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1. Diagnostic evaluation of paediatric autoimmune lymphoproliferative immunodeficiencies (ALPID): a prospective cohort study

Author

Abinun, Mario, Abrahamsen, Tore G., Albert, Michael H., Almalky, Mohamed, Altaf, Sadaf, Babayeva, Royala, Bakhtiar, Shahrzad, Baris, Safa, Baumann, Ulrich, Becker, Martina, Beier, Rita, Berger, Thomas, Biebl, Ariane, Bielack, Stefan S., Biskup, Saskia, Bode, Sebastian FN, Borchers, Regine, Boztug, Kaan, Brockmann, Knut, Bruwier, Annelyse, Buchholz, Bernd, Caballero-Oteyza, Andres, Cant, Andrew J., Castro, Carla N., Classen, Carl F., Claviez, Alexander, Crazzolara, Roman, Cuntz, Franziska, Dąbrowska-Leonik, Nel, Derichs, Ute, Dückers, Gregor, Eberl, Wolfgang, Ebetsberger-Dachs, Georg, Erlacher, Miriam, Fabre, Alexandre, Faletti, Laura, Farmand, Susan, Figueiredo, Antonio E., Fischer, Marco, Flaadt, Tim, Full, Hermann, Gambineri, Eleonora, Girschick, Hermann, Goldacker, Sigune, Grimbacher, Bodo, Groß, Miriam, Gruhn, Bernd, Haberfellner, Florian, Hague, Rosie, Hauch, Holger, Hauck, Fabian, Heine, Sabine, Huisman, Elise J., Jakovljevic, Gordana, James, Beki, Janda, Ales, Jones, Neil, Kaiser-Labusch, Petra, Kentouche, Karim, Knight, Julian C., Knirsch, Stephanie, Kontny, Udo, Körholz, Julia, Krenn, Thomas, Kuehnle, Ingrid, Kühne, Thomas, Lee-Dimroth, Jae-Yun, Lehmann, Hartwig, Leipold, Alfred, Meinhardt, Andrea, Minkov, Milen, Mönkemöller, Kirsten, Morbach, Henner, Mücke, Urs, Nathrath, Michaela, Naumann-Bartsch, Nora, Neth, Olaf, Niemeyer, Charlotte M., Olbrich, Peter, Ostró, Róbert, Owens, Stephen, Pac, Malgorzata, Pachlopnik Schmid, Jana, Page, Matthew J.T., Pekrun, Arnulf, Prader, Seraina, Proietti, Michele, Rajacic, Nada, Rothoeft, Tobias, Ryan, Clodagh, Salou, Sarah, Salzer, Elisabeth, Savic, Sinisa, Schilling, Freimut H., Schönberger, Stefan, Schuetz, Catharina, Schuez-Havupalo, Linnea, Schulte, Björn, Schulz, Ansgar, Schuster, Volker, Seidel, Markus G., Siepermann, Kathrin, Skomska-Pawliszak, Malgorzata, Smisek, Petr, Soomann, Maarja, Stiefel, Martina, Storck, Simone, Strahm, Brigitte, Streiter, Monika, Teltschik, Heiko-Manuel, Thalhammer, Julian, Tippelt, Stephan, Toskov, Vasil, Trück, Johannes, Vieth, Simon, von Bismarck, Philipp, Wegehaupt, Oliver, Wiesel, Thomas, Wittkowski, Helmut, Yalcin Gungoren, Ezgi, Hägele, Pauline, Staus, Paulina, Scheible, Raphael, Uhlmann, Annette, Heeg, Maximilian, Klemann, Christian, Maccari, Maria Elena, Ritterbusch, Henrike, Armstrong, Martin, Cutcutache, Ioana, Elliott, Katherine S, von Bernuth, Horst, Leahy, Timothy Ronan, Leyh, Jörg, Holzinger, Dirk, Lehmberg, Kai, Svec, Peter, Masjosthusmann, Katja, Hambleton, Sophie, Jakob, Marcus, Sparber-Sauer, Monika, Kager, Leo, Puzik, Alexander, Wolkewitz, Martin, Lorenz, Myriam Ricarda, Schwarz, Klaus, Speckmann, Carsten, Rensing-Ehl, Anne, and Ehl, Stephan

Published
2024
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2. A distinct CD38+CD45RA+ population of CD4+, CD8+, and double-negative T cells is controlled by FAS.

Author

Maccari, Maria, Fuchs, Sebastian, Kury, Patrick, Andrieux, Geoffroy, Völkl, Simon, Bengsch, Bertram, Lorenz, Myriam, Heeg, Maximilian, Rohr, Jan, Jägle, Sabine, Castro, Carla, Groß, Miriam, Warthorst, Ursula, König, Christoph, Fuchs, Ilka, Speckmann, Carsten, Thalhammer, Julian, Kapp, Friedrich, Seidel, Markus, Dückers, Gregor, Schönberger, Stefan, Schütz, Catharina, Führer, Marita, Kobbe, Robin, Holzinger, Dirk, Klemann, Christian, Smisek, Petr, Owens, Stephen, Horneff, Gerd, Kolb, Reinhard, Naumann-Bartsch, Nora, Miano, Maurizio, Staniek, Julian, Rizzi, Marta, Kalina, Tomas, Schneider, Pascal, Erxleben, Anika, Backofen, Rolf, Ekici, Arif, Niemeyer, Charlotte, Warnatz, Klaus, Grimbacher, Bodo, Eibel, Hermann, Mackensen, Andreas, Frei, Andreas, Schwarz, Klaus, Boerries, Melanie, Ehl, Stephan, and Rensing-Ehl, Anne

