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645 results on '"Bredius, Robbert"'

1. 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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2. Clinical Symptoms, Laboratory Parameters and Long-Term Follow-up in a National DADA2 Cohort

Author

Andriessen, Marie Valérie E., Legger, G. Elizabeth, Bredius, Robbert G. M., van Gijn, Marielle E., Hak, A. Elisabeth, Muller, Petra C. E. Hissink, Kamphuis, Sylvia, Klouwer, Femke C. C., Kuijpers, Taco W., Leavis, Helen L., Nierkens, Stefan, Rutgers, Abraham, van der Veken, Lars T., van Well, Gijs T. J., Mulders-Manders, Catharina M., and van Montfrans, Joris M.

Published
2023
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4. Outcomes and Treatment Strategies for Autoimmunity and Hyperinflammation in Patients with RAG Deficiency

Author

Farmer, Jocelyn R, Foldvari, Zsofia, Ujhazi, Boglarka, De Ravin, Suk See, Chen, Karin, Bleesing, Jack JH, Schuetz, Catharina, Al-Herz, Waleed, Abraham, Roshini S, Joshi, Avni Y, Costa-Carvalho, Beatriz T, Buchbinder, David, Booth, Claire, Reiff, Andreas, Ferguson, Polly J, Aghamohammadi, Asghar, Abolhassani, Hassan, Puck, Jennifer M, Adeli, Mehdi, Cancrini, Caterina, Palma, Paolo, Bertaina, Alice, Locatelli, Franco, Di Matteo, Gigliola, Geha, Raif S, Kanariou, Maria G, Lycopoulou, Lilia, Tzanoudaki, Marianna, Sleasman, John W, Parikh, Suhag, Pinero, Gloria, Fischer, Bernard M, Dbaibo, Ghassan, Unal, Ekrem, Patiroglu, Turkan, Karakukcu, Musa, Al-Saad, Khulood Khalifa, Dilley, Meredith A, Pai, Sung-Yun, Dutmer, Cullen M, Gelfand, Erwin W, Geier, Christoph B, Eibl, Martha M, Wolf, Hermann M, Henderson, Lauren A, Hazen, Melissa M, Bonfim, Carmem, Wolska-Kuśnierz, Beata, Butte, Manish J, Hernandez, Joseph D, Nicholas, Sarah K, Stepensky, Polina, Chandrakasan, Shanmuganathan, Miano, Maurizio, Westermann-Clark, Emma, Goda, Vera, Kriván, Gergely, Holland, Steven M, Fadugba, Olajumoke, Henrickson, Sarah E, Ozen, Ahmet, Karakoc-Aydiner, Elif, Baris, Safa, Kiykim, Ayca, Bredius, Robbert, Hoeger, Birgit, Boztug, Kaan, Pashchenko, Olga, Neven, Benedicte, Moshous, Despina, Villartay, Jean-Pierre de, Bousfiha, Ahmed Aziz, Hill, Harry R, Notarangelo, Luigi D, and Walter, Jolan E

Subjects
Autoimmune Disease, Genetics, Hematology, Inflammatory and immune system, Adolescent, Adult, Autoimmunity, Child, Child, Preschool, Female, Hematopoietic Stem Cell Transplantation, Homeodomain Proteins, Humans, Immunologic Deficiency Syndromes, Immunosuppressive Agents, Infant, Inflammation, Male, Middle Aged, Treatment Outcome, Young Adult, Recombination activating gene, Severe combined immunodeficiency, Immune dysregulation, Autoimmune cytopenias, Hematopoietic stem cell transplantation
Abstract

