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3. Low rate of nonrelapse mortality in under-4-year-olds with ALL given chemotherapeutic conditioning for HSCT: a phase 3 FORUM study

Author

Bader, Peter, Pötschger, Ulrike, Dalle, Jean-Hugues, Moser, Laura M., Balduzzi, Adriana, Ansari, Marc, Buechner, Jochen, Güngör, Tayfun, Ifversen, Marianne, Krivan, Gergely, Pichler, Herbert, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr, Stein, Jerry, Heusel, Jan Robert, Truong, Tony, Wachowiak, Jacek, Yesilipek, Akif, Locatelli, Franco, and Peters, Christina

Published
2024
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5. Umbilical Cord Blood Transplantation for Fanconi Anemia With a Special Focus on Late Complications: a Study on Behalf of Eurocord and SAAWP-EBMT

Author

Rafii, Hanadi, Volt, Fernanda, Bierings, Marc, Dalle, Jean-Hugues, Ayas, Mouhab, Rihani, Rawad, Faraci, Maura, de Simone, Giuseppina, Sengeloev, Henrik, Passweg, Jakob, Cavazzana, Marina, Costello, Regis, Maertens, Johan, Biffi, Alessandra, Johansson, Jan-Erik, Montoro, Juan, Guepin, Gabrielle Roth, Diaz, Miguel Angel, Sirvent, Anne, Kenzey, Chantal, Rivera Franco, Monica M., Cappelli, Barbara, Scigliuolo, Graziana Maria, Rocha, Vanderson, Ruggeri, Annalisa, Risitano, Antonio, De Latour, Regis Peffault, and Gluckman, Eliane

Published
2024
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6. Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation.

Author

Gluckman, Eliane, Cappelli, Barbara, Bernaudin, Francoise, Labopin, Myriam, Volt, Fernanda, Carreras, Jeanette, Pinto Simões, Belinda, Ferster, Alina, Dupont, Sophie, de la Fuente, Josu, Dalle, Jean-Hugues, Zecca, Marco, Walters, Mark C, Krishnamurti, Lakshmanan, Bhatia, Monica, Leung, Kathryn, Yanik, Gregory, Kurtzberg, Joanne, Dhedin, Nathalie, Kuentz, Mathieu, Michel, Gerard, Apperley, Jane, Lutz, Patrick, Neven, Bénédicte, Bertrand, Yves, Vannier, Jean Pierre, Ayas, Mouhab, Cavazzana, Marina, Matthes-Martin, Susanne, Rocha, Vanderson, Elayoubi, Hanadi, Kenzey, Chantal, Bader, Peter, Locatelli, Franco, Ruggeri, Annalisa, and Eapen, Mary

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Immunology, Hematology, Prevention, Sickle Cell Disease, Stem Cell Research, Rare Diseases, Clinical Research, Stem Cell Research - Nonembryonic - Human, Regenerative Medicine, Transplantation, Adolescent, Anemia, Sickle Cell, Child, Child, Preschool, Disease-Free Survival, Female, Graft Survival, HLA Antigens, Hematopoietic Stem Cell Transplantation, Histocompatibility, Humans, Infant, Male, Siblings, Surveys and Questionnaires, Survival Rate, Transplantation Conditioning, Treatment Outcome, Eurocord, the Pediatric Working Party of the European Society for Blood and Marrow Transplantation, and the Center for International Blood and Marrow Transplant Research, Cardiorespiratory Medicine and Haematology, Clinical Sciences, Paediatrics and Reproductive Medicine, Biochemistry and cell biology, Cardiovascular medicine and haematology, Paediatrics
Abstract

Despite advances in supportive therapy to prevent complications of sickle cell disease (SCD), access to care is not universal. Hematopoietic cell transplantation is, to date, the only curative therapy for SCD, but its application is limited by availability of a suitable HLA-matched donor and lack of awareness of the benefits of transplant. Included in this study are 1000 recipients of HLA-identical sibling transplants performed between 1986 and 2013 and reported to the European Society for Blood and Marrow Transplantation, Eurocord, and the Center for International Blood and Marrow Transplant Research. The primary endpoint was event-free survival, defined as being alive without graft failure; risk factors were studied using a Cox regression models. The median age at transplantation was 9 years, and the median follow-up was longer than 5 years. Most patients received a myeloablative conditioning regimen (n = 873; 87%); the remainder received reduced-intensity conditioning regimens (n = 125; 13%). Bone marrow was the predominant stem cell source (n = 839; 84%); peripheral blood and cord blood progenitors were used in 73 (7%) and 88 (9%) patients, respectively. The 5-year event-free survival and overall survival were 91.4% (95% confidence interval, 89.6%-93.3%) and 92.9% (95% confidence interval, 91.1%-94.6%), respectively. Event-free survival was lower with increasing age at transplantation (hazard ratio [HR], 1.09; P < .001) and higher for transplantations performed after 2006 (HR, 0.95; P = .013). Twenty-three patients experienced graft failure, and 70 patients (7%) died, with the most common cause of death being infection. The excellent outcome of a cohort transplanted over the course of 3 decades confirms the role of HLA-identical sibling transplantation for children and adults with SCD.