Subjects
ADP-ribosyl Cyclase 1, Adolescent, Adult, Aged, CD4-Positive T-Lymphocytes, CD8-Positive T-Lymphocytes, Child, Child, Preschool, Female, Humans, Infant, Infant, Newborn, Leukocyte Common Antigens, Lymphocyte Activation, Lymphoproliferative Disorders, Male, Middle Aged, Signal Transduction, Young Adult, fas Receptor
Abstract

The identification and characterization of rare immune cell populations in humans can be facilitated by their growth advantage in the context of specific genetic diseases. Here, we use autoimmune lymphoproliferative syndrome to identify a population of FAS-controlled TCRαβ+ T cells. They include CD4+, CD8+, and double-negative T cells and can be defined by a CD38+CD45RA+T-BET- expression pattern. These unconventional T cells are present in healthy individuals, are generated before birth, are enriched in lymphoid tissue, and do not expand during acute viral infection. They are characterized by a unique molecular signature that is unambiguously different from other known T cell differentiation subsets and independent of CD4 or CD8 expression. Functionally, FAS-controlled T cells represent highly proliferative, noncytotoxic T cells with an IL-10 cytokine bias. Mechanistically, regulation of this physiological population is mediated by FAS and CTLA4 signaling, and its survival is enhanced by mTOR and STAT3 signals. Genetic alterations in these pathways result in expansion of FAS-controlled T cells, which can cause significant lymphoproliferative disease.

Published
2021

4. Diagnostic evaluation of paediatric autoimmune lymphoproliferative immunodeficiencies (ALPID): a prospective cohort study

Author

Hägele, Pauline, primary, Staus, Paulina, additional, Scheible, Raphael, additional, Uhlmann, Annette, additional, Heeg, Maximilian, additional, Klemann, Christian, additional, Maccari, Maria Elena, additional, Ritterbusch, Henrike, additional, Armstrong, Martin, additional, Cutcutache, Ioana, additional, Elliott, Katherine S, additional, von Bernuth, Horst, additional, Leahy, Timothy Ronan, additional, Leyh, Jörg, additional, Holzinger, Dirk, additional, Lehmberg, Kai, additional, Svec, Peter, additional, Masjosthusmann, Katja, additional, Hambleton, Sophie, additional, Jakob, Marcus, additional, Sparber-Sauer, Monika, additional, Kager, Leo, additional, Puzik, Alexander, additional, Wolkewitz, Martin, additional, Lorenz, Myriam Ricarda, additional, Schwarz, Klaus, additional, Speckmann, Carsten, additional, Rensing-Ehl, Anne, additional, Ehl, Stephan, additional, Abinun, Mario, additional, Abrahamsen, Tore, additional, Albert, Michael, additional, Almalky, Mohamed, additional, Altaf, Sadaf, additional, Babayeva, Royala, additional, Bakhtiar, Shahrzad, additional, Baris, Safa, additional, Baumann, Ulrich, additional, Becker, Martina, additional, Berger, Thomas, additional, Biebl, Ariane, additional, Bielack, Stefan, additional, Biskup, Saskia, additional, Bismarck, Philipp, additional, Bode, Sebastian, additional, Borchers, Regine, additional, Boztug, Carl Friedrich, additional, Brockmann, Knut, additional, Bruwier, Annelyse, additional, Buchholz, Bernd, additional, Cant, Andrew, additional, Castro, Carla N, additional, Classen, Carl, additional, Claviez, Alexander, additional, Crazzolara, Roman, additional, Cuntz, Franziska, additional, Dąbrowska-Leonik, Nel, additional, Derichs, Ute, additional, Dückers, Gregor, additional, Eberl, Wolfgang, additional, Ebetsberger-Dachs, Georg, additional, Erlacher, Miriam, additional, Fabre, Alexandre, additional, Faletti, Laura, additional, Farmand, Susan, additional, Figueiredo, Antonio, additional, Fischer, Marco, additional, Flaadt, Tim, additional, Full, Hermann, additional, Gambineri, Eleonora, additional, Girschick, Hermann, additional, Goldacker, Sigune, additional, Grimbacher, Bodo, additional, Groß, Miriam, additional, Gruhn, Andrew J, additional, Gungoren, Ezgi, additional, Haberfellner, Florian, additional, Hague, Rosie, additional, Hauch, Holger, additional, Hauck, Fabian, additional, Heine, Sabine, additional, Huisman, Elise, additional, Jakovljevic, Gordana, additional, James, Beki, additional, Janda, Ales, additional, Janda, Małgorzata, additional, Jones, Neil, additional, Kaiser-Labusch, Petra, additional, Kentouche, Karim, additional, Knight, Julian, additional, Knirsch, Stephanie, additional, Kontny, Udo, additional, Körholz, Julia, additional, Krenn, Ezgi Yalcin, additional, Kuehnle, Ingrid, additional, Kühne, Thomas, additional, Lee-Dimroth, Jae-Yun, additional, Lehmann, Hartwig, additional, Leipold, Michael H., additional, Meinhardt, Andrea, additional, Mönkemöller, Kirsten, additional, Morbach, Henner, additional, Mücke, Urs, additional, Nathrath, Michaela, additional, Naumann-Bartsch, Nora, additional, Neth, Olaf, additional, Niemeyer, Charlotte, additional, Olbrich, Peter, additional, Ostró, Róbert, additional, Owens, Stephen, additional, Pac, Malgorzata, additional, Pachlopnik Schmid, Rita, additional, Page, Markus G., additional, Pekrun, Arnulf, additional, Prader, Seraina, additional, Proietti, Michele, additional, Rajacic, Nada, additional, Rothoeft, Tobias, additional, Ryan, Freimut H., additional, Salou, Sarah, additional, Salzer, Elisabeth, additional, Savic, Sinisa, additional, Schilling, Antonio E., additional, Schmid, Jana, additional, Schönberger, Stefan, additional, Schuetz, Catharina, additional, Schuez-Havupalo, Tore G., additional, Schulte, Björn, additional, Schulz, Ansgar, additional, Schuster, Volker, additional, Seidel, Markus, additional, Siepermann, Kathrin, additional, Smisek, Petr, additional, Soomann, Maarja, additional, Stiefel, Martina, additional, Storck, Simone, additional, Strahm, Brigitte, additional, Streiter, Elise J, additional, Teltschik, Charlotte M., additional, Thalhammer, Julian, additional, Tippelt, Stephan, additional, Toskov, Vasil, additional, Trück, Johannes, additional, Vieth, Simon, additional, Wegehaupt, Oliver, additional, and Wiesel, Thomas, additional

