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101. 162 A phase II study of decitabine in advanced chronic myelomonocytic leukemia (CMML)

Author

Braun, T., primary, Droin, N., additional, Itzykson, R., additional, de Renzis, B., additional, Dreyfus, F., additional, Laribi, K., additional, Boabdallah, K., additional, Charbonnier, A., additional, Cordonnier, C., additional, Lafon, I., additional, Recher, C., additional, Vey, N., additional, Bastie, J.-N., additional, Besançon, A., additional, Beyne-Rauzy, O., additional, Joly, B., additional, Jourdan, E., additional, Legros, L., additional, Marjanovic, Z., additional, Petitdidier, C., additional, Royer, B., additional, Sanhes, L., additional, Tertian, G., additional, Vaida, I., additional, Vallantin, X., additional, Vekhoff, A., additional, Milic, M., additional, Gardin, C., additional, Ades, L., additional, Fenaux, P., additional, and Solary, E., additional

Published
2011
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102. 124 AZACITIDINE VERSUS CONVENTIONAL CARE REGIMENS (CCR) IN OLDER PATIENTS WITH NEWLY DIAGNOSED AML (>30% MARROW BLASTS) WITH MYELODYSPLASIA-RELATED CHANGES: SUBGROUP ANALYSIS OF THE AZA-AML-001 STUDY

Author

Seymour, J.F., Döhner, H., Butrym, A., Wierzbowska, A., Selleslag, D., Jang, J.H., Cavenagh, J.D., Kumar, R., Schuh, A.C., Candoni, A., Récher, C., Sandhu, I., del Castillo, T. Bernal, Al-Ali, H.K., Martinelli, G., Falantes, J., Nopenney, R., Stone, R.M., Minden, M.D., McIntyre, H., Songer, S., Lucy, L.M., Beach, C.L., and Dombret, H.

Published
2015
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103. 96 OVERALL SURVIVAL IN OLDER PATIENTS WITH NEWLY DIAGNOSED AML WITH >30% BONE MARROW BLASTS AND POOR-RISK CYTOGENETICS TREATED WITH AZACITIDINE: SUBANALYSIS OF THE AZA-AML-001 STUDY

Author

Döhner, H., Seymour, J.F., Butrym, A., Wierzbowska, A., Selleslag, D., Jang, J.H., Cavenagh, J.D., Kumar, R., Schuh, A.C., Candoni, A., Récher, C., Sandhu, I., del Castillo, T. Bernal, Al-Ali, H.K., Martinelli, G., Falantes, J., Nopenney, R., Stone, R.M., Minden, M.D., McIntyre, H., Songer, S., Lucy, L.M., Beach, C.L., and Dombret, H.

Published
2015
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106. R114: Leukemic stem cells are rare and heterogeneous in human acute myelogenous leukemia

Author

Sarry, J.E., primary, Murphy, K., additional, Perry, R., additional, Sanchez, P.V., additional, Secreto, A., additional, Keefer, C., additional, Swider, C.R., additional, Strzelecki, A.C., additional, Cavelier, C., additional, Recher, C., additional, Mansat-De Mas, V., additional, Delabesse, É., additional, Danet-Desnoyers, G., additional, and Carroll, M., additional

Published
2010
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110. Rituximab compared to observation after high-dose consolidative first-line chemotherapy (HDC) with autologous stem cell transplantation in poor-risk diffuse large B-cell lymphoma: Updated results of the LNH98-B3 GELA study

Author

Haioun, C., primary, Mounier, N., additional, Emile, J. F., additional, Bologna, S., additional, Coiffier, B., additional, Tilly, H., additional, Recher, C., additional, Fermé, C., additional, Morschhauser, F., additional, and Gisselbrecht, C., additional

Published
2007
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112. No Benefit of Adding High-Dose Melphalan (HDM) Suppported by Autologous Stem Cell Transplantation (SCT) over Treatment with Conventional Induction, HD ARAC Consolidation, and Auto SCT (Busulfan + HDM) for Acute Myelogenous Leukemia (AML) under 60: First Results of the Randomized AML 2001 Trial.

Author

Lioure, B., primary, Pigneux, A., primary, Recher, C., primary, Witz, F., primary, Lamy, T., primary, Fegueux, N., primary, Bulabois, C.E., primary, Ifrah, N., primary, Luquet, I., primary, Bouscary, D., primary, Vey, N., primary, Delain, M., primary, Bay, J.O., primary, Turlure, P., primary, and Harousseau, Jean-Luc, primary

Published
2006
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113. Safety of Prophylactic Use of Darbepoetin Alfa in Patients with Diffuse Large B-Cell Lymphoma (DLBCL) Treated with R-CHOP 14 or R-CHOP21: Preliminary Results of the LNH03-6B Randomized GELA Study.

