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1. A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID

Author

Cesana, Daniela, Cicalese, Maria Pia, Calabria, Andrea, Merli, Pietro, Caruso, Roberta, Volpin, Monica, Rudilosso, Laura, Migliavacca, Maddalena, Barzaghi, Federica, Fossati, Claudia, Gazzo, Francesco, Pizzi, Simone, Ciolfi, Andrea, Bruselles, Alessandro, Tucci, Francesca, Spinozzi, Giulio, Pais, Giulia, Benedicenti, Fabrizio, Barcella, Matteo, Merelli, Ivan, Gallina, Pierangela, Giannelli, Stefania, Dionisio, Francesca, Scala, Serena, Casiraghi, Miriam, Strocchio, Luisa, Vinti, Luciana, Pacillo, Lucia, Draghi, Eleonora, Cesana, Marcella, Riccardo, Sara, Colantuono, Chiara, Six, Emmanuelle, Cavazzana, Marina, Carlucci, Filippo, Schmidt, Manfred, Cancrini, Caterina, Ciceri, Fabio, Vago, Luca, Cacchiarelli, Davide, Gentner, Bernhard, Naldini, Luigi, Tartaglia, Marco, Montini, Eugenio, Locatelli, Franco, and Aiuti, Alessandro

Published
2024
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3. Mechanisms of Immune Resistance

Author

Vago, Luca, Dazzi, Francesco, Sureda, Anna, editor, Corbacioglu, Selim, editor, Greco, Raffaella, editor, Kröger, Nicolaus, editor, and Carreras, Enric, editor

Published
2024
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4. Dynamics of polyclonal immuno-reconstitution after allogeneic transplant with post-transplant cyclophosphamide and letermovir

Author

Orofino, Giorgio, Xue, Elisabetta, Doglio, Matteo, Noviello, Maddalena, Tassi, Elena, Cristante, Matteo, Acerbis, Andrea, Clerici, Daniela, Farina, Francesca, Campodonico, Edoardo, Bruno, Alessandro, Piemontese, Simona, Mastaglio, Sara, Diral, Elisa, Milani, Raffaella, Ruggeri, Annalisa, Corti, Consuelo, Vago, Luca, Bonini, Chiara, Peccatori, Jacopo, Ciceri, Fabio, Lupo Stanghellini, Maria Teresa, and Greco, Raffaella

Published
2023
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6. Monitoring for virus-specific T-cell responses and viremia in allogeneic HSCT recipients: a survey from the EBMT Cellular Therapy & Immunobiology Working Party

Author

Greco, Raffaella, Hoogenboom, Jorinde D., Bonneville, Edouard F., Anagnostopoulos, Achilles, Cuoghi, Angela, Dalle, Jean-Hugues, Weissinger, Eva M., Lang, Peter, Galaverna, Federica, Martino, Massimo, Maschan, Alexei, Mauz-Körholz, Christine, Noviello, Maddalena, Passweg, Jakob, Peccatori, Jacopo, Rovira, Montserrat, Solano, Carlos, Veelken, Hendrik, Velardi, Andrea, Wagner-Drouet, Eva Maria, Zhang, Xi, Ciceri, Fabio, Bonini, Chiara, Vago, Luca, Ruggeri, Annalisa, and Chabannon, Christian

Published
2023
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7. Longitudinal single-cell profiling of chemotherapy response in acute myeloid leukemia

Author

Naldini, Matteo Maria, Casirati, Gabriele, Barcella, Matteo, Rancoita, Paola Maria Vittoria, Cosentino, Andrea, Caserta, Carolina, Pavesi, Francesca, Zonari, Erika, Desantis, Giacomo, Gilioli, Diego, Carrabba, Matteo Giovanni, Vago, Luca, Bernardi, Massimo, Di Micco, Raffaella, Di Serio, Clelia, Merelli, Ivan, Volpin, Monica, Montini, Eugenio, Ciceri, Fabio, and Gentner, Bernhard

Published
2023
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9. Neural stem cell transplantation in patients with progressive multiple sclerosis: an open-label, phase 1 study

Author

Genchi, Angela, Brambilla, Elena, Sangalli, Francesca, Radaelli, Marta, Bacigaluppi, Marco, Furlan, Roberto, Andolfo, Annapaola, Drago, Denise, Magagnotti, Cinzia, Scotti, Giulia Maria, Greco, Raffaella, Vezzulli, Paolo, Ottoboni, Linda, Bonopane, Marco, Capilupo, Daniela, Ruffini, Francesca, Belotti, Daniela, Cabiati, Benedetta, Cesana, Stefania, Matera, Giada, Leocani, Letizia, Martinelli, Vittorio, Moiola, Lucia, Vago, Luca, Panina-Bordignon, Paola, Falini, Andrea, Ciceri, Fabio, Uglietti, Anna, Sormani, Maria Pia, Comi, Giancarlo, Battaglia, Mario Alberto, Rocca, Maria A., Storelli, Loredana, Pagani, Elisabetta, Gaipa, Giuseppe, and Martino, Gianvito

Published
2023
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10. Human herpesvirus 6–specific T-cell immunity in allogeneic hematopoietic stem cell transplant recipients

Author

Noviello, Maddalena, Lorentino, Francesca, Xue, Elisabetta, Racca, Sara, Furnari, Giulia, Valtolina, Veronica, Campodonico, Edoardo, Dvir, Roee, Lupo-Stanghellini, Maria Teresa, Giglio, Fabio, Piemontese, Simona, Clerici, Daniela, Oltolini, Chiara, Tassi, Elena, Beretta, Valeria, Farina, Francesca, Mannina, Daniele, Ardemagni, Anna, Vago, Luca, Bernardi, Massimo, Corti, Consuelo, Peccatori, Jacopo, Clementi, Massimo, Ciceri, Fabio, Bonini, Chiara, and Greco, Raffaella

Published
2023
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11. Tumour Escape from CAR-T Cells

Author

Rasche, Leo, Vago, Luca, Mutis, Tuna, Kröger, Nicolaus, editor, Gribben, John, editor, Chabannon, Christian, editor, Yakoub-Agha, Ibrahim, editor, and Einsele, Hermann, editor

Published
2022
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12. Targeting MDM2 enhances antileukemia immunity after allogeneic transplantation via MHC-II and TRAIL-R1/2 upregulation

Author

Ho, Jenny N.H.G., Schmidt, Dominik, Lowinus, Theresa, Ryoo, Jeongmin, Dopfer, Elaine-Pashupati, Gonzalo Núñez, Nicolás, Costa-Pereira, Sara, Toffalori, Cristina, Punta, Marco, Fetsch, Viktor, Wertheimer, Tobias, Rittmann, Marie-Claire, Braun, Lukas M., Follo, Marie, Briere, Christelle, Vinnakota, Janaki Manoja, Langenbach, Marlene, Koppers, Felicitas, Shoumariyeh, Khalid, Engel, Helena, Rückert, Tamina, Märklin, Melanie, Holzmayer, Samuel, Illert, Anna L., Magon, Federica, Andrieux, Geoffroy, Duquesne, Sandra, Pfeifer, Dietmar, Staniek, Julian, Rizzi, Marta, Miething, Cornelius, Köhler, Natalie, Duyster, Justus, Menssen, Hans D., Boerries, Melanie, Buescher, Joerg M., Cabezas-Wallscheid, Nina, Blazar, Bruce R., Apostolova, Petya, Vago, Luca, Pearce, Erika L., Becher, Burkhard, and Zeiser, Robert