Published
2017

7. Low rate of nonrelapse mortality in under 4-year-olds with ALL given chemo-conditioning for HSCT: Phase III FORUM study

Author

Bader, P, Poetschger, U, Dalle, J, Moser, L, Balduzzi, A, Ansari, M, Buechner, J, Güngör, T, Ifversen, M, Kriván, G, Pichler, H, Renard, M, Staciuk, R, Sedlacek, P, Stein, J, Heusel, J, Truong, T, Wachowiak, J, Yeşilipek, M, Locatelli, F, Peters, C, Bader, Peter, Poetschger, Ulrike, Dalle, Jean-Hugues, Moser, Laura M, Balduzzi, Adriana Cristina, Ansari, Marc, Buechner, Jochen, Güngör, Tayfun, Ifversen, Marianne, Kriván, Gergely, Pichler, Herbert, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr, Stein, Jerry, Heusel, Jan Robert, Truong, Tony, Wachowiak, Jacek, Yeşilipek, M Akif, Locatelli, Franco, Peters, Christina, Bader, P, Poetschger, U, Dalle, J, Moser, L, Balduzzi, A, Ansari, M, Buechner, J, Güngör, T, Ifversen, M, Kriván, G, Pichler, H, Renard, M, Staciuk, R, Sedlacek, P, Stein, J, Heusel, J, Truong, T, Wachowiak, J, Yeşilipek, M, Locatelli, F, Peters, C, Bader, Peter, Poetschger, Ulrike, Dalle, Jean-Hugues, Moser, Laura M, Balduzzi, Adriana Cristina, Ansari, Marc, Buechner, Jochen, Güngör, Tayfun, Ifversen, Marianne, Kriván, Gergely, Pichler, Herbert, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr, Stein, Jerry, Heusel, Jan Robert, Truong, Tony, Wachowiak, Jacek, Yeşilipek, M Akif, Locatelli, Franco, and Peters, Christina

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) is highly effective for treating pediatric high-risk or relapsed acute lymphoblastic leukemia (ALL). For young children, total body irradiation (TBI) is associated with severe late sequelae. In the FORUMstudy (NCT01949129), we assessed safety, event-free survival (EFS), and overall survival (OS) of 2 TBIfree conditioning regimens in children aged <4 years with ALL. Patients received fludarabine (Flu), thiotepa (Thio), and either busulfan (Bu) or treosulfan (Treo) before HSCT. From 2013 to 2021, 191 children received transplantation and were observed for ≥6 months (median followup: 3 years). The 3-year OS was 0.63 (95% confidence interval [95% CI], 0.52-0.72) and 0.76 (95% CI, 0.64-0.84) for Flu/Thio/Bu and Flu/Thio/Treo (P = .075), respectively. Three-year EFSwas 0.52 (95% CI, 0.41-0.61) and 0.51 (95% CI, 0.39-0.62), respectively (P = .794). Cumulative incidence of nonrelapse mortality (NRM)and relapse at 3 years were 0.06 (95% CI, 0.02-0.12) vs 0.03 (95%CI: [removed]1 acute graft-versus-host disease (GVHD) occurred in 29% of patients receiving Flu/Thio/ Bu and 17% of those receiving Flu/Thio/Treo (P = .049), whereas grade 3/4 occurred in 10% and 9%, respectively (P = .813). The 3-year incidence of chronic GVHD was 0.07 (95% CI, 0.03-0.13) vs 0.05 (95% CI, 0.02-0.11), respectively (P = .518). In conclusion, both chemotherapeutic conditioning regimens were well tolerated and NRM was low. However, relapse was the major cause of treatment failure.

Published
2024

8. Low rate of nonrelapse mortality in under-4-year-olds with ALL given chemotherapeutic conditioning for HSCT: a phase 3 FORUM study

Author

Bader, Peter; https://orcid.org/0000-0003-4554-0265, Pötschger, Ulrike, Dalle, Jean-Hugues; https://orcid.org/0000-0002-8406-0793, Moser, Laura M, Balduzzi, Adriana; https://orcid.org/0000-0002-5879-0610, Ansari, Marc; https://orcid.org/0000-0002-9649-6498, Buechner, Jochen; https://orcid.org/0000-0001-5848-4501, Güngör, Tayfun; https://orcid.org/0000-0002-3261-1186, Ifversen, Marianne, Krivan, Gergely, Pichler, Herbert; https://orcid.org/0000-0001-5107-5123, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr; https://orcid.org/0000-0001-7895-2886, Stein, Jerry, Heusel, Jan Robert; https://orcid.org/0000-0002-5753-0817, Truong, Tony, Wachowiak, Jacek, Yesilipek, Akif, Locatelli, Franco; https://orcid.org/0000-0002-7976-3654, Peters, Christina; https://orcid.org/0000-0003-0369-8515, Bader, Peter; https://orcid.org/0000-0003-4554-0265, Pötschger, Ulrike, Dalle, Jean-Hugues; https://orcid.org/0000-0002-8406-0793, Moser, Laura M, Balduzzi, Adriana; https://orcid.org/0000-0002-5879-0610, Ansari, Marc; https://orcid.org/0000-0002-9649-6498, Buechner, Jochen; https://orcid.org/0000-0001-5848-4501, Güngör, Tayfun; https://orcid.org/0000-0002-3261-1186, Ifversen, Marianne, Krivan, Gergely, Pichler, Herbert; https://orcid.org/0000-0001-5107-5123, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr; https://orcid.org/0000-0001-7895-2886, Stein, Jerry, Heusel, Jan Robert; https://orcid.org/0000-0002-5753-0817, Truong, Tony, Wachowiak, Jacek, Yesilipek, Akif, Locatelli, Franco; https://orcid.org/0000-0002-7976-3654, and Peters, Christina; https://orcid.org/0000-0003-0369-8515

