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1. Graft-versus-host disease after anti-CD19 chimeric antigen receptor T-cell therapy following allogeneic hematopoietic cell transplantation: a transplant complications and paediatric diseases working parties joint EBMT study: IMMUNOTHERAPY

Author

Ortí, Guillermo, Peczynski, Christophe, Boreland, William, O’Reilly, Maeve, von Bonin, Malte, Balduzzi, Adriana, Besley, Caroline, Kalwak, Krzysztof, Ryhänen, Samppa, Güngör, Tayfun, Wynn, Robert F., Bader, Peter, Mielke, Stephan, Blaise, Didier, Amrolia, Persis, Yakoub-Agha, Ibrahim, Calkoen, Friso, Schubert, Maria-Luisa, Potter, Victoria, Pichler, Herbert, Kröger, Nicolaus, Kwon, Mi, Sengeloev, Henrik, Torrent, Anna, Chalandon, Yves, van Gorkom, Gwendolyn, Koenecke, Christian, Graham, Charlotte, Schoemans, Helene, Moiseev, Ivan, Penack, Olaf, and Peric, Zinaida

Published
2025
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2. Outcomes of patients undergoing allogeneic haematopoietic stem cell transplantation for congenital amegakaryocytic thrombocytopenia; a study on behalf of the PDWP of the EBMT

Author

Aldebert, Clémence, Fahd, Mony, Galimard, Jacques-Emmanuel, Ghemlas, Ibrahim A., Zecca, Marco, Silva, Juliana, Mohseny, Alexander, Kupesiz, Alphan, Hamladji, Rose-Marie, Miranda, Nuno, Güngör, Tayfun, Wynn, Robert F., Merli, Pietro, Sundin, Mikael, Faraci, Maura, Diaz-de-Heredia, Cristina, Burkhardt, Birgit, Bordon, Victoria, Angoso, Marie, Bader, Peter, Ifversen, Marianne, Herrera Arroyo, Concepcion, Maximova, Natalia, Riesco, Susana, Stein, Jerry, Dalissier, Arnaud, Locatelli, Franco, Kalwak, Krzysztof, Dalle, Jean-Hugues, and Corbacioglu, Selim

Published
2024
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4. Comparable outcomes after busulfan- or treosulfan-based conditioning for allo-HSCT in children with ALL: results of FORUM

Author

Kalwak, Krzysztof, Moser, Laura M., Pötschger, Ulrike, Bader, Peter, Kleinschmidt, Katharina, Meisel, Roland, Dalle, Jean-Hugues, Yesilipek, Akif, Balduzzi, Adriana, Krivan, Gergely, Goussetis, Evgenios, Staciuk, Raquel, Sedlacek, Petr, Pichler, Herbert, Svec, Peter, Gabriel, Melissa, Güngör, Tayfun, Bilic, Ernest, Buechner, Jochen, Renard, Marleen, Vettenranta, Kim, Ifversen, Marianne, Diaz-de-Heredia, Cristina, Stein, Jerry, Toporski, Jacek, Bierings, Marc, Peters, Christina, Ansari, Marc, and Locatelli, Franco

Published
2025
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5. Association of busulfan exposure and outcomes after HCT for patients with an inborn error of immunity

Author

Bognàr, Tim, Garcia-Rosa, Moises, Lalmohamed, Arief, Güngör, Tayfun, Hauri-Hohl, Mathias, Prockop, Susan, Oram, Layne, Pai, Sung-Yun, Brooks, Jordan, Savic, Rada M., Dvorak, Christopher C., Long-Boyle, Janel R., Krajinovic, Maja, Bittencourt, Henrique, Teyssier, Anne-Charlotte, Théorêt, Yves, Martinez, Cary, Egberts, Toine C. G., Morales, Erin, Slatter, Mary, Cuvelier, Geoffrey D. E., Chiesa, Robert, Wynn, Robert F., Coussons, Mary, Cicalese, Maria P., Ansari, Marc, Long, Susan E., Ebens, Christen L., Lust, Hannah, Chaudhury, Sonali, Nath, Christa E., Shaw, Peter J., Keogh, Steven J., van der Stoep, M. Y. Eileen C., Bredius, Robbert, Lindemans, Caroline A., Boelens, Jaap-Jan, and Bartelink, Imke H.

Published
2024
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6. Ruxolitinib in treatment-naive or corticosteroid-refractory paediatric patients with chronic graft-versus-host disease (REACH5): interim analysis of a single-arm, multicentre, phase 2 study

Author

Locatelli, Franco, Antmen, Bulent, Kang, Hyoung Jin, Koh, Katsuyoshi, Takahashi, Yoshiyuki, Kupesiz, Alphan, Dias Matos, Maria Gabriela A, Chopra, Yogi, Bhat, Sunil, Im, Ho Joon, Güngör, Tayfun, Lu, Meng-Yao, Stefanelli, Tommaso, Rosko, Christine, St Pierre, Annie, Burock, Karin, Smith, Yvonne, Sinclair, Karen, and Diaz-de-Heredia, Cristina

Published
2024
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8. Low rate of nonrelapse mortality in under-4-year-olds with ALL given chemotherapeutic conditioning for HSCT: a phase 3 FORUM study

Author

Bader, Peter, Pötschger, Ulrike, Dalle, Jean-Hugues, Moser, Laura M., Balduzzi, Adriana, Ansari, Marc, Buechner, Jochen, Güngör, Tayfun, Ifversen, Marianne, Krivan, Gergely, Pichler, Herbert, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr, Stein, Jerry, Heusel, Jan Robert, Truong, Tony, Wachowiak, Jacek, Yesilipek, Akif, Locatelli, Franco, and Peters, Christina

Published
2024
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9. Artificial thymic organoids represent a reliable tool to study T-cell differentiation in patients with severe T-cell lymphopenia

Author

Bosticardo, Marita, Pala, Francesca, Calzoni, Enrica, Delmonte, Ottavia M, Dobbs, Kerry, Gardner, Cameron L, Sacchetti, Nicolo’, Kawai, Tomoki, Garabedian, Elizabeth K, Draper, Debbie, Bergerson, Jenna RE, DeRavin, Suk See, Freeman, Alexandra F, Güngör, Tayfun, Hartog, Nicholas, Holland, Steven M, Kohn, Donald B, Malech, Harry L, Markert, Mary Louise, Weinacht, Katja G, Villa, Anna, Seet, Christopher S, Montel-Hagen, Amelie, Crooks, Gay M, and Notarangelo, Luigi D

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Hematology, Stem Cell Research - Nonembryonic - Human, Clinical Research, Stem Cell Research, 2.1 Biological and endogenous factors, Inflammatory and immune system, Antigens, CD34, Cell Differentiation, Hematopoietic Stem Cells, Humans, Lymphopenia, Organoids, Cardiovascular medicine and haematology
Abstract

