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

Author

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

Subjects
Humans, Child, Female, Lung Injury, Male, Hematopoietic Stem Cell Transplantation, Child, Preschool, Adolescent, Bronchoalveolar Lavage Fluid, Dysbiosis, Microbiota, Infant, Lung
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 childrens 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

4. Multicenter Long-Term Follow-Up of Allogeneic Hematopoietic Cell Transplantation with Omidubicel: A Pooled Analysis of Five Prospective Clinical Trials.

Author

Lin, Chenyu, Schwarzbach, Aurelie, Sanz, Jaime, Montesinos, Pau, Stiff, Patrick, Parikh, Suhag, Brunstein, Claudio, Cutler, Corey, Lindemans, Caroline, Hanna, Rabi, Koh, Liang, Jagasia, Madan, Valcarcel, David, Maziarz, Richard, Keating, Amy, Hwang, William, Rezvani, Andrew, Karras, Nicole, Fernandes, Juliana, Rocha, Vanderson, Badell, Isabel, Ram, Ron, Schiller, Gary, Volodin, Leonid, Walters, Mark, Hamerschlak, Nelson, Cilloni, Daniela, Frankfurt, Olga, McGuirk, Joseph, Kurtzberg, Joanne, Sanz, Guillermo, Simantov, Ronit, and Horwitz, Mitchell

Subjects
Allogeneic stem cell transplantation, Clinical trial, Cord blood, Ex vivo expansion, Long-term follow-up, Humans, Follow-Up Studies, Prospective Studies, Hematopoietic Stem Cell Transplantation, Disease-Free Survival, Multicenter Studies as Topic
Abstract

Omidubicel is an umbilical cord blood (UCB)-derived ex vivo-expanded cellular therapy product that has demonstrated faster engraftment and fewer infections compared with unmanipulated UCB in allogeneic hematopoietic cell transplantation. Although the early benefits of omidubicel have been established, long-term outcomes remain unknown. We report on a planned pooled analysis of 5 multicenter clinical trials including 105 patients with hematologic malignancies or sickle cell hemoglobinopathy who underwent omidubicel transplantation at 26 academic transplantation centers worldwide. With a median follow-up of 22 months (range, .3 to 122 months), the 3-year estimated overall survival and disease-free survival were 62.5% and 54.0%, respectively. With up to 10 years of follow-up, omidubicel showed durable trilineage hematopoiesis. Serial quantitative assessments of CD3+, CD4+, CD8+, CD19+, CD116+CD56+, and CD123+ immune subsets revealed median counts remaining within normal ranges through up to 8 years of follow-up. Secondary graft failure occurred in 5 patients (5%) in the first year, with no late cases reported. One case of donor-derived myeloid neoplasm was reported at 40 months post-transplantation. This was also observed in a control arm patient who received only unmanipulated UCB. Overall, omidubicel demonstrated stable trilineage hematopoiesis, immune competence, and graft durability in extended follow-up.

Published
2023

5. Corticosteroids impair epithelial regeneration in immune-mediated intestinal damage

Author

Arnhold, Viktor, Chang, Winston Y., Jansen, Suze A., Thangavelu, Govindarajan, Calafiore, Marco, Vinci, Paola, Fu, Ya-Yuan, Ito, Takahiro, Takashima, Shuichiro, Egorova, Anastasiya, Kuttiyara, Jason, Perlstein, Adam, van Hoesel, Marliek, Liu, Chen, Blazar, Bruce R., Lindemans, Caroline A., and Hanash, Alan M.

Subjects
Thermo Fisher Scientific Inc., Scientific equipment and supplies industry -- Analysis, Gene expression -- Analysis, T cells -- Analysis, Corticosteroids -- Analysis, Genes -- Analysis, Intestines -- Analysis, Stem cells -- Analysis, Immunotherapy -- Analysis, Disease susceptibility -- Analysis, Health care industry, The Jackson Laboratory, Memorial Sloan-Kettering Cancer Center
Abstract