Published
2022
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13. Allogeneic hematopoietic stem cell transplantation in patients older than 65 years with acute myeloid leukemia and myelodysplastic syndrome: a 15-year experience

Author

Piemontese, Simona, Lazzari, Lorenzo, Ruggeri, Annalisa, Marcatti, Magda, Lupo Stanghellini, Maria Teresa, Giglio, Fabio, Greco, Raffaella, Lorentino, Francesca, Clerici, Daniela, Assanelli, Andrea, Farina, Francesca, Mastaglio, Sara, Xue, Elisabetta, Marktel, Sarah, Vago, Luca, Gentner, Bernhard, Secco, Chiara, Corti, Consuelo, Carrabba, Matteo Giovanni, Bernardi, Massimo, Peccatori, Jacopo, and Ciceri, Fabio

Published
2022
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14. Targeting intracellular WT1 in AML with a novel RMF-peptide-MHC-specific T-cell bispecific antibody

Author

Augsberger, Christian, Hänel, Gerulf, Xu, Wei, Pulko, Vesna, Hanisch, Lydia Jasmin, Augustin, Angelique, Challier, John, Hunt, Katharina, Vick, Binje, Rovatti, Pier Eduardo, Krupka, Christina, Rothe, Maurine, Schönle, Anne, Sam, Johannes, Lezan, Emmanuelle, Ducret, Axel, Ortiz-Franyuti, Daniela, Walz, Antje-Christine, Benz, Jörg, Bujotzek, Alexander, Lichtenegger, Felix S., Gassner, Christian, Carpy, Alejandro, Lyamichev, Victor, Patel, Jigar, Konstandin, Nikola, Tunger, Antje, Schmitz, Marc, von Bergwelt-Baildon, Michael, Spiekermann, Karsten, Vago, Luca, Jeremias, Irmela, Marrer-Berger, Estelle, Umaña, Pablo, Klein, Christian, and Subklewe, Marion

Published
2021
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15. Atypical acute myeloid leukemia-specific transcripts generate shared and immunogenic MHC class-I-associated epitopes

Author

Ehx, Grégory, Larouche, Jean-David, Durette, Chantal, Laverdure, Jean-Philippe, Hesnard, Leslie, Vincent, Krystel, Hardy, Marie-Pierre, Thériault, Catherine, Rulleau, Caroline, Lanoix, Joël, Bonneil, Eric, Feghaly, Albert, Apavaloaei, Anca, Noronha, Nandita, Laumont, Céline M., Delisle, Jean-Sébastien, Vago, Luca, Hébert, Josée, Sauvageau, Guy, Lemieux, Sébastien, Thibault, Pierre, and Perreault, Claude

Published
2021
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16. Natural killer cell alloreactivity in HLA-haploidentical hematopoietic transplantation: a study on behalf of the CTIWP of the EBMT

Author

Ruggeri, Loredana, Vago, Luca, Eikema, Diderik-Jan, de Wreede, Liesbeth C., Ciceri, Fabio, Diaz, Miguel Angel, Locatelli, Franco, Jindra, Pavel, Milone, Giuseppe, Diez-Martin, Josè Luis, Pérez-Simón, Jose Antonio, Merluzzi, Mara, Koster, Linda, van der Werf, Steffie, van Biezen, Anja, Toubert, Antoine, Nagler, Arnon, Chabannon, Christian, Bonini, Chiara, and Velardi, Andrea

Published
2021
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17. Sorafenib maintenance in FLT3-ITD mutated AML after allogeneic HCT: a real-world, single-center experience.

Author

Diral, Elisa, Furnari, Giulia, Bruno, Alessandro, Greco, Raffaella, Clerici, Daniela, Marktel, Sarah, Farina, Francesca, Mastaglio, Sara, Vago, Luca, Piemontese, Simona, Peccatori, Jacopo, Corti, Consuelo, Bernardi, Massimo, Ciceri, Fabio, and Lupo-Stanghellini, Maria Teresa

Subjects
SORAFENIB, ACUTE myeloid leukemia, HEMATOPOIETIC stem cells, STEM cell transplantation, OVERALL survival
Abstract

Despite allogeneic hematopoietic stem cell transplant (allo-HCT) and the development of novel FLT3 inhibitors in both induction (midostaurin) and in the relapsed/refractory setting (gilteritinib), FLT3-ITD mutated leukemia (FLT3- ITD+ AML) still represents a challenge for modern hematology. Sorafenib is, to this date, the only inhibitor that demonstrated efficacy in improving both progression-free and overall survival as post-HCT maintenance therapy, even if its use in this setting has not been approved so far by regulatory agencies. The aim of our study was to evaluate the feasibility, safety, and efficacy of sorafenib maintenance in preventing early relapse in FLT3-ITD+ AML after HCT in a singlecenter experience. We analyzed 26 consecutive patients who received post-HCT 2-year maintenance with sorafenib at our center between 2017 and 2023. The median time from HCT to sorafenib start was 130 days, and the median dosage was 200 mg per day. Two (8%) and three (12%) patients discontinued maintenance due to toxicity and disease relapse, respectively. Eight (31%) patients terminated the 2-year maintenance and stopped sorafenib, while 13 patients are still under treatment. Overall, 21/26 patients (81%) are alive and in stable complete remission as outlined by a 2-year disease-free survival of 83.61%. No major long-term toxicity was reported at the last follow-up. Our real-world experience supports the use of sorafenib as a feasible and effective therapeutic option in post-HCT maintenance for FLT3-ITD+ AML. [ABSTRACT FROM AUTHOR]

Published
2024
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19. TIM-3, LAG-3, or 2B4 gene disruptions increase the anti-tumor response of engineered T cells

Author

Cianciotti, Beatrice Claudia, primary, Magnani, Zulma Irene, additional, Ugolini, Alessia, additional, Camisa, Barbara, additional, Merelli, Ivan, additional, Vavassori, Valentina, additional, Potenza, Alessia, additional, Imparato, Antonio, additional, Manfredi, Francesco, additional, Abbati, Danilo, additional, Perani, Laura, additional, Spinelli, Antonello, additional, Shifrut, Eric, additional, Ciceri, Fabio, additional, Vago, Luca, additional, Di Micco, Raffaella, additional, Naldini, Luigi, additional, Genovese, Pietro, additional, Ruggiero, Eliana, additional, and Bonini, Chiara, additional

Published
2024
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20. Immune escape and immunotherapy of acute myeloid leukemia

Author

Vago, Luca and Gojo, Ivana

Subjects
Cancer treatment -- Health aspects, Nivolumab -- Health aspects, Immunotherapy -- Health aspects, Acute myelocytic leukemia -- Development and progression -- Drug therapy, Antibodies, Intelligence gathering, Toxicity, Biological markers, Myeloid leukemia, Tumors, Diseases, Health care industry
Abstract

In spite of the recent approval of new promising targeted therapies, the clinical outcome of patients with acute myeloid leukemia (AML) remains suboptimal, prompting the search for additional and synergistic therapeutic rationales. It is increasingly evident that the bone marrow immune environment of AML patients is profoundly altered, contributing to the severity of the disease but also providing several windows of opportunity to prompt or rewire a proficient antitumor immune surveillance. In this Review, we present current evidence on immune defects in AML, discuss the challenges with selective targeting of AML cells, and summarize the clinical results and immunologic insights from studies that are testing the latest immunotherapy approaches to specifically target AML cells (antibodies, cellular therapies) or more broadly reactivate antileukemia immunity (vaccines, checkpoint blockade). Given the complex interactions between AML cells and the many components of their environment, it is reasonable to surmise that the future of immunotherapy in AML lies in the rational combination of complementary immunotherapeutic strategies with chemotherapeutics or other oncogenic pathway inhibitors. Identifying reliable biomarkers of response to improve patient selection and avoid toxicities will be critical in this process., Introduction Acute myeloid leukemia (AML) is a genetically, epigenetically, and clinically heterogeneous disease characterized by accumulation and expansion of immature myeloid cells in the bone marrow (BM) and peripheral blood [...]

