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1. Impact of childhood leukemia on siblings: their long-term perception of family functioning and its relationship with their psychosocial characteristics using structural equation modeling

Author

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

Published
2024
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3. CAR T-cells for T-cell acute lymphoblastic leukemia

Author

Marie Emilie Dourthe and André Baruchel

Subjects
T-cell acute lymphoblastic leukemia, Immunotherapy, Allogeneic CAR T-cells, Donor-derived CAR T-cells, Neoplasms. Tumors. Oncology. Including cancer and carcinogens, RC254-282
Abstract

T-cell acute lymphoblastic leukemia (T-ALL) has a dismal prognosis in case of relapsed or refractory disease. Contrary to B-ALL, few immunotherapies are available for T-ALL. Use of autologous CAR T-cells is challenging due to shared antigen between leukemic and normal T-cells responsible for fratricide and T-cell aplasia in case of persistence of CAR T-cells. Moreover, risk of contamination of the apheresis product by lymphoblasts remains an issue. To counteract these challenges several methods are used to edit T-cell such as protein expression blocker, CRISPR/Cas9 and base-editing. Other possibility is to use autologous T-cells naturally selected in vitro or donor-derived T-cells allowing gene edition and reduction of the risk of graft vs host disease. Encouraging results are obtained in preclinical and clinical studies for early response rate but several questions remain. Is the persistence of these cells requiring for maintaining the remission? Is it feasible to recover a target-negative T-cell population without risk of profound immunosuppression? Has an allogeneic stem cell transplantation to be planned for patients after CAR T-cells infusion? What about the risk of engineered T-cells in the long term?

Published
2024
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4. Daratumumab in pediatric relapsed/refractory acute lymphoblastic leukemia or lymphoblastic lymphoma: the DELPHINUS study

Author

Bhatla, Teena, Hogan, Laura E., Teachey, David T., Bautista, Francisco, Moppett, John, Velasco Puyó, Pablo, Micalizzi, Concetta, Rossig, Claudia, Shukla, Neerav, Gilad, Gil, Locatelli, Franco, Baruchel, André, Zwaan, C. Michel, Bezler, Natalie S., Rubio-San-Simón, Alba, Taussig, David C., Raetz, Elizabeth A., Mao, Zhengwei J., Wood, Brent L., Alvarez Arias, Diana, Krevvata, Maria, Nnane, Ivo, Bandyopadhyay, Nibedita, Lopez Solano, Lorena, Dennis, Robyn M., Carson, Robin, and Vora, Ajay

Published
2024
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5. NGS-based stratification refines the risk stratification in T-ALL and identifies a very-high-risk subgroup of patients

Author

Simonin, Mathieu, Vasseur, Loïc, Lengliné, Etienne, Lhermitte, Ludovic, Cabannes-Hamy, Aurélie, Balsat, Marie, Schmidt, Aline, Dourthe, Marie-Emilie, Touzart, Aurore, Graux, Carlos, Grardel, Nathalie, Cayuela, Jean-Michel, Arnoux, Isabelle, Gandemer, Virginie, Huguet, Françoise, Ducassou, Stéphane, Lhéritier, Véronique, Chalandon, Yves, Ifrah, Norbert, Dombret, Hervé, Macintyre, Elizabeth, Petit, Arnaud, Rousselot, Philippe, Lambert, Jérôme, Baruchel, André, Boissel, Nicolas, and Asnafi, Vahid

Published
2024
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7. ATM germ line pathogenic variants affect outcomes in children with ataxia-telangiectasia and hematological malignancies

Author

Elitzur, Sarah, Shiloh, Ruth, Loeffen, Jan L. C., Pastorczak, Agata, Takagi, Masatoshi, Bomken, Simon, Baruchel, Andre, Lehrnbecher, Thomas, Tasian, Sarah K., Abla, Oussama, Arad-Cohen, Nira, Astigarraga, Itziar, Ben-Harosh, Miriam, Bodmer, Nicole, Brozou, Triantafyllia, Ceppi, Francesco, Chugaeva, Liliia, Dalla Pozza, Luciano, Ducassou, Stephane, Escherich, Gabriele, Farah, Roula, Gibson, Amber, Hasle, Henrik, Hoveyan, Julieta, Jacoby, Elad, Jazbec, Janez, Junk, Stefanie, Kolenova, Alexandra, Lazic, Jelena, Lo Nigro, Luca, Mahlaoui, Nizar, Miller, Lane, Papadakis, Vassilios, Pecheux, Lucie, Pillon, Marta, Sarouk, Ifat, Stary, Jan, Stiakaki, Eftichia, Strullu, Marion, Tran, Thai Hoa, Ussowicz, Marek, Verdu-Amoros, Jaime, Wakulinska, Anna, Zawitkowska, Joanna, Stoppa-Lyonnet, Dominique, Taylor, A. Malcolm, Shiloh, Yosef, Izraeli, Shai, Minard-Colin, Veronique, Schmiegelow, Kjeld, Nirel, Ronit, Attarbaschi, Andishe, and Borkhardt, Arndt