Corticosteroid treatment (CST) failure is associated with poor outcomes for patients with gastrointestinal (GI) graft-versus-host disease (GVHD). CST is intended to target the immune system, but the glucocorticoid receptor (GR) is widely expressed, including within the intestines, where its effects are poorly understood. Here, we report that corticosteroids (CS) directly targeted intestinal epithelium, potentially worsening immune-mediated GI damage. CS administered to mice in vivo and intestinal organoid cultures ex vivo reduced epithelial proliferation. Following irradiation, immediate CST mitigated GI damage but delayed treatment attenuated regeneration and exacerbated damage. In a murine steroid-refractory (SR) GVHD model, CST impaired epithelial regeneration, worsened crypt loss, and reduced intestinal stem cell (ISC) frequencies. CST also exacerbated immune-mediated damage in organoid cultures with SR, GR-deficient T cells or IFN-[gamma]. These findings correlated with CS-dependent changes in apoptosis-related gene expression and STAT3-related epithelial proliferation. Conversely, IL-22 administration enhanced STAT3 activity and overcame CS-mediated attenuation of regeneration, reducing crypt loss and promoting ISC expansion in steroid-treated mice with GVHD. Therefore, CST has the potential to exacerbate GI damage if it fails to control the damage-inducing immune response, but this risk may be countered by strategies augmenting epithelial regeneration, thus providing a rationale for clinical approaches combining such tissue- targeted therapies with immunosuppression., Introduction The epithelial lining of the gastrointestinal (GI) tract undergoes turnover every 5-7 days (1). This renewal is maintained by the cycling of leucine-rich, repeat-containing GPCR 5-positive ([Lgr5.sup.+]) intestinal stem [...]

Published
2024
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10. Pulmonary microbiome and gene expression signatures differentiate lung function in pediatric hematopoietic cell transplant candidates

Author

Zinter, Matt S, Versluys, A Birgitta, Lindemans, Caroline A, Mayday, Madeline Y, Reyes, Gustavo, Sunshine, Sara, Chan, Marilynn, Fiorino, Elizabeth K, Cancio, Maria, Prevaes, Sabine, Sirota, Marina, Matthay, Michael A, Kharbanda, Sandhya, Dvorak, Christopher C, Boelens, Jaap J, and DeRisi, Joseph L

Subjects
Lung, Genetics, Pediatric, Transplantation, Clinical Research, Rare Diseases, Aetiology, 2.1 Biological and endogenous factors, Respiratory, Good Health and Well Being, Child, Hematopoietic Stem Cell Transplantation, Humans, Microbiota, Pulmonary Fibrosis, Transcriptome, Biological Sciences, Medical and Health Sciences
Abstract

Impaired baseline lung function is associated with mortality after pediatric allogeneic hematopoietic cell transplantation (HCT), yet limited knowledge of the molecular pathways that characterize pretransplant lung function has hindered the development of lung-targeted interventions. In this study, we quantified the association between bronchoalveolar lavage (BAL) metatranscriptomes and paired pulmonary function tests performed a median of 1 to 2 weeks before allogeneic HCT in 104 children in The Netherlands. Abnormal pulmonary function was recorded in more than half the cohort, consisted most commonly of restriction and impaired diffusion, and was associated with both all-cause and lung injury-related mortality after HCT. Depletion of commensal supraglottic taxa, such as Haemophilus, and enrichment of nasal and skin taxa, such as Staphylococcus, in the BAL microbiome were associated with worse measures of lung capacity and gas diffusion. In addition, BAL gene expression signatures of alveolar epithelial activation, epithelial-mesenchymal transition, and down-regulated immunity were associated with impaired lung capacity and diffusion, suggesting a postinjury profibrotic response. Detection of microbial depletion and abnormal epithelial gene expression in BAL enhanced the prognostic utility of pre-HCT pulmonary function tests for the outcome of post-HCT mortality. These findings suggest a potentially actionable connection between microbiome depletion, alveolar injury, and pulmonary fibrosis in the pathogenesis of pre-HCT lung dysfunction.

Published
2022

11. Survival in primary hemophagocytic lymphohistiocytosis, 2016 to 2021: etoposide is better than its reputation

Author

Böhm, Svea, Wustrau, Katharina, Pachlopnik Schmid, Jana, Prader, Seraina, Ahlmann, Martina, Yacobovich, Joanne, Beier, Rita, Speckmann, Carsten, Behnisch, Wolfgang, Ifversen, Marianne, Jordan, Michael, Marsh, Rebecca, Naumann-Bartsch, Nora, Mauz-Körholz, Christine, Hönig, Manfred, Schulz, Ansgar, Malinowska, Iwona, Hines, Melissa, Nichols, Kim E., Gil-Herrera, Juana, Talano, Julie-An, Crooks, Bruce, Formankova, Renata, Jorch, Norbert, Bakhtiar, Shahrzad, Kühnle, Ingrid, Streiter, Monika, Nathrath, Michaela, Russo, Alexandra, Dürken, Matthias, Lang, Peter, Lindemans, Caroline, Henter, Jan-Inge, Lehmberg, Kai, and Ehl, Stephan

Published
2024
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12. Newborn screening in metachromatic leukodystrophy – European consensus-based recommendations on clinical management

Author

Laugwitz, Lucia, Schoenmakers, Daphne H., Adang, Laura A., Beck-Woedl, Stefanie, Bergner, Caroline, Bernard, Geneviève, Bley, Annette, Boyer, Audrey, Calbi, Valeria, Dekker, Hanka, Eichler, Florian, Eklund, Erik, Fumagalli, Francesca, Gavazzi, Francesco, Grønborg, Sabine W., van Hasselt, Peter, Langeveld, Mirjam, Lindemans, Caroline, Mochel, Fanny, Oberg, Andreas, Ram, Dipak, Saunier-Vivar, Elise, Schöls, Ludger, Scholz, Michael, Sevin, Caroline, Zerem, Ayelet, Wolf, Nicole I., and Groeschel, Samuel

