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2. Retrospective, Landmark Analysis of Long-term Adult Morbidity Following Allogeneic HSCT for Inborn Errors of Immunity in Infancy and Childhood

Author

Day, James W., Elfeky, Reem, Nicholson, Bethany, Goodman, Rupert, Pearce, Rachel, Fox, Thomas A., Worth, Austen, Booth, Claire, Veys, Paul, Carpenter, Ben, Hough, Rachael, Gaspar, H. Bobby, Titman, Penny, Ridout, Deborah, Workman, Sarita, Hernandes, Fernando, Sandford, Kit, Laurence, Arian, Campbell, Mari, Burns, Siobhan O., and Morris, Emma C.

Published
2022
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5. 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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8. CAR T cells with dual targeting of CD19 and CD22 in pediatric and young adult patients with relapsed or refractory B cell acute lymphoblastic leukemia: a phase 1 trial

Author

Cordoba, Shaun, Onuoha, Shimobi, Thomas, Simon, Pignataro, Daniela Soriano, Hough, Rachael, Ghorashian, Sara, Vora, Ajay, Bonney, Denise, Veys, Paul, Rao, Kanchan, Lucchini, Giovanna, Chiesa, Robert, Chu, Jan, Clark, Liz, Fung, Mei Mei, Smith, Koval, Peticone, Carlotta, Al-Hajj, Muhammad, Baldan, Vania, Ferrari, Mathieu, Srivastava, Saket, Jha, Ram, Arce Vargas, Frederick, Duffy, Kevin, Day, William, Virgo, Paul, Wheeler, Lucy, Hancock, Jeremy, Farzaneh, Farzin, Domning, Sabine, Zhang, Yiyun, Khokhar, Nushmia Z., Peddareddigari, Vijay G. R., Wynn, Robert, Pule, Martin, and Amrolia, Persis J.

Published
2021
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9. Standardizing Definitions of Hematopoietic Recovery, Graft Rejection, Graft Failure, Poor Graft Function, and Donor Chimerism in Allogeneic Hematopoietic Cell Transplantation: A Report on Behalf of the American Society for Transplantation and Cellular Therapy

Author

Kharfan-Dabaja, Mohamed A., Kumar, Ambuj, Ayala, Ernesto, Aljurf, Mahmoud, Nishihori, Taiga, Marsh, Rebecca, Burroughs, Lauri M., Majhail, Navneet, Al-Homsi, A. Samer, Al-Kadhimi, Zaid S., Bar, Merav, Bertaina, Alice, Boelens, Jaap J., Champlin, Richard, Chaudhury, Sonali, DeFilipp, Zachariah, Dholaria, Bhagirathbhai, El-Jawahri, Areej, Fanning, Suzanne, Fraint, Ellen, Gergis, Usama, Giralt, Sergio, Hamilton, Betty K., Hashmi, Shahrukh K., Horn, Biljana, Inamoto, Yoshihiro, Jacobsohn, David A, Jain, Tania, Johnston, Laura, Kanate, Abraham S., Kansagra, Ankit, Kassim, Adetola, Kean, Leslie S., Kitko, Carrie L., Knight-Perry, Jessica, Kurtzberg, Joanne, Liu, Hien, MacMillan, Margaret L., Mahmoudjafari, Zahra, Mielcarek, Marco, Mohty, Mohamad, Nagler, Arnon, Nemecek, Eneida, Olson, Timothy S., Oran, Betul, Perales, Miguel-Angel, Prockop, Susan E., Pulsipher, Michael A., Pusic, Iskra, Riches, Marcie L., Rodriguez, Cesar, Romee, Rizwan, Rondon, Gabriela, Saad, Ayman, Shah, Nina, Shaw, Peter J., Shenoy, Shalini, Sierra, Jorge, Talano, Julie, Verneris, Michael R., Veys, Paul, Wagner, John E., Savani, Bipin N., Hamadani, Mehdi, and Carpenter, Paul A.

