ex vivo response to chemotherapy in a validation cohort of 95 individuals with acute myeloid leukemia treated with daunorubicin and cytarabine. Ex vivo drug response profiles improved ELN-22 risk stratification in individuals with adverse risk. We conclude that ex vivo drug response profiling is clinically feasible and has the potential to predict chemotherapy response in individuals with hematologic malignancies beyond clinically established genetic markers. | ||
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