Published
2020
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23. NK cell recovery after haploidentical HSCT with posttransplant cyclophosphamide: dynamics and clinical implications

Author

Russo, Antonio, Oliveira, Giacomo, Berglund, Sofia, Greco, Raffaella, Gambacorta, Valentina, Cieri, Nicoletta, Toffalori, Cristina, Zito, Laura, Lorentino, Francesca, Piemontese, Simona, Morelli, Mara, Giglio, Fabio, Assanelli, Andrea, Stanghellini, Maria Teresa Lupo, Bonini, Chiara, Peccatori, Jacopo, Ciceri, Fabio, Luznik, Leo, and Vago, Luca

Published
2018
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24. Nanosphere’s Verigene® Blood Culture Assay to Detect Multidrug-Resistant Gram-Negative Bacterial Outbreak: A Prospective Study on 79 Hematological Patients in a Country with High Prevalence of Antimicrobial Resistance

Author

Greco, Raffaella, Barbanti, Maria Chiara, Mancini, Nicasio, Infurnari, Laura, Pasciuta, Renée, Forcina, Alessandra, Oltolini, Chiara, Casirati, Gabriele, Mannina, Daniele, Giglio, Fabio, Messina, Carlo, Morelli, Mara, Lorentino, Francesca, Mastaglio, Sara, Perini, Tommaso, Vago, Luca, Scarpellini, Paolo, Carrabba, Matteo Giovanni, Stanghellini, Maria Teresa Lupo, Marktel, Sarah, Assanelli, Andrea, Bernardi, Massimo, Corti, Consuelo, Peccatori, Jacopo, Clementi, Massimo, and Ciceri, Fabio

Published
2019
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25. Beneficial role of CD8+ T-cell reconstitution after HLA-haploidentical stem cell transplantation for high-risk acute leukaemias: results from a clinico-biological EBMT registry study mostly in the T-cell-depleted setting

Author

Bondanza, Attilio, Ruggeri, Loredana, Noviello, Maddalena, Eikema, Dirk-Jan, Bonini, Chiara, Chabannon, Christian, van der Werf, Steffie, van Biezen, Anja, de Wreede, Liesbeth C., Crucitti, Lara, Vago, Luca, Merluzzi, Mara, Massei, Maria Speranza, Veelken, Hendrik, Koc, Yener, Bader, Peter, Gruhn, Bernd, Locatelli, Franco, Ciceri, Fabio, Toubert, Antoine, Velardi, Andrea, and on behalf of the EBMT Cell Therapy and Immunobiology Working Party

Published
2019
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26. Mother Donors Improve Outcomes after HLA Haploidentical Transplantation: A Study by the Cellular Therapy and Immunobiology Working Party of the European Society for Blood and Marrow Transplantation

Author

Ruggeri, Loredana, Eikema, Dirk-Jan, Bondanza, Attilio, Noviello, Maddalena, van Biezen, Anja, de Wreede, Liesbeth C., Crucitti, Lara, Vago, Luca, Ciardelli, Sara, Bader, Peter, Koc, Yener, Locatelli, Franco, Veelken, Joan H., Gruhn, Bernd, Evans, Pamela, Chabannon, Christian, Toubert, Antoine, and Velardi, Andrea

Published
2022
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27. Supplementary Materials and methods from Preexisting Immunity Drives the Response to Neoadjuvant Chemotherapy in Esophageal Adenocarcinoma

Author

Arbore, Giuseppina, primary, Albarello, Luca, primary, Bucci, Gabriele, primary, Punta, Marco, primary, Cossu, Andrea, primary, Fanti, Lorella, primary, Maurizio, Aurora, primary, Di Mauro, Francesco, primary, Bilello, Vito, primary, Arrigoni, Gianluigi, primary, Bonfiglio, Silvia, primary, Biancolini, Donatella, primary, Puccetti, Francesco, primary, Elmore, Ugo, primary, Vago, Luca, primary, Cascinu, Stefano, primary, Tonon, Giovanni, primary, Rosati, Riccardo, primary, Casorati, Giulia, primary, and Dellabona, Paolo, primary

Published
2023
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28. Data from Preexisting Immunity Drives the Response to Neoadjuvant Chemotherapy in Esophageal Adenocarcinoma

Author

Arbore, Giuseppina, primary, Albarello, Luca, primary, Bucci, Gabriele, primary, Punta, Marco, primary, Cossu, Andrea, primary, Fanti, Lorella, primary, Maurizio, Aurora, primary, Di Mauro, Francesco, primary, Bilello, Vito, primary, Arrigoni, Gianluigi, primary, Bonfiglio, Silvia, primary, Biancolini, Donatella, primary, Puccetti, Francesco, primary, Elmore, Ugo, primary, Vago, Luca, primary, Cascinu, Stefano, primary, Tonon, Giovanni, primary, Rosati, Riccardo, primary, Casorati, Giulia, primary, and Dellabona, Paolo, primary

Published
2023
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29. Supplementary Tables from Preexisting Immunity Drives the Response to Neoadjuvant Chemotherapy in Esophageal Adenocarcinoma

Author

Arbore, Giuseppina, primary, Albarello, Luca, primary, Bucci, Gabriele, primary, Punta, Marco, primary, Cossu, Andrea, primary, Fanti, Lorella, primary, Maurizio, Aurora, primary, Di Mauro, Francesco, primary, Bilello, Vito, primary, Arrigoni, Gianluigi, primary, Bonfiglio, Silvia, primary, Biancolini, Donatella, primary, Puccetti, Francesco, primary, Elmore, Ugo, primary, Vago, Luca, primary, Cascinu, Stefano, primary, Tonon, Giovanni, primary, Rosati, Riccardo, primary, Casorati, Giulia, primary, and Dellabona, Paolo, primary

Published
2023
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30. Supplementary Figures from Preexisting Immunity Drives the Response to Neoadjuvant Chemotherapy in Esophageal Adenocarcinoma

Author

Arbore, Giuseppina, primary, Albarello, Luca, primary, Bucci, Gabriele, primary, Punta, Marco, primary, Cossu, Andrea, primary, Fanti, Lorella, primary, Maurizio, Aurora, primary, Di Mauro, Francesco, primary, Bilello, Vito, primary, Arrigoni, Gianluigi, primary, Bonfiglio, Silvia, primary, Biancolini, Donatella, primary, Puccetti, Francesco, primary, Elmore, Ugo, primary, Vago, Luca, primary, Cascinu, Stefano, primary, Tonon, Giovanni, primary, Rosati, Riccardo, primary, Casorati, Giulia, primary, and Dellabona, Paolo, primary

