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

  • Cornelis M. van Tilburg
    1Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
  • Elke Pfaff
    1Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
  • Kristian W. Pajtler
    1Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
  • Karin P.S. Langenberg
    8Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands.
  • Petra Fiesel
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Barbara C. Jones
    1Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
  • Gnana Prakash Balasubramanian
    1Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
  • Sebastian Stark
    1Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
  • Pascal D. Johann
    1Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
  • Mirjam Blattner-Johnson
    1Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
  • Kathrin Schramm
    1Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
  • Nicola Dikow
    12Institute of Human Genetics, Heidelberg University Hospital, Heidelberg, Germany.
  • Steffen Hirsch
    1Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
  • Christian Sutter
    12Institute of Human Genetics, Heidelberg University Hospital, Heidelberg, Germany.
  • Kerstin Grund
    12Institute of Human Genetics, Heidelberg University Hospital, Heidelberg, Germany.
  • Arend von Stackelberg
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Andreas E. Kulozik
    1Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
  • Andrej Lissat
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Arndt Borkhardt
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Roland Meisel
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Dirk Reinhardt
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Jan-Henning Klusmann
    18Department of Pediatric Hematology, Oncology and Hemostaseology, Clinic for Pediatrics, University Hospital of Frankfurt, Goethe-University Frankfurt, Frankfurt/Main, Germany.
  • Gudrun Fleischhack
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Stephan Tippelt
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Dietrich von Schweinitz
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Irene Schmid
    20Department of Pediatric Oncology and Hematology, Dr. von Hauner Children's Hospital, Ludwig-Maximilians-University Munich, Munich, Germany.
  • Christof M. Kramm
    21Division of Pediatric Hematology and Oncology, University Medical Center Göttingen, Göttingen, Germany.
  • André O. von Bueren
    22Department of Pediatrics, Obstetrics and Gynecology, Division of Pediatric Hematology and Oncology, University Hospital Geneva, Geneva, Switzerland.
  • Gabriele Calaminus
    23Department of Pediatric Oncology and Hematology, University Hospital Bonn, Bonn, Germany.
  • Peter Vorwerk
    24Department of Pediatric Oncology and Hematology, University Hospital Magdeburg, Magdeburg, Germany.
  • Norbert Graf
    25Department of Pediatric Oncology and Hematology, University Hospital Saarland, Saarland, Germany.
  • Frank Westermann
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Matthias Fischer
    5National Center for Tumor Diseases (NCT) Network, Germany.
  • Angelika Eggert
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Birgit Burkhardt
    28Clinic of Pediatric Oncology and Hematology, University Hospital Münster, Muenster, Germany.
  • Wilhelm Wößmann
    29Department of Pediatric Oncology and Hematology, University Hospital Hamburg, Hamburg, Germany.
  • Michaela Nathrath
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Stefanie Hecker-Nolting
    5National Center for Tumor Diseases (NCT) Network, Germany.
  • Michael C. Frühwald
    5National Center for Tumor Diseases (NCT) Network, Germany.
  • Dominik T. Schneider
    33Clinic of Pediatrics, Municipal Hospital Dortmund, Dortmund, Germany.
  • Ines B. Brecht
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Petra Ketteler
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Simone Fulda
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Ewa Koscielniak
    5National Center for Tumor Diseases (NCT) Network, Germany.
  • Michael T. Meister
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Monika Scheer
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Simone Hettmer
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Matthias Schwab
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Roman Tremmel
    37Dr. Margarete Fischer-Bosch Institute of Clinical Pharmacology, Stuttgart, Germany.
  • Ingrid Øra
    39Department of Pediatric Oncology and Hematology, Skane University Hospital Lund, and HOPE-ITCC Unit, Pediatric Oncology, Karolinska University Hospital, Stockholm, Sweden.
  • Caroline Hutter
    40St. 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
    41Department of Oncology, University Children's Hospital, Zurich, Switzerland.
  • Olli Lohi
    42Tampere Center for Child Health Research and Tays Cancer Centre, Faculty of Medicine and Health Technology, Tampere University, Tampere, Finland.
  • Bernarda Kazanowska
    43Department of Pediatric Hematology/Oncology and BMT, Wroclaw Medical University, Wroclaw, Poland.
  • Antonis Kattamis
    44Division of Pediatric Hematology-Oncology, First Department of Pediatrics, National and Kapodistrian University of Athens, Athens, Greece.
  • Maria Filippidou
    1Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
  • Bianca Goemans
    8Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands.
  • C. Michel Zwaan
    8Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands.
  • Till Milde
    1Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
  • Natalie Jäger
    1Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
  • Stephan Wolf
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • David Reuss
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Felix Sahm
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Andreas von Deimling
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Uta Dirksen
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Angelika Freitag
    47NCT Trial Center, National Center for Tumor Diseases, Heidelberg, Germany, and German Cancer Research Center (DKFZ), Heidelberg, Germany.
  • Ruth Witt
    1Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
  • Peter Lichter
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Annette Kopp-Schneider
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • David T.W. Jones
    1Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
  • Jan J. Molenaar
    8Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands.
  • David Capper
    4German Cancer Consortium (DKTK), Heidelberg, Germany.
  • Stefan M. Pfister
    1Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
  • Olaf Witt
    1Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.

抄録

<jats:title>Abstract</jats:title> <jats:sec> <jats:title /> <jats:p>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; P = 0.011) in all other patients. The respective molecular targets were shown to be predictive for matched treatment response and not prognostic surrogates for improved outcome. Hereditary cancer predisposition syndromes were identified in 7.5% of patients, half of which were newly identified through the study. Integrated molecular analyses resulted in a change or refinement of diagnoses in 8.2% of cases.</jats:p> </jats:sec> <jats:sec> <jats:title>Significance:</jats:title> <jats:p>The pediatric precision oncology INFORM registry prospectively tested a target prioritization algorithm in a real-world, multinational setting and identified subgroups of patients benefiting from matched targeted treatment with improved progression-free survival, refinement of diagnosis, and identification of hereditary cancer predisposition syndromes.</jats:p> <jats:p>See related commentary by Eggermont et al., p. 2677 .</jats:p> <jats:p>This article is highlighted in the In This Issue feature, p. 2659</jats:p> </jats:sec>

収録刊行物

  • Cancer Discovery

    Cancer Discovery 11 (11), 2764-2779, 2021-08-09

    American Association for Cancer Research (AACR)

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