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

Author

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

Published
2023
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16. Meningococcal ACWY conjugate vaccine immunogenicity and safety in adolescents with juvenile idiopathic arthritis and inflammatory bowel disease: A prospective observational cohort study

Author

Ohm, Milou, van Straalen, Joeri W., Zijlstra, Marieke, de Joode-Smink, Gerrie, Sellies, Anne Jasmijn, Swart, Joost F., Vastert, Sebastiaan J., van Montfrans, Joris M., Bartels, Marije, van Royen-Kerkhof, Annet, Wildenbeest, Joanne G., Lindemans, Caroline A., Wolters, Victorien M., Wennink, Roos A.W., de Boer, Joke H., Knol, Mirjam J., Heijstek, Marloes W., Sanders, Elisabeth A.M., Verduyn-Lunel, Frans M., Berbers, Guy A.M., Wulffraat, Nico M., and Jansen, Marc H.A.

Published
2023
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17. Bloodstream Infection Due to Vancomycin-resistant Enterococcus Is Associated With Increased Mortality After Hematopoietic Cell Transplantation for Acute Leukemia and Myelodysplastic Syndrome: A Multicenter, Retrospective Cohort Study

Author

Papanicolaou, Genovefa A, Ustun, Celalettin, Young, Jo-Anne H, Chen, Min, Kim, Soyoung, Ahn, Kwang Woo, Komanduri, Krishna, Lindemans, Caroline, Auletta, Jeffery J, Riches, Marcie L, Abdel-Azim, Hisham, Ahmed, Ibrahim A, Aljurf, Mahmoud, Antin, Joseph, Ballen, Karen Kuhn, Beitinjaneh, Amer, Brown, Valerie I, Cerny, Jan, Champlin, Richard, Chao, Nelson, Chhabra, Saurabh, Dahi, Parastoo B, Daly, Andrew, Dandoy, Christopher, Dvorak, Christopher C, Forman, Stephen, Ganguly, Siddhartha, Hashmi, Shahrukh K, Kharfan-Dabaja, Mohamed A, Lazarus, Hillard, Ljungman, Per, Malone, Adriana K, Murthy, Nishihori, Taiga, Page, Kristin, Pingali, Ravi, Reddy, Vijay, Saad, Ayman, Savani, Bipin N, Seftel, Matthew, Szer, Jeffrey, Vij, Ravi, Weisdorf, Daniel, William, Basem M, Williams, Kirsten, Wirk, Baldeep, and Yared, Jean

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Clinical Sciences, Cancer, Vaccine Related, Transplantation, Rare Diseases, Hematology, Emerging Infectious Diseases, Clinical Research, Good Health and Well Being, Adolescent, Adult, Aged, Anti-Bacterial Agents, Bacteremia, Child, Child, Preschool, Gram-Positive Bacterial Infections, Hematopoietic Stem Cell Transplantation, Humans, Infant, Leukemia, Myeloid, Acute, Middle Aged, Myelodysplastic Syndromes, Retrospective Studies, Transplantation Conditioning, Vancomycin, Vancomycin-Resistant Enterococci, Young Adult, vancomycin-resistant Enterococcus, bacteremia, hematopoietic stem cell transplantation, mortality, CIBMTR® Infection and Immune Reconstitution Working Committee, Biological Sciences, Medical and Health Sciences, Microbiology, Clinical sciences
Abstract

BackgroundWe examined the impact of vancomycin-resistant Enterococcus (VRE) bloodstream infection (BSI) on outcomes of allogeneic hematopoietic cell transplantation (HCT) utilizing the Center for International Blood and Marrow Transplant Research database.MethodsAdult and pediatric patients (N = 7128) who underwent first HCT for acute leukemia or myelodysplastic syndrome from 2008 through 2012 were analyzed as 3 groups-VRE BSI, non-VRE BSI, without BSI-according to BSI status at 100 days (D100) after allogeneic HCT. Multivariable models examined the effect of VRE BSI for overall survival (OS) and nonrelapse mortality (NRM) at 1 year.ResultsOf 7128 patients, 258 (3.2%) had VRE BSI, 2398 (33.6%) had non-VRE BSI, and 4472 (63%) had no BSI. The median time to VRE BSI and non-VRE BSI were D11 and D15, respectively. Compared with non-VRE BSI patients, VRE BSI patients were older, had advanced-stage acute leukemia, and received umbilical cord blood (UCB) allografts. In multivariable models, VRE BSI was associated with lower OS (relative risk [RR], 2.9;(99% confidence interval [CI], 2.2-3.7) and increased NRM (RR, 4.7; 99% CI, 3.6-6.2) (P < .0001) for both. Other predictors for worse OS and increased NRM were non-VRE BSI, older age, advanced disease stage, UCB allograft, - mismatch, comorbidity index ≥3, and cytomegalovirus seropositivity (P < .001 for all variables).ConclusionsVRE BSI is associated with lowest OS and highest NRM compared with patients without BSI or non-VRE BSI. Novel interventions that address the pathophysiology of VRE BSI have the potential of improving survival after HCT.

