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3. 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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5. Measurable residual disease analysis in paediatric acute lymphoblastic leukaemia patients with ABL-class fusions

Author

Venn, Nicola C., Huang, Libby, Hovorková, Lenka, Muskovic, Walter, Wong, Marie, Law, Tamara, Heatley, Susan L., Khaw, Seong Lin, Revesz, Tom, Dalla Pozza, Luciano, Shaw, Peter J., Fraser, Chris, Moore, Andrew S., Cross, Siobhan, Bendak, Katerina, Norris, Murray D., Henderson, Michelle J., White, Deborah L., Cowley, Mark J., Trahair, Toby N., Zuna, Jan, and Sutton, Rosemary

Published
2022
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6. Outcomes after Second Hematopoietic Cell Transplantation in Children and Young Adults with Relapsed Acute Leukemia

Author

Lund, Troy C, Ahn, Kwang Woo, Tecca, Heather R, Hilgers, Megan V, Abdel-Azim, Hisham, Abraham, Allistair, Diaz, Miguel Angel, Badawy, Sherif M, Broglie, Larisa, Brown, Valerie, Dvorak, Christopher C, Gonzalez-Vicent, Marta, Hashem, Hasan, Hayashi, Robert J, Jacobsohn, David A, Kent, Michael W, Li, Chi-Kong, Margossian, Steven P, Martin, Paul L, Mehta, Parinda, Myers, Kasiani, Olsson, Richard, Page, Kristin, Pulsipher, Michael A, Shaw, Peter J, Smith, Angela R, Triplett, Brandon M, Verneris, Michael R, and Eapen, Mary

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Cancer, Transplantation, Hematology, Childhood Leukemia, Regenerative Medicine, Pediatric Cancer, Rare Diseases, Orphan Drug, Pediatric, Acute Disease, Adolescent, Adult, Child, Child, Preschool, Disease-Free Survival, Female, Hematopoietic Stem Cell Transplantation, Humans, Infant, Leukemia, Male, Recurrence, Survival Rate, Time Factors, Young Adult, relapse acute leukemia second transplant, Clinical Sciences, Immunology, Cardiovascular medicine and haematology
Abstract

Children with acute leukemia who relapse after hematopoietic cell transplantation (HCT) have few therapeutic options. We studied 251 children and young adults with acute myelogenous or lymphoblastic leukemia who underwent a second HCT for relapse after their first HCT. The median age at second HCT was 11 years, and the median interval between first and second HCT was 17 months. Most of the patients (n = 187; 75%) were in remission, received a myeloablative conditioning regimen (n = 157; 63%), and underwent unrelated donor HCT (n = 230; 92%). The 2-year probability of leukemia-free survival (LFS) was 33% after transplantation in patients in remission, compared with 19% after transplantation in patients not in remission (P = .02). The corresponding 8-year probabilities were 24% and 10% (P = .003). A higher rate of relapse contributed to the difference in LFS. The 2-year probability of relapse after transplantation was 42% in patients in remission and 56% in those in relapse (P = .05). The corresponding 8-year probabilities were 49% and 64% (P = .04). These data extend the findings of others showing that patients with a low disease burden are more likely to benefit from a second transplantation. Late relapse led to a 10% decrement in LFS beyond the second year after second HCT. This differs from first HCT, in which most relapses occur within 2 years after HCT.

Published
2019

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

Author

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

Published
2022
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9. 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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11. Outcomes of pediatric patients with therapy-related myeloid neoplasms

Author

Sharma, Akshay, Huang, Sujuan, Li, Ying, Brooke, Russell J., Ahmed, Ibrahim, Allewelt, Heather B., Amrolia, Persis, Bertaina, Alice, Bhatt, Neel S., Bierings, Marc B., Bies, Joshua, Brisset, Claire, Brondon, Jennifer E., Dahlberg, Ann, Dalle, Jean-Hugues, Eissa, Hesham, Fahd, Mony, Gassas, Adam, Gloude, Nicholas J., Goebel, W. Scott, Goeckerman, Erika S., Harris, Katherine, Ho, Richard, Hudspeth, Michelle P., Huo, Jeffrey S., Jacobsohn, David, Kasow, Kimberly A., Katsanis, Emmanuel, Kaviany, Saara, Keating, Amy K., Kernan, Nancy A., Ktena, Yiouli P., Lauhan, Colette R., López-Hernandez, Gerardo, Martin, Paul L., Myers, Kasiani C., Naik, Swati, Olaya-Vargas, Alberto, Onishi, Toshihiro, Radhi, Mohamed, Ramachandran, Shanti, Ramos, Kristie, Rangarajan, Hemalatha G., Roehrs, Philip A., Sampson, Megan E., Shaw, Peter J., Skiles, Jodi L., Somers, Katherine, Symons, Heather J., de Tersant, Marie, Uber, Allison N., Versluys, Birgitta, Cheng, Cheng, and Triplett, Brandon M.

