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1. Novel prognostic scoring systems for severe CRS and ICANS after anti-CD19 CAR T cells in large B-cell lymphoma

Author

Sesques, Pierre, Kirkwood, Amy A., Kwon, Mi, Rejeski, Kai, Jain, Michael D., Di Blasi, Roberta, Brisou, Gabriel, Gros, François-Xavier, le Bras, Fabien, Bories, Pierre, Choquet, Sylvain, Rubio, Marie-Thérèse, Iacoboni, Gloria, O’Reilly, Maeve, Casasnovas, René-Olivier, Bay, Jacques-Olivier, Mohty, Mohamad, Joris, Magalie, Abraham, Julie, Castilla Llorente, Cristina, Loschi, Mickael, Carras, Sylvain, Chauchet, Adrien, La Rochelle, Laurianne Drieu, Hermine, Olivier, Guidez, Stéphanie, Cony-Makhoul, Pascale, Fogarty, Patrick, Le Gouill, Steven, Morschhauser, Franck, Gastinne, Thomas, Cartron, Guillaume, Subklewe, Marion, Locke, Frederick L., Sanderson, Robin, Barba, Pere, Houot, Roch, and Bachy, Emmanuel

Published
2024
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2. Prospective validation of a biomarker-driven response prediction model to romiplostim in lower-risk myelodysplastic neoplasms – results of the EUROPE trial by EMSCO

Author

Kubasch, Anne Sophie, Giagounidis, Aristoteles, Metzgeroth, Georgia, Jonasova, Anna, Herbst, Regina, Diaz, Jose Miguel Torregrosa, De Renzis, Benoit, Götze, Katharina S., Huetter-Kroenke, Marie-Luise, Gourin, Marie-Pierre, Slama, Borhane, Dimicoli-Salazar, Sophie, Cony-Makhoul, Pascale, Laribi, Kamel, Park, Sophie, Jersemann, Katja, Schipp, Dorothea, Metzeler, Klaus H., Tiebel, Oliver, Sockel, Katja, Gloaguen, Silke, Mies, Anna, Chermat, Fatiha, Thiede, Christian, Sapena, Rosa, Schlenk, Richard F., Fenaux, Pierre, Platzbecker, Uwe, and Adès, Lionel

Published
2022
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3. Germline ATG2B/GSKIP-containing 14q32 duplication predisposes to early clonal hematopoiesis leading to myeloid neoplasms

Author

Pegliasco, Jean, Hirsch, Pierre, Marzac, Christophe, Isnard, Françoise, Meniane, Jean-Côme, Deswarte, Caroline, Pellet, Philippe, Lemaitre, Céline, Leroy, Gwendoline, Rabadan Moraes, Graciela, Guermouche, Hélène, Schmaltz-Panneau, Barbara, Pasquier, Florence, Colas, Chrystelle, Benusiglio, Patrick R., Bera, Odile, Bourhis, Jean-Henri, Brissot, Eolia, Caron, Olivier, Chraibi, Samy, Cony-Makhoul, Pascale, Delaunay-Darivon, Christine, Lapusan, Simona, de Fontbrune, Flore Sicre, Fuseau, Pascal, Najman, Albert, Vainchenker, William, Delhommeau, François, Micol, Jean-Baptiste, Plo, Isabelle, and Bellanné-Chantelot, Christine

Published
2022
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6. P1186: A LARGE FRENCH REAL WORLD MULTICENTRIC PROSPECTIVE COHORT OF PATIENTS WITH LYMPHOMA (REALYSA STUDY): DESCRIPTION OF THE DIFFUSE LARGE B CELL LYMPHOMA PATIENTS IN REAL WORLD IN FRANCE

Author

H. Ghesquieres, F. Cherblanc, A. Belot, V. Camus, K. Thokagevistk, K.-K. Bouabdallah, C. Esnault, L.-M. Fornecker, S. Micon, F. Bijou, C. Haioun, N. Morineau, L. Ysebaert, G. Damaj, S. Le Gouill, S. Guidez, F. Morschhauser, C. Thiéblemont, A. Chauchet, R. Gressin, F. Jardin, C. Fruchart, G. Labouré, L. Fouillet, P. Lionne-Huyghe, A. Bonnet, L. Lebras, S. Amorim, C. Leyronnas, G. Olivier, R. Guieze, T. Lamy, V. Launay, B. Drenou, O. Fitoussi, L. Detourmignies, J. Abraham, C. Soussain, F. Lachenal, P. Fogarty, P. Cony-Makhoul, A. Bernier, S. Le Guyader-Peyrou, A. Monnereau, F. Boissard, and C. Rossi

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Published
2022
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7. S169: CLINICAL AND MOLECULAR MARKERS FOR PREDICTING RESPONSE TO ROMIPLOSTIM TREATMENT IN LOWER-RISK MYELODYSPLASTIC SYNDROMES

Author

A. S. Kubasch, A. Giagounidis, G. Metzgeroth, A. Jonasova, R. Herbst, J. M. T. Diaz, B. De Renzis, K. S. Götze, M.-L. Huetter-Kroenke, M.-P. Gourin, B. Slama, S. Dimicoli-Salazar, P. Cony-Makhoul, K. Laribi, S. Park, K. Jersemann, D. Schipp, K. H. Metzeler, O. Tiebel, K. Sockel, S. Gloaguen, A. Mies, F. Chermat, C. Thiede, R. Sapena, R. F. Schlenk, P. Fenaux, U. Platzbecker, and L. Ades

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Published
2022
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8. Exome sequencing identifies germline variants in DIS3 in familial multiple myeloma

Author

Pertesi, Maroulio, Vallée, Maxime, Wei, Xiaomu, Revuelta, Maria V., Galia, Perrine, Demangel, Delphine, Oliver, Javier, Foll, Matthieu, Chen, Siwei, Perrial, Emeline, Garderet, Laurent, Corre, Jill, Leleu, Xavier, Boyle, Eileen M., Decaux, Olivier, Rodon, Philippe, Kolb, Brigitte, Slama, Borhane, Mineur, Philippe, Voog, Eric, Le Bris, Catherine, Fontan, Jean, Maigre, Michel, Beaumont, Marie, Azais, Isabelle, Sobol, Hagay, Vignon, Marguerite, Royer, Bruno, Perrot, Aurore, Fuzibet, Jean-Gabriel, Dorvaux, Véronique, Anglaret, Bruno, Cony-Makhoul, Pascale, Berthou, Christian, Desquesnes, Florence, Pegourie, Brigitte, Leyvraz, Serge, Mosser, Laurent, Frenkiel, Nicole, Augeul-Meunier, Karine, Leduc, Isabelle, Leyronnas, Cécile, Voillat, Laurent, Casassus, Philippe, Mathiot, Claire, Cheron, Nathalie, Paubelle, Etienne, Moreau, Philippe, Bignon, Yves–Jean, Joly, Bertrand, Bourquard, Pascal, Caillot, Denis, Naman, Hervé, Rigaudeau, Sophie, Marit, Gérald, Macro, Margaret, Lambrecht, Isabelle, Cliquennois, Manuel, Vincent, Laure, Helias, Philippe, Avet-Loiseau, Hervé, Moreno, Victor, Reis, Rui Manuel, Varkonyi, Judit, Kruszewski, Marcin, Vangsted, Annette Juul, Jurczyszyn, Artur, Zaucha, Jan Maciej, Sainz, Juan, Krawczyk-Kulis, Malgorzata, Wątek, Marzena, Pelosini, Matteo, Iskierka-Jażdżewska, Elzbieta, Grząśko, Norbert, Martinez-Lopez, Joaquin, Jerez, Andrés, Campa, Daniele, Buda, Gabriele, Lesueur, Fabienne, Dudziński, Marek, García-Sanz, Ramón, Nagler, Arnon, Rymko, Marcin, Jamroziak, Krzysztof, Butrym, Aleksandra, Canzian, Federico, Obazee, Ofure, Nilsson, Björn, Klein, Robert J., Lipkin, Steven M., McKay, James D., and Dumontet, Charles