Published
2024
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8. Genomic imbalance analysis provides new insight into prognostic factors in adult and pediatric T-ALL

Author

Balducci, Estelle, Simonin, Mathieu, Duployez, Nicolas, Steimlé, Thomas, Dourthe, Marie-Emilie, Villarese, Patrick, Ducassou, Stéphane, Arnoux, Isabelle, Cayuela, Jean-Michel, Balsat, Marie, Courtois, Lucien, Andrieu, Guillaume, Touzart, Aurore, Huguet, Françoise, Petit, Arnaud, Ifrah, Norbert, Dombret, Hervé, Baruchel, André, Macintyre, Elizabeth, Preudhomme, Claude, Boissel, Nicolas, and Asnafi, Vahid

Published
2024
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11. TREC mediated oncogenesis in human immature T lymphoid malignancies preferentially involves ZFP36L2

Author

Balducci, Estelle, Steimlé, Thomas, Smith, Charlotte, Villarese, Patrick, Feroul, Mélanie, Payet-Bornet, Dominique, Kaltenbach, Sophie, Couronné, Lucile, Lhermitte, Ludovic, Touzart, Aurore, Dourthe, Marie-Emilie, Simonin, Mathieu, Baruchel, André, Dombret, Hervé, Ifrah, Norbert, Boissel, Nicolas, Nadel, Bertrand, Macintyre, Elizabeth, Cieslak, Agata, and Asnafi, Vahid

Published
2023
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12. Harnessing the MYB-dependent TAL1 5’super-enhancer for targeted therapy in T-ALL

Author

Smith, Charlotte, Touzart, Aurore, Simonin, Mathieu, Tran-Quang, Christine, Hypolite, Guillaume, Latiri, Mehdi, Andrieu, Guillaume P., Balducci, Estelle, Dourthe, Marie-Émilie, Goyal, Ashish, Huguet, Françoise, Petit, Arnaud, Ifrah, Norbert, Baruchel, André, Dombret, Hervé, Macintyre, Elizabeth, Plass, Christoph, Ghysdael, Jacques, Boissel, Nicolas, and Asnafi, Vahid

Published
2023
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13. Prognostic impact of RUNX1 mutations and deletions in pediatric acute myeloid leukemia: results from the French ELAM02 study group

Author

Lew-Derivry, Lucille, Marceau-Renaut, Alice, Fenwarth, Laurène, Cuccuini, Wendy, Ballerini, Paola, Ferreboeuf, Maxime, Guilmatre, Audrey, Petit, Arnaud, Gandemer, Virginie, Rialland, Fanny, Schneider, Pascale, Michel, Gérard, Bertrand, Yves, Baruchel, Andre, Preudhomme, Claude, Leverger, Guy, and Lapillonne, Hélène

Published
2023
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14. Hematopoietic stem cell transplantation for acute lymphoblastic leukemia: why do adolescents and young adults outcomes differ from those of children? A retrospective study on behalf of the Francophone Society of Stem Cell Transplantation and Cellular Therapy (SFGM-TC)

Author

Grain, Audrey, Rialland-Battisti, Fanny, Chevallier, Patrice, Blin, Nicolas, Dalle, Jean-Hugues, Michel, Gérard, Dhédin, Nathalie, Peffault de Latour, Regis, Pochon, Cécile, Yakoub-Agha, Ibrahim, Bertrand, Yves, Sirvent, Anne, Jubert, Charlotte, Forcade, Edouard, Berceanu, Ana, Gandemer, Virginie, Schneider, Pascale, Bay, Jacques-Olivier, Rohrlich, Pierre-Simon, Brissot, Eolia, Paillard, Catherine, Plantaz, Dominique, Nguyen Quoc, Stéphanie, Gonzales, Fanny, Maillard, Natacha, Planche, Lucie, and Baruchel, André