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) is highly effective for treating pediatric high-risk or relapsed acute lymphoblastic leukemia (ALL). For young children, total body irradiation (TBI) is associated with severe late sequelae. In the FORUM study (NCT01949129), we assessed safety, event-free survival (EFS), and overall survival (OS) of 2 TBI-free conditioning regimens in children aged <4 years with ALL. Patients received fludarabine (Flu), thiotepa (Thio), and either busulfan (Bu) or treosulfan (Treo) before HSCT. From 2013 to 2021, 191 children received transplantation and were observed for ≥6 months (median follow-up: 3 years). The 3-year OS was 0.63 (95% confidence interval [95% CI], 0.52-0.72) and 0.76 (95% CI, 0.64-0.84) for Flu/Thio/Bu and Flu/Thio/Treo (P = .075), respectively. Three-year EFS was 0.52 (95% CI, 0.41-0.61) and 0.51 (95% CI, 0.39-0.62), respectively (P = .794). Cumulative incidence of nonrelapse mortality (NRM) and relapse at 3 years were 0.06 (95% CI, 0.02-0.12) vs 0.03 (95% CI: <0.01-0.09) (P = .406) and 0.42 (95% CI, 0.31-0.52) vs 0.45 (95% CI, 0.34-0.56) (P = .920), respectively. Grade >1 acute graft-versus-host disease (GVHD) occurred in 29% of patients receiving Flu/Thio/Bu and 17% of those receiving Flu/Thio/Treo (P = .049), whereas grade 3/4 occurred in 10% and 9%, respectively (P = .813). The 3-year incidence of chronic GVHD was 0.07 (95% CI, 0.03-0.13) vs 0.05 (95% CI, 0.02-0.11), respectively (P = .518). In conclusion, both chemotherapeutic conditioning regimens were well tolerated and NRM was low. However, relapse was the major cause of treatment failure. This trial was registered at www.clinicaltrials.gov as #NCT01949129.

Published
2024

11. Ethical Issues in HSCT

Author

El-Ghariani, Khaled, Dalle, Jean-Hugues, Carreras, Enric, editor, Dufour, Carlo, editor, Mohty, Mohamad, editor, and Kröger, Nicolaus, editor

Published
2019
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13. Genetic Susceptibility to Hepatic Sinusoidal Obstruction Syndrome in Pediatric Patients Undergoing Hematopoietic Stem Cell Transplantation

Author

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

Published
2020
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14. A predictive classifier of poor prognosis in transplanted patients with juvenile myelomonocytic leukemia: a study on behalf of the Société Francophone de Greffe de Moelle et de Thérapie Cellulaire

Author

Meyran, Déborah, primary, Arfeuille, Chloé, additional, Chevret, Sylvie, additional, Neven, Quentin, additional, Caye-Eude, Aurélie, additional, Lainey, Elodie, additional, Petit, Arnaud, additional, Rialland, Fanny, additional, Michel, Gérard, additional, Plantaz, Dominique, additional, Jubert, Charlotte, additional, Theron, Alexandre, additional, Gandemer, Virginie, additional, Ouachée-Chardin, Marie, additional, Paillard, Catherine, additional, Bruno, Bénédicte, additional, Buchbinder, Nimrod, additional, Pochon, Cécile, additional, Calvo, Charlotte, additional, Fahd, Mony, additional, Baruchel, André, additional, Cavé, Hélène, additional, Dalle, Jean-Hugues, additional, and Strullu, Marion, additional

Published
2024
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15. 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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16. Treatment Duration and Complete Response (CR) in Adult and Pediatric Patients (Pts) Treated with Defibrotide (DF) for Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) after Hematopoietic Cell Transplantation (HCT) in the Defifrance and EBMT PASS Registries

Author

Mohty, Mohamad, primary, Locatelli, Franco, additional, Blaise, Didier, additional, Latour, Régis Peffault de, additional, Labussière-Wallet, Hélène, additional, Amber, Vian, additional, Gandemer, Virginie, additional, Petitprez, Amelie, additional, Dronamraju, Nalina, additional, Dalle, Jean-Hugues, additional, and Srour, Micha, additional

Published
2024
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17. Ovarian Function and Spontaneous Pregnancy After Hematopoietic Stem Cell Transplantation for Leukemia Before Puberty: An L.E.A. Cohort Study

Author

Chabut, Mathilde, Schneider, Pascale, Courbiere, Blandine, Saultier, Paul, Bertrand, Yves, Tabone, Marie-Dominique, Pochon, Cécile, Ducassou, Stéphane, Paillard, Catherine, Gandemer, Virginie, Kanold, Justyna, Dalle, Jean-Hugues, Poiree, Maryline, Plat, Geneviève, Thouvenin, Sandrine, Plantaz, Dominique, Sirvent, Nicolas, Weinhard, Sara, Berbis, Julie, Baruchel, André, Leverger, Guy, Hamidou, Zeinab, Auquier, Pascal, and Michel, Gérard