Author

Delarue, R., primary, Mounier, N., additional, Haioun, C., additional, Coiffier, B., additional, Gisselbrecht, C., additional, Ghesquieres, H., additional, Lederlin, P., additional, Blanc, M., additional, Recher, C., additional, Hermine, O., additional, Reyes, F., additional, Tilly, H., additional, and Bosly, A., additional

Published
2006
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114. Extramedullary relapse in acute promyelocytic leukemia treated with all-trans retinoic acid and chemotherapy

Author

de Botton, S, primary, Sanz, M A, additional, Chevret, S, additional, Dombret, H, additional, Martin, G, additional, Thomas, X, additional, Mediavilla, J D, additional, Recher, C, additional, Ades, L, additional, Quesnel, B, additional, Brault, P, additional, Fey, M, additional, Wandt, H, additional, Machover, D, additional, Guerci, A, additional, Maloisel, F, additional, Stoppa, A M, additional, Rayon, C, additional, Ribera, J M, additional, Chomienne, C, additional, Degos, L, additional, and Fenaux, P, additional

Published
2005
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115. Autologous Stem Cell Transplantation (ASCT) Versus Oral Melphalan and High-Dose Dexamethasone in Patients with AL (Primary) Amyloidosis: Results of the French Multicentric Randomized Trial (MAG and IFM Intergroup).

Author

Jaccard, A., primary, Moreau, P., primary, Leblond, V., primary, Leleu, X., primary, Benboubker, L., primary, Hermine, O., primary, Recher, C., primary, Malphette, M., primary, Lioure, B., primary, Royer, B., primary, Grosbois, B., primary, Jaubert, J., primary, Preux, P.M., primary, Cogne, M., primary, and Fermand, J.P., primary

Published
2005
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116. Early allogeneic stem cell transplantation for young adults with acute myeloblastic leukemia in first complete remission: An intent-to-treat analysis of the long-term experience of the BGMT group

Author

Jourdan, E., primary, Boiron, J.M., additional, Dastugue, N., additional, Vey, N., additional, Marit, G., additional, Rigal Huguet, F., additional, Molina, L., additional, Fegueux, N., additional, Pigneux, A., additional, Recher, C., additional, Rossi, J.F., additional, Attal, M., additional, Sotto, J.J., additional, Maraninchi, D., additional, Reiffers, J., additional, Bardou, V.J., additional, and Blaise, D., additional

Published
2005
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124. High-dose imatinib mesylate combined with vincristine and dexamethasone (DIV regimen) as induction therapy in patients with resistant Philadelphia-positive acute lymphoblastic leukemia and lymphoid blast crisis of chronic myeloid leukemia.

Author

D. Rea, Legros, L., Raffoux, E., Thomas, X., Turlure, P., Maury, S., Dupriez, B., Pigneux, A., Choufi, B., Reman, O., Stéphane, D., Royer, B., Vigier, M., Ojeda-Uribe, M., Recher, C., Dombret, H., Huguet, F., and Rousselot, P.

Subjects
ANTINEOPLASTIC agents, IMATINIB, VINCRISTINE, COMBINATION drug therapy, LYMPHOBLASTIC leukemia treatment, ACUTE leukemia
Abstract

Imatinib combined with high-dose chemotherapy is now becoming the gold standard for treatment of Philadelphia chromosome-positive acute leukemias. However, in all studies imatinib dosage was tapered to 400–600 mg per day. We decided to initiate a clinical trial to evaluate an opposite strategy based on high-dose imatinib (800 mg per day) combined with a less intensive chemotherapeutic regimen (vincristine and dexamethasone), which we called the DIV induction regimen. Thirty-one patients (18 relapsing or refractory Ph+ acute lymphoblastic leukemias and 13 lymphoid blast crisis chronic myelogenous leukemias) were enrolled. Complete remission (CR) was obtained in 28 out of 30 assessable patients. The median bcr-abl/abl ratio after the induction course was 0.1%. Median time to neutrophil recovery was 21 days. Fungus infections were observed in six patients out of 31 and possibly related to dexamethasone. Neuropathy due to vincristine was noted in 14 cases. Nine out of 19 patients under 55 years received allogenic stem cell transplantation after a median time of 78 days post-CR. Patients older than 55 years experienced a 90% CR rate without additional toxicities, suggesting the DIV regimen may also be proposed as a front line therapy in older patients.Leukemia (2006) 20, 400–403. doi:10.1038/sj.leu.2404115; published online 26 January 2006 [ABSTRACT FROM AUTHOR]