Published
2023
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15. TREC mediated oncogenesis in human immature T lymphoid malignancies preferentially involves ZFP36L2

Author

Estelle Balducci, Thomas Steimlé, Charlotte Smith, Patrick Villarese, Mélanie Feroul, Dominique Payet-Bornet, Sophie Kaltenbach, Lucile Couronné, Ludovic Lhermitte, Aurore Touzart, Marie-Emilie Dourthe, Mathieu Simonin, André Baruchel, Hervé Dombret, Norbert Ifrah, Nicolas Boissel, Bertrand Nadel, Elizabeth Macintyre, Agata Cieslak, and Vahid Asnafi

Subjects
T-cell receptor excision circles (TREC), Oncogenesis, Cancer, Neoplasms. Tumors. Oncology. Including cancer and carcinogens, RC254-282
Abstract

Abstract The reintegration of excised signal joints resulting from human V(D)J recombination was described as a potent source of genomic instability in human lymphoid cancers. However, such molecular events have not been recurrently reported in clinical patient lymphoma/leukemia samples. Using a specifically designed NGS-capture pipeline, we here demonstrated the reintegration of T-cell receptor excision circles (TRECs) in 20/1533 (1.3%) patients with T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL). Remarkably, the reintegration of TREC recurrently targeted the tumor suppressor gene, ZFP36L2, in 17/20 samples. Thus, our data identified a new and hardly detectable mechanism of gene deregulation in lymphoid cancers providing new insights in human oncogenesis.

Published
2023
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16. 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

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

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Abstract

Juvenile myelomonocytic leukemia (JMML) is an aggressive pediatric myeloproliferative neoplasm requiring hematopoietic stem cell transplantation (HSCT) in most cases. We retrospectively analyzed 119 JMML patients who underwent first allogeneic HSCT between 2002 and 2021. The majority (97%) carried a RAS-pathway mutation, and 62% exhibited karyotypic alterations or additional mutations in SETBP1, ASXL1, JAK3 and/or the RAS pathway. Relapse was the primary cause of death, with a 5-year cumulative incidence of 24.6% (95%CI: 17.1-32.9). Toxic deaths occurred in 12 patients, resulting in treatmentrelated mortality (TRM) of 9.0% (95%CI: 4.6-15.3). The 5-year overall (OS) and event-free survival were 73.6% (95%CI: 65.7-82.4) and 66.4% (95%CI: 58.2-75.8), respectively. Four independent adverse prognostic factors for OS were identified: age at diagnosis >2 years, time from diagnosis to HSCT >6 months, monocyte count at diagnosis >7.2x109/L, and the presence of additional genetic alterations. Based on these factors, we proposed a predictive classifier. Patients with three or more predictors (21% of the cohort) had a 5-year OS of 34.2%, whereas those with none (7%) had a 5-year OS of 100%. Our study demonstrates improved transplant outcomes compared to prior published data, which can be attributed to the synergistic impacts of a low TRM and a reduced yet still substantial relapse incidence. By integrating genetic information with clinical and hematological features, we have devised a predictive classifier. This classifier effectively identifies a subgroup of patients who are at a heightened risk of unfavorable post-transplant outcomes who would benefit novel therapeutic agents and post-transplant strategies.

Published
2024
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17. Educational outcomes in siblings of childhood leukemia survivors: Factors associated with school difficulties and comparison with general population

Author

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

Subjects
epidemiology and prevention, pediatric cancer, psychosocial studies, survival, Neoplasms. Tumors. Oncology. Including cancer and carcinogens, RC254-282
Abstract

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.

Published
2024
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18. A non-symmetric divide-and-conquer recursive formula for the convolution of polynomials and power series

Author

Baruchel, Thomas

Subjects
Mathematics - Number Theory
Abstract

Some changes in a recent convolution formula are performed here in order to clean it up by using more conventional notations and by making use of more referrenced and documented components (namely Sierpi\'nski's polynomials, the Thue-Morse sequence, the binomial modulo~2 transform and its inverse). Several variants are published here, by reading afterwards summed coefficients in another order; the last formula is then turned back from a summation to a new divide-and-conquer recursive formula.