Published
2023
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19. 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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20. Transplantation in Children and Adolescents with Acute Lymphoblastic Leukemia from a Matched Donor versus an HLA-Identical Sibling: Is the Outcome Comparable? Results from the International BFM ALL SCT 2007 Study

Author

Balduzzi, Adriana, Dalle, Jean-Hugues, Wachowiak, Jacek, Yaniv, Isaac, Yesilipek, Akif, Sedlacek, Petr, Bierings, Marc, Ifversen, Marianne, Sufliarska, Sabina, Kalwak, Krzysztof, Lankester, Arjan, Toporski, Jacek, Di Maio, Lucia, Glogova, Evgenia, Poetschger, Ulrike, and Peters, Christina

Published
2019
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21. 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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22. Unrelated Cord Blood Transplantation in Children, Adolescents, and Young Adults with Acute Leukemia or Myelodysplastic Syndrome: A Retrospective Comparative Study from the French Society for Bone Marrow Transplantation and Cellular Therapy Between Real-World Data and Previously Reported Results of a Randomized Clinical Trial

Author

Teyssier, Anne-Charlotte, Michel, Gérard, Jubert, Charlotte, Rialland, Fanny, Visentin, Sandrine, Ouachée, Marie, Bilger, Karin, Gandemer, Virginie, Beguin, Yves, Marie-Cardine, Aude, Chalandon, Yves, Ansari, Marc, Baumstarck, Karine, Loundou, Anderson, Dalle, Jean-Hugues, and Sirvent, Anne

Published
2022
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23. Allogeneic Stem Cell Transplantation from HLA-Mismatched Donors for Pediatric Patients with Acute Lymphoblastic Leukemia Treated According to the 2003 BFM and 2007 International BFM Studies: Impact of Disease Risk on Outcomes

Author

Dalle, Jean-Hugues, Balduzzi, Adriana, Bader, Peter, Lankester, Arjan, Yaniv, Isaac, Wachowiak, Jacek, Pieczonka, Anna, Bierings, Marc, Yesilipek, Akif, Sedlaçek, Petr, Ifversen, Marianne, Sufliarska, Sabina, Toporski, Jacek, Glogova, Evgenia, Poetschger, Ulrike, and Peters, Christina

Published
2018
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24. Low Body Mass Index Is Associated with Increased Risk of Acute GVHD after Umbilical Cord Blood Transplantation in Children and Young Adults with Acute Leukemia: A Study on Behalf of Eurocord and the EBMT Pediatric Disease Working Party

Author

Paviglianiti, Annalisa, Dalle, Jean Hugues, Ayas, Mouhab, Boelens, Jan Jaap, Volt, Fernanda, Iori, Anna Paola, de Souza, Mair Pedro, Diaz, Miguel Angel, Michel, Gerard, Locatelli, Franco, Jubert, Charlotte, Yakoub-Agha, Ibrahim, Bittencourt, Henrique, Bertrand, Yves, Kenzey, Chantal, Tozatto Maio, Karina, Hayashi, Hiromi, Rocha, Vanderson, Bader, Peter, Gluckman, Eliane, and Ruggeri, Annalisa

Published
2018
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25. A landscape of germ line mutations in a cohort of inherited bone marrow failure patients

Author

Bluteau, Olivier, Sebert, Marie, Leblanc, Thierry, Peffault de Latour, Régis, Quentin, Samuel, Lainey, Elodie, Hernandez, Lucie, Dalle, Jean-Hugues, Sicre de Fontbrune, Flore, Lengline, Etienne, Itzykson, Raphael, Clappier, Emmanuelle, Boissel, Nicolas, Vasquez, Nadia, Da Costa, Mélanie, Masliah-Planchon, Julien, Cuccuini, Wendy, Raimbault, Anna, De Jaegere, Louis, Adès, Lionel, Fenaux, Pierre, Maury, Sébastien, Schmitt, Claudine, Muller, Marc, Domenech, Carine, Blin, Nicolas, Bruno, Bénédicte, Pellier, Isabelle, Hunault, Mathilde, Blanche, Stéphane, Petit, Arnaud, Leverger, Guy, Michel, Gérard, Bertrand, Yves, Baruchel, André, Socié, Gérard, and Soulier, Jean

Published
2018
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26. Use of letermovir in umbilical cord blood transplantation based on risk scores

Author

Rivera Franco, Monica M., primary, Rafii, Hanadi, additional, Volt, Fernanda, additional, Kenzey, Chantal, additional, Cappelli, Barbara, additional, Scigliuolo, Graziana Maria, additional, Rocha, Vanderson, additional, Raus, Nicole, additional, Dalle, Jean-Hugues, additional, Chevallier, Patrice, additional, Robin, Marie, additional, Rubio, Marie Thérèse, additional, Ruggeri, Annalisa, additional, and Gluckman, Eliane, additional

Published
2023
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27. Long-Term Outcomes of Cord Blood Transplantation from an HLA-Identical Sibling for Patients with Bone Marrow Failure Syndromes: A Report From Eurocord, Cord Blood Committee and Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation

Author

Pagliuca, Simona, Peffault de Latour, Régis, Volt, Fernanda, Locatelli, Franco, Zecca, Marco, Dalle, Jean-Hugues, Comoli, Patrizia, Vettenranta, Kim, Diaz, Miguel Angel, Reuven, Or, Bertrand, Yves, Diaz de Heredia, Cristina, Nagler, Arnon, Ghavamzadeh, Ardeshir, Sufliarska, Sabina, Lawson, Sarah, Kenzey, Chantal, Rocha, Vanderson, Dufour, Carlo, Gluckman, Eliane, Passweg, Jakob, and Ruggeri, Annalisa

Published
2017
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28. Late Effects Screening Guidelines after Hematopoietic Cell Transplantation for Inherited Bone Marrow Failure Syndromes: Consensus Statement From the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects After Pediatric HCT

Author

Dietz, Andrew C., Savage, Sharon A., Vlachos, Adrianna, Mehta, Parinda A., Bresters, Dorine, Tolar, Jakub, Bonfim, Carmem, Dalle, Jean Hugues, de la Fuente, Josu, Skinner, Roderick, Boulad, Farid, Duncan, Christine N., Baker, K. Scott, Pulsipher, Michael A., Lipton, Jeffrey M., Wagner, John E., and Alter, Blanche P.

Published
2017
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29. Current Knowledge and Priorities for Future Research in Late Effects after Hematopoietic Cell Transplantation for Inherited Bone Marrow Failure Syndromes: Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric Hematopoietic Cell Transplantation

Author

Dietz, Andrew C., Mehta, Parinda A., Vlachos, Adrianna, Savage, Sharon A., Bresters, Dorine, Tolar, Jakub, Boulad, Farid, Dalle, Jean Hugues, Bonfim, Carmem, de la Fuente, Josu, Duncan, Christine N., Baker, K. Scott, Pulsipher, Michael A., Lipton, Jeffrey M., Wagner, John E., and Alter, Blanche P.

Published
2017
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31. Educational outcomes in siblings of childhood leukemia survivors: Factors associated with school difficulties and comparison with general population.

Author

Faust, Cindy, Auquier, Pascal, Gandemer, Virginie, Bertrand, Yves, Tabone, Marie‐Dominique, Ansoborlo, Sophie, Baruchel, André, Bonneau, Jacinthe, Dalle, Jean‐Hugues, Chastagner, Pascal, Kanold, Justyna, Poirée, Maryline, Theron, Alexandre, Olivier, Laura, Pellier, Isabelle, Michel, Gérard, and Berbis, Julie

Subjects
EDUCATIONAL outcomes, ADULT child abuse victims, SIBLINGS, HEMATOPOIETIC stem cells, GRADE repetition, STEM cell donors, BENCH press
Abstract

Background: To investigate the educational outcomes of siblings of childhood leukemia survivors, explore determinants of school difficulties, and compare the rates of repeating grades between siblings and the general population. Methods: A cross‐sectional study of childhood leukemia survivors' siblings recruited through the Leucémies de l'Enfant et de l'Adolescent cohort, a French long‐term follow‐up program, was conducted, and education‐related data were obtained via self‐report questionnaires. Adjusted logistic regression models were used to identify variables associated with school difficulties and time since diagnosis. Rates of repeating a grade in middle school were compared between siblings and the general population of the same generation. Results: A total of 564 siblings with a mean time from diagnosis of 14.1 ± 6.4 years were included, among whom 139 (24.6%) repeated a grade, at an average of 6.4 ± 4.5 years after diagnosis. In multivariate analysis, the risk factors for repeating a grade were older siblings (odds ratio [OR] 2.3, p = 0.006), family financial difficulties (OR 2.8, p = 0.008), and history of repetition in survivors (OR, 2.5, p = 0.001). Sibling hematopoietic stem cell donors were at greater risk of repeating a grade long‐term after diagnosis (p = 0.018). Overall, siblings did not have a higher risk of educational delays at the end of middle school than the general population. Conclusion: Although the results are reassuring, socioeconomic and cancer‐related factors may have an impact on siblings' schooling long after diagnosis. Paying attention to siblings contributes to identifying the most vulnerable families, allowing more attention and appropriate resources to avoid long‐term repercussions. Additionally, supportive and targeted interventions can be developed to improve the organization of education and the health care system. [ABSTRACT FROM AUTHOR]

Published
2024
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32. The uterine volume is dramatically decreased after hematopoietic stem cell transplantation during childhood regardless of the conditioning regimen

Author

Courbiere, Blandine, primary, Drikes, Benjamin, additional, Grob, Anaïs, additional, Hamidou, Zeinab, additional, Saultier, Paul, additional, Bertrand, Yves, additional, Gandemer, Virginie, additional, Plantaz, Dominique, additional, Plat, Geneviève, additional, Poirée, Maryline, additional, Ducassou, Stéphane, additional, Pochon, Cécile, additional, Dalle, Jean-Hugues, additional, Thouvenin, Sandrine, additional, Paillard, Catherine, additional, Kanold, Justyna, additional, Sirvent, Anne, additional, Rousset-Jablonski, Christine, additional, Duros, Solène, additional, Gueniffey, Aurore, additional, Cohade, Clementine, additional, Boukaidi, Samir, additional, Frantz, Sandrine, additional, Agopiantz, Mikael, additional, Poirot, Catherine, additional, Genod, Anne, additional, Pirrello, Olivier, additional, Gremeau, Anne-Sophie, additional, Bringer-Deutsch, Sophie, additional, Auquier, Pascal, additional, and Michel, Gérard, additional