Published
2021
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12. Outcome of hematopoietic cell transplantation for DNA double-strand break repair disorders

Author

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

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

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

Published
2018

14. 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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17. Pathobiological signatures of dysbiotic lung injury in pediatric patients undergoing stem cell transplantation

Author

MS Algemene Pediatrie Onderzoek 2, Haematologie patientenzorg, SCT patientenzorg, Child Health, Infection & Immunity, Regenerative Medicine and Stem Cells, CTI Nierkens, Zinter, Matt S., Dvorak, Christopher C., Mayday, Madeline Y., Reyes, Gustavo, Simon, Miriam R., Pearce, Emma M., Kim, Hanna, Shaw, Peter J., Rowan, Courtney M., Auletta, Jeffrey J., Martin, Paul L., Godder, Kamar, Duncan, Christine N., Lalefar, Nahal R., Kreml, Erin M., Hume, Janet R., Abdel-Azim, Hisham, Hurley, Caitlin, Cuvelier, Geoffrey D.E., Keating, Amy K., Qayed, Muna, Killinger, James S., Fitzgerald, Julie C., Hanna, Rabi, Mahadeo, Kris M., Quigg, Troy C., Satwani, Prakash, Castillo, Paul, Gertz, Shira J., Moore, Theodore B., Hanisch, Benjamin, Abdel-Mageed, Aly, Phelan, Rachel, Davis, Dereck B., Hudspeth, Michelle P., Yanik, Greg A., Pulsipher, Michael A., Sulaiman, Imran, Segal, Leopoldo N., Versluys, Birgitta A., Lindemans, Caroline A., Boelens, Jaap J., DeRisi, Joseph L., the Pediatric Transplantation and Cell Therapy Consortium, MS Algemene Pediatrie Onderzoek 2, Haematologie patientenzorg, SCT patientenzorg, Child Health, Infection & Immunity, Regenerative Medicine and Stem Cells, CTI Nierkens, Zinter, Matt S., Dvorak, Christopher C., Mayday, Madeline Y., Reyes, Gustavo, Simon, Miriam R., Pearce, Emma M., Kim, Hanna, Shaw, Peter J., Rowan, Courtney M., Auletta, Jeffrey J., Martin, Paul L., Godder, Kamar, Duncan, Christine N., Lalefar, Nahal R., Kreml, Erin M., Hume, Janet R., Abdel-Azim, Hisham, Hurley, Caitlin, Cuvelier, Geoffrey D.E., Keating, Amy K., Qayed, Muna, Killinger, James S., Fitzgerald, Julie C., Hanna, Rabi, Mahadeo, Kris M., Quigg, Troy C., Satwani, Prakash, Castillo, Paul, Gertz, Shira J., Moore, Theodore B., Hanisch, Benjamin, Abdel-Mageed, Aly, Phelan, Rachel, Davis, Dereck B., Hudspeth, Michelle P., Yanik, Greg A., Pulsipher, Michael A., Sulaiman, Imran, Segal, Leopoldo N., Versluys, Birgitta A., Lindemans, Caroline A., Boelens, Jaap J., DeRisi, Joseph L., and the Pediatric Transplantation and Cell Therapy Consortium

Published
2024

18. Outcomes from hematopoietic stem cell transplantation following treosulfan‐based conditioning: A clinical and pharmacokinetic analysis.

Author

Rosser, Sebastian P. A., Brewer, Alice, Gabriel, Melissa, Wong, Melanie, Chung, Jason, McLachlan, Andrew J., Nath, Christa E., Keogh, Steven J., and Shaw, Peter J.