Published
2024
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5. Four Additional Doses of PEG-L-Asparaginase During the Consolidation Phase in the AIEOP-BFM ALL 2009 Protocol Do Not Improve Outcome and Increase Toxicity in High-Risk ALL: Results of a Randomized Study.

Author

Conter, Valentino, Valsecchi, Maria Grazia, Cario, Gunnar, Zimmermann, Martin, Attarbaschi, Andishe, Stary, Jan, Niggli, Felix, Dalla Pozza, Luciano, Elitzur, Sarah, Silvestri, Daniela, Locatelli, Franco, Möricke, Anja, Engstler, Gernot, Smisek, Petr, Bodmer, Nicole, Barbaric, Draga, Izraeli, Shai, Rizzari, Carmelo, Boos, Joachim, and Buldini, Barbara

Published
2024
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6. Therapeutic Drug Monitoring of Asparaginase: Intra-individual Variability and Predictivity in Children With Acute Lymphoblastic Leukemia Treated With PEG-Asparaginase in the AIEOP-BFM Acute Lymphoblastic Leukemia 2009 Study

Author

Würthwein, Gudrun, Lanvers-Kaminsky, Claudia, Gerss, Joachim, Möricke, Anja, Zimmermann, Martin, Stary, Jan, Smisek, Petr, Attarbaschi, Andishe, Nath, Christa, Zucchetti, Massimo, Rizzari, Carmelo, Schrappe, Martin, and Boos, Joachim

Published
2020
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8. Impact of Additional Intensive L-Asparaginase Therapy during Consolidation Phase for High-Risk Acute Lymphoblastic Leukemia: Results of a Randomized Controlled Trial in the AIEOP-BFM ALL 2009 Protocol

Author

Conter, Valentino, primary, Valsecchi, Maria Grazia, additional, Cario, Gunnar, additional, Zimmermann, Martin, additional, Attarbaschi, Andishe, additional, Stary, Jan, additional, Niggli, Felix, additional, Dalla Pozza, Luciano, additional, Elitzur, Sarah, additional, Silvestri, Daniela, additional, Locatelli, Franco, additional, Moericke, Anja, additional, Engstler, Gernot, additional, Smisek, Petr, additional, Bodmer, Nicole, additional, Barbaric, Draga, additional, Izraeli, Shai, additional, Rizzari, Carmelo, additional, Boos, Joachim, additional, Buldini, Barbara, additional, Lanvers-Kaminsky, Claudia, additional, Cazzaniga, Giovanni, additional, Gruhn, Bernd, additional, Biondi, Andrea, additional, and Schrappe, Martin, additional

Published
2022
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10. Guideline for management of non-Down syndrome neonates with a myeloproliferative disease on behalf of the I-BFM AML Study Group and EWOG-MDS

Author

Bertrums, Eline J.M., Zwaan, C. Michel, Hasegawa, Daisuke, de Haas, Valerie, Reinhardt, Dirk N., Locatelli, Franco, de Moerloose, Barbara, Dworzak, Michael, Buijs, Arjan, Smisek, Petr, Kolenova, Alexandra, Pronk, Cornelis Jan, Klusmann, Jan Henning, Carboné, Ana, Ferster, Alina, Antoniou, Evangelia, Meshinchi, Soheil, Raimondi, Susana C., Niemeyer, Charlotte M., Hasle, Henrik, van den Heuvel-Eibrink, Marry M., Goemans, Bianca F., Bertrums, Eline J.M., Zwaan, C. Michel, Hasegawa, Daisuke, de Haas, Valerie, Reinhardt, Dirk N., Locatelli, Franco, de Moerloose, Barbara, Dworzak, Michael, Buijs, Arjan, Smisek, Petr, Kolenova, Alexandra, Pronk, Cornelis Jan, Klusmann, Jan Henning, Carboné, Ana, Ferster, Alina, Antoniou, Evangelia, Meshinchi, Soheil, Raimondi, Susana C., Niemeyer, Charlotte M., Hasle, Henrik, van den Heuvel-Eibrink, Marry M., and Goemans, Bianca F.

