The Pediatric Precision Oncology INFORM Registry: Clinical Outcome and Benefit for Patients with Very High-Evidence Targets.


Journal

Cancer discovery
ISSN: 2159-8290
Titre abrégé: Cancer Discov
Pays: United States
ID NLM: 101561693

Informations de publication

Date de publication:
11 2021
Historique:
received: 22 01 2021
revised: 26 05 2021
accepted: 28 07 2021
pubmed: 11 8 2021
medline: 17 3 2022
entrez: 10 8 2021
Statut: ppublish

Résumé

INFORM is a prospective, multinational registry gathering clinical and molecular data of relapsed, progressive, or high-risk pediatric patients with cancer. This report describes long-term follow-up of 519 patients in whom molecular alterations were evaluated according to a predefined seven-scale target prioritization algorithm. Mean turnaround time from sample receipt to report was 25.4 days. The highest target priority level was observed in 42 patients (8.1%). Of these, 20 patients received matched targeted treatment with a median progression-free survival of 204 days [95% confidence interval (CI), 99-not applicable], compared with 117 days (95% CI, 106-143;

Identifiants

pubmed: 34373263
pii: 2159-8290.CD-21-0094
doi: 10.1158/2159-8290.CD-21-0094
pmc: PMC9414287
doi:

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

2764-2779

Commentaires et corrections

Type : CommentIn

Informations de copyright

©2021 The Authors; Published by the American Association for Cancer Research.

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Auteurs

Cornelis M van Tilburg (CM)

Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany. cornelis.vantilburg@kitz-heidelberg.de.
Clinical Cooperation Unit Pediatric Oncology, German Cancer Research Center (DKFZ), Heidelberg, Germany.
Department of Pediatric Oncology, Hematology, Immunology and Pulmonology, Heidelberg University Hospital, Heidelberg, Germany.
German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.

Elke Pfaff (E)

Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
Department of Pediatric Oncology, Hematology, Immunology and Pulmonology, Heidelberg University Hospital, Heidelberg, Germany.
German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Pediatric Glioma Research Group, German Cancer Research Center (DKFZ), Heidelberg, Germany.

Kristian W Pajtler (KW)

Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
Department of Pediatric Oncology, Hematology, Immunology and Pulmonology, Heidelberg University Hospital, Heidelberg, Germany.
German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Division of Pediatric Neurooncology, German Cancer Research Center (DKFZ), Heidelberg, Germany.

Karin P S Langenberg (KPS)

Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands.

Petra Fiesel (P)

German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Clinical Cooperation Unit Neuropathology, German Cancer Research Center (DKFZ), Heidelberg, Germany.
Department of Neuropathology, Institute of Pathology, Heidelberg University Hospital, Heidelberg, Germany.

Barbara C Jones (BC)

Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
Department of Pediatric Oncology, Hematology, Immunology and Pulmonology, Heidelberg University Hospital, Heidelberg, Germany.
German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Pediatric Glioma Research Group, German Cancer Research Center (DKFZ), Heidelberg, Germany.

Gnana Prakash Balasubramanian (GP)

Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Division of Pediatric Neurooncology, German Cancer Research Center (DKFZ), Heidelberg, Germany.

Sebastian Stark (S)

Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
Department of Pediatric Oncology, Hematology, Immunology and Pulmonology, Heidelberg University Hospital, Heidelberg, Germany.
German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Pediatric Glioma Research Group, German Cancer Research Center (DKFZ), Heidelberg, Germany.

Pascal D Johann (PD)

Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
Department of Pediatric Oncology, Hematology, Immunology and Pulmonology, Heidelberg University Hospital, Heidelberg, Germany.
German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Division of Pediatric Neurooncology, German Cancer Research Center (DKFZ), Heidelberg, Germany.
Swabian Children's Cancer Center, Paediatric and Adolescent Medicine, University Medical Center Augsburg, Augsburg, Germany.