Published
2021
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10. Hematopoietic Stem Cell Transplantation Resolves the Immune Deficit Associated with STAT3-Dominant-Negative Hyper-IgE Syndrome

Author

Harrison, Stephanie C., Tsilifis, Christo, Slatter, Mary A., Nademi, Zohreh, Worth, Austen, Veys, Paul, Ponsford, Mark J., Jolles, Stephen, Al-Herz, Waleed, Flood, Terence, Cant, Andrew J., Doffinger, Rainer, Barcenas-Morales, Gabriela, Carpenter, Ben, Hough, Rachael, Haraldsson, Ásgeir, Heimall, Jennifer, Grimbacher, Bodo, Abinun, Mario, and Gennery, Andrew R.

Published
2021
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11. 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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14. Indications for Hematopoietic Cell Transplantation and Immune Effector Cell Therapy: Guidelines from the American Society for Transplantation and Cellular Therapy

Author

Kanate, Abraham S., Majhail, Navneet S., Savani, Bipin N., Bredeson, Christopher, Champlin, Richard E., Crawford, Stephen, Giralt, Sergio A., LeMaistre, Charles F., Marks, David I., Omel, James L., Orchard, Paul J., Palmer, Jeanne, Saber, Wael, Veys, Paul A., Carpenter, Paul A., and Hamadani, Mehdi

Published
2020
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15. 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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16. 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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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 ML, Dvorak, Christopher C, Savic, Rada M, Zwaveling, Juliette, Bredius, Robbert GM, Egberts, Antoine CG, Bierings, Marc, Kletzel, Morris, Shaw, Peter J, Nath, Christa E, Hempel, George, Ansari, Marc, Krajinovic, Maja, Théorêt, Yves, Duval, Michel, Keizer, Ron J, Bittencourt, Henrique, Hassan, Moustapha, Güngör, Tayfun, Wynn, Robert F, Veys, Paul, Cuvelier, Geoff DE, Marktel, Sarah, Chiesa, Robert, Cowan, Morton J, Slatter, Mary A, Stricherz, Melisa K, Jennissen, Cathryn, Long-Boyle, Janel R, and Boelens, Jaap Jan

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

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

Published
2016

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

Author

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

Published
2020
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25. New graft manipulation strategies improve the outcome of mismatched stem cell transplantation in children with primary immunodeficiencies

Author

Elfeky, Reem, Shah, Ravi M., Unni, Mohamed N.M., Ottaviano, Giorgio, Rao, Kanchan, Chiesa, Robert, Amrolia, Persis, Worth, Austen, Flood, Terry, Abinun, Mario, Hambleton, Sophie, Cant, Andrew J., Gilmour, Kimberly, Adams, Stuart, Ahsan, Gul, Barge, Dawn, Gennery, Andrew R., Qasim, Waseem, Slatter, Mary, and Veys, Paul

Published
2019
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26. 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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28. 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

29. A trial of alemtuzumab adjunctive therapy in allogeneic hematopoietic cell transplantation with minimal conditioning for severe combined immunodeficiency.

Author

Dvorak, Christopher C, Horn, Biljana N, Puck, Jennifer M, Adams, Stuart, Veys, Paul, Czechowicz, Agnieszka, and Cowan, Morton J

Subjects
Humans, Severe Combined Immunodeficiency, Histocompatibility Testing, Treatment Outcome, Transplantation Conditioning, Chemotherapy, Adjuvant, Hematopoietic Stem Cell Transplantation, Transplantation, Homologous, Prospective Studies, Pilot Projects, Child, Infant, California, Male, Antibodies, Monoclonal, Humanized, Alemtuzumab, alemtuzumab, engraftment, haploidentical, severe combined immunodeficiency, Engraftment, Haploidentical, Severe combined immunodeficiency, Chemotherapy, Adjuvant, Transplantation, Homologous, Antibodies, Monoclonal, Humanized, Surgery, Paediatrics and Reproductive Medicine
Abstract

For infants with SCID the ideal conditioning regimen before allogeneic HCT would omit cytotoxic chemotherapy to minimize short- and long-term complications. We performed a prospective pilot trial with alemtuzumab monotherapy to overcome NK-cell mediated immunologic barriers to engraftment. We enrolled four patients who received CD34-selected haploidentical cells, two of whom failed to engraft donor T cells. The two patients who engrafted had delayed T-cell reconstitution, despite rapid clearance of circulating alemtuzumab. Although well-tolerated, alemtuzumab failed to overcome immunologic barriers to donor engraftment. Furthermore, alemtuzumab may slow T-cell development in patients with SCID in the setting of a T-cell depleted graft.