Published
2022

11. Guideline for management of non-Down syndrome neonates with a myeloproliferative disease on behalf of the I-BFM AML Study Group and EWOG-MDS

Author

Speerpunt, Genetica Sectie Genoomdiagnostiek, Cancer, Zorg en O&O, Child Health, Bertrums, Eline J M, Zwaan, C Michel, Hasegawa, Daisuke, De Haas, Valerie, Reinhardt, Dirk N, Locatelli, Franco, De Moerloose, Barbara, Dworzak, Michael, Buijs, Arjan, Smisek, Petr, Kolenova, Alexandra, Pronk, Cornelis Jan, Klusmann, Jan-Henning, Carboné, Ana, Ferster, Alina, Antoniou, Evangelia, Meshinchi, Soheil, Raimondi, Susana C, Niemeyer, Charlotte M, Hasle, Henrik, Van den Heuvel-Eibrink, Marry M, Goemans, Bianca F, Speerpunt, Genetica Sectie Genoomdiagnostiek, Cancer, Zorg en O&O, Child Health, Bertrums, Eline J M, Zwaan, C Michel, Hasegawa, Daisuke, De Haas, Valerie, Reinhardt, Dirk N, Locatelli, Franco, De Moerloose, Barbara, Dworzak, Michael, Buijs, Arjan, Smisek, Petr, Kolenova, Alexandra, Pronk, Cornelis Jan, Klusmann, Jan-Henning, Carboné, Ana, Ferster, Alina, Antoniou, Evangelia, Meshinchi, Soheil, Raimondi, Susana C, Niemeyer, Charlotte M, Hasle, Henrik, Van den Heuvel-Eibrink, Marry M, and Goemans, Bianca F

Published
2022

13. Guideline for management of non-Down syndrome neonates with a myeloproliferative disease on behalf of the I-BFM AML Study Group and EWOG-MDS

Author

Bertrums, Eline J.M., primary, Zwaan, C. Michel, additional, Hasegawa, Daisuke, additional, De Haas, Valerie, additional, Reinhardt, Dirk N., additional, Locatelli, Franco, additional, De Moerloose, Barbara, additional, Dworzak, Michael, additional, Buijs, Arjan, additional, Smisek, Petr, additional, Kolenova, Alexandra, additional, Pronk, Cornelis Jan, additional, Klusmann, Jan-Henning, additional, Carboné, Ana, additional, Ferster, Alina, additional, Antoniou, Evangelia, additional, Meshinchi, Soheil, additional, Raimondi, Susana C., additional, Niemeyer, Charlotte M., additional, Hasle, Henrik, additional, Van den Heuvel-Eibrink, Marry M., additional, and Goemans, Bianca F., additional

Published
2021
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14. A distinct CD38(+)CD45RA(+) population of CD4(+), CD8(+), and double-negative T cells is controlled by FAS

Author

Maccari, Maria Elena, Fuchs, Sebastian, Kury, Patrick, Andrieux, Geoffroy, Volkl, Simon, Bengsch, Bertram, Lorenz, Myriam Ricarda, Heeg, Maximilian, Rohr, Jan, Jagle, Sabine, Castro, Carla N., Gross, Miriam, Warthorst, Ursula, Koenig, Christoph, Fuchs, Ilka, Speckmann, Carsten, Thalhammer, Julian, Kapp, Friedrich G., Seidel, Markus G., Duckers, Gregor, Schoenberger, Stefan, Schuetz, Catharina, Fuhrer, Marita, Kobbe, Robin, Holzinger, Dirk, Klemann, Christian, Smisek, Petr, Owens, Stephen, Horneff, Gerd, Kolb, Reinhard, Naumann-Bartsch, Nora, Miano, Maurizio, Staniek, Julian, Rizzi, Marta, Kalina, Tomas, Schneider, Pascal, Erxleben, Anika, Backofen, Rolf, Ekici, Arif, Niemeyer, Charlotte M., Warnatz, Klaus, Grimbacher, Bodo, Eibel, Hermann, Mackensen, Andreas, Frei, Andreas Philipp, Schwarz, Klaus, Boerries, Melanie, Ehl, Stephan, Rensing-Ehl, Anne, Maccari, Maria Elena, Fuchs, Sebastian, Kury, Patrick, Andrieux, Geoffroy, Volkl, Simon, Bengsch, Bertram, Lorenz, Myriam Ricarda, Heeg, Maximilian, Rohr, Jan, Jagle, Sabine, Castro, Carla N., Gross, Miriam, Warthorst, Ursula, Koenig, Christoph, Fuchs, Ilka, Speckmann, Carsten, Thalhammer, Julian, Kapp, Friedrich G., Seidel, Markus G., Duckers, Gregor, Schoenberger, Stefan, Schuetz, Catharina, Fuhrer, Marita, Kobbe, Robin, Holzinger, Dirk, Klemann, Christian, Smisek, Petr, Owens, Stephen, Horneff, Gerd, Kolb, Reinhard, Naumann-Bartsch, Nora, Miano, Maurizio, Staniek, Julian, Rizzi, Marta, Kalina, Tomas, Schneider, Pascal, Erxleben, Anika, Backofen, Rolf, Ekici, Arif, Niemeyer, Charlotte M., Warnatz, Klaus, Grimbacher, Bodo, Eibel, Hermann, Mackensen, Andreas, Frei, Andreas Philipp, Schwarz, Klaus, Boerries, Melanie, Ehl, Stephan, and Rensing-Ehl, Anne

Abstract

The identification and characterization of rare immune cell populations in humans can be facilitated by their growth advantage in the context of specific genetic diseases. Here, we use autoimmune lymphoproliferative syndrome to identify a population of FAS-controlled TCR alpha beta(+) T cells. They include CD4(+), CD8(+), and double-negative T cells and can be defined by a CD38(+)CD45RA(+)T-BET- expression pattern. These unconventional T cells are present in healthy individuals, are generated before birth, are enriched in lymphoid tissue, and do not expand during acute viral infection. They are characterized by a unique molecular signature that is unambiguously different from other known T cell differentiation subsets and independent of CD4 or CD8 expression. Functionally, FAS-controlled T cells represent highly proliferative, noncytotoxic T cells with an IL-10 cytokine bias. Mechanistically, regulation of this physiological population is mediated by FAS and CTLA4 signaling, and its survival is enhanced by mTOR and STAT3 signals. Genetic alterations in these pathways result in expansion of FAS-controlled T cells, which can cause significant lymphoproliferative disease.