Mirjam Blattner-Johnson (M)

Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Pediatric Glioma Research Group, German Cancer Research Center (DKFZ), Heidelberg, Germany.

Kathrin Schramm (K)

Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Pediatric Glioma Research Group, German Cancer Research Center (DKFZ), Heidelberg, Germany.

Nicola Dikow (N)

Institute of Human Genetics, Heidelberg University Hospital, Heidelberg, Germany.

Steffen Hirsch (S)

Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
Institute of Human Genetics, Heidelberg University Hospital, Heidelberg, Germany.

Christian Sutter (C)

Institute of Human Genetics, Heidelberg University Hospital, Heidelberg, Germany.

Kerstin Grund (K)

Institute of Human Genetics, Heidelberg University Hospital, Heidelberg, Germany.

Arend von Stackelberg (A)

German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Pediatric Oncology and Hematology Department, Charité-Campus Virchow Klinikum, Berlin, Germany.

Andreas E Kulozik (AE)

Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
Department of Pediatric Oncology, Hematology, Immunology and Pulmonology, Heidelberg University Hospital, Heidelberg, Germany.
German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Clinical Cooperation Unit Pediatric Leukemia, German Cancer Research Center (DKFZ), Heidelberg, Germany.

Andrej Lissat (A)

German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Pediatric Oncology and Hematology Department, Charité-Campus Virchow Klinikum, Berlin, Germany.

Arndt Borkhardt (A)

German Cancer Consortium (DKTK), Heidelberg, Germany.
Department of Pediatric Oncology and Hematology, Medical Faculty, Heinrich Heine University, Düsseldorf, Germany.

Roland Meisel (R)

German Cancer Consortium (DKTK), Heidelberg, Germany.
Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf, Germany.

Dirk Reinhardt (D)

German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
West German Cancer Center, Pediatrics III, University Hospital Essen, Essen, Germany.

Jan-Henning Klusmann (JH)

Department of Pediatric Hematology, Oncology and Hemostaseology, Clinic for Pediatrics, University Hospital of Frankfurt, Goethe-University Frankfurt, Frankfurt/Main, Germany.

Gudrun Fleischhack (G)

German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
West German Cancer Center, Pediatrics III, University Hospital Essen, Essen, Germany.

Stephan Tippelt (S)

German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
West German Cancer Center, Pediatrics III, University Hospital Essen, Essen, Germany.

Dietrich von Schweinitz (D)

German Cancer Consortium (DKTK), Heidelberg, Germany.
Department of Pediatric Surgery, Dr. von Hauner Children's Hospital, Ludwig-Maximilians-University Munich, Munich, Germany.

Irene Schmid (I)

Department of Pediatric Oncology and Hematology, Dr. von Hauner Children's Hospital, Ludwig-Maximilians-University Munich, Munich, Germany.

Christof M Kramm (CM)

Division of Pediatric Hematology and Oncology, University Medical Center Göttingen, Göttingen, Germany.

André O von Bueren (AO)

Department of Pediatrics, Obstetrics and Gynecology, Division of Pediatric Hematology and Oncology, University Hospital Geneva, Geneva, Switzerland.

Gabriele Calaminus (G)

Department of Pediatric Oncology and Hematology, University Hospital Bonn, Bonn, Germany.

Peter Vorwerk (P)

Department of Pediatric Oncology and Hematology, University Hospital Magdeburg, Magdeburg, Germany.

Norbert Graf (N)

Department of Pediatric Oncology and Hematology, University Hospital Saarland, Saarland, Germany.

Frank Westermann (F)

German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Division of Neuroblastoma Genomics, German Cancer Research Center (DKFZ), Heidelberg, Germany.

Matthias Fischer (M)

National Center for Tumor Diseases (NCT) Network, Germany.
Department of Experimental Pediatric Oncology, University Hospital Köln, and Center for Molecular Medicine (CMMC), Medical Faculty, Cologne, Germany.

