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657 results on '"Röllig, Christoph"'

1. How to customize common data models for rare diseases: an OMOP-based implementation and lessons learned

Author

Ahmadi, Najia, Zoch, Michele, Guengoeze, Oya, Facchinello, Carlo, Mondorf, Antonia, Stratmann, Katharina, Musleh, Khader, Erasmus, Hans-Peter, Tchertov, Jana, Gebler, Richard, Schaaf, Jannik, Frischen, Lena S., Nasirian, Azadeh, Dai, Jiabin, Henke, Elisa, Tremblay, Douglas, Srisuwananukorn, Andrew, Bornhäuser, Martin, Röllig, Christoph, Eckardt, Jan-Niklas, Middeke, Jan Moritz, Wolfien, Markus, and Sedlmayr, Martin

Published
2024
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2. Mimicking clinical trials with synthetic acute myeloid leukemia patients using generative artificial intelligence

Author

Eckardt, Jan-Niklas, Hahn, Waldemar, Röllig, Christoph, Stasik, Sebastian, Platzbecker, Uwe, Müller-Tidow, Carsten, Serve, Hubert, Baldus, Claudia D., Schliemann, Christoph, Schäfer-Eckart, Kerstin, Hanoun, Maher, Kaufmann, Martin, Burchert, Andreas, Thiede, Christian, Schetelig, Johannes, Sedlmayr, Martin, Bornhäuser, Martin, Wolfien, Markus, and Middeke, Jan Moritz

Published
2024
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3. Biallelic TET2 mutation sensitizes to 5’-azacitidine in acute myeloid leukemia

Author

Stölzel, Friedrich, Fordham, Sarah E, Nandana, Devi, Lin, Wei-Yu, Blair, Helen J, Elstob, Claire J, Bell, Hayden L, Mohr, Brigitte, Ruhnke, Leo, Kunadt, Desiree, Dill, Claudia, Allsop, Daniel A, Piddock, Rachel E, Soura, Emmanouela-Niki, Park, Catherine Vida, Fadly, Mohd, Rahman, Thahira, Alharbi, Abrar A, Wobus, Manja, Altmann, Heidi, Röllig, Christoph, Wagenführ, Lisa, Jones, Gail L, Menne, Tobias, Jackson, Graham H, Marr, Helen J, Fitzgibbon, Jude, Onel, Kenan, Meggendorfer, Manja, Robinson, Amber, Bziuk, Zuzanna, Bowes, Emily, Heidenreich, Olaf, Haferlach, Torsten, Villar, Sara, Ariceta, Beñat, Diaz, Rosa Ayala, Altschuler, Steven J, Wu, Lani, Prosper, Felipe, Montesinos, Pau, Martinez-Lopez, Joaquin, Bornhäuser, Martin, and Allan, James M

Subjects
Biomedical and Clinical Sciences, Oncology and Carcinogenesis, Cancer, Hematology, Genetics, Pediatric, Pediatric Cancer, Rare Diseases, Human Genome, 2.1 Biological and endogenous factors, Aetiology, Good Health and Well Being, Humans, Mice, Animals, Azacitidine, Leukemia, Myeloid, Acute, Kaplan-Meier Estimate, Mutation, DNA-Binding Proteins, Dioxygenases, Leukemias, Molecular genetics, Biomedical and clinical sciences, Health sciences
Abstract

Precision medicine can significantly improve outcomes for patients with cancer, but implementation requires comprehensive characterization of tumor cells to identify therapeutically exploitable vulnerabilities. Here, we describe somatic biallelic TET2 mutations in an elderly patient with acute myeloid leukemia (AML) that was chemoresistant to anthracycline and cytarabine but acutely sensitive to 5'-azacitidine (5'-Aza) hypomethylating monotherapy, resulting in long-term morphological remission. Given the role of TET2 as a regulator of genomic methylation, we hypothesized that mutant TET2 allele dosage affects response to 5'-Aza. Using an isogenic cell model system and an orthotopic mouse xenograft, we demonstrate that biallelic TET2 mutations confer sensitivity to 5'-Aza compared with cells with monoallelic mutations. Our data argue in favor of using hypomethylating agents for chemoresistant disease or as first-line therapy in patients with biallelic TET2-mutated AML and demonstrate the importance of considering mutant allele dosage in the implementation of precision medicine for patients with cancer.

Published
2023

4. Impact of BMI on patient outcome in acute myeloid leukaemia patients receiving intensive induction therapy: a real-world registry experience

Author

Enßle, Julius C., Wolf, Sebastian, Scheich, Sebastian, Weber, Sarah, Kramer, Michael, Ruhnke, Leo, Schliemann, Christoph, Mikesch, Jan-Henrik, Krause, Stefan, Sauer, Tim, Hanoun, Maher, Reinhardt, Hans Christian, Kraus, Sabrina, Kaufmann, Martin, Hänel, Mathias, Fransecky, Lars, Burchert, Andreas, Neubauer, Andreas, Crysandt, Martina, Jost, Edgar, Niemann, Dirk, Schäfer-Eckart, Kerstin, Held, Gerhard, Kaiser, Ulrich, Wass, Maxi, Schaich, Markus, Müller-Tidow, Carsten, Platzbecker, Uwe, Baldus, Claudia D., Bornhäuser, Martin, Röllig, Christoph, Serve, Hubert, and Steffen, Björn

Published
2023
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5. Q-HAM: a multicenter upfront randomized phase II trial of quizartinib and high-dose Ara-C plus mitoxantrone in relapsed/refractory AML with FLT3-ITD

Author

Jaramillo, Sonia, Le Cornet, Lucian, Kratzmann, Markus, Krisam, Johannes, Görner, Martin, Hänel, Mathias, Röllig, Christoph, Wass, Maxi, Scholl, Sebastian, Ringhoffer, Mark, Reichart, Alexander, Steffen, Björn, Kayser, Sabine, Mikesch, Jan-Henrik, Schaefer-Eckart, Kerstin, Schubert, Jörg, Geer, Thomas, Martin, Sonja, Kieser, Meinhard, Sauer, Tim, Kriegsmann, Katharina, Hundemer, Michael, Serve, Hubert, Bornhäuser, Martin, Müller-Tidow, Carsten, and Schlenk, Richard F.

Published
2023
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6. UBTF tandem duplications are rare but recurrent alterations in adult AML and associated with younger age, myelodysplasia, and inferior outcome

Author

Georgi, Julia-Annabell, Stasik, Sebastian, Eckardt, Jan-Niklas, Zukunft, Sven, Hartwig, Marita, Röllig, Christoph, Middeke, Jan Moritz, Oelschlägel, Uta, Krug, Utz, Sauer, Tim, Scholl, Sebastian, Hochhaus, Andreas, Brümmendorf, Tim H., Naumann, Ralph, Steffen, Björn, Einsele, Hermann, Schaich, Markus, Burchert, Andreas, Neubauer, Andreas, Schäfer-Eckart, Kerstin, Schliemann, Christoph, Krause, Stefan W., Hänel, Mathias, Noppeney, Richard, Kaiser, Ulrich, Baldus, Claudia D., Kaufmann, Martin, Müller-Tidow, Carsten, Platzbecker, Uwe, Berdel, Wolfgang E., Serve, Hubert, Ehninger, Gerhard, Bornhäuser, Martin, Schetelig, Johannes, Kroschinsky, Frank, and Thiede, Christian

Published
2023
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7. Unsupervised meta-clustering identifies risk clusters in acute myeloid leukemia based on clinical and genetic profiles

Author

Eckardt, Jan-Niklas, Röllig, Christoph, Metzeler, Klaus, Heisig, Peter, Stasik, Sebastian, Georgi, Julia-Annabell, Kroschinsky, Frank, Stölzel, Friedrich, Platzbecker, Uwe, Spiekermann, Karsten, Krug, Utz, Braess, Jan, Görlich, Dennis, Sauerland, Cristina, Woermann, Bernhard, Herold, Tobias, Hiddemann, Wolfgang, Müller-Tidow, Carsten, Serve, Hubert, Baldus, Claudia D., Schäfer-Eckart, Kerstin, Kaufmann, Martin, Krause, Stefan W., Hänel, Mathias, Berdel, Wolfgang E., Schliemann, Christoph, Mayer, Jiri, Hanoun, Maher, Schetelig, Johannes, Wendt, Karsten, Bornhäuser, Martin, Thiede, Christian, and Middeke, Jan Moritz