Published
2023
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31. Phase II trial of hypomethylating agent combined with nivolumab for acute myeloid leukaemia relapse after allogeneic haematopoietic cell transplantation—Immune signature correlates with response

Author

Apostolova, Petya, primary, Kreutmair, Stefanie, additional, Toffalori, Cristina, additional, Punta, Marco, additional, Unger, Susanne, additional, Burk, Ann‐Cathrin, additional, Wehr, Claudia, additional, Maas‐Bauer, Kristina, additional, Melchinger, Wolfgang, additional, Haring, Eileen, additional, Hoefflin, Rouven, additional, Shoumariyeh, Khalid, additional, Hupfer, Valerie, additional, Lauer, Eliza Maria, additional, Duquesne, Sandra, additional, Lowinus, Theresa, additional, Gonzalo Núñez, Nicolás, additional, Alberti, Chiara, additional, da Costa Pereira, Sara, additional, Merten, Carla Helena, additional, Power, Laura, additional, Weiss, Matthias, additional, Böke, Caroline, additional, Pfeifer, Dietmar, additional, Marks, Reinhard, additional, Bertz, Hartmut, additional, Wäsch, Ralph, additional, Ihorst, Gabriele, additional, Gentner, Bernhard, additional, Duyster, Justus, additional, Boerries, Melanie, additional, Andrieux, Geoffroy, additional, Finke, Juergen, additional, Becher, Burkhard, additional, Vago, Luca, additional, and Zeiser, Robert, additional

Published
2023
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32. P436: CUTTING-EDGE TRANSCRIPTOMICS TO IDENTIFY LEUKEMIA-INTRINSIC AND -EXTRINSIC CORRELATES OF IMMUNE ESCAPE AND POST-TRANSPLANTATION RELAPSE

Author

Edoardo Rovatti, Pier, primary, Punta, Marco, additional, Maria Naldini, Matteo, additional, Barcella, Matteo, additional, Zito, Laura, additional, Giovanni Carrabba, Matteo, additional, Bernardi, Massimo, additional, Peccatori, Jacopo, additional, Merelli, Ivan, additional, Gentner, Bernhard, additional, Ciceri, Fabio, additional, Vago, Luca, additional, and Toffalori, Cristina, additional

Published
2023
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33. P429: THERAPY-INDUCED SENESCENCE TRIGGERS UPREGULATION OF ANTIGEN PRESENTATION VIA PRC2 INHIBITION AND PROMOTES ANTI-TUMOUR IMMUNITY IN ACUTE MYELOID LEUKAEMIA

Author

Gilioli, Diego, primary, Fusco, Simona, additional, Tavella, Teresa, additional, Giannetti, Kety, additional, Conti, Anastasia, additional, Antonella, Santoro, additional, Carsana, Edoardo, additional, Beretta, Stefano, additional, Schönlein, Martin, additional, Gambacorta, Valentina, additional, Aletti, Federico, additional, Giovanni Carrabba, Matteo, additional, Bonini, Chiara, additional, Ciceri, Fabio, additional, Merelli, Ivan, additional, Vago, Luca, additional, Schmitt, Clemens A., additional, and DI Micco, Raffaella, additional

Published
2023
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34. PB2426: SORAFENIB MAINTENANCE TAILORED TO FLT3-MUTATED AML AFTER ALLOGENEIC HCT IS HIGHLY FEASIBLE AND EFFECTIVE: A REAL-LIFE EXPERIENCE

Author

Diral, Elisa, primary, Furnari, Giulia, additional, Farina, Francesca, additional, Clerici, Daniela, additional, Marktel, Sarah, additional, Greco, Raffaella, additional, Catalano, Gloria, additional, Bruno, Alessandro, additional, Campodonico, Edoardo, additional, Xue, Elisabetta, additional, Mastaglio, Sara, additional, Vago, Luca, additional, Stefano Luca Bernardi, Massimo, additional, Carrabba, Matteo Giovanni, additional, Corti, Consuelo, additional, Peccatori, Jacopo, additional, Ciceri, Fabio, additional, and Teresa Lupo Stanghellini, Maria, additional

Published
2023
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35. NY-ESO-1 TCR single edited stem and central memory T cells to treat multiple myeloma without graft-versus-host disease

Author

Mastaglio, Sara, Genovese, Pietro, Magnani, Zulma, Ruggiero, Eliana, Landoni, Elisa, Camisa, Barbara, Schiroli, Giulia, Provasi, Elena, Lombardo, Angelo, Reik, Andreas, Cieri, Nicoletta, Rocchi, Martina, Oliveira, Giacomo, Escobar, Giulia, Casucci, Monica, Gentner, Bernhard, Spinelli, Antonello, Mondino, Anna, Bondanza, Attilio, Vago, Luca, Ponzoni, Maurilio, Ciceri, Fabio, Holmes, Michael C., Naldini, Luigi, and Bonini, Chiara

Published
2017
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36. Impact of predicted HLA class I immunopeptidome on viral reservoir in a cohort of people living with HIV in Italy.

Author

Rovatti, Pier Edoardo, Muccini, Camilla, Punta, Marco, Galli, Laura, Mainardi, Ilaria, Ponta, Giacomo, Vago, Luca Aldo Edoardo, and Castagna, Antonella

Subjects
HIV-positive persons, CYTOTOXIC T cells, KILLER cell receptors, GAG proteins, ANTIRETROVIRAL agents
Abstract

The class I HLA genotype has been widely recognized as a factor influencing HIV disease progression in treatment‐naïve subjects. However, little is known regarding its role in HIV disease course and how it influences the size of the viral reservoir once anti‐retroviral therapy (ART) is started. Here, leveraging on cutting‐edge bioinformatic tools, we explored the relationship between HLA class I and the HIV reservoir in a cohort of 90 people living with HIV (PLWH) undergoing ART and who achieved viral suppression. Analysis of HLA allele distribution among patients with high and low HIV reservoir allowed us to document a predominant role of HLA‐B and ‐C genes in regulating the size of HIV reservoir. We then focused on the analysis of HIV antigen (Ag) repertoire, by investigating immunogenetic parameters such as the degree of homozygosity, HLA evolutionary distance and Ag load. In particular, we used two different bioinformatic algorithms, NetMHCpan and MixMHCpred, to predict HLA presentation of immunogenic HIV‐derived peptides and identified HLA‐B*57:01 and HLA‐B*58:01 among the highest ranking HLAs in terms of total load, suggesting that their previously reported protective role against HIV disease progression might be linked to a more effective viral recognition and presentation to Cytotoxic T lymphocytes (CTLs). Further, we speculated that some peptide‐HLA complexes, including those produced by the interaction between HLA‐B*27 and the HIV Gag protein, might be particularly relevant for the efficient regulation of HIV replication and containment of the HIV reservoir. Last, we provide evidence of a possible synergistic effect between the CCR5 ∆32 mutation and Ag load in controlling HIV reservoir. [ABSTRACT FROM AUTHOR]

Published
2024
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37. Posttransplantation Cyclophosphamide- and Sirolimus-Based Graft-Versus-Host-Disease Prophylaxis in Allogeneic Stem Cell Transplant