BACKGROUND:Although autoimmunity and hyperinflammation secondary to recombination activating gene (RAG) deficiency have been associated with delayed diagnosis and even death, our current understanding is limited primarily to small case series. OBJECTIVE:Understand the frequency, severity, and treatment responsiveness of autoimmunity and hyperinflammation in RAG deficiency. METHODS:In reviewing the literature and our own database, we identified 85 patients with RAG deficiency, reported between 2001 and 2016, and compiled the largest case series to date of 63 patients with prominent autoimmune and/or hyperinflammatory pathology. RESULTS:Diagnosis of RAG deficiency was delayed a median of 5 years from the first clinical signs of immune dysregulation. Most patients (55.6%) presented with more than 1 autoimmune or hyperinflammatory complication, with the most common etiologies being cytopenias (84.1%), granulomas (23.8%), and inflammatory skin disorders (19.0%). Infections, including live viral vaccinations, closely preceded the onset of autoimmunity in 28.6% of cases. Autoimmune cytopenias had early onset (median, 1.9, 2.1, and 2.6 years for autoimmune hemolytic anemia, immune thrombocytopenia, and autoimmune neutropenia, respectively) and were refractory to intravenous immunoglobulin, steroids, and rituximab in most cases (64.7%, 73.7%, and 71.4% for autoimmune hemolytic anemia, immune thrombocytopenia, and autoimmune neutropenia, respectively). Evans syndrome specifically was associated with lack of response to first-line therapy. Treatment-refractory autoimmunity/hyperinflammation prompted hematopoietic stem cell transplantation in 20 patients. CONCLUSIONS:Autoimmunity/hyperinflammation can be a presenting sign of RAG deficiency and should prompt further evaluation. Multilineage cytopenias are often refractory to immunosuppressive treatment and may require hematopoietic cell transplantation for definitive management.

Published
2019

7. 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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8. 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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9. Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study.

Author

Barzaghi, Federica, Amaya Hernandez, Laura Cristina, Neven, Benedicte, Ricci, Silvia, Kucuk, Zeynep Yesim, Bleesing, Jack J, Nademi, Zohreh, Slatter, Mary Anne, Ulloa, Erlinda Rose, Shcherbina, Anna, Roppelt, Anna, Worth, Austen, Silva, Juliana, Aiuti, Alessandro, Murguia-Favela, Luis, Speckmann, Carsten, Carneiro-Sampaio, Magda, Fernandes, Juliana Folloni, Baris, Safa, Ozen, Ahmet, Karakoc-Aydiner, Elif, Kiykim, Ayca, Schulz, Ansgar, Steinmann, Sandra, Notarangelo, Lucia Dora, Gambineri, Eleonora, Lionetti, Paolo, Shearer, William Thomas, Forbes, Lisa R, Martinez, Caridad, Moshous, Despina, Blanche, Stephane, Fisher, Alain, Ruemmele, Frank M, Tissandier, Come, Ouachee-Chardin, Marie, Rieux-Laucat, Frédéric, Cavazzana, Marina, Qasim, Waseem, Lucarelli, Barbarella, Albert, Michael H, Kobayashi, Ichiro, Alonso, Laura, Diaz De Heredia, Cristina, Kanegane, Hirokazu, Lawitschka, Anita, Seo, Jong Jin, Gonzalez-Vicent, Marta, Diaz, Miguel Angel, Goyal, Rakesh Kumar, Sauer, Martin G, Yesilipek, Akif, Kim, Minsoo, Yilmaz-Demirdag, Yesim, Bhatia, Monica, Khlevner, Julie, Richmond Padilla, Erick J, Martino, Silvana, Montin, Davide, Neth, Olaf, Molinos-Quintana, Agueda, Valverde-Fernandez, Justo, Broides, Arnon, Pinsk, Vered, Ballauf, Antje, Haerynck, Filomeen, Bordon, Victoria, Dhooge, Catharina, Garcia-Lloret, Maria Laura, Bredius, Robbert G, Kałwak, Krzysztof, Haddad, Elie, Seidel, Markus Gerhard, Duckers, Gregor, Pai, Sung-Yun, Dvorak, Christopher C, Ehl, Stephan, Locatelli, Franco, Goldman, Frederick, Gennery, Andrew Richard, Cowan, Mort J, Roncarolo, Maria-Grazia, Bacchetta, Rosa, and Primary Immune Deficiency Treatment Consortium (PIDTC) and the Inborn Errors Working Party (IEWP) of the European Society for Blood and Marrow Transplantation (EBMT)

Subjects
Primary Immune Deficiency Treatment Consortium (PIDTC) and the Inborn Errors Working Party (IEWP) of the European Society for Blood and Marrow Transplantation, Humans, Genetic Diseases, X-Linked, Diabetes Mellitus, Type 1, Immune System Diseases, Diarrhea, Disease-Free Survival, Hematopoietic Stem Cell Transplantation, Survival Rate, Retrospective Studies, Follow-Up Studies, Mutation, Adolescent, Adult, Child, Child, Preschool, Infant, Female, Male, Forkhead Transcription Factors, Allografts, Immunosuppression Therapy, FOXP3, IPEX, Treg cells, enteropathy, genetic autoimmunity, hematopoietic stem cell transplantation, immunosuppression, neonatal diabetes, primary immune deficiency, rapamycin, Clinical Research, Stem Cell Research, Regenerative Medicine, Pediatric, Genetics, Transplantation, Aetiology, 2.1 Biological and endogenous factors, Immunology, Allergy
Abstract

