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1. Allogeneic Hematopoietic Stem Cell Transplantation in Immunodeficiency—Centromeric Instability—Facial Dysmorphism (ICF) Syndrome: an EBMT/ESID Inborn Errors Working Party Study

Author

Berghuis, Dagmar, Mehyar, Lubna S., Abu-Arja, Rolla, Albert, Michael H., Barnum, Jessie L., von Bernuth, Horst, Elfeky, Reem, Lewalle, Philippe, Laberko, Alexandra, Ghosh, Sujal, Slatter, Mary A., Weemaes, Corry M. R., Yesilipek, Akif, Sirait, Tiarlan, Neven, Bénédicte, Gennery, Andrew R., and Lankester, Arjan C.

Published
2024
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3. Hematopoietic stem cell transplantation for CTLA-4 insufficiency across Europe: A European Society for Blood and Marrow Transplantation Inborn Errors Working Party study

Author

Tsilifis, Christo, Speckmann, Carsten, Lum, Su Han, Fox, Thomas A., Soler, Adriana Margarit, Mozo, Yasmina, Corral, Dolores, Ewins, Anna-Maria, Hague, Rosie, Oikonomopoulou, Christina, Kałwak, Krzysztof, Drabko, Katarzyna, Wynn, Robert, Morris, Emma C., Elcombe, Suzanne, Bigley, Venetia, Lougaris, Vassilios, Malagola, Michele, Hauck, Fabian, Sedlacek, Petr, Laberko, Alexandra, Tjon, Jennifer M.L., Buddingh, Emilie P., Wehr, Claudia, Grimbacher, Bodo, Gennery, Andrew R., Lankester, Arjan C., Albert, Michael H., Neven, Bénédicte, and Slatter, Mary A.

Published
2024
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5. International retrospective study of allogeneic hematopoietic cell transplantation for activated PI3K-delta syndrome

Author

Dimitrova, Dimana, Nademi, Zohreh, Maccari, Maria Elena, Ehl, Stephan, Uzel, Gulbu, Tomoda, Takahiro, Okano, Tsubasa, Imai, Kohsuke, Carpenter, Benjamin, Ip, Winnie, Rao, Kanchan, Worth, Austen JJ, Laberko, Alexandra, Mukhina, Anna, Néven, Bénédicte, Moshous, Despina, Speckmann, Carsten, Warnatz, Klaus, Wehr, Claudia, Abolhassani, Hassan, Aghamohammadi, Asghar, Bleesing, Jacob J, Dara, Jasmeen, Dvorak, Christopher C, Ghosh, Sujal, Kang, Hyoung Jin, Markelj, Gašper, Modi, Arunkumar, Bayer, Diana K, Notarangelo, Luigi D, Schulz, Ansgar, Garcia-Prat, Marina, Soler-Palacín, Pere, Karakükcü, Musa, Yilmaz, Ebru, Gambineri, Eleonora, Menconi, Mariacristina, Masmas, Tania N, Holm, Mette, Bonfim, Carmem, Prando, Carolina, Hughes, Stephen, Jolles, Stephen, Morris, Emma C, Kapoor, Neena, Koltan, Sylwia, Paneesha, Shankara, Steward, Colin, Wynn, Robert, Duffner, Ulrich, Gennery, Andrew R, Lankester, Arjan C, Slatter, Mary, and Kanakry, Jennifer A

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Hematology, Rare Diseases, Cancer, Transplantation, Regenerative Medicine, Adolescent, Adult, Aged, Child, Child, Preschool, Class I Phosphatidylinositol 3-Kinases, Female, Graft Rejection, Hematopoietic Stem Cell Transplantation, Humans, Kaplan-Meier Estimate, MTOR Inhibitors, Male, Middle Aged, Phosphatidylinositol 3-Kinases, Primary Immunodeficiency Diseases, Retrospective Studies, Transplantation, Homologous, Treatment Outcome, Young Adult, Primary immunodeficiency, activated phosphoinositide 3-kinase delta syndrome, lymphoproliferation, allogeneic hemato-poietic cell transplantation, graft failure, mTOR inhibitor, serotherapy, allogeneic hematopoietic cell transplantation, Immunology, Allergy
Abstract