Author

Greco, Raffaella, Lorentino, Francesca, Albanese, Serena, Lupo Stanghellini, Maria Teresa, Giglio, Fabio, Piemontese, Simona, Clerici, Daniela, Lazzari, Lorenzo, Marcatti, Magda, Mastaglio, Sara, Xue, Elisabetta, Farina, Francesca, Pavesi, Francesca, Assanelli, Andrea, Carrabba, Matteo G., Marktel, Sarah, Vago, Luca, Bonini, Chiara, Corti, Consuelo, Bernardi, Massimo, Ciceri, Fabio, and Peccatori, Jacopo

Published
2021
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38. Sorafenib promotes graft-versus-leukemia activity in mice and humans through IL-15 production in FLT3-ITD-mutant leukemia cells

Author

Mathew, Nimitha R, Baumgartner, Francis, Braun, Lukas, O'Sullivan, David, Thomas, Simone, Waterhouse, Miguel, Müller, Tony A, Hanke, Kathrin, Taromi, Sanaz, Apostolova, Petya, Illert, Anna L, Melchinger, Wolfgang, Duquesne, Sandra, Schmitt-Graeff, Annette, Osswald, Lena, Yan, Kai-Li, Weber, Arnim, Tugues, Sonia, Spath, Sabine, Pfeifer, Dietmar, Follo, Marie, Claus, Rainer, Lübbert, Michael, Rummelt, Christoph, Bertz, Hartmut, Wäsch, Ralph, Haag, Johanna, Schmidts, Andrea, Schultheiss, Michael, Bettinger, Dominik, Thimme, Robert, Ullrich, Evelyn, Tanriver, Yakup, Vuong, Giang Lam, Arnold, Renate, Hemmati, Philipp, Wolf, Dominik, Ditschkowski, Markus, Jilg, Cordula, Wilhelm, Konrad, Leiber, Christian, Gerull, Sabine, Halter, Jörg, Lengerke, Claudia, Pabst, Thomas, Schroeder, Thomas, Kobbe, Guido, Rösler, Wolf, Doostkam, Soroush, Meckel, Stephan, Stabla, Kathleen, Metzelder, Stephan K, Halbach, Sebastian, Brummer, Tilman, Hu, Zehan, Dengjel, Joern, Hackanson, Björn, Schmid, Christoph, Holtick, Udo, Scheid, Christof, Spyridonidis, Alexandros, Stölzel, Friedrich, Ordemann, Rainer, Müller, Lutz P, Sicre-de-Fontbrune, Flore, Ihorst, Gabriele, Kuball, Jürgen, Ehlert, Jan E, Feger, Daniel, Wagner, Eva-Maria, Cahn, Jean-Yves, Schnell, Jacqueline, Kuchenbauer, Florian, Bunjes, Donald, Chakraverty, Ronjon, Richardson, Simon, Gill, Saar, Kröger, Nicolaus, Ayuk, Francis, Vago, Luca, Ciceri, Fabio, Müller, Antonia M, Kondo, Takeshi, Teshima, Takanori, Klaeger, Susan, Kuster, Bernhard, Weisdorf, Daniel, van der Velden, Walter, Dörfel, Daniela, Bethge, Wolfgang, Hilgendorf, Inken, Hochhaus, Andreas, Andrieux, Geoffroy, Börries, Melanie, Busch, Hauke, Magenau, John, Reddy, Pavan, Labopin, Myriam, Antin, Joseph H, Henden, Andrea S, Hill, Geoffrey R, Kennedy, Glen A, Bar, Merav, Sarma, Anita, McLornan, Donal, Mufti, Ghulam, Oran, Betul, Rezvani, Katayoun, Shah, Omid, Negrin, Robert S, Nagler, Arnon, Prinz, Marco, Burchert, Andreas, Neubauer, Andreas, Beelen, Dietrich, Mackensen, Andreas, von Bubnoff, Nikolas, Herr, Wolfgang, Becher, Burkhard, Socié, Gerard, Caligiuri, Michael A, Ruggiero, Eliana, Bonini, Chiara, Häcker, Georg, Duyster, Justus, Finke, Jürgen, Pearce, Erika, Blazar, Bruce R, and Zeiser, Robert

Subjects
Interleukin-15 -- Health aspects, Gene mutation -- Health aspects, Sorafenib -- Patient outcomes, Gene expression -- Health aspects, Acute myelocytic leukemia -- Genetic aspects -- Development and progression -- Drug therapy, Biological sciences, Health
Abstract

Individuals with acute myeloid leukemia (AML) harboring an internal tandem duplication (ITD) in the gene encoding Fms-related tyrosine kinase 3 (FLT3) who relapse after allogeneic hematopoietic cell transplantation (allo-HCT) have a 1-year survival rate below 20%. We observed that sorafenib, a multitargeted tyrosine kinase inhibitor, increased IL-15 production by FLT3-ITD[sup.+] leukemia cells. This synergized with the allogeneic CD8[sup.+] T cell response, leading to long-term survival in six mouse models of FLT3-ITD[sup.+] AML. Sorafenib-related IL-15 production caused an increase in CD8[sup.+]CD107a[sup.+]IFN-[gamma][sup.+] T cells with features of longevity (high levels of Bcl-2 and reduced PD-1 levels), which eradicated leukemia in secondary recipients. Mechanistically, sorafenib reduced expression of the transcription factor ATF4, thereby blocking negative regulation of interferon regulatory factor 7 (IRF7) activation, which enhanced IL-15 transcription. Both IRF7 knockdown and ATF4 overexpression in leukemia cells antagonized sorafenib-induced IL-15 production in vitro. Human FLT3-ITD[sup.+] AML cells obtained from sorafenib responders following sorafenib therapy showed increased levels of IL-15, phosphorylated IRF7, and a transcriptionally active IRF7 chromatin state. The mitochondrial spare respiratory capacity and glycolytic capacity of CD8[sup.+] T cells increased upon sorafenib treatment in sorafenib responders but not in nonresponders. Our findings indicate that the synergism of T cells and sorafenib is mediated via reduced ATF4 expression, causing activation of the IRF7-IL-15 axis in leukemia cells and thereby leading to metabolic reprogramming of leukemia-reactive T cells in humans. Therefore, sorafenib treatment has the potential to contribute to an immune-mediated cure of FLT3-ITD-mutant AML relapse, an otherwise fatal complication after allo-HCT., Author(s): Nimitha R Mathew [1, 2]; Francis Baumgartner [1]; Lukas Braun [1]; David O'Sullivan [3]; Simone Thomas [4]; Miguel Waterhouse [1]; Tony A Müller [1]; Kathrin Hanke [1, 2]; Sanaz [...]