Published
2019

19. An exploratory open-label multicentre phase I/II trial evaluating the safety and efficacy of postnatal or prenatal and postnatal administration of allogeneic expanded fetal mesenchymal stem cells for the treatment of severe osteogenesis imperfecta in infants and fetuses: the BOOSTB4 trial protocol

Author

Sagar, Rachel L, primary, Åström, Eva, additional, Chitty, Lyn S, additional, Crowe, Belinda, additional, David, Anna L, additional, DeVile, Catherine, additional, Forsmark, Annabelle, additional, Franzen, Vera, additional, Hermeren, Göran, additional, Hill, Melissa, additional, Johansson, Mats, additional, Lindemans, Caroline, additional, Lindgren, Peter, additional, Nijhuis, Wouter, additional, Oepkes, Dick, additional, Rehberg, Mirko, additional, Sahlin, Nils-Eric, additional, Sakkers, Ralph, additional, Semler, O, additional, Sundin, Mikael, additional, Walther-Jallow, Lilian, additional, Verweij, E J T Joanne, additional, Westgren, Magnus, additional, and Götherström, Cecilia, additional

Published
2024
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21. 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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22. Increased overall and bacterial infections following myeloablative allogeneic HCT for patients with AML in CR1

Author

Ustun, Celalettin, Kim, Soyoung, Chen, Min, Beitinjaneh, Amer M, Brown, Valerie I, Dahi, Parastoo B, Daly, Andrew, Diaz, Miguel Angel, Freytes, Cesar O, Ganguly, Siddhartha, Hashmi, Shahrukh, Hildebrandt, Gerhard C, Lazarus, Hillard M, Nishihori, Taiga, Olsson, Richard F, Page, Kristin M, Papanicolaou, Genovefa, Saad, Ayman, Seo, Sachiko, William, Basem M, Wingard, John R, Wirk, Baldeep, Yared, Jean A, Perales, Miguel-Angel, Auletta, Jeffery J, Komanduri, Krishna V, Lindemans, Caroline A, and Riches, Marcie L

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Stem Cell Research - Nonembryonic - Human, Hematology, Infectious Diseases, Transplantation, Stem Cell Research, Clinical Research, Infection, Good Health and Well Being, Adult, Bacterial Infections, Hematopoietic Stem Cell Transplantation, Humans, Infections, Leukemia, Myeloid, Acute, Myeloablative Agonists, Remission Induction, Time Factors, Transplantation Conditioning, Transplantation, Homologous, Treatment Outcome, Cardiovascular medicine and haematology
Abstract

Presumably, reduced-intensity/nonmyeloablative conditioning (RIC/NMA) for allogeneic hematopoietic cell transplantation (alloHCT) results in reduced infections compared with myeloablative conditioning (MAC) regimens; however, published evidence is limited. In this Center for International Blood and Marrow Transplant Research study, 1755 patients (aged ≥40 years) with acute myeloid leukemia in first complete remission were evaluated for infections occurring within 100 days after T-cell replete alloHCT. Patients receiving RIC/NMA (n = 777) compared with those receiving MAC (n = 978) were older and underwent transplantation more recently; however, the groups were similar regarding Karnofsky performance score, HCT-comorbidity index, and cytogenetic risk. One or more infections occurred in 1045 (59.5%) patients (MAC, 595 [61%]; RIC/NMA, 450 [58%]; P = .21) by day 100. The median time to initial infection after MAC conditioning occurred earlier (MAC, 15 days [range,

Published
2019

23. Virus detection in the cerebrospinal fluid of hematopoietic stem cell transplant recipients is associated with poor patient outcomes: a CIBMTR contemporary longitudinal study

Author

Abidi, Maheen Z, Hari, Parameswaran, Chen, Min, Kim, Soyoung, Battiwala, Minoo, Dahi, Parastoo Bahrami, Diaz, Miguel Angel, Gale, Robert Peter, Ganguly, Siddhartha, Gergis, Usama, Green, Jaime, Hildebrandt, Gerhard, Hill, Joshua A, Komanduri, Krishna, Lazarus, Hillard, Marks, David, Nishihori, Taiga, Olsson, Richard, Seo, Sachiko, Ustun, Celalettin, Yared, Jean, Yin, Dwight, Wingard, John, Wirk, Baldeep Mona, Auletta, Jeffrey, Lindemans, Caroline, and Riches, Marcie