BackgroundActivated phosphoinositide 3-kinase delta syndrome (APDS) is a combined immunodeficiency with a heterogeneous phenotype considered reversible by allogeneic hematopoietic cell transplantation (HCT).ObjectivesThis study sought to characterize HCT outcomes in APDS.MethodsRetrospective data were collected on 57 patients with APDS1/2 (median age, 13 years; range, 2-66 years) who underwent HCT.ResultsPre-HCT comorbidities such as lung, gastrointestinal, and liver pathology were common, with hematologic malignancy in 26%. With median follow-up of 2.3 years, 2-year overall and graft failure-free survival probabilities were 86% and 68%, respectively, and did not differ significantly by APDS1 versus APDS2, donor type, or conditioning intensity. The 2-year cumulative incidence of graft failure following first HCT was 17% overall but 42% if mammalian target of rapamycin inhibitor(s) (mTORi) were used in the first year post-HCT, compared with 9% without mTORi. Similarly, 2-year cumulative incidence of unplanned donor cell infusion was overall 28%, but 65% in the context of mTORi receipt and 23% without. Phenotype reversal occurred in 96% of evaluable patients, of whom 17% had mixed chimerism. Vulnerability to renal complications continued post-HCT, adding new insights into potential nonimmunologic roles of phosphoinositide 3-kinase not correctable through HCT.ConclusionsGraft failure, graft instability, and poor graft function requiring unplanned donor cell infusion were major barriers to successful HCT. Post-HCT mTORi use may confer an advantage to residual host cells, promoting graft instability. Longer-term post-HCT follow-up of more patients is needed to elucidate the kinetics of immune reconstitution and donor chimerism, establish approaches that reduce graft instability, and assess the completeness of phenotype reversal over time.

Published
2022

7. JAK inhibitor treatment for inborn errors of JAK/STAT signaling: An ESID/EBMT-IEWP retrospective study

Author

Fischer, Marco, Olbrich, Peter, Hadjadj, Jérôme, Aumann, Volker, Bakhtiar, Shahrzad, Barlogis, Vincent, von Bismarck, Philipp, Bloomfield, Markéta, Booth, Claire, Buddingh, Emmeline P., Cagdas, Deniz, Castelle, Martin, Chan, Alice Y., Chandrakasan, Shanmuganathan, Chetty, Kritika, Cougoul, Pierre, Crickx, Etienne, Dara, Jasmeen, Deyà-Martínez, Angela, Farmand, Susan, Formankova, Renata, Gennery, Andrew R., Gonzalez-Granado, Luis Ignacio, Hagin, David, Hanitsch, Leif Gunnar, Hanzlikovà, Jana, Hauck, Fabian, Ivorra-Cortés, José, Kisand, Kai, Kiykim, Ayca, Körholz, Julia, Leahy, Timothy Ronan, van Montfrans, Joris, Nademi, Zohreh, Nelken, Brigitte, Parikh, Suhag, Plado, Silvi, Ramakers, Jan, Redlich, Antje, Rieux-Laucat, Frédéric, Rivière, Jacques G., Rodina, Yulia, Júnior, Pérsio Roxo, Salou, Sarah, Schuetz, Catharina, Shcherbina, Anna, Slatter, Mary A., Touzot, Fabien, Unal, Ekrem, Lankester, Arjan C., Burns, Siobhan, Seppänen, Mikko R.J., Neth, Olaf, Albert, Michael H., Ehl, Stephan, Neven, Bénédicte, and Speckmann, Carsten

Published
2024
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10. Outcome of haematopoietic cell transplantation in children with lysosomal acid lipase deficiency: a study on behalf of the EBMT Inborn Errors Working Party

Author

Lum, Su Han, Minkov, Milen, Jones, Simon A., Hazelaar, Sheree, Sirait, Tiarlan, Potter, Jane E., Stepensky, Polina, Garban, Frederic, Pichler, Herbert, Stein, Jerry, Kaya, Zuhre, Schulz, Ansgar, Mellgren, Karin, Diaz de Heredia, Cristina, Pochon, Cecile, Riesco, Susana, Diaz, Miguel Angel, Michel, Gérard, Lindemans, Caroline, Gruhn, Bernd, Albert, Michael H., Lankester, Arjan C., Neven, Bénédicte, and Wynn, Robert

Published
2023
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13. 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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14. Indications for haematopoietic cell transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe, 2022

Author

Snowden, John A., Sánchez-Ortega, Isabel, Corbacioglu, Selim, Basak, Grzegorz W., Chabannon, Christian, de la Camara, Rafael, Dolstra, Harry, Duarte, Rafael F., Glass, Bertram, Greco, Raffaella, Lankester, Arjan C., Mohty, Mohamad, Neven, Bénédicte, de Latour, Régis Peffault, Pedrazzoli, Paolo, Peric, Zinaida, Yakoub-Agha, Ibrahim, Sureda, Anna, and Kröger, Nicolaus

Published
2022
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16. Phenotype, genotype, treatment, and survival outcomes in patients with X-linked inhibitor of apoptosis deficiency