Published
2023
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33. The Impact of Donor Type on Long-Term Health Status and Quality of Life after Allogeneic Hematopoietic Stem Cell Transplantation for Childhood Acute Leukemia: A Leucémie de l'Enfant et de L'Adolescent Study

Author

Visentin, Sandrine, Auquier, Pascal, Bertrand, Yves, Baruchel, André, Tabone, Marie-Dominique, Pochon, Cécile, Jubert, Charlotte, Poirée, Maryline, Gandemer, Virginie, Sirvent, Anne, Bonneau, Jacinthe, Paillard, Catherine, Freycon, Claire, Kanold, Justyna, Villes, Virginie, Berbis, Julie, Oudin, Claire, Galambrun, Claire, Pellier, Isabelle, Plat, Geneviève, Chambost, Hervé, Leverger, Guy, Dalle, Jean-Hugues, and Michel, Gérard

Published
2016
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34. Single- vs double-unit cord blood transplantation for children and young adults with acute leukemia or myelodysplastic syndrome

Author

Michel, Gérard, Galambrun, Claire, Sirvent, Anne, Pochon, Cecile, Bruno, Benedicte, Jubert, Charlotte, Loundou, Anderson, Yakoub-Agha, Ibrahim, Milpied, Noel, Lutz, Patrick, Marie-Cardine, Aude, Gandemer, Virginie, Blaise, Didier, Michallet, Mauricette, Rialland, Fanny, Renard, Cecile, Oudin, Claire, Esmiol, Sophie, Seux, Mylene, Baumstarck, Karine, Mohty, Mohamad, Rocha, Vanderson, and Dalle, Jean-Hugues

Published
2016
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37. Atidarsagene Autotemcel, a European Post-Regulatory Approval Model for Delivery of Autologous Hematopoietic Stem Cell Gene Therapy Products Via a Network of Qualified Treatment Centers (QTCs)

Author

Wynn, Robert F., primary, Jones, Simon, additional, Fumagalli, Francesca, additional, Calbi, Valeria, additional, Aiuti, Alessandro, additional, Gröschel, Samuel, additional, Lang, Peter, additional, van Hasselt, Peter, additional, Lindemans, Caroline A., additional, Sevin, Caroline, additional, Dalle, Jean-Hugues, additional, and Snell, Katie, additional

Published
2023
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38. Outcomes of Defibrotide-Treated Patients (Pts) with Anicteric/Icteric Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) after Hematopoietic Cell Transplantation (HCT): Results from the Defifrance Registry Study

Author

Gandemer, Virginie, primary, Guenounou, Sarah, additional, Izadifar-Legrand, Faezeh, additional, Blaise, Didier, additional, de Latour, Régis Peffault, additional, Duchêne, François, additional, Dronamraju, Nalina, additional, Yakoub-Agha, Ibrahim, additional, Labopin, Myriam, additional, Dalle, Jean-Hugues, additional, and Mohty, Mohamad, additional

Published
2023
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39. Treatment Duration, Symptom Resolution, and Survival in Defibrotide-Treated Patients (Pts) with Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) after Hematopoietic Cell Transplantation (HCT): Pooled Analysis of Defifrance and EBMT PASS Registries

Author

Mohty, Mohamad, primary, Blaise, Didier, additional, de Latour, Régis Peffault, additional, Labopin, Myriam, additional, Labussière-Wallet, Hélène, additional, Amber, Vian, additional, Gandemer, Virginie, additional, Gutierrez, Deborah, additional, Dronamraju, Nalina, additional, Dalle, Jean-Hugues, additional, and Yakoub-Agha, Ibrahim, additional

Published
2023
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40. Clonal hematopoiesis driven by chromosome 1q/MDM4 trisomy defines a canonical route toward leukemia in Fanconi anemia

Author

Sebert, Marie, primary, Gachet, Stéphanie, additional, Leblanc, Thierry, additional, Rousseau, Alix, additional, Bluteau, Olivier, additional, Kim, Rathana, additional, Ben Abdelali, Raouf, additional, Sicre de Fontbrune, Flore, additional, Maillard, Loïc, additional, Fedronie, Carèle, additional, Murigneux, Valentine, additional, Bellenger, Léa, additional, Naouar, Naira, additional, Quentin, Samuel, additional, Hernandez, Lucie, additional, Vasquez, Nadia, additional, Da Costa, Mélanie, additional, Prata, Pedro H., additional, Larcher, Lise, additional, de Tersant, Marie, additional, Duchmann, Matthieu, additional, Raimbault, Anna, additional, Trimoreau, Franck, additional, Fenneteau, Odile, additional, Cuccuini, Wendy, additional, Gachard, Nathalie, additional, Auger, Nathalie, additional, Tueur, Giulia, additional, Blanluet, Maud, additional, Gazin, Claude, additional, Souyri, Michèle, additional, Langa Vives, Francina, additional, Mendez-Bermudez, Aaron, additional, Lapillonne, Hélène, additional, Lengline, Etienne, additional, Raffoux, Emmanuel, additional, Fenaux, Pierre, additional, Adès, Lionel, additional, Forcade, Edouard, additional, Jubert, Charlotte, additional, Domenech, Carine, additional, Strullu, Marion, additional, Bruno, Bénédicte, additional, Buchbinder, Nimrod, additional, Thomas, Caroline, additional, Petit, Arnaud, additional, Leverger, Guy, additional, Michel, Gérard, additional, Cavazzana, Marina, additional, Gluckman, Eliane, additional, Bertrand, Yves, additional, Boissel, Nicolas, additional, Baruchel, André, additional, Dalle, Jean-Hugues, additional, Clappier, Emmanuelle, additional, Gilson, Eric, additional, Deriano, Ludovic, additional, Chevret, Sylvie, additional, Sigaux, François, additional, Socié, Gérard, additional, Stoppa-Lyonnet, Dominique, additional, de Thé, Hugues, additional, Antoniewski, Christophe, additional, Bluteau, Dominique, additional, Peffault de Latour, Régis, additional, and Soulier, Jean, additional