Published
2014

30. Primary Immune Deficiency Treatment Consortium (PIDTC) report.

Author

Griffith, Linda M, Cowan, Morton J, Notarangelo, Luigi D, Kohn, Donald B, Puck, Jennifer M, Pai, Sung-Yun, Ballard, Barbara, Bauer, Sarah C, Bleesing, Jack JH, Boyle, Marcia, Brower, Amy, Buckley, Rebecca H, van der Burg, Mirjam, Burroughs, Lauri M, Candotti, Fabio, Cant, Andrew J, Chatila, Talal, Cunningham-Rundles, Charlotte, Dinauer, Mary C, Dvorak, Christopher C, Filipovich, Alexandra H, Fleisher, Thomas A, Bobby Gaspar, Hubert, Gungor, Tayfun, Haddad, Elie, Hovermale, Emily, Huang, Faith, Hurley, Alan, Hurley, Mary, Iyengar, Sumathi, Kang, Elizabeth M, Logan, Brent R, Long-Boyle, Janel R, Malech, Harry L, McGhee, Sean A, Modell, Fred, Modell, Vicki, Ochs, Hans D, O'Reilly, Richard J, Parkman, Robertson, Rawlings, David J, Routes, John M, Shearer, William T, Small, Trudy N, Smith, Heather, Sullivan, Kathleen E, Szabolcs, Paul, Thrasher, Adrian, Torgerson, Troy R, Veys, Paul, Weinberg, Kenneth, Zuniga-Pflucker, Juan Carlos, and workshop participants

Subjects
workshop participants, Humans, Immunologic Deficiency Syndromes, Neonatal Screening, Hematopoietic Stem Cell Transplantation, Pilot Projects, Infant, Newborn, Societies, Scientific, ADA, Adenosine deaminase, Allogeneic hematopoietic cell transplantation, CGD, CIBMTR, Center for International Blood and Marrow Transplant Research, Chronic granulomatous disease, EBMT, ESID, European Group for Blood and Marrow Transplantation, European Society for Immunodeficiencies, GT, GVHD, Gene therapy, Graft-versus-host disease, HCT, Hematopoietic cell transplantation, IEWP, Inborn Errors Working Party, MAC, MUD, Matched unrelated donor, Myeloablative conditioning, NBS, NIAID, NIH, NK, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Natural killer, Newborn screening for SCID, PID, PIDTC, Primary Immune Deficiency Treatment Consortium, Primary immunodeficiency, RIC, Reduced-intensity conditioning, SCETIDE, SCID, Severe combined immunodeficiency, Stem Cell Transplantation for Immunodeficiencies in Europe, USIDNET, United States Immunodeficiency Network, WAS, Wiskott-Aldrich syndrome, clinical trial, gene therapy, primary immunodeficiency, Rare Diseases, Pediatric, Clinical Research, Inflammatory and immune system, Immunology, Allergy
Abstract

The Primary Immune Deficiency Treatment Consortium (PIDTC) is a network of 33 centers in North America that study the treatment of rare and severe primary immunodeficiency diseases. Current protocols address the natural history of patients treated for severe combined immunodeficiency (SCID), Wiskott-Aldrich syndrome, and chronic granulomatous disease through retrospective, prospective, and cross-sectional studies. The PIDTC additionally seeks to encourage training of junior investigators, establish partnerships with European and other International colleagues, work with patient advocacy groups to promote community awareness, and conduct pilot demonstration projects. Future goals include the conduct of prospective treatment studies to determine optimal therapies for primary immunodeficiency diseases. To date, the PIDTC has funded 2 pilot projects: newborn screening for SCID in Navajo Native Americans and B-cell reconstitution in patients with SCID after hematopoietic stem cell transplantation. Ten junior investigators have received grant awards. The PIDTC Annual Scientific Workshop has brought together consortium members, outside speakers, patient advocacy groups, and young investigators and trainees to report progress of the protocols and discuss common interests and goals, including new scientific developments and future directions of clinical research. Here we report the progress of the PIDTC to date, highlights of the first 2 PIDTC workshops, and consideration of future consortium objectives.