Author

Sediva, Anna, Neven, Benedicte, Hauck, Fabian, Warnatz, Klaus, Pac, Malgorzata, Carrabba, Maria, Palacin, Pere, Jandus, Peter, Gardulf, Ann, Mahlaoui, Nizar, Pergent, Martine, Schutz, Catharina, Sharapova, Svetlana, Vassilios, Lougaris, Candotti, Fabio, Volpi, Stephano, Yang, Linlin, Booth, Claire, Speckmann, Carsten, Seidel, Markus G., Worth, Austen J.J., Kindle, Gerhard, Lankester, Arjan C., Grimbacher, Bodo, Gennery, Andrew R., Seppanen, Mikko R.J., Morris, Emma C., and Burns, Siobhan O.

Published
2022
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17. 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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18. Allogeneic hematopoietic stem cell transplantation for adult HLH: a retrospective study by the chronic malignancies and inborn errors working parties of EBMT

Author

Machowicz, Rafal, Suarez, Felipe, Wiktor-Jedrzejczak, Wieslaw, Eikema, Diderik-Jan, de Wreede, Liesbeth C., Blok, Henric-Jan, Isaksson, Cecilia, Einsele, Hermann, Poiré, Xavier, van Dorp, Suzanne, Nikolousis, Emmanouil, Johansson, Jan-Erik, Kobbe, Guido, Zecca, Marco, Arnold, Renate, Gerbitz, Armin, Finke, Jürgen, Díez-Martín, Jose Luis, Bonifazi, Francesca, McQuaker, Grant, Lenhoff, Stig, Rohrlich, Pierre-Simon, Theobald, Matthias, Ljungman, Per, Collin, Matthew, Albert, Michael H., Ehninger, Gerhard, Carlson, Kristina, Halaburda, Kazimierz, Lehmberg, Kai, Schönland, Stefan, Yakoub-Agha, Ibrahim, Gennery, Andrew R., Lankester, Arjan C., and Kröger, Nicolaus

Published
2022
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19. 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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23. Mechanisms of genotype-phenotype correlation in autosomal dominant anhidrotic ectodermal dysplasia with immune deficiency

Author

Petersheim, Daniel, Massaad, Michel J, Lee, Saetbyul, Scarselli, Alessia, Cancrini, Caterina, Moriya, Kunihiko, Sasahara, Yoji, Lankester, Arjan C, Dorsey, Morna, Di Giovanni, Daniela, Bezrodnik, Liliana, Ohnishi, Hidenori, Nishikomori, Ryuta, Tanita, Kay, Kanegane, Hirokazu, Morio, Tomohiro, Gelfand, Erwin W, Jain, Ashish, Secord, Elizabeth, Picard, Capucine, Casanova, Jean-Laurent, Albert, Michael H, Torgerson, Troy R, and Geha, Raif S

Subjects
Genetics, Aetiology, 2.1 Biological and endogenous factors, Carrier Proteins, Chemokine CCL20, Ectodermal Dysplasia, Genetic Diseases, X-Linked, Genotype, HEK293 Cells, Humans, Immunologic Deficiency Syndromes, Intercellular Adhesion Molecule-1, Interleukin-6, NF-KappaB Inhibitor alpha, NF-kappa B p52 Subunit, Point Mutation, Primary Immunodeficiency Diseases, Proto-Oncogene Mas, Signal Transduction, Ectodermal dysplasia with immune deficiency, NF-kappa B inhibitor alpha, canonical nuclear factor kappa B pathway, noncanonical nuclear factor kappa B pathway, lymphorganogenesis, hematopoietic stem cell transplantation, NF-κB inhibitor α, canonical nuclear factor κB pathway, noncanonical nuclear factor κB pathway, Immunology, Allergy
Abstract