Published
2021

16. Pre-existing antibodies against polyethylene glycol reduce asparaginase activities on first administration of pegylated E. coli asparaginase in children with acute lymphocytic leukemia

Author

Khalil, Alaeddin, primary, Würthwein, Gudrun, additional, Golitsch, Jana, additional, Hempel, Georg, additional, Fobker, Manfred, additional, Gerss, Joachim, additional, Möricke, Anja, additional, Zimmermann, Martin, additional, Smisek, Petr, additional, Zucchetti, Massimo, additional, Nath, Christa, additional, Attarbaschi, Andishe, additional, Von Stackelberg, Arend, additional, Gökbuget, Nicola, additional, Rizzari, Carmelo, additional, Conter, Valentino, additional, Schrappe, Martin, additional, Boos, Joachim, additional, and Lanvers-Kaminsky, Claudia, additional

Published
2020
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17. A distinct CD38+CD45RA+ population of CD4+, CD8+, and double-negative T cells is controlled by FAS

Author

Maccari, Maria Elena, primary, Fuchs, Sebastian, additional, Kury, Patrick, additional, Andrieux, Geoffroy, additional, Völkl, Simon, additional, Bengsch, Bertram, additional, Lorenz, Myriam Ricarda, additional, Heeg, Maximilian, additional, Rohr, Jan, additional, Jägle, Sabine, additional, Castro, Carla N., additional, Groß, Miriam, additional, Warthorst, Ursula, additional, König, Christoph, additional, Fuchs, Ilka, additional, Speckmann, Carsten, additional, Thalhammer, Julian, additional, Kapp, Friedrich G., additional, Seidel, Markus G., additional, Dückers, Gregor, additional, Schönberger, Stefan, additional, Schütz, Catharina, additional, Führer, Marita, additional, Kobbe, Robin, additional, Holzinger, Dirk, additional, Klemann, Christian, additional, Smisek, Petr, additional, Owens, Stephen, additional, Horneff, Gerd, additional, Kolb, Reinhard, additional, Naumann-Bartsch, Nora, additional, Miano, Maurizio, additional, Staniek, Julian, additional, Rizzi, Marta, additional, Kalina, Tomas, additional, Schneider, Pascal, additional, Erxleben, Anika, additional, Backofen, Rolf, additional, Ekici, Arif, additional, Niemeyer, Charlotte M., additional, Warnatz, Klaus, additional, Grimbacher, Bodo, additional, Eibel, Hermann, additional, Mackensen, Andreas, additional, Frei, Andreas Philipp, additional, Schwarz, Klaus, additional, Boerries, Melanie, additional, Ehl, Stephan, additional, and Rensing-Ehl, Anne, additional

Published
2020
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18. A homozygous deletion in the SLC19A1 gene as a cause of folate-dependent recurrent megaloblastic anemia

Author

Svaton, Michael, primary, Skvarova Kramarzova, Karolina, primary, Kanderova, Veronika, primary, Mancikova, Andrea, primary, Smisek, Petr, primary, Jesina, Pavel, primary, Krijt, Jakub, primary, Stiburkova, Blanka, primary, Dobrovolny, Robert, primary, Sokolova, Jitka, primary, Bakardjieva-Mihaylova, Violeta, primary, Vodickova, Elena, primary, Rackova, Marketa, primary, Stuchly, Jan, primary, Kalina, Tomas, primary, Stary, Jan, primary, Trka, Jan, primary, Fronkova, Eva, primary, and Kozich, Viktor, primary

Published
2020
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20. Asparagine levels in the cerebrospinal fluid of children with acute lymphoblastic leukemia treated with pegylated-asparaginase in the induction phase of the AIEOP-BFM ALL 2009 study

Author

Rizzari, Carmelo, primary, Lanvers-Kaminsky, Claudia, additional, Valsecchi, Maria Grazia, additional, Ballerini, Andrea, additional, Matteo, Cristina, additional, Gerss, Joachim, additional, Wuerthwein, Gudrun, additional, Silvestri, Daniela, additional, Colombini, Antonella, additional, Conter, Valentino, additional, Biondi, Andrea, additional, Schrappe, Martin, additional, Moericke, Anja, additional, Zimmermann, Martin, additional, von Stackelberg, Arend, additional, Linderkamp, Christin, additional, Frühwald, Michael C., additional, Legien, Sabine, additional, Attarbaschi, Andishe, additional, Reismüller, Bettina, additional, Kasper, David, additional, Smisek, Petr, additional, Stary, Jan, additional, Vinti, Luciana, additional, Barisone, Elena, additional, Parasole, Rosanna, additional, Micalizzi, Concetta, additional, Zucchetti, Massimo, additional, and Boos, Joachim, additional

Published
2019
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21. Population Pharmacokinetics of PEGylated Asparaginase in Children with Acute Lymphoblastic Leukemia: Treatment Phase Dependency and Predictivity in Case of Missing Data.

