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1. Pathophysiologic implications of elevated prevalence of hereditary alpha-tryptasemia in all mastocytosis subtypes

Author

Polivka, Laura, Madrange, Marine, Bulai-Livideanu, Cristina, Barete, Stéphane, Ballul, Thomas, Neuraz, Antoine, Greco, Celine, Agopian, Julie, Brenet, Fabienne, Dubreuil, Patrice, Burdet, Charles, Lemal, Richard, Tournilhac, Olivier, Terriou, Louis, Launay, David, Bouillet, Laurence, Gourguechon, Clément, Damaj, Ghandi, Frenzel, Laurent, Meni, Cécile, Bouktit, Hassiba, Collange, Anne Florence, Gaudy-Marqueste, Caroline, Gousseff, Marie, Le Mouel, Edwige, Hamidou, Mohamed, Neel, Antoine, Ranta, Dana, Jaussaud, Roland, Guilpain, Philippe, Canioni, Danielle, Molina, Thierry Jo, Bruneau, Julie, Lhermitte, Ludovic, Garcelon, Nicolas, Javier, Rose-Marie, Pelletier, Fabien, Castelain, Florence, Retornaz, Frederique, Cabrera, Quentin, Zunic, Patricia, Gourin, Marie Pierre, Wierzbicka-Hainaut, Ewa, Viallard, Jean François, Lavigne, Christian, Hoarau, Cyrille, Durieu, Isabelle, Heiblig, Maël, Dimicoli-Salazar, Sophie, Torregrosa-Diaz, Jose Miguel, Soria, Angèle, Arock, Michel, Lortholary, Olivier, Bodemer, Christine, Hermine, Olivier, and Rossignol, Julien

Published
2024
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2. CPX-351 in higher risk myelodysplastic syndrome and chronic myelomonocytic leukaemia: a multicentre, single-arm, phase 2 study

Author

Peterlin, Pierre, Le Bris, Yannick, Turlure, Pascal, Chevallier, Patrice, Ménard, Audrey, Gourin, Marie-Pierre, Dumas, Pierre-Yves, Thepot, Sylvain, Berceanu, Ana, Park, Sophie, Hospital, Marie-Anne, Cluzeau, Thomas, Bouzy, Simon, Torregrosa-Diaz, Jose-Miguel, Drevon, Louis, Sapena, Rosa, Chermat, Fatiha, Ades, Lionel, Dimicoli-Salazar, Sophie, Chevret, Sylvie, Béné, Marie-Christine, and Fenaux, Pierre

Published
2023
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3. Vaccinations avant et après greffe de cellules hématopoïétiques autologues chez les patients atteints de maladies auto-immunes : propositions du groupe Maladies Auto-immunes et Thérapie Cellulaire de la SFGM-TC

Author

Maria, Alexandre Thibault Jacques, Campidelli, Arnaud, Castilla-Llorente, Cristina, Lansiaux, Pauline, Marjanovic, Zora, Pugnet, Grégory, Torregrosa-Diaz, Jose Miguel, Terriou, Louis, Algayres, Jean-Pierre, Urbain, Fanny, Yakoub-Agha, Ibrahim, and Farge, Dominique

Published
2023
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4. Indications de l’autogreffe de cellules hématopoïétiques dans la Maladie de Crohn : recommandations de la Société francophone de greffe de moelle et de thérapie cellulaire

Author

Puyade, Mathieu, Treton, Xavier, Alric, Laurent, Badoglio, Manuela, Castilla Llorente, Cristina, Fotsing, Ginette, Marjanovic, Zora, Rovira, Montserrat, Terriou, Louis, Torregrosa Díaz Jose, Miguel, Tran-Minh, My-Linh, Allez, Matthieu, Pugnet, Grégory, Laharie, David, Yakoub-Agha, Ibrahim, and Farge, Dominique

Published
2020
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5. Ropeginterferon alfa-2b versus standard therapy for polycythaemia vera (PROUD-PV and CONTINUATION-PV): a randomised, non-inferiority, phase 3 trial and its extension study