Published
2014

31. Treatment dilemmas in asymptomatic children with primary hemophagocytic lymphohistiocytosis

Author

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

Published
2018
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35. Challenges and Opportunities for International Cooperative Studies in Pediatric Hematopoeitic Cell Transplantation: Priorities of the Westhafen Intercontinental Group

Author

Schultz, Kirk R, Baker, Kevin Scott, Boelens, Jaap J, Bollard, Catherine M, Egeler, R Maarten, Cowan, Mort, Ladenstein, Ruth, Lankester, Arjan, Locatelli, Franco, Lawitschka, Anita, Levine, John E, Loh, Mignon, Nemecek, Eneida, Niemeyer, Charlotte, Prasad, Vinod K, Rocha, Vanderson, Shenoy, Shalini, Strahm, Brigitte, Veys, Paul, Wall, Donna, Bader, Peter, Grupp, Stephan A, Pulsipher, Michael A, and Peters, Christina

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Pediatric, Clinical Trials and Supportive Activities, Pediatric Research Initiative, Stem Cell Research, Clinical Research, Hematology, Transplantation, Regenerative Medicine, Adolescent, Age Factors, Child, Hematopoietic Stem Cell Transplantation, Humans, International Cooperation, Transplantation, Homologous, Pediatrics, Hematopoietic cell transplantation, Clinical Sciences, Immunology, Cardiovascular medicine and haematology
Abstract

More than 20% of allogeneic hematopoietic cell transplantations (HCTs) are performed in children and adolescents at a large number of relatively small centers. Unlike adults, at least one-third of HCTs in children are performed for rare, nonmalignant indications. Clinical trials to improve HCT outcomes in children have been limited by small numbers and these pediatric-specific features. The need for a larger number of pediatric HCT centers to participate in trials has led to the involvement of international collaborative groups. Representatives of the Pediatric Blood and Marrow Transplant Consortium, European Group for Blood and Marrow Transplantation's Pediatric Working Group, International Berlin-Frankfurt-Munster (iBFm) Stem Cell Transplantation Committee, and Children's Oncology Group's Hematopoietic Stem Cell Transplantation Discipline Committee met on October 3, 2012, in Frankfurt, Germany to develop a consensus on the highest priorities in pediatric HCT. In addition, it explored the creation of an international consortium to develop studies focused on HCT in children and adolescents. This meeting led to the creation of an international HCT network, dubbed the Westhafen Intercontinental Group, to develop worldwide priorities and strategies to address pediatric HCT issues. This review outlines the priorities of need as identified by this consensus group.

Published
2013

36. Enhanced CAR T cell expansion and prolonged persistence in pediatric patients with ALL treated with a low-affinity CD19 CAR

Author

Ghorashian, Sara, Kramer, Anne Marijn, Onuoha, Shimobi, Wright, Gary, Bartram, Jack, Richardson, Rachel, Albon, Sarah J., Casanovas-Company, Joan, Castro, Fernanda, Popova, Bilyana, Villanueva, Krystle, Yeung, Jenny, Vetharoy, Winston, Guvenel, Aleks, Wawrzyniecka, Patrycja A., Mekkaoui, Leila, Cheung, Gordon Weng-Kit, Pinner, Danielle, Chu, Jan, Lucchini, Giovanna, Silva, Juliana, Ciocarlie, Oana, Lazareva, Arina, Inglott, Sarah, Gilmour, Kimberly C., Ahsan, Gulrukh, Ferrari, Mathieu, Manzoor, Somayya, Champion, Kim, Brooks, Tony, Lopes, Andre, Hackshaw, Allan, Farzaneh, Farzin, Chiesa, Robert, Rao, Kanchan, Bonney, Denise, Samarasinghe, Sujith, Goulden, Nicholas, Vora, Ajay, Veys, Paul, Hough, Rachael, Wynn, Robert, Pule, Martin A., and Amrolia, Persis J.

