Journal Article

DKFZ-2023-01992

http://join2-wiki.gsi.de/foswiki/pub/Main/Artwork/join2_logo100x88.png Ex vivo drug response profiling for response and outcome prediction in hematologic malignancies: the prospective non-interventional SMARTrial.

Liebers, N. (First author)DKFZ* ; Bruch, P.-M. ; Terzer, T.DKFZ* ; Hernandez-Hernandez, M. ; Paramasivam, N. ; Fitzgerald, D. ; Altmann, H. ; Roider, T. ; Kolb, C. ; Knoll, M. ; Lenze, A. ; Platzbecker, U. ; Röllig, C. ; Baldus, C. ; Serve, H. ; Bornhäuser, M. ; Hübschmann, D.DKFZ* ; Müller-Tidow, C.DKFZ* ; Stölzel, F. ; Huber, W. ; Benner, A.DKFZ* ; Zenz, T. ; Lu, J. ; Dietrich, S. (Last author)DKFZ*

2023
Nature Research London

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Please use a persistent id in citations: doi:10.1038/s43018-023-00645-5

Abstract: 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.

Note: 2023 Dec;4(12):1648-1659 / #EA:B340#LA:B340#

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Contributing Institute(s):

1. Translationale Medizinische Onkologie (B340)
2. C060 Biostatistik (C060)
3. DKTK HD zentral (HD01)

Research Program(s):

1. 312 - Funktionelle und strukturelle Genomforschung (POF4-312) (POF4-312)

Appears in the scientific report 2023

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Database coverage:
; BIOSIS Previews ; Biological Abstracts ; Clarivate Analytics Master Journal List ; Current Contents - Clinical Medicine ; DEAL Nature ; Essential Science Indicators ; IF >= 20 ; JCR ; SCOPUS ; Science Citation Index Expanded ; Web of Science Core Collection

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