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1. 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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8. 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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10. 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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11. 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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16. 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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17. 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 M L, Dvorak, Christopher C, Savic, Rada M, Zwaveling, Juliette, Bredius, Robbert G M, Egberts, Antoine C G, 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 D E, Marktel, Sarah, Chiesa, Robert, Cowan, Morton J, Slatter, Mary A, Stricherz, Melisa K, Jennissen, Cathryn, Long-Boyle, Janel R, and Boelens, Jaap Jan

Published
2016
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18. 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

22. Development and Validation of an Efficient and Highly Sensitive Enzyme-Linked Immunosorbent Assay for Alemtuzumab Quantification in Human Serum and Plasma

Author

Achini-Gutzwiller, Federica R., primary, Jol-van der Zijde, Cornelia M., additional, Jansen-Hoogendijk, Anja M., additional, Lankester, Arjan C., additional, Bredius, Robbert G. M., additional, van Tol, Maarten J. D., additional, Moes, Dirk Jan A. R., additional, and Schilham, Marco W., additional

Published
2022
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25. 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, Lalmohamed, Arief, Afd Pharmacoepi & Clinical Pharmacology, Pharmacoepidemiology and Clinical Pharmacology, Afd Pharmacoepi & Clinical Pharmacology, Pharmacoepidemiology and Clinical Pharmacology, Clinical pharmacology and pharmacy, and CCA - Cancer Treatment and quality of life

Subjects
Medicine(all), Transplantation, Transplantation Conditioning, Hematopoietic cell transplantation, Sinusoidal obstruction syndrome, Veno-occlusive disease, Hematopoietic Stem Cell Transplantation, Hepatic Veno-Occlusive Disease, Hematopoietic cell, Hematology, Cell Biology, Young Adult, Humans, Immunology and Allergy, Molecular Medicine, Administration, Intravenous, Busulfan exposure, Child, Busulfan
Abstract

Intravenous busulfan is widely used as part of myeloablative conditioning regimens in children and young adults undergoing allogeneic hematopoietic cell transplantation (HCT). Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a serious clinical problem observed with busulfan-based conditioning HCT. The development of VOD/SOS may be associated with busulfan exposure. Getting more insight into the association between busulfan exposure and the development of VOD/SOS enables further optimization of dos-ing and treatment strategies. The objective of this study was to assess the association between the magnitude of busulfan exposure and the occurrence of VOD/SOS in children and young adults undergoing myeloablative con-ditioning with a busulfan-containing regimen before allogeneic HCT. In this observational study we included all patients who underwent allogeneic HCT with intravenous busulfan as part of the conditioning regimen at 15 pediatric transplantation centers between 2000 and 2015. The endpoint was the development of VOD/SOS. The magnitude of busulfan exposure was estimated using nonlinear mixed effect modeling and expressed as the maximal concentration (Cmax; day 1 and day 1 to 4 Cmax), cumulative area under the curve (AUC; day 1, high-est 1-day AUC in 4 days, and 4-day cumulative AUC), cumulative time above a concentration of 300 mg/L, and clearance on day 1. A total of 88 out of 697 patients (12.6%) developed VOD/SOS. The number of alkylators in the conditioning regimen was a strong effect modifier; therefore we stratified the regression analysis for the number of alkylators. For patients receiving only busulfan as one alkylator (36.3%, n = 253), cumulative busulfan exposure (>78 mg x h/L) was associated with increased VOD/SOS risk (12.6% versus 4.7%; odds ratio [OR] = 2.95, 95% confidence interval [CI] 1.13 to 7.66). For individuals receiving busulfan with one or two addi-tional alkylators (63.7%, n = 444), cumulative busulfan exposure (78 mg x h/L) did not further increase the risk of VOD/SOS (15.4% versus 15.2%; OR = 1.03, 95% CI 0.61 to 1.75). The effect of the magnitude of busulfan exposure on VOD/SOS risk in children and young adults undergoing HCT is dependent on the number of alkylators. In patients receiving busulfan as the only alkylator, higher cumulative busulfan exposure increased the risk of VOD/SOS, whereas in those receiving multiple alkylators, the magnitude of busulfan expo-sure did not further increase this risk. (c) 2022 The American Society for Transplantation and Cellular Therapy. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/)