Published
2019

20. Brothers and sisters of childhood acute leukemia survivors: Their long‐term quality of life and its determinants

Author

Cindy Faust, Pascal Auquier, Zeinab Hamidou, Yves Bertrand, Marie‐Dominique Tabone, Sophie Ansoborlo, André Baruchel, Virginie Gandemer, Jean‐Hugues Dalle, Pascal Chastagner, Justyna Kanold, Maryline Poirée, Nicolas Sirvent, Geneviève Plat, Isabelle Pellier, Gérard Michel, and Julie Berbis

Subjects
childhood leukemia, long‐term follow‐up, quality of life, siblings, survivors, Neoplasms. Tumors. Oncology. Including cancer and carcinogens, RC254-282
Abstract

Abstract Background Childhood cancer confront the whole family with a traumatic event. Because brothers and sisters may encounter emotional problems that can remain for a long time and that only few studies have assessed their long‐term outcome, our present objectives were to describe the long‐term quality of life (QoL) of childhood leukemia survivors' siblings and to explore its determinant. Methods Brothers and sisters (from 8‐year‐old) of survivors included in the French LEA Cohort completed a QoL questionnaire (according to their age). Scores were compared with those reported by age‐ and gender‐matched French general population and by survivors. Using a clustering method, siblings were categorized into 3 groups depending on their level of QoL's scores and factors likely to be linked with these clusters were explored with multivariate analyses. Results We included 689 brothers and sisters (313 minors, 376 adults) and the mean time from diagnosis was 13.2 ± 6.6 years. Minor siblings reported higher QoL scores than general population (p

Published
2023
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23. Properties of the cumulated deficient binary digit sum

Author

Baruchel, Thomas

Subjects
Mathematics - Number Theory
Abstract

The sequence A268289 from the On-Line Encyclopedia of Integer Sequences, namely the cumulated differences between the number of digits 1 and the number of digits 0 in the binary expansion of consecutive integers, is studied here. This sequence happens to match a sequence of cardinalities of some specific sets. Furthermore, it can also be expressed by using the Takagi function. The three different definitions have their own properties and combining them together lead to some new identities.

Published
2019

24. Flattening Karatsuba's recursion tree into a single summation

Author

Baruchel, Thomas

Subjects
Mathematics - Number Theory, Computer Science - Discrete Mathematics
Abstract

The recursion tree resulting from Karatsuba's formula is built here by using an interleaved splitting scheme rather than the traditional left/right one. This allows an easier access to the nodes of the tree and $2n-1$ of them are initially flattened all at once into a single recursive formula. The whole tree is then flattened further into a convolution formula involving less elementary multiplications than the usual Cauchy product. Unlike the traditional splitting scheme, the interleaved approach may also be applied to infinite power series, and corresponding formulas are also given.

Published
2019
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25. Harnessing the MYB-dependent TAL1 5’super-enhancer for targeted therapy in T-ALL

Author

Charlotte Smith, Aurore Touzart, Mathieu Simonin, Christine Tran-Quang, Guillaume Hypolite, Mehdi Latiri, Guillaume P. Andrieu, Estelle Balducci, Marie-Émilie Dourthe, Ashish Goyal, Françoise Huguet, Arnaud Petit, Norbert Ifrah, André Baruchel, Hervé Dombret, Elizabeth Macintyre, Christoph Plass, Jacques Ghysdael, Nicolas Boissel, and Vahid Asnafi

Subjects
Super-enhancer, Oncogene, Targeted therapy, Cancer, Neoplasms. Tumors. Oncology. Including cancer and carcinogens, RC254-282
Abstract

Abstract The acquisition of genetic abnormalities engendering oncogene dysregulation underpins cancer development. Certain proto-oncogenes possess several dysregulation mechanisms, yet how each mechanism impacts clinical outcome is unclear. Using T-cell acute lymphoblastic leukemia (T-ALL) as an example, we show that patients harboring 5’super-enhancer (5’SE) mutations of the TAL1 oncogene identifies a specific patient subgroup with poor prognosis irrespective of the level of oncogene dysregulation. Remarkably, the MYB dependent oncogenic 5’SE can be targeted using Mebendazole to induce MYB protein degradation and T-ALL cell death. Of note Mebendazole treatment demonstrated efficacy in vivo in T-ALL preclinical models. Our work provides proof of concept that within a specific oncogene driven cancer, the mechanism of oncogene dysregulation rather than the oncogene itself can identify clinically distinct patient subgroups and pave the way for future super-enhancer targeting therapy.