Published
2023
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8. Alterations of cohesin complex genes in acute myeloid leukemia: differential co-mutations, clinical presentation and impact on outcome

Author

Eckardt, Jan-Niklas, Stasik, Sebastian, Röllig, Christoph, Sauer, Tim, Scholl, Sebastian, Hochhaus, Andreas, Crysandt, Martina, Brümmendorf, Tim H., Naumann, Ralph, Steffen, Björn, Kunzmann, Volker, Einsele, Hermann, Schaich, Markus, Burchert, Andreas, Neubauer, Andreas, Schäfer-Eckart, Kerstin, Schliemann, Christoph, Krause, Stefan W., Herbst, Regina, Hänel, Mathias, Hanoun, Maher, Kaiser, Ulrich, Kaufmann, Martin, Rácil, Zdenek, Mayer, Jiri, Cerqueira, Tiago, Kroschinsky, Frank, Berdel, Wolfgang E., Serve, Hubert, Müller-Tidow, Carsten, Platzbecker, Uwe, Baldus, Claudia D., Schetelig, Johannes, Siepmann, Timo, Bornhäuser, Martin, Middeke, Jan Moritz, and Thiede, Christian

Published
2023
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9. Venetoclax-based salvage therapy as a bridge to transplant is feasible and effective in patients with relapsed/refractory AML

Author

Unglaub, Julia M., Schlenk, Richard F., Middeke, Jan Moritz, Krause, Stefan W., Kraus, Sabrina, Einsele, Hermann, Kramer, Michael, Zukunft, Sven, Kauer, Joseph, Renders, Simon, Katelari, Elena, Schliemann, Christoph, Pabst, Caroline, Luft, Thomas, Dreger, Peter, Röllig, Christoph, Bornhäuser, Martin, Müller-Tidow, Carsten, and Sauer, Tim

Published
2024
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10. Large case-control study indicates no association of KIR genotype and risk of developing acute myeloid leukemia

Author

Heidenreich, Falk, Falk, Bose, Baldauf, Henning, Massalski, Carolin, Schäfer, Gesine, Rücker-Braun, Elke, Altmann, Heidi, Sauter, Jürgen, Solloch, Ute V., Lange, Vinzenz, Stölzel, Friedrich, Röllig, Christoph, Middeke, Jan M., von Bonin, Malte, Thiede, Christian, Schäfer-Eckart, Kerstin, Müller-Tidow, Carsten, Krause, Stefan W., Kraus, Sabrina, Kaufmann, Martin, Hänel, Mathias, Serve, Hubert, Neubauer, Andreas, Bornhäuser, Martin, Schmidt, Alexander H., and Schetelig, Johannes

Published
2023
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11. Retained functional normal and preleukemic HSCs at diagnosis are associated with good prognosis in DNMT3AmutNPM1mut AMLs

Author

Donato, Elisa, Correia, Nadia, Andresen, Carolin, Karpova, Darja, Würth, Roberto, Klein, Corinna, Sohn, Markus, Przybylla, Adriana, Zeisberger, Petra, Rothfelder, Kathrin, Salih, Helmut, Bonig, Halvard, Stasik, Sebastian, Röllig, Christoph, Dolnik, Anna, Bullinger, Lars, Buchholz, Frank, Thiede, Christian, Hübschmann, Daniel, and Trumpp, Andreas

Published
2023
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12. Overlapping features of therapy-related and de novo NPM1-mutated AML

Author

Othman, Jad, Meggendorfer, Manja, Tiacci, Enrico, Thiede, Christian, Schlenk, Richard, Dillon, Richard, Stasik, Sebastian, Venanzi, Alessandra, Bertoli, Sarah, Delabesse, Eric, Dumas, Pierre-Yves, Pigneux, Arnaud, Bidet, Audrey, Gilkes, Amanda F., Thomas, Ian, Voso, Maria Teresa, Rambaldi, Alessandro, Brunetti, Lorenzo, Perriello, Vincenzo M., Andresen, Vibeke, Gjertsen, Bjorn T., Martelli, Maria Paola, Récher, Christian, Röllig, Christoph, Bornhäuser, Martin, Serve, Hubert, Müller-Tidow, Carsten, Baldus, Claudia D., Haferlach, Tortsten, Russell, Nigel, and Falini, Brunangelo

Published
2023
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13. Venetoclax synergizes with gilteritinib in FLT3 wild-type high-risk acute myeloid leukemia by suppressing MCL-1

Author

Janssen, Maike, Schmidt, Christina, Bruch, Peter-Martin, Blank, Maximilian F., Rohde, Christian, Waclawiczek, Alexander, Heid, Daniel, Renders, Simon, Göllner, Stefanie, Vierbaum, Lisa, Besenbeck, Birgit, Herbst, Sophie A., Knoll, Mareike, Kolb, Carolin, Przybylla, Adriana, Weidenauer, Katharina, Ludwig, Anne Kathrin, Fabre, Margarete, Gu, Muxin, Schlenk, Richard F., Stölzel, Friedrich, Bornhäuser, Martin, Röllig, Christoph, Platzbecker, Uwe, Baldus, Claudia, Serve, Hubert, Sauer, Tim, Raffel, Simon, Pabst, Caroline, Vassiliou, George, Vick, Binje, Jeremias, Irmela, Trumpp, Andreas, Krijgsveld, Jeroen, Müller-Tidow, Carsten, and Dietrich, Sascha

Published
2022
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14. Diagnosis and management of AML in adults: 2022 recommendations from an international expert panel on behalf of the ELN

Author

Döhner, Hartmut, Wei, Andrew H., Appelbaum, Frederick R., Craddock, Charles, DiNardo, Courtney D., Dombret, Hervé, Ebert, Benjamin L., Fenaux, Pierre, Godley, Lucy A., Hasserjian, Robert P., Larson, Richard A., Levine, Ross L., Miyazaki, Yasushi, Niederwieser, Dietger, Ossenkoppele, Gert, Röllig, Christoph, Sierra, Jorge, Stein, Eytan M., Tallman, Martin S., Tien, Hwei-Fang, Wang, Jianxiang, Wierzbowska, Agnieszka, and Löwenberg, Bob

Published
2022
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15. Idasanutlin plus cytarabine in relapsed or refractory acute myeloid leukemia: results of the MIRROS trial

Author

Konopleva, Marina Y., Röllig, Christoph, Cavenagh, Jamie, Deeren, Dries, Girshova, Larisa, Krauter, Jürgen, Martinelli, Giovanni, Montesinos, Pau, Schäfer, Jonas A., Ottmann, Oliver, Petrini, Mario, Pigneux, Arnaud, Rambaldi, Alessandro, Recher, Christian, Rodriguez-Veiga, Rebeca, Taussig, David, Vey, Norbert, Yoon, Sung-Soo, Ott, Marion, Muehlbauer, Susanne, Beckermann, Benjamin M., Catalani, Olivier, Genevray, Magali, Mundt, Kirsten, Jamois, Candice, Fenaux, Pierre, and Wei, Andrew H.

