Evidence map›Paper›PMID 34112699›Full record

Observational studyCancer discovery2021

Comprehensive Genomic and Transcriptomic Analysis for Guiding Therapeutic Decisions in Patients with Rare Cancers.

Peter Horak, Christoph Heining, Simon Kreutzfeldt, Barbara Hutter, Andreas Mock, Jennifer Hüllein, Martina Fröhlich, Sebastian Uhrig, Arne Jahn, Andreas Rump and 49 more

Registry-linked trialAbstract readObservational Study
PubMed Publisher
In one paragraph

Observational study in Cancer discovery, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05852522 (NCT MASTER Plattform Molecularly Aided Stratification for Tumor Eradication Research), which is not on this map. Cited by 158 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
158citing papers in PubMed, 2 pooled it
17.0field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT05852522 recruitingnot on this map

NCT MASTER Plattform Molecularly Aided Stratification for Tumor Eradication Research

Typeobservational_patient_registrySponsorGerman Cancer Research CenterRan2018 to 2029Enrolled10,000ConditionsRare Cancers, Multiomics
3 · Its place in the literature

Who cites it

158 citing papers in PubMed, 2 syntheses or guidelines pooled it, 270 citations in OpenAlex.

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98 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

59 authors at 18 institutions in 5 countries.

Peter Horak *Department of Translational Medical Oncology, National Center for Tumor Diseases (NCT) Heidelberg and German Cancer Research Center (DKFZ), Heidelberg, Germany.ORCID 0000-0003-4536-9306
Christoph Heining *Department of Translational Medical Oncology, NCT Dresden and DKFZ, Dresden, Germany.
Simon Kreutzfeldt *Department of Translational Medical Oncology, National Center for Tumor Diseases (NCT) Heidelberg and German Cancer Research Center (DKFZ), Heidelberg, Germany.ORCID 0000-0003-3278-4586
Barbara Hutter *Computational Oncology Group, Molecular Diagnostics Program, NCT Heidelberg and DKFZ, Heidelberg, Germany.ORCID 0000-0002-9034-0329
Andreas Mock *Department of Translational Medical Oncology, National Center for Tumor Diseases (NCT) Heidelberg and German Cancer Research Center (DKFZ), Heidelberg, Germany.ORCID 0000-0002-3332-9166
Jennifer Hüllein *Computational Oncology Group, Molecular Diagnostics Program, NCT Heidelberg and DKFZ, Heidelberg, Germany.
Martina Fröhlich *Computational Oncology Group, Molecular Diagnostics Program, NCT Heidelberg and DKFZ, Heidelberg, Germany.
Sebastian Uhrig *Computational Oncology Group, Molecular Diagnostics Program, NCT Heidelberg and DKFZ, Heidelberg, Germany.ORCID 0000-0002-0068-5957
Arne JahnCenter for Personalized Oncology, University Hospital Carl Gustav Carus, Technical University Dresden, Dresden, Germany.ORCID 0000-0003-1045-1908
Andreas RumpCenter for Personalized Oncology, University Hospital Carl Gustav Carus, Technical University Dresden, Dresden, Germany.ORCID 0000-0001-7116-6364
Laura GieldonInstitute for Clinical Genetics, Faculty of Medicine Carl Gustav Carus, Technical University Dresden, Dresden, Germany.
Lino MöhrmannDepartment of Translational Medical Oncology, NCT Dresden and DKFZ, Dresden, Germany.ORCID 0000-0003-4650-6827
Dorothea HanfDepartment of Translational Medical Oncology, NCT Dresden and DKFZ, Dresden, Germany.
Veronica TeleanuDepartment of Translational Medical Oncology, National Center for Tumor Diseases (NCT) Heidelberg and German Cancer Research Center (DKFZ), Heidelberg, Germany.
Christoph E HeiligDepartment of Translational Medical Oncology, National Center for Tumor Diseases (NCT) Heidelberg and German Cancer Research Center (DKFZ), Heidelberg, Germany.ORCID 0000-0001-8869-1421
Daniel B LipkaDepartment of Translational Medical Oncology, National Center for Tumor Diseases (NCT) Heidelberg and German Cancer Research Center (DKFZ), Heidelberg, Germany.ORCID 0000-0001-5081-7869
Michael AllgäuerInstitute of Pathology, Heidelberg University Hospital, Heidelberg, Germany.ORCID 0000-0003-4518-7887
Leo RuhnkeDepartment of Translational Medical Oncology, NCT Dresden and DKFZ, Dresden, Germany.
Andreas LaßmannDepartment of Translational Medical Oncology, National Center for Tumor Diseases (NCT) Heidelberg and German Cancer Research Center (DKFZ), Heidelberg, Germany.
Volker EndrisInstitute of Pathology, Heidelberg University Hospital, Heidelberg, Germany.
Olaf NeumannInstitute of Pathology, Heidelberg University Hospital, Heidelberg, Germany.ORCID 0000-0003-2684-9187
Roland PenzelInstitute of Pathology, Heidelberg University Hospital, Heidelberg, Germany.