Published
2019
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9. Lenalidomide with or without erythropoietin in transfusion-dependent erythropoiesis-stimulating agent-refractory lower-risk MDS without 5q deletion

Author

Toma, A, Kosmider, O, Chevret, S, Delaunay, J, Stamatoullas, A, Rose, C, Beyne-Rauzy, O, Banos, A, Guerci-Bresler, A, Wickenhauser, S, Caillot, D, Laribi, K, De Renzis, B, Bordessoule, D, Gardin, C, Slama, B, Sanhes, L, Gruson, B, Cony-Makhoul, P, Chouffi, B, Salanoubat, C, Benramdane, R, Legros, L, Wattel, E, Tertian, G, Bouabdallah, K, Guilhot, F, Taksin, A L, Cheze, S, Maloum, K, Nimuboma, S, Soussain, C, Isnard, F, Gyan, E, Petit, R, Lejeune, J, Sardnal, V, Renneville, A, Preudhomme, C, Fontenay, M, Fenaux, P, and Dreyfus, F

Published
2016
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10. A randomised phase II trial of hydroxychloroquine and imatinib versus imatinib alone for patients with chronic myeloid leukaemia in major cytogenetic response with residual disease

Author

Annie Latif, Arunima Mukhopadhyay, Mhairi Copland, Fiona Thomson, Dragana Milojkovic, Letizia Foroni, G. M. Smith, Jenny Byrne, Gillian A. Horne, Paolo Gallipoli, Philippe Schafhausen, Judith Dixon-Hughes, Franck E. Nicolini, P. Cony-Makhoul, Richard E. Clark, Wenjuan Cong, Gudmundur Vignir Helgason, Jon Stobo, Steffen Koschmieder, T H Brümmendorf, Lynn McMahon, Caroline Kelly, and Tessa L. Holyoake

Subjects
Male, 0301 basic medicine, Cancer Research, medicine.medical_specialty, Fusion Proteins, bcr-abl, Disease, Chronic myeloid leukaemia, Gastroenterology, Article, 03 medical and health sciences, 0302 clinical medicine, Chloroquine, Internal medicine, hemic and lymphatic diseases, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Antineoplastic Combined Chemotherapy Protocols, medicine, Clinical endpoint, Humans, Aged, Retrospective Studies, business.industry, Cancer stem cells, Imatinib, Hydroxychloroquine, Hematology, Middle Aged, Translational research, Prognosis, Survival Rate, 030104 developmental biology, Oncology, 030220 oncology & carcinogenesis, Cytogenetic Analysis, Leukemia, Myeloid, Chronic-Phase, Imatinib Mesylate, Female, Major Cytogenetic Response, Stem cell, business, Follow-Up Studies, medicine.drug
Abstract

1 In chronic-phase chronic myeloid leukaemia (CP-CML), residual BCR-ABL1+ leukaemia stem cells are 2 responsible for disease persistence despite TKI. Based on in vitro data, CHOICES (CHlorOquine and 3 Imatinib Combination to Eliminate Stem cells) was an international, randomised phase II trial designed 4 to study the safety and efficacy of imatinib (IM) and hydroxychloroquine (HCQ) compared to IM alone in 5 CP-CML patients in major cytogenetic remission with residual disease detectable by qPCR. Sixty-two 6 patients were randomly assigned to either arm. Treatment ‘successes’ was the primary end-point, 7 defined as ≥0.5 log reduction in 12-month qPCR level from trial entry. Selected secondary study end-8 points were 24-month treatment ‘successes’, molecular response and progression at 12 and 24 months, 9 comparison of IM levels, and achievement of blood HCQ levels >2000ng/ml. At 12 months, there was no 10 difference in ‘success’ rate (p=0.58); MMR was achieved in 80% (IM) vs 92% (IM/HCQ) (p=0.21). At 24 11 months, the ‘success’ rate was 20.8% higher with IM/HCQ (p=0.059). No patients progressed. 12 Seventeen adverse events, including four serious adverse reactions, were reported; diarrhoea occurred 13 more frequently with combination. IM/HCQ is tolerable in CP-CML, with modest improvement in qPCR 14 levels at 12 and 24 months, suggesting autophagy inhibition maybe of clinical value in CP-CML.

Published
2020
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17. Granulocyte–macrophage colony-stimulating factor (GM-CSF) to increase efficacy of intensive sequential chemotherapy with etoposide, mitoxantrone and cytarabine (EMA) in previously treated acute myeloid leukemia: a multicenter randomized placebo-controlled trial (EMA91 Trial)

Author

Thomas, X, Fenaux, P, Dombret, H, Delair, S, Dreyfus, F, Tilly, H, Vekhoff, A, Cony-Makhoul, P, Leblond, V, Troussard, X, Cordonnier, C, de Revel, T, Simon, M, Nicolini, F, Stoppa, AM, Janvier, M, Bordessoule, D, Rousselot, P, Ffrench, M, Marie, JP, and Archimbaud, E

Published
1999
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21. Caractéristiques cliniques, évolution à long terme et facteurs prédictifs de rechute après arrêt de l’Imatinib au cours de leucémie chronique à éosinophiles associée au réarrangement FIP1L1-PDGFRA: étude rétrospective à propos de 151 patients

Author

Mathilde Hunault-Berger, E. Lengline, F. Lhomme, M. Hamidou, Guillaume Lefèvre, P. Rousselot, Irène Machelart, M. Ebbo, P. Cony Makhoul, Jean-Emmanuel Kahn, Franck E. Nicolini, Aurélien Guffroy, Julien Rohmer, L. Galicier, Olivier Fain, J.S. Bladé, S. Tavitian, M. Groh, and Vincent Cottin

Subjects
Gastroenterology, Internal Medicine
Abstract

Introduction La leucemie chronique a eosinophiles associeeau transcrit FIP1L1-PDGFRA (LCE F/P) est un syndrome hypereosinophilique(SHE) clonal qui presente une grande sensibilite al’Imatinib Mesylate (IM) et dont l’incidence n’est pas connue. Les series publiees a ce jour sont de petite taille, avec un suivi limite et bien que les caracteristiques cliniques ainsi que la possibilite d’arret de l’IM soient connues, aucun facteur predictif de rechute n’a ete mis en evidence. Patients et methodes Etude francaise, multicentrique, retrospective en partenariat avec le groupe des biologistes moleculaires des hemopathies malignes incluant l’ensemble des patients ayant eu une recherche de F/P positive entre 2003 et 2019. Les patients avec une hemopathie aigue ou des donnees cliniques manquantes ont ete exclus. Un modele de regression logistique a ete utilise pour identifier des facteurs de risque de rechute. Resultats Parmi les 197 transcrits F/P positifs, 151 patients ont ete inclus (97 % d’hommes, âge moyen 49 ans). L’incidence de la LCE F/P etait estimee a 0,18 cas par an par million d’habitants. Comme precedemment decrit, les organes les plus frequemment atteints etaient la rate (44 %), la peau (32 %), le poumon (30 %), le coeur (19 %) et le Systeme Nerveux Central (10 %). Par ailleurs, des atteintes vasculaires (thrombose, occlusion arterielle, vascularite), articulaire et renale ont egalement ete decrites. Le pic d’eosinophilie moyen etait de 10,3 (± 6) G/l et les taux de vitamine B12, de tryptase et d’IgE totales etaient eleves dans 74/79 (94 %), 45/57 (79 %) et 12/86 (14 %) des cas tandis qu’une elevation franche de la CRP (i.e. > 40 mg/l) n’etait retrouvee que chez 5 patients. Trente et un (20 %) patients ont ete traites par corticoides sans aucune remission complete tandis que tous les patients traites par IM (n = 148) ont eu une reponse hematologique et moleculaire (n = 84 tests) complete. Parmi les 46 patients ayant arrete l’IM, 20 (47 %) ont presente une rechute. En analyse multivariee, le delai d’introduction du traitement (HR continu : 1,02 [1,00–1,03] ; p = 0,01) et la duree du traitement (HR continu : 0,97 [0,95–0,99] ; p = 0,01) etait des facteurs associes a la rechute. La duree de suivi moyenne etait de 80 (± 56) mois avec une survie globale a 1, 5 et 10 ans de 99 %, 95 % et 84 % respectivement. Conclusion Cette etude avec un large effectif confirme la predominance masculine de cette pathologie ainsi que la frequence des atteintes respiratoire, cardiaque, cutanee et splenique. Apres arret de l’IM une courte duree de traitement prealable et un delais prolonge d’introduction de l’IM etaient associes a la rechute.