Author

Bauer, Franz, Berbec, Nicoleta, Besses Raebel, Carlos, Borbenyi, Zita, Bumbea, Horia, Buxhofer-Ausch, Veronika, Calbecka, Malgorzata, Cayssials-Caylus, Emilie, Cazzola, Mario, Cerna, Olga, Cucuianu, Andrei, Dima, Delia Monica, Forjan, Ernst, Gheorghita, Emanuil, Greil, Richard, Hatalova, Antonia, Hrubisko, Mikulas, Jakucs, Janos, Kaplan, Polina, Klymenko, Sergiy, Koschmieder, Steffen, Lazaroiu, Mihaela, Lysa, Tamila, Masliak, Zvenyslava, Masszi, Tamas, Mihaylov, Georgi, Myasnikov, Alexander, Platzbecker, Uwe, Puyade, Mathieu, Rey, Jerome, Roy, Lydia, Schwarz, Jiri, Skotnicki, Aleksander, Sokolova, Irina, Soroka-Wojtaszko, Maria, Starzak-Gwozdz, Jolanta, Stoeva, Vera, Torregrosa-Diaz, Jose Miguel, Vallova, Anna, Volodicheva, Elena, Warzocha, Krzysztof, Willenbacher, Ella, Wolf, Dominik, Gisslinger, Heinz, Klade, Christoph, Georgiev, Pencho, Krochmalczyk, Dorota, Gercheva-Kyuchukova, Liana, Egyed, Miklos, Rossiev, Viktor, Dulicek, Petr, Illes, Arpad, Pylypenko, Halyna, Sivcheva, Lylia, Mayer, Jiri, Yablokova, Vera, Krejcy, Kurt, Grohmann-Izay, Barbara, Hasselbalch, Hans C, Kralovics, Robert, and Kiladjian, Jean-Jacques

Published
2020
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6. Indications de l’autogreffe de cellules souches hématopoïétiques dans la polyradiculonévrite inflammatoire démyélinisante chronique : recommandations de la Société francophone de greffe de moelle et de thérapie cellulaire (SFGM-TC)

Author

Puyade, Mathieu, Labeyrie, Céline, Badoglio, Manuela, Cintas, Pascal, Guenounou, Sarah, Lansiaux, Pauline, Marjanovic, Zora, Nicolas, Guillaume, Pomies, Amélie, Terriou, Louis, Torregrosa Diaz, Jose-Miguel, Baron, Clément, Castilla Llorente, Cristina, Yakoub-Agha, Ibrahim, and Farge, Dominique

Published
2020
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7. Decitabine Versus Hydroxyurea for Advanced Proliferative Chronic Myelomonocytic Leukemia: Results of a Randomized Phase III Trial Within the EMSCO Network

Author

Itzykson, Raphael, primary, Santini, Valeria, additional, Thepot, Sylvain, additional, Ades, Lionel, additional, Chaffaut, Cendrine, additional, Giagounidis, Aristoteles, additional, Morabito, Margot, additional, Droin, Nathalie, additional, Lübbert, Michael, additional, Sapena, Rosa, additional, Nimubona, Stanislas, additional, Goasguen, Jean, additional, Wattel, Eric, additional, Zini, Gina, additional, Torregrosa Diaz, Jose Miguel, additional, Germing, Ulrich, additional, Pelizzari, Anna Maria, additional, Park, Sophie, additional, Jaekel, Nadja, additional, Metzgeroth, Georgia, additional, Onida, Francesco, additional, Navarro, Robert, additional, Patriarca, Andrea, additional, Stamatoullas, Aspasia, additional, Götze, Katharina, additional, Puttrich, Martin, additional, Mossuto, Sandra, additional, Solary, Eric, additional, Gloaguen, Silke, additional, Chevret, Sylvie, additional, Chermat, Fatiha, additional, Platzbecker, Uwe, additional, and Fenaux, Pierre, additional

Published
2022
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8. Molecular Response Analysis By High Throughput Sequencing in Higher Risk Myelodysplastic Syndrome (HR-MDS) Treated Intensively with CPX-351

