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293 results on '"Bittencourt, H."'

1. Association study of candidate DNA-repair gene variants and acute graft versus host disease in pediatric patients receiving allogeneic hematopoietic stem-cell transplantation

Author

Uppugunduri, C. R. S., Huezo-Diaz Curtis, P., Nava, T., Rezgui, M. A., Mlakar, V., Mlakar, S. Jurkovic, Waespe, N., Théoret, Y., Gumy-Pause, F., Bernard, F., Chalandon, Y., Boelens, J. J., Bredius, R. G. M., Dalle, J. H., Nath, C., Corbacioglu, S., Peters, C., Bader, P., Shaw, P., Bittencourt, H., Krajinovic, M., and Ansari, M.

Published
2022
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2. CTL019 (tisagenlecleucel): CAR-T therapy for relapsed and refractory B-cell acute lymphoblastic leukemia

Author

Vairy S, Lopes Garcia J, Teira P, and Bittencourt H

Subjects
CTL019, Tisagenlecleucel, B-cell acute lymphoblastic leukemia, Therapeutics. Pharmacology, RM1-950
Abstract

Stephanie Vairy,* Julia Lopes Garcia,* Pierre Teira, Henrique Bittencourt Division of Haematology and Oncology, Department of Pediatrics, CHU Sainte-Justine, Université de Montréal, Montréal, QC, Canada *These authors contributed equally to this work Abstract: Over the past decades, survival of patients with acute lymphoblastic leukemia (ALL) has dramatically improved, but the subgroup of patients with relapsed/refractory ALL still continues to have dismal prognosis. As an emerging therapeutic approach, chimeric antigen receptor-modified T-cells (CAR-T) represent one of the few practice-changing therapies for this subgroup of patients. Originally conceived and built in Philadelphia (University of Pennsylvania), CTL019 or tisagenlecleucel, the first CAR-T approved by the US Food and Drug Administration, showed impressive results in refractory/relapsed ALL since the publication on two pediatric patients in 2013. It is in this context that we provide a review of this product in terms of manufacturing, pharmacology, toxicity, and efficacy studies. Evaluation and management of toxicities, particularly cytokine release syndrome and neurotoxicity, is recognized as an essential part of the patient treatment with broader use of IL-6 receptor inhibitor. An under-assessed aspect, the quality of life of patients entering CAR-T cells treatment, will also be reviewed. By their unique nature, CAR-T cells such as tisagenlecleucel operate in a different way than typical drugs, but also provide unique hope for B-cell malignancies. Keywords: CTL019, tisagenlecleucel, B-cell acute lymphoblastic leukemia

Published
2018

3. Tisagenlecleucel in pediatric and young adult patients with Down syndrome-associated relapsed/refractory acute lymphoblastic leukemia

Author

Laetsch, T, Maude, S, Balduzzi, A, Rives, S, Bittencourt, H, Boyer, M, Buechner, J, De Moerloose, B, Qayed, M, Phillips, C, Pulsipher, M, Hiramatsu, H, Tiwari, R, Grupp, S, Laetsch T. W., Maude S. L., Balduzzi A., Rives S., Bittencourt H., Boyer M. W., Buechner J., De Moerloose B., Qayed M., Phillips C. L., Pulsipher M. A., Hiramatsu H., Tiwari R., Grupp S. A., Laetsch, T, Maude, S, Balduzzi, A, Rives, S, Bittencourt, H, Boyer, M, Buechner, J, De Moerloose, B, Qayed, M, Phillips, C, Pulsipher, M, Hiramatsu, H, Tiwari, R, Grupp, S, Laetsch T. W., Maude S. L., Balduzzi A., Rives S., Bittencourt H., Boyer M. W., Buechner J., De Moerloose B., Qayed M., Phillips C. L., Pulsipher M. A., Hiramatsu H., Tiwari R., and Grupp S. A.

Abstract

Down syndrome-associated acute lymphoblastic leukemia (DS-ALL) patients suffer risk of chemotherapy-associated toxicities and poor outcomes. We evaluated tisagenlecleucel in 16 patients with DS-ALL in two phase 2 trials (ELIANA [NCT02435849], ENSIGN [NCT02228096]) and a phase 3b, managed access protocol (B2001X [NCT03123939]). Patients were 5–22 years old, had a median of two prior lines of therapy (range, 1–4), and four (25%) had prior stem cell transplants. Fourteen of 16 patients (88%) achieved complete remission (CR) or CR with incomplete blood count recovery (CRi); 12 of 14 (86%) with CR/CRi were minimal residual disease-negative. With a median follow-up of 13.2 months (range, 0.5–49.3 months), six patients (43%) relapsed after CR (three, CD19-negative; three, unknown) between 80–721 days post-infusion. Ongoing remissions in nine patients ranged from 6–48 months. Any-grade and grade 3/4 AEs occurred in 16 and 14 patients, respectively; 44% experienced grade 3/4 cytokine release syndrome and 13% experienced grade 3/4 neurological events. Grade 3/4 prolonged cytopenias occurred in 44% of patients. No grade 3/4 infections were observed. Tisagenlecleucel expansion and long-term persistence were consistent with previous reports. Comparable to ALL patients without DS, tisagenlecleucel produced high remission rates, manageable side-effects, and promising long-term outcomes in pediatric/young adult patients with DS-ALL.