BackgroundImmunodysregulation polyendocrinopathy enteropathy x-linked (IPEX) syndrome is a monogenic autoimmune disease caused by FOXP3 mutations. Because it is a rare disease, the natural history and response to treatments, including allogeneic hematopoietic stem cell transplantation (HSCT) and immunosuppression (IS), have not been thoroughly examined.ObjectiveThis analysis sought to evaluate disease onset, progression, and long-term outcome of the 2 main treatments in long-term IPEX survivors.MethodsClinical histories of 96 patients with a genetically proven IPEX syndrome were collected from 38 institutions worldwide and retrospectively analyzed. To investigate possible factors suitable to predict the outcome, an organ involvement (OI) scoring system was developed.ResultsWe confirm neonatal onset with enteropathy, type 1 diabetes, and eczema. In addition, we found less common manifestations in delayed onset patients or during disease evolution. There is no correlation between the site of mutation and the disease course or outcome, and the same genotype can present with variable phenotypes. HSCT patients (n = 58) had a median follow-up of 2.7 years (range, 1 week-15 years). Patients receiving chronic IS (n = 34) had a median follow-up of 4 years (range, 2 months-25 years). The overall survival after HSCT was 73.2% (95% CI, 59.4-83.0) and after IS was 65.1% (95% CI, 62.8-95.8). The pretreatment OI score was the only significant predictor of overall survival after transplant (P = .035) but not under IS.ConclusionsPatients receiving chronic IS were hampered by disease recurrence or complications, impacting long-term disease-free survival. When performed in patients with a low OI score, HSCT resulted in disease resolution with better quality of life, independent of age, donor source, or conditioning regimen.

Published
2018

12. Initial presenting manifestations in 16,486 patients with inborn errors of immunity include infections and noninfectious manifestations