Author

Le Bris, Yannick, primary, Bouzy, Simon, additional, Ménard, Audrey, additional, Turlure, Pascal, additional, Chevallier, Patrice, additional, Gourin, Marie-Pierre, additional, Dumas, Pierre-Yves, additional, Thepot, Sylvain, additional, Berceanu, Anna, additional, Park, Sophie, additional, Hospital, Marie-Anne, additional, Cluzeau, Thomas, additional, Torregrosa Diaz, Jose-Miguel, additional, Devron, Louis, additional, Sapena, Rosa, additional, Chermat, Fatiha, additional, Dimicoli Salazar, Sophie, additional, Béné, Marie C, additional, Fenaux, Pierre, additional, and Peterlin, Pierre, additional

Published
2022
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9. Decitabine Versus Hydroxyurea for Advanced Proliferative Chronic Myelomonocytic Leukemia: Results of a Randomized Phase III Trial Within the EMSCO Network.

Author

Itzykson, Raphael, Santini, Valeria, Thepot, Sylvain, Ades, Lionel, Chaffaut, Cendrine, Giagounidis, Aristoteles, Morabito, Margot, Droin, Nathalie, Lübbert, Michael, Sapena, Rosa, Nimubona, Stanislas, Goasguen, Jean, Wattel, Eric, Zini, Gina, Torregrosa Diaz, Jose Miguel, Germing, Ulrich, Pelizzari, Anna Maria, Park, Sophie, Jaekel, Nadja, and Metzgeroth, Georgia

Published
2023
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11. CPX 351 As First Line Treatment in Higher Risk MDS. a Phase II Trial By the GFM

Author

Peterlin, Pierre, primary, Turlure, Pascal, additional, Chevallier, Patrice, additional, Gourin, Marie-Pierre, additional, Dumas, Pierre-Yves, additional, Thepot, Sylvain, additional, Berceanu, Anna, additional, Park, Sophie, additional, Hospital, Marie Anne, additional, Cluzeau, Thomas, additional, Torregrosa Diaz, Jose Miguel, additional, Devron, Louis, additional, Chevret, Sylvie, additional, Bene, Marie C, additional, Le Bris, Yannick, additional, Sapena, Rosa, additional, Chermat, Fatiha, additional, Dimicoli-Salazar, Sophie, additional, and Fenaux, Pierre, additional

Published
2021
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12. Risk Stratification of Elderly Patients with Newly Diagnosed Acute Myeloid Leukemia Unfit for Intensive Chemotherapy

Author

Gardeney, Helene, primary, Torregrosa Diaz, Jose Miguel, additional, Basle, Caroline, additional, Cayssials, Emilie, additional, Bouyer, Sabrina, additional, Moya, Niels, additional, Gruchet, Cécile, additional, Sabirou, Florence, additional, Bobin, Arthur, additional, Levy, Anthony, additional, Nsiala, Laly, additional, Cailly, Laura, additional, Vonfeld, Mathilde, additional, Desmier, Deborah, additional, Leleu, Xavier, additional, and Gallego Hernanz, Maria Pilar, additional

Published
2021
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13. Efficacy and Safety of Luspatercept +/- Erythropoiesis-Stimulating Agent (ESA) in Patients with Myelodysplastic Syndromes with Ring Sideroblasts (MDS-RS): A French Multicenter Prospective Real-Life Registry

Author

Comont, Thibault, D'Aveni, Maud, Schenone, Laurence, Torregrosa-Díaz, Jose Miguel, Aguinaga, Lorea, Pardo, Juan, Sebert, Marie, Visanica, Sorin, Santana, Clemence, Le Clech, Lenaig, Stamatoullas, Aspasia, Thepot, Sylvain, Daguindau, Etienne, Beziat, Guillaume, Botin, Teresa, Dimicoli, Sophie, Park, Sophie, Paubelle, Etienne, Slama, Bohrane, Willems, Lise, Debarri, Houria, Gyan, Emmanuel, Calmettes, Claire, Rauzy, Odile, Chermat, Fatiha, Fontenay, Michaela, Ades, Lionel, and Fenaux, Pierre