The study of early T-cell development in humans is challenging because of limited availability of thymic samples and the limitations of in vitro T-cell differentiation assays. We used an artificial thymic organoid (ATO) platform generated by aggregating a DLL4-expressing stromal cell line (MS5-hDLL4) with CD34+ cells isolated from bone marrow or mobilized peripheral blood to study T-cell development from CD34+ cells of patients carrying hematopoietic intrinsic or thymic defects that cause T-cell lymphopenia. We found that AK2 deficiency is associated with decreased cell viability and an early block in T-cell development. We observed a similar defect in a patient carrying a null IL2RG mutation. In contrast, CD34+ cells from a patient carrying a missense IL2RG mutation reached full T-cell maturation, although cell numbers were significantly lower than in controls. CD34+ cells from patients carrying RAG mutations were able to differentiate to CD4+CD8+ cells, but not to CD3+TCRαβ+ cells. Finally, normal T-cell differentiation was observed in a patient with complete DiGeorge syndrome, consistent with the extra-hematopoietic nature of the defect. The ATO system may help determine whether T-cell deficiency reflects hematopoietic or thymic intrinsic abnormalities and define the exact stage at which T-cell differentiation is blocked.

Published
2020

11. Second allogeneic stem cell transplantation can rescue a significant proportion of patients with JMML relapsing after first allograft

Author

Vinci, Luca, Flotho, Christian, Noellke, Peter, Lebrecht, Dirk, Masetti, Riccardo, de Haas, Valerie, De Moerloose, Barbara, Dworzak, Michael, Hasle, Henrik, Güngör, Tayfun, Starý, Jan, Turkiewicz, Dominik, Ussowicz, Marek, de Heredia, Cristina Diaz, Buechner, Jochen, Jahnukainen, Kirsi, Kallay, Krisztian, Bodova, Ivana, Smith, Owen P., Zecca, Marco, Bresters, Dorine, Lang, Peter, Masmas, Tania Nicole, Meisel, Roland, Pichler, Herbert, Erlacher, Miriam, Göhring, Gudrun, Locatelli, Franco, Strahm, Brigitte, Niemeyer, Charlotte M., and Yoshimi, Ayami

Published
2023
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13. Incidence of subsequent malignancies after total body irradiation-based allogeneic HSCT in children with ALL – long-term follow-up from the prospective ALL-SCT 2003 trial

Author

Eichinger, Anna, Poetschger, Ulrike, Glogova, Evgenia, Bader, Peter, Basu, Oliver, Beier, Rita, Burkhardt, Birgit, Classen, Carl-Friedrich, Claviez, Alexander, Corbacioglu, Selim, Deubzer, Hedwig E., Greil, Johann, Gruhn, Bernd, Güngör, Tayfun, Kafa, Kinan, Kühl, Jörn-Sven, Lang, Peter, Lange, Bjoern Soenke, Meisel, Roland, Müller, Ingo, Sauer, Martin G., Schlegel, Paul-Gerhardt, Schulz, Ansgar, Stachel, Daniel, Strahm, Brigitte, Wawer, Angela, Peters, Christina, and Albert, Michael H.

Published
2022
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14. Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study

Author

Aiuti, Alessandro, Maschan, Alexei, Aljurf, Mahmoud, Gedde-Dahl, Tobias, Gurman, Gunhan, Bordon, Victoria, Kriván, Gergely, Locatelli, Franco, Porta, Fulvio, Valcárcel, David, Beguin, Yves, Faraci, Maura, Kröger, Nicolaus, Kulagin, Aleksandr, Shaw, Peter J., Veelken, Joan Hendrik, Diaz de Heredia, Cristina, Fagioli, Franca, Felber, Matthias, Gruhn, Bernd, Holter, Wolfgang, Rössig, Claudia, Sedlacek, Petr, Apperley, Jane, Ayas, Mouhab, Bodova, Ivana, Choi, Goda, Cornelissen, J.J., Sirvent, Anne, Khan, Anjum, Kupesiz, Alphan, Lenhoff, Stig, Ozdogu, Hakan, von der Weid, Nicolas, Rovira, Montserrat, Schots, Rik, Vinh, Donald C., Albert, Michael H., Sirait, Tiarlan, Eikema, Dirk-Jan, Bakunina, Katerina, Wehr, Claudia, Suarez, Felipe, Fox, Maria Laura, Mahlaoui, Nizar, Gennery, Andrew R., Lankester, Arjan C., Beier, Rita, Bernardo, Maria Ester, Bigley, Venetia, Lindemans, Caroline A., Burns, Siobhan O., Carpenter, Ben, Dybko, Jaroslaw, Güngör, Tayfun, Hauck, Fabian, Lum, Su Han, Balashov, Dmitry, Meisel, Roland, Moshous, Despina, Schulz, Ansgar, Speckmann, Carsten, Slatter, Mary A., Strahm, Brigitte, Uckan-Cetinkaya, Duygu, Meyts, Isabelle, Vallée, Tanja C., Wynn, Robert, Neven, Bénédicte, and Morris, Emma C.

Published
2022
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15. Mulch pneumonitis in chronic granulomatous disease: More than just a fungal infection.

Author

Soomann, Maarja, Prader, Seraina, Güngör, Tayfun, Pachlopnik Schmid, Jana, Warris, Adilia, and Trück, Johannes

Subjects
HEMATOPOIETIC stem cell transplantation, CHRONIC granulomatous disease, INTERSTITIAL lung diseases, ADULT respiratory distress syndrome, SYMPTOMS, PEER review of students
Abstract

Mulch pneumonitis is a rare form of acute fungal pneumonitis that can occur in patients with chronic granulomatous disease (CGD) following exposure to decaying organic matter. A case study of an 8-year-old girl in Europe highlights the challenges in diagnosing and treating this condition, which can lead to severe respiratory complications. The patient responded well to a combination of antifungal therapy and steroids, ultimately requiring hematopoietic stem cell transplantation for long-term management. The case underscores the importance of timely testing and functional results in diagnosing CGD and optimizing therapeutic decisions. [Extracted from the article]

Published
2024
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16. Hematopoietic cell transplantation in severe combined immunodeficiency: The SCETIDE 2006-2014 European cohort