Author

Boztug, Kaan, Brunner, Juergen, Demel, Ulrike F., Förster-Waldl, Elisabeth, Gasteiger, Lukas M., Göschl, Lisa, Kojić, Marina, Schroll, Andrea, Seidel, Markus G., Wintergerst, Uwe, Wisgrill, Lukas, Sharapova, Svetlana O., Goffard, Jean-Christophe, Kerre, Tessa, Meyts, Isabelle, Roosens, Fine, Smet, Julie, Haerynck, Filomeen, Eric, Zelimir Pavle, Milenova, Veneta, Gagro, Alenka, Richter, Darko, Chovancova, Zita, Hlavackova, Eva, Litzman, Jiri, Milota, Tomas, Sediva, Anna, Elaziz, Dalia Abd, Alkady, Radwa Salaheldin, El Sayed El Hawary, Rabab, Eldash, Alia S., Galal, Nermeen, Lotfy, Sohilla, Meshaal, Safa S., Reda, Shereen M., Sobh, Ali, Elmarsafy, Aisha, Seppänen, Mikko R.J., Brosselin, Pauline, Courteille, Virginie, De Vergnes, Nathalie, Kracker, Sven, Pergent, Martine, Randrianomenjanahary, Philippe, Ahrenstorf, Gerrit, Albert, Michael H., Ankermann, Tobias, Atschekzei, Faranaz, Baumann, Ulrich, Becker, Benjamin C., Behrends, Uta, Belohradsky, Bernd H., Biegner, Anika-Kerstin, Binder, Nadine, Bode, Sebastian F.N., Boesecke, Christoph, Boetticher, Benedikt, Borte, Michael, Borte, Stephan, Classen, Carl Friedrich, Dirks, Johannes, Dückers, Gregor, El-Helou, Sabine, Ernst, Diana, Fasshauer, Maria, Fecker, Gisela, Felgentreff, Kerstin, Foell, Dirk, Ghosh, Sujal, Girschick, Hermann J., Goldacker, Sigune, Graf, Norbert, Graf, Dagmar, Greil, Johann, Hanitsch, Leif Gunnar, Hauck, Fabian, Heeg, Maximilian, Heine, Sabine I., Henes, Joerg C., Hoenig, Manfred, Holzer, Ursula, Holzinger, Dirk, Horneff, Gerd, Hundsdoerfer, Patrick, Jablonka, Alexandra, Jakoby, Donate, Joean, Oana, Kaiser-Labusch, Petra, Klemann, Christian, Kobbe, Robin, Körholz, Julia, Kramm, Christof M., Krüger, Renate, Landwehr-Kenzel, Sybille, Lehmberg, Kai, Liese, Johannes G., Lippert, Conrad Ferdinand, Maccari, Maria Elena, Masjosthusmann, Katja, Meinhardt, Andrea, Metzler, Markus, Morbach, Henner, Müller, Ingo, Naumann-Bartsch, Nora, Neubert, Jennifer, Niehues, Tim, Peter, Hans-Hartmut, Rieber, Nikolaus, Ritterbusch, Henrike, Rockstroh, Jürgen Kurt, Roesler, Joachim, Schauer, Uwe, Scheible, Raphael, Schmalzing, Marc, Schmidt, Reinhold Ernst, Schneider, Dominik T., Schreiber, Stefan, Schuetz, Catharina, Schulz, Ansgar, Schulze-Koops, Hendrik, Schulze-Sturm, Ulf, Schuster, Volker, Schwaneck, Eva C., Schwarz, Klaus, Schwarze-Zander, Carolynne, Sirin, Mehtap, Skapenko, Alla, Sogkas, Georgios, Sparber-Sauer, Monika, Speckmann, Carsten, Steinmann, Sandra, Stiehler, Sophie, Tenbrock, Klaus, von Bernuth, Horst, Warnatz, Klaus, Wasmuth, Jan-Christian, Weiss, Michael, Witte, Torsten, Wittke, Kirsten, Wittkowski, Helmut, Zeuner, Rainald A., Farmaki, Evangelia, Hatzistilianou, Maria N., Kakkas, Ioannis, Kanariou, Maria G., Kapousouzi, Androniki, Liatsis, Emmanouil, Maggina, Paraskevi, Papadopoulou-Alataki, Efimia, Raptaki, Maria, Speletas, Matthaios, Tantou, Sofia, Goda, Vera, Kriván, Gergely, Marodi, Laszlo, Abolhassani, Hassan, Aghamohammadi, Asghar, Rezaei, Nima, Feighery, Conleth, Leahy, Timothy Ronan, Ryan, Paul, Batzir, Nurit Assia, Garty, Ben Zion, Tamary, Hannah, Aiuti, Alessandro, Amodio, Donato, Azzari, Chiara, Barzaghi, Federica, Baselli, Lucia A., Cancrini, Caterina, Carrabba, Maria, Cazzaniga, Marco, Cesaro, Simone, Chinello, Matteo, Danieli, Maria Giovanna, Dellepiane, Rosa Maria, Fabio, Giovanna, Gambineri, Eleonora, Lodi, Lorenzo, Lougaris, Vassilios, Marasco, Carolina, Martire, Baldassarre, Marzollo, Antonio, Milito, Cinzia, Moschese, Viviana, Pignata, Claudio, Plebani, Alessandro, Porta, Fulvio, Quinti, Isabella, Ricci, Silvia, Soresina, Annarosa, Tommasini, Alberto, Vacca, Angelo, Vanessa, Clementina, Blažienė, Audra, Sitkauskiene, Brigita, Gowin, Ewelina, Heropolitańska-Pliszka, Edyta, Pietrucha, Barbara, Szaflarska, Anna, Więsik-Szewczyk, Ewa, Wolska-Kuśnierz, Beata, Esteves, Isabel, Faria, Emilia, Marques, Laura Hora, Neves, João Farela, Silva, Susana L., Teixeira, Carla, Pereira da Silva, Sara, Capilna, Brindusa Ruxandra, Guseva, Marina N., Shcherbina, Anna, Bobcakova, Anna, Ciznar, Peter, Gabzdilova, Juliana, Jesenak, Milos, Kapustova, Lenka, Orosova, Jaroslava, Petrovicova, Otilia, Raffac, Stefan, Kopač, Peter, Allende, Luis M., Antolí, Arnau, Blanch, Gemma Rocamora, Carbone, Javier, Dieli-Crimi, Romina, Garcia-Prat, Marina, Gil-Herrera, Juana, Gonzalez-Granado, Luis Ignacio, Agulló, Pilar Llobet, Olbrich, Peter, Parra-Martínez, Alba, Paz-Artal, Estela, Pleguezuelo, Daniel E., Rodríguez, Nerea Salmón, Sánchez-Ramón, Silvia, Santos-Pérez, Juan Luis, Solanich, Xavier, Soler-Palacin, Pere, González-Amores, Miriam, Ekwall, Olov, Fasth, Anders, Bitzenhofer-Grüber, Michaela, Candotti, Fabio, Dimitriou, Florentia, Heininger, Ulrich, Holbro, Andreas, Jandus, Peter, Kolios, Antonios G.A., Marschall, Karin, Schmid, Jana Pachlopnik, Posfay-Barbe, Klara M., Prader, Seraina, Reichenbach, Janine, Steiner, Urs C., Trück, Johannes, Bredius, Robbert G., de Kruijf- Bazen, Suzanne, de Vries, Esther, Henriet, Stefanie S.V., Kuijpers, Taco W., Potjewijd, Judith, Rutgers, Abraham, Stol, Kim, van Aerde, Koen J., Van den Berg, J. Merlijn, van de Ven, Annick A.J.M., Montfrans, Jorisvan, Aydemir, Sezin, Baris, Safa, Dogu, Figen, Ikinciogullari, Aydan, Karakoc-Aydiner, Elif, Kilic, Sara S., Kiykim, Ayca, Kökçü Karadağ, Şefika İlknur, Kutukculer, Necil, Ocak, Suheyla, UNAL, Ekrem, Boyarchuk, Oksana, Hilfanova, Anna, Kostyuchenko, Larysa V., Alachkar, Hana, Arkwright, Peter D., Baxendale, Helen E., Bernatoniene, Jolanta, Coulter, Tanya I., Garcez, Tomaz, Goddard, Sarah, Gompels, Mark M., Grigoriadou, Sofia, Herriot, Richard, Herwadkar, Archana, Huissoon, Aarnoud, Ibberson, Lisa, Nademi, Zoreh, Noorani, Sadia, Parvin, Shahnaz, Steele, Cathal Laurence, Thomas, Moira, Waruiru, Catherine, Yong, Patrick F.K., Bourne, Helen, Thalhammer, Julian, Kindle, Gerhard, Nieters, Alexandra, Rusch, Stephan, Fischer, Alain, Grimbacher, Bodo, Edgar, David, Buckland, Matthew, Mahlaoui, Nizar, and Ehl, Stephan