Published
2022

4. Three-Year Update of Tisagenlecleucel in Pediatric and Young Adult Patients With Relapsed/Refractory Acute Lymphoblastic Leukemia in the ELIANA Trial

Author

Laetsch, T, Maude, S, Rives, S, Hiramatsu, H, Bittencourt, H, Bader, P, Baruchel, A, Boyer, M, De Moerloose, B, Qayed, M, Buechner, J, Pulsipher, M, Myers, G, Stefanski, H, Martin, P, Nemecek, E, Peters, C, Yanik, G, Khaw, S, Davis, K, Krueger, J, Balduzzi, A, Boissel, N, Tiwari, R, O'Donovan, D, Grupp, S, Laetsch, Theodore W, Maude, Shannon L, Rives, Susana, Hiramatsu, Hidefumi, Bittencourt, Henrique, Bader, Peter, Baruchel, André, Boyer, Michael, De Moerloose, Barbara, Qayed, Muna, Buechner, Jochen, Pulsipher, Michael A, Myers, Gary Douglas, Stefanski, Heather E, Martin, Paul L, Nemecek, Eneida, Peters, Christina, Yanik, Gregory, Khaw, Seong Lin, Davis, Kara L, Krueger, Joerg, Balduzzi, Adriana, Boissel, Nicolas, Tiwari, Ranjan, O'Donovan, Darragh, Grupp, Stephan A, Laetsch, T, Maude, S, Rives, S, Hiramatsu, H, Bittencourt, H, Bader, P, Baruchel, A, Boyer, M, De Moerloose, B, Qayed, M, Buechner, J, Pulsipher, M, Myers, G, Stefanski, H, Martin, P, Nemecek, E, Peters, C, Yanik, G, Khaw, S, Davis, K, Krueger, J, Balduzzi, A, Boissel, N, Tiwari, R, O'Donovan, D, Grupp, S, Laetsch, Theodore W, Maude, Shannon L, Rives, Susana, Hiramatsu, Hidefumi, Bittencourt, Henrique, Bader, Peter, Baruchel, André, Boyer, Michael, De Moerloose, Barbara, Qayed, Muna, Buechner, Jochen, Pulsipher, Michael A, Myers, Gary Douglas, Stefanski, Heather E, Martin, Paul L, Nemecek, Eneida, Peters, Christina, Yanik, Gregory, Khaw, Seong Lin, Davis, Kara L, Krueger, Joerg, Balduzzi, Adriana, Boissel, Nicolas, Tiwari, Ranjan, O'Donovan, Darragh, and Grupp, Stephan A

Abstract

Clinical trials frequently include multiple end points that mature at different times. The initial report, typically based on the primary end point, may be published when key planned co-primary or secondary analyses are not yet available. Clinical Trial Updates provide an opportunity to disseminate additional results from studies, published in JCO or elsewhere, for which the primary end point has already been reported.In the primary analysis of the global phase II ELIANA trial (ClinicalTrials.gov identifier: NCT02435849), tisagenlecleucel provided an overall remission rate of 81% in pediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (R/R B-ALL), with 59% of responders remaining relapse-free at 12 months. Here, we report an update on efficacy, safety, and patient-reported quality of life in 79 pediatric and young adult patients with R/R B-ALL following a median follow-up of 38.8 months. The overall remission rate was 82%. The median event-free survival was 24 months, and the median overall survival was not reached. Event-free survival was 44% (95% CI, 31 to 57) and overall survival was 63% (95% CI, 51 to 73) at 3 years overall (most events occur within the first 2 years). The estimated 3-year relapse-free survival with and without censoring for subsequent therapy was 52% (95% CI, 37 to 66) and 48% (95% CI, 34 to 60), respectively. No new or unexpected long-term adverse events were reported. Grade 3/4 adverse events were reported in 29% of patients > 1 year after infusion; grade 3/4 infection rate did not increase > 1 year after infusion. Patients reported improvements in quality of life up to 36 months after infusion. These findings demonstrate favorable long-term safety and suggest tisagenlecleucel as a curative treatment option for heavily pretreated pediatric and young adult patients with R/R B-ALL.

Published
2023

6. S112: TISAGENLECLEUCEL IN PEDIATRIC AND YOUNG ADULT PATIENTS (PTS) WITH RELAPSED/REFRACTORY (R/R) B-CELL ACUTE LYMPHOBLASTIC LEUKEMIA (B-ALL): FINAL ANALYSES FROM THE ELIANA STUDY

Author

Rives, S., primary, Maude, S. L., additional, Hiramatsu, H., additional, Baruchel, A., additional, Bader, P., additional, Bittencourt, H., additional, Buechner, J., additional, Laetsch, T., additional, De Moerloose, B., additional, Qayed, M., additional, Stefanski, H. E., additional, Davis, K. L., additional, Martin, P. L., additional, Nemecek, E., additional, Peters, C., additional, Yanik, G., additional, Balduzzi, A., additional, Boissel, N., additional, Khaw, S. L., additional, Krueger, J., additional, Levine, J., additional, Davies, S., additional, Myers, G. D., additional, Yeo, A., additional, O’Donovan, D., additional, Ramos, R., additional, Pulsipher, M., additional, and Grupp, S., additional

Published
2022
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7. Tisagenlecleucel in pediatric and young adult patients with Down syndrome-associated relapsed/refractory acute lymphoblastic leukemia

Author

Laetsch TW, Maude SL, Balduzzi A, Rives-Sola S, Bittencourt H, Boyer MW, Buechner J, De Moerloose B, Qayed M, Phillips CL, Pulsipher MA, Hiramatsu H, Tiwari R, and Grupp SA