BackgroundAutosomal dominant anhidrotic ectodermal dysplasia with immune deficiency (AD EDA-ID) is caused by heterozygous point mutations at or close to serine 32 and serine 36 or N-terminal truncations in IκBα that impair its phosphorylation and degradation and thus activation of the canonical nuclear factor κ light chain enhancer of activated B cells (NF-κB) pathway. The outcome of hematopoietic stem cell transplantation is poor in patients with AD EDA-ID despite achievement of chimerism. Mice heterozygous for the serine 32I mutation in IκBα have impaired noncanonical NF-κB activity and defective lymphorganogenesis.ObjectiveWe sought to establish genotype-phenotype correlation in patients with AD EDA-ID.MethodsA disease severity scoring system was devised. Stability of IκBα mutants was examined in transfected cells. Immunologic, biochemical, and gene expression analyses were performed to evaluate canonical and noncanonical NF-κB signaling in skin-derived fibroblasts.ResultsDisease severity was greater in patients with IκBα point mutations than in those with truncation mutations. IκBα point mutants were expressed at significantly higher levels in transfectants compared with truncation mutants. Canonical NF-κB-dependent IL-6 secretion and upregulation of the NF-κB subunit 2/p100 and RELB proto-oncogene, NF-κB subunit (RelB) components of the noncanonical NF-κB pathway were diminished significantly more in patients with point mutations compared with those with truncations. Noncanonical NF-κB-driven generation of the transcriptionally active p100 cleavage product p52 and upregulation of CCL20, intercellular adhesion molecule 1 (ICAM1), and vascular cell adhesion molecule 1 (VCAM1), which are important for lymphorganogenesis, were diminished significantly more in LPS plus α-lymphotoxin β receptor-stimulated fibroblasts from patients with point mutations compared with those with truncations.ConclusionsIκBα point mutants accumulate at higher levels compared with truncation mutants and are associated with more severe disease and greater impairment of canonical and noncanonical NF-κB activity in patients with AD EDA-ID.

Published
2018

27. Extended clinical and immunological phenotype and transplant outcome in CD27 and CD70 deficiency

Author

Ghosh, Sujal, Köstel Bal, Sevgi, Edwards, Emily S.J., Pillay, Bethany, Jiménez Heredia, Raúl, Erol Cipe, Funda, Rao, Geetha, Salzer, Elisabeth, Zoghi, Samaneh, Abolhassani, Hassan, Momen, Tooba, Gostick, Emma, Price, David A., Zhang, Yu, Oler, Andrew J., Gonzaga-Jauregui, Claudia, Erman, Baran, Metin, Ayse, Ilhan, Inci, Haskologlu, Sule, Islamoglu, Candan, Baskin, Kubra, Ceylaner, Serdar, Yilmaz, Ebru, Unal, Ekrem, Karakukcu, Musa, Berghuis, Dagmar, Cole, Theresa, Gupta, Aditya K., Hauck, Fabian, Kogler, Hubert, Hoepelman, Andy I.M., Baris, Safa, Karakoc-Aydiner, Elif, Ozen, Ahmet, Kager, Leo, Holzinger, Dirk, Paulussen, Michael, Krüger, Renate, Meisel, Roland, Oommen, Prasad T., Morris, Emma, Neven, Benedicte, Worth, Austen, van Montfrans, Joris, Fraaij, Pieter L.A., Choo, Sharon, Dogu, Figen, Davies, E. Graham, Burns, Siobhan, Dückers, Gregor, Becker, Ruy Perez, von Bernuth, Horst, Latour, Sylvain, Faraci, Maura, Gattorno, Marco, Su, Helen C., Pan-Hammarström, Qiang, Hammarström, Lennart, Lenardo, Michael J., Ma, Cindy S., Niehues, Tim, Aghamohammadi, Asghar, Rezaei, Nima, Ikinciogullari, Aydan, Tangye, Stuart G., Lankester, Arjan C., and Boztug, Kaan

Published
2020
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28. Proceeding of the European Group for Blood and Marrow Transplantation (EBMT) congress on sickle cell disease, 16–17 may 2019, Regensburg, Germany: What is the impact of antithymocyte globulin pharmacokinetics on haploidentical hematopoietic stem cell transplantation?

Author

Oostenbrink, Lisa V.E., Jol-van der Zijde, Cornelia M., Jansen-Hoogendijk, Anja M., Pool, Emma S., van Halteren, Astrid G.S., Moes, Dirk Jan A.R., Bredius, Robbert G.M., Mohseny, Alex B., Smiers, Frans J.W., van Tol, Maarten J.D., Schilham, Marco W., and Lankester, Arjan C.

Published
2020
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29. 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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30. Anti-T-lymphocyte globulin exposure is associated with acute graft-versus-host disease and relapse in pediatric acute lymphoblastic leukemia patients undergoing hematopoietic stem cell transplantation: a multinational prospective study

Author

Oostenbrink, Lisa V.E., primary, Von Asmuth, Erik G.J., additional, Jol-van der Zijde, Cornelia M., additional, Jansen-Hoogendijk, Anja M., additional, Vervat, Carly, additional, Bredius, Robbert G.M., additional, Van Tol, Maarten J.D., additional, Schilham, Marco W., additional, Sedlacek, Petr, additional, Ifversen, Marianne, additional, Balduzzi, Adriana, additional, Bader, Peter, additional, Peters, Christina, additional, Moes, Dirk Jan A.R., additional, and Lankester, Arjan C., additional

