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270 results on '"van Tilburg, Cornelis M."'

1. LOGGIC/FIREFLY-2: a phase 3, randomized trial of tovorafenib vs. chemotherapy in pediatric and young adult patients with newly diagnosed low-grade glioma harboring an activating RAF alteration

Author

van Tilburg, Cornelis M., Kilburn, Lindsay B., Perreault, Sébastien, Schmidt, Rene, Azizi, Amedeo A., Cruz-Martínez, Ofelia, Zápotocký, Michal, Scheinemann, Katrin, Meeteren, Antoinette Y. N. Schouten-van, Sehested, Astrid, Opocher, Enrico, Driever, Pablo Hernáiz, Avula, Shivaram, Ziegler, David S., Capper, David, Koch, Arend, Sahm, Felix, Qiu, Jiaheng, Tsao, Li-Pen, Blackman, Samuel C., Manley, Peter, Milde, Till, Witt, Ruth, Jones, David T. W., Hargrave, Darren, and Witt, Olaf

Published
2024
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3. MAPK inhibitor sensitivity scores predict sensitivity driven by the immune infiltration in pediatric low-grade gliomas

Author

Sigaud, Romain, Albert, Thomas K., Hess, Caroline, Hielscher, Thomas, Winkler, Nadine, Kocher, Daniela, Walter, Carolin, Münter, Daniel, Selt, Florian, Usta, Diren, Ecker, Jonas, Brentrup, Angela, Hasselblatt, Martin, Thomas, Christian, Varghese, Julian, Capper, David, Thomale, Ulrich W., Hernáiz Driever, Pablo, Simon, Michèle, Horn, Svea, Herz, Nina Annika, Koch, Arend, Sahm, Felix, Hamelmann, Stefan, Faria-Andrade, Augusto, Jabado, Nada, Schuhmann, Martin U., Schouten-van Meeteren, Antoinette Y. N., Hoving, Eelco, Brummer, Tilman, van Tilburg, Cornelis M., Pfister, Stefan M., Witt, Olaf, Jones, David T. W., Kerl, Kornelius, and Milde, Till

Published
2023
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5. Larotrectinib in patients with TRK fusion-positive solid tumours: a pooled analysis of three phase 1/2 clinical trials

Author

Hong, David S, DuBois, Steven G, Kummar, Shivaani, Farago, Anna F, Albert, Catherine M, Rohrberg, Kristoffer S, van Tilburg, Cornelis M, Nagasubramanian, Ramamoorthy, Berlin, Jordan D, Federman, Noah, Mascarenhas, Leo, Geoerger, Birgit, Dowlati, Afshin, Pappo, Alberto S, Bielack, Stefan, Doz, François, McDermott, Ray, Patel, Jyoti D, Schilder, Russell J, Tahara, Makoto, Pfister, Stefan M, Witt, Olaf, Ladanyi, Marc, Rudzinski, Erin R, Nanda, Shivani, Childs, Barrett H, Laetsch, Theodore W, Hyman, David M, and Drilon, Alexander

Subjects
Biomedical and Clinical Sciences, Clinical Sciences, Oncology and Carcinogenesis, Clinical Trials and Supportive Activities, Clinical Research, Cancer, Evaluation of treatments and therapeutic interventions, 6.1 Pharmaceuticals, Good Health and Well Being, Adolescent, Adult, Aged, Aged, 80 and over, Child, Child, Preschool, Female, Humans, Infant, Infant, Newborn, Male, Middle Aged, Neoplasms, Proteins, Pyrazoles, Pyrimidines, Young Adult, Oncology & Carcinogenesis, Oncology and carcinogenesis
Abstract

BackgroundThe selective TRK inhibitor larotrectinib was approved for paediatric and adult patients with advanced TRK fusion-positive solid tumours based on a primary analysis set of 55 patients. The aim of our analysis was to explore the efficacy and long-term safety of larotrectinib in a larger population of patients with TRK fusion-positive solid tumours.MethodsPatients were enrolled and treated in a phase 1 adult, a phase 1/2 paediatric, or a phase 2 adolescent and adult trial. Some eligibility criteria differed between these studies. For this pooled analysis, eligible patients were aged 1 month or older, with a locally advanced or metastatic non-CNS primary, TRK fusion-positive solid tumour, who had received standard therapy previously if available. This analysis set includes the 55 patients on which approval of larotrectinib was based. Larotrectinib was administered orally (capsule or liquid formulation), on a continuous 28-day schedule, to adults mostly at a dose of 100 mg twice daily, and to paediatric patients mostly at a dose of 100 mg/m2 (maximum of 100 mg) twice daily. The primary endpoint was objective response as assessed by local investigators in an intention-to-treat analysis. Contributing trials are registered with ClinicalTrials.gov, NCT02122913 (active not recruiting), NCT02637687 (recruiting), and NCT02576431 (recruiting).FindingsBetween May 1, 2014, and Feb 19, 2019, 159 patients with TRK fusion-positive cancer were enrolled and treated with larotrectinib. Ages ranged from less than 1 month to 84 years. The proportion of patients with an objective response according to investigator assessment was 121 (79%, 95% CI 72-85) of 153 evaluable patients, with 24 (16%) having complete responses. In a safety population of 260 patients treated regardless of TRK fusion status, the most common grade 3 or 4 larotrectinib-related adverse events were increased alanine aminotransferase (eight [3%] of 260 patients), anaemia (six, 2%), and decreased neutrophil count (five [2%]). The most common larotrectinib-related serious adverse events were increased alanine aminotransferase (two [

Published
2020

6. Molecular diagnostics enables detection of actionable targets: the Pediatric Targeted Therapy 2.0 registry

Author

Ecker, Jonas, Selt, Florian, Sturm, Dominik, Sill, Martin, Korshunov, Andrey, Hirsch, Steffen, Capper, David, Dikow, Nicola, Sutter, Christian, Müller, Carina, Sigaud, Romain, Eggert, Angelika, Simon, Thorsten, Niehues, Tim, von Deimling, Andreas, Pajtler, Kristian W., van Tilburg, Cornelis M., Jones, David T.W., Sahm, Felix, Pfister, Stefan M., Witt, Olaf, and Milde, Till

Published
2023
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7. Resistance, rebound, and recurrence regrowth patterns in pediatric low-grade glioma treated by MAPK inhibition: A modified Delphi approach to build international consensus-based definitions—International Pediatric Low-Grade Glioma Coalition

Author

O’Hare, Patricia, primary, Cooney, Tabitha, additional, de Blank, Peter, additional, Gutmann, David H, additional, Kieran, Mark, additional, Milde, Till, additional, Fangusaro, Jason, additional, Fisher, Michael, additional, Avula, Shivaram, additional, Packer, Roger, additional, Fukuoka, Kohei, additional, Mankad, Kshitij, additional, Mueller, Sabine, additional, Waanders, Angela J, additional, Opocher, Enrico, additional, Bouffet, Eric, additional, Raabe, Eric, additional, Werle, Natacha Entz, additional, Azizi, Amedeo A, additional, Robison, Nathan J, additional, Hernáiz Driever, Pablo, additional, Russo, Mark, additional, Schouten, Netteke, additional, van Tilburg, Cornelis M, additional, Sehested, Astrid, additional, Grill, Jacques, additional, Bandopadhayay, Pratiti, additional, Kilday, John-Paul, additional, Witt, Olaf, additional, Ashley, David M, additional, Ertl-Wagner, Birgit Betina, additional, Tabori, Uri, additional, and Hargrave, Darren R, additional

Published
2024
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8. Drug sensitivity profiling of 3D tumor tissue cultures in the pediatric precision oncology program INFORM