Abstract

Down syndrome-associated acute lymphoblastic leukemia (DS-ALL) patients suffer risk of chemotherapy-associated toxicities and poor outcomes. We evaluated tisagenlecleucel in 16 patients with DS-ALL in two phase 2 trials (ELIANA [NCT02435849], ENSIGN [NCT02228096]) and a phase 3b, managed access protocol (B2001X [NCT03123939]). Patients were 5-22 years old, had a median of two prior lines of therapy (range, 1-4), and four (25%) had prior stem cell transplants. Fourteen of 16 patients (88%) achieved complete remission (CR) or CR with incomplete blood count recovery (CRi); 12 of 14 (86%) with CR/CRi were minimal residual disease-negative. With a median follow-up of 13.2 months (range, 0.5-49.3 months), six patients (43%) relapsed after CR (three, CD19-negative; three, unknown) between 80-721 days post-infusion. Ongoing remissions in nine patients ranged from 6-48 months. Any-grade and grade 3/4 AEs occurred in 16 and 14 patients, respectively; 44% experienced grade 3/4 cytokine release syndrome and 13% experienced grade 3/4 neurological events. Grade 3/4 prolonged cytopenias occurred in 44% of patients. No grade 3/4 infections were observed. Tisagenlecleucel expansion and long-term persistence were consistent with previous reports. Comparable to ALL patients without DS, tisagenlecleucel produced high remission rates, manageable side-effects, and promising long-term outcomes in pediatric/young adult patients with DS-ALL. Plain Language Summary Children with Down syndrome have a 20 times higher risk of developing a type of blood cancer called Down syndrome-associated acute lymphoblastic leukemia (ALL). Children who develop Down syndrome-associated ALL typically receive chemotherapy to treat their cancer; however, they can experience severe toxicity or other consequences from these therapies, especially stem cell transplant, and have a poor prognosis if their disease returns after treatment. These children need an effective but less toxic treatment option. Tisagenlecleucel is a chimeric antigen receptor-T cell therapy that specially modifies the patient's own T-cells to recognize and attack the cancer cells. Tisagenlecleucel is approved for use in children and young adults with ALL whose disease reappears after two or more treatments or whose disease doesn't respond to treatment. Here we present data from 16 patients across three clinical studies showing that tisagenlecleucel is well-tolerated and an effective treatment option for children and young adults with Down syndrome-associated ALL, and was similar to what is observed in patients without Down syndrome. Taken together, patients with Down syndrome-associated ALL have unique medical needs, and tisagenlecleucel may help them live longer, avoid stem cell transplantation, and the toxicity from chemotherapy.

Published
2022

8. Correction to: GSTM1 and GSTT1 double null genotypes determining cell fate and proliferation as potential risk factors of relapse in children with hematological malignancies after hematopoietic stem cell transplantation

Author

Mlakar, S.J., Uppugunduri, S.C.R., Nava, T., Mlakar, V., Golay, H., Robin, S., Waespe, N., Rezgui, M.A., Chalandon, Y., Boelens, J.J., Bredius, R.G.M., Dalle, J.H., Peters, C., Corbacioglu, S., Bittencourt, H., Krajinovic, M., Ansari, M., and European Soc Blood Marrow Transpla

Subjects
Cancer Research, Oncology, General Medicine
Published
2021

10. Comparison of outcomes after single or double cord blood transplantation in adults with acute leukemia using different types of myeloablative conditioning regimen, a retrospective study on behalf of Eurocord and the Acute Leukemia Working Party of EBMT

Author

Ruggeri, A, Sanz, G, Bittencourt, H, Sanz, J, Rambaldi, A, Volt, F, Yakoub-Agha, I, Ribera, J M, Mannone, L, Sierra, J, Mohty, M, Solano, C, Nabhan, S, Arcese, W, Gluckman, E, Labopin, M, and Rocha, V

Published
2014
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11. Impact of HLA mismatch direction on outcomes after umbilical cord blood transplantation for hematological malignant disorders: a retrospective Eurocord-EBMT analysis

Author

Cunha, R, Loiseau, P, Ruggeri, A, Sanz, G, Michel, G, PaolaIori, A, Socié, G, Arcese, W, Picardi, A, Dias de Heredia, C, Rio, B, Locatelli, F, O'Brien, T A, Yakoub-Agha, I, Angel Diaz, M, Milpied, N, Bittencourt, H, Pedro Souza, M, Aljurf, M, Charron, D, Boudjedir, K, Labopin, M, Gluckman, E, and Rocha, V

Published
2014
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12. Association study of candidate DNA-repair gene variants and acute graft versus host disease in pediatric patients receiving allogeneic hematopoietic stem-cell transplantation

Author

Uppugunduri, C. R. S., primary, Huezo-Diaz Curtis, P., additional, Nava, T., additional, Rezgui, M. A., additional, Mlakar, V., additional, Mlakar, S. Jurkovic, additional, Waespe, N., additional, Théoret, Y., additional, Gumy-Pause, F., additional, Bernard, F., additional, Chalandon, Y., additional, Boelens, J. J., additional, Bredius, R. G. M., additional, Dalle, J. H., additional, Nath, C., additional, Corbacioglu, S., additional, Peters, C., additional, Bader, P., additional, Shaw, P., additional, Bittencourt, H., additional, Krajinovic, M., additional, and Ansari, M., additional

Published
2021
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21. Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia

Author

Maude, S, Laetsch, T, Buechner, J, Rives, S, Boyer, M, Bittencourt, H, Bader, P, Verneris, M, Stefanski, H, Myers, G, Qayed, M, De Moerloose, B, Hiramatsu, H, Schlis, K, Davis, K, Martin, P, Nemecek, E, Yanik, G, Peters, C, Baruchel, A, Boissel, N, Mechinaud, F, Balduzzi, A, Krueger, J, June, C, Levine, B, Wood, P, Taran, T, Leung, M, Mueller, K, Zhang, Y, Sen, K, Lebwohl, D, Pulsipher, M, Grupp, S, Maude SL, Laetsch TW, Buechner J, Rives S, Boyer M, Bittencourt H, Bader P, Verneris MR, Stefanski HE, Myers GD, Qayed M, De Moerloose B, Hiramatsu H, Schlis K, Davis KL, Martin PL, Nemecek ER, Yanik GA, Peters C, Baruchel A, Boissel N, Mechinaud F, Balduzzi A, Krueger J, June CH, Levine BL, Wood P, Taran T, Leung M, Mueller KT, Zhang Y, Sen K, Lebwohl D, Pulsipher MA, Grupp SA, Maude, S, Laetsch, T, Buechner, J, Rives, S, Boyer, M, Bittencourt, H, Bader, P, Verneris, M, Stefanski, H, Myers, G, Qayed, M, De Moerloose, B, Hiramatsu, H, Schlis, K, Davis, K, Martin, P, Nemecek, E, Yanik, G, Peters, C, Baruchel, A, Boissel, N, Mechinaud, F, Balduzzi, A, Krueger, J, June, C, Levine, B, Wood, P, Taran, T, Leung, M, Mueller, K, Zhang, Y, Sen, K, Lebwohl, D, Pulsipher, M, Grupp, S, Maude SL, Laetsch TW, Buechner J, Rives S, Boyer M, Bittencourt H, Bader P, Verneris MR, Stefanski HE, Myers GD, Qayed M, De Moerloose B, Hiramatsu H, Schlis K, Davis KL, Martin PL, Nemecek ER, Yanik GA, Peters C, Baruchel A, Boissel N, Mechinaud F, Balduzzi A, Krueger J, June CH, Levine BL, Wood P, Taran T, Leung M, Mueller KT, Zhang Y, Sen K, Lebwohl D, Pulsipher MA, and Grupp SA

Abstract

BACKGROUND In a single-center phase 1-2a study, the anti-CD19 chimeric antigen receptor (CAR) T-cell therapy tisagenlecleucel produced high rates of complete remission and was associated with serious but mainly reversible toxic effects in children and young adults with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL). METHODS We conducted a phase 2, single-cohort, 25-center, global study of tisagenlecleucel in pediatric and young adult patients with CD19+ relapsed or refractory B-cell ALL. The primary end point was the overall remission rate (the rate of complete remission or complete remission with incomplete hematologic recovery) within 3 months. RESULTS For this planned analysis, 75 patients received an infusion of tisagenlecleucel and could be evaluated for efficacy. The overall remission rate within 3 months was 81%, with all patients who had a response to treatment found to be negative for minimal residual disease, as assessed by means of flow cytometry. The rates of event-free survival and overall survival were 73% (95% confidence interval [CI], 60 to 82) and 90% (95% CI, 81 to 95), respectively, at 6 months and 50% (95% CI, 35 to 64) and 76% (95% CI, 63 to 86) at 12 months. The median duration of remission was not reached. Persistence of tisagenlecleucel in the blood was observed for as long as 20 months. Grade 3 or 4 adverse events that were suspected to be related to tisagenlecleucel occurred in 73% of patients. The cytokine release syndrome occurred in 77% of patients, 48% of whom received tocilizumab. Neurologic events occurred in 40% of patients and were managed with supportive care, and no cerebral edema was reported. CONCLUSIONS In this global study of CAR T-cell therapy, a single infusion of tisagenlecleucel provided durable remission with long-term persistence in pediatric and young adult patients with relapsed or refractory B-cell ALL, with transient high-grade toxic effects.

Published
2018

38. Sickle cell disease: An international survey of results of HLA-identical sibling hematopoietic stem cell transplantation