Published
2018
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40. Immune monitoring in allogeneic hematopoietic stem cell transplant recipients: a survey from the EBMT-CTIWP

Author

Greco, Raffaella, Ciceri, Fabio, Noviello, Maddalena, Bondanza, Attilio, Vago, Luca, Oliveira, Giacomo, Peccatori, Jacopo, Cieri, Nicoletta, Ruggeri, Annalisa, Koehl, Ulrike, Fleischhauer, Katharina, Rocha, Vanderson, Dazzi, Francesco, van der Werf, Steffie Maria, Eikema, Dirk-Jan, Terwel, Sofie Rosanne, Kuball, Jürgen, Toubert, Antoine, Chabannon, Christian, Bonini, Chiara, and on behalf of EBMT Cellular Therapy and Immunobiology Working Party (CTIWP)

Published
2018
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41. Allelic HLA Matching and Pair Origin Are Favorable Prognostic Factors for Unrelated Hematopoietic Stem Cell Transplantation in Neoplastic Hematologic Diseases: An Italian Analysis by the Gruppo Italiano Trapianto di Cellule Staminali e Terapie Cellulari, Italian Bone Marrow Donor Registry, and Associazione Italiana di Immunogenetica e Biologia dei Trapianti

Author

Picardi, Alessandra, Sacchi, Nicoletta, Miotti, Valeria, Lorentino, Francesca, Oldani, Elena, Rambaldi, Alessandro, Sessa, Mariarosaria, Bruno, Benedetto, Cerno, Michela, Vago, Luca, Bernasconi, Paolo, Arcese, William, Benedetti, Fabio, Pioltelli, Pietro, Russo, Domenico, Farina, Lucia, Fagioli, Franca, Guidi, Stefano, Saporiti, Giorgia, Zallio, Francesco, Chiusolo, Patrizia, Borghero, Carlo, Papalinetti, Gabriele, La Rocca, Ursula, Milone, Giuseppe, Lamparelli, Teresa, Carella, Angelo M., Luppi, Mario, Olivieri, Attilio, Martino, Massimo, Carluccio, Paola, Celeghini, Ivana, Andreani, Marco, Gallina, Anna M., Patriarca, Francesca, Pollichieni, Simona, Mammoliti, Sonia, Miccichè, Silvia, Mangione, Ilaria, Ciceri, Fabio, and Bonifazi, Francesca

Published
2021
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42. Adjuvant role of SeptiFast to improve the diagnosis of sepsis in a large cohort of hematological patients

Author

Greco, Raffaella, Barbanti, Maria Chiara, Mancini, Nicasio, Crucitti, Lara, Oltolini, Chiara, Forcina, Alessandra, Lorentino, Francesca, Vago, Luca, Messina, Carlo, Clerici, Daniela, Morelli, Mara, Giglio, Fabio, Lupo Stanghellini, Maria Teresa, Infurnari, Laura, Carrabba, Matteo G., Marktel, Sarah, Assanelli, Andrea, Scarpellini, Paolo, Bernardi, Massimo, Peccatori, Jacopo, Corti, Consuelo, Clementi, Massimo, and Ciceri, Fabio

Published
2018
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43. Real-World Validation of Molecular International Prognostic Scoring System for Myelodysplastic Syndromes

Author

Sauta, Elisabetta, primary, Robin, Marie, additional, Bersanelli, Matteo, additional, Travaglino, Erica, additional, Meggendorfer, Manja, additional, Zhao, Lin-Pierre, additional, Caballero Berrocal, Juan Carlos, additional, Sala, Claudia, additional, Maggioni, Giulia, additional, Bernardi, Massimo, additional, Di Grazia, Carmen, additional, Vago, Luca, additional, Rivoli, Giulia, additional, Borin, Lorenza, additional, D'Amico, Saverio, additional, Tentori, Cristina Astrid, additional, Ubezio, Marta, additional, Campagna, Alessia, additional, Russo, Antonio, additional, Mannina, Daniele, additional, Lanino, Luca, additional, Chiusolo, Patrizia, additional, Giaccone, Luisa, additional, Voso, Maria Teresa, additional, Riva, Marta, additional, Oliva, Esther Natalie, additional, Zampini, Matteo, additional, Riva, Elena, additional, Nibourel, Olivier, additional, Bicchieri, Marilena, additional, Bolli, Niccolo’, additional, Rambaldi, Alessandro, additional, Passamonti, Francesco, additional, Savevski, Victor, additional, Santoro, Armando, additional, Germing, Ulrich, additional, Kordasti, Shahram, additional, Santini, Valeria, additional, Diez-Campelo, Maria, additional, Sanz, Guillermo, additional, Sole, Francesc, additional, Kern, Wolfgang, additional, Platzbecker, Uwe, additional, Ades, Lionel, additional, Fenaux, Pierre, additional, Haferlach, Torsten, additional, Castellani, Gastone, additional, and Della Porta, Matteo Giovanni, additional

Published
2023
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44. Data from Integrated Multiomic Profiling Identifies the Epigenetic Regulator PRC2 as a Therapeutic Target to Counteract Leukemia Immune Escape and Relapse

Author

Gambacorta, Valentina, primary, Beretta, Stefano, primary, Ciccimarra, Martina, primary, Zito, Laura, primary, Giannetti, Kety, primary, Andrisani, Angela, primary, Gnani, Daniela, primary, Zanotti, Lucia, primary, Oliveira, Giacomo, primary, Carrabba, Matteo Giovanni, primary, Cittaro, Davide, primary, Merelli, Ivan, primary, Ciceri, Fabio, primary, Di Micco, Raffaella, primary, and Vago, Luca, primary

Published
2023
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45. Integrating biological HLA-DPB1 mismatch models to predict survival after unrelated hematopoietic cell transplantation

Author

Ruggeri, Annalisa, De Wreede, Liesbeth C, Müller, Carlheinz R, Crivello, Pietro, Bonneville, Edouard F, Petersdorf, Effie W, Socié, Gerard, Dubois, Valérie, Niittyvuopio, Riitta, Peräsaari, Juha, Yakoub-Agha, Ibrahim, Cornelissen, Jan J, Wieten, Lotte, Gedde-Dahl, Tobias, Forcade, Edouard, Crawley, Charles R, Marsh, Steven G E, Gandemer, Virginie, Tholouli, Eleni, Bulabois, Claude-Eric, Huynh, Anne, Choi, Goda, Deconinck, Eric, Itäla-Remes, Maija, Lenhoff, Stig, Bengtsson, Mats, Johansson, Jan-Erik, Van Gorkom, Gwendolyn, Hoogenboom, Jorinde D, Vago, Luca, Rocha, Vanderson, Bonini, Chiara, Chabannon, Christian, Fleischhauer, Katharina, Ruggeri, Annalisa, De Wreede, Liesbeth C, Müller, Carlheinz R, Crivello, Pietro, Bonneville, Edouard F, Petersdorf, Effie W, Socié, Gerard, Dubois, Valérie, Niittyvuopio, Riitta, Peräsaari, Juha, Yakoub-Agha, Ibrahim, Cornelissen, Jan J, Wieten, Lotte, Gedde-Dahl, Tobias, Forcade, Edouard, Crawley, Charles R, Marsh, Steven G E, Gandemer, Virginie, Tholouli, Eleni, Bulabois, Claude-Eric, Huynh, Anne, Choi, Goda, Deconinck, Eric, Itäla-Remes, Maija, Lenhoff, Stig, Bengtsson, Mats, Johansson, Jan-Erik, Van Gorkom, Gwendolyn, Hoogenboom, Jorinde D, Vago, Luca, Rocha, Vanderson, Bonini, Chiara, Chabannon, Christian, and Fleischhauer, Katharina

Published
2023
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46. Real-World Validation of Molecular International Prognostic Scoring System for Myelodysplastic Syndromes