Angelika Eggert (A)

German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Pediatric Oncology and Hematology Department, Charité-Campus Virchow Klinikum, Berlin, Germany.

Birgit Burkhardt (B)

Clinic of Pediatric Oncology and Hematology, University Hospital Münster, Muenster, Germany.

Wilhelm Wößmann (W)

Department of Pediatric Oncology and Hematology, University Hospital Hamburg, Hamburg, Germany.

Michaela Nathrath (M)

German Cancer Consortium (DKTK), Heidelberg, Germany.
Department of Pediatric Hematology and Oncology, Klinikum Kassel, Kassel, Germany.
Department of Pediatrics, Technical University Munich, Munich, Germany.

Stefanie Hecker-Nolting (S)

National Center for Tumor Diseases (NCT) Network, Germany.
Department of Pediatric Oncology, Hematology and Immunology, Klinikum Stuttgart, Olgahospital, Stuttgart, Germany.

Michael C Frühwald (MC)

National Center for Tumor Diseases (NCT) Network, Germany.
Swabian Children's Cancer Center, Paediatric and Adolescent Medicine, University Medical Center Augsburg, Augsburg, Germany.

Dominik T Schneider (DT)

Clinic of Pediatrics, Municipal Hospital Dortmund, Dortmund, Germany.

Ines B Brecht (IB)

German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Department of Pediatric Oncology and Hematology, University Hospital Tübingen, Tübingen, Germany.

Petra Ketteler (P)

German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
West German Cancer Center, Pediatrics III, University Hospital Essen, Essen, Germany.

Simone Fulda (S)

German Cancer Consortium (DKTK), Heidelberg, Germany.
Institute for Experimental Cancer Research in Pediatrics, Goethe University Frankfurt, Frankfurt, Germany.

Ewa Koscielniak (E)

National Center for Tumor Diseases (NCT) Network, Germany.
Department of Pediatric Oncology, Hematology and Immunology, Klinikum Stuttgart, Olgahospital, Stuttgart, Germany.

Michael T Meister (MT)

German Cancer Consortium (DKTK), Heidelberg, Germany.
Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands.

Monika Scheer (M)

German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Pediatric Oncology and Hematology Department, Charité-Campus Virchow Klinikum, Berlin, Germany.

Simone Hettmer (S)

German Cancer Consortium (DKTK), Heidelberg, Germany.
Department of Pediatrics and Adolescent Medicine, Division of Pediatric Hematology and Oncology, University Medical Center Freiburg, Faculty of Medicine, University of Freiburg, Freiburg, Germany.

Matthias Schwab (M)

German Cancer Consortium (DKTK), Heidelberg, Germany.
Dr. Margarete Fischer-Bosch Institute of Clinical Pharmacology, Stuttgart, Germany.
Departments of Clinical Pharmacology, Pharmacy and Biochemistry, and Cluster of Excellence iFIT (EXC 2180) "Image-Guided and Functionally Instructed Tumor Therapies," University of Tübingen, Tübingen, Germany.

Roman Tremmel (R)

Dr. Margarete Fischer-Bosch Institute of Clinical Pharmacology, Stuttgart, Germany.

Ingrid Øra (I)

Department of Pediatric Oncology and Hematology, Skane University Hospital Lund, and HOPE-ITCC Unit, Pediatric Oncology, Karolinska University Hospital, Stockholm, Sweden.

Caroline Hutter (C)

St. Anna Children's Hospital, Department of Pediatrics, Medical University of Vienna, and St. Anna Children's Cancer Research Institute (CCRI), Vienna, Austria.

Nicolas U Gerber (NU)

Department of Oncology, University Children's Hospital, Zurich, Switzerland.

Olli Lohi (O)

Tampere Center for Child Health Research and Tays Cancer Centre, Faculty of Medicine and Health Technology, Tampere University, Tampere, Finland.

Bernarda Kazanowska (B)

Department of Pediatric Hematology/Oncology and BMT, Wroclaw Medical University, Wroclaw, Poland.