Katja BeckDepartment of Translational Medical Oncology, National Center for Tumor Diseases (NCT) Heidelberg and German Cancer Research Center (DKFZ), Heidelberg, Germany.
Daniela RichterDepartment of Translational Medical Oncology, NCT Dresden and DKFZ, Dresden, Germany.
Ulrike WinterDepartment of Translational Medical Oncology, National Center for Tumor Diseases (NCT) Heidelberg and German Cancer Research Center (DKFZ), Heidelberg, Germany.
Stephan WolfGerman Cancer Consortium (DKTK), Heidelberg, Germany.
Katrin PfützeGerman Cancer Consortium (DKTK), Heidelberg, Germany.
Christina GeörgGerman Cancer Consortium (DKTK), Heidelberg, Germany.
Bettina MeißburgerGerman Cancer Consortium (DKTK), Heidelberg, Germany.
Ivo BuchhalterOmics IT and Data Management Core Facility, DKFZ, Heidelberg, Germany.ORCID 0000-0003-0764-5832
Marinela AugustinDepartment of Hematology and Oncology, Klinikum Nuremberg, Paracelsus Medical University, Nuremberg, Germany.ORCID 0000-0003-0494-0422
Walter E AulitzkyDepartment of Hematology, Oncology and Palliative Medicine, Robert Bosch Hospital, Stuttgart, Germany.
Peter HohenbergerSarcoma Unit, Interdisciplinary Tumor Center Mannheim, Mannheim University Medical Center, Heidelberg University, Mannheim, Germany.
Matthias KroissDepartment of Medicine I, Würzburg University Hospital, Würzburg, Germany.ORCID 0000-0002-7628-3161
Peter SchirmacherGerman Cancer Consortium (DKTK), Heidelberg, Germany.
Richard F SchlenkGerman Cancer Consortium (DKTK), Heidelberg, Germany.ORCID 0000-0003-2215-2059
Ulrich KeilholzCharité Comprehensive Cancer Center, Charité - Universitätsmedizin Berlin, Berlin, Germany.
Frederick KlauschenDKTK, Berlin, Germany.
Gunnar FolprechtDKTK, Dresden, Germany.ORCID 0000-0002-9321-9911
Sebastian BauerDepartment of Medical Oncology, West German Cancer Center, University Hospital Essen, Essen, Germany.ORCID 0000-0001-5949-8120
Jens Thomas SivekeDKTK, Essen, Germany.ORCID 0000-0002-8772-4778
Christian H BrandtsUniversity Cancer Center (UCT) Frankfurt, University Hospital Frankfurt, Goethe University, Frankfurt, Germany.
Thomas KindlerUCT Mainz, Johannes Gutenberg University Mainz, Mainz, Germany.
Melanie BoerriesComprehensive Cancer Center Freiburg, University of Freiburg Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany.ORCID 0000-0002-3670-0602
Anna L IllertComprehensive Cancer Center Freiburg, University of Freiburg Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
Nikolas von BubnoffDepartment of Internal Medicine I, University of Freiburg Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
Philipp J JostDepartment of Hematology and Oncology, Klinikum rechts der Isar, Technical University Munich, Munich, Germany.ORCID 0000-0003-2454-0362
Karsten SpiekermannDKTK, Munich, Germany.ORCID 0000-0002-5139-4957
Michael BitzerDepartment of Internal Medicine I, University Hospital Tübingen, Tübingen, Germany.
Klaus Schulze-OsthoffDKTK, Tübingen, Germany.
Christof von KalleClinical Study Center, Charité - Universitätsmedizin Berlin, and Berlin Institute of Health, Berlin, Germany.ORCID 0000-0001-9221-3297
Barbara KlinkCenter for Personalized Oncology, University Hospital Carl Gustav Carus, Technical University Dresden, Dresden, Germany.ORCID 0000-0002-8753-8238
Benedikt BrorsGerman Cancer Consortium (DKTK), Heidelberg, Germany.ORCID 0000-0001-5940-3101
Albrecht StenzingerGerman Cancer Consortium (DKTK), Heidelberg, Germany.
Evelin SchröckCenter for Personalized Oncology, University Hospital Carl Gustav Carus, Technical University Dresden, Dresden, Germany.
Daniel HübschmannGerman Cancer Consortium (DKTK), Heidelberg, Germany.
Wilko WeichertDKTK, Munich, Germany.
Hanno Glimm *Department of Translational Medical Oncology, NCT Dresden and DKFZ, Dresden, Germany. stefan.froehling@nct-heidelberg.de hanno.glimm@nct-dresden.de.
Stefan Fröhling *Department of Translational Medical Oncology, National Center for Tumor Diseases (NCT) Heidelberg and German Cancer Research Center (DKFZ), Heidelberg, Germany. stefan.froehling@nct-heidelberg.de hanno.glimm@nct-dresden.de.ORCID 0000-0001-7907-4595
German Cancer Research Center · DEHeidelberg University · DEKrankenhaus Dresden-Friedrichstadt · DEVKTA · DEUniversity of Freiburg · DEElekta (Switzerland) · CHSRH Hochschule der Populären Künste · DEBerlin Institute of Health at Charité - Universitätsmedizin Berlin · DEComprehensive Cancer Center Mainfranken · DEEssen University Hospital · DEGoethe University Frankfurt · DELuxembourg Institute of Health · LUMedical University of Graz · ATNuremberg Hospital · DERobert Bosch Hospital · DEUniversity Children's Hospital Tübingen · DEUniversity of Applied Sciences Mainz · DEUniversity of Tübingen · DE