Author

Würthwein, Gudrun, Lanvers-Kaminsky, Claudia, Siebel, Christian, Gerß, Joachim, Möricke, Anja, Zimmermann, Martin, Stary, Jan, Smisek, Petr, Schrappe, Martin, Rizzari, Carmelo, Zucchetti, Massimo, Hempel, Georg, Wicha, Sebastian G., and Boos, Joachim

Abstract

Background and Objectives: The pharmacokinetics of polyethylene glycol-conjugated asparaginase (PEG-ASNase) are characterized by an increase in elimination over time, a marked increase in ASNase activity levels from induction to reinduction, and high inter- and intraindividual variability. A population pharmacokinetic (PopPK) model is required to estimate individual dose intensity, despite gaps in monitoring compliance. Methods: In the AIEOP-BFM ALL 2009 trial, two PEG-ASNase administrations (2500 U/m2 intravenously) during induction (14-day interval) and one administration during reinduction were administered in children with acute lymphoblastic leukemia. ASNase activity levels were monitored weekly. A PopPK model was used for covariate modeling and external validation. The predictivity of the model in case of missing data was tested for observations, as well as for the derived parameters of the area under the concentration time curve (AUC0-∞) and time above different thresholds. Results: Compared to the first administration in induction (1374 patients, 6069 samples), the initial clearance and volume of distribution decreased by 11.0% and 15.9%, respectively, during the second administration during induction and by 41.2% and 28.4% during reinduction. Furthermore, the initial clearance linearly increased for children aged > 8 years and was 7.1% lower for females. The model was successfully externally validated (1253 patients, 5523 samples). In case of missing data, > 52% of the predictions for observations and > 82% for derived parameters were within ± 20% of the nominal value. Conclusion: A PopPK model that describes the complex pharmacokinetics of PEG-ASNase was successfully externally validated. AUC0−∞ or time above different thresholds, which are parameters describing dose intensity, can be estimated with high predictivity, despite missing data. (www.clinicaltrials.gov, NCT01117441, first submitted date: May 3, 2010). [ABSTRACT FROM AUTHOR]

Published
2021
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22. Folate-Dependent Normocytic Anemia Caused By a Hypomorphic Mutation in SLC19A1 gene

Author

Svaton, Michael, primary, Skvarova Kramarzova, Karolina, additional, Kanderova, Veronika, additional, Smisek, Petr, additional, Jesina, Pavel, additional, Krijt, Jakub, additional, Stiburkova, Blanka, additional, Mancikova, Andrea, additional, Dobrovolny, Robert, additional, Bakardjieva-Mihaylova, Violeta, additional, Vodickova, Elena, additional, Stuchly, Jan, additional, Kalina, Tomas, additional, Stary, Jan, additional, Trka, Jan, additional, Fronkova, Eva, additional, and Kozich, Viktor, additional

Published
2018
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23. The Prognostic Impact of Cytogenetics and Karyotype Changes in Pediatric Patients with Relapsed Acute Myeloid Leukemia: A Retrospective Cohort Study within the Relapsed AML 2001/01 Study

Author

Klein, Kim, primary, Zimmermann, Martin, additional, Beverloo, Berna, additional, von Neuhoff, Christine, additional, De Haas, Valerie, additional, van Weelderen, Romy, additional, Raimondi, Susana C., additional, Cloos, Jacqueline, additional, Abrahamsson, Jonas, additional, Bertrand, Yves, additional, Dworzak, Michael, additional, Elitzur, Sarah, additional, Fynn, Alcira, additional, Gibson, Brenda, additional, Ha, Shau Yin, additional, Harrison, Christine, additional, Hasle, Henrik, additional, Leverger, Guy, additional, Maschan, Alexey, additional, Razzouk, Bassem I., additional, Reinhardt, Dirk, additional, Rizzari, Carmelo, additional, Smisek, Petr, additional, Creutzig, Ursula, additional, and Kaspers, Gertjan J.L., additional

Published
2016
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26. Improved Outcome in Pediatric Relapsed Acute Myeloid Leukemia: Results of a Randomized Trial on Liposomal Daunorubicin by the International BFM Study Group

Author

Kaspers, Gertjan J.L., primary, Zimmermann, Martin, additional, Reinhardt, Dirk, additional, Gibson, Brenda E.S., additional, Tamminga, Rienk Y.J., additional, Aleinikova, Olga, additional, Armendariz, Hortensia, additional, Dworzak, Michael, additional, Ha, Shau-Yin, additional, Hasle, Henrik, additional, Hovi, Liisa, additional, Maschan, Alexei, additional, Bertrand, Yves, additional, Leverger, Guy G., additional, Razzouk, Bassem I., additional, Rizzari, Carmelo, additional, Smisek, Petr, additional, Smith, Owen, additional, Stark, Batia, additional, and Creutzig, Ursula, additional

Published
2013
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28. Central Nervous System (CNS) Involvement In Pediatric Relapsed Acute Myeloid Leukemia: Results and Lessons From Study Relapsed AML 2001/01

Author

Kaspers, Gertjan, primary, Zimmermann, Martin, additional, Reinhardt, Dirk, additional, Gibson, Brenda, additional, Tamminga, Rienk, additional, Aleinikova, Olga, additional, Armendariz, Hortensia, additional, Dworzak, Michael, additional, Ha, S., additional, Hovi, Lisa, additional, Maschan, Alexei, additional, Baruchel, Andre, additional, Bertrand, Yves, additional, Razzouk, Bassem, additional, Rizzari, Carmelo, additional, Smisek, Petr, additional, Smith, Owen, additional, Stark, Batia, additional, and Creutzig, Ursula, additional

Published
2010
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29. B Precursor ALL Subset with Aberrant CD2 Expression and a Specific Predisposition to Early Monocytic Transdifferentiation

Author

Mejstrikova, Ester, primary, Slamova, Lucie, additional, Fronkova, Eva, additional, Zuna, Jan, additional, Bourquin, Jean-Pierre, additional, Vodickova, Elena, additional, Muzikova, Katerina, additional, Zemanova, Zuzana, additional, Pelkova, Vendula, additional, Van Delft, Frederik, additional, Kalina, Tomas, additional, Fiser, Karel, additional, Smisek, Petr, additional, Trka, Jan, additional, Stary, Jan, additional, and Hrusak, Ondrej, additional

Published
2010
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30. Acquisition of Isochromosome 7 Is a Late Change in the Pathogenesis of Hepatosplenic Lymphoma, Documented On a Case of Adolescent Girl 5 Years After Renal Transplant with Preceding TCR Gamma-Delta Positive LGL Leukemia.