Published
2024
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31. Outcomes of HLA-mismatched HSCT with TCRαβ/CD19 depletion or post-HSCT cyclophosphamide for inborn errors of immunity

Author

Lum, Su Han, primary, Albert, Michael H, additional, Gilbert, Patrick, additional, Sirait, Tiarlan, additional, Algeri, Mattia, additional, Muratori, Rafaella, additional, Fournier, Benjamin, additional, Laberko, Alexandra, additional, Karakukcu, Musa, additional, Unal, Ekrem, additional, Ayas, Mouhab F, additional, Yadav, Satya Prakash, additional, Fisgin, Tunc, additional, Elfeky, Reem, additional, Fernandes, Juliana Folloni, additional, Faraci, Maura, additional, Cole, Theresa, additional, Schulz, Ansgar S, additional, Meisel, Roland, additional, Zecca, Marco, additional, Ifversen, Marianne, additional, Biffi, Alessandra, additional, Diana, Jean-Sebastien, additional, Vallée, Tanja C., additional, Giardino, Stefano, additional, Ersoy, Gizem Zengin, additional, Moshous, Despina, additional, Gennery, Andrew R, additional, Balashov, Dmitry, additional, Bonfim, Carmem M.S., additional, Locatelli, Franco, additional, Lankester, Arjan C, additional, Neven, Bénédicte, additional, and Slatter, Mary A, additional

Published
2024
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34. 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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35. Anti-T-lymphocyte globulin exposure is associated with acute graft-versus-host disease and relapse in pediatric acute lymphoblastic leukemia patients undergoing hematopoietic stem cell transplantation: a multinational prospective study

Author

Oostenbrink, L, Von Asmuth, E, Jol-van der Zijde, C, Jansen-Hoogendijk, A, Vervat, C, Bredius, R, Van Tol, M, Schilham, M, Sedlacek, P, Ifversen, M, Balduzzi, A, Bader, P, Peters, C, Moes, D, Lankester, A, Oostenbrink, Lisa V. E., Von Asmuth, Erik G. J., Jol-van der Zijde, Cornelia M., Jansen-Hoogendijk, Anja M., Vervat, Carly, Bredius, Robbert G. M., Van Tol, Maarten J. D., Schilham, Marco W., Sedlacek, Petr, Ifversen, Marianne, Balduzzi, Adriana, Bader, Peter, Peters, Christina, Moes, Dirk Jan A. R., Lankester, Arjan C., Oostenbrink, L, Von Asmuth, E, Jol-van der Zijde, C, Jansen-Hoogendijk, A, Vervat, C, Bredius, R, Van Tol, M, Schilham, M, Sedlacek, P, Ifversen, M, Balduzzi, A, Bader, P, Peters, C, Moes, D, Lankester, A, Oostenbrink, Lisa V. E., Von Asmuth, Erik G. J., Jol-van der Zijde, Cornelia M., Jansen-Hoogendijk, Anja M., Vervat, Carly, Bredius, Robbert G. M., Van Tol, Maarten J. D., Schilham, Marco W., Sedlacek, Petr, Ifversen, Marianne, Balduzzi, Adriana, Bader, Peter, Peters, Christina, Moes, Dirk Jan A. R., and Lankester, Arjan C.

Abstract

Anti T-lymphocyte globulin (ATLG) is used in hematopoietic stem cell transplantation (HSCT) to prevent graft-versus-host disease (GvHD) and graft failure. To date, insight in ATLG pharmacokinetics and -dynamics (PK/PD) is limited, and population PK (POPPK) models are lacking. In this prospective study, we describe ATLG POPPK using NONMEM® and the impact of ATLG exposure on clinical outcome and immune reconstitution in a homogeneous cohort of pediatric acute lymphoblastic leukemia (ALL) patients transplanted with a matched unrelated donor and receiving uniform ATLG dosing. Based on 121 patients and 812 samples for POPPK analysis, a two-compartmental model with parallel linear and non-linear clearance and bodyweight as covariate, best described the ATLG concentrationtime data. The level of ATLG exposure (day active ATLG day 16 8.2%, p.