Published
2023
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28. Chimeric Antigen Receptor (CAR) T-Cell Products for Pediatric Cancers: Why Alternative Development Paths Are Needed

Author

Rossig, C, Pearson, A, Vassal, G, Scobie, N, Bird, N, Blanc, P, Vormoor, H, Calkoen, F, Locatelli, F, del Bufalo, F, Rives, S, Jacoby, E, Balduzzi, A, Bourquin, J, Baruchel, A, Rossig C., Pearson A. D., Vassal G., Scobie N., Bird N., Blanc P., Vormoor H. J., Calkoen F. G., Locatelli F., del Bufalo F., Rives S., Jacoby E., Balduzzi A., Bourquin J. P., Baruchel A., Rossig, C, Pearson, A, Vassal, G, Scobie, N, Bird, N, Blanc, P, Vormoor, H, Calkoen, F, Locatelli, F, del Bufalo, F, Rives, S, Jacoby, E, Balduzzi, A, Bourquin, J, Baruchel, A, Rossig C., Pearson A. D., Vassal G., Scobie N., Bird N., Blanc P., Vormoor H. J., Calkoen F. G., Locatelli F., del Bufalo F., Rives S., Jacoby E., Balduzzi A., Bourquin J. P., and Baruchel A.

Published
2024

31. P321: TNF-MEDIATED CELL DEATH: AN ACTIONABLE TARGET FOR IMMUNOTHERAPY IN T-CELL ACUTE LYMPHOBLASTIC LEUKEMIA

Author

Marie-Emilie Dourthe, Etienne Lengline, Guillaume Charbonnier, Mehdi Latiri, Chaimae Saji, Agata Cieslak, Mathieu Simonin, Norbert Ifrah, Hervé Dombret, Olivier Hermine, Nicolas Boissel, André Baruchel, Jacques Ghysdael, Elizabeth Macintyre, Christine Tran-Quang, Guillaume Andrieu, and Vahid Asnafi

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Published
2023
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32. P501: ISATUXIMAB PLUS CHEMOTHERAPY FOR PEDIATRIC RELAPSED/REFRACTORY ACUTE LYMPHOBLASTIC LEUKEMIA OR ACUTE MYELOID LEUKEMIA (ISAKIDS): INTERIM EFFICACY ANALYSIS

Author

André Baruchel, Yves Bertrand, Karsten Nysom, Hyoung Jin Kang, Monica Makiya, Jonas Abrahamsson, Oscar González-Llano, Willy Quinones Choque, Carmelo Rizzari, Jochen Buechner, Simone Cesaro, Ximo Duarte, Franca Fagioli, Antonis Kattamis, Guy Leverger, Brigitte Nelken, Lynn Wang, Sandrine Mace, Corina Oprea, Giovanni Abbadessa, and Michel Zwaan

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Published
2023
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33. P1408: UPDATED RESULTS OF THE PHASE I BALLI-01 TRIAL OF UCART22, AN ANTI-CD22 ALLOGENEIC CAR-T CELL PRODUCT, IN PATIENTS WITH RELAPSED OR REFRACTORY (R/R) CD22+ B-CELL ACUTE LYMPHOBLASTIC LEUKEMIA (B-ALL)

Author

Nicolas Boissel, Patrice Chevallier, Kevin Curran, Gary Schiller, Hongtau Liu, Richard Larson, Daniel J. Deangelo, Jean-Baptiste Mear, Stephan Grupp, André Baruchel, Marina Konopleva, Alexandra Lacroce, Caroline Roudet, Ana Korngold, Katie Newhall, Eric Laille, Daniel Lee, Mark Frattini, and Nitin Jain

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Published
2023
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34. Down syndrome and leukemia: from basic mechanisms to clinical advances

Author

André Baruchel, Jean-Pierre Bourquin, John Crispino, Sergi Cuartero, Henrik Hasle, Johann Hitzler, Jan-Henning Klusmann, Shai Izraeli, Andrew A. Lane, Sébastien Malinge, Karen R. Rabin, Irene Roberts, Sandra Ryeom, Sarah K. Tasian, and Elvin Wagenblast

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Abstract

Children with Down syndrome (DS, trisomy 21) are at a significantly higher risk of developing acute leukemia compared to the overall population. Many studies investigating the link between trisomy 21 and leukemia initiation and progression have been conducted over the last two decades. Despite improved treatment regimens and significant progress in iden - tifying genes on chromosome 21 and the mechanisms by which they drive leukemogenesis, there is still much that is unknown. A focused group of scientists and clinicians with expertise in leukemia and DS met in October 2022 at the Jérôme Lejeune Foundation in Paris, France for the 1st International Symposium on Down Syndrome and Leukemia. This meeting was held to discuss the most recent advances in treatment regimens and the biology underlying the initiation, progression, and relapse of acute lymphoblastic leukemia and acute myeloid leukemia in children with DS. This review provides a summary of what is known in the field, challenges in the management of DS patients with leukemia, and key questions in the field.