Published
2020
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23. Real-world study of children and young adults with myeloproliferative neoplasms: identifying risks and unmet needs

Author

Sobas, Marta, Kiladjian, Jean-Jacques, Beauverd, Yan, Curto-Garcia, Natalia, Sadjadian, Parvis, Shih, Lee Yung, Devos, Timothy, Krochmalczyk, Dorota, Galli, Serena, Bieniaszewska, Maria, Seferynska, Ilona, McMullin, Mary Frances, Armatys, Anna, Spalek, Adrianna, Waclaw, Joanna, Zdrenghea, Mihnea, Legros, Laurence, Girodon, François, Lewandowski, Krzysztof, Angona Figueras, Anna, Samuelsson, Jan, Abuin Blanco, Aitor, Cony-Makhoul, Pascale, Collins, Angela, James, Chloé, Kusec, Rajko, Lauermannova, Marie, Noya, Maria Sol, Skowronek, Malgorzata, Szukalski, Lukasz, Szmigielska-Kaplon, Anna, Wondergem, Marielle, Dudchenko, Iryna, Gora Tybor, Joanna, Laribi, Kamel, Kulikowska de Nalecz, Anna, Demory, Jean-Loup, Le Du, Katell, Zweegman, Sonja, Besses Raebel, Carlos, Skoda, Radek, Giraudier, Stéphane, Griesshammer, Martin, Harrison, Claire N., and Ianotto, Jean-Christophe

Abstract

Myeloproliferative neoplasms (MPNs) are uncommon in children/young adults. Here, we present data on unselected patients diagnosed before 25 years of age included from 38 centers in 15 countries. Sequential patients were included. We identified 444 patients, with median follow-up 9.7 years (0-47.8). Forty-nine (11.1%) had a history of thrombosis at diagnosis, 49 new thrombotic events were recorded (1.16% patient per year [pt/y]), perihepatic vein thromboses were most frequent (47.6% venous events), and logistic regression identified JAK2V617F mutation (P= .016) and hyperviscosity symptoms (visual disturbances, dizziness, vertigo, headache) as risk factors (P= .040). New hemorrhagic events occurred in 44 patients (9.9%, 1.04% pt/y). Disease transformation occurred in 48 patients (10.9%, 1.13% pt/y), usually to myelofibrosis (7.5%) with splenomegaly as a novel risk factor for transformation in essential thrombocythemia (ET) (P= .000) in logistical regression. Eight deaths (1.8%) were recorded, 3 after allogeneic stem cell transplantation. Concerning conventional risk scores: International Prognostic Score for Essential Thrombocythemia-Thrombosis and new International Prognostic Score for Essential Thrombocythemia-Thrombosis differentiated ET patients in terms of thrombotic risk. Both scores identified high-risk patients with the same median thrombosis-free survival of 28.5 years. No contemporary scores were able to predict survival for young ET or polycythemia vera patients. Our data represents the largest real-world study of MPN patients age < 25 years at diagnosis. Rates of thrombotic events and transformation were higher than expected compared with the previous literature. Our study provides new and reliable information as a basis for prospective studies, trials, and development of harmonized international guidelines for the specific management of young patients with MPN.

Published
2022
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28. Efficacy of weekly paclitaxel-bevacizumab combination in advanced non squamous non-small cell lung cancer (NSCLC) progressing after immune checkpoint inhibitors - AVATAX , a retrospective multicentric study: Preliminary data

Author

Christos Chouaid, J. Pinsolle, S. Valery, P. Cony-Makhoul, G. Rousseau-Bussac, M. Geier, I. Zaccaria, P. Boré, Chantal Decroisette, Isabelle Monnet, F. Ennahdi, Renaud Descourt, Gilles Robinet, G. Bilger, and Anne-Claire Toffart

Subjects
medicine.medical_specialty, business.industry, Immune checkpoint inhibitors, Weekly paclitaxel, Hematology, Oncology, Third line, Non squamous, Family medicine, Honorarium, medicine, business, Accommodation, Toxicity profile, Third line treatment
Abstract

Background ULTIMATE, a phase III trial, showed a significant superiority regarding progression free survival (PFS) of the combination paclitaxel-bevacizumab versus docetaxel as second or third line treatment for advanced non-small cell lung cancer (NSCLC). With the increase use of immunotherapy in first-line setting, second line strategies must be redefined. Paclitaxel-bevacizumab combination could be an option. Methods The main objectives were to describe safety and efficacy of this combination in metastatic non-squamous NSCLC as second-line therapy or beyond with a special attention paid to the sub-group treated just after immune checkpoint inhibitors. It was a multicentric retrospective study. Results This analysis included from 1st September 2010 to 1st april 2018, 76 patients: 42 (55%) male, 18 (24%) and 36 (47%) treated in second and third line respectively, and 22 (29%) in fourth line or more. Overall response rate (ORR) was 37% (28/76) and disease control rate 74 % (56/76). Median PFS and OS were 5,7 [95%CI: 4,1-6,9] months and 11,2 [95%CI: 8-not reached] months respectively. In second and third line, ORR was respectively 39% and 42%, PFS 4 months [95%CI: 2,5-7,7] and 6 months [95%CI: 4-7], OS 9,4 months [95%CI: 2,7-not reached] and was not reached in third line at 12 months. Grade 3–4 adverse events included asthenia 5% (4/76), neurotoxicity 3% (2/76), bleeding events 4% (3/76), and hematological toxicity 1% (1/76). In the subset of patients treated in third-line or beyond, immediately after immunotherapy (33/76), we note with interest that ORR was 42% (14/33), median PFS was 6,2 [95% CI: 4,6-7,7] months, and median overall survival was not reached at 12 months. Conclusion This study shows an acceptable toxicity profile associated with encouraging efficacy of the combination paclitaxel-bevacizumab as second-line treatment or beyond for non –squamous NSCLC patients. Moreover, these results seems to be very promising preliminary findings for patients treated just after immunotherapy. AVATAX is ongoing and should collect data from about 200 patients. Legal entity responsible for the study Chantal Decroisette. Funding Has not received any funding. Disclosure C. Chouaid: Speaker Bureau / Expert testimony, Research grant / Funding (self), Research grant / Funding (institution), Travel / Accommodation / Expenses: AZ; Speaker Bureau / Expert testimony, Research grant / Funding (self), Research grant / Funding (institution), Travel / Accommodation / Expenses: BI; Speaker Bureau / Expert testimony, Research grant / Funding (self), Research grant / Funding (institution), Travel / Accommodation / Expenses: Bayer and Amgen; Speaker Bureau / Expert testimony, Research grant / Funding (self), Research grant / Funding (institution), Travel / Accommodation / Expenses: Takeda; Speaker Bureau / Expert testimony, Research grant / Funding (self), Research grant / Funding (institution), Travel / Accommodation / Expenses: Pfizer; Speaker Bureau / Expert testimony, Research grant / Funding (self), Research grant / Funding (institution), Travel / Accommodation / Expenses: Novartis; Speaker Bureau / Expert testimony, Research grant / Funding (self), Research grant / Funding (institution), Travel / Accommodation / Expenses: Lilly; Speaker Bureau / Expert testimony, Research grant / Funding (self), Research grant / Funding (institution), Travel / Accommodation / Expenses: MSD; Speaker Bureau / Expert testimony, Research grant / Funding (self), Research grant / Funding (institution), Travel / Accommodation / Expenses: Bristol-Myers Squibb; Speaker Bureau / Expert testimony, Research grant / Funding (self), Research grant / Funding (institution), Travel / Accommodation / Expenses: Sanofi Aventis; Speaker Bureau / Expert testimony, Research grant / Funding (self), Research grant / Funding (institution), Travel / Accommodation / Expenses: Roche; Speaker Bureau / Expert testimony, Research grant / Funding (self), Research grant / Funding (institution), Travel / Accommodation / Expenses: GSK. G. Rousseau-Bussac: Travel / Accommodation / Expenses: AstraZeneca; Honoraria (self): roche; Travel / Accommodation / Expenses: Bristol-Myers-Squibb, Travel / Accommodation / Expenses: Takeda; Travel / Accommodation / Expenses: Roche; Travel / Accommodation / Expenses: Pfizer; Travel / Accommodation / Expenses: Novartis; Travel / Accommodation / Expenses: MSD; Travel / Accommodation / Expenses: Mundipharma; Travel / Accommodation / Expenses: Lilly; Travel / Accommodation / Expenses: Janssen-Cilag; Travel / Accommodation / Expenses: Boehringer Ingelheim. J. Pinsolle: Speaker Bureau / Expert testimony, Travel / Accommodation / Expenses: MSD; Speaker Bureau / Expert testimony, Travel / Accommodation / Expenses: Takeda; Speaker Bureau / Expert testimony, Travel / Accommodation / Expenses: Pfizer; Speaker Bureau / Expert testimony, Travel / Accommodation / Expenses: Roche; Speaker Bureau / Expert testimony, Travel / Accommodation / Expenses: Pierre Fabre. R. Descourt: Speaker Bureau / Expert testimony, Travel / Accommodation / Expenses: Boehringer Ingelheim; Speaker Bureau / Expert testimony, Travel / Accommodation / Expenses: Pfizer; Speaker Bureau / Expert testimony, Travel / Accommodation / Expenses: Roche; Speaker Bureau / Expert testimony, Travel / Accommodation / Expenses: Bristol-Myers Squibb; Speaker Bureau / Expert testimony, Travel / Accommodation / Expenses: MSD; Speaker Bureau / Expert testimony, Travel / Accommodation / Expenses: AstraZeneca; Speaker Bureau / Expert testimony, Travel / Accommodation / Expenses: Takeda. M. Geier: Speaker Bureau / Expert testimony, Travel / Accommodation / Expenses: MSD; Speaker Bureau / Expert testimony, Travel / Accommodation / Expenses: Bristol-Myers Squibb. C. Decroisette: Advisory / Consultancy, Travel / Accommodation / Expenses: Roche; Speaker Bureau / Expert testimony, Travel / Accommodation / Expenses: Pfizer; Speaker Bureau / Expert testimony, Travel / Accommodation / Expenses: Takeda; Speaker Bureau / Expert testimony, Travel / Accommodation / Expenses: Boehringer Ingelheim; Speaker Bureau / Expert testimony, Travel / Accommodation / Expenses: AstraZeneca; Speaker Bureau / Expert testimony, Travel / Accommodation / Expenses: MSD; Speaker Bureau / Expert testimony, Travel / Accommodation / Expenses: Bristol-Myers Squibb. All other authors have declared no conflicts of interest.