Published
2024

36. JAK inhibitor treatment for inborn errors of JAK/STAT signaling: An ESID/EBMT-IEWP retrospective study

Author

PMC Medisch specialisten, Cluster B, Immuno/reuma patientenzorg, Child Health, Infection & Immunity, Fischer, Marco, Olbrich, Peter, Hadjadj, Jérôme, Aumann, Volker, Bakhtiar, Shahrzad, Barlogis, Vincent, von Bismarck, Philipp, Bloomfield, Markéta, Booth, Claire, Buddingh, Emmeline P., Cagdas, Deniz, Castelle, Martin, Chan, Alice Y., Chandrakasan, Shanmuganathan, Chetty, Kritika, Cougoul, Pierre, Crickx, Etienne, Dara, Jasmeen, Deyà-Martínez, Angela, Farmand, Susan, Formankova, Renata, Gennery, Andrew R., Gonzalez-Granado, Luis Ignacio, Hagin, David, Hanitsch, Leif Gunnar, Hanzlikovà, Jana, Hauck, Fabian, Ivorra-Cortés, José, Kisand, Kai, Kiykim, Ayca, Körholz, Julia, Leahy, Timothy Ronan, van Montfrans, Joris, Nademi, Zohreh, Nelken, Brigitte, Parikh, Suhag, Plado, Silvi, Ramakers, Jan, Redlich, Antje, Rieux-Laucat, Frédéric, Rivière, Jacques G., Rodina, Yulia, Júnior, Pérsio Roxo, Salou, Sarah, Schuetz, Catharina, Shcherbina, Anna, Slatter, Mary A., Touzot, Fabien, Unal, Ekrem, Lankester, Arjan C., Burns, Siobhan, Seppänen, Mikko R.J., Neth, Olaf, Albert, Michael H., Ehl, Stephan, Neven, Bénédicte, Speckmann, Carsten, PMC Medisch specialisten, Cluster B, Immuno/reuma patientenzorg, Child Health, Infection & Immunity, Fischer, Marco, Olbrich, Peter, Hadjadj, Jérôme, Aumann, Volker, Bakhtiar, Shahrzad, Barlogis, Vincent, von Bismarck, Philipp, Bloomfield, Markéta, Booth, Claire, Buddingh, Emmeline P., Cagdas, Deniz, Castelle, Martin, Chan, Alice Y., Chandrakasan, Shanmuganathan, Chetty, Kritika, Cougoul, Pierre, Crickx, Etienne, Dara, Jasmeen, Deyà-Martínez, Angela, Farmand, Susan, Formankova, Renata, Gennery, Andrew R., Gonzalez-Granado, Luis Ignacio, Hagin, David, Hanitsch, Leif Gunnar, Hanzlikovà, Jana, Hauck, Fabian, Ivorra-Cortés, José, Kisand, Kai, Kiykim, Ayca, Körholz, Julia, Leahy, Timothy Ronan, van Montfrans, Joris, Nademi, Zohreh, Nelken, Brigitte, Parikh, Suhag, Plado, Silvi, Ramakers, Jan, Redlich, Antje, Rieux-Laucat, Frédéric, Rivière, Jacques G., Rodina, Yulia, Júnior, Pérsio Roxo, Salou, Sarah, Schuetz, Catharina, Shcherbina, Anna, Slatter, Mary A., Touzot, Fabien, Unal, Ekrem, Lankester, Arjan C., Burns, Siobhan, Seppänen, Mikko R.J., Neth, Olaf, Albert, Michael H., Ehl, Stephan, Neven, Bénédicte, and Speckmann, Carsten

Published
2024

38. Solid organ transplantation after hematopoietic stem cell transplantation in childhood: A multicentric retrospective survey

Author

Faraci, Maura, Bertaina, Alice, Dalissier, Arnaud, Ifversen, Marianne, Schulz, Ansgar, Gennery, Andrew, Burkhardt, Birgit, Badell Serra, Isabel, Diaz-de-Heredia, Cristina, Lanino, Edoardo, Lankester, Arjan C., Gruhn, Bernd, Matthes-Martin, Susanne, Kühl, Joern S., Varotto, Stefania, Paillard, Catherine, Guilmatre, Audrey, Sastre, Ana, Abecasis, Manuel, Garwer, Birgit, Sedlacek, Petr, Boelens, Jaap J., Beohou, Eric, and Bader, Peter

Published
2019
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40. Hematopoietic stem cell transplantation for CD40 ligand deficiency: Results from an EBMT/ESID-IEWP-SCETIDE-PIDTC study