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Immunology, Stem Cell Research, HIV/AIDS, Regenerative Medicine, Stem Cell Research - Nonembryonic - Human, Cancer, Infectious Diseases, Transplantation, Infection, Good Health and Well Being, Adolescent, Adult, Aged, Child, Child, Preschool, DNA, Viral, Female, Hematopoietic Stem Cell Transplantation, Humans, Longitudinal Studies, Male, Middle Aged, Transplantation Conditioning, Virus Diseases, Young Adult, Clinical Sciences, Oncology and Carcinogenesis, Cardiovascular medicine and haematology, Oncology and carcinogenesis
Abstract

Limited data exist on characteristics of central nervous system viruses (CNS-V) in allogeneic hematopoietic stem cell transplant (HCT) recipients. Between 2007 and 2015, the Center for International Blood and Marrow Transplant Research (CIBMTR) received information on 27,532 patients undergoing HCT. Of these, centers reported 165 HCT recipients with CNS-V detected in cerebrospinal fluid within 6 months after HCT. CNS viruses predominantly included human herpes virus 6 (HHV-6) (73%), followed by Epstein-Barr Virus (10%), cytomegalovirus (3%), varicella zoster virus (3%), herpes simplex virus (3%) and Adenovirus (3%). Median time of viral detection in CNS was 31 days after HCT; and viral detection was earlier in patients with CNS HHV-6. Concurrent viremia occurred in 52% of patients. Cord blood transplant recipients (CBT) accounted for the majority (53%) of patients with CNS-V. Myeloablative conditioning (65%), use of fludarabine (63%), or use of anti-thymocyte globulin (61%) were also predominant. Overall survival from the time of detection of CNS-V was 50% at 6 months and 30% at 5 years. Infections were the leading cause of death (32%). In summary, CBT recipients predominated in the population with CNS-V. Outcomes after CNS-V were poor with significant mortality seen in the first 6 months.

Published
2019

24. Bacterial blood stream infections (BSIs), particularly post-engraftment BSIs, are associated with increased mortality after allogeneic hematopoietic cell transplantation

Author

Ustun, Celalettin, Young, Jo-Anne H, Papanicolaou, Genovefa A, Kim, Soyoung, Ahn, Kwang Woo, Chen, Min, Abdel-Azim, Hisham, Aljurf, Mahmoud, Beitinjaneh, Amer, Brown, Valerie, Cerny, Jan, Chhabra, Saurabh, Kharfan-Dabaja, Mohamed A, Dahi, Parastoo B, Daly, Andrew, Dandoy, Christopher E, Dvorak, Christopher C, Freytes, Cesar O, Hashmi, Shahrukh, Lazarus, Hillard, Ljungman, Per, Nishihori, Taiga, Page, Kristin, Pingali, Sai RK, Saad, Ayman, Savani, Bipin N, Weisdorf, Daniel, Williams, Kirsten, Wirk, Baldeep, Auletta, Jeffery J, Lindemans, Caroline A, Komanduri, Krishna, and Riches, Marcie

Subjects
Biomedical and Clinical Sciences, Clinical Sciences, Clinical Research, Hematology, Good Health and Well Being, Adolescent, Adult, Bacteremia, Child, Female, Hematopoietic Stem Cell Transplantation, Humans, Male, Middle Aged, Mortality, Risk Factors, Transplantation Conditioning, Transplantation, Homologous, Young Adult, Oncology and Carcinogenesis, Immunology, Cardiovascular medicine and haematology, Oncology and carcinogenesis
Abstract

We analyzed CIBMTR data to evaluate the incidence of non-relapse mortality (NRM) and association with overall survival (OS) for bacterial blood stream infections (BSIs) occurring within 100 days of alloHCT in 2 different phases: pre-/peri-engraftment (BSI very early phase, BSI-VEP) and BSI post-engraftment (BSI occurring between 2 weeks after engraftment and day 100, late early phase, BSI-LEP). Of the 7128 alloHCT patients, 2656 (37%) had ≥1 BSI by day 100. BSI-VEP, BSI-LEP, and BSI-Both constituted 56% (n = 1492), 31% (n = 824), and 13% (n = 340) of total BSI, respectively. Starting in 2009, we observed a gradual decline in BSI incidence through 2012 (61-48%). Patients with BSI-VEP were more likely to receive a myeloablative conditioning (MAC) regimen with total body irradiation (TBI). NRM was significantly higher in patients with any BSI (RR 1.82 95% CI 1.63-2.04 for BSI-VEP, RR 2.46, 95% CI 2.05-2.96 for BSI-LEP, and RR 2.29, 95% CI 1.87-2.81 for BSI-Both) compared with those without BSI. OS was significantly lower in patients with any BSI compared with patients without BSI (RR 1.36, 95% CI 1.26-1.47 for BSI-VEP; RR 1.83, 95% CI 1.58-2.12 for BSI-LEP: RR 1.66, 95% CI 1.43-1.94 for BSI-Both). BSIs within day 100 after alloHCT are common and remain a risk factor for mortality.