Author

Gluckman, E., Cappelli, B., Bernaudin, F., Labopin, M., Volt, F., Carreras, J., Simoes, B. P., Ferster, A., Dupont, S., De La Fuente, J., Dalle, J. -H., Zecca, M., Walters, M. C., Krishnamurti, L., Bhatia, M., Leung, K., Yanik, G., Kurtzberg, J., Dhedin, N., Kuentz, M., Michel, G., Apperley, J., Lutz, P., Neven, B., Bertrand, Y., Vannier, J. P., Ayas, M., Cavazzana, M., Matthes-Martin, S., Rocha, V., Elayoubi, H., Kenzey, C., Bader, P., Locatelli, Franco, Ruggeri, A., Eapen, M., Bordon, V., Labarque, V., Pereira, M., Bittencourt, H., Petersen, H., Deconninck, E., Jubert, C., Perrin, J., Cahn, J. Y., Bruno, B., Bordigoni, P., Mechinaud, F., Vernant, J. P., Stephan, J. L., Suttorp, M., Strahm, B., Da Cunha, C. B., Garwer, B., Rothmayer, M., Wendelin, K., Graphakos, S., Tbakhi, A., Naeimi, N., Zuckerman, T., Sharon, P. B., Yaniv, I., Amos, T., Prete, A., Lo Nigro, L., Lanino, E., Faraci, M., Ciceri, F., Marktel, S., De Simone, G., Messina, C., Bartolomeo, P. D. I., Santarone, S., Vallisa, D., Bertaina, A., Arcese, W., Foa, R., Berger, M., Maximova, N., Wallet, S., Bazuaye, G. N., Maschan, A., De Heredia, C. D., Bieler, C. B., Pato, J. R., Heras, I., Trevor, R., Abayomi, K., Thomson, J., Fasth, A., Frodin, U., Ljugman, P., Ansari, M., Gungor, T., Unal, E., Pehlivan, M., Anak, S., Ozturk, G., Unal, A., Lawson, S., Keshani, J., Drake, A., Wynn, R., Williams, J., Jagsia, M., Leung, W., Abraham, A., Sahdey, I., Margolis, D., Eames, G., Horwitz, E., Cowan, M., Kapoor, N., Rowley, S., Megason, G., Rogers, Z., Bolanos-Meade, J., Hudspeth, M., Rosenthal, J., Olson, T., Kassow, K., Selby, G., Haines, H., Chaudhury, S., Gluckman, E., Cappelli, B., Bernaudin, F., Labopin, M., Volt, F., Carreras, J., Simoes, B. P., Ferster, A., Dupont, S., De La Fuente, J., Dalle, J. -H., Zecca, M., Walters, M. C., Krishnamurti, L., Bhatia, M., Leung, K., Yanik, G., Kurtzberg, J., Dhedin, N., Kuentz, M., Michel, G., Apperley, J., Lutz, P., Neven, B., Bertrand, Y., Vannier, J. P., Ayas, M., Cavazzana, M., Matthes-Martin, S., Rocha, V., Elayoubi, H., Kenzey, C., Bader, P., Locatelli, F., Ruggeri, A., Eapen, M., Bordon, V., Labarque, V., Pereira, M., Bittencourt, H., Petersen, H., Deconninck, E., Jubert, C., Perrin, J., Cahn, J. Y., Bruno, B., Bordigoni, P., Mechinaud, F., Vernant, J. P., Stephan, J. L., Suttorp, M., Strahm, B., Da Cunha, C. B., Garwer, B., Rothmayer, M., Wendelin, K., Graphakos, S., Tbakhi, A., Naeimi, N., Zuckerman, T., Sharon, P. B., Yaniv, I., Amos, T., Prete, A., Lo Nigro, L., Lanino, E., Faraci, M., Ciceri, F., Marktel, S., De Simone, G., Messina, C., Bartolomeo, P. D. I., Santarone, S., Vallisa, D., Bertaina, A., Arcese, W., Foa, R., Berger, M., Maximova, N., Wallet, S., Bazuaye, G. N., Maschan, A., De Heredia, C. D., Bieler, C. B., Pato, J. R., Heras, I., Trevor, R., Abayomi, K., Thomson, J., Fasth, A., Frodin, U., Ljugman, P., Ansari, M., Gungor, T., Unal, E., Pehlivan, M., Anak, S., Ozturk, G., Unal, A., Lawson, S., Keshani, J., Drake, A., Wynn, R., Williams, J., Jagsia, M., Leung, W., Abraham, A., Sahdey, I., Margolis, D., Eames, G., Horwitz, E., Cowan, M., Kapoor, N., Rowley, S., Megason, G., Rogers, Z., Bolanos-Meade, J., Hudspeth, M., Rosenthal, J., Olson, T., Kassow, K., Selby, G., Haines, H., Chaudhury, S., France Monacord, Centre Scientifique de Monaco (CSM), CHI Créteil, Centre de Recherche Saint-Antoine (CR Saint-Antoine), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Pierre et Marie Curie - Paris 6 (UPMC), Eurocord, Assistance publique - Hôpitaux de Paris (AP-HP) (APHP)-Institut Universitaire d'Hématologie (IUH), Université Paris Diderot - Paris 7 (UPD7)-Université Paris Diderot - Paris 7 (UPD7), Hôpital Universitaire des Enfants - Reine Fabiola, Université Libre de Bruxelles, Equipe NEMESIS - Centre de Recherches de l'Institut du Cerveau et de la Moelle épinière (NEMESIS-CRICM), Centre de Recherche de l'Institut du Cerveau et de la Moelle épinière (CRICM), Centre National de la Recherche Scientifique (CNRS)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Pierre et Marie Curie - Paris 6 (UPMC)-Centre National de la Recherche Scientifique (CNRS)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Pierre et Marie Curie - Paris 6 (UPMC), Unité d'Hémato-Immunologie pédiatrique [Hôpital Robert Debré, Paris], Service d'Immuno-hématologie pédiatrique [Hôpital Robert Debré, Paris], Hôpital Robert Debré-Hôpital Robert Debré, Duke University Medical Center, Service de Greffe de Moelle - Unité AJA, Assistance publique - Hôpitaux de Paris (AP-HP) (APHP)-Groupe Hospitalier Saint Louis - Lariboisière - Fernand Widal [Paris], Assistance publique - Hôpitaux de Paris (AP-HP) (APHP), Service d'hématologie clinique, Assistance publique - Hôpitaux de Paris (AP-HP) (APHP)-Hôpital Henri Mondor-Université Paris-Est Créteil Val-de-Marne - Paris 12 (UPEC UP12), Hôpital de la Timone [CHU - APHM] (TIMONE), Department of Haematology, Hôpital Civil, Hopital Civil, Département d'Immunologie, hématologie et rhumatologie pédiatriques [Hôpital Necker-Enfants malades - APHP], CHU Necker - Enfants Malades [AP-HP], Centre Hospitalier Lyon Sud [CHU - HCL] (CHLS), Hospices Civils de Lyon (HCL), Hemostase, Endothelium, Angiogenese (UMR_S_553), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris Diderot - Paris 7 (UPD7), Physiopathologie, Autoimmunité, maladies Neuromusculaires et THErapies Régénératrices (PANTHER), Université de Rouen Normandie (UNIROUEN), Normandie Université (NU)-Normandie Université (NU)-Institut National de la Santé et de la Recherche Médicale (INSERM), Imagine - Institut des maladies génétiques (IMAGINE - U1163), Centre National de la Recherche Scientifique (CNRS)-Université Paris Descartes - Paris 5 (UPD5)-Institut National de la Santé et de la Recherche Médicale (INSERM), Churchill Hospital [Breast Care Unit], Churchill Hospital Oxford Centre for Haematology, Santa Lucia Foundation, IRCSS, Rome, Medical College of Wisconsin, National Institute for Health Research, Centre de Recherche Saint-Antoine (UMRS893), Université Pierre et Marie Curie - Paris 6 (UPMC)-Institut National de la Santé et de la Recherche Médicale (INSERM), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Institut Universitaire d'Hématologie (IUH), Hôpital Universitaire des Enfants Reine Fabiola [Bruxelles, Belgique] (HUDERF), Université Pierre et Marie Curie - Paris 6 (UPMC)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-Université Pierre et Marie Curie - Paris 6 (UPMC)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Groupe Hospitalier Saint Louis - Lariboisière - Fernand Widal [Paris], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Hôpital Henri Mondor-Université Paris-Est Créteil Val-de-Marne - Paris 12 (UPEC UP12), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Université Paris Diderot - Paris 7 (UPD7)-Institut National de la Santé et de la Recherche Médicale (INSERM), Université Paris Descartes - Paris 5 (UPD5)-Institut National de la Santé et de la Recherche Médicale (INSERM), UCL - SSS/IREC/PEDI - Pôle de Pédiatrie, and UCL - (SLuc) Service d'hématologie et d'oncologie pédiatrique