Published
2019
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29. PS1472 CURRENT PRACTICE IN THE MANAGEMENT OF ESSENTIAL THROMBOCYTEMIA AND POLYCYTHEMIA VERA: A SURVEY FROM THE FRENCH INTERGROUP OF MYELOPROLIFERATIVE NEOPLASMS (FIM) AMONG 120 HEMATOLOGISTS AND INTERNISTS

Author

L. Roy, P. Cony-Makhoul, P. Lionne-Huyghe, J.-J. Kiladjian, E. Lippert, S. Giraudier, B. Dupriez, G. Fouquet, V. Ugo, and M. Wémeau

Subjects
medicine.medical_specialty, Polycythemia vera, Current practice, business.industry, medicine, Essential Thrombocytemia, Hematology, medicine.disease, business, Intensive care medicine
Published
2019
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30. Impact of the COVID-19 Epidemic on Patients with Myeloproliferative Neoplasia: The French Prospective Observational Study Covim

Author

Legros, Laurence, Boyer perrard, Françoise, Ranta, Dana, Roy, Lydia, Ojeda-Uribe, Mario, Giraudier, Stephane, Parquet, Nathalie, Ianotto, Jean Christophe, Wemeau, Mathieu, Plantier, Isabelle, Nicolini, Franck E., Lippert, Eric, Cony-Makhoul, Pascale, Barraco, Fiorenza, Stalnikiewicz, Laure, Garban, Frederic, Dupriez, Brigitte, Girodon, Francois, Brignier, Anne, Arkam, Yazid, Meunier, Mathieu, James, Chloe, Rolland-Neyret, Valérie, Ugo, Valerie, Morel, Pierre, Bene, Marie C, and Kiladjian, Jean-Jacques

Published
2022
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32. Genomic analysis of primary and secondary myelofibrosis redefines the prognostic impact of ASXL1 mutations: a FIM study

Author

Luque Paz, Damien, Riou, Jérémie, Verger, Emmanuelle, Cassinat, Bruno, Chauveau, Aurélie, Ianotto, Jean-Christophe, Dupriez, Brigitte, Boyer, Françoise, Renard, Maxime, Mansier, Olivier, Murati, Anne, Rey, Jérôme, Etienne, Gabriel, Mansat-De Mas, Véronique, Tavitian, Suzanne, Nibourel, Olivier, Girault, Stéphane, Le Bris, Yannick, Girodon, François, Ranta, Dana, Chomel, Jean-Claude, Cony-Makhoul, Pascale, Sujobert, Pierre, Robles, Margot, Ben Abdelali, Raouf, Kosmider, Olivier, Cottin, Laurane, Roy, Lydia, Sloma, Ivan, Vacheret, Fabienne, Wemeau, Mathieu, Mossuz, Pascal, Slama, Borhane, Cussac, Vincent, Denis, Guillaume, Walter-Petrich, Anouk, Burroni, Barbara, Jézéquel, Nathalie, Giraudier, Stéphane, Lippert, Eric, Socié, Gérard, Kiladjian, Jean-Jacques, and Ugo, Valérie

Abstract

We aimed to study the prognostic impact of the mutational landscape in primary and secondary myelofibrosis. The study included 479 patients with myelofibrosis recruited from 24 French Intergroup of Myeloproliferative Neoplasms (FIM) centers. The molecular landscape was studied by high-throughput sequencing of 77 genes. A Bayesian network allowed the identification of genomic groups whose prognostic impact was studied in a multistate model considering transitions from the 3 conditions: myelofibrosis, acute leukemia, and death. Results were validated using an independent, previously published cohort (n = 276). Four genomic groups were identified: patients with TP53 mutation; patients with ≥1 mutation in EZH2, CBL, U2AF1, SRSF2, IDH1, IDH2, NRAS, or KRAS (high-risk group); patients with ASXL1-only mutation (ie, no associated mutation in TP53 or high-risk genes); and other patients. A multistate model found that both TP53 and high-risk groups were associated with leukemic transformation (hazard ratios [HRs] [95% confidence interval], 8.68 [3.32-22.73] and 3.24 [1.58-6.64], respectively) and death from myelofibrosis (HRs, 3.03 [1.66-5.56] and 1.77 [1.18-2.67], respectively). ASXL1-only mutations had no prognostic value that was confirmed in the validation cohort. However, ASXL1 mutations conferred a worse prognosis when associated with a mutation in TP53 or high-risk genes. This study provides a new definition of adverse mutations in myelofibrosis with the addition of TP53, CBL, NRAS, KRAS, and U2AF1 to previously described genes. Furthermore, our results argue that ASXL1 mutations alone cannot be considered detrimental.