Published
2019

25. Fludarabine exposure predicts outcome after CD19 CAR T-cell therapy in children and young adults with acute leukemia

Author

Dekker, Linde, Calkoen, Friso G., Jiang, Yilin, Blok, Hilly, Veldkamp, Saskia R., De Koning, Coco, Spoon, Maike, Admiraal, Rick, Hoogerbrugge, Peter, Vormoor, Britta, Vormoor, H. Josef, Visscher, Henk, Bierings, Marc, Van Der Vlugt, Marieke, Van Tinteren, Harm, Nijstad, A. Laura, Huitema, Alwin D.R., Van Der Elst, Kim C.M., Pieters, Rob, Lindemans, Caroline A., and Nierkens, Stefan

Published
2022
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26. Modified Delphi procedure-based expert consensus on endpoints for an international disease registry for Metachromatic Leukodystrophy: The European Metachromatic Leukodystrophy initiative (MLDi)

Author

Schoenmakers, Daphne H., Beerepoot, Shanice, van den Berg, Sibren, Adang, Laura, Bley, Annette, Boelens, Jaap-Jan, Fumagalli, Francesca, Goettsch, Wim G., Grønborg, Sabine, Groeschel, Samuel, van Hasselt, Peter M., Hollak, Carla E. M., Lindemans, Caroline, Mochel, Fanny, Mol, Peter G. M., Sevin, Caroline, Zerem, Ayelet, Schöls, Ludger, and Wolf, Nicole I.

Published
2022
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27. 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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29. Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy

Author

Chiesa, Robert, Boelens, Jaap Jan, Duncan, Christine N., Kühl, Jörn-Sven, Sevin, Caroline, Kapoor, Neena, Prasad, Vinod K., Lindemans, Caroline A., Jones, Simon A., Amartino, Hernan M., Algeri, Mattia, Bunin, Nancy, Diaz-de-Heredia, Cristina, Loes, Daniel J., Shamir, Esther, Timm, Alison, McNeil, Elizabeth, Dietz, Andrew C., and Orchard, Paul J.

Published
2022
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31. Longitudinal volumetric analysis of gray matter atrophy in metachromatic leukodystrophy.

Author

Al‐Saady, Murtadha L., Galabova, Hristina, Schoenmakers, Daphne H., Beerepoot, Shanice, Lindemans, Caroline, van Hasselt, Peter M., van der Knaap, Marjo S., Wolf, Nicole I., and Pouwels, Petra J. W.

Abstract

Metachromatic leukodystrophy (MLD) is an inherited lysosomal storage disorder characterized by arylsulfatase A (ASA) deficiency, leading to sulfatide accumulation and myelin degeneration in the central nervous system. While primarily considered a white matter (WM) disease, gray matter (GM) is also affected in MLD, and hematopoietic stem cell transplantation (HSCT) may have limited effect on GM atrophy. We cross‐sectionally and longitudinally studied GM volumes using volumetric MRI in a cohort of 36 (late‐infantile, juvenile and adult type) MLD patients containing untreated and HSCT treated subjects. Cerebrum, cortical GM, (total) CSF, cerebellum, deep gray matter (DGM) (excluding thalamus) and thalamus volumes were analyzed. Longitudinal correlations with measures of cognitive and motor functioning were assessed. Cross‐sectionally, juvenile and adult type patients (infantiles excluded based on limited numbers) were compared with controls at earliest scan, before possible treatment. Patients had lower cerebrum, cortical GM, DGM and thalamus volumes. Differences were most pronounced for adult type patients. Longitudinal analyses showed substantial and progressive atrophy of all regions and increase of CSF in untreated patients. Similar, albeit less pronounced, effects were seen in treated patients for cerebrum, cortical GM, CSF and thalamus volumes. Deterioration in motor performance (all patients) was related to atrophy, and increase of CSF, in all regions. Cognitive functioning (data available for treated patients) was related to cerebral, cortical GM and thalamus atrophy; and to CSF increase. Our findings illustrate the importance of recognizing GM pathology as a potentially substantial, clinically relevant part of MLD, apparently less amenable to treatment. [ABSTRACT FROM AUTHOR]

Published
2024
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33. International Recommendations for the Diagnosis and Management of Patients With Adrenoleukodystrophy: A Consensus-Based Approach