Author

Lankester, Arjan C., Neven, Benedicte, Mahlaoui, Nizar, von Asmuth, Erik G.J., Courteille, Virginie, Alligon, Mikael, Albert, Michael H., Serra, Isabelle Badell, Bader, Peter, Balashov, Dmitry, Beier, Rita, Bertrand, Yves, Blanche, Stephane, Bordon, Victoria, Bredius, Robbert G., Cant, Andrew, Cavazzana, Marina, Diaz-de-Heredia, Cristina, Dogu, Figen, Ehlert, Karoline, Entz-Werle, Natacha, Fasth, Anders, Ferrua, Francesca, Ferster, Alina, Formankova, Renata, Friedrich, Wilhelm, Gonzalez-Vicent, Marta, Gozdzik, Jolanta, Güngör, Tayfun, Hoenig, Manfred, Ikinciogullari, Aydan, Kalwak, Krzysztof, Kansoy, Savas, Kupesiz, Alphan, Lanfranchi, Arnalda, Lindemans, Caroline A., Meisel, Roland, Michel, Gerard, Miranda, Nuno A.A., Moraleda, Jose, Moshous, Despina, Pichler, Herbert, Rao, Kanchan, Sedlacek, Petr, Slatter, Mary, Soncini, Elena, Speckmann, Carsten, Sundin, Mikael, Toren, Amos, Vettenranta, Kim, Worth, Austen, Yeşilipek, Mehmet A., Zecca, Marco, Porta, Fulvio, Schulz, Ansgar, Veys, Paul, Fischer, Alain, and Gennery, Andrew R.

Published
2022
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17. Association Between the Magnitude of Intravenous Busulfan Exposure and Development of Hepatic Veno-Occlusive Disease in Children and Young Adults Undergoing Myeloablative Allogeneic Hematopoietic Cell Transplantation

Author

Bognàr, Tim, Bartelink, Imke H., Egberts, Toine C.G., Rademaker, Carin M.A., Versluys, A. Birgitta, Slatter, Mary A., Kletzel, Morris, Nath, Christa E., Cuvelier, Geoffrey D.E, Savic, Rada M., Dvorak, Christopher, Long-Boyle, Janel R., Cowan, Morton J., Bittencourt, Henrique, Bredius, Robbert G.M., Güngör, Tayfun, Shaw, Peter J., Ansari, Marc, Hassan, Moustapha, Krajinovic, Maja, Hempel, Georg, Marktel, Sarah, Chiesa, Robert, Théoret, Yves, Lund, Troy, Orchard, Paul J., Wynn, Robert F., Boelens, Jaap Jan, and Lalmohamed, Arief

Published
2022
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18. Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis

Author

Albert, Michael H., Slatter, Mary A., Gennery, Andrew R., Güngör, Tayfun, Bakunina, Katerina, Markovitch, Benyamin, Hazelaar, Sheree, Sirait, Tiarlan, Courteille, Virginie, Aiuti, Alessandro, Aleinikova, Olga V., Balashov, Dmitry, Bernardo, Maria Ester, Bodova, Ivana, Bruno, Benedicte, Cavazzana, Marina, Chiesa, Robert, Fischer, Alain, Hauck, Fabian, Ifversen, Marianne, Kałwak, Krzysztof, Klein, Christoph, Kulagin, Alexander, Kupesiz, Alphan, Kuskonmaz, Baris, Lindemans, Caroline A., Locatelli, Franco, Lum, Su Han, Maschan, Alexey, Meisel, Roland, Moshous, Despina, Porta, Fulvio, Sauer, Martin G., Sedlacek, Petr, Schulz, Ansgar, Suarez, Felipe, Vallée, Tanja C., Winiarski, Jacek H., Zecca, Marco, Neven, Bénédicte, Veys, Paul, and Lankester, Arjan C.

Published
2022
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22. Outcome of hematopoietic cell transplantation for DNA double-strand break repair disorders

Author

Slack, James, Albert, Michael H, Balashov, Dmitry, Belohradsky, Bernd H, Bertaina, Alice, Bleesing, Jack, Booth, Claire, Buechner, Jochen, Buckley, Rebecca H, Ouachée-Chardin, Marie, Deripapa, Elena, Drabko, Katarzyna, Eapen, Mary, Feuchtinger, Tobias, Finocchi, Andrea, Gaspar, H Bobby, Ghosh, Sujal, Gillio, Alfred, Gonzalez-Granado, Luis I, Grunebaum, Eyal, Güngör, Tayfun, Heilmann, Carsten, Helminen, Merja, Higuchi, Kohei, Imai, Kohsuke, Kalwak, Krzysztof, Kanazawa, Nubuo, Karasu, Gülsün, Kucuk, Zeynep Y, Laberko, Alexandra, Lange, Andrzej, Mahlaoui, Nizar, Meisel, Roland, Moshous, D, Muramatsu, Hideki, Parikh, Suhag, Pasic, Srdjan, Schmid, Irene, Schuetz, Catharina, Schulz, Ansgar, Schultz, Kirk R, Shaw, Peter J, Slatter, Mary A, Sykora, Karl-Walter, Tamura, Shinobu, Taskinen, Mervi, Wawer, Angela, Wolska-Kuśnierz, Beata, Cowan, Morton J, Fischer, Alain, and Gennery, Andrew R

Subjects
Rare Diseases, Transplantation, Hematology, Genetics, Cancer, Pediatric, Adolescent, Alleles, Child, Child, Preschool, DNA Breaks, Double-Stranded, DNA Repair, DNA Repair-Deficiency Disorders, Female, Follow-Up Studies, Graft vs Host Disease, Hematopoietic Stem Cell Transplantation, Humans, Infant, Kaplan-Meier Estimate, Male, Mutation, Prognosis, Treatment Outcome, Virus Diseases, Young Adult, Ataxia-telangiectasia, Cernunnos-XLF deficiency, DNA repair disorders, DNA ligase IV deficiency, hematopoietic stem cell transplantation, Nijmegen breakage syndrome, Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation and the European Society for Immunodeficiencies, Stem Cell Transplant for Immunodeficiencies in Europe, Center for International Blood and Marrow Transplant Research, Primary Immunodeficiency Treatment Consortium, Immunology, Allergy
Abstract