Published
2022
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16. Using stroma-anchoring cytokines to augment ADCC: a phase 1 trial of F16IL2 and BI 836858 for posttransplant AML relapse

Author

Berdel, Andrew F., Ruhnke, Leo, Angenendt, Linus, Wermke, Martin, Röllig, Christoph, Mikesch, Jan-Henrik, Scheller, Annika, Hemmerle, Teresa, Matasci, Mattia, Wethmar, Klaus, Kessler, Torsten, Gerwing, Mirjam, Hescheler, Daniel, Schäfers, Michael, Hartmann, Wolfgang, Altvater, Bianca, Rossig, Claudia, Bornhäuser, Martin, Lenz, Georg, Stelljes, Matthias, Rueter, Bjoern, Neri, Dario, Berdel, Wolfgang E., and Schliemann, Christoph

Published
2022
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18. Deep learning identifies Acute Promyelocytic Leukemia in bone marrow smears

Author

Eckardt, Jan-Niklas, Schmittmann, Tim, Riechert, Sebastian, Kramer, Michael, Sulaiman, Anas Shekh, Sockel, Katja, Kroschinsky, Frank, Schetelig, Johannes, Wagenführ, Lisa, Schuler, Ulrich, Platzbecker, Uwe, Thiede, Christian, Stölzel, Friedrich, Röllig, Christoph, Bornhäuser, Martin, Wendt, Karsten, and Middeke, Jan Moritz

Published
2022
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19. Impact of IDH1 and IDH2 mutational subgroups in AML patients after allogeneic stem cell transplantation

Author

Kunadt, Desiree, Stasik, Sebastian, Metzeler, Klaus H., Röllig, Christoph, Schliemann, Christoph, Greif, Philipp A., Spiekermann, Karsten, Rothenberg-Thurley, Maja, Krug, Utz, Braess, Jan, Krämer, Alwin, Hochhaus, Andreas, Scholl, Sebastian, Hilgendorf, Inken, Brümmendorf, Tim H., Jost, Edgar, Steffen, Björn, Bug, Gesine, Einsele, Hermann, Görlich, Dennis, Sauerland, Cristina, Schäfer-Eckart, Kerstin, Krause, Stefan W., Hänel, Mathias, Hanoun, Maher, Kaufmann, Martin, Wörmann, Bernhard, Kramer, Michael, Sockel, Katja, Egger-Heidrich, Katharina, Herold, Tobias, Ehninger, Gerhard, Burchert, Andreas, Platzbecker, Uwe, Berdel, Wolfgang E., Müller-Tidow, Carsten, Hiddemann, Wolfgang, Serve, Hubert, Stelljes, Matthias, Baldus, Claudia D., Neubauer, Andreas, Schetelig, Johannes, Thiede, Christian, Bornhäuser, Martin, Middeke, Jan M., and Stölzel, Friedrich

Published
2022
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20. Author Correction: Genome-wide association study identifies susceptibility loci for acute myeloid leukemia

Author

Lin, Wei-Yu, Fordham, Sarah E., Hungate, Eric, Sunter, Nicola J., Elstob, Claire, Xu, Yaobo, Park, Catherine, Quante, Anne, Strauch, Konstantin, Gieger, Christian, Skol, Andrew, Rahman, Thahira, Sucheston-Campbell, Lara, Wang, Junke, Hahn, Theresa, Clay-Gilmour, Alyssa I., Jones, Gail L., Marr, Helen J., Jackson, Graham H., Menne, Tobias, Collin, Mathew, Ivey, Adam, Hills, Robert K., Burnett, Alan K., Russell, Nigel H., Fitzgibbon, Jude, Larson, Richard A., Le Beau, Michelle M., Stock, Wendy, Heidenreich, Olaf, Alharbi, Abrar, Allsup, David J., Houlston, Richard S., Norden, Jean, Dickinson, Anne M., Douglas, Elisabeth, Lendrem, Clare, Daly, Ann K., Palm, Louise, Piechocki, Kim, Jeffries, Sally, Bornhäuser, Martin, Röllig, Christoph, Altmann, Heidi, Ruhnke, Leo, Kunadt, Desiree, Wagenführ, Lisa, Cordell, Heather J., Darlay, Rebecca, Andersen, Mette K., Fontana, Maria C., Martinelli, Giovanni, Marconi, Giovanni, Sanz, Miguel A., Cervera, José, Gómez-Seguí, Inés, Cluzeau, Thomas, Moreilhon, Chimène, Raynaud, Sophie, Sill, Heinz, Voso, Maria Teresa, Lo-Coco, Francesco, Dombret, Hervé, Cheok, Meyling, Preudhomme, Claude, Gale, Rosemary E., Linch, David, Gaal-Wesinger, Julia, Masszi, Andras, Nowak, Daniel, Hofmann, Wolf-Karsten, Gilkes, Amanda, Porkka, Kimmo, Milosevic Feenstra, Jelena D., Kralovics, Robert, Grimwade, David, Meggendorfer, Manja, Haferlach, Torsten, Krizsán, Szilvia, Bödör, Csaba, Stölzel, Friedrich, Onel, Kenan, and Allan, James M.

Published
2022
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21. Molecular profiling and clinical implications of patients with acute myeloid leukemia and extramedullary manifestations

Author

Eckardt, Jan-Niklas, Stölzel, Friedrich, Kunadt, Desiree, Röllig, Christoph, Stasik, Sebastian, Wagenführ, Lisa, Jöhrens, Korinna, Kuithan, Friederike, Krämer, Alwin, Scholl, Sebastian, Hochhaus, Andreas, Crysandt, Martina, Brümmendorf, Tim H., Naumann, Ralph, Steffen, Björn, Kunzmann, Volker, Einsele, Hermann, Schaich, Markus, Burchert, Andreas, Neubauer, Andreas, Schäfer-Eckart, Kerstin, Schliemann, Christoph, Krause, Stefan W., Herbst, Regina, Hänel, Mathias, Hanoun, Maher, Kaiser, Ulrich, Kaufmann, Martin, Rácil, Zdenek, Mayer, Jiri, Kroschinsky, Frank, Berdel, Wolfgang E., Ehninger, Gerhard, Serve, Hubert, Müller-Tidow, Carsten, Platzbecker, Uwe, Baldus, Claudia D., Schetelig, Johannes, Bornhäuser, Martin, Thiede, Christian, and Middeke, Jan Moritz

Published
2022
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22. Differential impact of IDH1/2 mutational subclasses on outcome in adult AML: results from a large multicenter study

Author

Middeke, Jan M., Metzeler, Klaus H., Röllig, Christoph, Krämer, Michael, Eckardt, Jan-Niklas, Stasik, Sebastian, Greif, Philipp A., Spiekermann, Karsten, Rothenberg-Thurley, Maja, Krug, Utz, Braess, Jan, Krämer, Alwin, Hochhaus, Andreas, Brümmendorf, Tim H., Naumann, Ralph, Steffen, Björn, Einsele, Hermann, Schaich, Markus, Burchert, Andreas, Neubauer, Andreas, Görlich, Dennis, Sauerland, Cristina, Schäfer-Eckart, Kerstin, Schliemann, Christoph, Krause, Stefan W., Hänel, Mathias, Frickhofen, Norbert, Noppeney, Richard, Kaiser, Ulrich, Kaufmann, Martin, Kunadt, Desiree, Wörmann, Bernhard, Sockel, Katja, von Bonin, Malte, Herold, Tobias, Müller-Tidow, Carsten, Platzbecker, Uwe, Berdel, Wolfgang E., Serve, Hubert, Baldus, Claudia D., Ehninger, Gerhard, Schetelig, Johannes, Hiddemann, Wolfgang, Bornhäuser, Martin, Stölzel, Friedrich, and Thiede, Christian

Published
2022
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23. CEBPA mutations in 4708 patients with acute myeloid leukemia: differential impact of bZIP and TAD mutations on outcome

Author

Taube, Franziska, Georgi, Julia Annabell, Kramer, Michael, Stasik, Sebastian, Middeke, Jan Moritz, Röllig, Christoph, Krug, Utz, Krämer, Alwin, Scholl, Sebastian, Hochhaus, Andreas, Brümmendorf, Tim H., Naumann, Ralph, Petzold, Andreas, Mulet-Lazaro, Roger, Valk, Peter J.M., Steffen, Björn, Einsele, Hermann, Schaich, Markus, Burchert, Andreas, Neubauer, Andreas, Schäfer-Eckart, Kerstin, Schliemann, Christoph, Krause, Stefan W., Hänel, Mathias, Noppeney, Richard, Kaiser, Ulrich, Baldus, Claudia D., Kaufmann, Martin, Herold, Sylvia, Stölzel, Friedrich, Sockel, Katja, von Bonin, Malte, Müller-Tidow, Carsten, Platzbecker, Uwe, Berdel, Wolfgang E., Serve, Hubert, Ehninger, Gerhard, Bornhäuser, Martin, Schetelig, Johannes, and Thiede, Christian

