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1. Outcomes of allogeneic haematopoietic cell transplantation for myelofibrosis in children and adolescents: the retrospective study of the EBMT Paediatric Diseases WP

Author

Wachowiak, Jacek, Galimard, Jacques-Emmanuel, Dalissier, Arnaud, Rihani, Rawad, AlSaedi, Hawazen, Wynn, Robert F., Dalle, Jean-Hugues, Peffault de Latour, Régis, Sedlacek, Petr, Balduzzi, Adriana, Schroeder, Thomas, Bodova, Ivana, Gonzalez Vicent, Marta, Gruhn, Bernd, Hamladji, Rose-Marie, Krivan, Gergely, Patrick, Katharine, Sobkowiak-Sobierajska, Agnieszka, Stepensky, Polina, Unal, Ali, Amrolia, Persis, Perez Martinez, Antonio, Rialland, Fanny, Aljurf, Mahmoud, Isgro, Antonella, Toren, Amos, Bierings, Marc, Corbacioglu, Selim, and Kałwak, Krzysztof

Published
2024
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2. Increased risk of subsequent neoplasm after hematopoietic stem cell transplantation in 5-year survivors of childhood acute lymphoblastic leukemia

Author

Westerveld, Aimée S. R., Roesthuis, Pien, van der Pal, Helena J. H., Bresters, Dorine, Bierings, Marc, Loonen, Jacqueline, de Vries, Andrica C. H., Louwerens, Marloes, Koopman, Maria M. W., van den Heuvel-Eibrink, Marry M., van der Heiden-van der Loo, Margriet, Hoogerbrugge, Peter, Janssens, Geert O., de Krijger, Ronald R., Ronckers, Cecile M., Pieters, Rob, Kremer, Leontien C. M., and Teepen, Jop C.

Published
2024
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3. Hematopoietic stem cell transplantation for pediatric patients with non-anaplastic peripheral T-cell lymphoma. An EBMT pediatric diseases working party study

Author

Moser, Olga, Ngoya, Maud, Galimard, Jacques-Emmanuel, Dalissier, Arnaud, Dalle, Jean Hugues, Kalwak, Krzysztof, Wössmann, Wilhelm, Burkhardt, Birgit, Bierings, Marc, Gonzalez-Vicent, Marta, López Corral, Lucía, Mellgren, Karin, Attarbaschi, Andishe, Bourhis, Jean Henri, Carlson, Kristina, Corbacioglu, Selim, Drabko, Katarzyna, Sundin, Mikael, Toporski, Jacek, Cario, Gunnar, and Kontny, Udo

Published
2024
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6. Outcomes of hematopoietic stem cell transplantation in 813 pediatric patients with Fanconi anemia

Author

Lum, Su Han, Eikema, Dirk-Jan, Piepenbroek, Brian, Wynn, Robert F., Samarasinghe, Sujith, Dalissier, Arnaud, Kalwak, Krysztof, Ayas, Mouhab, Hamladji, Rose-Marie, Yesilipek, Akif, Dalle, Jean-Hugues, Uckan-Cetinkaya, Duygu, Bierings, Marc, Kupesiz, Alphan, Halahleh, Khalid, Skorobogatova, Elena, Öztürk, Gülyüz, Faraci, Maura, Renard, Cecile, Evans, Pamela, Corbacioglu, Selim, Locatelli, Franco, Dufour, Carlo, Risitano, Antonio, and Peffault de Latour, Régis

Published
2024
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9. Influence of invasive aspergillosis during acute leukaemia treatment on survival after allogeneic stem cell transplantation: a prospective study of the EBMT Infectious Diseases Working Party

Author

Penack, Olaf, Tridello, Gloria, Salmenniemi, Urpu, Martino, Rodrigo, Khanna, Nina, Perruccio, Katia, Fagioli, Franca, Richert-Przygonska, Monika, Labussière-Wallet, Hélène, Maertens, Johan, Jubert, Charlotte, Aljurf, Mahmoud, Pichler, Herbert, Kriván, Gergely, Kunadt, Desiree, Popova, Marina, Gabriel, Melissa, Calore, Elisabetta, Blau, Igor Wolfgang, Benedetti, Fabio, Itäla-Remes, Maija, de Kort, Elizabeth, Russo, Domenico, Faraci, Maura, Ménard, Anne-Lise, Borne, Peter von dem, Poiré, Xavier, Yesilipek, Akif, Gozdzik, Jolanta, Yeğin, Zeynep Arzu, Yañez, Lucrecia, Facchini, Luca, Van Gorkom, Gwendolyn, Thurner, Lorenz, Kocak, Ulker, Sampol, Antònia, Zuckerman, Tsila, Bierings, Marc, Mielke, Stephan, Ciceri, Fabio, Wendel, Lotus, Knelange, Nina, Mikulska, Malgorzata, Averbuch, Dina, Styczynski, Jan, Camara, Rafael de la, and Cesaro, Simone

Published
2024
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10. Comprehensive single-cell genome analysis at nucleotide resolution using the PTA Analysis Toolbox