Subjects
Male, HYDROXYUREA, Transplantation Conditioning, [SDV]Life Sciences [q-bio], medicine.medical_treatment, CHILDREN, Hematopoietic stem cell transplantation, Biochemistry, THALASSEMIA, 0302 clinical medicine, HLA Antigens, Surveys and Questionnaires, 1114 Paediatrics And Reproductive Medicine, Child, ComputingMilieux_MISCELLANEOUS, CÉLULAS-TRONCO, Hazard ratio, Graft Survival, BONE-MARROW TRANSPLANT, Hematopoietic Stem Cell Transplantation, Hematology, Sickle cell anemia, 3. Good health, Survival Rate, medicine.anatomical_structure, Treatment Outcome, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, 030220 oncology & carcinogenesis, Child, Preschool, Histocompatibility, [SDV.IMM]Life Sciences [q-bio]/Immunology, Female, Life Sciences & Biomedicine, medicine.medical_specialty, Adolescent, Anemia, Immunology, Anemia, Sickle Cell, 1102 Cardiovascular Medicine And Haematology, Disease-Free Survival, 03 medical and health sciences, Internal medicine, MANAGEMENT, medicine, Humans, ANEMIA, Survival rate, Transplantation, Science & Technology, business.industry, Siblings, Sickle cell disease, Infant, 1103 Clinical Sciences, ADULTS, Cell Biology, medicine.disease, LIFE, EXPERT PANEL, Bone marrow, FOLLOW-UP, business, 030215 immunology
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

42. Erratum: Impact of HLA mismatch direction on outcomes after umbilical cord blood transplantation for hematological malignant disorders: a retrospective Eurocord-EBMT analysis

Author

Cunha, R, Loiseau, P, Ruggeri, A, Sanz, G, Michel, G, Iori, A P, Socié, G, Arcese, W, Picardi, A, Dias de Heredia, C, Rio, B, Locatelli, F, O'Brien, T A, Yakoub-Agha, I, Diaz, M A, Milpied, N, Bittencourt, H, Souza, M P, Aljurf, M, Charron, D, Boudjedir, K, Labopin, M, Gluckman, E, and Rocha, V

Published
2014
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43. 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, A., Dalle, J. H., Ayas, M., Boelens, J. J., Volt, F., Iori, A. P., de Souza, M. P., Diaz, M. A., Michel, G., Locatelli, Franco, Jubert, C., Yakoub-Agha, I., Bittencourt, H., Bertrand, Y., Kenzey, C., Tozatto Maio, K., Hayashi, H., Rocha, V., Bader, P., Gluckman, E., Ruggeri, A., Locatelli F. (ORCID:0000-0002-7976-3654), Paviglianiti, A., Dalle, J. H., Ayas, M., Boelens, J. J., Volt, F., Iori, A. P., de Souza, M. P., Diaz, M. A., Michel, G., Locatelli, Franco, Jubert, C., Yakoub-Agha, I., Bittencourt, H., Bertrand, Y., Kenzey, C., Tozatto Maio, K., Hayashi, H., Rocha, V., Bader, P., Gluckman, E., Ruggeri, A., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

Body mass index (BMI) may influence outcomes after allogeneic hematopoietic stem cell transplantation (HSCT). However, the impact of BMI on survival in children undergoing HSCT is not well defined, with conflicting results being reported on this issue. We analyzed 855 patients age 2 to 20 years with diagnosis of acute leukemia who underwent umbilical cord blood transplantation (UCBT) from 1990 to 2015. Patients were classified according to BMI as normal (fifth to 85th percentile), underweight (less than fifth percentile), overweight (85th to 95th percentile), and obese (>95th percentile) using growth charts for age and sex. All patients received single-unit UCBT after a myeloablative conditioning regimen. Diagnosis was acute lymphoblastic leukemia in 68% of the patients. Sixty-one percent of patients (n = 523) were in the normal BMI category, 11% (n = 96) were underweight, 16% (n = 137) overweight, and 12% (n = 99) obese. The cumulative incidence of grade II to IV acute graft-versus-host disease (aGVHD) was 35% (32% to 38%). According to pretransplantation BMI, aGVHD was 46% (33% to 59%) for underweight, 34% (31% to 42%) for normal, 36% (18% to 38%) for overweight, and 27% (15% to 37%) for obese (P =.04). In multivariate analysis, a BMI less than the fifth percentile was associated with higher incidence of acute grade II to IV GVHD compared with normal-BMI patients (hazard ratio, 1.61; 95% confidence interval, 1.15 to 2.26; P =.006). Our results show that being underweight at the time of transplantation is associated with an increased risk of aGVHD, highlighting the importance of nutritional status before UCBT.