Published
2019
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37. Long-Term Survival and Late Deaths after Hematopoietic Cell Transplantation for Primary Immunodeficiency Diseases and Inborn Errors of Metabolism

Author

Eapen, Mary, Ahn, Kwang Woo, Orchard, Paul J, Cowan, Morton J, Davies, Stella M, Fasth, Anders, Hassebroek, Anna, Ayas, Mouhab, Bonfim, Carmem, O’Brien, Tracey A, Gross, Thomas G, Horwitz, Mitchell, Horwitz, Edwin, Kapoor, Neena, Kurtzberg, Joanne, Majhail, Navneet, Ringden, Olle, Szabolcs, Paul, Veys, Paul, and Baker, K Scott

Subjects
Clinical Research, Transplantation, Hematology, Pediatric, Good Health and Well Being, Adolescent, Child, Child, Preschool, Chronic Disease, Europe, Female, Graft vs Host Disease, Hematopoietic Stem Cell Transplantation, Humans, Immunologic Deficiency Syndromes, Infant, International Cooperation, Male, Metabolism, Inborn Errors, Severe Combined Immunodeficiency, Survival Analysis, Time Factors, Transplantation Chimera, Transplantation Conditioning, Transplantation, Homologous, United States, Unrelated Donors, Mortality, Graft-versus-host disease, Non-malignant hematologic diseases, Clinical Sciences, Immunology
Abstract

It is uncertain whether late mortality rates after hematopoietic cell transplantation for severe combined immunodeficiency (SCID), non-SCID primary immunodeficiency diseases (non-SCID PIDD), and inborn errors of metabolism (IEM) return to rates observed in the general population, matched for age, sex, and nationality. We studied patients with SCID (n = 201), non-SCID PIDD (n = 405), and IEM (n = 348) who survived for at least 2 years after transplantation with normal T cell function (SCID) or >95% donor chimerism (non-SCID PIDD and IEM). Importantly, mortality rate was significantly higher in these patients compared with the general population for several years after transplantation. The rate decreased toward the normal rate in patients with SCID and non-SCID PIDD beyond 6 years after transplantation, but not in patients with IEM. Active chronic graft-versus-host disease at 2 years was associated with increased risk of late mortality for all diseases (hazard ratio [HR], 1.87; P = .05). In addition, late mortality was higher in patients with non-SCID PIDD who received T cell-depleted grafts (HR 4.16; P = .007) and in patients with IEM who received unrelated donor grafts (HR, 2.72; P = .03) or mismatched related donor grafts (HR, 3.76; P = .01). The finding of higher mortality rates in these long-term survivors for many years after transplantation confirms the need for long-term surveillance.

Published
2012

38. Hematopoietic stem cell transplantation in 29 patients hemizygous for hypomorphic IKBKG/NEMO mutations

Author

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

Published
2017
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39. Allele-level HLA matching for umbilical cord blood transplantation for non-malignant diseases in children: a retrospective analysis

Author

Eapen, Mary, Wang, Tao, Veys, Paul A, Boelens, Jaap J, St Martin, Andrew, Spellman, Stephen, Bonfim, Carmem Sales, Brady, Colleen, Cant, Andrew J, Dalle, Jean-Hugues, Davies, Stella M, Freeman, John, Hsu, Katherine C, Fleischhauer, Katharina, Kenzey, Chantal, Kurtzberg, Joanne, Michel, Gerard, Orchard, Paul J, Paviglianiti, Annalisa, Rocha, Vanderson, Veneris, Michael R, Volt, Fernanda, Wynn, Robert, Lee, Stephanie J, Horowitz, Mary M, Gluckman, Eliane, and Ruggeri, Annalisa

Published
2017
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40. Reticular dysgenesis: international survey on clinical presentation, transplantation, and outcome

Author

Hoenig, Manfred, Lagresle-Peyrou, Chantal, Pannicke, Ulrich, Notarangelo, Luigi D., Porta, Fulvio, Gennery, Andrew R., Slatter, Mary, Cowan, Morton J., Stepensky, Polina, Al-Mousa, Hamoud, Al-Zahrani, Daifulah, Pai, Sung-Yun, Al Herz, Waleed, Gaspar, Hubert B., Veys, Paul, Oshima, Koichi, Imai, Kohsuke, Yabe, Hiromasa, Noroski, Lenora M., Wulffraat, Nico M., Sykora, Karl-Walter, Soler-Palacin, Pere, Muramatsu, Hideki, Al Hilali, Mariam, Moshous, Despina, Debatin, Klaus-Michael, Schuetz, Catharina, Jacobsen, Eva-Maria, Schulz, Ansgar S., Schwarz, Klaus, Fischer, Alain, Friedrich, Wilhelm, and Cavazzana, Marina