Published
2022

26. 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

Afd Pharmacoepi & Clinical Pharmacology, Pharmacoepidemiology and Clinical Pharmacology, 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, Lalmohamed, Arief, Afd Pharmacoepi & Clinical Pharmacology, Pharmacoepidemiology and Clinical Pharmacology, 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

27. 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

Apotheek Opleiding, Apotheek Onderzoek, Child Health, Infection & Immunity, Regenerative Medicine and Stem Cells, Brain, SCT patientenzorg, CTI Nierkens, Apotheek O&O&O, Cancer, 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, Lalmohamed, Arief, Apotheek Opleiding, Apotheek Onderzoek, Child Health, Infection & Immunity, Regenerative Medicine and Stem Cells, Brain, SCT patientenzorg, CTI Nierkens, Apotheek O&O&O, Cancer, 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

29. Pharmacokinetics of rituximab in a pediatric patient with therapy-resistant nephrotic syndrome

Author

Counsilman, Clare E., Jol-van der Zijde, Cornelia M., Stevens, Jasper, Cransberg, Karlien, Bredius, Robbert G. M., and Sukhai, Ram N.

Subjects
Nephrotic syndrome -- Research -- Drug therapy -- Patient outcomes -- Complications and side effects, Rituximab -- Dosage and administration -- Research, Pharmacokinetics -- Research, Health
Abstract

Background Rituximab (RTX) has recently been introduced as a second-line therapy for nephrotic syndrome in children. Studies show that RTX given during the nephrotic state may be less effective than treatment during a non-nephrotic state, possibly due to loss of RTX in the urine. Case-Diagnosis/Treatment We describe a 10-year-old boy with steroid-resistant nephrotic syndrome (SRNS) treated with RTX during a phase of active non-selective proteinuria. The serum half-life of RTX in this patient was less than 1 day compared to 20 days in patients without protein losses. Urinary clearance was at least 25 %, compared to approximately 0 % in control patients. However, RTX loss in the urine, as well as in pleural effusion and ascites, only partly explains the rapid drop in the serum RTX concentration of this patient. Conclusions Serum half-life of RTX can be extremely short, partly due to excessive urinary losses in therapy-resistant nephrotic syndrome with non-selective proteinuria, as seen in our patient. These findings may help to explain the poor results of RTX treatment in patients with active proteinuria. Electronic supplementary material The online version of this article (doi:10.1007/s00467-015-3120-8) contains supplementary material, which is available to authorized users., Author(s): Clare E. Counsilman[sup.1] , Cornelia M. Jol-van der Zijde[sup.1] , Jasper Stevens[sup.3] , Karlien Cransberg[sup.2] , Robbert G. M. Bredius[sup.1] , Ram N. Sukhai[sup.1] Author Affiliations: (1) Department of [...]

Published
2015
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33. Phenotypic variability in patients with ADA2 deficiency due to identical homozygous R169Q mutations

Author

Van Montfrans, Joris M., Hartman, Esther A. R., Braun, Kees P. J., Hennekam, Eric A. M., Hak, Elisabeth A., Nederkoorn, Paul J., Westendorp, Willeke F., Bredius, Robbert G. M., Kollen, Wouter J. W., Schölvinck, Elisabeth H., Legger, G. Elizabeth, Meyts, Isabelle, Liston, Adrian, Lichtenbelt, Klaske D., Giltay, Jacques C., Van Haaften, Gijs, De Vries Simons, Gaby M., Leavis, Helen, Sanders, Cornelis J. G., Bierings, Marc B., Nierkens, Stefan, and Van Gijn, Marielle E.

Published
2016
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36. Precision dosing of intravenous busulfan in pediatric hematopoietic stem cell transplantation: Results from a multicenter population pharmacokinetic study

Author

Ben Hassine, Khalil, primary, Nava, Tiago, additional, Théoret, Yves, additional, Nath, Christa E., additional, Daali, Youssef, additional, Kassir, Nastya, additional, Lewis, Victor, additional, Bredius, Robbert G. M., additional, Shaw, Peter J., additional, Bittencourt, Henrique, additional, Krajinovic, Maja, additional, Uppugunduri, Chakradhara Rao Satyanarayana, additional, and Ansari, Marc, additional

Published
2021
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38. IL-7 and IL-15 Levels Reflect the Degree of T Cell Depletion during Lymphopenia and Are Associated with an Expansion of Effector Memory T Cells after Pediatric Hematopoietic Stem Cell Transplantation