Author

Peterziel, Heike, Jamaladdin, Nora, ElHarouni, Dina, Gerloff, Xenia F., Herter, Sonja, Fiesel, Petra, Berker, Yannick, Blattner-Johnson, Mirjam, Schramm, Kathrin, Jones, Barbara C., Reuss, David, Turunen, Laura, Friedenauer, Aileen, Holland-Letz, Tim, Sill, Martin, Weiser, Lena, Previti, Christopher, Balasubramanian, Gnanaprakash, Gerber, Nicolas U., Gojo, Johannes, Hutter, Caroline, Øra, Ingrid, Lohi, Olli, Kattamis, Antonis, de Wilde, Bram, Westermann, Frank, Tippelt, Stephan, Graf, Norbert, Nathrath, Michaela, Sparber-Sauer, Monika, Sehested, Astrid, Kramm, Christof M., Dirksen, Uta, Kallioniemi, Olli, Pfister, Stefan M., van Tilburg, Cornelis M., Jones, David T. W., Saarela, Jani, Pietiäinen, Vilja, Jäger, Natalie, Schlesner, Matthias, Kopp-Schneider, Annette, Oppermann, Sina, Milde, Till, Witt, Olaf, and Oehme, Ina

Published
2022
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10. Gene regulatory network landscape of Group 3/4 medulloblastoma

Author

Joshi, Piyush, primary, Stelzer, Tamina, additional, Okonechnikov, Konstantin, additional, Sarropoulos, Ioannis, additional, Sepp, Mari, additional, Pour-Jamnani, Mischan V., additional, Rademacher, Anne, additional, Yamada-Saito, Tetsuya, additional, Schneider, Céline, additional, Schmidt, Julia, additional, Schäfer, Philipp, additional, Leiss, Kevin, additional, Bortolomeazzi, Michele, additional, Mallm, Jan-Philipp, additional, da Silva, Patricia B.G., additional, Statz, Britta, additional, Wittmann, Andrea, additional, Schramm, Kathrin, additional, Blattner-Johnson, Mirjam, additional, Fiesel, Petra, additional, Jones, Barbara, additional, Milde, Till, additional, Pajtler, Kristian, additional, van Tilburg, Cornelis M., additional, Witt, Olaf, additional, Rippe, Karsten, additional, Korshunov, Andrey, additional, Jones, David T.W., additional, Hovestadt, Volker, additional, Northcott, Paul A., additional, Thongjuea, Supat, additional, Jäger, Natalie, additional, Kaessmann, Henrik, additional, Pfister, Stefan M., additional, and Kutscher, Lena M., additional

Published
2024
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11. Implementing data on targeted therapy from the INFORM registry platform for children with relapsed cancer in Sweden

Author

Wallin, Sofia, Ora, Ingrid, Prochazka, Gabriela, Sandgren, Johanna, Bjorklund, Caroline, Ljungman, Gustaf, Vogt, Hartmut, Ek, Torben, van Tilburg, Cornelis M., Nilsson, Anna, Wallin, Sofia, Ora, Ingrid, Prochazka, Gabriela, Sandgren, Johanna, Bjorklund, Caroline, Ljungman, Gustaf, Vogt, Hartmut, Ek, Torben, van Tilburg, Cornelis M., and Nilsson, Anna

Abstract

Background Advances in treatment of childhood malignancies have improved overall cure rates to 80%. Nevertheless, cancer is still the most common cause of childhood mortality in Sweden. The prognosis is particularly poor for relapse of high-risk malignancies. In the international INFORM registry, tumor tissue from patients with relapsed, refractory, or progressive pediatric cancer as well as from very-high risk primary tumors is biologically characterized using next-generation sequencing to identify possible therapeutic targets. We analyzed data from Swedish children included in the INFORM registry concerning patient characteristics, survival, sequencing results and whether targeted treatment was administered to the children based on the molecular findings.Methods A registry-based descriptive analysis of 184 patients included in the INFORM registry in Sweden during 2016-2021.Results The most common diagnoses were soft tissue and bone sarcomas followed by high grade gliomas [including diffuse intrinsic pontine glioma (DIPG)]. Complete molecular analysis was successful for 203/212 samples originating from 184 patients. In 88% of the samples, at least one actionable target was identified. Highly prioritized targets, according to a preset scale, were identified in 48 (24%) samples from 40 patients and 24 of these patients received matched targeted treatment but only six children within a clinical trial. No statistically significant benefit in terms of overall survival or progression free survival was observed between children treated with matched targeted treatment compared to all others.Conclusion This international collaborative study demonstrate feasibility regarding sequencing of pediatric high-risk tumors providing molecular data regarding potential actionable targets to clinicians. For a few individuals the INFORM analysis was of utmost importance and should be regarded as a new standard of care with the potential to guide targeted therapy., Funding Agencies|Swedish Childhood Cancer Fund (BCF); Entrepreneurs for good; ARMEC Lindeberg Foundation; German Cancer Research Center (DKFZ); German Cancer Consortium (DKTK); German Federal Ministry of Education and Research (BMBF); German Federal Ministry of Health (BMG); Ministry of Science, Research and the Arts of the State of Baden-Wurttemberg; German Cancer Aid; German Childhood Cancer Foundation; Aid organization BILD hilft e.V. (Ein Herz fur Kinder); RTL television

Published
2024
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12. Diagnostik und Therapie von Tumoren mit NTRK-Genfusionen

Author

Stenzinger, Albrecht, van Tilburg, Cornelis M., Tabatabai, Ghazaleh, Länger, Florian, Graf, Norbert, Griesinger, Frank, Heukamp, Lukas C., Hummel, Michael, Klingebiel, Thomas, Hettmer, Simone, Vokuhl, Christian, Merkelbach-Bruse, Sabine, Overkamp, Friedrich, Reichardt, Peter, Scheer, Monika, Weichert, Wilko, Westphalen, C. Benedikt, Bokemeyer, Carsten, Ivanyi, Philipp, Loges, Sonja, Schirmacher, Peter, Wörmann, Bernhard, Bielack, Stefan, and Seufferlein, Thomas T. W.

Published
2021
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14. Radiation-induced gliomas represent H3-/IDH-wild type pediatric gliomas with recurrent PDGFRA amplification and loss of CDKN2A/B

Author

Deng, Maximilian Y., Sturm, Dominik, Pfaff, Elke, Sill, Martin, Stichel, Damian, Balasubramanian, Gnana Prakash, Tippelt, Stephan, Kramm, Christof, Donson, Andrew M., Green, Adam L., Jones, Chris, Schittenhelm, Jens, Ebinger, Martin, Schuhmann, Martin U., Jones, Barbara C., van Tilburg, Cornelis M., Wittmann, Andrea, Golanov, Andrey, Ryzhova, Marina, Ecker, Jonas, Milde, Till, Witt, Olaf, Sahm, Felix, Reuss, David, Sumerauer, David, Zamecnik, Josef, Korshunov, Andrey, von Deimling, Andreas, Pfister, Stefan M., and Jones, David T. W.