BackgroundRare DNA breakage repair disorders predispose to infection and lymphoreticular malignancies. Hematopoietic cell transplantation (HCT) is curative, but coadministered chemotherapy or radiotherapy is damaging because of systemic radiosensitivity. We collected HCT outcome data for Nijmegen breakage syndrome, DNA ligase IV deficiency, Cernunnos-XRCC4-like factor (Cernunnos-XLF) deficiency, and ataxia-telangiectasia (AT).MethodsData from 38 centers worldwide, including indication, donor, conditioning regimen, graft-versus-host disease, and outcome, were analyzed. Conditioning was classified as myeloablative conditioning (MAC) if it contained radiotherapy or alkylators and reduced-intensity conditioning (RIC) if no alkylators and/or 150 mg/m2 fludarabine or less and 40 mg/kg cyclophosphamide or less were used.ResultsFifty-five new, 14 updated, and 18 previously published patients were analyzed. Median age at HCT was 48 months (range, 1.5-552 months). Twenty-nine patients underwent transplantation for infection, 21 had malignancy, 13 had bone marrow failure, 13 received pre-emptive transplantation, 5 had multiple indications, and 6 had no information. Twenty-two received MAC, 59 received RIC, and 4 were infused; information was unavailable for 2 patients. Seventy-three of 77 patients with DNA ligase IV deficiency, Cernunnos-XLF deficiency, or Nijmegen breakage syndrome received conditioning. Survival was 53 (69%) of 77 and was worse for those receiving MAC than for those receiving RIC (P = .006). Most deaths occurred early after transplantation, suggesting poor tolerance of conditioning. Survival in patients with AT was 25%. Forty-one (49%) of 83 patients experienced acute GvHD, which was less frequent in those receiving RIC compared with those receiving MAC (26/56 [46%] vs 12/21 [57%], P = .45). Median follow-up was 35 months (range, 2-168 months). No secondary malignancies were reported during 15 years of follow-up. Growth and developmental delay remained after HCT; immune-mediated complications resolved.ConclusionRIC HCT resolves DNA repair disorder-associated immunodeficiency. Long-term follow-up is required for secondary malignancy surveillance. Routine HCT for AT is not recommended.

Published
2018

23. Multisystem inflammation and susceptibility to viral infections in human ZNFX1 deficiency

Author

Vavassori, Stefano, Chou, Janet, Faletti, Laura Eva, Haunerdinger, Veronika, Opitz, Lennart, Joset, Pascal, Fraser, Christopher J., Prader, Seraina, Gao, Xianfei, Schuch, Luise A., Wagner, Matias, Hoefele, Julia, Maccari, Maria Elena, Zhu, Ying, Elakis, George, Gabbett, Michael T., Forstner, Maria, Omran, Heymut, Kaiser, Thomas, Kessler, Christina, Olbrich, Heike, Frosk, Patrick, Almutairi, Abduarahman, Platt, Craig D., Elkins, Megan, Weeks, Sabrina, Rubin, Tamar, Planas, Raquel, Marchetti, Tommaso, Koovely, Danil, Klämbt, Verena, Soliman, Neveen A., von Hardenberg, Sandra, Klemann, Christian, Baumann, Ulrich, Lenz, Dominic, Klein-Franke, Andreas, Schwemmle, Martin, Huber, Michael, Sturm, Ekkehard, Hartleif, Steffen, Häffner, Karsten, Gimpel, Charlotte, Brotschi, Barbara, Laube, Guido, Güngör, Tayfun, Buckley, Michael F., Kottke, Raimund, Staufner, Christian, Hildebrandt, Friedhelm, Reu-Hofer, Simone, Moll, Solange, Weber, Achim, Kaur, Hundeep, Ehl, Stephan, Hiller, Sebastian, Geha, Raif, Roscioli, Tony, Griese, Matthias, and Pachlopnik Schmid, Jana

Published
2021
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24. Hematopoietic Cell Transplantation Cures Adenosine Deaminase 2 Deficiency: Report on 30 Patients

Author

Hashem, Hasan, Bucciol, Giorgia, Ozen, Seza, Unal, Sule, Bozkaya, Ikbal Ok, Akarsu, Nurten, Taskinen, Mervi, Koskenvuo, Minna, Saarela, Janna, Dimitrova, Dimana, Hickstein, Dennis D., Hsu, Amy P., Holland, Steven M., Krance, Robert, Sasa, Ghadir, Kumar, Ashish R., Müller, Ingo, de Sousa, Monica Abreu, Delafontaine, Selket, Moens, Leen, Babor, Florian, Barzaghi, Federica, Cicalese, Maria Pia, Bredius, Robbert, van Montfrans, Joris, Baretta, Valentina, Cesaro, Simone, Stepensky, Polina, Benedicte, Neven, Moshous, Despina, Le Guenno, Guillaume, Boutboul, David, Dalal, Jignesh, Brooks, Joel P., Dokmeci, Elif, Dara, Jasmeen, Lucas, Carrie L., Hambleton, Sophie, Wilson, Keith, Jolles, Stephen, Koc, Yener, Güngör, Tayfun, Schnider, Caroline, Candotti, Fabio, Steinmann, Sandra, Schulz, Ansgar, Chambers, Chip, Hershfield, Michael, Ombrello, Amanda, Kanakry, Jennifer A., and Meyts, Isabelle

Published
2021
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26. Hematopoietic cell transplantation in chronic granulomatous disease: a study of 712 children and adults

Author

Chiesa, Robert, Wang, Junfeng, Blok, Henric-Jan, Hazelaar, Sheree, Neven, Benedicte, Moshous, Despina, Schulz, Ansgar, Hoenig, Manfred, Hauck, Fabian, Al Seraihy, Amal, Gozdzik, Jolanta, Ljungman, Per, Lindemans, Caroline A., Fernandes, Juliana F., Kalwak, Krzysztof, Strahm, Brigitte, Schanz, Urs, Sedlacek, Petr, Sykora, Karl-Walter, Aksoylar, Serap, Locatelli, Franco, Stepensky, Polina, Wynn, Robert, Lum, Su Han, Zecca, Marco, Porta, Fulvio, Taskinen, Mervi, Gibson, Brenda, Matthes, Susanne, Karakukcu, Musa, Hauri-Hohl, Mathias, Veys, Paul, Gennery, Andrew R., Lucchini, Giovanna, Felber, Matthias, Albert, Michael H., Balashov, Dmitry, Lankester, Arjan, Güngör, Tayfun, and Slatter, Mary A.

Published
2020
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27. Targeted busulfan-based reduced-intensity conditioning and HLA-matched HSCT cure hemophagocytic lymphohistiocytosis

Author

Felber, Matthias, Steward, Colin G., Kentouche, Karim, Fasth, Anders, Wynn, Robert F., Zeilhofer, Ulrike, Haunerdinger, Veronika, Volkmer, Benjamin, Prader, Seraina, Gruhn, Bernd, Ehl, Stephan, Lehmberg, Kai, Müller, Daniel, Gennery, Andrew R., Albert, Michael H., Hauck, Fabian, Rao, Kanchan, Veys, Paul, Hassan, Moustapha, Lankester, Arjan C., Schmid, Jana Pachlopnik, Hauri-Hohl, Mathias M., and Güngör, Tayfun

Published
2020
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28. Favorable outcomes of hematopoietic stem cell transplantation in children and adolescents with Diamond-Blackfan anemia

Author

Strahm, Brigitte, Loewecke, Felicia, Niemeyer, Charlotte M., Albert, Michael, Ansari, Marc, Bader, Peter, Bertrand, Yves, Burkhardt, Birgit, Da Costa, Lydie M., Ferster, Alina, Fischer, Alexandra, Güngör, Tayfun, Gruhn, Bernd, Hainmann, Ina, Kapp, Friedrich, Lang, Peter, Müller, Ingo, Schulz, Ansgar, Szvetnik, Amina, Wlodarski, Marcin, Noellke, Peter, Leblanc, Thierry, and Dalle, Jean-Hugues