Author

Sauta, E, Robin, M, Bersanelli, M, Travaglino, E, Meggendorfer, M, Zhao, L, Caballero Berrocal, J, Sala, C, Maggioni, G, Bernardi, M, Di Grazia, C, Vago, L, Rivoli, G, Borin, L, D'Amico, S, Tentori, C, Ubezio, M, Campagna, A, Russo, A, Mannina, D, Lanino, L, Chiusolo, P, Giaccone, L, Voso, M, Riva, M, Oliva, E, Zampini, M, Riva, E, Nibourel, O, Bicchieri, M, Bolli, N, Rambaldi, A, Passamonti, F, Savevski, V, Santoro, A, Germing, U, Kordasti, S, Santini, V, Diez-Campelo, M, Sanz, G, Sole, F, Kern, W, Platzbecker, U, Ades, L, Fenaux, P, Haferlach, T, Castellani, G, Della Porta, M, Sauta, Elisabetta, Robin, Marie, Bersanelli, Matteo, Travaglino, Erica, Meggendorfer, Manja, Zhao, Lin-Pierre, Caballero Berrocal, Juan Carlos, Sala, Claudia, Maggioni, Giulia, Bernardi, Massimo, Di Grazia, Carmen, Vago, Luca, Rivoli, Giulia, Borin, Lorenza, D'Amico, Saverio, Tentori, Cristina Astrid, Ubezio, Marta, Campagna, Alessia, Russo, Antonio, Mannina, Daniele, Lanino, Luca, Chiusolo, Patrizia, Giaccone, Luisa, Voso, Maria Teresa, Riva, Marta, Oliva, Esther Natalie, Zampini, Matteo, Riva, Elena, Nibourel, Olivier, Bicchieri, Marilena, Bolli, Niccolo', Rambaldi, Alessandro, Passamonti, Francesco, Savevski, Victor, Santoro, Armando, Germing, Ulrich, Kordasti, Shahram, Santini, Valeria, Diez-Campelo, Maria, Sanz, Guillermo, Sole, Francesc, Kern, Wolfgang, Platzbecker, Uwe, Ades, Lionel, Fenaux, Pierre, Haferlach, Torsten, Castellani, Gastone, Della Porta, Matteo Giovanni, Sauta, E, Robin, M, Bersanelli, M, Travaglino, E, Meggendorfer, M, Zhao, L, Caballero Berrocal, J, Sala, C, Maggioni, G, Bernardi, M, Di Grazia, C, Vago, L, Rivoli, G, Borin, L, D'Amico, S, Tentori, C, Ubezio, M, Campagna, A, Russo, A, Mannina, D, Lanino, L, Chiusolo, P, Giaccone, L, Voso, M, Riva, M, Oliva, E, Zampini, M, Riva, E, Nibourel, O, Bicchieri, M, Bolli, N, Rambaldi, A, Passamonti, F, Savevski, V, Santoro, A, Germing, U, Kordasti, S, Santini, V, Diez-Campelo, M, Sanz, G, Sole, F, Kern, W, Platzbecker, U, Ades, L, Fenaux, P, Haferlach, T, Castellani, G, Della Porta, M, Sauta, Elisabetta, Robin, Marie, Bersanelli, Matteo, Travaglino, Erica, Meggendorfer, Manja, Zhao, Lin-Pierre, Caballero Berrocal, Juan Carlos, Sala, Claudia, Maggioni, Giulia, Bernardi, Massimo, Di Grazia, Carmen, Vago, Luca, Rivoli, Giulia, Borin, Lorenza, D'Amico, Saverio, Tentori, Cristina Astrid, Ubezio, Marta, Campagna, Alessia, Russo, Antonio, Mannina, Daniele, Lanino, Luca, Chiusolo, Patrizia, Giaccone, Luisa, Voso, Maria Teresa, Riva, Marta, Oliva, Esther Natalie, Zampini, Matteo, Riva, Elena, Nibourel, Olivier, Bicchieri, Marilena, Bolli, Niccolo', Rambaldi, Alessandro, Passamonti, Francesco, Savevski, Victor, Santoro, Armando, Germing, Ulrich, Kordasti, Shahram, Santini, Valeria, Diez-Campelo, Maria, Sanz, Guillermo, Sole, Francesc, Kern, Wolfgang, Platzbecker, Uwe, Ades, Lionel, Fenaux, Pierre, Haferlach, Torsten, Castellani, Gastone, and Della Porta, Matteo Giovanni

Abstract

Purpose: Myelodysplastic syndromes (MDS) are heterogeneous myeloid neoplasms in which a risk-adapted treatment strategy is needed. Recently, a new clinical-molecular prognostic model, the Molecular International Prognostic Scoring System (IPSS-M) was proposed to improve the prediction of clinical outcome of the currently available tool (Revised International Prognostic Scoring System [IPSS-R]). We aimed to provide an extensive validation of IPSS-M. Methods: A total of 2,876 patients with primary MDS from the GenoMed4All consortium were retrospectively analyzed. Results: IPSS-M improved prognostic discrimination across all clinical end points with respect to IPSS-R (concordance was 0.81 v 0.74 for overall survival and 0.89 v 0.76 for leukemia-free survival, respectively). This was true even in those patients without detectable gene mutations. Compared with the IPSS-R based stratification, the IPSS-M risk group changed in 46% of patients (23.6% and 22.4% of subjects were upstaged and downstaged, respectively).In patients treated with hematopoietic stem cell transplantation (HSCT), IPSS-M significantly improved the prediction of the risk of disease relapse and the probability of post-transplantation survival versus IPSS-R (concordance was 0.76 v 0.60 for overall survival and 0.89 v 0.70 for probability of relapse, respectively). In high-risk patients treated with hypomethylating agents (HMA), IPSS-M failed to stratify individual probability of response; response duration and probability of survival were inversely related to IPSS-M risk.Finally, we tested the accuracy in predicting IPSS-M when molecular information was missed and we defined a minimum set of 15 relevant genes associated with high performance of the score. Conclusion: IPSS-M improves MDS prognostication and might result in a more effective selection of candidates to HSCT. Additional factors other than gene mutations can be involved in determining HMA sensitivity. The definition of a minimum set of relevan