Published
2023
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14. FLT3 Ligand Kinetic Profile Predicts Response to Treatment and Event-Free Survival (EFS) in Adults with High-Risk MDS/CMML Receiving CPX-351: A GFM Study

Author

Peterlin, Pierre, Gaschet, Joëlle, Turlure, Pascal, Gourin, Marie-Pierre, Dumas, Pierre-Yves, Thepot, Sylvain, Berceanu, Ana, Park, Sophie, Hospital, Marie Anne, Cluzeau, Thomas, Torregrosa-Diaz, Jose-Miguel, Drevon, Louis, Sapena, Rosa, Chermat, Fatiha, Dimicoli-Salazar, Sophie, Jullien, Maxime, Fenaux, Pierre, and Chevallier, Patrice

Published
2023
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15. Prognostic Characterization of Advanced Mastocytosis Patients Treated with Midostaurin According to Diagnosis and Mutation-Adjusted Risk Score: A Nation-Wide Ceremast Study of 170 Patients

Author

Heiblig, Mael, Gourguechon, Clement, Guilpain, Philippe, Livideanu, Cristina Bulai, Barete, Stéphane, Agopian, Julie, Brenet, Fabienne, Dubreuil, Patrice, Lemal, Richard, Tournilhac, Olivier, Terriou, Louis, Launay, David, Bouillet, Laurence, Damaj, Gandhi Laurent, Ballul, Thomas, Greco, Céline, Polivka, Laura, Frenzel, Laurent, Meni, Cecile, Bouktit, Hassiba, Gaudy-Marqueste, Caroline, Gousseff, Marie, Le Mouel, Edwige, Neel, Antoine, Ranta, Dana, Jaussaud, Roland, Molina, Thierry Jo, Bruneau, Julie, Lhermitte, Ludovic, Temple, Marie, Kosmider, Olivier, Javier, Rose-Marie, Pelletier, Fabien, Castelain, Florence, Retornaz, Frédérique, Cabrera, Quentin, Zunic, Patricia, Gourin, Marie-Pierre, Wierzbicka-Hainaut, Ewa, Viallard, Jean-Francois, Lavigne, Christian, Hoarau, Cyrille, Durieu, Isabelle, Dimicoli-Salazar, Sophie, Torregrosa-Diaz, Jose-Miguel, Soria, Angele, Arock, Michel, Bodemer, Christine, Lortholary, Olivier, Hermine, Olivier, and Rossignol, Julien

Published
2023
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17. Decitabine Versus Hydroxyurea for Advanced Proliferative CMML: Results of the Emsco Randomized Phase 3 Dacota Trial

Author

Itzykson, Raphael, primary, Santini, Valeria, additional, Chaffaut, Cendrine, additional, Lionel, Ades, additional, Thepot, Sylvain, additional, Giagounidis, Aristoteles, additional, Morabito, Margot, additional, Droin, Nathalie, additional, Luebbert, Michael, additional, Sapena, Rosa, additional, Nimubona, Stanislas, additional, Goasguen, Jean E., additional, Wattel, Eric, additional, Zini, Gina, additional, Torregrosa Diaz, Jose Miguel, additional, Germing, Ulrich, additional, Pelizzari, Anna Maria, additional, Park, Sophie, additional, Jaekel, Nadja, additional, Metzgeroth, Georgia, additional, Onida, Francesco, additional, Navarro, Robert, additional, Patriarca, Andrea, additional, Stamatoulas Bastard, Aspasia, additional, Puttrich, Martin, additional, Mossuto, Sandra, additional, Solary, Eric, additional, Gloaguen, Silke, additional, Chevret, Sylvie, additional, Chermat, Fatiha, additional, Platzbecker, Uwe, additional, and Fenaux, Pierre, additional

Published
2020
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18. A Molecular-Based Response Prediction Model to Romiplostim in Patients with Lower-Risk Myelodysplastic Syndrome and Severe Thrombocytopenia