Author

Kielsen, Katrine, primary, Oostenbrink, Lisa V. E., additional, von Asmuth, Erik G. J., additional, Jansen-Hoogendijk, Anja M., additional, van Ostaijen-ten Dam, Monique M., additional, Ifversen, Marianne, additional, Heilmann, Carsten, additional, Schilham, Marco W., additional, van Halteren, Astrid G. S., additional, Bredius, Robbert G. M., additional, Lankester, Arjan C., additional, Jol-van der Zijde, Cornelia M., additional, van Tol, Maarten J. D., additional, and Müller, Klaus, additional

Published
2021
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39. Immunoglobulin Replacement Therapy Versus Antibiotic Prophylaxis as Treatment for Incomplete Primary Antibody Deficiency

Author

Immuno/reuma onderzoek 1 (Vastert), Infection & Immunity, MS Interne Geneeskunde, Infectieziekten patientenzorg, Immuno/reuma onderzoek 7 (Montfrans), Immuno/reuma patientenzorg, Child Health, 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, van Montfrans, Joris M, Immuno/reuma onderzoek 1 (Vastert), Infection & Immunity, MS Interne Geneeskunde, Infectieziekten patientenzorg, Immuno/reuma onderzoek 7 (Montfrans), Immuno/reuma patientenzorg, Child Health, 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

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

Author

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

Published
2020
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45. Outcome of Non-hematological Autoimmunity After Hematopoietic Cell Transplantation in Children with Primary Immunodeficiency

Author

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

Published
2020
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48. Hematopoietic stem cell transplantation for CD40 ligand deficiency: Results from an EBMT/ESID-IEWP-SCETIDE-PIDTC study

Author

Ferrua, F, Galimberti, S, Courteille, V, Slatter, M, Booth, C, Moshous, D, Neven, B, Blanche, S, Laberko, A, Shcherbina, A, Balashov, D, Soncini, E, Porta, F, Al-Mousa, H, Al-Saud, B, Al-Dhekri, H, Arnaout, R, Formankova, R, Bertrand, Y, Lange, A, Smart, J, Wolska-Kusnierz, B, Aquino, V, Dvorak, C, Fasth, A, Fouyssac, F, Heilmann, C, Hoenig, M, Schuetz, C, Kelečić, J, Bredius, R, Lankester, A, Lindemans, C, Suarez, F, Sullivan, K, Albert, M, Kałwak, K, Barlogis, V, Bhatia, M, Bordon, V, Czogala, W, Alonso, L, Dogu, F, Gozdzik, J, Ikinciogullari, A, Kriván, G, Ljungman, P, Meyts, I, Mustillo, P, Smith, A, Speckmann, C, Sundin, M, Keogh, S, Shaw, P, Boelens, J, Schulz, A, Sedlacek, P, Veys, P, Mahlaoui, N, Janda, A, Davies, E, Fischer, A, Cowan, M, Gennery, A, Ferrua, Francesca, Galimberti, Stefania, Courteille, Virginie, Slatter, Mary Anne, Booth, Claire, Moshous, Despina, Neven, Benedicte, Blanche, Stephane, 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, Gennery, Andrew Richard, Ferrua, F, Galimberti, S, Courteille, V, Slatter, M, Booth, C, Moshous, D, Neven, B, Blanche, S, Laberko, A, Shcherbina, A, Balashov, D, Soncini, E, Porta, F, Al-Mousa, H, Al-Saud, B, Al-Dhekri, H, Arnaout, R, Formankova, R, Bertrand, Y, Lange, A, Smart, J, Wolska-Kusnierz, B, Aquino, V, Dvorak, C, Fasth, A, Fouyssac, F, Heilmann, C, Hoenig, M, Schuetz, C, Kelečić, J, Bredius, R, Lankester, A, Lindemans, C, Suarez, F, Sullivan, K, Albert, M, Kałwak, K, Barlogis, V, Bhatia, M, Bordon, V, Czogala, W, Alonso, L, Dogu, F, Gozdzik, J, Ikinciogullari, A, Kriván, G, Ljungman, P, Meyts, I, Mustillo, P, Smith, A, Speckmann, C, Sundin, M, Keogh, S, Shaw, P, Boelens, J, Schulz, A, Sedlacek, P, Veys, P, Mahlaoui, N, Janda, A, Davies, E, Fischer, A, Cowan, M, Gennery, A, Ferrua, Francesca, Galimberti, Stefania, Courteille, Virginie, Slatter, Mary Anne, Booth, Claire, Moshous, Despina, Neven, Benedicte, Blanche, Stephane, 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