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The clinical relevance of comprehensive molecular analysis in rare cancers is not established. We analyzed the molecular profiles and clinical outcomes of 1,310 patients (rare cancers, 75.5%) enrolled in a prospective observational study by the German Cancer Consortium that applies whole-genome/exome and RNA sequencing to inform the care of adults with incurable cancers. On the basis of 472 single and six composite biomarkers, a cross-institutional molecular tumor board provided evidence-based management recommendations, including diagnostic reevaluation, genetic counseling, and experimental treatment, in 88% of cases. Recommended therapies were administered in 362 of 1,138 patients (31.8%) and resulted in significantly improved overall response and disease control rates (23.9% and 55.3%) compared with previous therapies, translating into a progression-free survival ratio >1.3 in 35.7% of patients. These data demonstrate the benefit of molecular stratification in rare cancers and represent a resource that may promote clinical trial access and drug approvals in this underserved patient population. SIGNIFICANCE: Rare cancers are difficult to treat; in particular, molecular pathogenesis-oriented medical therapies are often lacking. This study shows that whole-genome/exome and RNA sequencing enables molecularly informed treatments that lead to clinical benefit in a substantial proportion of patients with advanced rare cancers and paves the way for future clinical trials.

Indexed as

NeoplasmsTranscriptomeAdultExome SequencingGene Expression ProfilingGenomicsHumans

Identifiers

PMID34112699
OpenAlexW3171689169

What OpenQuestion holds

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.