Published
2006
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136. IMPROVED SURVIVAL IN PATIENTS >= 60 WITH FIRST RELAPSED/REFRACTORY ACUTE MYELOID LEUKEMIA TREATED WITH VOSAROXIN PLUS CYTARABINE VS PLACEBO PLUS CYTARABINE: RESULTS FROM THE PHASE 3 VALOR STUDY

Author

Carella, A. -M, Ravandi, F., Ritchie, E. K., Sayar, H., Lancet, J. E., Craig, M. D., Norbert Vey, Strickland, S. A., Schiller, G. J., Jabbour, E., Erba, H. P., Pigneux, A., Horst, H. -A, Recher, C., Klimek, V. M., Cortes, J., Roboz, G. J., Craig, A. R., Fox, J. A., Ward, R., Smith, J. A., Acton, G., Mehta, C., Kantarjian, H. M., and Stuart, R. K.

139. Rituximab given after high dose therapy and autologous stem cell transplantation induces durable clearance of minimal residual disease in about half of the patients with follicular non Hodgkin's lymphoma: Interim 24 months results of a multicenter open label phase II trial (M39012 trial)

Author

Morschhauser, F., Recher, C., Galoin, S., Milpied, N., Gressin, R., Gilles Salles, Brice, P., Stoppa, Am, Haioun, C., Colombat, P., Bourhis, Jh, Rossi, Jf, Decominck, D., Bergougnoux, L., Berriot-Varoqueaux, N., Delsol, G., and Attal, M.

140. OVERALL SURVIVAL IN OLDER PATIENTS WITH NEWLY DIAGNOSED AML WITH > 30% BONE MARROW BLASTS AND POOR-RISK CYTOGENETICS TREATED WITH AZACITIDINE: SUBANALYSIS OF THE AZA-AML-001 STUDY

Author

Doehner, H., Seymour, J. F., Butrym, A., Wierzbowska, A., Selleslag, D., Jang, J. H., Cavenagh, J. D., Kumar, R., Schuh, A. C., anna candoni, Recher, C., Sandhu, I., Bernal Del Castillo, T., Al-Ali, H. K., Martinelli, G., Falantes, J., Nopenney, R., Stone, R. M., Minden, M. D., Mcintyre, H., Songer, S., Lucy, L. M., Beach, C. L., and Dombret, H.

145. Eltrombopag Added to Immunosuppression in Severe Aplastic Anemia.

Author

de Latour, R. Peffault, Kulasekararaj, A., Iacobelli, S., Terwel, S. R., Cook, R., Griffin, M., Halkes, C. J. M., Recher, C., Barraco, F., Forcade, E., Vallejo, J.-C., Drexler, B., Mear, J.-B., Smith, A. E., Angelucci, E., Raymakers, R. A. P., de Groot, M. R., Daguindau, E., Nur, E., and Barcellini, W.

Abstract

BACKGROUND: A single-group, phase 1-2 study indicated that eltrombopag improved the efficacy of standard immunosuppressive therapy that entailed horse antithymocyte globulin (ATG) plus cyclosporine in patients with severe aplastic anemia. METHODS: In this prospective, investigator-led, open-label, multicenter, randomized, phase 3 trial, we compared the efficacy and safety of horse ATG plus cyclosporine with or without eltrombopag as front-line therapy in previously untreated patients with severe aplastic anemia. The primary end point was a hematologic complete response at 3 months. RESULTS: Patients were assigned to receive immunosuppressive therapy (Group A, 101 patients) or immunosuppressive therapy plus eltrombopag (Group B, 96 patients). The percentage of patients who had a complete response at 3 months was 10% in Group A and 22% in Group B (odds ratio, 3.2; 95% confidence interval [CI], 1.3 to 7.8; P = 0.01). At 6 months, the overall response rate (the percentage of patients who had a complete or partial response) was 41% in Group A and 68% in Group B. The median times to the first response were 8.8 months (Group A) and 3.0 months (Group B). The incidence of severe adverse events was similar in the two groups. With a median follow-up of 24 months, a karyotypic abnormality that was classified as myelodysplastic syndrome developed in 1 patient (Group A) and 2 patients (Group B); event-free survival was 34% and 46%, respectively. Somatic mutations were detected in 29% (Group A) and 31% (Group B) of the patients at baseline; these percentages increased to 66% and 55%, respectively, at 6 months, without affecting the hematologic response and 2-year outcome. CONCLUSIONS: The addition of eltrombopag to standard immunosuppressive therapy improved the rate, rapidity, and strength of hematologic response among previously untreated patients with severe aplastic anemia, without additional toxic effects. (Funded by Novartis and others; RACE ClinicalTrials.gov number, NCT02099747; EudraCT number, 2014-000363-40.). [ABSTRACT FROM AUTHOR]