Abstract

Background: CD40 ligand (CD40L) deficiency, an X-linked primary immunodeficiency, causes recurrent sinopulmonary, Pneumocystis and Cryptosporidium species infections. Long-term survival with supportive therapy is poor. Currently, the only curative treatment is hematopoietic stem cell transplantation (HSCT). Objective: We performed an international collaborative study to improve patients' management, aiming to individualize risk factors and determine optimal HSCT characteristics. Methods: We retrospectively collected data on 130 patients who underwent HSCT for CD40L deficiency between 1993-2015. We analyzed outcome and variables' relevance with respect to survival and cure. Results: Overall survival (OS), event-free survival (EFS), and disease-free survival (DFS) were 78.2%, 58.1%, and 72.3% 5 years after HSCT. Results were better in transplantations performed in 2000 or later and in children less than 10 years old at the time of HSCT. Pre-existing organ damage negatively influenced outcome. Sclerosing cholangitis was the most important risk factor. After 2000, superior OS was achieved with matched donors. Use of myeloablative regimens and HSCT at 2 years or less from diagnosis associated with higher OS and DFS. EFS was best with matched sibling donors, myeloablative conditioning (MAC), and bone marrow–derived stem cells. Most rejections occurred after reduced-intensity or nonmyeloablative conditioning, which associated with poor donor cell engraftment. Mortality occurred mainly early after HSCT, predominantly from infections. Among survivors who ceased immunoglobulin replacement, T-lymphocyte chimerism was 50% or greater donor in 85.2%. Conclusion: HSCT is curative in patients with CD40L deficiency, with improved outcome if performed before organ damage development. MAC is associated with better OS, EFS, and DFS. Prospective studies are required to compare the risks of HSCT with those of lifelong supportive therapy.

Published
2019

49. Differential Elimination of Anti-Thymocyte Globulin of Fresenius and Genzyme Impacts T-Cell Reconstitution After Hematopoietic Stem Cell Transplantation

Author

Oostenbrink, Lisa V E, Jol-van der Zijde, Cornelia M, Kielsen, Katrine, Jansen-Hoogendijk, Anja M, Ifversen, Marianne, Müller, Klaus G, Lankester, Arjan C, van Halteren, Astrid G S, Bredius, Robbert G M, Schilham, Marco W, van Tol, Maarten J D, Oostenbrink, Lisa V E, Jol-van der Zijde, Cornelia M, Kielsen, Katrine, Jansen-Hoogendijk, Anja M, Ifversen, Marianne, Müller, Klaus G, Lankester, Arjan C, van Halteren, Astrid G S, Bredius, Robbert G M, Schilham, Marco W, and van Tol, Maarten J D

Abstract

Anti-thymocyte globulin (ATG) is a lymphocyte depleting agent applied in hematopoietic stem cell transplantation (HSCT) to prevent rejection and Graft-vs.-Host Disease (GvHD). In this study, we compared two rabbit ATG products, ATG-Genzyme (ATG-GENZ), and ATG-Fresenius (ATG-FRES), with respect to dosing, clearance of the active lymphocyte binding component, post-HSCT immune reconstitution and clinical outcome. Fifty-eigth pediatric acute leukemia patients (n = 42 ATG-GENZ, n = 16 ATG-FRES), who received a non-depleted bone marrow or peripheral blood stem cell graft from an unrelated donor were included. ATG-GENZ was given at a dosage of 6-10 mg/kg; ATG-FRES at 45-60 mg/kg. The active component of ATG from both products was cleared at different rates. Within the ATG-FRES dose range no differences were found in clearance of active ATG or T-cell re-appearance. However, the high dosage of ATG-GENZ (10 mg/kg), in contrast to the low dosage (6-8 mg/kg), correlated with prolonged persistence of active ATG and delayed T-cell reconstitution. Occurrence of serious acute GvHD (grade III-IV) was highest in the ATG-GENZ-low dosage group. These results imply that dosing of ATG-GENZ is more critical than dosing of ATG-FRES due to the difference in clearance of active ATG. This should be taken into account when designing clinical protocols.

Published
2019

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