Published
2022
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24. Impact of PTPN11 mutations on clinical outcome analyzed in 1529 patients with acute myeloid leukemia

Author

Stasik, Sebastian, Eckardt, Jan-Niklas, Kramer, Michael, Röllig, Christoph, Krämer, Alwin, Scholl, Sebastian, Hochhaus, Andreas, Crysandt, Martina, Brümmendorf, Tim H., Naumann, Ralph, Steffen, Björn, Kunzmann, Volker, Einsele, Hermann, Schaich, Markus, Burchert, Andreas, Neubauer, Andreas, Schäfer-Eckart, Kerstin, Schliemann, Christoph, Krause, Stefan, Herbst, Regina, Hänel, Mathias, Frickhofen, Norbert, Noppeney, Richard, Kaiser, Ulrich, Baldus, Claudia D., Kaufmann, Martin, Rácil, Zdenek, Platzbecker, Uwe, Berdel, Wolfgang E., Mayer, Jiri, Serve, Hubert, Müller-Tidow, Carsten, Ehninger, Gerhard, Bornhäuser, Martin, Schetelig, Johannes, Middeke, Jan M., and Thiede, Christian

Published
2021
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25. A scoring system for AML patients aged 70 years or older, eligible for intensive chemotherapy: a study based on a large European data set using the DATAML, SAL, and PETHEMA registries

Author

Bérard, Emilie, Röllig, Christoph, Bertoli, Sarah, Pigneux, Arnaud, Tavitian, Suzanne, Kramer, Michael, Serve, Hubert, Bornhäuser, Martin, Platzbecker, Uwe, Müller-Tidow, Carsten, Baldus, Claudia D., Martínez-Cuadrón, David, Serrano, Josefina, Martínez-Sánchez, Pilar, Arbolí, Eduardo Rodríguez, Gil, Cristina, Bergua, Juan, Bernal, Teresa, de la Fuente Burguera, Adolfo, Delabesse, Eric, Bidet, Audrey, Dumas, Pierre-Yves, Montesinos, Pau, and Récher, Christian

Published
2022
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26. Selective inhibition of FLT3 by gilteritinib in relapsed or refractory acute myeloid leukaemia: a multicentre, first-in-human, open-label, phase 1–2 study

Author

Perl, Alexander E, Altman, Jessica K, Cortes, Jorge, Smith, Catherine, Litzow, Mark, Baer, Maria R, Claxton, David, Erba, Harry P, Gill, Stan, Goldberg, Stuart, Jurcic, Joseph G, Larson, Richard A, Liu, Chaofeng, Ritchie, Ellen, Schiller, Gary, Spira, Alexander I, Strickland, Stephen A, Tibes, Raoul, Ustun, Celalettin, Wang, Eunice S, Stuart, Robert, Röllig, Christoph, Neubauer, Andreas, Martinelli, Giovanni, Bahceci, Erkut, and Levis, Mark

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Oncology and Carcinogenesis, Rare Diseases, Clinical Trials and Supportive Activities, Patient Safety, Lung, Clinical Research, Cancer, Infectious Diseases, Genetics, Hematology, Evaluation of treatments and therapeutic interventions, 6.1 Pharmaceuticals, Aged, Aniline Compounds, Antineoplastic Agents, Blood Platelets, Female, Humans, Leukemia, Myeloid, Acute, Male, Maximum Tolerated Dose, Middle Aged, Phosphorylation, Pyrazines, Recurrence, Retreatment, fms-Like Tyrosine Kinase 3, Oncology & Carcinogenesis, Oncology and carcinogenesis
Abstract

BackgroundInternal tandem duplication mutations in FLT3 are common in acute myeloid leukaemia and are associated with rapid relapse and short overall survival. The clinical benefit of FLT3 inhibitors in patients with acute myeloid leukaemia has been limited by rapid generation of resistance mutations, particularly in codon Asp835 (D835). We aimed to assess the highly selective oral FLT3 inhibitor gilteritinib in patients with relapsed or refractory acute myeloid leukaemia.MethodsIn this phase 1-2 trial, we enrolled patients aged 18 years or older with acute myeloid leukaemia who either were refractory to induction therapy or had relapsed after achieving remission with previous treatment. Patients were enrolled into one of seven dose-escalation or dose-expansion cohorts assigned to receive once-daily doses of oral gilteritinib (20 mg, 40 mg, 80 mg, 120 mg, 200 mg, 300 mg, or 450 mg). Cohort expansion was based on safety and tolerability, FLT3 inhibition in correlative assays, and antileukaemic activity. Although the presence of an FLT3 mutation was not an inclusion criterion, we required ten or more patients with locally confirmed FLT3 mutations (FLT3mut+) to be enrolled in expansion cohorts at each dose level. On the basis of emerging findings, we further expanded the 120 mg and 200 mg dose cohorts to include FLT3mut+ patients only. The primary endpoints were the safety, tolerability, and pharmacokinetics of gilteritinib. Safety and tolerability were assessed in the safety analysis set (all patients who received at least one dose of gilteritinib). Responses were assessed in the full analysis set (all patients who received at least one dose of study drug and who had at least one datapoint post-treatment). Pharmacokinetics were assessed in a subset of the safety analysis set for which sufficient data for concentrations of gilteritinib in plasma were available to enable derivation of one or more pharmacokinetic variables. This study is registered with ClinicalTrials.gov, number NCT02014558, and is ongoing.FindingsBetween Oct 15, 2013, and Aug 27, 2015, 252 adults with relapsed or refractory acute myeloid leukaemia received oral gilteritinib once daily in one of seven dose-escalation (n=23) or dose-expansion (n=229) cohorts. Gilteritinib was well tolerated; the maximum tolerated dose was established as 300 mg/day when two of three patients enrolled in the 450 mg dose-escalation cohort had two dose-limiting toxicities (grade 3 diarrhoea and grade 3 elevated aspartate aminotransferase). The most common grade 3-4 adverse events irrespective of relation to treatment were febrile neutropenia (97 [39%] of 252), anaemia (61 [24%]), thrombocytopenia (33 [13%]), sepsis (28 [11%]), and pneumonia (27 [11%]). Commonly reported treatment-related adverse events were diarrhoea (92 [37%] of 252]), anaemia (86 [34%]), fatigue (83 [33%]), elevated aspartate aminotransferase (65 [26%]), and increased alanine aminotransferase (47 [19%]). Serious adverse events occurring in 5% or more of patients were febrile neutropenia (98 [39%] of 252; five related to treatment), progressive disease (43 [17%]), sepsis (36 [14%]; two related to treatment), pneumonia (27 [11%]), acute renal failure (25 [10%]; five related to treatment), pyrexia (21 [8%]; three related to treatment), bacteraemia (14 [6%]; one related to treatment), and respiratory failure (14 [6%]). 95 people died in the safety analysis set, of which seven deaths were judged possibly or probably related to treatment (pulmonary embolism [200 mg/day], respiratory failure [120 mg/day], haemoptysis [80 mg/day], intracranial haemorrhage [20 mg/day], ventricular fibrillation [120 mg/day], septic shock [80 mg/day], and neutropenia [120 mg/day]). An exposure-related increase in inhibition of FLT3 phosphorylation was noted with increasing concentrations in plasma of gilteritinib. In-vivo inhibition of FLT3 phosphorylation occurred at all dose levels. At least 90% of FLT3 phosphorylation inhibition was seen by day 8 in most patients receiving a daily dose of 80 mg or higher. 100 (40%) of 249 patients in the full analysis set achieved a response, with 19 (8%) achieving complete remission, ten (4%) complete remission with incomplete platelet recovery, 46 (18%) complete remission with incomplete haematological recovery, and 25 (10%) partial remission INTERPRETATION: Gilteritinib had a favourable safety profile and showed consistent FLT3 inhibition in patients with relapsed or refractory acute myeloid leukaemia. These findings confirm that FLT3 is a high-value target for treatment of relapsed or refractory acute myeloid leukaemia; based on activity data, gilteritinib at 120 mg/day is being tested in phase 3 trials.FundingAstellas Pharma, National Cancer Institute (Leukemia Specialized Program of Research Excellence grant), Associazione Italiana Ricerca sul Cancro.