Author

Engelen, Marc, van Ballegoij, Wouter J.C., Mallack, Eric James, Van Haren, Keith P., Köhler, Wolfgang, Salsano, Ettore, van Trotsenburg, A.S.P., Mochel, Fanny, Sevin, Caroline, Regelman, Molly O, Tritos, Nicholas A, Halper, Alyssa, Lachmann, Robin H, Davison, James, Raymond, Gerald V., Lund, Troy, Orchard, Paul J., Kuehl, Joern-Sven, Lindemans, Caroline A., Caruso, Paul, Turk, Bela Rui, Moser, Ann B., Vaz, Frederic M, Ferdinandusse, Sacha, Kemp, Stephan, Fatemi, Ali, Eichler, Florian S., and Huffnagel, Irene C.

Published
2022
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35. Inventory of current practices regarding hematopoietic stem cell transplantation in metachromatic leukodystrophy in Europe and neighboring countries

Author

Schoenmakers, Daphne H., primary, Mochel, Fanny, additional, Adang, Laura A., additional, Boelens, Jaap-Jan, additional, Calbi, Valeria, additional, Eklund, Erik A., additional, Grønborg, Sabine W., additional, Fumagalli, Francesca, additional, Groeschel, Samuel, additional, Lindemans, Caroline, additional, Sevin, Caroline, additional, Schöls, Ludger, additional, Ram, Dipak, additional, Zerem, Ayelet, additional, Graessner, Holm, additional, and Wolf, Nicole I., additional

Published
2024
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37. Evaluation of Mobilization, Apheresis, and Conditioning Regimen and Engraftment in Patients Receiving One-Time Gene Therapy with Elivaldogene Autotemcel (Eli-cel) for Cerebral Adrenoleukodystrophy (CALD)

Author

Duncan, Christine N., primary, Orchard, Paul J., additional, Eichler, Florian S., additional, Kühl, Jörn-Sven, additional, De Oliveira, Satiro N., additional, Thrasher, Adrian J., additional, Chiesa, Robert, additional, Dalle, Jean-Hugues, additional, Shah, Ami J., additional, Locatelli, Franco, additional, Smith, Nicholas J.C., additional, Fernandes, Juliana Folloni, additional, Amartino, Hernán M., additional, Lindemans, Caroline A., additional, Zhao, Jack, additional, Singh, Ajay, additional, Thakar, Himal L., additional, Prasad, Vinod K., additional, Dietz, Andrew C., additional, and Williams, David A., additional

Published
2024
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38. Design of a Multi-Center Randomized Active Controlled Phase 3 Clinical Trial (HURCULES) Evaluating the Safety and Efficacy of OTL-203 in Patients with MPS-IH Versus Standard of Care with Allogeneic Hematopoietic Stem Cell Transplantation

Author

Gupta, Ashish O., primary, Wynn, Robert F, additional, Kharbanda, Sandhya, additional, Lindemans, Caroline A., additional, Ahrens-Nicklas, Rebecca, additional, van Hasselt, Peter M., additional, Lund, Troy C., additional, Olson, Timothy S., additional, Tucci, Francesca, additional, Martin, Leonie, additional, Boeglin, Nathalie, additional, Brooks, Jean, additional, Syonmez, Su, additional, Campbell, Laura, additional, Harmatz, Paul, additional, Jones, Simon A., additional, Orchard, Paul J., additional, and Bernardo, Maria Ester, additional

Published
2024
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39. Real World Use of the Pediatric Disease Risk Index for Allogeneic Hematopoietic Cell Transplantation in Patients with Acute Lymphoblastic Leukemia and Acute Myeloid Leukemia: A Multicenter Study

Author

Lucas, Alexandre G. Troullioud, primary, Bhoopalan, Senthil Velan, additional, Nierkens, Stefan, additional, de Koning, Coco, additional, Keerthi, Dinesh, additional, Seetharaman, Aditya, additional, Naik, Swati, additional, Prockop, Susan E., additional, Sharma, Akshay, additional, Boelens, Jaap Jan, additional, and Lindemans, Caroline A., additional

Published
2024
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41. Model-Based Dosing of Anti-Thymocyte Globulin in Pediatric Allogeneic Hematopoietic Cell Transplantation Improves Survival Chances: Updated Results from the Single Arm Phase II Parachute-Trial Combined with Real-World Data

Author

Admiraal, Rick, primary, Nierkens, Stefan, additional, Bierings, Marc, additional, Belderbos, Mirjam, additional, Huitema, Alwin D.R., additional, Bredius, Robbert, additional, Jiang, Yilin, additional, Curran, Kevin J., additional, Harris, Andrew C., additional, Scaradavou, Andromachi, additional, Cancio, Maria I., additional, Klein, Elizabeth, additional, Kollen, Wouter, additional, Bresters, Dorine, additional, Calkoen, Friso, additional, Versluijs, A. Birgitta, additional, Zwaan, Christian M., additional, Boelens, Jaap-Jan, additional, and Lindemans, Caroline A., additional