Published
2023
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36. CD19 CAR T-cells for pediatric relapsed acute lymphoblastic leukemia with active CNS involvement: a retrospective international study

Author

Jacoby, Elad, Ghorashian, Sara, Vormoor, Britta, De Moerloose, Barbara, Bodmer, Nicole, Molostova, Olga, Yanir, Asaf D, Buechner, Jochen, Elhasid, Ronit, Bielorai, Bella, Rogosic, Srdan, Dourthe, Marie-Emilie, Maschan, Michael, Rossig, Claudia, Toren, Amos, von Stackelberg, Arend, Locatelli, Franco, Bader, Peter, Zimmermann, Martin, Bourquin, Jean Pierre, and Baruchel, Andre

Published
2022
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37. The use of ICU resources in CAR-T cell recipients: a hospital-wide study

Author

Sandrine Valade, Michael Darmon, Lara Zafrani, Eric Mariotte, Virginie Lemiale, Swann Bredin, Guillaume Dumas, Nicolas Boissel, Florence Rabian, André Baruchel, Isabelle Madelaine, Jérôme Larghero, Anne Brignier, Etienne Lengliné, Stéphanie Harel, Bertrand Arnulf, Roberta Di Blasi, Catherine Thieblemont, and Elie Azoulay

Subjects
Anti-CD19 chimeric antigen receptor, Intensive care, Hematological malignancies, Sepsis, Performance status, Medical emergencies. Critical care. Intensive care. First aid, RC86-88.9
Abstract

Abstract Background CAR-T cell (chimeric antigen receptor T) therapy has emerged as an effective treatment of refractory hematological malignancies. Intensive care management is intrinsic to CAR-T cell therapy. We aim to describe and to assess outcomes in critically ill CAR-T cell recipients. Study design and methods Hospital-wide retrospective study. Consecutive CAR-T cell recipients requiring ICU admission from July 2017 and December 2020 were included. Results 71 patients (median age 60 years [37–68]) were admitted to the ICU 6 days [4–7] after CAR-T cell infusion. Underlying malignancies included diffuse large B cell lymphoma (n = 53, 75%), acute lymphoblastic leukemia (17 patients, 24%) and multiple myeloma (n = 1, 1.45%). Performance status (PS) was 1 [1–2]. Shock was the main reason for ICU admission (n = 40, 48%). Isolated cytokine release syndrome (CRS) was the most common complication (n = 33, 46%), while 21 patients (30%) had microbiologically documented bacterial infection (chiefly catheter-related infection). Immune effector cell-associated neurotoxicity syndrome was reported in 26 (37%) patients. At ICU admission, vasopressors were required in 18 patients (25%) and invasive mechanical ventilation in two. Overall, 49 (69%) and 40 patients (56%) received tocilizumab or steroids, respectively. Determinant of mortality were the reason for ICU admission (disease progression vs. sepsis or CRS (HR 4.02 [95%CI 1.10–14.65]), Performance status (HR 1.97/point [95%CI 1.14–3.41]) and SOFA score (HR 1.16/point [95%CI 1.01–1.33]). Conclusions Meaningful survival could be achieved in up to half the CAR-T cell recipients. The severity of organ dysfunction is a major determinant of death, especially in patients with altered performance status or disease progression.

Published
2022
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38. Tisagenlecleucel therapy for relapsed or refractory B-cell acute lymphoblastic leukaemia in infants and children younger than 3 years of age at screening: an international, multicentre, retrospective cohort study

Author

Ghorashian, Sara, Jacoby, Elad, De Moerloose, Barbara, Rives, Susana, Bonney, Denise, Shenton, Geoff, Bader, Peter, Bodmer, Nicole, Quintana, Agueda Molinos, Herrero, Blanca, Algeri, Mattia, Locatelli, Franco, Vettenranta, Kim, Gonzalez, Berta, Attarbaschi, Andishe, Harris, Stephen, Bourquin, Jean Pierre, and Baruchel, André

Published
2022
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42. The use of ICU resources in CAR-T cell recipients: a hospital-wide study