Published
2017

27. Allogeneic Stem Cell Transplantation with Sequential Melphalan-Based Conditioning in AML: Residual Morphological Blast Count Determines the Risk of Relapse

Author

Sockel, Katja, Stölzel, Friedrich, Hönl, Franziska, Baldauf, Henning, Röllig, Christoph, Wermke, Martin, Bonin, Malte von, Teipel, Raphael, Link-Rachner, Cornelia, Brandt, Kalina, Kroschinsky, Frank, Hänel, Mathias, Morgner, Anke, Klesse, Christian, Ehninger, Gerhard, Platzbecker, Uwe, Bornhäuser, Martin, Schetelig, Johannes, Moritz Middeke, Jan, Sockel, Katja, Stölzel, Friedrich, Hönl, Franziska, Baldauf, Henning, Röllig, Christoph, Wermke, Martin, Bonin, Malte von, Teipel, Raphael, Link-Rachner, Cornelia, Brandt, Kalina, Kroschinsky, Frank, Hänel, Mathias, Morgner, Anke, Klesse, Christian, Ehninger, Gerhard, Platzbecker, Uwe, Bornhäuser, Martin, Schetelig, Johannes, and Moritz Middeke, Jan

Abstract

Introduction: Allogeneic hematopoietic cell transplantation (HCT) during chemotherapy-induced aplasia may offer long-term survival in acute myeloid leukemia (AML) with otherwise poor prognosis including ELN adverse risk, relapsed or refractory disease. However, the value of residual morphologic disease prior HCT in this context has not been conclusively settled until yet. Therefore, we aimed to investigate variables predicting outcome in this unique setting of sequential conditioning therapy, with a focus on pretreatment morphologic blast count. In contrast to the most popular FLAMSA-RIC protocol, we used a melphalan-based conditioning regimen during aplasia. Methods: We retrospectively analyzed data from 173 AML patients who underwent a sequential melphalan-based conditioning therapy between 2003 and 2015 at our centre. All patients participated either in the prospective Phase 2 BRIDGE trial (NCT01295307), the Phase 3 AML2003 study (NCT00180102) or were treated according to this protocol and underwent allogeneic HCT after melphalan-based conditioning in treatment-induced aplasia. Results: Median bone marrow blast count prior to conditioning was 10% (range, 0–96%). Four year probabilities of EFS and OS were 34% (95% CI, 28–43%) and 43% (95% CI, 36–52%), respectively. In multivariate analysis, blast count >20% was associated with worse EFS (HR = 1.93; p = 0.009) and OS (HR = 1.80; p = 0.026). This effect was not significant anymore for HCT during 1st line therapy. Conclusion: Allogeneic HCT in aplasia with a melphalan-based conditioning regimen has the potential to cure a subset of adverse risk AML patients, even with persistent morphological disease prior HCT. However, a high pre-transplant blast count still indicates patients with a dismal prognosis, especially in the relapsed patient group, for whom post-transplant strategies should be considered to further optimize post HCT outcome.

Published
2024

28. Prognostic impact of CEBPA mutational subgroups in adult AML

Author

Georgi, Julia Annabell, Stasik, Sebastian, Kramer, Michael, Meggendorfer, Manja, Röllig, Christoph, Haferlach, Torsten, Valk, Peter, Linch, David, Herold, Tobias, Duployez, Nicolas, Taube, Franziska, Middeke, Jan Moritz, Platzbecker, Uwe, Serve, Hubert, Baldus, Claudia D., Muller-Tidow, Carsten, Haferlach, Claudia, Koch, Sarah, Berdel, Wolfgang E., Woermann, Bernhard J., Krug, Utz, Braess, Jan, Hiddemann, Wolfgang, Spiekermann, Karsten, Boertjes, Emma L., Hills, Robert K., Burnett, Alan, Ehninger, Gerhard, Metzeler, Klaus, Rothenberg-Thurley, Maja, Dufour, Annika, Dombret, Hervé, Pautas, Cecile, Preudhomme, Claude, Fenwarth, Laurene, Bornhäuser, Martin, Gale, Rosemary, Thiede, Christian, Georgi, Julia Annabell, Stasik, Sebastian, Kramer, Michael, Meggendorfer, Manja, Röllig, Christoph, Haferlach, Torsten, Valk, Peter, Linch, David, Herold, Tobias, Duployez, Nicolas, Taube, Franziska, Middeke, Jan Moritz, Platzbecker, Uwe, Serve, Hubert, Baldus, Claudia D., Muller-Tidow, Carsten, Haferlach, Claudia, Koch, Sarah, Berdel, Wolfgang E., Woermann, Bernhard J., Krug, Utz, Braess, Jan, Hiddemann, Wolfgang, Spiekermann, Karsten, Boertjes, Emma L., Hills, Robert K., Burnett, Alan, Ehninger, Gerhard, Metzeler, Klaus, Rothenberg-Thurley, Maja, Dufour, Annika, Dombret, Hervé, Pautas, Cecile, Preudhomme, Claude, Fenwarth, Laurene, Bornhäuser, Martin, Gale, Rosemary, and Thiede, Christian

Abstract

Despite recent refinements in the diagnostic and prognostic assessment of CEBPA mutations in AML, several questions remain open, i.e. implications of different types of basic region leucin zipper (bZIP) mutations, the role of co-mutations and the allelic state. Using pooled primary data analysis on 1010 CEBPA-mutant adult AML patients, a comparison was performed taking into account the type of mutation (bZIP: either typical in-frame insertion/deletion (InDel) mutations (bZIP InDel), frameshift InDel or nonsense mutations inducing translational stop (bZIP STOP) or single base-pair missense alterations (bZIP ms), and transcription activation domain (TAD) mutations) and the allelic state (single (smCEBPA) vs. double mutant (dmCEBPA)). Only bZIP InDel patients had significantly higher rates of complete remission and longer relapse free and overall survival (OS) compared with all other CEBPA-mutant subgroups. Moreover, co-mutations in bZIP InDel patients (e.g. GATA2, FLT3, WT1 as well as ELN2022 adverse risk aberrations) had no independent impact on OS, whereas in non-bZIP InDel patients, grouping according to ELN2022 recommendations added significant prognostic information. In conclusion, these results demonstrate bZIP InDel mutations to be the major independent determinant of outcome in CEBPA-mutant AML, thereby refining current classifications according to WHO (including all dmCEBPA and smCEBPA bZIP) as well as ELN2022 and ICC recommendations (including CEBPA bZIP ms). (Figure presented.)

Published
2024

29. Clinical outcomes in patients with relapsed/refractory FLT3-mutated acute myeloid leukemia treated with gilteritinib who received prior midostaurin or sorafenib

Author

Perl, Alexander E., Hosono, Naoko, Montesinos, Pau, Podoltsev, Nikolai, Martinelli, Giovanni, Panoskaltsis, Nicki, Recher, Christian, Smith, Catherine C., Levis, Mark J., Strickland, Stephen, Röllig, Christoph, Groß-Langenhoff, Marco, Chou, Wen-Chien, Lee, Je-Hwan, Yokoyama, Hisayuki, Hasabou, Nahla, Lu, Qiaoyang, Tiu, Ramon V., and Altman, Jessica K.