Published
2021
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16. Implementation of paediatric precision oncology into clinical practice: The Individualized Therapies for Children with cancer program ‘iTHER’

Author

Langenberg, Karin P.S., Meister, Michael T., Bakhuizen, Jette J., Boer, Judith M., van Eijkelenburg, Natasha K.A., Hulleman, Esther, Ilan, Uri, Looze, Eleonora J., Dierselhuis, Miranda P., van der Lugt, Jasper, Breunis, Willemijn, Schild, Linda G., Ober, Kimberley, van Hooff, Sander R., Scheijde-Vermeulen, Marijn A., Hiemcke-Jiwa, Laura S., Flucke, Uta E., Kranendonk, Mariette E.G., Wesseling, Pieter, Sonneveld, Edwin, Punt, Simone, Boltjes, Arjan, van Dijk, Freerk, Verwiel, Eugene T.P., Volckmann, Richard, Hehir-Kwa, Jayne Y., Kester, Lennart A., Koudijs, Marco M.J., Waanders, Esme, Holstege, Frank C.P., Vormoor, H. Josef, Hoving, Eelco W., van Noesel, Max M., Pieters, Rob, Kool, Marcel, Stumpf, Miriam, Blattner-Johnson, Mirjam, Balasubramanian, Gnana P., Van Tilburg, Cornelis M., Jones, Barbara C., Jones, David T.W., Witt, Olaf, Pfister, Stefan M., Jongmans, Marjolijn C.J., Kuiper, Roland P., de Krijger, Ronald R., Wijnen, Marc H.W., den Boer, Monique L., Zwaan, C. Michel, Kemmeren, Patrick, Koster, Jan, Tops, Bastiaan B.J., Goemans, Bianca F., Molenaar, Jan J., Dep Scheikunde, Afd Pharmaceutics, Pharmaceutics, Dep Scheikunde, Afd Pharmaceutics, Pharmaceutics, Center of Experimental and Molecular Medicine, Oncogenomics, and CCA - Cancer Treatment and Quality of Life

Subjects
Cancer Research, Adolescent, Molecular biology, Precision medicine, High-Throughput Nucleotide Sequencing, Medical Oncology, Hereditary, Oncology, Neoplasms, Mutation, Exome Sequencing, Next-generation sequencing, Humans, Molecular targeted therapy, Prospective Studies, Child, Cancer
Abstract

iTHER is a Dutch prospective national precision oncology program aiming to define tumour molecular profiles in children and adolescents with primary very high-risk, relapsed, or refractory paediatric tumours. Between April 2017 and April 2021, 302 samples from 253 patients were included. Comprehensive molecular profiling including low-coverage whole genome sequencing (lcWGS), whole exome sequencing (WES), RNA sequencing (RNA-seq), Affymetrix, and/or 850k methylation profiling was successfully performed for 226 samples with at least 20% tumour content. Germline pathogenic variants were identified in 16% of patients (35/219), of which 22 variants were judged causative for a cancer predisposition syndrome. At least one somatic alteration was detected in 204 (90.3%), and 185 (81.9%) were considered druggable, with clinical priority very high (6.1%), high (21.3%), moderate (26.0%), intermediate (36.1%), and borderline (10.5%) priority. iTHER led to revision or refinement of diagnosis in 8 patients (3.5%). Temporal heterogeneity was observed in paired samples of 15 patients, indicating the value of sequential analyses. Of 137 patients with follow-up beyond twelve months, 21 molecularly matched treatments were applied in 19 patients (13.9%), with clinical benefit in few. Most relevant barriers to not applying targeted therapies included poor performance status, as well as limited access to drugs within clinical trial. iTHER demonstrates the feasibility of comprehensive molecular profiling across all ages, tumour types and stages in paediatric cancers, informing of diagnostic, prognostic, and targetable alterations as well as reportable germline variants. Therefore, WES and RNA-seq is nowadays standard clinical care at the Princess Máxima Center for all children with cancer, including patients at primary diagnosis. Improved access to innovative treatments within biology-driven combination trials is required to ultimately improve survival.

Published
2022

18. Outcome of Children and Adolescents With Relapsed/Refractory/Progressive Malignancies Treated With Molecularly Informed Targeted Drugs in the Pediatric Precision Oncology Registry INFORM

Author

Heipertz, Anna-Elisa, primary, Pajtler, Kristian W., additional, Pfaff, Elke, additional, Schramm, Kathrin, additional, Blattner-Johnson, Mirjam, additional, Milde, Till, additional, Jones, Barbara C., additional, Zuliani, Cecilia, additional, Hutter, Caroline, additional, Lohi, Olli, additional, Kattamis, Antonis, additional, Dachowska-Kalwak, Iwona, additional, Nilsson, Anna, additional, Gerber, Nicolas U., additional, Langenberg, Karin P.S., additional, Goemans, Bianca F., additional, Zwaan, C. Michel, additional, Molenaar, Jan J., additional, Jäger, Natalie, additional, Dirksen, Uta, additional, Witt, Ruth, additional, Pfister, Stefan M., additional, Jones, David T.W., additional, Kopp-Schneider, Annette, additional, Witt, Olaf, additional, and van Tilburg, Cornelis M., additional

Published
2023
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19. Clinical outcome of pediatric medulloblastoma patients with Li–Fraumeni syndrome

Author

Kolodziejczak, Anna S, primary, Guerrini-Rousseau, Lea, additional, Planchon, Julien Masliah, additional, Ecker, Jonas, additional, Selt, Florian, additional, Mynarek, Martin, additional, Obrecht, Denise, additional, Sill, Martin, additional, Autry, Robert J, additional, Stutheit-Zhao, Eric, additional, Hirsch, Steffen, additional, Amouyal, Elsa, additional, Dufour, Christelle, additional, Ayrault, Olivier, additional, Torrejon, Jacob, additional, Waszak, Sebastian M, additional, Ramaswamy, Vijay, additional, Pentikainen, Virve, additional, Demir, Haci Ahmet, additional, Clifford, Steven C, additional, Schwalbe, Ed C, additional, Massimi, Luca, additional, Snuderl, Matija, additional, Galbraith, Kristyn, additional, Karajannis, Matthias A, additional, Hill, Katherine, additional, Li, Bryan K, additional, Walsh, Mike, additional, White, Christine L, additional, Redmond, Shelagh, additional, Loizos, Loizou, additional, Jakob, Marcus, additional, Kordes, Uwe R, additional, Schmid, Irene, additional, Hauer, Julia, additional, Blattmann, Claudia, additional, Filippidou, Maria, additional, Piccolo, Gianluca, additional, Scheurlen, Wolfram, additional, Farrag, Ahmed, additional, Grund, Kerstin, additional, Sutter, Christian, additional, Pietsch, Torsten, additional, Frank, Stephan, additional, Schewe, Denis M, additional, Malkin, David, additional, Ben-Arush, Myriam, additional, Sehested, Astrid, additional, Wong, Tai-Tong, additional, Wu, Kuo-Sheng, additional, Liu, Yen-Lin, additional, Carceller, Fernando, additional, Mueller, Sabine, additional, Stoller, Schuyler, additional, Taylor, Michael D, additional, Tabori, Uri, additional, Bouffet, Eric, additional, Kool, Marcel, additional, Sahm, Felix, additional, von Deimling, Andreas, additional, Korshunov, Andrey, additional, von Hoff, Katja, additional, Kratz, Christian P, additional, Sturm, Dominik, additional, Jones, David T W, additional, Rutkowski, Stefan, additional, van Tilburg, Cornelis M, additional, Witt, Olaf, additional, Bougeard, Gaëlle, additional, Pajtler, Kristian W, additional, Pfister, Stefan M, additional, Bourdeaut, Franck, additional, and Milde, Till, additional

Published
2023
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20. Class I HDAC inhibitor entinostat synergizes with PLK1 inhibitors in MYC-amplified medulloblastoma cells

Author

Valinciute, Gintvile, primary, Ecker, Jonas, additional, Selt, Florian, additional, Hielscher, Thomas, additional, Sigaud, Romain, additional, Ridinger, Johannes, additional, Thatikonda, Venu, additional, Gatzweiler, Charlotte, additional, Robinson, Sarah, additional, Talbot, Julie, additional, Bernardi, Flavia, additional, Picard, Daniel, additional, Blattner-Johnson, Mirjam, additional, Schmid, Simone, additional, Jones, David T., additional, van Tilburg, Cornelis M., additional, Capper, David, additional, Kool, Marcel, additional, Remke, Marc, additional, Oehme, Ina, additional, Pfister, Stefan M., additional, Roussel, Martine F., additional, Ayrault, Olivier, additional, Witt, Olaf, additional, and Milde, Till, additional