Published
2021
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14. Genetic susceptibility to acute graft versus host disease in pediatric patients undergoing HSCT

Author

Ansari, Marc, Petrykey, Kateryna, Rezgui, Mohamed Aziz, Del Vecchio, Veronica, Cortyl, Jacques, Ameur, Milad, Nava, Tiago, Beaulieu, Patrick, St-Onge, Pascal, Mlakar, Simona Jurkovic, Uppugunduri, Chakradhara Rao S., Théoret, Yves, Bartelink, Imke H., Boelens, Jaap-Jan, Bredius, Robbert G. M., Dalle, Jean-Hugues, Lewis, Victor, Kangarloo, Bill S., Corbacioglu, Selim, Sinnett, Daniel, Bittencourt, Henrique, and Krajinovic, Maja

Published
2021
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15. 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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18. Allogeneic hematopoietic stem cell transplantation in leukocyte adhesion deficiency type I and III

Author

Bakhtiar, Shahrzad, Salzmann-Manrique, Emilia, Blok, Henric-Jan, Eikema, Dirk-Jan, Hazelaar, Sheree, Ayas, Mouhab, Toren, Amos, Goldstein, Gal, Moshous, Despina, Locatelli, Franco, Merli, Pietro, Michel, Gerard, Öztürk, Gülyüz, Schulz, Ansgar, Heilmann, Carsten, Ifversen, Marianne, Wynn, Rob F., Aleinikova, Olga, Bertrand, Yves, Tbakhi, Abdelghani, Veys, Paul, Karakukcu, Musa, Kupesiz, Alphan, Ghavamzadeh, Ardeshir, Handgretinger, Rupert, Unal, Emel, Perez-Martinez, Antonio, Gokce, Muge, Porta, Fulvio, Aksu, Tekin, Karasu, Gülsün, Badell, Isabel, Ljungman, Per, Skorobogatova, Elena, Yesilipek, Akif, Zuckerman, Tsila, Bredius, Robbert R.G., Stepensky, Polina, Shadur, Bella, Slatter, Mary, Gennery, Andrew R., Albert, Michael H., Bader, Peter, and Lankester, Arjan