Published
2023
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41. The Impact of Pre-Transplant Extramedullary Disease on the Outcome of Allogeneic Hematopoietic Cell Transplantation for Acute Myeloid Leukemia in Children- on Behalf of PDWP/EBMT

Author

Silva, Juliana, primary, Galimard, Jacques-Emmanuel, additional, Dalle, Jean-Hugues, additional, Locatelli, Franco, additional, Alsaedi, Hawazen S., additional, Diaz, Miguel Angel, additional, Jubert, Charlotte, additional, Bertrand, Yves, additional, Simone, Giuseppina, additional, Bader, Peter, additional, Fagioli, Franca, additional, Kitra Roussou, Vassiliki, additional, Furness, Caroline, additional, Wynn, Robert, additional, Burkhardt, Birgit, additional, Biffi, Alessandra, additional, Bierings, Marc, additional, Díaz de Heredia, Cristina, additional, Prete, Arcangelo, additional, Toren, Amos, additional, Patrick, Katharine, additional, Holter, Wolfgang, additional, Dalissier, Arnaud, additional, Rao, Kanchan, additional, and Corbacioglu, Selim, additional

Published
2022
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42. Impact of Hydroxyurea and Vaso-Occlusive Crisis on Ovarian Follicle Density in Girls and Young Females with Sickle Cell Disease

Author

Diesch, Tamara, primary, Poirot, Catherine, additional, Sanchez, Carlos, additional, Atkinson, Andrew, additional, Pondarré, Corinne, additional, Dhedin, Nathalie, additional, Neven, Benedicte, additional, Pirenne, France, additional, Leanour, Gilles, additional, Broncheriou, Isabelle, additional, Bernaudin, Françoise, additional, and Dalle, Jean-Hugues, additional

Published
2022
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43. Cardiac and Vascular Risks Are the Essential Parameters for Long-Term Surveillance of Lower Risk Childhood Acute Leukemia Survivors

Author

Saultier, Paul, primary, El Riachy, Nour, additional, Bertrand, Yves, additional, Tabone, Marie-Dominique, additional, Leverger, Guy, additional, Dalle, Jean-Hugues, additional, Baruchel, Andre, additional, Pochon, Cecile, additional, Paillard, Catherine, additional, Ducassou, Stephane, additional, Poiree, Marilyne, additional, Plantaz, Dominique, additional, Kanold, Justyna, additional, Gandemer, Virginie, additional, Sirvent, Nicolas, additional, Plat, Genevieve, additional, Pellier, Isabelle, additional, Reguerre, Yves, additional, Thouvenin, Sandrine, additional, Petit, Audrey Françoise, additional, Auquier, Pascal, additional, Berbis, Julie, additional, and Michel, Gerard, additional

Published
2022
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44. Superior Graft-Versus-Leukemia Effect in Matched Unrelated Donor Versus HLA-Identical Sibling Pediatric Recipients Transplanted for Acute Lymphoblastic Leukemia within the Forum Study

Author

Dalle, Jean-Hugues, primary, Bader, Peter, additional, Yesilipek, Akif, additional, Locatelli, Franco, additional, Palma, Julia, additional, Wachowiak, Jacek, additional, Pichler, Herbert, additional, Ifversen, Marianne, additional, Kriván, Gergely, additional, Buechner, Jochen, additional, Díaz-de-Heredia, Cristina, additional, Bierings, Marc, additional, Staciuk, Raquel, additional, Gungor, Tayfun, additional, Toporski, Jacek, additional, Balduzzi, Adriana, additional, Poetschger, Ulrike, additional, Peters, Christina, additional, and Sedlacek, Petr, additional

Published
2022
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45. Treatment Duration, Symptom Resolution, and Survival in Defibrotide-Treated Patients with Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome after Hematopoietic Cell Transplantation: Pooled Analysis of Defifrance and EBMT PASS Registries

Author

Mohty, Mohamad, primary, Locatelli, Franco, additional, Blaise, Didier, additional, Peffault de Latour, Régis, additional, Labopin, Myriam, additional, Labussière-Wallet, Hélène, additional, Amber, Vian, additional, Gandemer, Virginie, additional, Gutierrez, Deborah, additional, Dronamraju, Nalina, additional, Dalle, Jean-Hugues, additional, and Yakoub-Agha, Ibrahim, additional