Published
2018

44. A risk factor analysis of outcomes after unrelated cord blood transplantation for children with Wiskott-Aldrich syndrome.

Author

Shekhovtsova, Z, Bonfim, C, Ruggeri, A, Nichele, S, Page, K, AlSeraihy, A, Barriga, F, de Toledo Codina, JS, Veys, P, Boelens, JJ, Mellgren, K, Bittencourt, H, O'Brien, T, Shaw, PJ, Chybicka, A, Volt, F, Giannotti, F, Gluckman, E, Kurtzberg, J, Gennery, AR, Rocha, V, Eurocord, Cord Blood Committee of Cellular Therapy and Immunobiology Working Party of the EBMT, Federal University of Parana, Duke University Medical Center and Inborn Errors Working Party of the EBMT, Shekhovtsova, Z, Bonfim, C, Ruggeri, A, Nichele, S, Page, K, AlSeraihy, A, Barriga, F, de Toledo Codina, JS, Veys, P, Boelens, JJ, Mellgren, K, Bittencourt, H, O'Brien, T, Shaw, PJ, Chybicka, A, Volt, F, Giannotti, F, Gluckman, E, Kurtzberg, J, Gennery, AR, Rocha, V, and Eurocord, Cord Blood Committee of Cellular Therapy and Immunobiology Working Party of the EBMT, Federal University of Parana, Duke University Medical Center and Inborn Errors Working Party of the EBMT

Abstract

Wiskott-Aldrich syndrome is a severe X-linked recessive immune deficiency disorder. A scoring system of Wiskott-Aldrich syndrome severity (0.5-5) distinguishes two phenotypes: X-linked thrombocytopenia and classic Wiskott-Aldrich syndrome. Hematopoietic cell transplantation is curative for Wiskott-Aldrich syndrome; however, the use of unrelated umbilical cord blood transplantation has seldom been described. We analyzed umbilical cord blood transplantation outcomes for 90 patients. The median age at umbilical cord blood transplantation was 1.5 years. Patients were classified according to clinical scores [2 (23%), 3 (30%), 4 (23%) and 5 (19%)]. Most patients underwent HLA-mismatched umbilical cord blood transplantation and myeloablative conditioning with anti-thymocyte globulin. The cumulative incidence of neutrophil recovery at day 60 was 89% and that of grade II-IV acute graft-versus-host disease at day 100 was 38%. The use of methotrexate for graft-versus-host disease prophylaxis delayed engraftment (P=0.02), but decreased acute graft-versus-host disease (P=0.03). At 5 years, overall survival and event-free survival rates were 75% and 70%, respectively. The estimated 5-year event-free survival rates were 83%, 73% and 55% for patients with a clinical score of 2, 4-5 and 3, respectively. In multivariate analysis, age <2 years at the time of the umbilical cord blood transplant and a clinical phenotype of X-linked thrombocytopenia were associated with improved event-free survival. Overall survival tended to be better in patients transplanted after 2007 (P=0.09). In conclusion, umbilical cord blood transplantation is a good alternative option for young children with Wiskott-Aldrich syndrome lacking an HLA identical stem cell donor.

Published
2017

45. Unrelated cord blood transplantation for childhood acute myelogenous leukemia: The influence of cytogenetic risk group stratification

Author

Michel, G., Cunha, R., Ruggeri, A., O'Brien, T. A., Bittencourt, H., Dalle, J. H., Locatelli, Franco, Iori, A. P., Mauad, M., Oudin, C., Giannotti, F., Volt, F., Gluckman, E., Bader, P., Rocha, V., Locatelli F. (ORCID:0000-0002-7976-3654), Michel, G., Cunha, R., Ruggeri, A., O'Brien, T. A., Bittencourt, H., Dalle, J. H., Locatelli, Franco, Iori, A. P., Mauad, M., Oudin, C., Giannotti, F., Volt, F., Gluckman, E., Bader, P., Rocha, V., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

No abstract available

Published
2016

46. Erratum: Impact of HLA mismatch direction on outcomes after umbilical cord blood transplantation for hematological malignant disorders: A retrospective Eurocord-EBMT analysis (Bone Marrow Transplantation (2014) 49 (864))

Author

Cunha, R., Loiseau, P., Ruggeri, A., Sanz, G., Michel, G., Iori, A. P., Socie, G., Arcese, W., Picardi, A., Dias De Heredia, C., Rio, B., Locatelli, F., O'Brien, T. A., Yakoub-Agha, I., Diaz, M. A., Milpied, N., Bittencourt, H., Souza, M. P., Aljurf, M., Charron, D., Boudjedir, K., Labopin, M., Gluckman, E., and Rocha, V.

Published
2014

48. Effect of Different Computer-aided Design/Computer-aided Manufacturing (CAD/CAM) Materials and Thicknesses on the Fracture Resistance of Occlusal Veneers.

Author

Andrade, J. P., Stona, D., Bittencourt, H. R., Borges, G. A., Burnett Júnior, L. H., and Spohr, A. M.