Author

Middelkamp, Sjors, Manders, Freek, Peci, Flavia, van Roosmalen, Markus J., González, Diego Montiel, Bertrums, Eline J.M., van der Werf, Inge, Derks, Lucca L.M., Groenen, Niels M., Verheul, Mark, Trabut, Laurianne, Pleguezuelos-Manzano, Cayetano, Brandsma, Arianne M., Antoniou, Evangelia, Reinhardt, Dirk, Bierings, Marc, Belderbos, Mirjam E., and van Boxtel, Ruben

Published
2023
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11. Use of eculizumab in children with allogeneic haematopoietic stem cell transplantation associated thrombotic microangiopathy - a multicentre retrospective PDWP and IEWP EBMT study

Author

Svec, Peter, Elfeky, Reem, Galimard, Jacques-Emmanuel, Higham, Christine S., Dalissier, Arnaud, Quigg, Troy C., Bueno Sanchez, David, Han Lum, Su, Faraci, Maura, Cole, Theresa, Pichler, Herbert, Benítez-Carabante, Maria Isabel, Horakova, Julia, Gonzalez -Vicent, Marta, Yanir, Asaf, Fagioli, Franca, Wölfl, Matthias, von der Weid, Nicolas, Protheroe, Rachel, Krivan, Gergely, Speckmann, Carsten, James, Beki, Avcin, Simona Lucija, Bertrand, Yves, Verna, Marta, Riha, Petr, Patrick, Katharine, Cesaro, Simone, Kalwak, Krzysztof, Bierings, Marc, Büchner, Jochen, Mellgren, Karin, Prohászka, Zoltán, Neven, Bénédicte, Lankester, Arjan, and Corbacioglu, Selim

Published
2023
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12. Fludarabine exposure predicts outcome after CD19 CAR T-cell therapy in children and young adults with acute leukemia

Author

Dekker, Linde, Calkoen, Friso G., Jiang, Yilin, Blok, Hilly, Veldkamp, Saskia R., De Koning, Coco, Spoon, Maike, Admiraal, Rick, Hoogerbrugge, Peter, Vormoor, Britta, Vormoor, H. Josef, Visscher, Henk, Bierings, Marc, Van Der Vlugt, Marieke, Van Tinteren, Harm, Nijstad, A. Laura, Huitema, Alwin D.R., Van Der Elst, Kim C.M., Pieters, Rob, Lindemans, Caroline A., and Nierkens, Stefan

Published
2022
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15. Umbilical Cord Blood Transplantation for Fanconi Anemia With a Special Focus on Late Complications: a Study on Behalf of Eurocord and SAAWP-EBMT

Author

Rafii, Hanadi, Volt, Fernanda, Bierings, Marc, Dalle, Jean-Hugues, Ayas, Mouhab, Rihani, Rawad, Faraci, Maura, de Simone, Giuseppina, Sengeloev, Henrik, Passweg, Jakob, Cavazzana, Marina, Costello, Regis, Maertens, Johan, Biffi, Alessandra, Johansson, Jan-Erik, Montoro, Juan, Guepin, Gabrielle Roth, Diaz, Miguel Angel, Sirvent, Anne, Kenzey, Chantal, Rivera Franco, Monica M., Cappelli, Barbara, Scigliuolo, Graziana Maria, Rocha, Vanderson, Ruggeri, Annalisa, Risitano, Antonio, De Latour, Regis Peffault, and Gluckman, Eliane

Published
2024
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16. HEV infection in stem cell transplant recipients—retrospective study of EBMT Infectious Diseases Working Party

Author

Mikulska, Malgorzata, Penack, Olaf, Wendel, Lotus, Knelange, Nina, Cornelissen, Jan J., Blijlevens, Nicole, Passweg, Jakob, Kroger, Nicolaus, Bruns, Anke, Koenecke, Christian, Bierings, Marc, Piñana, José Luis, Labussiere-Wallet, Helene, Ghesquieres, Herve, Diaz, Miguel Angel, Sampol, Antonia, Averbuch, Diana, de la Camara, Rafael, and Styczynski, Jan

Published
2022
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17. Outcomes of pediatric patients with therapy-related myeloid neoplasms

Author

Sharma, Akshay, Huang, Sujuan, Li, Ying, Brooke, Russell J., Ahmed, Ibrahim, Allewelt, Heather B., Amrolia, Persis, Bertaina, Alice, Bhatt, Neel S., Bierings, Marc B., Bies, Joshua, Brisset, Claire, Brondon, Jennifer E., Dahlberg, Ann, Dalle, Jean-Hugues, Eissa, Hesham, Fahd, Mony, Gassas, Adam, Gloude, Nicholas J., Goebel, W. Scott, Goeckerman, Erika S., Harris, Katherine, Ho, Richard, Hudspeth, Michelle P., Huo, Jeffrey S., Jacobsohn, David, Kasow, Kimberly A., Katsanis, Emmanuel, Kaviany, Saara, Keating, Amy K., Kernan, Nancy A., Ktena, Yiouli P., Lauhan, Colette R., López-Hernandez, Gerardo, Martin, Paul L., Myers, Kasiani C., Naik, Swati, Olaya-Vargas, Alberto, Onishi, Toshihiro, Radhi, Mohamed, Ramachandran, Shanti, Ramos, Kristie, Rangarajan, Hemalatha G., Roehrs, Philip A., Sampson, Megan E., Shaw, Peter J., Skiles, Jodi L., Somers, Katherine, Symons, Heather J., de Tersant, Marie, Uber, Allison N., Versluys, Birgitta, Cheng, Cheng, and Triplett, Brandon M.