Published
2022
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31. Genome-wide association study identifies susceptibility loci for acute myeloid leukemia

Author

Lin, Wei-Yu, Fordham, Sarah E., Hungate, Eric, Sunter, Nicola J., Elstob, Claire, Xu, Yaobo, Park, Catherine, Quante, Anne, Strauch, Konstantin, Gieger, Christian, Skol, Andrew, Rahman, Thahira, Sucheston-Campbell, Lara, Wang, Junke, Hahn, Theresa, Clay-Gilmour, Alyssa I., Jones, Gail L., Marr, Helen J., Jackson, Graham H., Menne, Tobias, Collin, Mathew, Ivey, Adam, Hills, Robert K., Burnett, Alan K., Russell, Nigel H., Fitzgibbon, Jude, Larson, Richard A., Le Beau, Michelle M., Stock, Wendy, Heidenreich, Olaf, Alharbi, Abrar, Allsup, David J., Houlston, Richard S., Norden, Jean, Dickinson, Anne M., Douglas, Elisabeth, Lendrem, Clare, Daly, Ann K., Palm, Louise, Piechocki, Kim, Jeffries, Sally, Bornhäuser, Martin, Röllig, Christoph, Altmann, Heidi, Ruhnke, Leo, Kunadt, Desiree, Wagenführ, Lisa, Cordell, Heather J., Darlay, Rebecca, Andersen, Mette K., Fontana, Maria C., Martinelli, Giovanni, Marconi, Giovanni, Sanz, Miguel A., Cervera, José, Gómez-Seguí, Inés, Cluzeau, Thomas, Moreilhon, Chimène, Raynaud, Sophie, Sill, Heinz, Voso, Maria Teresa, Lo-Coco, Francesco, Dombret, Hervé, Cheok, Meyling, Preudhomme, Claude, Gale, Rosemary E., Linch, David, Gaal-Wesinger, Julia, Masszi, Andras, Nowak, Daniel, Hofmann, Wolf-Karsten, Gilkes, Amanda, Porkka, Kimmo, Milosevic Feenstra, Jelena D., Kralovics, Robert, Grimwade, David, Meggendorfer, Manja, Haferlach, Torsten, Krizsán, Szilvia, Bödör, Csaba, Stölzel, Friedrich, Onel, Kenan, and Allan, James M.

Published
2021
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32. Rationale and design of the 2 by 2 factorial design GnG-trial: a randomized phase-III study to compare two schedules of gemtuzumab ozogamicin as adjunct to intensive induction therapy and to compare double-blinded intensive postremission therapy with or without glasdegib in older patients with newly diagnosed AML

Author

Jaramillo, Sonia, Krisam, Johannes, Le Cornet, Lucian, Kratzmann, Markus, Baumann, Lukas, Sauer, Tim, Crysandt, Martina, Rank, Andreas, Behringer, Dirk, Teichmann, Lino, Görner, Martin, Trappe, Ralf-Ulrich, Röllig, Christoph, Krause, Stefan, Hanoun, Maher, Hopfer, Olaf, Held, Gerhard, Buske, Sebastian, Fransecky, Lars, Kayser, Sabine, Schliemann, Christoph, Schaefer-Eckart, Kerstin, Al-Fareh, Yousef, Schubert, Jörg, Geer, Thomas, Kaufmann, Martin, Brecht, Arne, Niemann, Dirk, Kieser, Meinhard, Bornhäuser, Martin, Platzbecker, Uwe, Serve, Hubert, Baldus, Claudia D., Müller-Tidow, Carsten, and Schlenk, Richard F.

Published
2021
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33. Real-world experience of CPX-351 as first-line treatment for patients with acute myeloid leukemia

Author

Rautenberg, Christina, Stölzel, Friedrich, Röllig, Christoph, Stelljes, Matthias, Gaidzik, Verena, Lauseker, Michael, Kriege, Oliver, Verbeek, Mareike, Unglaub, Julia Marie, Thol, Felicitas, Krause, Stefan W., Hänel, Mathias, Neuerburg, Charlotte, Vucinic, Vladan, Jehn, Christian-Friedrich, Severmann, Julia, Wass, Maxi, Fransecky, Lars, Chemnitz, Jens, Holtick, Udo, Schäfer-Eckart, Kerstin, Schröder, Josephine, Kraus, Sabrina, Krüger, William, Kaiser, Ulrich, Scholl, Sebastian, Koch, Kathrin, Henning, Lea, Kobbe, Guido, Haas, Rainer, Alakel, Nael, Röhnert, Maximilian-Alexander, Sockel, Katja, Hanoun, Maher, Platzbecker, Uwe, Holderried, Tobias A. W., Morgner, Anke, Heuser, Michael, Sauer, Tim, Götze, Katharina S., Wagner-Drouet, Eva, Döhner, Konstanze, Döhner, Hartmut, Schliemann, Christoph, Schetelig, Johannes, Bornhäuser, Martin, Germing, Ulrich, Schroeder, Thomas, and Middeke, Jan Moritz

Published
2021
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35. Germline variant GFI1-36N affects DNA repair and sensitizes AML cells to DNA damage and repair therapy

Author

Frank, Daria, primary, Patnana, Pradeep Kumar, additional, Vorwerk, Jan, additional, Mao, Lianghao, additional, Mokada Gopal, Lavanya, additional, Jung, Noelle, additional, Hennig, Thorben, additional, Ruhnke, Leo, additional, Frenz, Joris Maximillian, additional, Kuppusamy, Maithreyan, additional, Autry, Robert J, additional, Wei, Lanying, additional, Sun, Kaiyan, additional, Ahmed, Helal, additional, Künstner, Axel, additional, Busch, Hauke, additional, Müller, Heiko, additional, Hutter, Stephan, additional, Hoermann, Gregor, additional, Liu, Longlong, additional, Xie, Xiaoqing, additional, Al-Matary, Yahya, additional, Nimmagadda, Subbaiah Chary, additional, Charles Cano, Fiorella, additional, Heuser, Michael, additional, Thol, Felicitas R., additional, Göhring, Gudrun, additional, Steinemann, Doris, additional, Thomale, Jürgen, additional, Leitner, Theo, additional, Fischer, Anja, additional, Rad, Roland, additional, Röllig, Christoph, additional, Altmann, Heidi, additional, Kunadt, Desiree, additional, Berdel, Wolfgang E., additional, Hüve, Jana, additional, Neumann, Felix, additional, Klingauf, Jürgen, additional, Calderon, Virginie, additional, Opalka, Bertram, additional, Dührsen, Ulrich, additional, Rosenbauer, Frank, additional, Dugas, Martin, additional, Varghese, Julian, additional, Reinhardt, H. Christian H., additional, von Bubnoff, Nikolas, additional, Möröy, Tarik, additional, Lenz, Georg, additional, Batcha, Aarif M.N., additional, Giorgi, Marianna, additional, Selvam, Murugan, additional, Wang, Eunice S., additional, McWeeney, Shannon K., additional, Tyner, Jeffrey W., additional, Stölzel, Friedrich, additional, Mann, Matthias, additional, Jayavelu, Ashok Kumar, additional, and Khandanpour, Cyrus, additional

Published
2023
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36. PB1759: RETAINED FUNCTIONAL NORMAL AND/OR PRE-LEUKEMIC HSCS IN THE BONE MARROW ARE ASSOCIATED TO GOOD PROGNOSIS IN DNMT3AMUTNPM1MUT AML PATIENTS

Author

Donato, Elisa, primary, Correia, Nadia, additional, Andresen, Carolin, additional, Karpova, Darja, additional, Würth, Roberto, additional, Klein, Corinna, additional, Sohn, Markus, additional, Przybylla, Adriana, additional, Zeisberger, Petra, additional, Rothfelder, Kathrin, additional, Salih, Helmut, additional, Bonig, Halvard, additional, Stasik, Sebastian, additional, Röllig, Christoph, additional, Dolnik, Anna, additional, Bullinger, Lars, additional, Buchholz, Frank, additional, Thiede, Christian, additional, Hübschmann, Daniel, additional, and Trumpp, Andreas, additional

Published
2023
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37. S123: ACUTE MYELOID LEUKEMIA STEM CELL SUBTYPES DEFINED BY MUTATIONS AND DIFFERENTIATION STATE REGULATE APOPTOTIC DEPENDENCY AND CLINICAL RESPONSE TO VENETOCLAX.