Published
2023
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21. Corrigendum to ‘Molecular diagnostics enables detection of actionable targets: the Pediatric Targeted Therapy 2.0 registry’ [Eur J Cancer 180 (2023) 71–84]

Author

Ecker, Jonas, primary, Selt, Florian, additional, Sturm, Dominik, additional, Sill, Martin, additional, Korshunov, Andrey, additional, Hirsch, Steffen, additional, Capper, David, additional, Dikow, Nicola, additional, Sutter, Christian, additional, Müller, Carina, additional, Sigaud, Romain, additional, Eggert, Angelika, additional, Simon, Thorsten, additional, Niehues, Tim, additional, von Deimling, Andreas, additional, Pajtler, Kristian W., additional, van Tilburg, Cornelis M., additional, Jones, David T.W., additional, Sahm, Felix, additional, Pfister, Stefan M., additional, Witt, Olaf, additional, and Milde, Till, additional

Published
2023
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22. INFORM2 NivEnt: The first trial of the INFORM2 biomarker driven phase I/II trial series: the combination of nivolumab and entinostat in children and adolescents with refractory high-risk malignancies

Author

van Tilburg, Cornelis M., Witt, Ruth, Heiss, Melanie, Pajtler, Kristian W., Plass, Christoph, Poschke, Isabel, Platten, Michael, Harting, Inga, Sedlaczek, Oliver, Freitag, Angelika, Meyrath, David, Taylor, Lenka, Balasubramanian, Gnana Prakash, Jäger, Natalie, Pfaff, Elke, Jones, Barbara C., Milde, Till, Pfister, Stefan M., Jones, David T. W., Kopp-Schneider, Annette, and Witt, Olaf

Published
2020
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23. Phase I/II intra-patient dose escalation study of vorinostat in children with relapsed solid tumor, lymphoma, or leukemia

Author

van Tilburg, Cornelis M., Milde, Till, Witt, Ruth, Ecker, Jonas, Hielscher, Thomas, Seitz, Angelika, Schenk, Jens-Peter, Buhl, Juliane L., Riehl, Dennis, Frühwald, Michael C., Pekrun, Arnulf, Rossig, Claudia, Wieland, Regina, Flotho, Christian, Kordes, Uwe, Gruhn, Bernd, Simon, Thorsten, Linderkamp, Christin, Sahm, Felix, Taylor, Lenka, Freitag, Angelika, Burhenne, Jürgen, Foerster, Kathrin I., Meid, Andreas D., Pfister, Stefan M., Karapanagiotou-Schenkel, Irini, and Witt, Olaf

Published
2019
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24. LOGGIC Core BioClinical Data Bank: Added clinical value of RNA-Seq in an international molecular diagnostic registry for pediatric low-grade glioma patients

Author

Hardin, Emily C, primary, Schmid, Simone, additional, Sommerkamp, Alexander, additional, Bodden, Carina, additional, Heipertz, Anna-Elisa, additional, Sievers, Philipp, additional, Wittmann, Andrea, additional, Milde, Till, additional, Pfister, Stefan M, additional, von Deimling, Andreas, additional, Horn, Svea, additional, Herz, Nina A, additional, Simon, Michèle, additional, Perera, Ashwyn A, additional, Azizi, Amedeo, additional, Cruz, Ofelia, additional, Curry, Sarah, additional, Van Damme, An, additional, Garami, Miklos, additional, Hargrave, Darren, additional, Kattamis, Antonis, additional, Kotnik, Barbara Faganel, additional, Lähteenmäki, Päivi, additional, Scheinemann, Katrin, additional, Schouten-van Meeteren, Antoinette Y N, additional, Sehested, Astrid, additional, Viscardi, Elisabetta, additional, Wormdal, Ole Mikal, additional, Zapotocky, Michal, additional, Ziegler, David S, additional, Koch, Arend, additional, Driever, Pablo Hernáiz, additional, Witt, Olaf, additional, Capper, David, additional, Sahm, Felix, additional, Jones, David T W, additional, and van Tilburg, Cornelis M, additional

Published
2023
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25. Abstract CT228: Concordance between tissue and circulating tumor DNA (ctDNA) testing for neurotrophic tyrosine receptor kinase (NTRK) gene fusions in larotrectinib (laro) clinical trials

Author

Rudzinski, Erin R., primary, Ladanyi, Marc, additional, Ritterhouse, Lauren L., additional, Sahm, Felix, additional, Norenberg, Ricarda, additional, Zheng, Hong, additional, Chen, Chi, additional, Bernard-Gauthier, Vadim, additional, Brega, Nicoletta, additional, van Tilburg, Cornelis M., additional, Lin, Jessica J., additional, Brose, Marcia S., additional, Lassen, Ulrik N., additional, McDermott, Ray, additional, Laetsch, Theodore W., additional, Hong, David S., additional, Drilon, Alexander, additional, and Roy-Chowdhuri, Sinchita, additional

Published
2023
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26. Supplementary Table from The Pediatric Precision Oncology INFORM Registry: Clinical Outcome and Benefit for Patients with Very High-Evidence Targets

Author

van Tilburg, Cornelis M., primary, Pfaff, Elke, primary, Pajtler, Kristian W., primary, Langenberg, Karin P.S., primary, Fiesel, Petra, primary, Jones, Barbara C., primary, Balasubramanian, Gnana Prakash, primary, Stark, Sebastian, primary, Johann, Pascal D., primary, Blattner-Johnson, Mirjam, primary, Schramm, Kathrin, primary, Dikow, Nicola, primary, Hirsch, Steffen, primary, Sutter, Christian, primary, Grund, Kerstin, primary, von Stackelberg, Arend, primary, Kulozik, Andreas E., primary, Lissat, Andrej, primary, Borkhardt, Arndt, primary, Meisel, Roland, primary, Reinhardt, Dirk, primary, Klusmann, Jan-Henning, primary, Fleischhack, Gudrun, primary, Tippelt, Stephan, primary, von Schweinitz, Dietrich, primary, Schmid, Irene, primary, Kramm, Christof M., primary, von Bueren, André O., primary, Calaminus, Gabriele, primary, Vorwerk, Peter, primary, Graf, Norbert, primary, Westermann, Frank, primary, Fischer, Matthias, primary, Eggert, Angelika, primary, Burkhardt, Birgit, primary, Wößmann, Wilhelm, primary, Nathrath, Michaela, primary, Hecker-Nolting, Stefanie, primary, Frühwald, Michael C., primary, Schneider, Dominik T., primary, Brecht, Ines B., primary, Ketteler, Petra, primary, Fulda, Simone, primary, Koscielniak, Ewa, primary, Meister, Michael T., primary, Scheer, Monika, primary, Hettmer, Simone, primary, Schwab, Matthias, primary, Tremmel, Roman, primary, Øra, Ingrid, primary, Hutter, Caroline, primary, Gerber, Nicolas U., primary, Lohi, Olli, primary, Kazanowska, Bernarda, primary, Kattamis, Antonis, primary, Filippidou, Maria, primary, Goemans, Bianca, primary, Zwaan, C. Michel, primary, Milde, Till, primary, Jäger, Natalie, primary, Wolf, Stephan, primary, Reuss, David, primary, Sahm, Felix, primary, von Deimling, Andreas, primary, Dirksen, Uta, primary, Freitag, Angelika, primary, Witt, Ruth, primary, Lichter, Peter, primary, Kopp-Schneider, Annette, primary, Jones, David T.W., primary, Molenaar, Jan J., primary, Capper, David, primary, Pfister, Stefan M., primary, and Witt, Olaf, primary