Published
2021
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18. Comparison of outcomes of immunosuppressive therapy with rabbit versus horse antithymocyte globulin and cyclosporine a in children with acquired severe aplastic anemia

Author

Yoshimi, Ayami, primary, Noellke, Peter, additional, Starý, Jan, additional, Kállay, Krisztián, additional, Smith, Owen, additional, Locatelli, Franco, additional, Buechner, Jochen, additional, Bodova, Ivana, additional, Sevilla, Julian, additional, Schmugge, Markus, additional, Bierings, Marc, additional, Masmas, Tania, additional, Dworzak, Michael, additional, Labarque, Veerle, additional, Pawelec, Katarzyna, additional, Jahnukainen, Kirsi, additional, Polychronopoulou, Sophia, additional, Kjollerstrom, Paula, additional, Kavcic, Marko, additional, Erlacher, Miriam, additional, Niemeyer, Charlotte M., additional, and Strahm, Brigitte, additional

Published
2024
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19. CD4+ T-cell reconstitution predicts survival outcomes after acute graft-versus-host-disease: a dual-center validation

Author

de Koning, Coco, Prockop, Susan, van Roessel, Ichelle, Kernan, Nancy, Klein, Elizabeth, Langenhorst, Jurgen, Szanto, Celina, Belderbos, Mirjam, Bierings, Marc, Boulad, Farid, Bresters, Dorine, Cancio, Maria, Curran, Kevin, Kollen, Wouter, O'Reilly, Richard, Scaradavou, Andromachi, Spitzer, Barbara, Versluijs, Birgitta, Huitema, Alwin, Lindemans, Caroline, Nierkens, Stefan, and Boelens, Jaap Jan

Published
2021
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20. Prophylactic use of liposomal amphotericin B in children and adolescents undergoing allogeneic hematopoietic cell transplantation: A 10-years single center experience

Author

Cancer, Infection & Immunity, CTI Nierkens, Infectieziekten patientenzorg, SCT patientenzorg, Child Health, Regenerative Medicine and Stem Cells, Infectiezieken, Rotte, Laura G.Y., de Koning, Coco C.H., Loeffen, Yvette G.T., Bierings, Marc B., Jan Boelens, Jaap, Lindemans, Caroline A., Wolfs, Tom F.W., Cancer, Infection & Immunity, CTI Nierkens, Infectieziekten patientenzorg, SCT patientenzorg, Child Health, Regenerative Medicine and Stem Cells, Infectiezieken, Rotte, Laura G.Y., de Koning, Coco C.H., Loeffen, Yvette G.T., Bierings, Marc B., Jan Boelens, Jaap, Lindemans, Caroline A., and Wolfs, Tom F.W.

Published
2024

21. High-dose individualized antithymocyte globulin with therapeutic drug monitoring in high-risk cord blood transplant

Author

Arts-assistenten Kinderen, Haematologie patientenzorg, Apotheek O&O&O, Cancer, Bureau Onderzoek, CTI Nierkens, SCT patientenzorg, Child Health, Infection & Immunity, Regenerative Medicine and Stem Cells, CTI Research, Admiraal, Rick, Versluijs, A Birgitta, Huitema, Alwin D R, Ebskamp, Lysette, Lacna, Amelia, de Kanter, C T Klaartje, Bierings, Marc B, Boelens, Jaap Jan, Lindemans, Caroline A, Nierkens, Stefan, Arts-assistenten Kinderen, Haematologie patientenzorg, Apotheek O&O&O, Cancer, Bureau Onderzoek, CTI Nierkens, SCT patientenzorg, Child Health, Infection & Immunity, Regenerative Medicine and Stem Cells, CTI Research, Admiraal, Rick, Versluijs, A Birgitta, Huitema, Alwin D R, Ebskamp, Lysette, Lacna, Amelia, de Kanter, C T Klaartje, Bierings, Marc B, Boelens, Jaap Jan, Lindemans, Caroline A, and Nierkens, Stefan

Published
2024

22. Continuous PEGasparaginase Dosing Reduces Hypersensitivity Reactions in Pediatric ALL:A Dutch Childhood Oncology Group ALL11 Randomized Trial

Author

Van Der Sluis, Inge M., Brigitha, Leiah J., Fiocco, Marta, De Groot-Kruseman, Hester A., Bierings, Marc, Van Den Bos, Cor, De Haas, Valerie, Hoogerbrugge, Peter M., Tissing, Wim J.E., Veening, Margreet A., Pieters, Rob, Van Der Sluis, Inge M., Brigitha, Leiah J., Fiocco, Marta, De Groot-Kruseman, Hester A., Bierings, Marc, Van Den Bos, Cor, De Haas, Valerie, Hoogerbrugge, Peter M., Tissing, Wim J.E., Veening, Margreet A., and Pieters, Rob