Published
2021
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33. Genomic analysis of primary and secondary myelofibrosis redefines the prognostic impact of ASXL1mutations: a FIM study

Author

Luque Paz, Damien, Riou, Jérémie, Verger, Emmanuelle, Cassinat, Bruno, Chauveau, Aurélie, Ianotto, Jean-Christophe, Dupriez, Brigitte, Boyer, Françoise, Renard, Maxime, Mansier, Olivier, Murati, Anne, Rey, Jérôme, Etienne, Gabriel, Mansat-De Mas, Véronique, Tavitian, Suzanne, Nibourel, Olivier, Girault, Stéphane, Le Bris, Yannick, Girodon, François, Ranta, Dana, Chomel, Jean-Claude, Cony-Makhoul, Pascale, Sujobert, Pierre, Robles, Margot, Ben Abdelali, Raouf, Kosmider, Olivier, Cottin, Laurane, Roy, Lydia, Sloma, Ivan, Vacheret, Fabienne, Wemeau, Mathieu, Mossuz, Pascal, Slama, Borhane, Cussac, Vincent, Denis, Guillaume, Walter-Petrich, Anouk, Burroni, Barbara, Jézéquel, Nathalie, Giraudier, Stéphane, Lippert, Eric, Socié, Gérard, Kiladjian, Jean-Jacques, and Ugo, Valérie

Abstract

We aimed to study the prognostic impact of the mutational landscape in primary and secondary myelofibrosis. The study included 479 patients with myelofibrosis recruited from 24 French Intergroup of Myeloproliferative Neoplasms (FIM) centers. The molecular landscape was studied by high-throughput sequencing of 77 genes. A Bayesian network allowed the identification of genomic groups whose prognostic impact was studied in a multistate model considering transitions from the 3 conditions: myelofibrosis, acute leukemia, and death. Results were validated using an independent, previously published cohort (n = 276). Four genomic groups were identified: patients with TP53mutation; patients with ≥1 mutation in EZH2, CBL, U2AF1, SRSF2, IDH1, IDH2, NRAS, or KRAS(high-risk group); patients with ASXL1-only mutation (ie, no associated mutation in TP53or high-risk genes); and other patients. A multistate model found that both TP53and high-risk groups were associated with leukemic transformation (hazard ratios [HRs] [95% confidence interval], 8.68 [3.32-22.73] and 3.24 [1.58-6.64], respectively) and death from myelofibrosis (HRs, 3.03 [1.66-5.56] and 1.77 [1.18-2.67], respectively). ASXL1-only mutations had no prognostic value that was confirmed in the validation cohort. However, ASXL1mutations conferred a worse prognosis when associated with a mutation in TP53or high-risk genes. This study provides a new definition of adverse mutations in myelofibrosis with the addition of TP53, CBL, NRAS, KRAS, and U2AF1to previously described genes. Furthermore, our results argue that ASXL1mutations alone cannot be considered detrimental.

Published
2021
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34. Guide de recommandations d’utilisation des bisphosphonates dans les lésions osseuses malignes des tumeurs solides et du myélome multiple

Author

C. Sebban, A.-G. Bodard, C. Roemer-Becuwe, P. Biron, J.-F. Brantus, P. Collet, H. Ghesquières, J P Larbre, I. Krakowski, S. Salino, J.-P. Guastalla, F. Farsi, Bertrand Favier, V. Balestrière, A. Fontana, G. Blanc, F. Brocard, T. Thomas, S. Martinon, Michel Pavic, Philippe Debourdeau, and P. Cony-Makhoul

Subjects
medicine.medical_specialty, business.industry, medicine.medical_treatment, Gastroenterology, MEDLINE, Cancer, Guideline, medicine.disease, Radiation therapy, Spinal cord compression, Bone surgery, Internal Medicine, medicine, In patient, Radiology, business, Multiple myeloma
Abstract

Bisphosphonates are indicated for the treatment of bone lesions in patients with solid tumours or multiple myeloma. Bisphosphonates have proven their effectiveness in reducing the number of bone complications (hypercalcemia, pain, disease-related fractures, spinal cord compression) and delaying their occurrence in patients with bone tumours; they have also been shown to reduce the need for bone surgery and palliative or pain-relieving radiotherapy in these patients. International recommendations for the treatment of bone lesions related to malignant solid tumours and multiple myeloma have been established. We have elaborated clinical practice guidelines on the use of bisphosphonates to assist treatment decision-making in bone oncology. The guide contains decision trees and tables with information to guide pre-treatment evaluation and patient follow-up, as well as indications and conditions of use of bisphosphonates. In 2007, the regional cancer network of Rhone-Alpes, ONCORA, formed a working group (GIP ONCORA) to elaborate the guideline. The final version was then discussed and adopted at a plenary session in July 2009, during a collaborative workshop on supportive care recommendations organized by ONCORA and the regional cancer network of Lorraine.

Published
2011
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35. Long-term outcome of imatinib 400 mg compared to imatinib 600 mg or imatinib 400 mg daily in combination with cytarabine or pegylated interferon alpha 2a for chronic myeloid leukaemia: results from the French SPIRIT phase III randomised trial

Author

Guilhot, Francois, Rigal-Huguet, Françoise, Guilhot, Joëlle, Guerci-Bresler, Agnès-Paule, Maloisel, Frédéric, Rea, Delphine, Coiteux, Valérie, Gardembas, Martine, Berthou, Christian, Vekhoff, Anne, Jourdan, Eric, Berger, Marc, Fouillard, Loïc, Alexis, Magda, Legros, Laurence, Rousselot, Philippe, Delmer, Alain, Lenain, Pascal, Escoffre Barbe, Martine, Gyan, Emmanuel, Bulabois, Claude-Eric, Dubruille, Viviane, Joly, Bertrand, Pollet, Bertrand, Cony-Makhoul, Pascale, Johnson-Ansah, Hyacinthe, Mercier, Melanie, Caillot, Denis, Charbonnier, Aude, Kiladjian, Jean-Jacques, Chapiro, Jacques, Penot, Amélie, Dorvaux, Véronique, Vaida, Iona, Santagostino, Alberto, Roy, Lydia, Zerazhi, Hacene, Deconinck, Eric, Maisonneuve, Herve, Plantier, Isabelle, Lebon, Delphine, Arkam, Yazid, Cambier, Nathalie, Ghomari, Kamel, Miclea, Jean-Michel, Glaisner, Sylvie, Cayuela, Jean-Michel, Chomel, Jean-Claude, Muller, Marc, Lhermitte, Ludovic, Delord, Marc, Preudhomme, Claude, Etienne, Gabriel, Mahon, François-Xavier, and Nicolini, Franck- Emmanuel

Abstract

The STI571 prospective randomised trial (SPIRIT) French trial is a four-arm study comparing imatinib (IM) 400 mg versus IM 600 mg, IM 400 mg + cytarabine (AraC), and IM 400 mg + pegylated interferon alpha2a (PegIFN-α2a) for the front-line treatment of chronic-phase chronic myeloid leukaemia (CML). Long-term analyses included overall and progression-free survival, molecular responses to treatment, and severe adverse events. Starting in 2003, the trial included 787 evaluable patients. The median overall follow-up of the patients was 13.5 years (range 3 months to 16.7 years). Based on intention-to-treat analyses, at 15 years, overall and progression-free survival were similar across arms: 85%, 83%, 80%, and 82% and 84%, 87%, 79%, and 79% for the IM 400 mg (N= 223), IM 600 mg (N= 171), IM 400 mg + AraC (N= 172), and IM 400 mg + PegIFN-α2a (N= 221) arms, respectively. The rate of major molecular response at 12 months and deep molecular response (MR4) over time were significantly higher with the combination IM 400 mg + PegIFN-α2a than with IM 400 mg: p= 0.0001 and p= 0.0035, respectively. Progression to advanced phases and secondary malignancies were the most frequent causes of death. Toxicity was the main reason for stopping AraC or PegIFN-α2a treatment.