Published
2023
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27. Suppl. Table S2 from A Cell-Based MAPK Reporter Assay Reveals Synergistic MAPK Pathway Activity Suppression by MAPK Inhibitor Combination in BRAF-Driven Pediatric Low-Grade Glioma Cells

Author

Usta, Diren, primary, Sigaud, Romain, primary, Buhl, Juliane L., primary, Selt, Florian, primary, Marquardt, Viktoria, primary, Pauck, David, primary, Jansen, Jennifer, primary, Pusch, Stefan, primary, Ecker, Jonas, primary, Hielscher, Thomas, primary, Vollmer, Johanna, primary, Sommerkamp, Alexander C., primary, Rubner, Tobias, primary, Hargrave, Darren, primary, van Tilburg, Cornelis M., primary, Pfister, Stefan M., primary, Jones, David T.W., primary, Remke, Marc, primary, Brummer, Tilman, primary, Witt, Olaf, primary, and Milde, Till, primary

Published
2023
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28. Data from A Cell-Based MAPK Reporter Assay Reveals Synergistic MAPK Pathway Activity Suppression by MAPK Inhibitor Combination in BRAF-Driven Pediatric Low-Grade Glioma Cells

Author

Usta, Diren, primary, Sigaud, Romain, primary, Buhl, Juliane L., primary, Selt, Florian, primary, Marquardt, Viktoria, primary, Pauck, David, primary, Jansen, Jennifer, primary, Pusch, Stefan, primary, Ecker, Jonas, primary, Hielscher, Thomas, primary, Vollmer, Johanna, primary, Sommerkamp, Alexander C., primary, Rubner, Tobias, primary, Hargrave, Darren, primary, van Tilburg, Cornelis M., primary, Pfister, Stefan M., primary, Jones, David T.W., primary, Remke, Marc, primary, Brummer, Tilman, primary, Witt, Olaf, primary, and Milde, Till, primary

Published
2023
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29. Supplementary Figures S1-S5 from A Cell-Based MAPK Reporter Assay Reveals Synergistic MAPK Pathway Activity Suppression by MAPK Inhibitor Combination in BRAF-Driven Pediatric Low-Grade Glioma Cells

Author

Usta, Diren, primary, Sigaud, Romain, primary, Buhl, Juliane L., primary, Selt, Florian, primary, Marquardt, Viktoria, primary, Pauck, David, primary, Jansen, Jennifer, primary, Pusch, Stefan, primary, Ecker, Jonas, primary, Hielscher, Thomas, primary, Vollmer, Johanna, primary, Sommerkamp, Alexander C., primary, Rubner, Tobias, primary, Hargrave, Darren, primary, van Tilburg, Cornelis M., primary, Pfister, Stefan M., primary, Jones, David T.W., primary, Remke, Marc, primary, Brummer, Tilman, primary, Witt, Olaf, primary, and Milde, Till, primary

Published
2023
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30. Supplemental Table Legend from A Cell-Based MAPK Reporter Assay Reveals Synergistic MAPK Pathway Activity Suppression by MAPK Inhibitor Combination in BRAF-Driven Pediatric Low-Grade Glioma Cells

Author

Usta, Diren, primary, Sigaud, Romain, primary, Buhl, Juliane L., primary, Selt, Florian, primary, Marquardt, Viktoria, primary, Pauck, David, primary, Jansen, Jennifer, primary, Pusch, Stefan, primary, Ecker, Jonas, primary, Hielscher, Thomas, primary, Vollmer, Johanna, primary, Sommerkamp, Alexander C., primary, Rubner, Tobias, primary, Hargrave, Darren, primary, van Tilburg, Cornelis M., primary, Pfister, Stefan M., primary, Jones, David T.W., primary, Remke, Marc, primary, Brummer, Tilman, primary, Witt, Olaf, primary, and Milde, Till, primary

Published
2023
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31. Data from The Pediatric Precision Oncology INFORM Registry: Clinical Outcome and Benefit for Patients with Very High-Evidence Targets

Author

van Tilburg, Cornelis M., primary, Pfaff, Elke, primary, Pajtler, Kristian W., primary, Langenberg, Karin P.S., primary, Fiesel, Petra, primary, Jones, Barbara C., primary, Balasubramanian, Gnana Prakash, primary, Stark, Sebastian, primary, Johann, Pascal D., primary, Blattner-Johnson, Mirjam, primary, Schramm, Kathrin, primary, Dikow, Nicola, primary, Hirsch, Steffen, primary, Sutter, Christian, primary, Grund, Kerstin, primary, von Stackelberg, Arend, primary, Kulozik, Andreas E., primary, Lissat, Andrej, primary, Borkhardt, Arndt, primary, Meisel, Roland, primary, Reinhardt, Dirk, primary, Klusmann, Jan-Henning, primary, Fleischhack, Gudrun, primary, Tippelt, Stephan, primary, von Schweinitz, Dietrich, primary, Schmid, Irene, primary, Kramm, Christof M., primary, von Bueren, André O., primary, Calaminus, Gabriele, primary, Vorwerk, Peter, primary, Graf, Norbert, primary, Westermann, Frank, primary, Fischer, Matthias, primary, Eggert, Angelika, primary, Burkhardt, Birgit, primary, Wößmann, Wilhelm, primary, Nathrath, Michaela, primary, Hecker-Nolting, Stefanie, primary, Frühwald, Michael C., primary, Schneider, Dominik T., primary, Brecht, Ines B., primary, Ketteler, Petra, primary, Fulda, Simone, primary, Koscielniak, Ewa, primary, Meister, Michael T., primary, Scheer, Monika, primary, Hettmer, Simone, primary, Schwab, Matthias, primary, Tremmel, Roman, primary, Øra, Ingrid, primary, Hutter, Caroline, primary, Gerber, Nicolas U., primary, Lohi, Olli, primary, Kazanowska, Bernarda, primary, Kattamis, Antonis, primary, Filippidou, Maria, primary, Goemans, Bianca, primary, Zwaan, C. Michel, primary, Milde, Till, primary, Jäger, Natalie, primary, Wolf, Stephan, primary, Reuss, David, primary, Sahm, Felix, primary, von Deimling, Andreas, primary, Dirksen, Uta, primary, Freitag, Angelika, primary, Witt, Ruth, primary, Lichter, Peter, primary, Kopp-Schneider, Annette, primary, Jones, David T.W., primary, Molenaar, Jan J., primary, Capper, David, primary, Pfister, Stefan M., primary, and Witt, Olaf, primary

Published
2023
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32. Supplementary Figure from The Pediatric Precision Oncology INFORM Registry: Clinical Outcome and Benefit for Patients with Very High-Evidence Targets