Abstract

PURPOSE:The primary objective of this randomized study was to determine whether a continuous dosing schedule (without the asparaginase-free interval) would result in less hypersensitivity reactions to PEGasparaginase (PEGasp) compared with the standard noncontinuous dosing schedule.METHODS:Eight hundred eighteen patients (age 1-18 years) with ALL were enrolled in the Dutch Childhood Oncology Group-ALL11 protocol and received PEGasp. Three hundred twelve patients stratified in the medium-risk arm were randomly assigned to receive 14 individualized PEGasp doses once every two weeks in either a noncontinuous or continuous schedule after the first three doses in induction (EudraCT: 2012-000067-25). Hypersensitivity reactions were defined as allergies, allergic-like reactions, and silent inactivation. Secondary end points were other asparaginase-related toxicities, asparaginase activity and antibody levels, and outcome.RESULTS:During induction, 27 of 818 patients (3.3%) experienced hypersensitivity reactions. After random assignment, 4 of 155 (2.6%) in the continuous treatment arm versus 17 of 157 (10.8%) patients in the noncontinuous treatment arm had hypersensitivity reactions (P <.01), of which two (1.3%) versus 13 (8.3%) were inactivating reactions (P <.01). The occurrence of inactivating hypersensitivity reactions was seven times lower in the continuous arm (odds ratio, 0.15 [0.032-0.653]). In addition, antibody levels were significantly lower in the continuous arm (P <.01). With exception of a lower incidence of increased amylase in the continuous arm, there were no significant differences in total number of asparaginase-associated toxicities between arms. However, the timing of the toxicities was associated with the timing of the asparaginase administrations. No difference in 5-year cumulative incidence of relapse, death, or disease-free survival was found between both treatment arms.CON

Published
2024

23. Increased risk of subsequent neoplasm after hematopoietic stem cell transplantation in 5-year survivors of childhood acute lymphoblastic leukemia

Author

Cancer, MS Radiotherapie, Pathologie Pathologen staf, Zorg en O&O, Child Health, Westerveld, Aimée S R, Roesthuis, Pien, van der Pal, Helena J H, Bresters, Dorine, Bierings, Marc, Loonen, Jacqueline, de Vries, Andrica C H, Louwerens, Marloes, Koopman, Maria M W, van den Heuvel-Eibrink, Marry M, van der Heiden-van der Loo, Margriet, Hoogerbrugge, Peter, Janssens, Geert O, de Krijger, Ronald R, Ronckers, Cecile M, Pieters, Rob, Kremer, Leontien C M, Teepen, Jop C, Cancer, MS Radiotherapie, Pathologie Pathologen staf, Zorg en O&O, Child Health, Westerveld, Aimée S R, Roesthuis, Pien, van der Pal, Helena J H, Bresters, Dorine, Bierings, Marc, Loonen, Jacqueline, de Vries, Andrica C H, Louwerens, Marloes, Koopman, Maria M W, van den Heuvel-Eibrink, Marry M, van der Heiden-van der Loo, Margriet, Hoogerbrugge, Peter, Janssens, Geert O, de Krijger, Ronald R, Ronckers, Cecile M, Pieters, Rob, Kremer, Leontien C M, and Teepen, Jop C

Published
2024

24. Increased risk of subsequent neoplasm after hematopoietic stem cell transplantation in 5-year survivors of childhood acute lymphoblastic leukemia

Author

Westerveld, Aimée S.R., Roesthuis, Pien, van der Pal, Helena J.H., Bresters, Dorine, Bierings, Marc, Loonen, Jacqueline, de Vries, Andrica C.H., Louwerens, Marloes, Koopman, Maria M.W., van den Heuvel-Eibrink, Marry M., van der Heiden-van der Loo, Margriet, Hoogerbrugge, Peter, Janssens, Geert O., de Krijger, Ronald R., Ronckers, Cecile M., Pieters, Rob, Kremer, Leontien C.M., Teepen, Jop C., Westerveld, Aimée S.R., Roesthuis, Pien, van der Pal, Helena J.H., Bresters, Dorine, Bierings, Marc, Loonen, Jacqueline, de Vries, Andrica C.H., Louwerens, Marloes, Koopman, Maria M.W., van den Heuvel-Eibrink, Marry M., van der Heiden-van der Loo, Margriet, Hoogerbrugge, Peter, Janssens, Geert O., de Krijger, Ronald R., Ronckers, Cecile M., Pieters, Rob, Kremer, Leontien C.M., and Teepen, Jop C.