Published
2021
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36. Germline ATG2B/GSKIP-containing 14q32 duplication predisposes to early clonal hematopoiesis leading to myeloid neoplasms

Author

Pegliasco, Jean, Hirsch, Pierre, Marzac, Christophe, Isnard, Françoise, Meniane, Jean-Côme, Deswarte, Caroline, Pellet, Philippe, Lemaitre, Céline, Leroy, Gwendoline, Rabadan Moraes, Graciela, Guermouche, Hélène, Schmaltz-Panneau, Barbara, Pasquier, Florence, Colas, Chrystelle, Benusiglio, Patrick R., Bera, Odile, Bourhis, Jean-Henri, Brissot, Eolia, Caron, Olivier, Chraibi, Samy, Cony-Makhoul, Pascale, Delaunay-Darivon, Christine, Lapusan, Simona, de Fontbrune, Flore Sicre, Fuseau, Pascal, Najman, Albert, Vainchenker, William, Delhommeau, François, Micol, Jean-Baptiste, Plo, Isabelle, and Bellanné-Chantelot, Christine

Abstract

The germline predisposition associated with the autosomal dominant inheritance of the 14q32 duplication implicating ATG2B/GSKIPgenes is characterized by a wide clinical spectrum of myeloid neoplasms. We analyzed 12 asymptomatic carriers and 52 patients aged 18–74 years from six families, by targeted sequencing of 41 genes commonly mutated in myeloid malignancies. We found that 75% of healthy carriers displayed early clonal hematopoiesis mainly driven by TET2mutations. Molecular landscapes of patients revealed two distinct routes of clonal expansion and leukemogenesis. The first route is characterized by the clonal dominance of myeloproliferative neoplasms (MPN)-driver events associated with TET2mutations in half of cases and mutations affecting splicing and/or the RAS pathway in one-third of cases, leading to the early development of MPN, mostly essential thrombocythemia, with a high risk of transformation (50% after 10 years). The second route is distinguished by the absence of MPN-driver mutations and leads to AML without prior MPN. These patients mostly harbored a genomic landscape specific to acute myeloid leukemia secondary to myelodysplastic syndrome. An unexpected result was the total absence of DNMT3Amutations in this cohort. Our results suggest that the germline duplication constitutively mimics hematopoiesis aging by favoring TET2clonal hematopoiesis.

Published
2021
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37. A randomised phase II trial of hydroxychloroquine and imatinib versus imatinib alone for patients with chronic myeloid leukaemia in major cytogenetic response with residual disease

Author

Horne, G. A., Stobo, J., Kelly, C., Mukhopadhyay, A., Latif, A. L., Dixon-Hughes, J., McMahon, L., Cony-Makhoul, P., Byrne, J., Smith, G., Koschmieder, S., BrÜmmendorf, T. H., Schafhausen, P., Gallipoli, P., Thomson, F., Cong, W., Clark, R. E., Milojkovic, D., Helgason, G. V., Foroni, L., Nicolini, F. E., Holyoake, T. L., and Copland, M.

Abstract

In chronic-phase chronic myeloid leukaemia (CP-CML), residual BCR-ABL1+ leukaemia stem cells are responsible for disease persistence despite TKI. Based on in vitro data, CHOICES (CHlorOquine and Imatinib Combination to Eliminate Stem cells) was an international, randomised phase II trial designed to study the safety and efficacy of imatinib (IM) and hydroxychloroquine (HCQ) compared with IM alone in CP-CML patients in major cytogenetic remission with residual disease detectable by qPCR. Sixty-two patients were randomly assigned to either arm. Treatment ‘successes’ was the primary end point, defined as ≥0.5 log reduction in 12-month qPCR level from trial entry. Selected secondary study end points were 24-month treatment ‘successes’, molecular response and progression at 12 and 24 months, comparison of IM levels, and achievement of blood HCQ levels >2000 ng/ml. At 12 months, there was no difference in ‘success’ rate (p= 0.58); MMR was achieved in 80% (IM) vs 92% (IM/HCQ) (p= 0.21). At 24 months, the ‘success’ rate was 20.8% higher with IM/HCQ (p= 0.059). No patients progressed. Seventeen serious adverse events, including four serious adverse reactions, were reported; diarrhoea occurred more frequently with combination. IM/HCQ is tolerable in CP-CML, with modest improvement in qPCR levels at 12 and 24 months, suggesting autophagy inhibition maybe of clinical value in CP-CML.

Published
2020
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38. Follow-Up of Complete Cytogenetic Remission in Patients With Chronic Myeloid Leukemia After Cessation of Interferon Alfa

Author

F X, Mahon, X, Delbrel, P, Cony-Makhoul, C, Fabères, J M, Boiron, C, Barthe, C, Bilhou-Nabéra, A, Pigneux, G, Marit, and J, Reiffers

Subjects
Adult, Male, Cancer Research, Time Factors, Remission Induction, Fusion Proteins, bcr-abl, Interferon-alpha, Antineoplastic Agents, Middle Aged, Disease-Free Survival, Statistics, Nonparametric, Survival Rate, Oncology, Recurrence, Risk Factors, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Humans, Female, Aged, Follow-Up Studies
Abstract

PURPOSE: A small proportion of patients with chronic myeloid leukemia (CML) achieve a complete cytogenetic response (CCR), defined as the disappearance of Philadelphia (Ph) chromosome–positive metaphases, after treatment with interferon alfa (IFN). In this population of patients, the question of whether treatment should then be withdrawn is not yet resolved. PATIENTS AND METHODS: In the present study, we followed 15 patients who stopped IFN after achieving CCR. In nine patients IFN was stopped in view of adverse reactions (n = 8) or patient’s choice (n = 1). For the remaining six patients, the treatment was stopped because no BCR/ABL rearrangement could be detected by reverse transcriptase polymerase chain reaction (RT-PCR) in four successive analyses using peripheral-blood samples. RESULTS: Loss of CCR and survival were not statistically different (P = .48; P = .7) for the 15 patients who stopped IFN compared with 41 other CCR patients who continued IFN therapy in our institution. The median follow-up after discontinuation of IFN treatment was 36 months (range, 6 to 108 months). Seven patients (47%) (females, or CCR > 24 months and RT-PCR negative before IFN cessation; P < .0001) did not relapse. Eight other patients (53%) relapsed (lost CCR) within 3 to 33 months of treatment discontinuation. One of them relapsed in major cytogenetic remission (MCR) and was still in MCR 87 months after stopping therapy without any treatment. CONCLUSION: It is possible to stop IFN treatment at least in some patients with CML who achieve a prolonged period of CCR. This study also illustrates the hypothesis that persistence of low numbers of Ph-positive cells does not necessarily imply hematologic relapse.

Published
2002
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39. Determination of cefepime and cefpirome in human serum by high-performance liquid chromatography using an ultrafiltration for antibiotics serum extraction

Author

Dominique Breilh, Marie-Claude Saux, A. Fratta, C Lavallee, P Cony-Makhoul, and Dominique Ducint

Subjects
Quality Control, Chromatography, medicine.diagnostic_test, medicine.drug_class, Chemistry, Cefepime, Antibiotics, Ultrafiltration, General Chemistry, Cefpirome, Sensitivity and Specificity, High-performance liquid chromatography, Cephalosporins, Minimum inhibitory concentration, Therapeutic drug monitoring, medicine, Humans, Drug Monitoring, Quantitative analysis (chemistry), Chromatography, High Pressure Liquid, medicine.drug
Abstract

The aim of this study was to describe a high-performance liquid chromatography (HPLC) assay for the determination of cefepime and cefpirome in human serum without changing chromatographic conditions. The assay consisted to measure cefepime and cefpirome which were unbound to proteins having a molecular mass of 10,000 or more by ultrafiltration followed by HPLC with a Supelcosil ABZ+ column and UV detection at a wavelength of 263 nm. The assay was been found to be linear and has been validated over the concentration range 200 to 0.50 microg/ml for both cefepime and cefpirome, from 200 microl serum, extracted. In future, the assay will support therapeutic drug monitoring for cefepime and cefpirome in neutropenic patients in correlation with microbiological parameters such as MIC90 (minimal inhibitory concentration of antibiotic which kills 90% of the initial bacterial inoculum) and clinical efficacy.