Published
2021
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19. 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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20. Genetic Susceptibility to Hepatic Sinusoidal Obstruction Syndrome in Pediatric Patients Undergoing Hematopoietic Stem Cell Transplantation

Author

Ansari, Marc, Petrykey, Kateryna, Rezgui, Mohamed Aziz, Del Vecchio, Veronica, Cortyl, Jacques, Ralph, Reginald-Olivier, Nava, Tiago, Beaulieu, Patrick, St-Onge, Pascal, Jurkovic Mlakar, Simona, Huezo-Diaz Curtis, Patricia, Uppugunduri, Chakradhara Rao S., Lesne, Laurence, Théoret, Yves, Chalandon, Yves, Bartelink, Imke H., Boelens, Jaap-Jan, Bredius, Robbert G.M., Dalle, Jean-Hugues, Lewis, Victor, Kangarloo, Bill S., Peters, Christina, Sinnett, Daniel, Bittencourt, Henrique, and Krajinovic, Maja

Published
2020
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21. 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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22. 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

23. 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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24. Immunoglobulin Replacement Therapy Versus Antibiotic Prophylaxis as Treatment for Incomplete Primary Antibody Deficiency

Author

Smits, Bas M., Kleine Budde, Ilona, de Vries, Esther, ten Berge, Ineke J. M., Bredius, Robbert G. M., van Deuren, Marcel, van Dissel, Jaap T., Ellerbroek, Pauline M., van der Flier, Michiel, van Hagen, P. Martin, Nieuwhof, Chris, Rutgers, Bram, Sanders, Lieke E. A. M., Simon, Anna, Kuijpers, Taco W., and van Montfrans, Joris M.

Published
2021
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25. Outcome of Non-hematological Autoimmunity After Hematopoietic Cell Transplantation in Children with Primary Immunodeficiency

Author

Lum, Su Han, Elfeky, Reem, Achini, Federica R., Margarit-Soler, Adriana, Cinicola, Bianca, Perez-Heras, Inigo, Nademi, Zohreh, Flood, Terry, Cheetham, Tim, Worth, Austen, Qasim, Waseem, Amin, Rakesh, Rao, Kanchan, Chiesa, Robert, Bredius, Robbert G. M., Amrolia, Persis, Abinun, Mario, Hambleton, Sophie, Veys, Paul, Gennery, Andrew R., Lankester, Arjan, and Slatter, Mary

Published
2021
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26. 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

29. 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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30. Hematopoietic Stem Cell Transplantation as Treatment for Patients with DOCK8 Deficiency

Author

Aydin, Susanne E., Freeman, Alexandra F., Al-Herz, Waleed, Al-Mousa, Hamoud A., Arnaout, Rand K., Aydin, Roland C., Barlogis, Vincent, Belohradsky, Bernd H., Bonfim, Carmem, Bredius, Robbert G., Chu, Julia I., Ciocarlie, Oana C., Doğu, Figen, Gaspar, Hubert B., Geha, Raif S., Gennery, Andrew R., Hauck, Fabian, Hawwari, Abbas, Hickstein, Dennis D., Hoenig, Manfred, Ikinciogullari, Aydan, Klein, Christoph, Kumar, Ashish, Ifversen, Marianne R.S., Matthes, Susanne, Metin, Ayse, Neven, Benedicte, Pai, Sung-Yun, Parikh, Suhag H., Picard, Capucine, Renner, Ellen D., Sanal, Özden, Schulz, Ansgar S., Schuster, Friedhelm, Shah, Nirali N., Shereck, Evan B., Slatter, Mary A., Su, Helen C., van Montfrans, Joris, Woessmann, Wilhelm, Ziegler, John B., and Albert, Michael H.

Published
2019
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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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33. Effect of weight and maturation on busulfan clearance in infants and small children undergoing hematopoietic cell transplantation.