Author

Ferrua, Francesca, Galimberti, Stefania, Courteille, Virginie, Slatter, Mary Anne, Booth, Claire, Moshous, Despina, Neven, Benedicte, Blanche, Stephane, Cavazzana, Marina, Laberko, Alexandra, Shcherbina, Anna, Balashov, Dmitry, Soncini, Elena, Porta, Fulvio, Al-Mousa, Hamoud, Al-Saud, Bandar, Al-Dhekri, Hasan, Arnaout, Rand, Formankova, Renata, Bertrand, Yves, Lange, Andrzej, Smart, Joanne, Wolska-Kusnierz, Beata, Aquino, Victor M., Dvorak, Christopher C., Fasth, Anders, Fouyssac, Fanny, Heilmann, Carsten, Hoenig, Manfred, Schuetz, Catharina, Kelečić, Jadranka, Bredius, Robbert G.M., Lankester, Arjan C., Lindemans, Caroline A., Suarez, Felipe, Sullivan, Kathleen E., Albert, Michael H., Kałwak, Krzysztof, Barlogis, Vincent, Bhatia, Monica, Bordon, Victoria, Czogala, Wojciech, Alonso, Laura, Dogu, Figen, Gozdzik, Jolanta, Ikinciogullari, Aydan, Kriván, Gergely, Ljungman, Per, Meyts, Isabelle, Mustillo, Peter, Smith, Angela R., Speckmann, Carsten, Sundin, Mikael, Keogh, Steven John, Shaw, Peter John, Boelens, Jaap Jan, Schulz, Ansgar S., Sedlacek, Petr, Veys, Paul, Mahlaoui, Nizar, Janda, Ales, Davies, E. Graham, Fischer, Alain, Cowan, Morton J., and Gennery, Andrew Richard

Published
2019
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42. 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

43. Potential role of B- and NK-cells in the pathogenesis of pediatric aplastic anemia through deep phenotyping.

Author

Vissers, Lotte T. W., van Ostaijen-ten Dam, Monique M., Melsen, Janine E., van der Spek, Yanna M., Kemna, Koen P., Lankester, Arjan C., van der Burg, Mirjam, and Mohseny, Alexander B.

Subjects
KILLER cells, HEMATOPOIETIC stem cell transplantation, CHILD patients, HEMATOPOIETIC stem cells, BONE marrow
Abstract

Introduction: Pediatric patients with unexplained bone marrow failure (BMF) are often categorized as aplastic anemia (AA). Based on the accepted hypothesis of an auto-immune mechanism underlying AA, immune suppressive therapy (IST) might be effective. However, due to the lack of diagnostic tools to identify immune AA and prognostic markers to predict IST response together with the unequaled curative potential of hematopoietic stem cell transplantation (HSCT), most pediatric severe AA patients are momentarily treated by HSCT if available. Although several studies indicate oligoclonal T-cells with cytotoxic activities towards the hematopoietic stem cells, increasing evidence points towards defective inhibitory mechanisms failing to inhibit auto-reactive T-cells. Methods: We aimed to investigate the role of NK- and B-cells in seven pediatric AA patients through a comprehensive analysis of paired bone marrow and peripheral blood samples with spectral flow cytometry in comparison to healthy age-matched bone marrow donors. Results: We observed a reduced absolute number of NK-cells in peripheral blood of AA patients with a skewed distribution towards CD56bright NK-cells in a subgroup of patients. The enriched CD56bright NK-cells had a lower expression of CD45RA and TIGIT and a higher expression of CD16, compared to healthy donors. Functional analysis revealed no differences in degranulation. However, IFN-g production and perforin expression of NK-cells were reduced in the CD56bright-enriched patient group. The diminished NK-cell function in this subgroup might underly the auto-immunity. Importantly, NK-function of AA patients with reduced CD56bright NK-cells was comparable to healthy donors. Also, B-cell counts were lower in AA patients. Subset analysis revealed a trend towards reduction of transitional B-cells in both absolute and relative numbers compared to healthy controls. As these cells were previously hypothesized as regulatory cells in AA, decreased numbers might be involved in defective inhibition of auto-reactive T-cells. Interestingly, even in patients with normal distribution of precursor B-cells, the transitional compartment was reduced, indicating partial differentiation failure from immature to transitional B-cells or a selective loss. Discussion: Our findings provide a base for future studies to unravel the role of transitional B-cells and CD56bright NK-cells in larger cohorts of pediatric AA patients as diagnostic markers for immune AA and targets for therapeutic interventions. [ABSTRACT FROM AUTHOR]

Published
2024
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44. Case report: Persistent hypogammaglobulinemia and mixed chimerism after HLA class-II disparate-hematopoietic stem cell transplant.