Published
1999
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40. DNA methylation profiling reveals a pathological signature that contributes to transcriptional defects of CD34+CD15− cells in early chronic‐phase chronic myeloid leukemia.

Author

Maupetit‐Mehouas, Stéphanie, Court, Franck, Bourgne, Céline, Guerci‐Bresler, Agnès, Cony‐Makhoul, Pascale, Johnson, Hyacinthe, Etienne, Gabriel, Rousselot, Philippe, Guyotat, Denis, Janel, Alexandre, Hermet, Eric, Saugues, Sandrine, Berger, Juliette, Arnaud, Philippe, and Berger, Marc G.

Abstract

Despite the high efficiency of tyrosine kinase inhibitors (TKI), some patients with chronic myeloid leukemia (CML) will display residual disease that can become resistant to treatment, indicating intraclonal heterogeneity in chronic‐phase CML (CP‐CML). To determine the basis of this heterogeneity, we conducted the first exhaustive characterization of the DNA methylation pattern of sorted CP‐CML CD34+CD15− (immature) and CD34−CD15+ (mature) cells at diagnosis (prior to any treatment) and compared it to that of CD34+CD15− and CD34−CD15+ cells isolated from healthy donors (HD). In both cell types, we identified several hundreds of differentially methylated regions (DMRs) showing DNA methylation changes between CP‐CML and HD samples, with only a subset of them in common between CD34+CD15− and CD34−CD15+ cells. This suggested DNA methylation variability within the same CML clone. We also identified 70 genes that could be aberrantly repressed upon hypermethylation and 171 genes that could be aberrantly expressed upon hypomethylation of some of these DMRs in CP‐CML cells, among which 18 and 81, respectively, were in CP‐CML CD34+CD15− cells only. We then validated the DNA methylation and expression defects of selected candidate genes. Specifically, we identified GAS2, a candidate oncogene, as a new example of gene the hypomethylation of which is associated with robust overexpression in CP‐CML cells. Altogether, we demonstrated that DNA methylation abnormalities exist at early stages of CML and can affect the transcriptional landscape of malignant cells. These observations could lead to the development of combination treatments with epigenetic drugs and TKI for CP‐CML. [ABSTRACT FROM AUTHOR]

Published
2018
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41. Exome sequencing identifies germline variants in DIS3in familial multiple myeloma

Author

Pertesi, Maroulio, Vallée, Maxime, Wei, Xiaomu, Revuelta, Maria V., Galia, Perrine, Demangel, Delphine, Oliver, Javier, Foll, Matthieu, Chen, Siwei, Perrial, Emeline, Garderet, Laurent, Corre, Jill, Leleu, Xavier, Boyle, Eileen M., Decaux, Olivier, Rodon, Philippe, Kolb, Brigitte, Slama, Borhane, Mineur, Philippe, Voog, Eric, Le Bris, Catherine, Fontan, Jean, Maigre, Michel, Beaumont, Marie, Azais, Isabelle, Sobol, Hagay, Vignon, Marguerite, Royer, Bruno, Perrot, Aurore, Fuzibet, Jean-Gabriel, Dorvaux, Véronique, Anglaret, Bruno, Cony-Makhoul, Pascale, Berthou, Christian, Desquesnes, Florence, Pegourie, Brigitte, Leyvraz, Serge, Mosser, Laurent, Frenkiel, Nicole, Augeul-Meunier, Karine, Leduc, Isabelle, Leyronnas, Cécile, Voillat, Laurent, Casassus, Philippe, Mathiot, Claire, Cheron, Nathalie, Paubelle, Etienne, Moreau, Philippe, Bignon, Yves–Jean, Joly, Bertrand, Bourquard, Pascal, Caillot, Denis, Naman, Hervé, Rigaudeau, Sophie, Marit, Gérald, Macro, Margaret, Lambrecht, Isabelle, Cliquennois, Manuel, Vincent, Laure, Helias, Philippe, Avet-Loiseau, Hervé, Moreno, Victor, Reis, Rui Manuel, Varkonyi, Judit, Kruszewski, Marcin, Vangsted, Annette Juul, Jurczyszyn, Artur, Zaucha, Jan Maciej, Sainz, Juan, Krawczyk-Kulis, Malgorzata, Wątek, Marzena, Pelosini, Matteo, Iskierka-Jażdżewska, Elzbieta, Grząśko, Norbert, Martinez-Lopez, Joaquin, Jerez, Andrés, Campa, Daniele, Buda, Gabriele, Lesueur, Fabienne, Dudziński, Marek, García-Sanz, Ramón, Nagler, Arnon, Rymko, Marcin, Jamroziak, Krzysztof, Butrym, Aleksandra, Canzian, Federico, Obazee, Ofure, Nilsson, Björn, Klein, Robert J., Lipkin, Steven M., McKay, James D., and Dumontet, Charles

Published
2019
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42. High efficacy of CD19 CAR T-cells in patients with transformed Waldenström macroglobulinemia.

Author

Durot, Eric, Roos-Weil, Damien, Chauchet, Adrien, Decroocq, Justine, Di Blasi, Roberta, Gastinne, Thomas, Bensaber, Hedi, Cheminant, Morgane, Jacquet, Caroline, Guidez, Stéphanie, Gros, François-Xavier, Bachy, Emmanuel, Coste, Arthur, Cony-Makhoul, Pascale, Treon, Steven P., Delmer, Alain, Reshef, Ran, Le Gouill, Steven, Castillo, Jorge J., and Houot, Roch

Abstract

Histological transformation of Waldenström macroglobulinemia (HT-WM) carries a poor prognosis with standard treatments. Here, we report the first series of HT-WM treated with CAR T-cells showing a high efficacy and no unexpected toxicity.

Published
2024
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43. [Practice guidelines of the use of bisphosphonates in solid tumours with bone metastases and in multiple myeloma]

Author

J F, Brantus, C, Roemer-Becuwe, P, Cony-Makhoul, S, Salino, A, Fontana, P, Debourdeau, T, Thomas, J P, Guastalla, H, Ghesquieres, C, Sebban, M, Pavic, P, Collet, J P, Larbre, S, Martinon, F, Brocard, A G, Bodard, G, Blanc, V, Balestrière, B, Favier, F, Farsi, I, Krakowski, and P, Biron

Subjects
Bone Density Conservation Agents, Diphosphonates, Decision Trees, Humans, Bone Neoplasms, Multiple Myeloma
Abstract

Bisphosphonates are indicated for the treatment of bone lesions in patients with solid tumours or multiple myeloma. Bisphosphonates have proven their effectiveness in reducing the number of bone complications (hypercalcemia, pain, disease-related fractures, spinal cord compression) and delaying their occurrence in patients with bone tumours; they have also been shown to reduce the need for bone surgery and palliative or pain-relieving radiotherapy in these patients. International recommendations for the treatment of bone lesions related to malignant solid tumours and multiple myeloma have been established. We have elaborated clinical practice guidelines on the use of bisphosphonates to assist treatment decision-making in bone oncology. The guide contains decision trees and tables with information to guide pre-treatment evaluation and patient follow-up, as well as indications and conditions of use of bisphosphonates. In 2007, the regional cancer network of Rhône-Alpes, ONCORA, formed a working group (GIP ONCORA) to elaborate the guideline. The final version was then discussed and adopted at a plenary session in July 2009, during a collaborative workshop on supportive care recommendations organized by ONCORA and the regional cancer network of Lorraine.