Published
2020
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31. 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
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Cancer, Transplantation, 6.1 Pharmaceuticals, 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, Cardiovascular medicine and haematology
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

32. Correction to: Hematopoietic Cell Transplantation Cures Adenosine Deaminase 2 Deficiency: Report on 30 Patients

Author

Hashem, Hasan, Bucciol, Giorgia, Ozen, Seza, Unal, Sule, Bozkaya, Ikbal Ok, Akarsu, Nurten, Taskinen, Mervi, Koskenvuo, Minna, Saarela, Janna, Dimitrova, Dimana, Hickstein, Dennis D., Hsu, Amy P., Holland, Steven M., Krance, Robert, Sasa, Ghadir, Kumar, Ashish R., Müller, Ingo, de Sousa, Monica Abreu, Delafontaine, Selket, Moens, Leen, Babor, Florian, Barzaghi, Federica, Cicalese, Maria Pia, Bredius, Robbert, van Montfrans, Joris, Baretta, Valentina, Cesaro, Simone, Stepensky, Polina, Benedicte, Neven, Moshous, Despina, Le Guenno, Guillaume, Boutboul, David, Dalal, Jignesh, Brooks, Joel P., Dokmeci, Elif, Dara, Jasmeen, Lucas, Carrie L., Hambleton, Sophie, Wilson, Keith, Jolles, Stephen, Koc, Yener, Güngör, Tayfun, Schnider, Caroline, Candotti, Fabio, Steinmann, Sandra, Schulz, Ansgar, Chambers, Chip, Hershfield, Michael, Ombrello, Amanda, Kanakry, Jennifer A., and Meyts, Isabelle

Published
2022
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33. Role of HLA-B exon 1 in graft-versus-host disease after unrelated haemopoietic cell transplantation: a retrospective cohort study

Author

Petersdorf, Effie W, Carrington, Mary, O'hUigin, Colm, Bengtsson, Mats, De Santis, Dianne, Dubois, Valerie, Gooley, Ted, Horowitz, Mary, Hsu, Katharine, Madrigal, J Alejandro, Maiers, Martin, Malkki, Mari, McKallor, Caroline, Morishima, Yasuo, Oudshoorn, Machteld, Spellman, Stephen, Villard, Jean, Stevenson, Phil, Apperley, Jane, Bardy, Peter, Bernard, Ghislaine, Bertrand, Yves, Bloor, Adrian, Bonini, Chiara, Buhler, Stephane, Bungener, Laura, Campbell, Helen, Carlson, Kristina, Carpenter, Ben, Cesbron, Anne, Chabannon, Christian, Chalandon, Yves, Chapman, Jeremy, Chebel, Réda, Chevallier, Patrice, Choi, Gerda, Collin, Matt, Cornelissen, Jan J, Crawley, Charles, D'Orsogna, Lloyd, Dalle, Jean-Hugues, Deconinck, Eric, DeMatteis, Muriel, Diviney, Mary, Dormoy, Anne, Gagne, Katia, Gibson, Brenda, Gilleece, Maria, Gottlieb, David, Gribben, John, Güngör, Tayfun, Haagenson, Mike, Hart, Cathie, Holdsworth, Rhonda, Humphreys, Ian, Kodera, Yoshihisa, Koh, Mickey, Labussière-Wallet, Hélène, Lankester, Arjan C, Lardy, Neubery, Lawson, Sarah, Leleu, Xavier, MacKinnon, Stephen, Malladi, Ram, Marsh, Steven GE, Martin, Murray, Mayor, Neema P, McQuaker, I Grant, Meijer, Ellen, Morishima, Satoko, Nikolousis, Emmanouil, Orchard, Kim, Passweg, Jacob, Patel, Amit, Patrick, Katherine, Pedron, Béatrice, Peniket, Andy, Perry, Julia, Petersen, Eefke, Potter, Victoria, Potter, Mike, Protheroe, Rachel, Raus, Nicole, Ruiz de Elvira, Carmen, Russell, Nigel, Schaap, Nicholaas PM, Schanz, Urs, Schouten, Harry, Skinner, Roderick, Snowden, John, Spierings, Eric, Steward, Colin, Tholouli, Eleni, Thornton, Alycia, Tilanus, Marcel, van de Meer, Arnold, Veelkens, Hendrik, Veys, Paul, Watson, Narelle, Weston, Lyanne, Wilson, Keith, Wilson, Marie, Wynn, Robert, Zsiros, József, Maiers, Martin J, and Spellman, Stephen R

Published
2020
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34. Technical comment on: Trottmann M, et al. Real-world expenditures and survival time after CAR-T treatment for large B-cell lymphoma in Switzerland: a retrospective study using insurance claims data

Author

Arber, Caroline, Baerlocher, Gabriela, Chalandon, Yves; https://orcid.org/0000-0001-9341-8104, Daskalakis, Michael; https://orcid.org/0000-0002-8138-0505, Duchosal, Michel, Fehr, Martin, Gerull, Sabine; https://orcid.org/0000-0002-6612-4220, Güngör, Tayfun; https://orcid.org/0000-0002-3261-1186, Nair, Gayathri, Pabst, Thomas; https://orcid.org/0000-0002-6055-5257, Passweg, Jakob R; https://orcid.org/0000-0001-7092-3351, Piccolruaz, Barbara, Renner, Christoph, Ruefer, Axel, Schneidawind, Dominik; https://orcid.org/0000-0002-2672-431X, Stüssi, Georg, Zeerleder, Sacha, Halter, Jörg P, Arber, Caroline, Baerlocher, Gabriela, Chalandon, Yves; https://orcid.org/0000-0001-9341-8104, Daskalakis, Michael; https://orcid.org/0000-0002-8138-0505, Duchosal, Michel, Fehr, Martin, Gerull, Sabine; https://orcid.org/0000-0002-6612-4220, Güngör, Tayfun; https://orcid.org/0000-0002-3261-1186, Nair, Gayathri, Pabst, Thomas; https://orcid.org/0000-0002-6055-5257, Passweg, Jakob R; https://orcid.org/0000-0001-7092-3351, Piccolruaz, Barbara, Renner, Christoph, Ruefer, Axel, Schneidawind, Dominik; https://orcid.org/0000-0002-2672-431X, Stüssi, Georg, Zeerleder, Sacha, and Halter, Jörg P

Published
2024

35. Low rate of nonrelapse mortality in under 4-year-olds with ALL given chemo-conditioning for HSCT: Phase III FORUM study