Author

Valade, Sandrine, Darmon, Michael, Zafrani, Lara, Mariotte, Eric, Lemiale, Virginie, Bredin, Swann, Dumas, Guillaume, Boissel, Nicolas, Rabian, Florence, Baruchel, André, Madelaine, Isabelle, Larghero, Jérôme, Brignier, Anne, Lengliné, Etienne, Harel, Stéphanie, Arnulf, Bertrand, Di Blasi, Roberta, Thieblemont, Catherine, and Azoulay, Elie

Published
2022
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43. Some conjectural continued fractions

Author

Baruchel, Thomas

Subjects
Mathematics - Number Theory
Abstract

Several conjectural continued fractions found with the help of various algorithms are published in this paper.

Published
2017

44. Outcomes of infants with very late relapse of acute lymphoblastic leukaemia initially treated in Interfant‐06.

Author

Lecornec, Nicolas, Fahd, Mony, De Lorenzo, Paola, Valsecchi, Maria Grazia, Micalizzi, Concetta, Diaz, Paulina, Pennella, Carla, Locatelli, Franco, Vinti, Luciana, Cavé, Hélène, Caye‐Eude, Aurélie, Stary, Jan, Pieters, Rob, Baruchel, André, and Brethon, Benoit

Subjects
HEMATOPOIETIC stem cell transplantation, TOTAL body irradiation, LEUCOCYTES, STEM cell transplantation, ADVERSE health care events, ADENOVIRUS diseases, BLAST injuries
Abstract

The article discusses the outcomes of infants with very late relapse of acute lymphoblastic leukaemia initially treated in Interfant-06. It highlights the characteristics and outcomes of six children who experienced a very late relapse of infant ALL, providing detailed information on their initial diagnosis, relapse, treatment, and follow-up. The study shows that all patients achieved a second complete remission after a lymphoid-style salvage regimen, with five patients receiving allogeneic hematopoietic stem cell transplantation and experiencing promising long-term results. The findings suggest that infants with very late relapses may have a more favorable outcome compared to those who relapse within the first year after diagnosis, emphasizing the importance of effective first-line therapies in reducing relapse rates. [Extracted from the article]

Published
2024
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45. EBV-driven lymphoid neoplasms associated with pediatric ALL maintenance therapy

Author

Elitzur, Sarah, Vora, Ajay, Burkhardt, Birgit, Inaba, Hiroto, Attarbaschi, Andishe, Baruchel, Andre, Escherich, Gabriele, Gibson, Brenda, Liu, Hsi-Che, Loh, Mignon, Moorman, Anthony V., Moricke, Anja, Pieters, Rob, Uyttebroeck, Anne, Baird, Susan, Bartram, Jack, Barzilai-Birenboim, Shlomit, Batra, Sandeep, Ben-Harosh, Miriam, Bertrand, Yves, Buitenkamp, Trudy, Caldwell, Kenneth, Drut, Ricardo, Geerlinks, Ashley V., Gilad, Gil, Grainger, John, Haouy, Stephanie, Heaney, Nicholas, Huang, Mary, Ingham, Danielle, Krenova, Zdenka, Kuhlen, Michaela, Lehrnbecher, Thomas, Manabe, Atsushi, Niggli, Felix, Paris, Claudia, Revel-Vilk, Shoshana, Rohrlich, Pierre, Sinno, Mohamad G., Szczepanski, Tomasz, Tamesberger, Melanie, Warrier, Rajasekharan, Wolfl, Matthias, Nirel, Ronit, Izraeli, Shai, Borkhardt, Arndt, and Schmiegelow, Kjeld

Published
2022
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47. Clinico-biological features of T-cell acute lymphoblastic leukemia with fusion proteins

Author

Thomas Steimlé, Marie-Emilie Dourthe, Marion Alcantara, Aurore Touzart, Mathieu Simonin, Johanna Mondesir, Ludovic Lhermitte, Jonathan Bond, Carlos Graux, Nathalie Grardel, Jean-Michel Cayuela, Isabelle Arnoux, Virginie Gandemer, Marie Balsat, Norbert Vey, Elizabeth Macintyre, Norbert Ifrah, Hervé Dombret, Arnaud Petit, André Baruchel, Philippe Ruminy, Nicolas Boissel, and Vahid Asnafi

Subjects
Neoplasms. Tumors. Oncology. Including cancer and carcinogens, RC254-282
Abstract