Subjects
HEMATOPOIETIC stem cell transplantation, PHARMACOKINETICS
Abstract

Background: The aims of this study are to report our experience with treosulfan‐based conditioning regimens for patients with non‐malignant hematologic conditions, correlating clinical outcomes at different time points post‐transplant with treosulfan exposure (AUC). Methods: This study was a single‐center observational study investigating overall survival (OS), disease‐free survival (DFS), and event‐free survival (EFS) end‐points post‐transplant. The consequences of treosulfan AUC with respect to toxicity, correction of underlying disease, and long‐term chimerism were also explored using pharmacokinetic analysis. Results: Forty‐six patients received 49 transplants with treosulfan and fludarabine‐based conditioning between 2005 and 2023. Twenty‐four patients also received thiotepa. Donor chimerism was assessed on either whole blood or sorted cell lines at different time points post‐transplant. Thirty‐nine patients received treosulfan pharmacokinetic assessment to evaluate cumulative AUC, with five infants receiving real‐time assessment to facilitate daily dose adjustment. OS, DFS, and EFS were 87%, 81%, and 69%, respectively. Median follow‐up was 32.1 months (range 0.82–160 months) following transplant. Lower EFS was associated with patient age (<1 year; p =.057) and lower cumulative treosulfan dose (<42 g/m2; p =.003). Stable donor chimerism in B‐cell, NK‐cell, and granulocyte lineages at 1‐year post‐transplant were more prevalent in patients receiving thiotepa conditioning. Two infants required daily dose adjustment to treosulfan to avoid high AUC. Conclusions: Excellent clinical outcomes and stable chimerism were observed in this patient series. The addition of thiotepa conferred no significant toxicity and trended toward sustained ongoing donor engraftment. Correlating treosulfan AUC with long‐term patient outcomes is required. [ABSTRACT FROM AUTHOR]

Published
2024
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20. Subsequent neoplasms and late mortality in children undergoing allogeneic transplantation for nonmalignant diseases

Author

Kahn, Justine M., Brazauskas, Ruta, Tecca, Heather R., Bo-Subait, Stephanie, Buchbinder, David, Battiwala, Minoo, Flowers, Mary E.D., Savani, Bipin N., Phelan, Rachel, Broglie, Larisa, Abraham, Allistair A., Keating, Amy K., Daly, Andrew, Wirk, Baldeep, George, Biju, Alter, Blanche P., Ustun, Celalettin, Freytes, Cesar O., Beitinjaneh, Amer M., Duncan, Christine, Copelan, Edward, Hildebrandt, Gerhard C., Murthy, Hemant S., Lazarus, Hillard M., Auletta, Jeffery J., Myers, Kasiani C., Williams, Kirsten M., Page, Kristin M., Vrooman, Lynda M., Norkin, Maxim, Byrne, Michael, Diaz, Miguel Angel, Kamani, Naynesh, Bhatt, Neel S., Rezvani, Andrew, Farhadfar, Nosha, Mehta, Parinda A., Hematti, Peiman, Shaw, Peter J., Kamble, Rammurti T., Schears, Raquel, Olsson, Richard F., Hayashi, Robert J., Gale, Robert Peter, Mayo, Samantha J., Chhabra, Saurabh, Rotz, Seth J., Badawy, Sherif M., Ganguly, Siddhartha, Pavletic, Steven, Nishihori, Taiga, Prestidge, Tim, Agrawal, Vaibhav, Hogan, William J., Inamoto, Yoshihiro, Shaw, Bronwen E., and Satwani, Prakash

Published
2020
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23. 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

24. Incidence of Sinusoidal Obstruction Syndrome/Veno-Occlusive Disease and Treatment with Defibrotide in Allogeneic Transplantation: A Multicenter Australasian Registry Study

Author

Coutsouvelis, John, Kirkpatrick, Carl M., Dooley, Michael, Spencer, Andrew, Kennedy, Glen, Chau, Maggie, Huang, Gillian, Doocey, Richard, Copeland, Tandy-Sue, Do, Louis, Bardy, Peter, Kerridge, Ian, Cole, Theresa, Fraser, Chris, Perera, Travis, Larsen, Stephen R., Mason, Kate, O'Brien, Tracey A., Shaw, Peter J., Teague, Lochie, Butler, Andrew, Watson, Anne-Marie, Ramachandran, Shanti, Marsh, Jodie, Khan, Zulekha, and Hamad, Nada