Published
2022
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146. Early death in acute promyelocytic leukemia (APL) in French centers: a multicenter study in 399 patients.

Author

Rahmé, R, Thomas, X, Recher, C, Vey, N, Delaunay, J, Deconinck, E, Hirsch, P, Bordessoule, D, Micol, J-B, Stamatoullas, A, Mariette, C, Pautas, C, Bories, P, Marolleau, J-P, Hunault-Berger, M, Fegueux, N, Raffoux, E, Dombret, H, Degos, L, and Fenaux, P

Subjects
*EARLY death, *CAUSES of death, *ACUTE promyelocytic leukemia, *RETINOIC acid receptors, *HOSPITAL admission & discharge
Abstract

The article analyzes the incidence and causes of early death (ED) in acute promyelocytic leukemia (APL) in French centers from December 2006 to December 2011. APL diagnosis is confirmed by the presence of t(15;17) translocation and/or promyelocytic leukemia gene-retinoic acid recepter alpha gene rearrangement. ED is caused by differentiation syndrome, myocardial infarction and multiple organ failure. The rapid admission of patients follows as all-trans retinoic acid is rapidly started.

Published
2014
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148. Gilteritinib or Chemotherapy for Relapsed or Refractory FLT3-Mutated AML.

Author

Perl, A. E., Martinelli, G., Cortes, J. E., Neubauer, A., Berman, E., Paolini, S., Montesinos, P., Baer, M. R., Larson, R. A., Ustun, C., Fabbiano, F., Erba, H. P., Di Stasi, A., Stuart, R., Olin, R., Kasner, M., Ciceri, F., Chou, W.-C., Podoltsev, N., and Recher, C.

Subjects
*FEBRILE neutropenia, *ACUTE myeloid leukemia, *PROTEIN-tyrosine kinases, *CANCER chemotherapy
Abstract

Background Patients with relapsed or refractory acute myeloid leukemia (AML) with mutations in the FMS-like tyrosine kinase 3 gene (FLT3) infrequently have a response to salvage chemotherapy. Gilteritinib is an oral, potent, selective FLT3 inhibitor with single-agent activity in relapsed or refractory FLT3-mutated AML. Methods In a phase 3 trial, we randomly assigned adults with relapsed or refractory FLT3-mutated AML in a 2:1 ratio to receive either gilteritinib (at a dose of 120 mg per day) or salvage chemotherapy. The two primary end points were overall survival and the percentage of patients who had complete remission with full or partial hematologic recovery. Secondary end points included event-free survival (freedom from treatment failure [i.e., relapse or lack of remission] or death) and the percentage of patients who had complete remission. Results Of 371 eligible patients, 247 were randomly assigned to the gilteritinib group and 124 to the salvage chemotherapy group. The median overall survival in the gilteritinib group was significantly longer than that in the chemotherapy group (9.3 months vs. 5.6 months; hazard ratio for death, 0.64; 95% confidence interval [CI], 0.49 to 0.83; P<0.001). The median event-free survival was 2.8 months in the gilteritinib group and 0.7 months in the chemotherapy group (hazard ratio for treatment failure or death, 0.79; 95% CI, 0.58 to 1.09). The percentage of patients who had complete remission with full or partial hematologic recovery was 34.0% in the gilteritinib group and 15.3% in the chemotherapy group (risk difference, 18.6 percentage points; 95% CI, 9.8 to 27.4); the percentages with complete remission were 21.1% and 10.5%, respectively (risk difference, 10.6 percentage points; 95% CI, 2.8 to 18.4). In an analysis that was adjusted for therapy duration, adverse events of grade 3 or higher and serious adverse events occurred less frequently in the gilteritinib group than in the chemotherapy group; the most common adverse events of grade 3 or higher in the gilteritinib group were febrile neutropenia (45.9%), anemia (40.7%), and thrombocytopenia (22.8%). Conclusions Gilteritinib resulted in significantly longer survival and higher percentages of patients with remission than salvage chemotherapy among patients with relapsed or refractory FLT3-mutated AML. (Funded by Astellas Pharma; ADMIRAL ClinicalTrials.gov number, NCT02421939. opens in new tab.) [ABSTRACT FROM AUTHOR]

Published
2019
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149. How should we diagnose and treat blastic plasmacytoid dendritic cell neoplasm patients?