Abstract T-cell acute lymphoblastic leukemias (T-ALL) represent 15% of pediatric and 25% of adult ALL. Since they have a particularly poor outcome in relapsed/refractory cases, identifying prognosis factors at diagnosis is crucial to adapting treatment for high-risk patients. Unlike acute myeloid leukemia and BCP ALL, chromosomal rearrangements leading to chimeric fusion-proteins with strong prognosis impact are sparsely reported in T-ALL. To address this issue an RT-MPLA assay was applied to a consecutive series of 522 adult and pediatric T-ALLs and identified a fusion transcript in 20% of cases. PICALM-MLLT10 (4%, n = 23), NUP214-ABL1 (3%, n = 19) and SET-NUP214 (3%, n = 18) were the most frequent. The clinico-biological characteristics linked to fusion transcripts in a subset of 235 patients (138 adults in the GRAALL2003/05 trials and 97 children from the FRALLE2000 trial) were analyzed to identify their prognosis impact. Patients with HOXA trans-deregulated T-ALLs with MLLT10, KMT2A and SET fusion transcripts (17%, 39/235) had a worse prognosis with a 5-year EFS of 35.7% vs 63.7% (HR = 1.63; p = 0.04) and a trend for a higher cumulative incidence of relapse (5-year CIR = 45.7% vs 25.2%, HR = 1.6; p = 0.11). Fusion transcripts status in T-ALL can be robustly identified by RT-MLPA, facilitating risk adapted treatment strategies for high-risk patients.

Published
2022
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48. Determinants of CD19-positive vs CD19-negative relapse after tisagenlecleucel for B-cell acute lymphoblastic leukemia

Author

Dourthe, Marie-Emilie, Rabian, Florence, Yakouben, Karima, Chevillon, Florian, Cabannes-Hamy, Aurélie, Méchinaud, Françoise, Grain, Audrey, Chaillou, Delphine, Rahal, Ilhem, Caillat-Zucman, Sophie, Lesprit, Emmanuelle, Naudin, Jérôme, Roupret-Serzec, Julie, Parquet, Nathalie, Brignier, Anne, Guérin-El Khourouj, Valérie, Lainey, Elodie, Caye-Eude, Aurélie, Cavé, Hélène, Clappier, Emmanuelle, Mathis, Stéphanie, Azoulay, Elie, Dalle, Jean Hugues, Dhédin, Nathalie, Madelaine, Isabelle, Larghero, Jérôme, Boissel, Nicolas, and Baruchel, André

Published
2021
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49. Translating regenerative medicine therapies in neonatal necrotizing enterocolitis

Author

Ganji, Niloofar, primary, Kalish, Brian, additional, Offringa, Martin, additional, Li, Bo, additional, Anderson, James, additional, Baruchel, Sylvain, additional, Blakely, Martin, additional, De Coppi, Paolo, additional, Eaton, Simon, additional, Gauda, Estelle, additional, Hall, Nigel, additional, Heath, Anna, additional, Livingston, Michael H., additional, McNair, Carol, additional, Mitchell, Robert, additional, Patel, Ketan, additional, Pechlivanoglou, Petros, additional, Pleasants-Terashita, Hazel, additional, Pryor, Erin, additional, Radisic, Milica, additional, Shah, Prakesh S., additional, Thébaud, Bernard, additional, Wang, Kasper, additional, Zani, Augusto, additional, and Pierro, Agostino, additional

Published
2024
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50. Success of donor‐derived CAR‐T cells after failure of autologous CD19 CAR‐T cells (tisagenlecleucel) in B‐cell acute lymphoblastic leukaemia

Author

Dourthe, Marie Emilie, primary, Yakouben, Karima, additional, David, Audrey, additional, Thouvenin, Sandrine, additional, Chaillou, Delphine, additional, Caillat‐Zucman, Sophie, additional, Cuffel, Alexis, additional, Tardy, Cléa, additional, Lainey, Elodie, additional, Caye‐Eude, Aurélie, additional, Arfeuille, Chloé, additional, Delaugerre, Constance, additional, Chaix‐Baudier, Marie‐Laure, additional, Naudin, Jérôme, additional, Auvin, Stéphane, additional, Bergaoui, Karim, additional, Merlat‐Guitard, Anne‐Isabelle, additional, De Jorna, Romain, additional, Mebarki, Miryam, additional, Dalle, Jean‐Hugues, additional, and Baruchel, André, additional

Published
2024
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