Antonis Kattamis (A)

Division of Pediatric Hematology-Oncology, First Department of Pediatrics, National and Kapodistrian University of Athens, Athens, Greece.

Maria Filippidou (M)

Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
Division of Pediatric Neurooncology, German Cancer Research Center (DKFZ), Heidelberg, Germany.
Division of Pediatric Hematology-Oncology, First Department of Pediatrics, National and Kapodistrian University of Athens, Athens, Greece.

Bianca Goemans (B)

Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands.

C Michel Zwaan (CM)

Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands.
Erasmus MC-Sophia Children's Hospital, Rotterdam, the Netherlands.

Till Milde (T)

Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
Clinical Cooperation Unit Pediatric Oncology, German Cancer Research Center (DKFZ), Heidelberg, Germany.
Department of Pediatric Oncology, Hematology, Immunology and Pulmonology, Heidelberg University Hospital, Heidelberg, Germany.
German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.

Natalie Jäger (N)

Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Division of Pediatric Neurooncology, German Cancer Research Center (DKFZ), Heidelberg, Germany.

Stephan Wolf (S)

German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Genomics and Proteomics Core Facility, German Cancer Research Center (DKFZ), Heidelberg, Germany.

David Reuss (D)

German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Clinical Cooperation Unit Neuropathology, German Cancer Research Center (DKFZ), Heidelberg, Germany.
Department of Neuropathology, Institute of Pathology, Heidelberg University Hospital, Heidelberg, Germany.

Felix Sahm (F)

German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Clinical Cooperation Unit Neuropathology, German Cancer Research Center (DKFZ), Heidelberg, Germany.
Department of Neuropathology, Institute of Pathology, Heidelberg University Hospital, Heidelberg, Germany.

Andreas von Deimling (A)

German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Clinical Cooperation Unit Neuropathology, German Cancer Research Center (DKFZ), Heidelberg, Germany.
Department of Neuropathology, Institute of Pathology, Heidelberg University Hospital, Heidelberg, Germany.

Uta Dirksen (U)

German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
West German Cancer Center, Pediatrics III, University Hospital Essen, Essen, Germany.

Angelika Freitag (A)

NCT Trial Center, National Center for Tumor Diseases, Heidelberg, Germany, and German Cancer Research Center (DKFZ), Heidelberg, Germany.

Ruth Witt (R)

Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
Clinical Cooperation Unit Pediatric Oncology, German Cancer Research Center (DKFZ), Heidelberg, Germany.
German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.

Peter Lichter (P)

German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Division of Molecular Genetics, German Cancer Research Center (DKFZ), Heidelberg, Germany.

Annette Kopp-Schneider (A)

German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Department Biostatistics, German Cancer Research Center (DKFZ), Heidelberg, Germany.

David T W Jones (DTW)

Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Pediatric Glioma Research Group, German Cancer Research Center (DKFZ), Heidelberg, Germany.

Jan J Molenaar (JJ)

Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands.
Utrecht University, Department of Pharmaceutical Sciences, Utrecht, the Netherlands.

David Capper (D)

German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Charité-Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Department of Neuropathology, Berlin, Germany.

Stefan M Pfister (SM)

Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
Department of Pediatric Oncology, Hematology, Immunology and Pulmonology, Heidelberg University Hospital, Heidelberg, Germany.
German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.
Division of Pediatric Neurooncology, German Cancer Research Center (DKFZ), Heidelberg, Germany.

Olaf Witt (O)

Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
Clinical Cooperation Unit Pediatric Oncology, German Cancer Research Center (DKFZ), Heidelberg, Germany.
Department of Pediatric Oncology, Hematology, Immunology and Pulmonology, Heidelberg University Hospital, Heidelberg, Germany.
German Cancer Consortium (DKTK), Heidelberg, Germany.
National Center for Tumor Diseases (NCT) Network, Germany.

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