Published
2023
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25. Central nervous system disorders after hematopoietic stem cell transplantation: a prospective study of the Infectious Diseases Working Party of EBMT

Author

Schmidt-Hieber, Martin, Engelhard, Dan, Ullmann, Andrew, Ljungman, Per, Maertens, Johan, Martino, Rodrigo, Rovira, Montserrat, Shaw, Peter J., Robin, Christine, Faraci, Maura, Byrne, Jenny, Schäfer-Eckart, Kerstin, Einsele, Hermann, Faber, Edgar, Rigacci, Luigi, Saccardi, Riccardo, Balaguer-Rosello, Aitana, Isaksson, Cecilia, Christopeit, Maximilian, Tridello, Gloria, Wang, Junfeng, Knelange, Nina, Mikulska, Malgorzata, Cesaro, Simone, and Styczynski, Jan

Published
2020
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26. More precisely defining risk peri-HCT in pediatric ALL: pre- vs post-MRD measures, serial positivity, and risk modeling

Author

Bader, Peter, Salzmann-Manrique, Emilia, Balduzzi, Adriana, Dalle, Jean-Hugues, Woolfrey, Ann E., Bar, Merav, Verneris, Michael R., Borowitz, Michael J., Shah, Nirali N., Gossai, Nathan, Shaw, Peter J., Chen, Allen R., Schultz, Kirk R., Kreyenberg, Hermann, Di Maio, Lucia, Cazzaniga, Gianni, Eckert, Cornelia, van der Velden, Vincent H.J., Sutton, Rosemary, Lankester, Arjan, Peters, Christina, Klingebiel, Thomas E., Willasch, Andre M., Grupp, Stephan A., and Pulsipher, Michael A.

Published
2019
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27. Harmonization of Busulfan Plasma Exposure Unit (BPEU): A Community-Initiated Consensus Statement

Author

McCune, Jeannine S., Quinones, Christine M., Ritchie, James, Carpenter, Paul A., van Maarseveen, Erik, Yeh, Rosa F., Anasetti, Claudio, Boelens, Jaap J., Hamerschlak, Nelson, Hassan, Moustapha, Kang, Hyoung Jin, Kanda, Yoshinobu, Paci, Angelo, Perales, Miguel-Angel, Shaw, Peter J., Seewaldt, Victoria L., Savani, Bipin N., Hsieh, Angela, Poon, Betsy, Mohty, Mohamad, Pulsipher, Michael A., Pasquini, Marcelo, and Dupuis, L. Lee

Published
2019
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28. Gonadal Function after Busulfan Compared with Treosulfan in Children and Adolescents Undergoing Allogeneic Hematopoietic Stem Cell Transplant

Author

Faraci, Maura, Diesch, Tamara, Labopin, Myriam, Dalissier, Arnaud, Lankester, Arian, Gennery, Andrew, Sundin, Mikael, Uckan-Cetinkaya, Duygu, Bierings, Marc, Peters, Anke M.J., Garwer, Martina, Schulz, Ansgar, Michel, Gerard, Giorgiani, Giovanna, Gruhn, Bernd, Locatelli, Franco, Giardino, Stefano, Uyttebroeck, Anne, Rialland, Fanny, Itäla-Remes, Maija, Dreger, Peter, Shaw, Peter J., Bordon, Victoria, Schlegel, Paul G., Mellgren, Karin, Moraleda, Jose M., Patrick, Katharine, Schneider, Pascale, Jubert, Charlotte, Lawitschka, Anita, Salooja, Nina, Basak, Grzegorz W., Corbacioglu, Selim, Duarte, Rafael, and Bader, Peter

Published
2019
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29. Ibrutinib Treatment of Pediatric Chronic Graft-versus-Host Disease: Primary Results from the Phase 1/2 iMAGINE Study