Author

van Tilburg, Cornelis M., primary, Pfaff, Elke, primary, Pajtler, Kristian W., primary, Langenberg, Karin P.S., primary, Fiesel, Petra, primary, Jones, Barbara C., primary, Balasubramanian, Gnana Prakash, primary, Stark, Sebastian, primary, Johann, Pascal D., primary, Blattner-Johnson, Mirjam, primary, Schramm, Kathrin, primary, Dikow, Nicola, primary, Hirsch, Steffen, primary, Sutter, Christian, primary, Grund, Kerstin, primary, von Stackelberg, Arend, primary, Kulozik, Andreas E., primary, Lissat, Andrej, primary, Borkhardt, Arndt, primary, Meisel, Roland, primary, Reinhardt, Dirk, primary, Klusmann, Jan-Henning, primary, Fleischhack, Gudrun, primary, Tippelt, Stephan, primary, von Schweinitz, Dietrich, primary, Schmid, Irene, primary, Kramm, Christof M., primary, von Bueren, André O., primary, Calaminus, Gabriele, primary, Vorwerk, Peter, primary, Graf, Norbert, primary, Westermann, Frank, primary, Fischer, Matthias, primary, Eggert, Angelika, primary, Burkhardt, Birgit, primary, Wößmann, Wilhelm, primary, Nathrath, Michaela, primary, Hecker-Nolting, Stefanie, primary, Frühwald, Michael C., primary, Schneider, Dominik T., primary, Brecht, Ines B., primary, Ketteler, Petra, primary, Fulda, Simone, primary, Koscielniak, Ewa, primary, Meister, Michael T., primary, Scheer, Monika, primary, Hettmer, Simone, primary, Schwab, Matthias, primary, Tremmel, Roman, primary, Øra, Ingrid, primary, Hutter, Caroline, primary, Gerber, Nicolas U., primary, Lohi, Olli, primary, Kazanowska, Bernarda, primary, Kattamis, Antonis, primary, Filippidou, Maria, primary, Goemans, Bianca, primary, Zwaan, C. Michel, primary, Milde, Till, primary, Jäger, Natalie, primary, Wolf, Stephan, primary, Reuss, David, primary, Sahm, Felix, primary, von Deimling, Andreas, primary, Dirksen, Uta, primary, Freitag, Angelika, primary, Witt, Ruth, primary, Lichter, Peter, primary, Kopp-Schneider, Annette, primary, Jones, David T.W., primary, Molenaar, Jan J., primary, Capper, David, primary, Pfister, Stefan M., primary, and Witt, Olaf, primary

Published
2023
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33. Table S4 from The Senescence-associated Secretory Phenotype Mediates Oncogene-induced Senescence in Pediatric Pilocytic Astrocytoma

Author

Buhl, Juliane L., primary, Selt, Florian, primary, Hielscher, Thomas, primary, Guiho, Romain, primary, Ecker, Jonas, primary, Sahm, Felix, primary, Ridinger, Johannes, primary, Riehl, Dennis, primary, Usta, Diren, primary, Ismer, Britta, primary, Sommerkamp, Alexander C., primary, Martinez-Barbera, J.P., primary, Wefers, Annika K., primary, Remke, Marc, primary, Picard, Daniel, primary, Pusch, Stefan, primary, Gronych, Jan, primary, Oehme, Ina, primary, van Tilburg, Cornelis M., primary, Kool, Marcel, primary, Kuhn, Daniela, primary, Capper, David, primary, von Deimling, Andreas, primary, Schuhmann, Martin U., primary, Herold-Mende, Christel, primary, Korshunov, Andrey, primary, Brummer, Tilman, primary, Pfister, Stefan M., primary, Jones, David T.W., primary, Witt, Olaf, primary, and Milde, Till, primary

Published
2023
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34. Supplemental Table Legend from The Senescence-associated Secretory Phenotype Mediates Oncogene-induced Senescence in Pediatric Pilocytic Astrocytoma

Author

Buhl, Juliane L., primary, Selt, Florian, primary, Hielscher, Thomas, primary, Guiho, Romain, primary, Ecker, Jonas, primary, Sahm, Felix, primary, Ridinger, Johannes, primary, Riehl, Dennis, primary, Usta, Diren, primary, Ismer, Britta, primary, Sommerkamp, Alexander C., primary, Martinez-Barbera, J.P., primary, Wefers, Annika K., primary, Remke, Marc, primary, Picard, Daniel, primary, Pusch, Stefan, primary, Gronych, Jan, primary, Oehme, Ina, primary, van Tilburg, Cornelis M., primary, Kool, Marcel, primary, Kuhn, Daniela, primary, Capper, David, primary, von Deimling, Andreas, primary, Schuhmann, Martin U., primary, Herold-Mende, Christel, primary, Korshunov, Andrey, primary, Brummer, Tilman, primary, Pfister, Stefan M., primary, Jones, David T.W., primary, Witt, Olaf, primary, and Milde, Till, primary

Published
2023
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35. Figure S1, Figure S2, Figure S3, Figure S4 from The Senescence-associated Secretory Phenotype Mediates Oncogene-induced Senescence in Pediatric Pilocytic Astrocytoma

Author

Buhl, Juliane L., primary, Selt, Florian, primary, Hielscher, Thomas, primary, Guiho, Romain, primary, Ecker, Jonas, primary, Sahm, Felix, primary, Ridinger, Johannes, primary, Riehl, Dennis, primary, Usta, Diren, primary, Ismer, Britta, primary, Sommerkamp, Alexander C., primary, Martinez-Barbera, J.P., primary, Wefers, Annika K., primary, Remke, Marc, primary, Picard, Daniel, primary, Pusch, Stefan, primary, Gronych, Jan, primary, Oehme, Ina, primary, van Tilburg, Cornelis M., primary, Kool, Marcel, primary, Kuhn, Daniela, primary, Capper, David, primary, von Deimling, Andreas, primary, Schuhmann, Martin U., primary, Herold-Mende, Christel, primary, Korshunov, Andrey, primary, Brummer, Tilman, primary, Pfister, Stefan M., primary, Jones, David T.W., primary, Witt, Olaf, primary, and Milde, Till, primary

Published
2023
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36. Outcome of Children and Adolescents With Relapsed/Refractory/Progressive Malignancies Treated With Molecularly Informed Targeted Drugs in the Pediatric Precision Oncology Registry INFORM

Author

Afd Pharmaceutics, Pharmaceutics, Heipertz, Anna-Elisa, Pajtler, Kristian W, Pfaff, Elke, Schramm, Kathrin, Blattner-Johnson, Mirjam, Milde, Till, Jones, Barbara C, Zuliani, Cecilia, Hutter, Caroline, Lohi, Olli, Kattamis, Antonis, Dachowska-Kalwak, Iwona, Nilsson, Anna, Gerber, Nicolas U, Langenberg, Karin P S, Goemans, Bianca, Zwaan, C Michel, Molenaar, Jan J, Jäger, Natalie, Dirksen, Uta, Witt, Ruth, Pfister, Stefan M, Jones, David T W, Kopp-Schneider, Annette, Witt, Olaf, van Tilburg, Cornelis M, Afd Pharmaceutics, Pharmaceutics, Heipertz, Anna-Elisa, Pajtler, Kristian W, Pfaff, Elke, Schramm, Kathrin, Blattner-Johnson, Mirjam, Milde, Till, Jones, Barbara C, Zuliani, Cecilia, Hutter, Caroline, Lohi, Olli, Kattamis, Antonis, Dachowska-Kalwak, Iwona, Nilsson, Anna, Gerber, Nicolas U, Langenberg, Karin P S, Goemans, Bianca, Zwaan, C Michel, Molenaar, Jan J, Jäger, Natalie, Dirksen, Uta, Witt, Ruth, Pfister, Stefan M, Jones, David T W, Kopp-Schneider, Annette, Witt, Olaf, and van Tilburg, Cornelis M

Published
2023

37. Original Research Molecular diagnostics enables detection of actionable targets: the Pediatric Targeted Therapy 2.0 registry

Author

Ecker, Jonas, Selt, Florian, Sturm, Dominik, Sill, Martin, Korshunov, Andrey, Hirsch, Steffen, Capper, David, Dikow, Nicola, Sutter, Christian, Mueller, Carina, Sigaud, Romain, Eggert, Angelika, Simon, Thorsten, Niehues, Tim, von Deimling, Andreas, Pajtler, Kristian W., van Tilburg, Cornelis M., Jones, David T. W., Sahm, Felix, Pfister, Stefan M., Witt, Olaf, Milde, Till, Ecker, Jonas, Selt, Florian, Sturm, Dominik, Sill, Martin, Korshunov, Andrey, Hirsch, Steffen, Capper, David, Dikow, Nicola, Sutter, Christian, Mueller, Carina, Sigaud, Romain, Eggert, Angelika, Simon, Thorsten, Niehues, Tim, von Deimling, Andreas, Pajtler, Kristian W., van Tilburg, Cornelis M., Jones, David T. W., Sahm, Felix, Pfister, Stefan M., Witt, Olaf, and Milde, Till