Author

Pai, Sung-Yun, Pereira, Luis, Bartelink, Imke, Boelens, Jaap, Bredius, Robbert, Wynn, Rob, Cuvelier, Geoff, Shaw, Peter, Slatter, Mary, Dvorak, Christopher, Long-Boyle, Janel, Cowan, Morton, and Savic, Radojka

Subjects
Busulfan, Hematopoietic cell transplantation, Pediatrics, Pharmacokinetics, Antineoplastic Agents, Alkylating, Busulfan, Child, Preschool, Female, Hematopoietic Stem Cell Transplantation, Humans, Infant, Infant, Newborn, Male, Retrospective Studies, Transplantation Conditioning, Transplantation, Autologous
Abstract

Little information is currently available regarding the pharmacokinetics (PK) of busulfan in infants and small children to help guide decisions for safe and efficacious drug therapy. The objective of this study was to develop an algorithm for individualized dosing of i.v. busulfan in infants and children weighing ≤12 kg, that would achieve targeted exposure with the first dose of busulfan. Population PK modeling was conducted using intensive time-concentration data collected through the routine therapeutic drug monitoring of busulfan in 149 patients from 8 centers. Busulfan PK was well described by a 1-compartment base model with linear elimination. The important clinical covariates affecting busulfan PK were actual body weight and age. Based on our model, the predicted clearance of busulfan increases approximately 1.7-fold between 6 weeks to 2 years of life. For infants age

Published
2013

34. Effect of Weight and Maturation on Busulfan Clearance in Infants and Small Children Undergoing Hematopoietic Cell Transplantation

Author

Savic, Radojka M, Cowan, Morton J, Dvorak, Christopher C, Pai, Sung-Yun, Pereira, Luis, Bartelink, Imke H, Boelens, Jaap J, Bredius, Robbert GM, Wynn, Rob F, Cuvelier, Geoff DE, Shaw, Peter J, Slatter, Mary A, and Long-Boyle, Janel

Subjects
Pharmacology and Pharmaceutical Sciences, Biomedical and Clinical Sciences, Transplantation, Pediatric, Perinatal Period - Conditions Originating in Perinatal Period, Clinical Research, Good Health and Well Being, Antineoplastic Agents, Alkylating, Busulfan, Child, Preschool, Female, Hematopoietic Stem Cell Transplantation, Humans, Infant, Infant, Newborn, Male, Retrospective Studies, Transplantation Conditioning, Transplantation, Autologous, Hematopoietic cell transplantation, Pharmacokinetics, Pediatrics, Clinical Sciences, Immunology, Cardiovascular medicine and haematology
Abstract

Little information is currently available regarding the pharmacokinetics (PK) of busulfan in infants and small children to help guide decisions for safe and efficacious drug therapy. The objective of this study was to develop an algorithm for individualized dosing of i.v. busulfan in infants and children weighing ≤12 kg, that would achieve targeted exposure with the first dose of busulfan. Population PK modeling was conducted using intensive time-concentration data collected through the routine therapeutic drug monitoring of busulfan in 149 patients from 8 centers. Busulfan PK was well described by a 1-compartment base model with linear elimination. The important clinical covariates affecting busulfan PK were actual body weight and age. Based on our model, the predicted clearance of busulfan increases approximately 1.7-fold between 6 weeks to 2 years of life. For infants age

Published
2013

36. 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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38. The Wiskott-Aldrich syndrome protein is required for iNKT cell maturation and function

Author

Locci, Michela, Draghici, Elena, Marangoni, Francesco, Bosticardo, Marita, Catucci, Marco, Aiuti, Alessandro, Cancrini, Caterina, Marodi, Laszlo, Espanol, Teresa, Bredius, Robbert GM, Thrasher, Adrian J, Schulz, Ansgar, Litzman, Jiri, Roncarolo, Maria Grazia, Casorati, Giulia, Dellabona, Paolo, and Villa, Anna

Subjects
Genetics, Rare Diseases, Aetiology, 2.1 Biological and endogenous factors, Inflammatory and immune system, Animals, Cytokines, Flow Cytometry, Humans, Hyaluronan Receptors, Lymphocyte Activation, Mice, Mice, Knockout, Mutation, Natural Killer T-Cells, Phenotype, Wiskott-Aldrich Syndrome, Medical and Health Sciences, Immunology
Abstract