Published
2024
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46. CD4+ T-cell reconstitution predicts survival outcomes after acute graft-versus-host-disease: a dual-center validation

Author

de Koning, Coco, Prockop, Susan, van Roessel, Ichelle, Kernan, Nancy, Klein, Elizabeth, Langenhorst, Jurgen, Szanto, Celina, Belderbos, Mirjam, Bierings, Marc, Boulad, Farid, Bresters, Dorine, Cancio, Maria, Curran, Kevin, Kollen, Wouter, O'Reilly, Richard, Scaradavou, Andromachi, Spitzer, Barbara, Versluijs, Birgitta, Huitema, Alwin, Lindemans, Caroline, Nierkens, Stefan, and Boelens, Jaap Jan

Published
2021
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49. Infection Rates among Acute Leukemia Patients Receiving Alternative Donor Hematopoietic Cell Transplantation

Author

Ballen, Karen, Ahn, Kwang Woo, Chen, Min, Abdel-Azim, Hisham, Ahmed, Ibrahim, Aljurf, Mahmoud, Antin, Joseph, Bhatt, Ami S, Boeckh, Michael, Chen, George, Dandoy, Christopher, George, Biju, Laughlin, Mary J, Lazarus, Hillard M, MacMillan, Margaret L, Margolis, David A, Marks, David I, Norkin, Maxim, Rosenthal, Joseph, Saad, Ayman, Savani, Bipin, Schouten, Harry C, Storek, Jan, Szabolcs, Paul, Ustun, Celalettin, Verneris, Michael R, Waller, Edmund K, Weisdorf, Daniel J, Williams, Kirsten M, Wingard, John R, Wirk, Baldeep, Wolfs, Tom, Young, Jo-Anne H, Auletta, Jeffrey, Komanduri, Krishna V, Lindemans, Caroline, and Riches, Marcie L

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Immunology, Rare Diseases, Stem Cell Research, Infectious Diseases, Clinical Research, Regenerative Medicine, Transplantation, Hematology, Stem Cell Research - Nonembryonic - Human, 2.2 Factors relating to the physical environment, Aetiology, Infection, Good Health and Well Being, Acute Disease, Adolescent, Adult, Aged, Cord Blood Stem Cell Transplantation, Female, Hematopoietic Stem Cell Transplantation, Histocompatibility, Humans, Incidence, Infections, Leukemia, Male, Middle Aged, Retrospective Studies, Survival Analysis, Unrelated Donors, Young Adult, Umbilical cord blood, Clinical Sciences, Cardiovascular medicine and haematology
Abstract

Alternative graft sources (umbilical cord blood [UCB], matched unrelated donors [MUD], or mismatched unrelated donors [MMUD]) enable patients without a matched sibling donor to receive potentially curative hematopoietic cell transplantation (HCT). Retrospective studies demonstrate comparable outcomes among different graft sources. However, the risk and types of infections have not been compared among graft sources. Such information may influence the choice of a particular graft source. We compared the incidence of bacterial, viral, and fungal infections in 1781 adults with acute leukemia who received alternative donor HCT (UCB, n= 568; MUD, n = 930; MMUD, n = 283) between 2008 and 2011. The incidences of bacterial infection at 1 year were 72%, 59%, and 65% (P

Published
2016

50. Survival in primary hemophagocytic lymphohistiocytosis 2016-2021: etoposide is better than its reputation

Author

Böhm, Svea, primary, Wustrau, Katharina, additional, Pachlopnik Schmid, Jana, additional, Prader, Seraina, additional, Ahlman, Martina, additional, Yacobovich, Joanne, additional, Beier, Rita, additional, Speckmann, Carsten, additional, Behnisch, Wolfgang, additional, Ifversen, Marianne, additional, Jordan, Michael B., additional, Marsh, Rebecca A, additional, Naumann-Bartsch, Nora, additional, Mauz-Koerholz, Christine, additional, Honig, Manfred, additional, Schulz, Ansgar S, additional, Malinowska, Iwona, additional, Hines, Melissa Ruth, additional, Nichols, Kim E, additional, Gil-Herrera, Juana, additional, Talano, Julie-An, additional, Crooks, Bruce, additional, Formankova, Renata, additional, Jorch, Norbert, additional, Bakhtiar, Shahrzad, additional, Kühnle, Ingrid, additional, Streiter, Monika, additional, Nathrath, Michaela, additional, Russo, Alexandra, additional, Duerken, Matthias, additional, Lang, Peter, additional, Lindemans, Caroline A., additional, Henter, Jan-Inge, additional, Lehmberg, Kai, additional, and Ehl, Stephan, additional

Published
2023
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