Author

Carpenter, Paul A., Kang, Hyoung Jin, Yoo, Keon Hee, Zecca, Marco, Cho, Bin, Lucchini, Giovanna, Nemecek, Eneida R., Schultz, Kirk R., Stepensky, Polina, Chaudhury, Sonali, Oshrine, Benjamin, Khaw, Seong Lin, Harris, Andrew C., Verna, Marta, Zubarovskaya, Liudmila, Lee, Yihua, Wahlstrom, Justin, Styles, Lori, Shaw, Peter J., and Dalle, Jean-Hugues

Published
2022
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30. Hematopoietic Stem Cell Transplantation for Hepatitis-associated Aplastic Anemia Following Liver Transplantation for Nonviral Hepatitis: A Retrospective Analysis and a Review of the Literature by the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation (SAAWP-EBMT)

Author

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

Published
2020
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31. Antimicrobial Resistance in Gram-Negative Rods Causing Bacteremia in Hematopoietic Stem Cell Transplant Recipients : Intercontinental Prospective Study of the Infectious Diseases Working Party of the European Bone Marrow Transplantation Group

Author

Averbuch, Diana, Tridello, Gloria, Hoek, Jennifer, Mikulska, Malgorzata, Akan, Hamdi, San Segundo, Lucrecia Yaňez, Pabst, Thomas, Özçelik, Tülay, Klyasova, Galina, Donnini, Irene, Wu, Depei, Gülbas, Zafer, Zuckerman, Tsila, de Sousa, Aida Botelho, Beguin, Yves, Xhaard, Aliénor, Bachy, Emmanuel, Ljungman, Per, de la Camara, Rafael, Rascon, Jelena, Camps, Isabel Ruiz, Vitek, Antonin, Patriarca, Francesca, Cudillo, Laura, Vrhovac, Radovan, Shaw, Peter J., Wolfs, Tom, O’Brien, Tracey, Avni, Batia, Silling, Gerda, Sabty, Firas Al, Graphakos, Stelios, Sankelo, Marja, Sengeloev, Henrik, Pillai, Srinivas, Matthes, Susanne, Melanthiou, Frederiki, Iacobelli, Simona, Styczynski, Jan, Engelhard, Dan, and Cesaro, Simone

Published
2017

32. Hematopoietic Cell Transplantation for Congenital Dyserythropoietic Anemia: A Report from the Pediatric Transplant and Cellular Therapy Consortium

Author

Rangarajan, Hemalatha G., Stanek, Joseph R., Abdel-Azim, Hisham, Modi, Arunkumar, Haight, Ann, McKinney, Christopher M., McKeone, Daniel J., Buchbinder, David K., Katsanis, Emmanuel, Abusin, Ghada A., Ahmed, Ibrahim, Law, Jason, Silva, Jorge Galvez, Mallhi, Kanwaldeep K., Burroughs, Lauri M., Shah, Niketa, Shaw, Peter J., Greiner, Robert, Shenoy, Shalini, Pulsipher, Michael A., and Abu-Arja, Rolla

Published
2022
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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. Upper and/or Lower Respiratory Tract Infection Caused by Human Metapneumovirus After Allogeneic Hematopoietic Stem Cell Transplantation

Author

Piñana, Jose Luis, primary, Tridello, Gloria, additional, Xhaard, Aliénor, additional, Wendel, Lotus, additional, Montoro, Juan, additional, Vazquez, Lourdes, additional, Heras, Inmaculada, additional, Ljungman, Per, additional, Mikulska, Malgorzata, additional, Salmenniemi, Urpu, additional, Perez, Ariadna, additional, Kröger, Nicolaus, additional, Cornelissen, Jan, additional, Sala, Elisa, additional, Martino, Rodrigo, additional, Geurten, Claire, additional, Byrne, Jenny, additional, Maertens, Johan, additional, Kerre, Tessa, additional, Martin, Murray, additional, Pascual, Maria Jesús, additional, Yeshurun, Moshe, additional, Finke, Jürgen, additional, Groll, Andreas H, additional, Shaw, Peter J, additional, Blijlevens, Nicole, additional, Arcese, William, additional, Ganser, Arnold, additional, Suarez-Lledo, Maria, additional, Alzahrani, Mohsen, additional, Choi, Goda, additional, Forcade, Edouard, additional, Paviglianiti, Annalisa, additional, Solano, Carlos, additional, Wachowiak, Jacek, additional, Zuckerman, Tsila, additional, Bader, Peter, additional, Clausen, Johannes, additional, Mayer, Jiri, additional, Schroyens, Wilfried, additional, Metafuni, Elisabetta, additional, Knelange, Nina, additional, Averbuch, Dina, additional, and de la Camara, Rafael, additional