Abstract

Background: Precision oncology requires diagnostic accuracy and robust detection of actionable alterations. The Pediatric Targeted Therapy (PTT) 2.0 program aims at improving diagnostic accuracy by addition of molecular analyses to the existing histological diagnosis and detection of actionable alterations for relapsed paediatric oncology patients, in cases with limited availability of tumour material.Methods: Paediatric patients diagnosed with relapse or progression of a central nervous system tumour (n = 178), a sarcoma (n = 41) or another solid tumour (n = 44) were included. DNA methylation array, targeted gene panel sequencing on tumour and blood (130 genes), RNA sequencing in selected cases and a pathway-specific immunohistochemistry (IHC) panel were performed using limited formalin-fixed paraffin embedded tissue from any disease episode available. The clinical impact of reported findings was assessed by a serial questionnaire-based follow-up.Results: Integrated molecular diagnostics resulted in refined or changed diagnosis in 117/263 (44%) tumours. Actionable targets were detected in 155/263 (59%) cases. Constitutional DNA variants with clinical relevance were identified in 16/240 (7%) of patients, half of which were previously unknown. Clinical follow-up showed that 26/263 (10%) of patients received mechanism-of-action based treatment matched to the molecular findings.Conclusion: Next-generation diagnostics adds robust and relevant information on diagnosis, actionable alterations and cancer predisposition syndromes even when tissue from the current disease episode is limited. 2022 Elsevier Ltd. All rights reserved.

Published
2023

38. Next-generation personalised medicine for high-risk paediatric cancer patients – The INFORM pilot study

Author

Worst, Barbara C., van Tilburg, Cornelis M., Balasubramanian, Gnana Prakash, Fiesel, Petra, Witt, Ruth, Freitag, Angelika, Boudalil, Miream, Previti, Christopher, Wolf, Stephan, Schmidt, Sabine, Chotewutmontri, Sasithorn, Bewerunge-Hudler, Melanie, Schick, Matthias, Schlesner, Matthias, Hutter, Barbara, Taylor, Lenka, Borst, Tobias, Sutter, Christian, Bartram, Claus R., Milde, Till, Pfaff, Elke, Kulozik, Andreas E., von Stackelberg, Arend, Meisel, Roland, Borkhardt, Arndt, Reinhardt, Dirk, Klusmann, Jan-Henning, Fleischhack, Gudrun, Tippelt, Stephan, Dirksen, Uta, Jürgens, Heribert, Kramm, Christof M., von Bueren, Andre O., Westermann, Frank, Fischer, Matthias, Burkhardt, Birgit, Wößmann, Wilhelm, Nathrath, Michaela, Bielack, Stefan S., Frühwald, Michael C., Fulda, Simone, Klingebiel, Thomas, Koscielniak, Ewa, Schwab, Matthias, Tremmel, Roman, Driever, Pablo Hernáiz, Schulte, Johannes H., Brors, Benedikt, von Deimling, Andreas, Lichter, Peter, Eggert, Angelika, Capper, David, Pfister, Stefan M., Jones, David T.W., and Witt, Olaf

Published
2016
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39. Safety and efficacy of mTOR inhibitor treatment in patients with tuberous sclerosis complex under 2 years of age – a multicenter retrospective study

Author

Saffari, Afshin, Brösse, Ines, Wiemer-Kruel, Adelheid, Wilken, Bernd, Kreuzaler, Paula, Hahn, Andreas, Bernhard, Matthias K., van Tilburg, Cornelis M., Hoffmann, Georg F., Gorenflo, Matthias, Hethey, Sven, Kaiser, Olaf, Kölker, Stefan, Wagner, Robert, Witt, Olaf, Merkenschlager, Andreas, Möckel, Andreas, Roser, Timo, Schlump, Jan-Ulrich, Serfling, Antje, Spiegler, Juliane, Milde, Till, Ziegler, Andreas, and Syrbe, Steffen

Published
2019
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40. Key Pharmacokinetic Parameters of 74 Pediatric Anticancer Drugs Providing Assistance in Preclinical Studies.

Author

Jamaladdin, Nora, Sigaud, Romain, Kocher, Daniela, Kolodziejczak, Anna S., Nonnenbroich, Leo F., Ecker, Jonas, Usta, Diren, Benzel, Julia, Peterziel, Heike, Pajtler, Kristian W., van Tilburg, Cornelis M., Oehme, Ina, Witt, Olaf, and Milde, Till

Subjects
ANTINEOPLASTIC agents, BLOOD-brain barrier, LITERATURE reviews, PHARMACOKINETICS, CHILD patients, PEDIATRIC oncology
Abstract

Novel drug treatments for pediatric patients with cancer are urgently needed. Success of drug development in pediatric oncology has been promising, but many drugs still fail in translation from preclinical to clinical phases. To increase the translational potential, several improvements have been implemented, including the use of clinically achievable concentrations in the drug testing phase. Although pharmacokinetic (PK) parameters of numerous investigated drugs are published, a comprehensive PK overview of the most common drugs in pediatric oncology could guide preclinical trial design and improve the translatability into clinical trials. A review of the literature was conducted for PK parameters of 74 anticancer drugs, from the drug sensitivity profiling library of the INdividualized Therapy FOr Relapsed Malignancies in Childhood (INFORM) registry. PK data in the pediatric population were reported and complemented by adult parameters when no pediatric data were available. In addition, blood–brain barrier (BBB)‐penetration assessment of drugs was provided by using the BBB score. Maximum plasma concentration was available for 73 (97%), area under the plasma concentration‐time curve for 69 (92%), plasma protein binding for 66 (88%), plasma half‐life for 57 (76%), time to maximum concentration for 54 (72%), clearance for 52 (69%), volume of distribution for 37 (49%), lowest plasma concentration reached by the drug before the next dose administration for 21 (28%), and steady‐state concentration for 4 (5%) of drugs. Pediatric PK data were available for 48 (65%) drugs. We provide a comprehensive review of PK data for 74 drugs studied in pediatric oncology. This data set can serve as a reference to design experiments more closely mimicking drug PK conditions in patients, and may thereby increase the probability of successful clinical translation. [ABSTRACT FROM AUTHOR]

Published
2023
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41. CTNI-62. LONG-TERM EFFICACY AND SAFETY OF LAROTRECTINIB IN PATIENTS WITH TROPOMYOSIN RECEPTOR KINASE (TRK) FUSION PRIMARY CENTRAL NERVOUS SYSTEM (CNS) TUMORS

Author

Perreault, Sébastien, primary, Drilon, Alexander, additional, Lassen, Ulrik N, additional, Geoerger, Birgit, additional, Nysom, Karsten, additional, Øra, Ingrid, additional, Gavrilovic, Igor T, additional, Norenberg, Ricarda, additional, Bernard-Gauthier, Vadim, additional, De La Cuesta, Esther, additional, Laetsch, Theodore W, additional, Doz, François, additional, and Van Tilburg, Cornelis M, additional

Published
2022
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42. BH3 mimetics targeting BCL-XL impact the senescent compartment of pilocytic astrocytoma