Author

Bader, P, Poetschger, U, Dalle, J, Moser, L, Balduzzi, A, Ansari, M, Buechner, J, Güngör, T, Ifversen, M, Kriván, G, Pichler, H, Renard, M, Staciuk, R, Sedlacek, P, Stein, J, Heusel, J, Truong, T, Wachowiak, J, Yeşilipek, M, Locatelli, F, Peters, C, Bader, Peter, Poetschger, Ulrike, Dalle, Jean-Hugues, Moser, Laura M, Balduzzi, Adriana Cristina, Ansari, Marc, Buechner, Jochen, Güngör, Tayfun, Ifversen, Marianne, Kriván, Gergely, Pichler, Herbert, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr, Stein, Jerry, Heusel, Jan Robert, Truong, Tony, Wachowiak, Jacek, Yeşilipek, M Akif, Locatelli, Franco, Peters, Christina, Bader, P, Poetschger, U, Dalle, J, Moser, L, Balduzzi, A, Ansari, M, Buechner, J, Güngör, T, Ifversen, M, Kriván, G, Pichler, H, Renard, M, Staciuk, R, Sedlacek, P, Stein, J, Heusel, J, Truong, T, Wachowiak, J, Yeşilipek, M, Locatelli, F, Peters, C, Bader, Peter, Poetschger, Ulrike, Dalle, Jean-Hugues, Moser, Laura M, Balduzzi, Adriana Cristina, Ansari, Marc, Buechner, Jochen, Güngör, Tayfun, Ifversen, Marianne, Kriván, Gergely, Pichler, Herbert, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr, Stein, Jerry, Heusel, Jan Robert, Truong, Tony, Wachowiak, Jacek, Yeşilipek, M Akif, Locatelli, Franco, and Peters, Christina

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) is highly effective for treating pediatric high-risk or relapsed acute lymphoblastic leukemia (ALL). For young children, total body irradiation (TBI) is associated with severe late sequelae. In the FORUMstudy (NCT01949129), we assessed safety, event-free survival (EFS), and overall survival (OS) of 2 TBIfree conditioning regimens in children aged <4 years with ALL. Patients received fludarabine (Flu), thiotepa (Thio), and either busulfan (Bu) or treosulfan (Treo) before HSCT. From 2013 to 2021, 191 children received transplantation and were observed for ≥6 months (median followup: 3 years). The 3-year OS was 0.63 (95% confidence interval [95% CI], 0.52-0.72) and 0.76 (95% CI, 0.64-0.84) for Flu/Thio/Bu and Flu/Thio/Treo (P = .075), respectively. Three-year EFSwas 0.52 (95% CI, 0.41-0.61) and 0.51 (95% CI, 0.39-0.62), respectively (P = .794). Cumulative incidence of nonrelapse mortality (NRM)and relapse at 3 years were 0.06 (95% CI, 0.02-0.12) vs 0.03 (95%CI: [removed]1 acute graft-versus-host disease (GVHD) occurred in 29% of patients receiving Flu/Thio/ Bu and 17% of those receiving Flu/Thio/Treo (P = .049), whereas grade 3/4 occurred in 10% and 9%, respectively (P = .813). The 3-year incidence of chronic GVHD was 0.07 (95% CI, 0.03-0.13) vs 0.05 (95% CI, 0.02-0.11), respectively (P = .518). In conclusion, both chemotherapeutic conditioning regimens were well tolerated and NRM was low. However, relapse was the major cause of treatment failure.

Published
2024

36. Low rate of nonrelapse mortality in under-4-year-olds with ALL given chemotherapeutic conditioning for HSCT: a phase 3 FORUM study

Author

Bader, Peter; https://orcid.org/0000-0003-4554-0265, Pötschger, Ulrike, Dalle, Jean-Hugues; https://orcid.org/0000-0002-8406-0793, Moser, Laura M, Balduzzi, Adriana; https://orcid.org/0000-0002-5879-0610, Ansari, Marc; https://orcid.org/0000-0002-9649-6498, Buechner, Jochen; https://orcid.org/0000-0001-5848-4501, Güngör, Tayfun; https://orcid.org/0000-0002-3261-1186, Ifversen, Marianne, Krivan, Gergely, Pichler, Herbert; https://orcid.org/0000-0001-5107-5123, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr; https://orcid.org/0000-0001-7895-2886, Stein, Jerry, Heusel, Jan Robert; https://orcid.org/0000-0002-5753-0817, Truong, Tony, Wachowiak, Jacek, Yesilipek, Akif, Locatelli, Franco; https://orcid.org/0000-0002-7976-3654, Peters, Christina; https://orcid.org/0000-0003-0369-8515, Bader, Peter; https://orcid.org/0000-0003-4554-0265, Pötschger, Ulrike, Dalle, Jean-Hugues; https://orcid.org/0000-0002-8406-0793, Moser, Laura M, Balduzzi, Adriana; https://orcid.org/0000-0002-5879-0610, Ansari, Marc; https://orcid.org/0000-0002-9649-6498, Buechner, Jochen; https://orcid.org/0000-0001-5848-4501, Güngör, Tayfun; https://orcid.org/0000-0002-3261-1186, Ifversen, Marianne, Krivan, Gergely, Pichler, Herbert; https://orcid.org/0000-0001-5107-5123, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr; https://orcid.org/0000-0001-7895-2886, Stein, Jerry, Heusel, Jan Robert; https://orcid.org/0000-0002-5753-0817, Truong, Tony, Wachowiak, Jacek, Yesilipek, Akif, Locatelli, Franco; https://orcid.org/0000-0002-7976-3654, and Peters, Christina; https://orcid.org/0000-0003-0369-8515

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) is highly effective for treating pediatric high-risk or relapsed acute lymphoblastic leukemia (ALL). For young children, total body irradiation (TBI) is associated with severe late sequelae. In the FORUM study (NCT01949129), we assessed safety, event-free survival (EFS), and overall survival (OS) of 2 TBI-free conditioning regimens in children aged <4 years with ALL. Patients received fludarabine (Flu), thiotepa (Thio), and either busulfan (Bu) or treosulfan (Treo) before HSCT. From 2013 to 2021, 191 children received transplantation and were observed for ≥6 months (median follow-up: 3 years). The 3-year OS was 0.63 (95% confidence interval [95% CI], 0.52-0.72) and 0.76 (95% CI, 0.64-0.84) for Flu/Thio/Bu and Flu/Thio/Treo (P = .075), respectively. Three-year EFS was 0.52 (95% CI, 0.41-0.61) and 0.51 (95% CI, 0.39-0.62), respectively (P = .794). Cumulative incidence of nonrelapse mortality (NRM) and relapse at 3 years were 0.06 (95% CI, 0.02-0.12) vs 0.03 (95% CI: <0.01-0.09) (P = .406) and 0.42 (95% CI, 0.31-0.52) vs 0.45 (95% CI, 0.34-0.56) (P = .920), respectively. Grade >1 acute graft-versus-host disease (GVHD) occurred in 29% of patients receiving Flu/Thio/Bu and 17% of those receiving Flu/Thio/Treo (P = .049), whereas grade 3/4 occurred in 10% and 9%, respectively (P = .813). The 3-year incidence of chronic GVHD was 0.07 (95% CI, 0.03-0.13) vs 0.05 (95% CI, 0.02-0.11), respectively (P = .518). In conclusion, both chemotherapeutic conditioning regimens were well tolerated and NRM was low. However, relapse was the major cause of treatment failure. This trial was registered at www.clinicaltrials.gov as #NCT01949129.