Published
2017
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41. Impact of Conditioning Regimen on Outcomes for Children with Acute Myeloid Leukemia Undergoing Transplantation in First Complete Remission. An Analysis on Behalf of the Pediatric Disease Working Party of the European Group for Blood and Marrow Transplantation

Author

Lucchini, Giovanna, Labopin, Myriam, Beohou, Eric, Dalissier, Arnauld, Dalle, Jean Hughes, Cornish, Jacqueline, Zecca, Marco, Samarasinghe, Sujith, Gibson, Brenda, Locatelli, Franco, Bertrand, Yves, Abdel-Rahman, Fawzi, Socie, Gerald, Sundin, Mikael, Lankester, Arjan, Sedlacek, Peter, Hamladji, Rose Marie, Heilmann, Carsten, Afanasyev, Boris, Hough, Rachel, Peters, Cristina, Bader, Peter, and Veys, Paul

Published
2017
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43. High transplant-related mortality associated with haematopoietic stem cell transplantation for paediatric therapy-related acute myeloid leukaemia (t-AML). A study on behalf of the United Kingdom Paediatric Blood and Bone Marrow Transplant Group

Author

Gassas, Adam, Sivaprakasam, Ponni, Cummins, Michelle, Breslin, Patricia, Patrick, Katharine, Slatter, Mary, Skinner, Roderick, Shenton, Geof, Gibson, Brenda, Lawson, Sarah, Petterson, Toni, Potter, Michael, James, Beki, Hough, Rachael, Hiwarkar, Prashant, Vora, Ajay, Veys, Paul, De La Fuente, Josu, Wynn, Robert, and Amrolia, Persis

Published
2018
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45. Hematopoietic stem cell transplantation outcomes for 11 patients with dedicator of cytokinesis 8 deficiency

Author

Al-Herz, Waleed, Chu, Julia I., van der Spek, Jet, Raghupathy, Raj, Massaad, Michel J., Keles, Sevgi, Biggs, Catherine M., Cockerton, Lucinda, Chou, Janet, Dbaibo, Ghassan, Elisofon, Scott A., Hanna-Wakim, Rima, Kim, Heung Bae, Lehmann, Leslie E., McDonald, Douglas R., Notarangelo, Luigi D., Veys, Paul, Chatila, Talal A., Geha, Raif S., Gaspar, H. Bobby, and Pai, Sung-Yun

Published
2016
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46. Treosulfan-based conditioning for allogeneic HSCT in children with chronic granulomatous disease: a multicenter experience

Author

Morillo-Gutierrez, Beatriz, Beier, Rita, Rao, Kanchan, Burroughs, Lauri, Schulz, Ansgar, Ewins, Anna-Maria, Gibson, Brenda, Sedlacek, Petr, Krol, Ladislav, Strahm, Brigitte, Zaidman, Irina, Kalwak, Krzysztof, Talano, Julie-An, Woolfrey, Ann, Fraser, Chris, Meyts, Isabelle, Müller, Ingo, Wachowiak, Jacek, Bernardo, Maria Ester, Veys, Paul, Sykora, Karl-Walter, Gennery, Andrew R., and Slatter, Mary

Published
2016
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47. The minimum required level of donor chimerism in hereditary hemophagocytic lymphohistiocytosis

Author

Hartz, Bernd, Marsh, Rebecca, Rao, Kanchan, Henter, Jan-Inge, Jordan, Michael, Filipovich, Lisa, Bader, Peter, Beier, Rita, Burkhardt, Birgit, Meisel, Roland, Schulz, Ansgar, Winkler, Beate, Albert, Michael H., Greil, Johann, Karasu, Gülsün, Woessmann, Wilhelm, Corbacioglu, Selim, Gruhn, Bernd, Holter, Wolfgang, Kühl, Jörn-Sven, Lang, Peter, Seidel, Markus G., Veys, Paul, Löfstedt, Alexandra, Ammann, Sandra, Ehl, Stephan, Janka, Gritta, Müller, Ingo, and Lehmberg, Kai

Published
2016
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