Author

Mejstrikova, Ester, primary, Kabickova, Edita, additional, Sumerauer, David, additional, Seeman, Tomas, additional, Fronkova, Eva, additional, Zemanova, Zuzana, additional, Lizcova, Libuse, additional, Sedláček, Petr, additional, Smisek, Petr, additional, Muzikova, Katerina, additional, Pospisilova, Klara, additional, Janda, Ales, additional, Kodet, Roman, additional, Capkova, Linda, additional, Stary, Jan, additional, and Hrusak, Ondrej, additional

Published
2009
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31. Composition of Cellular Subsets by Flow Cytometry Identifies Differences Between MDS Subtypes and Aplastic Anemia but No Differences Are Identified Between Cases with and without Monosomy 7.

Author

Mejstrikova, Ester, primary, Pelkova, Vendula, additional, Reiterová, Michaela, additional, Sukova, Martina, additional, Zemanova, Zuzana, additional, Vodickova, Elena, additional, Campr, Vit, additional, Michalova, Kyra, additional, Jarosova, Marie, additional, Formankova, Renata, additional, Smisek, Petr, additional, Sedlacek, Petr, additional, Pospisilova, Dagmar, additional, Mihal, Vladimir, additional, Hak, Jiri, additional, Jabali, Yahia, additional, Sterba, Jaroslav, additional, Blatny, Jan, additional, Zapletal, Ondrej, additional, Cerna, Zdenka, additional, Votava, Tomas, additional, Prochazkova, Daniela, additional, Trka, Jan, additional, Stary, Jan, additional, and Hrusak, Ondrej, additional

Published
2009
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33. Composition of Cellular Subsets by Flow Cytometry Identifies Differences Between MDS Subtypes and Aplastic Anemia but No Differences Are Identified Between Cases with and without Monosomy 7

Author

Mejstrikova, Ester, Pelkova, Vendula, Reiterova, Michaela, Sukova, Martina, Zemanova, Zuzana, Vodickova, Elena, Campr, Vit, Kyra Michalova, Jarosova, Marie, Formankova, Renata, Smisek, Petr, Sedlacek, Petr, Pospisilova, Dagmar, Mihal, Vladimir, Hak, Jiri, Jabali, Yahia, Sterba, Jaroslav, Blatny, Jan, Zapletal, Ondrej, Cerna, Zdenka, Votava, Tomas, Prochazkova, Daniela, Trka, Jan, Stary, Jan, and Hrusak, Ondrej

34. Guideline for management of non-Down syndrome neonates with a myeloproliferative disease on behalf of the I-BFM AML Study Group and EWOG-MDS.

Author

Bertrums EJM, Zwaan CM, Hasegawa D, De Haas V, Reinhardt DN, Locatelli F, De Moerloose B, Dworzak M, Buijs A, Smisek P, Kolenova A, Pronk CJ, Klusmann JH, Carboné A, Ferster A, Antoniou E, Meshinchi S, Raimondi SC, Niemeyer CM, Hasle H, Van den Heuvel-Eibrink MM, and Goemans BF

Subjects
Humans, Infant, Newborn, Leukemia, Myeloid, Acute complications, Leukemia, Myeloid, Acute diagnosis, Leukemia, Myeloid, Acute therapy, Myelodysplastic Syndromes, Myeloproliferative Disorders complications, Myeloproliferative Disorders diagnosis, Myeloproliferative Disorders drug therapy
Published
2022
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35. Pre-existing antibodies against polyethylene glycol reduce asparaginase activities on first administration of pegylated E. coli asparaginase in children with acute lymphocytic leukemia.

Author

Khalil A, Würthwein G, Golitsch J, Hempel G, Fobker M, Gerss J, Möricke A, Zimmermann M, Smisek P, Zucchetti M, Nath C, Attarbaschi A, Von Stackelberg A, Gökbuget N, Rizzari C, Conter V, Schrappe M, Boos J, and Lanvers-Kaminsky C

Subjects
Adult, Antibodies, Asparaginase therapeutic use, Child, Escherichia coli, Humans, Infant, Polyethylene Glycols therapeutic use, Antineoplastic Agents adverse effects, Precursor Cell Lymphoblastic Leukemia-Lymphoma drug therapy
Abstract

Antibodies against polyethylene glycol (PEG) in healthy subjects raise concerns about the efficacy of pegylated drugs. We evaluated the prevalence of antibodies against PEG among patients with acute lymphoblastic leukemia (ALL) prior to and/or immediately after their first dose of pegylated E.coli asparaginase (PEG-ASNase). Serum samples of 701 children, 673 with primary ALL, 28 with relapsed ALL, and 188 adults with primary ALL were analyzed for anti-PEG IgG and IgM. Measurements in 58 healthy infants served as reference to define cut-points for antibody-positive and -negative samples. Anti-PEG antibodies were detected in ALL patients prior the first PEG-ASNase with a prevalence of 13.9% (anti-PEG IgG) and 29.1% (anti-PEG IgM). After administration of PEG-ASNase the prevalence of anti-PEG antibodies decreased to 4.2% for anti-PEG IgG and to 4.5% for anti-PEG IgM. Pre-existing anti-PEG antibodies did not inhibit PEG-ASNase activity but significantly reduced PEGASNase activity levels in a concentration dependent manner. Although pre-existing anti-PEG antibodies did not boost, pre-existing anti-PEG IgG were significantly associated with firstexposure hypersensitivity reactions (CTCAE grade 2) (p.