Author

Waclawiczek, Alexander, primary, Leppä, Aino-Maija, additional, Renders, Simon, additional, Betz, Barbara, additional, Stumpf, Karolin, additional, Jansen, Maike, additional, Merbach, Anne Kathrin, additional, Shahswar, Rabia, additional, Röllig, Christoph, additional, Heuser, Michael, additional, Sauer, Tim, additional, Müller-Tidow, Carsten, additional, and Trumpp, Andreas, additional

Published
2023
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38. PB1887: STING AGONIST FOR THE TREATMENT OF RELAPSED/REFRACTORY ACUTE MYELOID LEUKEMIA AND HIGH-RISK MYELODYSPLASTIC SYNDROME: A FIRST-IN-CLINIC PHASE 1 STUDY OF GSK3745417

Author

Montesinos, Pau, primary, Al-Ali, Haifa Kathrin, additional, Dominguez, Juan Manuel Alonso, additional, Jentzsch, Madlen, additional, Lavrencic, Mojca, additional, Martelli, Maria Paola, additional, Röllig, Christoph, additional, Sica, Simona, additional, Riham, Iadevaia, additional, Yablonski, Kaitlin, additional, Wang, Tianli, additional, Mahmood, Zafar, additional, Koenen, Giedre, additional, Schmidt, Hank, additional, Yang, Jingsing, additional, and Yee, Karen, additional

Published
2023
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39. P528: CAN MULTIPARAMETRIC FLOW CYTOMETRY OF PERIPHERAL BLOOD CHALLENGE BONE MARROW ANALYSES FOR DETECTION OF MEASURABLE RESIDUAL DISEASE IN PATIENTS WITH ACUTE MYELOID LEUKEMIA?

Author

Schadt, Jonas, primary, Alexander Röhnert, Maximilian, additional, Röllig, Christoph, additional, F. Schlenk, Richard, additional, Schweigler, Dirk, additional, Zukunft, Sven, additional, Bornhäuser, Martin, additional, von Bonin, Malte, additional, and Oelschlägel, Uta, additional

Published
2023
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40. P556: A NEW MULTIPLEX PCR TOOL ENABLES TIMELY IDENTIFICATION OF ACTIONABLE TARGETS FOR MOLECULAR-GUIDED THERAPY IN PATIENTS WITH ACUTE MYELOID LEUKEMIA

Author

Stölzel, Friedrich, primary, Ruhnke, Leo, additional, Altmann, Heidi, additional, Röllig, Christoph, additional, Bornhauser, Martin, additional, Chavakis, Triantafyllos, additional, Nestler, Stephanie, additional, Preussler, Matthias, additional, Weber, Anke, additional, and Poitz, David, additional

Published
2023
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41. P554: RESULTS OF A RANDOMIZED PLACEBO-CONTROLLED PHASE 2 STUDY OF HYPOMETHYLATING AGENTS WITH OR WITHOUT ELTROMBOPAG IN ELDERLY AML PATIENTS (DELTA)

Author

Sockel, Katja, primary, Mütherig, Anke, additional, Crysandt, Martina, additional, Hänel, Mathias, additional, Noppeney, Richard, additional, Schaefer-Eckart, Kerstin, additional, Kaufmann, Martin, additional, Röllig, Christoph, additional, Schetelig, Johannes, additional, Zukunft, Sven, additional, Fiebig, Frank, additional, Giagounidis, Aristoteles, additional, Scholl, Sebastian, additional, Lück, Andreas, additional, Rieger, Kathrin, additional, Götze, Katharina, additional, Geer, Thomas, additional, Kiewe, Philipp, additional, Müller-Tidow, Carsten, additional, Serve, Hubert, additional, Baldus, Claudia, additional, Kaiser, Ulrich, additional, Mahlmann, Stefan, additional, Schmidt, Burkhard, additional, Parmentier, Stefani, additional, Illmer, Thomas, additional, Seggewiß-Bernhardt, Ruth, additional, Kiani, Alexander, additional, Linde, Hartmut, additional, Dürk, Heinz, additional, Kramer, Michael, additional, Kunadt, Desiree, additional, Schmidt-Brücken, Katharina, additional, Hase, Jana, additional, Helas, Susann, additional, Moritz Middeke, Jan, additional, von Bonin, Malte, additional, Oelschlaegel, Uta, additional, Ehninger, Gerhard, additional, Thiede, Christian, additional, Bornhauser, Martin, additional, and Platzbecker, Uwe, additional

Published
2023
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42. S138: VALIDATION OF THE REVISED 2022 EUROPEAN LEUKEMIANET (ELN) RISK STRATIFICATION IN ADULT PATIENTS WITH ACUTE MYELOID LEUKEMIA

Author

Bill, Marius, primary, Ruhnke, Leo, additional, Zukunft, Sven, additional, Schäfer, Silvia, additional, Eckardt, Jan-Niklas, additional, Stasik, Sebastian, additional, Hanoun, Maher, additional, Schroeder, Thomas, additional, Fransecky, Lars, additional, Steffen, Björn, additional, Krause, Stefan W., additional, Scholl, Sebastian, additional, Hochhaus, Andreas, additional, Sauer, Tim, additional, Kraus, Sabrina, additional, Schäfer-Eckart, Kerstin, additional, Kaufmann, Martin, additional, Jost, Edgar, additional, Brümmendorf, Tim H, additional, Schliemann, Christoph, additional, Mikesch, Jan-Henrik, additional, Krug, Utz, additional, Hänel, Mathias, additional, Morgner, Anke, additional, Schaich, Markus, additional, Neubauer, Andreas, additional, Repp, Roland, additional, Niemann, Dirk, additional, Seggewiss-Bernhardt, Ruth, additional, Meinhardt, Achim, additional, Kullmer, Johannes, additional, Kaiser, Ulrich, additional, Blau, Wolfgang, additional, Kiani, Alexander, additional, Grigoleit, Götz Ulrich, additional, Giagounidis, Aristoteles, additional, Wurm, Alexander, additional, Altmann, Heidi, additional, Middeke, Jan Moritz, additional, Schetelig, Johannes, additional, Müller-Tidow, Carsten, additional, Stölzel, Friedrich, additional, Baldus, Claudia, additional, Platzbecker, Uwe, additional, Serve, Hubert, additional, Bornhäuser, Martin, additional, Thiede, Christian, additional, and Röllig, Christoph, additional

Published
2023
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43. Long-Term Follow-Up and Impact of Comorbidity before Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Relapsed or Refractory Acute Myeloid Leukemia—Lessons Learned from the Prospective BRIDGE Trial

Author

Middeke, Jan Moritz, Herbst, Regina, Parmentier, Stefani, Bug, Gesine, Hänel, Mathias, Stuhler, Gernot, Schäfer-Eckart, Kerstin, Rösler, Wolf, Klein, Stefan, Bethge, Wolfgang, Bitz, Ulrich, Büttner, Bozena, Knoth, Holger, Alakel, Nael, Schaich, Markus, Morgner, Anke, Kramer, Michael, Sockel, Katja, von Bonin, Malte, Stölzel, Friedrich, Platzbecker, Uwe, Röllig, Christoph, Thiede, Christian, Ehninger, Gerhard, Bornhäuser, Martin, and Schetelig, Johannes

Published
2017
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45. Elotuzumab, lenalidomide, and dexamethasone in RRMM: final overall survival results from the phase 3 randomized ELOQUENT-2 study

Author

Dimopoulos, Meletios A., Lonial, Sagar, White, Darrell, Moreau, Philippe, Weisel, Katja, San-Miguel, Jesus, Shpilberg, Ofer, Grosicki, Sebastian, Špička, Ivan, Walter-Croneck, Adam, Magen, Hila, Mateos, Maria-Victoria, Belch, Andrew, Reece, Donna, Beksac, Meral, Spencer, Andrew, Oakervee, Heather, Orlowski, Robert Z., Taniwaki, Masafumi, Röllig, Christoph, Einsele, Hermann, Matsumoto, Morio, Wu, Ka Lung, Anderson, Kenneth C., Jou, Ying-Ming, Ganetsky, Alex, Singhal, Anil K., and Richardson, Paul G.