Published
2023
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38. Risk Factors for Subsequent Central Nervous System Tumors in Pediatric Allogeneic Hematopoietic Cell Transplant: A Study from the Center for International Blood and Marrow Transplant Research (CIBMTR)

Author

Gabriel, Melissa, Shaw, Bronwen E., Brazauskas, Ruta, Chen, Min, Margolis, David A., Sengelov, Henrik, Dahlberg, Ann, Ahmed, Ibrahim A., Delgado, David, Lazarus, Hillard M., Gibson, Brenda, Myers, Kasiani C., Kamble, Rammurti T., Abdel-Mageed, Aly, Li, Chi-Kong, Flowers, Mary E.D., Battiwalla, Minoo, Savani, Bipin N., Majhail, Navneet, and Shaw, Peter J.

Published
2017
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46. Preparative Regimens

Author

Levine, John E., Shaw, Peter J., Smith, Franklin O., Smith, Franklin O., editor, Reaman, Gregory H., editor, and Racadio, Judy M., editor

Published
2014
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48. Pathobiological signatures of dysbiotic lung injury in pediatric patients undergoing stem cell transplantation

Author

Zinter, Matt S., Dvorak, Christopher C., Mayday, Madeline Y., Reyes, Gustavo, Simon, Miriam R., Pearce, Emma M., Kim, Hanna, Shaw, Peter J., Rowan, Courtney M., Auletta, Jeffrey J., Martin, Paul L., Godder, Kamar, Duncan, Christine N., Lalefar, Nahal R., Kreml, Erin M., Hume, Janet R., Abdel-Azim, Hisham, Hurley, Caitlin, Cuvelier, Geoffrey D. E., Keating, Amy K., Qayed, Muna, Killinger, James S., Fitzgerald, Julie C., Hanna, Rabi, Mahadeo, Kris M., Quigg, Troy C., Satwani, Prakash, Castillo, Paul, Gertz, Shira J., Moore, Theodore B., Hanisch, Benjamin, Abdel-Mageed, Aly, Phelan, Rachel, Davis, Dereck B., Hudspeth, Michelle P., Yanik, Greg A., Pulsipher, Michael A., Sulaiman, Imran, Segal, Leopoldo N., Versluys, Birgitta A., Lindemans, Caroline A., Boelens, Jaap J., and DeRisi, Joseph L.

Abstract

Hematopoietic cell transplantation (HCT) uses cytotoxic chemotherapy and/or radiation followed by intravenous infusion of stem cells to cure malignancies, bone marrow failure and inborn errors of immunity, hemoglobin and metabolism. Lung injury is a known complication of the process, due in part to disruption in the pulmonary microenvironment by insults such as infection, alloreactive inflammation and cellular toxicity. How microorganisms, immunity and the respiratory epithelium interact to contribute to lung injury is uncertain, limiting the development of prevention and treatment strategies. Here we used 278 bronchoalveolar lavage (BAL) fluid samples to study the lung microenvironment in 229 pediatric patients who have undergone HCT treated at 32 children’s hospitals between 2014 and 2022. By leveraging paired microbiome and human gene expression data, we identified high-risk BAL compositions associated with in-hospital mortality (P= 0.007). Disadvantageous profiles included bacterial overgrowth with neutrophilic inflammation, microbiome contraction with epithelial fibroproliferation and profound commensal depletion with viral and staphylococcal enrichment, lymphocytic activation and cellular injury, and were replicated in an independent cohort from the Netherlands (P= 0.022). In addition, a broad array of previously occult pathogens was identified, as well as a strong link between antibiotic exposure, commensal bacterial depletion and enrichment of viruses and fungi. Together these lung–immune system–microorganism interactions clarify the important drivers of fatal lung injury in pediatric patients who have undergone HCT. Further investigation is needed to determine how personalized interpretation of heterogeneous pulmonary microenvironments may be used to improve pediatric HCT outcomes.

Published
2024
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