Published
2024

37. Reducing Mortality and Morbidity in Children with Severe Combined Immunodeficiency in Switzerland: the Role of Newborn Screening

Author

Soomann, Maarja; https://orcid.org/0000-0002-2237-5327, Prader, Seraina; https://orcid.org/0000-0002-2950-5411, Pinto Monteiro, Aline, Zeilhofer, Ulrike, Hauri-Hohl, Mathias, Güngör, Tayfun; https://orcid.org/0000-0002-3261-1186, Pachlopnik Schmid, Jana; https://orcid.org/0000-0002-6653-9047, Trück, Johannes; https://orcid.org/0000-0002-0418-7381, Felber, Matthias, Soomann, Maarja; https://orcid.org/0000-0002-2237-5327, Prader, Seraina; https://orcid.org/0000-0002-2950-5411, Pinto Monteiro, Aline, Zeilhofer, Ulrike, Hauri-Hohl, Mathias, Güngör, Tayfun; https://orcid.org/0000-0002-3261-1186, Pachlopnik Schmid, Jana; https://orcid.org/0000-0002-6653-9047, Trück, Johannes; https://orcid.org/0000-0002-0418-7381, and Felber, Matthias

Abstract

Newborn screening (NBS) for severe combined immunodeficiency (SCID) has been introduced in various countries with the aim of reducing morbidity and mortality. However, studies analyzing outcomes before and after the implementation of NBS programs remain limited. This study sought to compare the outcomes of SCID patients identified through Switzerland’s national SCID NBS program, introduced in January 2019, with those of a historical cohort diagnosed between 2007 and 2019. The study included seven patients (32%) identified through NBS, and 15 (68%) born before NBS implementation and diagnosed based on clinical signs. Children in the NBS group were younger at diagnosis (median age 9 days vs 9 months, P = .002) and at hematopoietic stem cell transplantation (HSCT, median age 5 months vs 11 months, P = .003) compared to the clinical group. The NBS group had a lower incidence of infections before HSCT (29% vs 93%, P = .004). Although not statistically significant, the overall survival rate on last follow-up was higher in the NBS group (86% vs 67%, P = .62). Importantly, patients with active infections undergoing HSCT had a significantly lower overall survival probability compared to those without (P = .01). In conclusion, the introduction of NBS in Switzerland has led to earlier and often asymptomatic diagnosis of affected children, enabling timely intervention, infection prevention, and prompt treatment. These factors have contributed to higher survival rates in the NBS group. These findings underscore the critical importance of NBS for SCID, offering potential life-saving benefits through early detection and intervention.

Published
2024

38. Long-term outcome after allogeneic hematopoietic stem cell transplantation for Shwachman–Diamond syndrome: a retrospective analysis and a review of the literature by the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation (SAAWP-EBMT)

Author

Cesaro, Simone, Pillon, Marta, Sauer, Martin, Smiers, Frans, Faraci, Maura, de Heredia, Cristina Diaz, Wynn, Robert, Greil, Johann, Locatelli, Franco, Veys, Paul, Uyttebroeck, Anne, Ljungman, Per, Chevalier, Patrice, Ansari, Marc, Badell, Isabel, Güngör, Tayfun, Salim, Rahuman, Tischer, Johanna, Tecchio, Cristina, Russell, Nigel, Chybicka, Alicja, Styczynski, Jan, Krivan, Gergely, Smith, Owen, Stein, Jerry, Afanasyev, Boris, Pochon, Cécile, Menconi, Maria Cristina, Bosman, Paul, Mauro, Margherita, Tridello, Gloria, de Latour, Regis Peffault, and Dufour, Carlo

Published
2020
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39. Supportive care during pediatric hematopoietic stem cell transplantation: beyond infectious diseases. A report from workshops on supportive care of the Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT)

Author

Nava, Tiago, Ansari, Marc, Dalle, Jean-Hugues, de Heredia, Christina Diaz, Güngör, Tayfun, Trigoso, Eugenia, Falkenberg, Ulrike, Bertaina, Alice, Gibson, Brenda, Jarisch, Andrea, Balduzzi, Adriana, Boenig, Halvard, Krivan, Gergely, Vettenranta, Kim, Matic, Toni, Buechner, Jochen, Kalwak, Krzysztof, Lawitschka, Anita, Yesilipek, Akif, Lucchini, Giovanna, Peters, Christina, Turkiewicz, Dominik, Niinimäki, Riitta, Diesch, Tamara, Lehrnbecher, Thomas, Sedlacek, Petr, Hutt, Daphna, Dalissier, Arnaud, Wachowiak, Jacek, Yaniv, Isaac, Stein, Jerry, Yalçin, Koray, Sisinni, Luisa, Deiana, Marco, Ifversen, Marianne, Kuhlen, Michaela, Meisel, Roland, Bakhtiar, Shahrzad, Cesaro, Simone, Willasch, Andre, Corbacioglu, Selim, and Bader, Peter

Published
2020
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40. High Grades of Amphiregulin Expression in Intestinal Tissue Samples of Pediatric Patients with Severe Acute Graft-Versus-Host Disease after Allogeneic Hematopoietic Cell Transplantation

Author

Zeka, Fjolla, primary, Angori, Silvia, additional, Rutishauser, Dr. Dorothea, additional, Moch, Holger, additional, Posovszky, Carsten, additional, Amin, Khalid, additional, Holtan, Shernan Grace, additional, Güngör, Tayfun, additional, and Drozdov, Dr. Daniel, additional

Published
2024
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41. Comparison of outcomes of hematopoietic stem cell transplantation without chemotherapy conditioning by using matched sibling and unrelated donors for treatment of severe combined immunodeficiency

Author

Dvorak, Christopher C, Hassan, Amel, Slatter, Mary A, Hönig, Manfred, Lankester, Arjan C, Buckley, Rebecca H, Pulsipher, Michael A, Davis, Jeffrey H, Güngör, Tayfun, Gabriel, Melissa, Bleesing, Jacob H, Bunin, Nancy, Sedlacek, Petr, Connelly, James A, Crawford, David F, Notarangelo, Luigi D, Pai, Sung-Yun, Hassid, Jake, Veys, Paul, Gennery, Andrew R, and Cowan, Morton J