Author

Xavier Cahu, Maxime Desmarets, Thibaut Leguay, Philippe Saas, Victoria Raggueneau, Delphine Binda, Maria Alessandra Rosenthal, Chrystelle Vidal, Olivier Adotevi, Fanny Angelot-Delettre, Françoise Solly, Anne-Cécile Galoisy, Alice Garnier, Sylvie Daliphard, Estelle Guérin, Marie-Pierre Gourin, Karim Maloum, Véronique Harrivel, Edouard Cornet, Felipe Suarez, Jacques Vargaftig, Fabrice Jardin, Caroline Mayeur-Rousse, Sylvain Thepot, Maïder Pagadoy, Thorsten Braun, Bernard Drenou, Yuriy Drebit, Marc Maynadié, Caroline Basle, Zehaira Benseddik, Frédéric Féger, Jean Feuillard, Christian Recher, Etienne Lengliné, Catherine Cordonnier, Rémi Letestu, Mathieu Puyade, Isabelle Arnoux, Remy Gressin, Nathalie Contentin, Jerome Tamburini, Pascale Saussoy, Mary Callanan, Elodie Dindinaud, Pierre-Simon Rohrlich, Julien Guy, Hind Bennani, Tony Petrella, Vincent Foissaud, Johann Rose, Natacha Maillard, Lucile Baseggio, Magali Le Garff-Tavernier, Vincent Barlogis, Denis Guyotat, Yohan Desbrosses, Caroline Bonmati, Damien Roos-Weil, Michel Ticchioni, Sandrine Puyraimond, Norbert Vey, Adriana Plesa, Blandine Guffroy, Daniel Lusina, Bérengère Gruson, Anne Roggy, Véronique Salaun, Eric Deconinck, Jean-Yves Cahn, Nathalie Jacques, Caroline Bret, Florian Renosi, Marie-Christine Béné, Alice Eischen, Stefan Wickenhauser, Benjamin Papoular, Francine Garnache-Ottou, François-Xavier Gros, Vahid Asnafi, Celia Salanoubat, Blandine Bénet, Elisabeth Macintyre, Lou Soret, Orianne Wagner-Ballon, Mohamad Mohty, Elsa Bera, Nicolas Freynet, Ludovic Lhermitte, Franck Trimoreau, Claude Preudhomme, Christophe Roumier, Sébastien Maury, Sabrina Bouyer, Eve Poret, Mikael Roussel, Romaric Lacroix, Christine Arnoulet, Françoise Schillinger, Patricia Okamba, Christine Lefebvre, Didier Blaise, Nicolas Lechevalier, Sabine Brechignac, Christophe Ferrand, Estelle Seilles, Richard Veyrat-Masson, Giorgia Battipaglia, Denis Caillot, Véronique Latger-Cannard, Bruno Quesnel, Didier Bouscary, Sophie Brun, Agathe Debliquis, Marie Loosveld, Franck Geneviève, Carinne Lafon, Lydia Campos, Thierry Fest, Ouda Ghoual, Marie-Christine Jacob, Pierre Peterlin, Valérie Bardet, Anne Arnaud, Véronique Dorvaux, Sabeha Biichle, Interactions hôte-greffon-tumeur, ingénierie cellulaire et génique - UFC (UMR INSERM 1098) (RIGHT), Institut National de la Santé et de la Recherche Médicale (INSERM)-Etablissement français du sang [Bourgogne-Franche-Comté] (EFS [Bourgogne-Franche-Comté])-Université de Franche-Comté (UFC), Université Bourgogne Franche-Comté [COMUE] (UBFC)-Université Bourgogne Franche-Comté [COMUE] (UBFC), Centre d'Investigation Clinique de Besançon (Inserm CIC 1431), Centre Hospitalier Régional Universitaire de Besançon (CHRU Besançon)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Etablissement français du sang [Bourgogne-Franche-Comté] (EFS [Bourgogne-Franche-Comté])-Université de Franche-Comté (UFC), Institut Necker Enfants-Malades (INEM - UM 111 (UMR 8253 / U1151)), Université Paris Descartes - Paris 5 (UPD5)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Centre de Recherche en Cancérologie et Immunologie Nantes-Angers (CRCINA), Université d'Angers (UA)-Université de Nantes (UN)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-Centre hospitalier universitaire de Nantes (CHU Nantes), Centre recherche en CardioVasculaire et Nutrition = Center for CardioVascular and Nutrition research (C2VN), Aix Marseille Université (AMU)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Institut National de Recherche pour l’Agriculture, l’Alimentation et l’Environnement (INRAE), Institut Paoli-Calmettes, Fédération nationale des Centres de lutte contre le Cancer (FNCLCC), Institut de génétique humaine (IGH), Université de Montpellier (UM)-Centre National de la Recherche Scientifique (CNRS), Centre Hospitalier Régional Universitaire [Montpellier] (CHRU Montpellier), Microenvironment, Cell Differentiation, Immunology and Cancer (MICMAC), Université de Rennes 1 (UR1), Université de Rennes (UNIV-RENNES)-Université de Rennes (UNIV-RENNES)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Structure Fédérative de Recherche en Biologie et Santé de Rennes ( Biosit : Biologie - Santé - Innovation Technologique ), CHU Pontchaillou [Rennes], Institut Universitaire du Cancer de Toulouse - Oncopole (IUCT Oncopole - UMR 1037), Université Toulouse III - Paul Sabatier (UT3), Université Fédérale Toulouse Midi-Pyrénées-Université Fédérale Toulouse Midi-Pyrénées-CHU Toulouse [Toulouse]-Institut National de la Santé et de la Recherche Médicale (INSERM), Lipides - Nutrition - Cancer [Dijon - U1231] (LNC), Université de Bourgogne (UB)-Institut National de la Santé et de la Recherche Médicale (INSERM)-AgroSup Dijon - Institut National Supérieur des Sciences Agronomiques, de l'Alimentation et de l'Environnement, Institute for Advanced Biosciences / Institut pour l'Avancée des Biosciences (Grenoble) (IAB), Centre Hospitalier Universitaire [Grenoble] (CHU)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Etablissement français du sang - Auvergne-Rhône-Alpes (EFS)-Centre National de la Recherche Scientifique (CNRS)-Université Grenoble Alpes [2016-2019] (UGA [2016-2019]), ANR-11-LABX-0024,ParaFrap,Alliance française contre les maladies parasitaires(2011), European Project: IC18CT980373, Université de Franche-Comté (UFC), Université Bourgogne Franche-Comté [COMUE] (UBFC)-Université Bourgogne Franche-Comté [COMUE] (UBFC)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre Hospitalier Régional Universitaire de Besançon (CHRU Besançon)-Etablissement français du sang [Bourgogne-Franche-Comté] (EFS [Bourgogne-Franche-Comté]), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université de Nantes - UFR de Médecine et des Techniques Médicales (UFR MEDECINE), Université de Nantes (UN)-Université de Nantes (UN)-Centre hospitalier universitaire de Nantes (CHU Nantes)-Centre National de la Recherche Scientifique (CNRS)-Université d'Angers (UA), Université de