Abstract

Acute lymphoblastic leukemia (ALL) survivors are at risk for developing subsequent neoplasms, but there is limited information on long-term risks and risk factors for both subsequent malignant neoplasms (SMNs) and subsequent non-malignant neoplasms (SNMNs). We analyzed long-term risk and risk factors for SMNs and SNMNs among 3291 5-year ALL survivors from the Dutch Childhood Cancer Survivor Study-LATER cohort (1963–2014). We calculated standardized incidence ratios (SIRs) and cumulative incidences and used multivariable Cox proportional hazard regression analyses for analyzing risk factors. A total of 97 survivors developed SMNs and 266 SNMNs. The 30-year cumulative incidence was 4.1% (95%CI: 3.5–5.3) for SMNs and 10.4%(95%CI: 8.9–12.1) for SNMNs. Risk of SMNs was elevated compared to the general population (SIR: 2.6, 95%CI: 2.1–3.1). Survivors treated with hematopoietic stem cell transplantation (HSCT) with total body irradiation (TBI) (HR:4.2, 95%CI: 2.3–7.9), and without TBI (HR:4.0,95%CI: 1.2–13.7) showed increased SMN risk versus non-transplanted survivors. Cranial radiotherapy (CRT) was also a risk factor for SMNs (HR:2.1, 95%CI: 1.4–4.0). In conclusion, childhood ALL survivors have an increased SMN risk, especially after HSCT and CRT. A key finding is that even HSCT-treated survivors without TBI treatment showed an increased SMN risk, possibly due to accompanied chemotherapy treatment. This emphasizes the need for careful follow-up of HSCT and/or CRT-treated survivors.

Published
2024

25. High-dose ERT, rituximab, and early HSCT in an infant with Wolman’s disease

Author

MS Algemene Pediatrie Onderzoek 2, UMC Krant, SCT patientenzorg, Child Health, Infection & Immunity, Regenerative Medicine and Stem Cells, Metabole ziekten patientenzorg, Eskandari, Siawosh K., Revenich, Elisabeth G.M., Pot, Dirk J., de Boer, Foekje, Bierings, Marc, van Spronsen, Francjan J., van Hasselt, Peter M., Lindemans, Caroline A., Lubout, Charlotte M.A., MS Algemene Pediatrie Onderzoek 2, UMC Krant, SCT patientenzorg, Child Health, Infection & Immunity, Regenerative Medicine and Stem Cells, Metabole ziekten patientenzorg, Eskandari, Siawosh K., Revenich, Elisabeth G.M., Pot, Dirk J., de Boer, Foekje, Bierings, Marc, van Spronsen, Francjan J., van Hasselt, Peter M., Lindemans, Caroline A., and Lubout, Charlotte M.A.

Published
2024

26. Predictors of outcomes in hematopoietic cell transplantation for Fanconi anemia

Author

SCT patientenzorg, Child Health, Infection & Immunity, Regenerative Medicine and Stem Cells, CTI De Witte, MS Hematologie, Cancer, Cancio, Maria, Troullioud Lucas, Alexandre G, Bierings, Marc, Klein, Elizabeth, de Witte, Moniek A, Smiers, Frans J, Bresters, Dorine, Boelens, Jaap Jan, Smetsers, Stephanie E, SCT patientenzorg, Child Health, Infection & Immunity, Regenerative Medicine and Stem Cells, CTI De Witte, MS Hematologie, Cancer, Cancio, Maria, Troullioud Lucas, Alexandre G, Bierings, Marc, Klein, Elizabeth, de Witte, Moniek A, Smiers, Frans J, Bresters, Dorine, Boelens, Jaap Jan, and Smetsers, Stephanie E

Published
2024

30. Continuous PEGasparaginase Dosing Reduces Hypersensitivity Reactions in Pediatric ALL: A Dutch Childhood Oncology Group ALL11 Randomized Trial

Author

van der Sluis, Inge M., primary, Brigitha, Leiah J., additional, Fiocco, Marta, additional, de Groot-Kruseman, Hester A., additional, Bierings, Marc, additional, van den Bos, Cor, additional, de Haas, Valerie, additional, Hoogerbrugge, Peter M., additional, Tissing, Wim J.E., additional, Veening, Margreet A., additional, and Pieters, Rob, additional

Published
2024
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31. Model-Based Dosing of Anti-Thymocyte Globulin in Pediatric Allogeneic Hematopoietic Cell Transplantation Improves Survival Chances: Updated Results from the Single Arm Phase II Parachute-Trial Combined with Real-World Data

Author

Admiraal, Rick, primary, Nierkens, Stefan, additional, Bierings, Marc, additional, Belderbos, Mirjam, additional, Huitema, Alwin D.R., additional, Bredius, Robbert, additional, Jiang, Yilin, additional, Curran, Kevin J., additional, Harris, Andrew C., additional, Scaradavou, Andromachi, additional, Cancio, Maria I., additional, Klein, Elizabeth, additional, Kollen, Wouter, additional, Bresters, Dorine, additional, Calkoen, Friso, additional, Versluijs, A. Birgitta, additional, Zwaan, Christian M., additional, Boelens, Jaap-Jan, additional, and Lindemans, Caroline A., additional

Published
2024
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32. Association of busulfan exposure with survival and toxicity after haemopoietic cell transplantation in children and young adults: a multicentre, retrospective cohort analysis