Published
2022
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36. The prognostic significance of early treatment response in pediatric relapsed acute myeloid leukemia: results of the international study Relapsed AML 2001/01.

Author

Creutzig U, Zimmermann M, Dworzak MN, Gibson B, Tamminga R, Abrahamsson J, Ha SY, Hasle H, Maschan A, Bertrand Y, Leverger G, von Neuhoff C, Razzouk B, Rizzari C, Smisek P, Smith OP, Stark B, Reinhardt D, and Kaspers GL

Subjects
Adolescent, Antineoplastic Combined Chemotherapy Protocols, Bone Marrow Cells pathology, Child, Child, Preschool, Drug Monitoring, Female, Humans, Leukemia, Myeloid, Acute mortality, Leukemia, Myeloid, Acute pathology, Male, Prognosis, Recurrence, Remission Induction, Survival Analysis, Antibiotics, Antineoplastic therapeutic use, Bone Marrow Cells drug effects, Daunorubicin therapeutic use, Leukemia, Myeloid, Acute diagnosis, Leukemia, Myeloid, Acute drug therapy
Abstract

The prognostic significance of early response to treatment has not been reported in relapsed pediatric acute myeloid leukemia. In order to identify an early and easily applicable prognostic factor allowing subsequent treatment modifications, we assessed leukemic blast counts in the bone marrow by morphology on days 15 and 28 after first reinduction in 338 patients of the international Relapsed-AML2001/01 trial. Both day 15 and day 28 status was classified as good (≤20% leukemic blasts) in 77% of patients. The correlation between day 15 and 28 blast percentages was significant, but not strong (Spearman correlation coefficient = 0.49, P<0.001). Survival probability decreased in a stepwise fashion along with rising blast counts at day 28. Patients with bone marrow blast counts at this time-point of ≤5%, 6-10%, 11-20% and >20% had 4-year probabilities of survival of 52%±3% versus 36%±10% versus 21%±9% versus 14%±4%, respectively, P<0.0001; this trend was not seen for day 15 results. Multivariate analysis showed that early treatment response at day 28 had the strongest prognostic significance, superseding even time to relapse (< or ≥12 months). In conclusion, an early response to treatment, measured on day 28, is a strong and independent prognostic factor potentially useful for treatment stratification in pediatric relapsed acute myeloid leukemia. This study was registered with ISRCTN code: 94206677., (Copyright© Ferrata Storti Foundation.)

Published
2014
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37. Sequential decisions on FAS sequencing guided by biomarkers in patients with lymphoproliferation and autoimmune cytopenia.

Author

Rensing-Ehl A, Janda A, Lorenz MR, Gladstone BP, Fuchs I, Abinun M, Albert M, Butler K, Cant A, Cseh AM, Ebinger M, Goldacker S, Hambleton S, Hebart H, Houet L, Kentouche K, Kühnle I, Lehmberg K, Mejstrikova E, Niemeyer C, Minkov M, Neth O, Dückers G, Owens S, Rösler J, Schilling FH, Schuster V, Seidel MG, Smisek P, Sukova M, Svec P, Wiesel T, Gathmann B, Schwarz K, Vach W, Ehl S, and Speckmann C

Subjects
Adolescent, Autoimmune Diseases blood, Autoimmune Diseases diagnosis, Autoimmune Diseases genetics, Biomarkers blood, Cohort Studies, Female, Humans, Lymphoproliferative Disorders diagnosis, Male, Fas Ligand Protein blood, Fas Ligand Protein genetics, Lymphoproliferative Disorders blood, Lymphoproliferative Disorders genetics, Vitamin B 12 blood
Abstract

Clinical and genetic heterogeneity renders confirmation or exclusion of autoimmune lymphoproliferative syndrome difficult. To re-evaluate and improve the currently suggested diagnostic approach to patients with suspected FAS mutation, the most frequent cause of autoimmune lymphoproliferative syndrome, we prospectively determined 11 biomarkers in 163 patients with splenomegaly or lymphadenopathy and presumed or proven autoimmune cytopenia(s). Among 98 patients sequenced for FAS mutations in CD3(+)TCRα/β(+)CD4(-)CD8(-) "double negative" T cells, 32 had germline and six had somatic FAS mutations. The best a priori predictor of FAS mutations was the combination of vitamin B12 and soluble FAS ligand (cut-offs 1255 pg/mL and 559 pg/mL, respectively), which had a positive predictive value of 92% and a negative predictive value of 97%. We used these data to develop a web-based probability calculator for FAS mutations using the three most discriminatory biomarkers (vitamin B12, soluble FAS ligand, interleukin-10) of the 11 tested. Since more than 60% of patients with lymphoproliferation and autoimmune cytopenia(s) in our cohort did not harbor FAS mutations, 15% had somatic FAS mutations, and the predictive value of double-negative T-cell values was rather low (positive and negative predictive values of 61% and 77%, respectively), we argue that the previously suggested diagnostic algorithm based on determination of double-negative T cells and germline FAS sequencing, followed by biomarker analysis, is not efficient. We propose vitamin B12 and soluble FAS ligand assessment as the initial diagnostic step with subsequent decision on FAS sequencing supported by a probability-calculating tool.

Published
2013
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