Author

Selt, Florian, primary, Sigaud, Romain, additional, Valinciute, Gintvile, additional, Sievers, Philipp, additional, Zaman, Julia, additional, Alcon, Clara, additional, Schmid, Simone, additional, Peterziel, Heike, additional, Tsai, Jessica W, additional, Guiho, Romain, additional, Martínez-Barbera, Juan Pedro, additional, Pusch, Stefan, additional, Deng, Jing, additional, Zhai, Yifan, additional, van Tilburg, Cornelis M, additional, Schuhman, Martin U, additional, El Damaty, Ahmed, additional, Bandopadhayay, Pratiti, additional, Herold-Mende, Christel, additional, von Deimling, Andreas, additional, Pfister, Stefan M, additional, Montero, Joan, additional, Capper, David, additional, Oehme, Ina, additional, Sahm, Felix, additional, Jones, David T W, additional, Witt, Olaf, additional, and Milde, Till, additional

Published
2022
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43. The first-in-class ERK inhibitor ulixertinib shows promising activity in mitogen-activated protein kinase (MAPK)-driven pediatric low-grade glioma models

Author

Sigaud, Romain, primary, Rösch, Lisa, additional, Gatzweiler, Charlotte, additional, Benzel, Julia, additional, von Soosten, Laura, additional, Peterziel, Heike, additional, Selt, Florian, additional, Najafi, Sara, additional, Ayhan, Simay, additional, Gerloff, Xenia F, additional, Hofmann, Nina, additional, Büdenbender, Isabel, additional, Schmitt, Lukas, additional, Foerster, Kathrin I, additional, Burhenne, Jürgen, additional, Haefeli, Walter E, additional, Korshunov, Andrey, additional, Sahm, Felix, additional, van Tilburg, Cornelis M, additional, Jones, David T W, additional, Pfister, Stefan M, additional, Knoerzer, Deborah, additional, Kreider, Brent L, additional, Sauter, Max, additional, Pajtler, Kristian W, additional, Zuckermann, Marc, additional, Oehme, Ina, additional, Witt, Olaf, additional, and Milde, Till, additional

Published
2022
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44. Abstract 5221: The first-in-class ERK inhibitor ulixertinib (BVD-523) shows activity in MAPK-driven pediatric low-grade glioma models as single agent and in combination with MEK inhibitors or senolytics

Author

Sigaud, Romain, primary, Rösch, Lisa, additional, Gatzweiler, Charlotte, additional, Benzel, Julia, additional, von Soosten, Laura, additional, Peterziel, Heike, additional, Najafi, Sara, additional, Ayhan, Simay, additional, Hofmann, Nina, additional, Förster, Kathrin I., additional, Burhenne, Jürgen, additional, Longuespée, Rémi, additional, van Tilburg, Cornelis M., additional, Jones, David T., additional, Pfister, Stefan M., additional, Knoerzer, Deborah, additional, Kreider, Brent, additional, Sauter, Max, additional, Pajtler, Kristian W., additional, Zuckermann, Marc, additional, Oehme, Ina, additional, Witt, Olaf, additional, and Milde, Till, additional

Published
2022
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45. Abstract CT545: Variability in NTRK gene fusions does not appear to impact response to larotrectinib

Author

Ladanyi, Marc, primary, Roy-Chowdhuri, Sinchita, additional, Ritterhouse, Lauren, additional, Sahm, Felix, additional, Norenberg, Ricarda, additional, Shen, Kui, additional, Chen, Chi, additional, Fellous, Marc, additional, Brega, Nicoletta, additional, van Tilburg, Cornelis M., additional, Lin, Jessica J., additional, Brose, Marcia S., additional, Lassen, Ulrik N., additional, McDermott, Ray, additional, Laetsch, Theodore W., additional, Hong, David S., additional, Drilon, Alexander, additional, and Rudzinski, Erin R., additional

Published
2022
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46. HGG-61.Landscape of cancer predisposition in pediatric high-grade glioma

Author

Blattner-Johnson, Mirjam, primary, Sturm, Dominik, additional, Sievers, Philipp, additional, Jones, Barbara, additional, Schramm, Kathrin, additional, Balasubramanian, Gnana Prakash, additional, Sill, Martin, additional, Jäger, Natalie, additional, van Tilburg, Cornelis M, additional, Milde, Till, additional, Pajtler, Kristian W, additional, Dikow, Nicola, additional, Hirsch, Steffen, additional, Sutter, Christian, additional, Sahm, Felix, additional, Witt, Olaf, additional, Pfister, Stefan M, additional, and Jones, David T W, additional

Published
2022
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47. OTHR-32. The Pediatric Targeted Therapy 2.0 registry: robust molecular diagnostics for precision oncology

Author

Ecker, Jonas, primary, Selt, Florian, additional, Sturm, Dominik, additional, Sill, Martin, additional, Korshunov, Andrey, additional, Capper, David, additional, Hirsch, Steffen, additional, Dikow, Nicola, additional, Sutter, Christian, additional, Müller, Carina, additional, Sigaud, Romain, additional, von Deimling, Andreas, additional, Pajtler, Kristian W, additional, van Tilburg, Cornelis M, additional, Jones, David T W, additional, Sahm, Felix, additional, Pfister, Stefan M, additional, Witt, Olaf, additional, and Milde, Till, additional

Published
2022
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48. LGG-25. The first-in-class ERK inhibitor ulixertinib (BVD-523) shows activity in MAPK-driven pediatric low-grade glioma models as single agent and in combination with MEK inhibitors or senolytics

Author

Sigaud, Romain, primary, Rösch, Lisa, additional, Gatzweiler, Charlotte, additional, Benzel, Julia, additional, von Soosten, Laura, additional, Peterziel, Heike, additional, Najafi, Sara, additional, Ayhan, Simay, additional, Gerloff, Xena F, additional, Hofmann, Nina, additional, Büdenbender, Isabel, additional, Foerster, Kathrin I, additional, Burhenne, Jürgen, additional, Longuespée, Rémi, additional, van Tilburg, Cornelis M, additional, Jones, David T W, additional, Pfister, Stefan M, additional, Knoerzer, Deborah, additional, Kreider, Brent, additional, Sauter, Max, additional, Pajtler, Kristian W, additional, Zuckermann, Marc, additional, Oehme, Ina, additional, Witt, Olaf, additional, and Milde, Till, additional

Published
2022
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49. HGG-18. Long-term efficacy and safety of larotrectinib in paediatric patients with tropomyosin receptor kinase (TRK) fusion-positive primary central nervous system (CNS) tumours

Author

Nysom, Karsten, primary, Doz, François, additional, Geoerger, Birgit, additional, Øra, Ingrid, additional, Perreault, Sebastien, additional, Norenberg, Ricarda, additional, Fellous, Marc, additional, De La Cuesta, Esther, additional, Laetsch, Theodore W, additional, and van Tilburg, Cornelis M, additional

Published
2022
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50. LGG-14. LOGGIC (Low Grade Glioma in Children) Core BioClinical Data Bank: Establishment and added clinical value of an international molecular diagnostic registry for pediatric low-grade glioma patients

Author

Hardin, Emily C, primary, Schmid, Simone, additional, Sommerkamp, Alexander, additional, le Simon, Michè, additional, Caspar, Claudia, additional, Bodden, Carina, additional, Heipertz, Anna-Elisa, additional, Sievers, Philipp, additional, Wittmann, Andrea, additional, Perera, Ashwyn A, additional, Milde, Till, additional, Pfister, Stefan M, additional, von Deimling, Andreas, additional, Driever, Pablo Hernáiz, additional, Koch, Arend, additional, Hargrave, Darren, additional, Witt, Olaf, additional, Capper, David, additional, Sahm, Felix, additional, Jones, David T W, additional, and van Tilburg, Cornelis M, additional

Published
2022
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