Published
2010

44. Syndrome lymphoprolifératif à lymphocytes granuleux de phénotype CD8+: une pathologie clonale d'évolution chronique

Author

Cl. Beylot, B. Leng, P. Cony-Makhoul, J.Ph. Merlio, Antoine Broustet, A.M. Ferrer, Th. Astier-Gin, Josy Reiffers, Jean-Luc Pellegrin, and A. De Mascharel

Subjects
Gastroenterology, Internal Medicine, Biology, Molecular biology
Abstract

Resume Les hyperlymphocytoses CD8 avec neutropenie constituent un groupe heterogene au sein duquel sont rassembles des etats cliniques benins reactionnels et d'authentiques pathologies neoplasiques entrant dans le cadre des leucemies a grands lymphocytes granuleux. Le pronostic de ces leucemies a LGL (Large Granular Lymphocyte) semble lie au phenotype: extremement severe et rapidement lethal lorsque le phenotype est de type NK, chronique et lentement evolutif lorsque le phenotype est de type T (CD8+). Les auteurs rapportent quatre observations d'hyperlymphocytose CD8+ avec neutropenie, correspondant a des proliferations lymphocytaires CD2±, CD3+, CD4-, CD8+, CD16-, CD56±, CD57+, associees a un rearrangement clonal du gene b du recepteur T. Dans deux des quatre observations, l'hyperlymphocytose sanguine caracteristique n'est apparue qu'apres la splenectomie, mais l'analyse retrospective des biopsies osteo-medullaires permet d'affirmer la preexistence de la pathologie clonale. Ces lymphocytes ont une faible activite Natural Killer contre la lignee K562. Il n'y a pas de sequence provirale HTLV1 integree dans l'ADN de ces cellules leucemiques. L'evolution de cette pathologie monoclonale est cliniquement benigne, chronique, avec un recul de plus de treize ans dans un cas. Si la splenctomie n'a pas corrige la neutropenie, elle a permis le controle d'une anemie hemolytique et d'une thrombopenie de type peripherique chez un patient.

Published
1992
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45. Une pathologie chevaleresque

Author

P. Cony-Makhoul, Jean-François Viallard, M. Parrens, Bernard Leng, Jean-Luc Pellegrin, Josy Reiffers, and M. Viallard

Subjects
business.industry, Gastroenterology, Internal Medicine, Medicine, business
Published
1999
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46. 87 A PHASE II STUDY OF THE EFFICACY AND SAFETY OF AN INTENSIFIED SCHEDULE OF AZACITIDINE IN HIGHER RISK MDS PATIENTS: A STUDY BY THE GFM

Author

Marie Sebert, Lionel Adès, S. Chevret, Agnès Guerci-Bresler, Jacques Delaunay, J. Lejeune, Pierre Fenaux, P. Cony Makhoul, Laurence Legros, and Christian Recher

Subjects
Oncology, Cancer Research, medicine.medical_specialty, Schedule, business.industry, Internal medicine, Azacitidine, Medicine, Phases of clinical research, Hematology, business, medicine.drug
Published
2015
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47. CD19-Targeting CAR T-Cell Therapy in Transformed Waldenström Macroglobulinemia/Lymphoplasmacytic Lymphoma: A Descar-T and US Collaborative Study

Author

Durot, Eric, Roos-Weil, Damien, Chauchet, Adrien, Decroocq, Justine, Di Blasi, Roberta, Gastinne, Thomas, Bensader, Hedi, Cheminant, Morgane, Jacquet, Caroline, Guidez, Stephanie, Gros, François-Xavier, Bachy, Emmanuel, Cony-Makhoul, Pascale, Treon, Steven P, Delmer, Alain Jacques, Reshef, Ran, Le Gouill, Steven, Castillo, Jorge J., and Houot, Roch

Abstract

Introduction:Patients with histological transformation (HT) of Waldenström macroglobulinemia (WM) who relapse or are refractory (R/R) have a poor prognosis with standard chemoimmunotherapy, notably patients who are unable to undergo high dose chemotherapy (HCT) and autologous stem cell transplantation (ASCT) or who relapse after HCT/ASCT. CD19-targeted chimeric antigen receptor (CAR) T-cell therapies can lead to durable responses in relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL) and thus may represent a promising therapeutic option in R/R transformed WM (tWM); however, data from pivotal trials is lacking. This study aimed to evaluate the efficacy and safety of anti-CD19 CAR T-cells in real-word patients with R/R tWM.

Published
2023
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48. Real World Data of Axicabtagene Ciloleucel As Second Line Therapy for Patients with Large B Cell Lymphoma: First Results of a Lysa Study from the French Descar-T Registry

Author

Brisou, Gabriel, Cartron, Guillaume, Bachy, Emmanuel, Thieblemont, Catherine, Castilla-Llorente, Cristina, Le Bras, Fabien, Gros, François-Xavier, Loschi, Michael, Houot, Roch, Dulery, Remy, Jardin, Fabrice, Joris, Magalie, Sylvain, Choquet, Morschhauser, Franck, Guidez, Stephanie, Hermine, Olivier, Drieu La Rochelle, Laurianne, Carras, Sylvain, Guffroy, Blandine, Bories, Pierre, Casasnovas, Rene-Olivier, Abraham, Julie, Le Gouill, Steven, Cony-Makhoul, Pascale, Gat, Elodie, and Tessoulin, Benoit

Abstract

Background and Significance:

Published
2023
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49. Impact of Cytoreductive Drugs upon Outcomes in a Contemporary Cohort of Adolescent and Young Adults with Essential Thrombocythemia and Polycythemia Vera

Author

Beauverd, Yan, Ianotto, Jean-Christophe, Thaw, Kyaw Htin, Sobas, Marta, Sadjadian, Parvis, Curto-Garcia, Natalia, Shih, Lee-Yung, Devos, Timothy, Krochmalczyk, Dorota, Galli, Serena, Bieniaszewska, Maria, Seferynska, Ilona, McMullin, Mary Frances, Armatys, Anna, Spalek, Adrianna, Waclaw, Joanna, Zdrenghea, Mihnea Tudor, Legros, Laurence, Girodon, Francois, Lewandowski, Krzysztof, Angona Figueras, Anna, Samuelsson, Jan, Abuin Blanco, Aitor, Cony-Makhoul, Pascale, Collins, Angela, James, Chloe, Kusec, Rajko, Lauermannova, Marie, Noya, Maria Soledad, Skowronek, Malgorzata, Szukalski, Lukasz, Szmigielska-Kaplon, Anna, Wondergem, Marielle, Dudchenko, Iryna, Gora-Tybor, Joanna, Laribi, Kamel, Kulikowska De Nalecz, Anna, Demory, Jean-Loup, Le Du, Katell, Zweegman, Sonja, Besses Raebel, Carlos, Skoda, Radek C., Giraudier, Stephane, Griesshammer, Martin, Kiladjian, Jean-Jacques, and Harrison, Claire N

Abstract

Introduction

Published
2023
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50. Characteristics, Outcomes, Tfr Rates in Young Adults with Chronic Myeloid Leukemia in the Tyrosine Kinase Inhibitor Era: A French Observational Study

Author

Legros, Laurence, Huguet, Francoise, Cony-Makhoul, Pascale, Touitou, Irit, Nicolini, Franck E., Rea, Delphine, Berger, Marc G., Uzunov, Madalina, Roy, Lydia, Escoffre Barbe, Martine, Guerci Bresler, Agnes, Etienne, Gabriel, Daguenet, Elisabeth, Penot, Amelie, Gardembas Pain, Martine, Ame, Shanti, Jourdan, Eric, Johnson-Ansah, Hyacinthe A., Coiteux, Valerie, Machet, Antoine, Lippert, Eric, Vekoff, Anne, Karsenti, Jean Michel, Morisset, Stéphane, and Mahon, Francois

Abstract

Introduction. Chronic myeloid leukemia (CML) is a myeloproliferative disorder with a median age of approximately 60 years. Our study emphasizes the fact that CML can present at younger ages. These patients have significantly different life challenges compared with older patients. The 2020 update of the ELN recommendations mentioned that“ TFR may be the main goal for any patient, irrespective of age, but it is clear that the younger the patient the stronger the case for achieving TFR”. We report here the main characteristics and TFR data in CML young adults.

Published
2023
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