Author

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

Published
2020
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19. Ropeginterferon alfa-2b versus standard therapy for polycythaemia vera (PROUD-PV and CONTINUATION-PV): a randomised, non-inferiority, phase 3 trial and its extension study

Author

Gisslinger, Heinz, primary, Klade, Christoph, additional, Georgiev, Pencho, additional, Krochmalczyk, Dorota, additional, Gercheva-Kyuchukova, Liana, additional, Egyed, Miklos, additional, Rossiev, Viktor, additional, Dulicek, Petr, additional, Illes, Arpad, additional, Pylypenko, Halyna, additional, Sivcheva, Lylia, additional, Mayer, Jiri, additional, Yablokova, Vera, additional, Krejcy, Kurt, additional, Grohmann-Izay, Barbara, additional, Hasselbalch, Hans C, additional, Kralovics, Robert, additional, Kiladjian, Jean-Jacques, additional, Bauer, Franz, additional, Berbec, Nicoleta, additional, Besses Raebel, Carlos, additional, Borbenyi, Zita, additional, Bumbea, Horia, additional, Buxhofer-Ausch, Veronika, additional, Calbecka, Malgorzata, additional, Cayssials-Caylus, Emilie, additional, Cazzola, Mario, additional, Cerna, Olga, additional, Cucuianu, Andrei, additional, Dima, Delia Monica, additional, Forjan, Ernst, additional, Gheorghita, Emanuil, additional, Greil, Richard, additional, Hatalova, Antonia, additional, Hrubisko, Mikulas, additional, Jakucs, Janos, additional, Kaplan, Polina, additional, Klymenko, Sergiy, additional, Koschmieder, Steffen, additional, Lazaroiu, Mihaela, additional, Lysa, Tamila, additional, Masliak, Zvenyslava, additional, Masszi, Tamas, additional, Mihaylov, Georgi, additional, Myasnikov, Alexander, additional, Platzbecker, Uwe, additional, Puyade, Mathieu, additional, Rey, Jerome, additional, Roy, Lydia, additional, Schwarz, Jiri, additional, Skotnicki, Aleksander, additional, Sokolova, Irina, additional, Soroka-Wojtaszko, Maria, additional, Starzak-Gwozdz, Jolanta, additional, Stoeva, Vera, additional, Torregrosa-Diaz, Jose Miguel, additional, Vallova, Anna, additional, Volodicheva, Elena, additional, Warzocha, Krzysztof, additional, Willenbacher, Ella, additional, and Wolf, Dominik, additional

Published
2020
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21. Biomarkers of Response to Romiplostim in Patients with Lower-Risk Myelodysplastic Syndrome (MDS) and Thrombocytopenia - Results of the Europe Trial By the Emsco Network

Author

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

Published
2019
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22. Vaccinations, incluant des propositions concernant le SARS-Cov-2, avant et après greffe de cellules hématopoïétiques autologues chez les patients atteints de maladies auto-immunes : propositions du groupe Maladies Auto-immunes et Thérapie Cellulaire de la Société Francophone de Greffe de Moelle et de Thérapie Cellulaire (MATHEC-SFGM-TC)

Author

Maria, Alexandre Thibault Jacques, Campidelli, Arnaud, Castilla-Llorente, Cristina, Lansiaux, Pauline, Marjanovic, Zora, Pugnet, Grégory, Torregrosa-Diaz, Jose Miguel, Terriou, Louis, Algayres, Jean-Pierre, Urbain, Fanny, Yakoub-Agha, Ibrahim, and Farge, Dominique

Abstract

Dans une démarche visant à uniformiser les procédures d’allogreffe et d’autogreffe de cellules hématopoïétiques, la Société Francophone de Greffe de Moelle et Thérapie cellulaire (SFGM-TC) a organisé les douzièmes ateliers d’harmonisation des pratiques en septembre 2021 à Lille. En l’absence de recommandation nationale ou internationale spécifique, le groupe Maladies Auto-Immunes et Thérapie Cellulaire (MATHEC) a rédigé des propositions de vaccinations encadrant les procédures de greffe de cellules hématopoïétiques autologues pour maladie auto-immune, incluant des propositions spécifiques au SARS-Cov-2.