The Wiskott-Aldrich syndrome (WAS) protein (WASp) is a regulator of actin cytoskeleton in hematopoietic cells. Mutations of the WASp gene cause WAS. Although WASp is involved in various immune cell functions, its role in invariant natural killer T (iNKT) cells has never been investigated. Defects of iNKT cells could indeed contribute to several WAS features, such as recurrent infections and high tumor incidence. We found a profound reduction of circulating iNKT cells in WAS patients, directly correlating with the severity of clinical phenotype. To better characterize iNKT cell defect in the absence of WASp, we analyzed was(-/-) mice. iNKT cell numbers were significantly reduced in the thymus and periphery of was(-/-) mice as compared with wild-type controls. Moreover analysis of was(-/-) iNKT cell maturation revealed a complete arrest at the CD44(+) NK1.1(-) intermediate stage. Notably, generation of BM chimeras demonstrated a was(-/-) iNKT cell-autonomous developmental defect. was(-/-) iNKT cells were also functionally impaired, as suggested by the reduced secretion of interleukin 4 and interferon gamma upon in vivo activation. Altogether, these results demonstrate the relevance of WASp in integrating signals critical for development and functional differentiation of iNKT cells and suggest that defects in these cells may play a role in WAS pathology.

Published
2009

39. Hematopoietic stem cell transplantation rescues the hematological, immunological, and vascular phenotype in DADA2

Author

Hashem, Hasan, Kumar, Ashish R., Müller, Ingo, Babor, Florian, Bredius, Robbert, Dalal, Jignesh, Hsu, Amy P., Holland, Steven M., Hickstein, Dennis D., Jolles, Stephen, Krance, Robert, Sasa, Ghadir, Taskinen, Mervi, Koskenvuo, Minna, Saarela, Janna, van Montfrans, Joris, Wilson, Keith, Bosch, Barbara, Moens, Leen, Hershfield, Michael, and Meyts, Isabelle

Published
2017
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41. Association between anti-thymocyte globulin exposure and survival outcomes in adult unrelated haemopoietic cell transplantation: a retrospective, pharmacodynamic cohort analysis

Author

Admiraal, Rick, Nierkens, Stefan, de Witte, Moniek A, Petersen, Eefke J, Fleurke, Ger-jan, Verrest, Luka, Belitser, Svetlana V, Bredius, Robbert G M, Raymakers, Reinier A P, Knibbe, Catherijne A J, Minnema, Monique C, van Kesteren, Charlotte, Kuball, Jurgen, and Boelens, Jaap J

Published
2017
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43. Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency

Author

Cicalese, Maria Pia, Ferrua, Francesca, Castagnaro, Laura, Pajno, Roberta, Barzaghi, Federica, Giannelli, Stefania, Dionisio, Francesca, Brigida, Immacolata, Bonopane, Marco, Casiraghi, Miriam, Tabucchi, Antonella, Carlucci, Filippo, Grunebaum, Eyal, Adeli, Mehdi, Bredius, Robbert G., Puck, Jennifer M., Stepensky, Polina, Tezcan, Ilhan, Rolfe, Katie, De Boever, Erika, Reinhardt, Rickey R., Appleby, Jonathan, Ciceri, Fabio, Roncarolo, Maria Grazia, and Aiuti, Alessandro

Published
2016
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45. Clinical Symptoms, Laboratory Parameters and Long-Term Follow-up in a National DADA2 Cohort

Author

Genetica Sectie Genoomdiagnostiek, MS Reumatologie/Immunologie/Infectie, Infection & Immunity, CTI Research, Cancer, Cluster B, Immuno/reuma patientenzorg, Child Health, Andriessen, Marie Valérie E, Legger, G Elizabeth, Bredius, Robbert G M, van Gijn, Marielle E, Hak, A Elisabeth, Muller, Petra C E Hissink, Kamphuis, Sylvia, Klouwer, Femke C C, Kuijpers, Taco W, Leavis, Helen L, Nierkens, Stefan, Rutgers, Abraham, van der Veken, Lars T, van Well, Gijs T J, Mulders-Manders, Catharina M, van Montfrans, Joris M, Genetica Sectie Genoomdiagnostiek, MS Reumatologie/Immunologie/Infectie, Infection & Immunity, CTI Research, Cancer, Cluster B, Immuno/reuma patientenzorg, Child Health, Andriessen, Marie Valérie E, Legger, G Elizabeth, Bredius, Robbert G M, van Gijn, Marielle E, Hak, A Elisabeth, Muller, Petra C E Hissink, Kamphuis, Sylvia, Klouwer, Femke C C, Kuijpers, Taco W, Leavis, Helen L, Nierkens, Stefan, Rutgers, Abraham, van der Veken, Lars T, van Well, Gijs T J, Mulders-Manders, Catharina M, and van Montfrans, Joris M

Published
2023

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