Author

Bartelink, Imke H, Lalmohamed, Arief, van Reij, Elisabeth ML, Dvorak, Christopher C, Savic, Rada M, Zwaveling, Juliette, Bredius, Robbert GM, Egberts, Antoine CG, Bierings, Marc, Kletzel, Morris, Shaw, Peter J, Nath, Christa E, Hempel, George, Ansari, Marc, Krajinovic, Maja, Théorêt, Yves, Duval, Michel, Keizer, Ron J, Bittencourt, Henrique, Hassan, Moustapha, Güngör, Tayfun, Wynn, Robert F, Veys, Paul, Cuvelier, Geoff DE, Marktel, Sarah, Chiesa, Robert, Cowan, Morton J, Slatter, Mary A, Stricherz, Melisa K, Jennissen, Cathryn, Long-Boyle, Janel R, and Boelens, Jaap Jan

Subjects
Cancer, Transplantation, Good Health and Well Being, Adolescent, Adult, Area Under Curve, Busulfan, Child, Child, Preschool, Cohort Studies, Disease-Free Survival, Dose-Response Relationship, Drug, Female, Graft Rejection, Graft Survival, Graft vs Host Disease, Hematopoietic Stem Cell Transplantation, Humans, Infant, Male, Recurrence, Retrospective Studies, Transplantation Conditioning, Transplantation, Homologous, Treatment Outcome, Cardiorespiratory Medicine and Haematology, Clinical Sciences
Abstract

BackgroundIntravenous busulfan combined with therapeutic drug monitoring to guide dosing improves outcomes after allogeneic haemopoietic cell transplantation (HCT). The best method to estimate busulfan exposure and optimum exposure in children or young adults remains unclear. We therefore assessed three approaches to estimate intravenous busulfan exposure (expressed as cumulative area under the curve [AUC]) and associated busulfan AUC with clinical outcomes in children or young adults undergoing allogeneic HCT.MethodsIn this retrospective analysis, patients from 15 centres in the Netherlands, USA, Canada, Switzerland, UK, Italy, Germany, and Australia who received a busulfan-based conditioning regimen between March 18, 2001, and Feb 12, 2015, were included. Cumulative AUC was calculated by numerical integration using non-linear mixed effect modelling (AUCNONMEM), non-compartmental analysis (AUC from 0 to infinity [AUC0-∞] and to the next dose [AUC0-τ]), and by individual centres using various approaches (AUCcentre). The main outcome of interest was event-free survival. Other outcomes of interest were graft failure or relapse, or both; transplantation-related mortality; acute toxicity (veno-occlusive disease or acute graft versus-host disease [GvHD]); chronic GvHD; overall survival; and chronic-GvHD-free event-free survival. We used propensity-score-adjusted Cox proportional hazard models, Weibull models, and Fine-Gray competing risk regressions for statistical analyses.Findings790 patients were enrolled, 674 of whom were included: 274 (41%) with malignant and 400 (59%) with non-malignant disease. Median age was 4·5 years (IQR 1·4-10·7). The median busulfan AUCNONMEM was 74·4 mg × h/L (95% CI 31·1-104·6), which correlated with the standardised method AUC0-∞ (r2=0·74), but the latter correlated poorly with AUCcentre (r2=0·35). Estimated 2-year event-free survival was 69·7% (95% CI 66·2-73·0). Event-free survival at 2 years was 77·0% (95% CI 72·1-82·9) in the 257 patients with an optimum intravenous busulfan AUC of 78-101 mg × h/L compared with 66·1% (60·9-71·4) in the 235 patients at the low historical target of 58-86 mg × h/L and 49·5% (29·2-66·0) in the 44 patients with a high (>101 mg × h/L) busulfan AUC (p=0·011). Compared with the low AUC group, graft failure or relapse occurred less frequently in the optimum AUC group (hazard ratio [HR] 0·57, 95% CI 0·39-0·84; p=0·0041). Acute toxicity (HR 1·69, 1·12-2·57; p=0·013) and transplantation-related mortality (2·99, 1·82-4·92; p101 mg × h/L) than in the low AUC group (

Published
2016

33. The impact of donor type on the outcome of pediatric patients with very high risk acute lymphoblastic leukemia. A study of the ALL SCT 2003 BFM-SG and 2007-BFM-International SG

Author

Dalle, Jean-Hugues, Balduzzi, Adriana, Bader, Peter, Pieczonka, Anna, Yaniv, Isaac, Lankester, Arjan, Bierings, Marc, Yesilipek, Akif, Sedlacek, Petr, Ifversen, Marianne, Svec, Peter, Toporski, Jacek, Gungor, Taifun, Wachowiak, Jacek, Glogova, Evgenia, Poetschger, Ulrike, and Peters, Christina

Published
2021
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34. Transplantation in Children and Adolescents with Acute Lymphoblastic Leukemia from a Matched Donor versus an HLA-Identical Sibling: Is the Outcome Comparable? Results from the International BFM ALL SCT 2007 Study