Published
2022
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46. Outcomes of Defibrotide-Treated Patients with Anicteric/Icteric Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) after Hematopoietic Cell Transplantation (HCT): Results from the Defifrance Registry Study

Author

Gandemer, Virginie, primary, Guenounou, Sarah, additional, Legrand, Faezeh, additional, Blaise, Didier, additional, Peffault de Latour, Régis, additional, Duchêne, François, additional, Dronamraju, Nalina, additional, Yakoub-Agha, Ibrahim, additional, Labopin, Myriam, additional, Dalle, Jean-Hugues, additional, and Mohty, Mohamad, additional

Published
2022
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47. Specific Cytogenetic Abnormalities at Diagnosis Predict Survival after Hematopoietic Cell Transplant in Poor-Risk Pediatric Acute Myeloid Leukemia: A PDWP/EBMT Study

Author

Sharma, Akshay, primary, Galimard, Jacques-Emmanuel, additional, Pryce, Angharad, additional, Bhoopalan, Senthil, additional, Dalissier, Arnaud, additional, Dalle, Jean-Hugues, additional, Locatelli, Franco, additional, Jubert, Charlotte, additional, Mirci-Danicar, Oana, additional, Kitra-Roussou, Vassiliki, additional, Bertrand, Yves, additional, Fagioli, Franca, additional, Rialland, Fanny, additional, Biffi, Alessandra, additional, Wynn, Robert F, additional, Michel, Gerard, additional, Tambaro, Francesco Paolo P, additional, Ahmari, Ali Abdallah, additional, Tbakhi, Abdelghani, additional, Furness, Caroline, additional, Díaz Pérez, Miguel Ángel, additional, Sedlacek, Petr, additional, Bodova, Ivana, additional, Faraci, Maura, additional, Rao, Kanchan, additional, Rubio, Marie Thérèse, additional, Gibson, Brenda, additional, Bhatt, Neel S., additional, and Corbacioglu, Selim, additional

Published
2022
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48. Outcome of Haematopoietic Cell Transplantation in 813 Children with Fanconi Anaemia: A Study on Behalf of the EBMT Severe Aplastic Anaemia Working Party and Paediatric Disease Working Party

Author

Lum, Su Han, primary, Samarasinghe, Sujith, additional, Eikema, Dirk-Jan, additional, Piepenbroek, Brian, additional, Dalissier, Arnaud, additional, Ayas, Mouhab, additional, Mousavi, Ashrafsadat, additional, Hamladji, Rose-Marie, additional, Yesilipek, Akif, additional, Dalle, Jean-Hugues, additional, Kansoy, Savas, additional, Locatelli, Franco, additional, Çetinkaya, Duygu, additional, Bierings, Marc, additional, Kupesiz, O. Alphan, additional, Tbakhi, Abdelghani, additional, Skorobogatova, Elena, additional, Öztürk, Gülyüz, additional, Faraci, Maura, additional, Bertrand, Yves, additional, Evans, Pamela, additional, Carbacioglu, Selim, additional, Dufour, Carlo, additional, Risitano, Antonio, additional, Wynn, Robert F, additional, and Peffault de Latour, Régis, additional

Published
2022
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49. Impact of Donor and Patient CMV Serology in Children Receiving First Geno-Identical or Unrelated HSCT for Malignant Hematological Disease: An EBMT Retrospective Registry Study

Author

Ince, Elif, primary, Galimard, Jacques-Emmanuel, additional, Ifversen, Marianne, additional, Dalissier, Arnaud, additional, Szmit, Zofia, additional, Locatelli, Franco, additional, Mirci-Danicar, Oana, additional, Sedlacek, Petr, additional, Dalle, Jean-Hugues, additional, Ayas, Mouhab, additional, Hamladji, Rose-Marie, additional, Biondi, Andrea, additional, Styczynski, Jan, additional, Bertrand, Yves, additional, Skorobogatova, Elena, additional, Tbakhi, Abdelghani, additional, Kielsen, Katrine, additional, Kitra-Roussou, Vassiliki, additional, Fagioli, Franca, additional, Holter, Wolfgang, additional, Groll, Andreas H., additional, Aljurf, Mahmoud, additional, Taskinen, Mervi, additional, Bierings, Marc, additional, Meisel, Roland, additional, Cesaro, Simone, additional, Kalwak, Krzysztof, additional, and Corbacioglu, Selim, additional

Published
2022
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50. Relapse Is the Most Common Treatment Failure Post HSCT in Children with ALL below 4 Years of Age Given a Chemo-Based Conditioning Regimen. Results from the Prospective Multinational Forum-Trial

Author

Peters, Christina, primary, Poetschger, Ulrike, additional, Dalle, Jean-Hugues, additional, Yesilipek, Akif, additional, Stein, Jerry, additional, Pichler, Herbert, additional, Balduzzi, Adriana, additional, Locatelli, Franco, additional, Sedlacek, Petr, additional, Wachowiak, Jacek, additional, Güngör, Tayfun, additional, Ifversen, Marianne, additional, Renard, Marleen, additional, Truong, Tony H, additional, Staciuk, Raquel, additional, Shaw, Peter J., additional, Kriván, Gergely, additional, Büchner, Jochen, additional, and Bader, Peter, additional

Published
2022
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