Author

de Gier, Melanie, Pico-Knijnenburg, Ingrid, van Ostaijen-ten Dam, Monique M., Berghuis, Dagmar, Smiers, Frans J., van Beek, Adriaan A., Jolink, Hetty, Jansen, Patty M., Lankester, Arjan C., and van der Burg, Mirjam

Subjects
STEM cell transplantation, CHIMERISM, HEMATOPOIETIC stem cell transplantation, AGAMMAGLOBULINEMIA, T helper cells
Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative treatment for various hematological, immunological and metabolic diseases, replacing the patient's hematopoietic system with donor-derived healthy hematopoietic stem cells. HSCT can be complicated by early and late events related to impaired immunological recovery such as prolonged hypogammaglobulinemia post- HSCT. We present a 16-year-old female patient with sickle-cell disease who underwent HSCT with stem cells from a human leukocyte antigen (HLA) class-II mismatched family donor.While cellular recoverywas good post-HSCT, the patient developed mixed chimerism and suffered from cervical lymphadenopathy, recurrent airway infections and cutaneous SLE. She presented with hypogammaglobulinemia and was started on immunoglobulin substitution therapy and antibiotic prophylaxis. B-cell phenotyping showed that she had increased transitional and naïve mature B cells, reduced memory B cells, and diminished marginal zone/natural effector cells. In-depth immunophenotyping and B-cell receptor repertoire sequencing ruled out an intrinsic B-cell defect by expression of activation-induced cytidine deaminase (AID), presence of somatic hypermutationsanddifferentiationintoIgG-andIgA-producingplasmacells in vitro. Immunohistochemistry and flow cytometry of lymph node tissue showed a clear block in terminal B-cell differentiation. Chimerism analysis of sorted lymph node populations showed that exclusively patient-derived B cells populated germinal centers, while only a minor fraction of follicular helper T cells was patient-derived. Given this discrepancy, we deduced that the HLA class-II disparity between patient and donor likely hinders terminal B-cell differentiation in the lymph node. This case highlights that studying disturbed cognate T-B interactions in the secondary lymphoid organs can provide unique insights when deciphering prolonged hypogammaglobulinemia post-HSCT. [ABSTRACT FROM AUTHOR]

Published
2024
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45. Restoration of T and B Cell Differentiation after RAG1 Gene Transfer in Human RAG1 Defective Hematopoietic Stem Cells.

Author

Sorel, Nataël, Díaz-Pascual, Francisco, Bessot, Boris, Sadek, Hanem, Mollet, Chloé, Chouteau, Myriam, Zahn, Marco, Gil-Farina, Irene, Tajer, Parisa, van Eggermond, Marja, Berghuis, Dagmar, Lankester, Arjan C., André, Isabelle, Gabriel, Richard, Cavazzana, Marina, Pike-Overzet, Kasrin, Staal, Frank J. T., and Lagresle-Peyrou, Chantal

Subjects
B cell differentiation, SEVERE combined immunodeficiency, T cell receptors, T cell differentiation, HEMATOPOIETIC stem cells
Abstract

Recombinase-activating gene (RAG)-deficient SCID patients lack B and T lymphocytes due to the inability to rearrange immunoglobulin and T cell receptor genes. The two RAG genes act as a required dimer to initiate gene recombination. Gene therapy is a valid treatment alternative for RAG-SCID patients who lack a suitable bone marrow donor, but developing such therapy for RAG1/2 has proven challenging. Using a clinically approved lentiviral vector with a codon-optimized RAG1 gene, we report here preclinical studies using CD34+ cells from four RAG1-SCID patients. We used in vitro T cell developmental assays and in vivo assays in xenografted NSG mice. The RAG1-SCID patient CD34+ cells transduced with the RAG1 vector and transplanted into NSG mice led to restored human B and T cell development. Together with favorable safety data on integration sites, these results substantiate an ongoing phase I/II clinical trial for RAG1-SCID. [ABSTRACT FROM AUTHOR]

Published
2024
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47. Hematopoietic Stem Cell Transplantation for Hepatitis-associated Aplastic Anemia Following Liver Transplantation for Nonviral Hepatitis: 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

Mohseny, Alexander B., Eikema, Dirk-Jan A., Neven, Benedicte, Kröger, Nicolaus, Shaw, Peter J., Damaj, Ghandi, Dalle, Jean-Hugues, Bosman, Paul V., Delehaye, Fanny, Lankester, Arjan C., Smiers, Frans J., and Peffault de Latour, Regis

Published
2020
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50. Impact of Treosulfan Exposure on Early and Long-Term Clinical Outcomes in Pediatric Allogeneic Hematopoietic Stem Cell Transplantation Recipients: A Prospective Multicenter Study

Author

van der Stoep, M.Y. Eileen C., Bertaina, Alice, Moes, Dirk Jan A.R., Algeri, Mattia, Bredius, Robbert G.M., Smiers, Frans J.W., Berghuis, Dagmar, Buddingh, Emilie P., Mohseny, Alexander B., Guchelaar, Henk-Jan, Locatelli, Franco, Zwaveling, Juliette, and Lankester, Arjan C.

Published
2022
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