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Transplantation, Cancer, Regenerative Medicine, Hematology, Clinical Research, Stem Cell Research - Nonembryonic - Human, Stem Cell Research, Adult, Australia, B-Lymphocytes, Child, Chimerism, Europe, Female, Graft vs Host Disease, Hematopoietic Stem Cell Transplantation, Histocompatibility, Histocompatibility Testing, Humans, Infant, Infant, Newborn, Male, North America, Retrospective Studies, Severe Combined Immunodeficiency, Siblings, Survival Analysis, T-Lymphocytes, Transplantation Conditioning, Treatment Outcome, Unrelated Donors, Volunteers, Severe combined immunodeficiency, hematopoietic cell transplantation, sibling donors, unrelated donors, umbilical cord blood, conditioning, serotherapy, Conditioning, Hematopoietic cell transplantation, Serotherapy, Sibling donors, Umbilical cord blood, Unrelated donors, Immunology, Allergy
Abstract

BackgroundPatients with severe combined immunodeficiency disease who have matched sibling donors (MSDs) can proceed to hematopoietic cell transplantation (HCT) without conditioning chemotherapy.ObjectiveWe sought to determine whether the results of HCT without chemotherapy-based conditioning from matched unrelated donors (URDs), either from volunteer adults or umbilical cord blood, are comparable with those from MSDs.MethodsWe performed a multicenter survey of severe combined immunodeficiency transplantation centers in North America, Europe, and Australia to compile retrospective data on patients who have undergone unconditioned HCT from either URDs (n = 37) or MSDs (n = 66).ResultsMost patients undergoing URD HCT (92%) achieved donor T-cell engraftment compared with 97% for those with MSDs; however, estimated 5-year overall and event-free survival were worse for URD recipients (71% and 60%, respectively) compared with MSD recipients (92% and 89%, respectively; P < .01 for both). URD recipients who received pre-HCT serotherapy had similar 5-year overall survival (100%) to MSD recipients. The incidences of grade II to IV acute and chronic graft-versus-host disease were higher in URD (50% and 39%, respectively) compared with MSD (22% and 5%, respectively) recipients (P < .01 for both). In the surviving patients there was no difference in T-cell reconstitution at the last follow-up between the URD and MSD recipients; however, MSD recipients were more likely to achieve B-cell reconstitution (72% vs 17%, P < .001).ConclusionUnconditioned URD HCT achieves excellent rates of donor T-cell engraftment similar to that seen in MSD recipients, and reconstitution rates are adequate. However, only a minority will have myeloid and B-cell reconstitution, and attention must be paid to graft-versus-host disease prophylaxis. This approach might be safer in children ineligible for intense regimens to spare the potential complications of chemotherapy.

Published
2014

45. Treatment dilemmas in asymptomatic children with primary hemophagocytic lymphohistiocytosis

Author

Lucchini, Giovanna, Marsh, Rebecca, Gilmour, Kimberly, Worth, Austen, Nademi, Zohreh, Rao, Anupama, Booth, Claire, Amrolia, Persis, Silva, Juliana, Chiesa, Robert, Wynn, Robert, Lehmberg, Kai, Astigarraga, Itziar, Güngör, Tayfun, Stary, Jan, Moshous, Despina, Ifversen, Marianne, Zinn, Daniel, Jordan, Michael, Kumar, Ashish, Yasumi, Takahiro, Veys, Paul, and Rao, Kanchan

Published
2018
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46. Presence of centromeric but absence of telomeric group B KIR haplotypes in stem cell donors improve leukaemia control after HSCT for childhood ALL

Author

Babor, Florian, Peters, Christina, Manser, Angela R., Glogova, Evgenia, Sauer, Martin, Pötschger, Ulrike, Ahlmann, Martina, Cario, Gunnar, Feuchtinger, Tobias, Gruhn, Bernd, Güngör, Tayfun, Horn, Peter A., Kremens, Bernhard, Lang, Peter, Mezger, Markus, Müller, Ingo, Mytilineos, Joannis, Oevermann, Lena, Pichler, Herbert, Scherenschlich, Nadine, Schuster, Friedhelm R., Siepermann, Meinolf, Stachel, Daniel, Strahm, Brigitte, Wössmann, Wilhelm, Escherich, Gabriele, Zimmermann, Martin, Schrappe, Martin, Borkhardt, Arndt, Eckert, Cornelia, Bader, Peter, Uhrberg, Markus, and Meisel, Roland

Published
2019
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47. High-resolution HLA phased haplotype frequencies to predict the success of unrelated donor searches and clinical outcome following hematopoietic stem cell transplantation

Author

Buhler, Stéphane, Baldomero, Helen, Ferrari-Lacraz, Sylvie, Nunes, José Manuel, Sanchez-Mazas, Alicia, Massouridi-Levrat, Stravroula, Heim, Dominik, Halter, Jörg, Nair, Gayathri, Chalandon, Yves, Schanz, Urs, Güngör, Tayfun, Nicoloso, Grazia, Tiercy, Jean-Marie, Passweg, Jakob, and Villard, Jean

Published
2019
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48. Hematopoietic stem cell transplantation in 29 patients hemizygous for hypomorphic IKBKG/NEMO mutations

Author

Miot, Charline, Imai, Kohsuke, Imai, Chihaya, Mancini, Anthony J., Kucuk, Zeynep Yesim, Kawai, Tokomki, Nishikomori, Ryuta, Ito, Etsuro, Pellier, Isabelle, Dupuis Girod, Sophie, Rosain, Jeremie, Sasaki, Shinya, Chandrakasan, Shanmuganathan, Pachlopnik Schmid, Jana, Okano, Tsubasa, Colin, Estelle, Olaya-Vargas, Alberto, Yamazaki-Nakashimada, Marco, Qasim, Waseem, Espinosa Padilla, Sara, Jones, Andrea, Krol, Alfons, Cole, Nyree, Jolles, Stephen, Bleesing, Jack, Vraetz, Thomas, Gennery, Andrew R., Abinun, Mario, Güngör, Tayfun, Costa-Carvalho, Beatriz, Condino-Neto, Antonio, Veys, Paul, Holland, Steven M., Uzel, Gulbu, Moshous, Despina, Neven, Benedicte, Blanche, Stéphane, Ehl, Stephan, Döffinger, Rainer, Patel, Smita Y., Puel, Anne, Bustamante, Jacinta, Gelfand, Erwin W., Casanova, Jean-Laurent, Orange, Jordan S., and Picard, Capucine

Published
2017
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