Published
2022
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23. Characteristics and outcomes associated with CD2 and CD25 expression on bone marrow mast cells in patients with systemic mastocytosis.

Author

Rossignol J, Georgin-Lavialle S, Canioni D, Beganovic O, Brouzes C, Fain O, Heiblig M, Gourguechon C, Guilpain P, Bulai-Livideanu C, Barete S, Agopian J, Brenet F, Dubreuil P, Lemal R, Tournilhac O, Terriou L, Launay D, Bouillet L, Chatain C, Damaj G, Ballul T, Greco C, Polivka L, Frenzel L, Meni C, Bouktit H, Benabou D, Devin C, Gaudy-Marqueste C, Gousseff M, Le Mouel E, Neel A, Ranta D, Jaussaud R, Jo Molina T, Bruneau J, Javier RM, Pelletier F, Castelain F, Retornaz F, Cabrera Q, Zunic P, Gourin MP, Wierzbicka-Hainaut E, Viallard JF, Lavigne C, Hoarau C, Durieu I, Dimicoli-Salazar S, Torregrosa-Diaz JM, Duval A, Garcelon N, Lespinasse J, Soria A, Chantran Y, Arock M, Bodemer C, Lortholary O, Asnafi V, Hermine O, and Lhermitte L

Abstract

Not available.

Published
2024
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24. Comparison of prognostic scores according to WHO classification in 170 patients with advanced mastocytosis and C-finding treated with midostaurin.

Author

Heiblig M, Gourguechon C, Guilpain P, Bulai-Livideanu C, Barete S, Chantran Y, Agopian J, Brenet F, Dubreuil P, Lespinasse J, Lemal R, Tournilhac O, Terriou L, Launay D, Bouillet L, Chatain C, Damaj G, Ballul T, Greco C, Polivka L, Frenzel L, Meni C, Bouktit H, Benabou D, Gaudy-Marqueste C, Gousseff M, Le Mouel E, Neel A, Ranta D, Jaussaud R, Molina TJ, Bruneau J, Villarese P, Lhermitte L, Maouche-Chrétien L, Temple M, Kosmider O, Javier RM, Pelletier F, Castelain F, Retornaz F, Cabrera Q, Zunic P, Gourin MP, Wierzbicka-Hainaut E, Viallard JF, Lavigne C, Hoarau C, Durieu I, Dimicoli-Salazar S, Torregrosa-Diaz JM, Wemeau M, Soria A, Arock M, Bodemer C, Lortholary O, Hermine O, and Rossignol J

Subjects
Humans, Male, Female, Middle Aged, Aged, Retrospective Studies, Prognosis, Adult, World Health Organization, Aged, 80 and over, Protein Kinase Inhibitors therapeutic use, Leukemia, Mast-Cell drug therapy, Hematologic Neoplasms drug therapy, Hematologic Neoplasms mortality, Staurosporine analogs & derivatives, Staurosporine therapeutic use, Mastocytosis, Systemic drug therapy, Mastocytosis, Systemic mortality, Mastocytosis, Systemic classification, Mastocytosis, Systemic diagnosis
Abstract

Advanced systemic mastocytosis (AdvSM) encompasses heterogeneous mastocytosis subtypes and is associated with poor outcomes. Although midostaurin was the first tyrosine kinase inhibitor to be approved for AdvSM patients, long-lasting responses are limited. The mutation-Adjusted Risk Score (MARS), the International Prognostic Scoring System for mastocytosis (IPSM) and the Global Prognostic Score for Systemic Mastocytosis (GPSM) have been established to characterize the outcomes of patients with overall AdvSM. However, given the outcome's dependency on the AdvSM subtype, prognostic characterization within each subtype is critical. We aimed to study the predictive ability using Harrell's concordance index of prognostic scores according to the AdvSM subtype. We conducted a nationwide retrospective study using the French mastocytosis reference center's registry and included all midostaurin-treated patients with C finding. Overall, 170 patients were identified: 46 aggressive SM (ASM), 11 mast cell leukemia (MCL), and 113 SM with associated hematological neoplasm (SM-AHN). All risk scores improved their discriminative value for overall survival (OS) when combined with the AdvSM subtype. The best predictive value was for adjusted MARS (C-index = 0.689), followed by GPSM (C-index = 0.677) and IPSM (C-index = 0.618). In a multivariable analysis, MARS stratification and the AdvSM subtype were both prognostic for OS. Accordingly, five subgroups of patients with AdvSM and a different median OS were identified: 9.9 months for MCL, 24 months for intermediate/high-risk SM-AHN, 33 months for intermediate/high-risk ASM, 58 months for low-risk SM-AHN and was not reached for low-risk ASM (p < 0.001). The AdvSM subtype and the MARS are the most predictive of OS and should prompt specific management., (© 2024 The Author(s). American Journal of Hematology published by Wiley Periodicals LLC.)