Published
2023

47. Neural stem cell transplantation in patients with progressive multiple sclerosis: an open-label, phase 1 study

Author

Genchi, A, Brambilla, E, Sangalli, F, Radaelli, M, Bacigaluppi, M, Furlan, R, Andolfo, A, Drago, D, Magagnotti, C, Scotti, G, Greco, R, Vezzulli, P, Ottoboni, L, Bonopane, M, Capilupo, D, Ruffini, F, Belotti, D, Cabiati, B, Cesana, S, Matera, G, Leocani, L, Martinelli, V, Moiola, L, Vago, L, Panina-Bordignon, P, Falini, A, Ciceri, F, Uglietti, A, Sormani, M, Comi, G, Battaglia, M, Rocca, M, Storelli, L, Pagani, E, Gaipa, G, Martino, G, Genchi, Angela, Brambilla, Elena, Sangalli, Francesca, Radaelli, Marta, Bacigaluppi, Marco, Furlan, Roberto, Andolfo, Annapaola, Drago, Denise, Magagnotti, Cinzia, Scotti, Giulia Maria, Greco, Raffaella, Vezzulli, Paolo, Ottoboni, Linda, Bonopane, Marco, Capilupo, Daniela, Ruffini, Francesca, Belotti, Daniela, Cabiati, Benedetta, Cesana, Stefania, Matera, Giada, Leocani, Letizia, Martinelli, Vittorio, Moiola, Lucia, Vago, Luca, Panina-Bordignon, Paola, Falini, Andrea, Ciceri, Fabio, Uglietti, Anna, Sormani, Maria Pia, Comi, Giancarlo, Battaglia, Mario Alberto, Rocca, Maria A, Storelli, Loredana, Pagani, Elisabetta, Gaipa, Giuseppe, Martino, Gianvito, Genchi, A, Brambilla, E, Sangalli, F, Radaelli, M, Bacigaluppi, M, Furlan, R, Andolfo, A, Drago, D, Magagnotti, C, Scotti, G, Greco, R, Vezzulli, P, Ottoboni, L, Bonopane, M, Capilupo, D, Ruffini, F, Belotti, D, Cabiati, B, Cesana, S, Matera, G, Leocani, L, Martinelli, V, Moiola, L, Vago, L, Panina-Bordignon, P, Falini, A, Ciceri, F, Uglietti, A, Sormani, M, Comi, G, Battaglia, M, Rocca, M, Storelli, L, Pagani, E, Gaipa, G, Martino, G, Genchi, Angela, Brambilla, Elena, Sangalli, Francesca, Radaelli, Marta, Bacigaluppi, Marco, Furlan, Roberto, Andolfo, Annapaola, Drago, Denise, Magagnotti, Cinzia, Scotti, Giulia Maria, Greco, Raffaella, Vezzulli, Paolo, Ottoboni, Linda, Bonopane, Marco, Capilupo, Daniela, Ruffini, Francesca, Belotti, Daniela, Cabiati, Benedetta, Cesana, Stefania, Matera, Giada, Leocani, Letizia, Martinelli, Vittorio, Moiola, Lucia, Vago, Luca, Panina-Bordignon, Paola, Falini, Andrea, Ciceri, Fabio, Uglietti, Anna, Sormani, Maria Pia, Comi, Giancarlo, Battaglia, Mario Alberto, Rocca, Maria A, Storelli, Loredana, Pagani, Elisabetta, Gaipa, Giuseppe, and Martino, Gianvito

Abstract

Innovative pro-regenerative treatment strategies for progressive multiple sclerosis (PMS), combining neuroprotection and immunomodulation, represent an unmet need. Neural precursor cells (NPCs) transplanted in animal models of multiple sclerosis have shown preclinical efficacy by promoting neuroprotection and remyelination by releasing molecules sustaining trophic support and neural plasticity. Here we present the results of STEMS, a prospective, therapeutic exploratory, non-randomized, open-label, single-dose-finding phase 1 clinical trial (NCT03269071, EudraCT 2016-002020-86), performed at San Raffaele Hospital in Milan, Italy, evaluating the feasibility, safety and tolerability of intrathecally transplanted human fetal NPCs (hfNPCs) in 12 patients with PMS (with evidence of disease progression, Expanded Disability Status Scale ≥6.5, age 18–55 years, disease duration 2–20 years, without any alternative approved therapy). The safety primary outcome was reached, with no severe adverse reactions related to hfNPCs at 2-year follow-up, clearly demonstrating that hfNPC therapy in PMS is feasible, safe and tolerable. Exploratory secondary analyses showed a lower rate of brain atrophy in patients receiving the highest dosage of hfNPCs and increased cerebrospinal fluid levels of anti-inflammatory and neuroprotective molecules. Although preliminary, these results support the rationale and value of future clinical studies with the highest dose of hfNPCs in a larger cohort of patients.

Published
2023

48. Human Herpesvirus 6 Infection Following Haploidentical Transplantation: Immune Recovery and Outcome

Author

Greco, Raffaella, Crucitti, Lara, Noviello, Maddalena, Racca, Sara, Mannina, Daniele, Forcina, Alessandra, Lorentino, Francesca, Valtolina, Veronica, Rolla, Serena, Dvir, Roee, Morelli, Mara, Giglio, Fabio, Barbanti, Maria Chiara, Lupo Stanghellini, Maria Teresa, Oltolini, Chiara, Vago, Luca, Scarpellini, Paolo, Assanelli, Andrea, Carrabba, Matteo G., Marktel, Sarah, Bernardi, Massimo, Corti, Consuelo, Clementi, Massimo, Peccatori, Jacopo, Bonini, Chiara, and Ciceri, Fabio

Published
2016
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49. Matching for the nonconventional MHC-I MICA gene significantly reduces the incidence of acute and chronic GVHD

Author

Carapito, Raphael, Jung, Nicolas, Kwemou, Marius, Untrau, Meiggie, Michel, Sandra, Pichot, Angélique, Giacometti, Gaëlle, Macquin, Cécile, Ilias, Wassila, Morlon, Aurore, Kotova, Irina, Apostolova, Petya, Schmitt-Graeff, Annette, Cesbron, Anne, Gagne, Katia, Oudshoorn, Machteld, van der Holt, Bronno, Labalette, Myriam, Spierings, Eric, Picard, Christophe, Loiseau, Pascale, Tamouza, Ryad, Toubert, Antoine, Parissiadis, Anne, Dubois, Valérie, Lafarge, Xavier, Maumy-Bertrand, Myriam, Bertrand, Frédéric, Vago, Luca, Ciceri, Fabio, Paillard, Catherine, Querol, Sergi, Sierra, Jorge, Fleischhauer, Katharina, Nagler, Arnon, Labopin, Myriam, Inoko, Hidetoshi, von dem Borne, Peter A., Kuball, Jürgen, Ota, Masao, Katsuyama, Yoshihiko, Michallet, Mauricette, Lioure, Bruno, Peffault de Latour, Régis, Blaise, Didier, Cornelissen, Jan J., Yakoub-Agha, Ibrahim, Claas, Frans, Moreau, Philippe, Milpied, Noël, Charron, Dominique, Mohty, Mohamad, Zeiser, Robert, Socié, Gérard, and Bahram, Seiamak

Published
2016
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50. Spontaneous remission of choroidal involvement by chronic myelomonocytic leukemia: a case report.

Author

Diral, Elisa, Catalano, Gloria, Cicinelli, Maria Vittoria, Distefano, Andrea, Mastaglio, Sara, Vago, Luca, Lupo Stanghellini, Maria Teresa, Bernardi, Massimo, Ponzoni, Maurilio, Ciceri, Fabio, and Carrabba, Matteo G.

Subjects
CHRONIC leukemia, STEM cell transplantation, ACUTE myeloid leukemia, BLOOD diseases, THERAPEUTICS, VISION disorders
Abstract

Chronic myelomonocytic leukemia (CMML) is a rare hematological disorder characterized by variable risk of evolution to acute myeloid leukemia; to date, allogeneic stem cell transplantation is the only curative treatment. We report a case of choroidal involvement in a woman affected by CMML and presenting only with visual impairment. The patient was initially evaluated for an intensive therapeutic approach, but after biopsy the ocular lesion spontaneously regressed. Thus a "watch and wait" strategy was preferred. One year and a half after initial diagnosis, the patient is alive, with stable hematological disease and without any ocular involvement. Therefore, a close, not invasive follow up could be useful to tailor treatment for patients affected by single ocular lesions in CMML. [ABSTRACT FROM AUTHOR]

Published
2024
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