Subjects
DENTAL veneers, CAD/CAM systems, DENTAL materials
Abstract

The aim was to evaluate, in vitro, the influence of different computer-aided design/computeraidedmanufacturing (CAD/CAM)materials (IPS e.max CAD, Vita Enamic, and Lava Ultimate) and thicknesses (0.6 mm and 1.5 mm) on the fracture resistance of occlusal veneers. Sixty human third molars were prepared to simulate advanced erosion of the occlusal surface, and the teeth were randomly divided into six experimental groups (n=10) according to thematerial and thickness used to build the veneers. Ten sound teeth formed the control group. The veneers were adhesively luted and submitted to mechanical cyclic loading (1 million cycles at 200-N load). The fracture resistance test was performed in a universal testing machine. The failures were classified as ''reparable'' and ''irreparable.'' According to two-way analysis of variance and the Tukey test, the interaction (material3thickness) was significant (p=0.013). The highest fracture resistance was obtained for IPS e.max CAD at a 1.5-mm thickness (4995 N) andwas significantly higher compared to the other experimental groups (p<0.05). The lowest fracture resistance was obtained for Vita Enamic at 0.6mm(2973N), although this resistance was not significantly different from those for IPS e.max CAD at 0.6 mm (3067 N), Lava Ultimate at 0.6 mm (3384 N), Vita Enamic at 1.5 mm (3540 N), and Lava Ultimate at 1.5 mm (3584 N) (p>0.05). The experimental groups did not differ significantly from the sound teeth (3991 N) (p>0.05). The failures were predominantly repairable. The occlusal veneers of IPS e.max CAD, Vita Enamic, and Lava Ultimate, with thicknesses of 0.6 mm and 1.5 mm, obtained fracture resistances similar to those associated with sound teeth. [ABSTRACT FROM AUTHOR]

Published
2018
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49. TR-16 * PERSONALIZED TARGETED THERAPY IN REFRACTORY OR RELAPSED CANCER IN CHILDHOOD (TRICEPS STUDY)

Author

Dumoucel, S., primary, Duval, M., additional, Marzouki, M., additional, Bittencourt, H., additional, Samson, Y., additional, Cellot, S., additional, Rivard, G.-E., additional, Teira, P., additional, Carret, A.-S., additional, Healy, J., additional, Dal Soglio, D., additional, Patey, N., additional, Piche, N., additional, Sontag, T., additional, Langlois, S., additional, Lajoie, M., additional, and Sinnett, D., additional

Published
2015
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50. Validation of the EBMT risk score in chronic myeloid leukemia in Brazil and allogeneic transplant outcome

Author

Carmino De Souza, Vigorito, A. C., Ruiz, M. A., Nucci, M., Dulley, F. L., Funcke, V., Tabak, D., Azevedo, A. M., Byington, R., Macedo, M. C., Saboya, R., Aranha, F. J. P., Oliveira, G. B., Zulli, R., Miranda, E. C. M., Azevedo, W. M., Lodi, F. M., Voltarelli, J. C., Simões, B. P., Colturato, V., Souza, M. P., Silla, L., Bittencourt, H., Piron-Ruiz, L., Maiolino, A., Gratwohl, A., and Pasquini, R.

Subjects
Adult, Male, Risk, Adolescent, Hematopoietic Stem Cell Transplantation, Infant, Middle Aged, Disease-Free Survival, Sex Factors, Treatment Outcome, Child, Preschool, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Humans, Transplantation, Homologous, Female, Child, Brazil, Retrospective Studies
Abstract

The management of chronic myeloid leukemia (CML) has changed radically since the introduction of imatinib therapy. The decision of whether to offer a patient a hematopoietic stem cell transplant (HSCT) must be based on the probability of success of the procedure. The aim of this retrospective analysis of 1,084 CML patients who received an allogeneic HSCT in 10 Brazilian Centers between February 1983 and March 2003 was to validate the EBMT risk score.The study population comprised 647 (60%) males and 437 (40%) females, with a median age of 32 years old (range 1 - 59); 898 (83%) were in chronic phase, 146 (13%) were in accelerated phase and 40 (4%) were in blast crisis; 151 (14%) were younger than 20 years old, 620 (57%) were between 20 and 40 and 313 (29%) were older than 40; 1,025 (94%) received an HLA fully matched sibling transplant and only 59 (6%) received an unrelated transplant. In 283 cases (26%) a male recipient received a graft from a female donor. The interval from diagnosis to transplantation was less than 12 months in 223 (21%) cases and greater in 861 (79%). The overall survival, disease-free survival, transplant-related mortality and relapse incidence were 49%, 50%, 45% and 25%, respectively.Of the 1084 patients, 179 (17%) had a risk score of 0 or 1, 397 (37%) had a score of 2, 345 (32%) had a score of 3, 135 (12%) had a score of 4 and 28 (2%) a score of 5 or 6. The overall survival (OS) rate in patients with risk scores 0-1 and 2 was similar (58% and 55%, respectively) but significantly better than that in patients with scores 3 or more (score 3 - 44%, 4 - 36 % and 5-6 - 27%, respectively) pp0.001). Disease-free survival (DFS) and transplant related mortality (TRM) in a patients with a score of 3 or more were 46% and 49%, respectively and the relapse rate beyond score 5-6 was 77%. Disease status had a negative impact on all outcomes (OS, DFS, TRM, and relapse). The OS rate for male recipients of a graft from a female donor was 40% compared to 52% among the other donor-recipient pairs (p=0.004). DFS and TRM were significant for disease phase and female donor-male recipient (p0.001 and p0.003, respectively). In our experience, age and interval between diagnosis and transplant did influence OS, DFS, TRM, and relapse rate.Our results validate the EBMT risk score in the context of a developing country and confirm its usefulness for making point decisions in the imatinib era.

Published
2005

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