Bourgogne (UB)-AgroSup Dijon - Institut National Supérieur des Sciences Agronomiques, de l'Alimentation et de l'Environnement-Institut National de la Santé et de la Recherche Médicale (INSERM), Centre Hospitalier Universitaire [Grenoble] (CHU)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-Etablissement français du sang - Auvergne-Rhône-Alpes (EFS)-Université Grenoble Alpes [2016-2019] (UGA [2016-2019]), Institut National de la Santé et de la Recherche Médicale (INSERM)-Etablissement français du sang [Bourgogne-Franche-Comté] (EFS BFC)-Université de Franche-Comté (UFC), Centre Hospitalier Régional Universitaire de Besançon (CHRU Besançon)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Etablissement français du sang [Bourgogne-Franche-Comté] (EFS BFC)-Université de Franche-Comté (UFC), Université de Rennes (UR)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Structure Fédérative de Recherche en Biologie et Santé de Rennes ( Biosit : Biologie - Santé - Innovation Technologique ), Université de Toulouse (UT)-Université de Toulouse (UT)-Centre Hospitalier Universitaire de Toulouse (CHU Toulouse)-Institut National de la Santé et de la Recherche Médicale (INSERM), UCL - SSS/IREC/SLUC - Pôle St.-Luc, UCL - (SLuc) Service de biologie hématologique, Garnache-Ottou, F., Vidal, C., Biichle, S., Renosi, F., Poret, E., Pagadoy, M., Desmarets, M., Roggy, A., Seilles, E., Soret, L., Schillinger, F., Puyraimond, S., Petrella, T., Preudhomme, C., Roumier, C., Macintyre, E. A., Harrivel, V., Desbrosses, Y., Gruson, B., Genevieve, F., Thepot, S., Drebit, Y., Leguay, T., Gros, F. -X., Lechevalier, N., Saussoy, P., Salaun, V., Cornet, E., Benseddik, Z., Veyrat-Masson, R., Wagner-Ballon, O., Salanoubat, C., Maynadie, M., Guy, J., Caillot, D., Jacob, M. -C., Cahn, J. -Y., Gressin, R., Rose, J., Quesnel, B., Guerin, E., Trimoreau, F., Feuillard, J., Gourin, M. -P., Plesa, A., Baseggio, L., Arnoux, I., Vey, N., Blaise, D., Lacroix, R., Arnoulet, C., Benet, B., Dorvaux, V., Bret, C., Drenou, B., Debliquis, A., Latger-Cannard, V., Bonmati, C., Bene, M. -C., Peterlin, P., Ticchioni, M., Rohrlich, P. -S., Arnaud, A., Wickenhauser, S., Bardet, V., Brechignac, S., Papoular, B., Raggueneau, V., Vargaftig, J., Letestu, R., Lusina, D., Braun, T., Foissaud, V., Tamburini, J., Bennani, H., Freynet, N., Cordonnier, C., Le Garff-Tavernier, M., Jacques, N., Maloum, K., Roos-Weil, D., Bouscary, D., Asnafi, V., Lhermitte, L., Suarez, F., Lengline, E., Feger, F., Battipaglia, G., Mohty, M., Bouyer, S., Ghoual, O., Dindinaud, E., Basle, C., Puyade, M., Lafon, C., Fest, T., Roussel, M., Cahu, X., Bera, E., Daliphard, S., Jardin, F., Campos, L., Solly, F., Guyotat, D., Galoisy, A. -C., Eischen, A., Mayeur-Rousse, C., Guffroy, B., Recher, C., Loosveld, M., Garnier, A., Barlogis, V., Rosenthal, M. A., Brun, S., Contentin, N., Maury, S., Callanan, M., Lefebvre, C., Maillard, N., Okamba, P., Ferrand, C., Adotevi, O., Saas, P., Angelot-Delettre, F., Binda, D., and Deconinck, E.