Author

Balduzzi, Adriana, Dalle, Jean-Hugues, Wachowiak, Jacek, Yaniv, Isaac, Yesilipek, Akif, Sedlacek, Petr, Bierings, Marc, Ifversen, Marianne, Sufliarska, Sabina, Kalwak, Krzysztof, Lankester, Arjan, Toporski, Jacek, Di Maio, Lucia, Glogova, Evgenia, Poetschger, Ulrike, and Peters, Christina

Published
2019
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35. Gonadal Function after Busulfan Compared with Treosulfan in Children and Adolescents Undergoing Allogeneic Hematopoietic Stem Cell Transplant

Author

Faraci, Maura, Diesch, Tamara, Labopin, Myriam, Dalissier, Arnaud, Lankester, Arian, Gennery, Andrew, Sundin, Mikael, Uckan-Cetinkaya, Duygu, Bierings, Marc, Peters, Anke M.J., Garwer, Martina, Schulz, Ansgar, Michel, Gerard, Giorgiani, Giovanna, Gruhn, Bernd, Locatelli, Franco, Giardino, Stefano, Uyttebroeck, Anne, Rialland, Fanny, Itäla-Remes, Maija, Dreger, Peter, Shaw, Peter J., Bordon, Victoria, Schlegel, Paul G., Mellgren, Karin, Moraleda, Jose M., Patrick, Katharine, Schneider, Pascale, Jubert, Charlotte, Lawitschka, Anita, Salooja, Nina, Basak, Grzegorz W., Corbacioglu, Selim, Duarte, Rafael, and Bader, Peter

Published
2019
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36. Allogeneic HSCT for classical BCR::ABL1-negative myeloproliferative neoplasms in children: the retrospective study of the EBMT Pediatric Diseases WP

Author

Wachowiak, Jacek, primary, Galimard, Jacques-Emmanuel, additional, Dalissier, Arnaud, additional, Rihani, Rawad, additional, ALSAEDI, HAWAZEN, additional, Wynn, Robert, additional, Dalle, Jean-Hugues, additional, de Latour, Regis Peffault, additional, Sedlacek, Petr, additional, Balduzzi, Adriana, additional, Schroeder, Thomas, additional, Bodova, Ivana, additional, Vicent, Marta Gonzalez, additional, Gruhn, Bernd, additional, Hamladji, Rose-Marie, additional, Kriván, Gergely, additional, Patrick, Katharine, additional, Sobkowiak-Sobierajska, Agnieszka, additional, Stepensky, Polina, additional, Unal, Ali, additional, Amrolia, Persis, additional, Perez-Martinez, Antonio, additional, Rialland, Fanny, additional, Aljurf, Mahmoud D, additional, Isgro', Antonella, additional, Toren, Amos, additional, Bierings, Marc, additional, Corbacioglu, Selim, additional, and Kalwak, Krzysztof, additional

Published
2023
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39. Allogeneic Stem Cell Transplantation from HLA-Mismatched Donors for Pediatric Patients with Acute Lymphoblastic Leukemia Treated According to the 2003 BFM and 2007 International BFM Studies: Impact of Disease Risk on Outcomes

Author

Dalle, Jean-Hugues, Balduzzi, Adriana, Bader, Peter, Lankester, Arjan, Yaniv, Isaac, Wachowiak, Jacek, Pieczonka, Anna, Bierings, Marc, Yesilipek, Akif, Sedlaçek, Petr, Ifversen, Marianne, Sufliarska, Sabina, Toporski, Jacek, Glogova, Evgenia, Poetschger, Ulrike, and Peters, Christina

Published
2018
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40. Intravenous Busulfan Compared with Total Body Irradiation Pretransplant Conditioning for Adults with Acute Lymphoblastic Leukemia

Author

Hogan, William, Battiwalla, Minoo, Copelan, Edward, Hildebrandt, Gerhard, Ganguly, Sid, Majhail, Navneet, Woolfrey, Ann, Nivison-Smith, Ian, Hertzberg, Mark, Diaz, Miguel Angel, Jakubowski, Ann, Ustun, Celalettin, Yong, Agnes, Freytes, Cesar, DeFilipp, Zachariah, Inamoto, Yoshi, Cahn, Jean-Yves, Savani, Bipin, Yared, Jean, Bajel, Ashish, Bacher, Ulrike, Uy, Geoffrey, Rizzieri, David, Wieduwilt, Matthew, Schultz, Kirk, Grunwald, Michael, Kamble, Rammurti, Qayed, Muna, Brammer, Jonathan, Ballen, Karen, Khera, Nandita, Schouten, Harry, Bierings, Marc, Kanakry, Christopher, Wood, William Allen, Saad, Ayman, Savoie, Lynn, Hamilton, Betty Ky, Aljurf, Mahmoud, Vij, Ravi, Pawarode, Attaphol, Olsson, Richard, Wirk, B. Mona, Beitinjaneh, Amer, Seo, Sachiko, Cerny, Jan, Akpek, Gorgun, Norkin, Maxim, Nishihori, Taiga, Lazaryan, Aleksandr, Sabloff, Mitchell, Kebriaei, Partow, Anasetti, Claudio, Zhang, Mei-Jie, Wang, Hai-Lin, Aldoss, Ibrahim, de Lima, Marcos, Khoury, H. Jean, Sandmaier, Brenda M., Horowitz, Mary M., Artz, Andrew, Bejanyan, Nelli, Ciurea, Stefan, Lazarus, Hillard M., Gale, Robert Peter, Litzow, Mark, Bredeson, Christopher, Seftel, Matthew D., Pulsipher, Michael A., Boelens, Jaap-Jan, Alvarnas, Joseph, Champlin, Richard, Forman, Stephen, Pullarkat, Vinod, Weisdorf, Daniel, and Marks, David I.