Published
2020
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47. Abstract CT124: A first-in-clinic phase 1 study of GSK3745417 STING agonist in relapsed/refractory acute myeloid leukemia and high-risk myelodysplastic syndrome

Author

Montesinos, Pau, primary, Al-Ali, Haifa, additional, Alonso-Dominguez, Juan M., additional, Jentzsch, Madlen, additional, Jongen-Lavrencic, Mojca, additional, Martelli, Maria Paola, additional, Röllig, Christoph, additional, Sica, Simona, additional, Iadevaia, Riham, additional, Yablonski, Kaitlin, additional, Wang, Tianli, additional, Mahmood, Zafar, additional, Koenen, Giedre, additional, Schmidt, Hank, additional, Yang, Jingsong, additional, and Yee, Karen, additional

Published
2023
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48. Biallelic TET2 mutations confer sensitivity to 5'-azacitidine in acute myeloid leukemia.

Author

Stölzel, Friedrich, Stölzel, Friedrich, Fordham, Sarah E, Nandana, Devi, Lin, Wei-Yu, Blair, Helen, Elstob, Claire, Bell, Hayden L, Mohr, Brigitte, Ruhnke, Leo, Kunadt, Desiree, Dill, Claudia, Allsop, Daniel, Piddock, Rachel, Soura, Emmanouela-Niki, Park, Catherine, Fadly, Mohd, Rahman, Thahira, Alharbi, Abrar, Wobus, Manja, Altmann, Heidi, Röllig, Christoph, Wagenführ, Lisa, Jones, Gail L, Menne, Tobias, Jackson, Graham H, Marr, Helen J, Fitzgibbon, Jude, Onel, Kenan, Meggendorfer, Manja, Robinson, Amber, Bziuk, Zuzanna, Bowes, Emily, Heidenreich, Olaf, Haferlach, Torsten, Villar, Sara, Ariceta, Beñat, Diaz, Rosa Ayala, Altschuler, Steven J, Wu, Lani F, Prosper, Felipe, Montesinos, Pau, Martinez-Lopez, Joaquin, Bornhäuser, Martin, Allan, James M, Stölzel, Friedrich, Stölzel, Friedrich, Fordham, Sarah E, Nandana, Devi, Lin, Wei-Yu, Blair, Helen, Elstob, Claire, Bell, Hayden L, Mohr, Brigitte, Ruhnke, Leo, Kunadt, Desiree, Dill, Claudia, Allsop, Daniel, Piddock, Rachel, Soura, Emmanouela-Niki, Park, Catherine, Fadly, Mohd, Rahman, Thahira, Alharbi, Abrar, Wobus, Manja, Altmann, Heidi, Röllig, Christoph, Wagenführ, Lisa, Jones, Gail L, Menne, Tobias, Jackson, Graham H, Marr, Helen J, Fitzgibbon, Jude, Onel, Kenan, Meggendorfer, Manja, Robinson, Amber, Bziuk, Zuzanna, Bowes, Emily, Heidenreich, Olaf, Haferlach, Torsten, Villar, Sara, Ariceta, Beñat, Diaz, Rosa Ayala, Altschuler, Steven J, Wu, Lani F, Prosper, Felipe, Montesinos, Pau, Martinez-Lopez, Joaquin, Bornhäuser, Martin, and Allan, James M

Abstract

Precision medicine can significantly improve outcomes for patients with cancer, but implementation requires comprehensive characterization of tumor cells to identify therapeutically exploitable vulnerabilities. Here, we describe somatic biallelic TET2 mutations in an elderly patient with acute myeloid leukemia (AML) that was chemoresistant to anthracycline and cytarabine but acutely sensitive to 5'-azacitidine (5'-Aza) hypomethylating monotherapy, resulting in long-term morphological remission. Given the role of TET2 as a regulator of genomic methylation, we hypothesized that mutant TET2 allele dosage affects response to 5'-Aza. Using an isogenic cell model system and an orthotopic mouse xenograft, we demonstrate that biallelic TET2 mutations confer sensitivity to 5'-Aza compared with cells with monoallelic mutations. Our data argue in favor of using hypomethylating agents for chemoresistant disease or as first-line therapy in patients with biallelic TET2-mutated AML and demonstrate the importance of considering mutant allele dosage in the implementation of precision medicine for patients with cancer.

Published
2023

49. Ex vivo drug response profiling for response and outcome prediction in hematologic malignancies: the prospective non-interventional SMARTrial

Author

Liebers, Nora, Bruch, Peter-Martin; https://orcid.org/0000-0002-9992-3109, Terzer, Tobias, Hernandez-Hernandez, Miguel, Paramasivam, Nagarajan, Fitzgerald, Donnacha, Altmann, Heidi, Roider, Tobias; https://orcid.org/0000-0002-6973-3531, Kolb, Carolin, Knoll, Mareike, Lenze, Angela, Platzbecker, Uwe, Röllig, Christoph, Baldus, Claudia; https://orcid.org/0000-0002-0748-834X, Serve, Hubert; https://orcid.org/0000-0001-8472-5516, Bornhäuser, Martin, Hübschmann, Daniel; https://orcid.org/0000-0002-6041-7049, Müller-Tidow, Carsten; https://orcid.org/0000-0002-7166-5232, Stölzel, Friedrich; https://orcid.org/0000-0003-0653-5332, Huber, Wolfgang; https://orcid.org/0000-0002-0474-2218, Benner, Axel, Zenz, Thorsten; https://orcid.org/0000-0001-7890-9845, Lu, Junyan, Dietrich, Sascha; https://orcid.org/0000-0002-0648-1832, Liebers, Nora, Bruch, Peter-Martin; https://orcid.org/0000-0002-9992-3109, Terzer, Tobias, Hernandez-Hernandez, Miguel, Paramasivam, Nagarajan, Fitzgerald, Donnacha, Altmann, Heidi, Roider, Tobias; https://orcid.org/0000-0002-6973-3531, Kolb, Carolin, Knoll, Mareike, Lenze, Angela, Platzbecker, Uwe, Röllig, Christoph, Baldus, Claudia; https://orcid.org/0000-0002-0748-834X, Serve, Hubert; https://orcid.org/0000-0001-8472-5516, Bornhäuser, Martin, Hübschmann, Daniel; https://orcid.org/0000-0002-6041-7049, Müller-Tidow, Carsten; https://orcid.org/0000-0002-7166-5232, Stölzel, Friedrich; https://orcid.org/0000-0003-0653-5332, Huber, Wolfgang; https://orcid.org/0000-0002-0474-2218, Benner, Axel, Zenz, Thorsten; https://orcid.org/0000-0001-7890-9845, Lu, Junyan, and Dietrich, Sascha; https://orcid.org/0000-0002-0648-1832

Abstract

Ex vivo drug response profiling is a powerful tool to study genotype-drug response associations and is being explored as a tool set for precision medicine in cancer. Here we conducted a prospective non-interventional trial to investigate feasibility of ex vivo drug response profiling for treatment guidance in hematologic malignancies (SMARTrial, NCT03488641 ). The primary endpoint to provide drug response profiling reports within 7 d was met in 91% of all study participants (N = 80). Secondary endpoint analysis revealed that ex vivo resistance to chemotherapeutic drugs predicted chemotherapy treatment failure in vivo. We confirmed the predictive value of ex vivo response to chemotherapy in a validation cohort of 95 individuals with acute myeloid leukemia treated with daunorubicin and cytarabine. Ex vivo drug response profiles improved ELN-22 risk stratification in individuals with adverse risk. We conclude that ex vivo drug response profiling is clinically feasible and has the potential to predict chemotherapy response in individuals with hematologic malignancies beyond clinically established genetic markers.

Published
2023

50. Allogeneic Hematopoietic Cell Transplantation in Multiple Myeloma: Focus on Longitudinal Assessment of Donor Chimerism, Extramedullary Disease, and High-Risk Cytogenetic Features

Author

Rasche, Leo, Röllig, Christoph, Stuhler, Gernot, Danhof, Sophia, Mielke, Stephan, Grigoleit, Goetz Ulrich, Dissen, Lea, Schemmel, Lea, Middeke, Jan Moritz, Rücker, Viktoria, Schreder, Martin, Schetelig, Johannes, Bornhäuser, Martin, Einsele, Hermann, Thiede, Christian, and Knop, Stefan

Published
2016
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