Subjects
Oncology, Vincristine, medicine.medical_specialty, Myeloid, Cyclophosphamide, Clinical Trials and Observations, medicine.medical_treatment, [SDV.CAN]Life Sciences [q-bio]/Cancer, Hematopoietic stem cell transplantation, Plasmacytoid dendritic cell, Immunophenotyping, Clonal Evolution, 03 medical and health sciences, 0302 clinical medicine, Bone Marrow, hemic and lymphatic diseases, Internal medicine, medicine, Humans, Neoplasm Metastasis, Neoplasm Staging, 030304 developmental biology, Chromosome Aberrations, 0303 health sciences, Leukemia, business.industry, Hematopoietic Stem Cell Transplantation, Disease Management, Myeloid leukemia, Dendritic Cells, Hematology, Prognosis, medicine.disease, Blood Cell Count, 3. Good health, Transplantation, Treatment Outcome, medicine.anatomical_structure, 030220 oncology & carcinogenesis, Acute Disease, business, Biomarkers, medicine.drug
Abstract

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and aggressive leukemia for which we developed a nationwide network to collect data from new cases diagnosed in France. In a retrospective, observational study of 86 patients (2000-2013), we described clinical and biological data focusing on morphologies and immunophenotype. We found expression of markers associated with plasmacytoid dendritic cell origin (HLA-DRhigh, CD303+, CD304+, and cTCL1+) plus CD4 and CD56 and frequent expression of isolated markers from the myeloid, B-, and T-lymphoid lineages, whereas specific markers (myeloperoxidase, CD14, cCD3, CD19, and cCD22) were not expressed. Fifty-one percent of cytogenetic abnormalities impact chromosomes 13, 12, 9, and 15. Myelemia was associated with an adverse prognosis. We categorized chemotherapeutic regimens into 5 groups: acute myeloid leukemia (AML)–like, acute lymphoid leukemia (ALL)–like, lymphoma (cyclophosphamide, doxorubicin, vincristine, and prednisone [CHOP])–like, high-dose methotrexate with asparaginase (Aspa-MTX) chemotherapies, and not otherwise specified (NOS) treatments. Thirty patients received allogeneic hematopoietic cell transplantation (allo-HCT), and 4 patients received autologous hematopoietic cell transplantation. There was no difference in survival between patients receiving AML-like, ALL-like, or Aspa-MTX regimens; survival was longer in patients who received AML-like, ALL-like, or Aspa-MTX regimens than in those who received CHOP-like regimens or NOS. Eleven patients are in persistent complete remission after allo-HCT with a median survival of 49 months vs 8 for other patients. Our series confirms a high response rate with a lower toxicity profile with the Aspa-MTX regimen, offering the best chance of access to hematopoietic cell transplantation and a possible cure.

Published
2019
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150. Gilteritinib or Chemotherapy for Relapsed or Refractory FLT3-Mutated AML

Author

Robert K. Stuart, Je-Hwan Lee, Andreas Neubauer, Jorge E. Cortes, Richard A. Larson, Timothy S. Pardee, Mark J. Levis, Chaofeng Liu, Harry P. Erba, Sung Soo Yoon, Celalettin Ustun, Ellin Berman, Maria R. Baer, Nikolai A. Podoltsev, Wen-Chien Chou, Giovanni Martinelli, Nahla Hasabou, Pau Montesinos, Antonio Di Stasi, Stefania Paolini, Alexander E. Perl, Francesco Fabbiano, Hisayuki Yokoyama, Xuan Liu, Margaret Kasner, Rebecca L. Olin, Naoko Hosono, Erkut Bahceci, Amir T. Fathi, Fabio Ciceri, Christian Recher, Perl, A. E., Martinelli, G., Cortes, J. E., Neubauer, A., Berman, E., Paolini, S., Montesinos, P., Baer, M. R., Larson, R. A., Ustun, C., Fabbiano, F., Erba, H. P., Di Stasi, A., Stuart, R., Olin, R., Kasner, M., Ciceri, F., Chou, W. -C., Podoltsev, N., Recher, C., Yokoyama, H., Hosono, N., Yoon, S. -S., Lee, J. -H., Pardee, T., Fathi, A. T., Liu, C., Hasabou, N., Liu, X., Bahceci, E., and Levis, M. J.

Subjects
Myeloid, medicine.medical_treatment, Salvage therapy, 030204 cardiovascular system & hematology, 03 medical and health sciences, chemistry.chemical_compound, fluids and secretions, 0302 clinical medicine, hemic and lymphatic diseases, Medicine, 030212 general & internal medicine, Quizartinib, Chemotherapy, business.industry, Myeloid leukemia, hemic and immune systems, General Medicine, medicine.disease, Leukemia, medicine.anatomical_structure, chemistry, embryonic structures, Fms-Like Tyrosine Kinase 3, Cancer research, business, Crenolanib
Abstract

BACKGROUND Patients with relapsed or refractory acute myeloid leukemia (AML) with mutations in the FMS-like tyrosine kinase 3 gene (FLT3) infrequently have a response to salvage chemotherapy. Gilteritinib is an oral, potent, selective FLT3 inhibitor with single-agent activity in relapsed or refractory FLT3-mutated AML. METHODS In a phase 3 trial, we randomly assigned adults with relapsed or refractory FLT3mutated AML in a 2:1 ratio to receive either gilteritinib (at a dose of 120 mg per day) or salvage chemotherapy. The two primary end points were overall survival and the percentage of patients who had complete remission with full or partial hematologic recovery. Secondary end points included event-free survival (freedom from treatment failure [i.e., relapse or lack of remission] or death) and the percentage of patients who had complete remission. RESULTS Of 371 eligible patients, 247 were randomly assigned to the gilteritinib group and 124 to the salvage chemotherapy group. The median overall survival in the gilteritinib group was significantly longer than that in the chemotherapy group (9.3 months vs. 5.6 months; hazard ratio for death, 0.64; 95% confidence interval [CI], 0.49 to 0.83; P

Published
2019
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