Published
2018
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42. Excellent leukemia control after second hematopoietic cell transplants with unrelated cord blood grafts for post-transplant relapse in pediatric patients

Author

Troullioud Lucas, Alexandre G., primary, Boelens, Jaap Jan, additional, Prockop, Susan E., additional, Curran, Kevin J., additional, Bresters, Dorine, additional, Kollen, Wouter, additional, Versluys, Birgitta, additional, Bierings, Marc B., additional, Archer, Anne, additional, Davis, Eric, additional, Klein, Elizabeth, additional, Kernan, Nancy A., additional, Lindemans, Caroline A., additional, and Scaradavou, Andromachi, additional

Published
2023
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44. Improved Outcome for ALL by Prolonging Therapy for IKZF1 Deletion and Decreasing Therapy for Other Risk Groups

Author

Pieters, Rob, primary, de Groot-Kruseman, Hester, additional, Fiocco, Marta, additional, Verwer, Femke, additional, Van Overveld, Merian, additional, Sonneveld, Edwin, additional, van der Velden, Vincent, additional, Beverloo, H. Berna, additional, Bierings, Marc, additional, Dors, Natasja, additional, de Haas, Valérie, additional, Hoogerbrugge, Peter, additional, Van der Sluis, Inge, additional, Tissing, Wim, additional, Veening, Margreet, additional, Boer, Judith, additional, and Den Boer, Monique, additional

Published
2023
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50. Improved Outcome for ALL by Prolonging Therapy for IKZF1 Deletion and Decreasing Therapy for Other Risk Groups

Author

Pieters, Rob, de Groot-Kruseman, Hester, Fiocco, Marta, Verwer, Femke, Van Overveld, Merian, Sonneveld, Edwin, van der Velden, Vincent, Beverloo, H. Berna, Bierings, Marc, Dors, Natasja, de Haas, Valérie, Hoogerbrugge, Peter, Van der Sluis, Inge, Tissing, Wim, Veening, Margreet, Boer, Judith, Den Boer, Monique, Pieters, Rob, de Groot-Kruseman, Hester, Fiocco, Marta, Verwer, Femke, Van Overveld, Merian, Sonneveld, Edwin, van der Velden, Vincent, Beverloo, H. Berna, Bierings, Marc, Dors, Natasja, de Haas, Valérie, Hoogerbrugge, Peter, Van der Sluis, Inge, Tissing, Wim, Veening, Margreet, Boer, Judith, and Den Boer, Monique

Abstract

PURPOSE: The ALL10 protocol improved outcomes for children with ALL by stratifying and adapting therapy into three minimal residual disease-defined risk groups: standard risk, medium risk (MR), and high risk. IKZF1-deleted (IKZF1del) ALL in the largest MR group still showed poor outcome, in line with protocols worldwide, accounting for a high number of overall relapses. ALL10 showed high toxicity in Down syndrome (DS) and excellent outcome in ETV6::RUNX1 ALL. Poor prednisone responders (PPRs) were treated as high risk in ALL10. In ALL11, we prolonged therapy for IKZF1del from 2 to 3 years. We reduced therapy for DS by omitting anthracyclines completely, for ETV6::RUNX1 in intensification, and for PPR by treatment as MR. METHODS:Eight hundred nineteen patients with ALL (age, 1-18 years) were enrolled on ALL11 and stratified as in ALL10. Results were compared with those in ALL10. RESULTS: The five-year overall survival (OS), event-free survival (EFS), cumulative risk of relapse (CIR), and death in complete remission on ALL11 were 94.2% (SE, 0.9%), 89.0% (1.2), 8.2% (1.1), and 2.3% (0.6), respectively. Prolonged maintenance for IKZF1del MR improved 5-year CIR by 2.2-fold (10.8% v 23.4%; P = .035) and EFS (87.1% v 72.3%; P = .019). Landmark analysis at 2 years from diagnosis showed a 2.9-fold reduction of CIR (25.6%-8.8%; P = .008) and EFS improvement (74.4%-91.2%; P = .007). Reduced therapy did not abrogate 5-year outcome for ETV6::RUNX1 (EFS, 98.3%; OS, 99.4%), DS (EFS, 87.0%; OS, 87.0%), and PPR (EFS, 81.1%; OS, 94.9%). CONCLUSION: Children with IKZF1del ALL seem to benefit from prolonged maintenance therapy. Chemotherapy was successfully reduced for patients with ETV6::RUNX1, DS, and PPR ALL. It has to be noted that these results were obtained in a nonrandomized study using a historical control group.

Published
2023

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