Published
2024
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25. Efficacy and safety of mammalian target of rapamycin inhibitors in systemic mastocytosis: A nationwide French pilot study.

Author

Moraly J, Rossignol J, Rouzaud C, Gabas T, Bouktit H, Lhermitte L, Canioni D, Fraitag S, Bruneau J, Barete S, Suarez F, Ballul T, Meni C, Polivka L, Terriou L, Launay D, Bouillet L, Gaudy-Marqueste C, Gousseff M, Le Mouel E, Neel A, Ranta D, Jaussaud R, Guilpain P, Frenzel L, Agopian J, Dubreuil P, Greco C, Dimicoli-Salazar S, Heiblig M, Gourguechon C, Tournilhac O, Javier RM, Castelain F, Cabrera Q, Gourin MP, Wierzbicka-Hainaut E, Torregrosa-Diaz JM, Bulai C, Lavigne C, Hoarau C, Arock M, Damaj G, Lortholary O, and Hermine O

Subjects
Humans, Pilot Projects, Female, Male, Middle Aged, Adult, France, Aged, Proto-Oncogene Proteins c-kit genetics, Proto-Oncogene Proteins c-kit antagonists & inhibitors, Everolimus therapeutic use, Everolimus adverse effects, Treatment Outcome, TOR Serine-Threonine Kinases antagonists & inhibitors, Aged, 80 and over, Mastocytosis, Systemic drug therapy, Sirolimus therapeutic use, Sirolimus adverse effects, MTOR Inhibitors therapeutic use
Abstract

Systemic mastocytosis (SM) corresponds to a rare and heterogeneous spectrum of diseases characterized by the accumulation of atypical mast cells (MCs). Advanced mastocytosis (Adv-SM) is associated with poor survival; in contrast, patients with non-advanced SM (non-Adv-SM) usually have a normal life expectancy but may experience poor quality of life. Despite recent therapeutic progress including tyrosine kinase inhibitors, new treatment options are needed for refractory and/or intolerant patients with both severely symptomatic and Adv-SM. In vitro, the mTOR pathway is activated in MCs from patients bearing the KIT D816V mutation. Furthermore, rapamycin induces the apoptosis of KIT D816V MCs selectively. In this nationwide study, we report the outcomes of patients diagnosed with SM and treated with a mammalian target of rapamycin inhibitor (imTOR) within the French National Reference Center for mastocytosis (CEREMAST). All patients registered were relapsing, treatment-refractory, or ineligible for other cytoreductive therapy. Non-Adv-SM patients received imTOR as a monotherapy (rapamycin/everolimus), and Adv-SM patients received imTOR as a monotherapy or in combination with cytarabine. The objective response rate (ORR) in non-Adv-SM was 60% (partial response in 40% and major response in 20%), including reductions in skin involvement, mediator release symptoms, and serum tryptase. In the Adv-SM group, the ORR was 20% (including one major response and one partial response, both in patients with a KIT D816V mutation), which enabled a successful bridge to allogeneic stem cell transplantation in one patient. Our results suggest that imTOR treatment has potential benefits in patients with SM harboring a KIT D816V mutation., (© 2024 The Authors. American Journal of Hematology published by Wiley Periodicals LLC.)

Published
2024
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