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51. Prenatal diagnosis of a large abdominal cyst – Recommendations and management

Author

Ramy Abramsky, Admit Ben Haim, Kyla Marks, Esther Maor, Dan Tirosh, Reli Hershkovitz, Ilan Shelef, Dragan Kravarusic, and Antonella Nahom Di Veroli

Subjects
Prenatal diagnosis, Abdominal cyst, Mesenchymal hamartoma, Pediatrics, RJ1-570, Surgery, RD1-811
Abstract

This case report describes a twin fetus diagnosed in the third trimester with an enlarging abdominal mass that was confirmed on fetal magnetic resonance imaging (MRI) to be a hepatic mesenchymal hamartoma (HMH) without involvement of the placenta. Serial ultrasonography demonstrated progressive enlargement of the mass and infant was delivered at 33 + 6 weeks gestational age following maternal complications. On the fifth day, at laparotomy, a huge mass connected to the lower portion of the liver was completely resected. Microscopic evaluation confirmed a hepatic mesenchymal hamartoma. Postoperative recovery was uneventful and the infant was discharged at 4 weeks. The antenatal diagnosis of the hepatic mesenchymal hamartoma by fetal MRI and subsequent follow-up by serial ultrasonography emphasizes the importance of combining these 2 modalities for optimal management of the pregnancy to allow a favorable outcome.

Published
2018
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52. Efficacy and Safety of Luspatercept in Adult Patients with Transfusion-Dependent Anemia Due to Very Low, Low and Intermediate Risk Myelodysplastic Syndromes (MDS) with Ring Sideroblasts, Who Had an Unsatisfactory Response to or Are Ineligible for Erythropoietin-Based Therapy: A Retrospective Multicenter Study By Fondazione Italiana Sindromi Mielodisplastiche (FiSiM ETS)

Author

Luca Lanino, Prassede Salutari, Alessandra Perego, Bruno Fattizzo, Marta Riva, Marta Ubezio, Pellegrino Musto, Daniela Cilloni, Esther Natalie Oliva, Maria Teresa Voso, Anna Maria Pelizzari, Antonella Poloni, Isabella Capodanno, Chiara Elena, Claudio Fozza, Fabrizio Pane, Massimo Breccia, Marco De Gobbi, Francesco Di Bassiano, Daniela Barraco, Elena Crisà, Dario Ferrero, Chiara Frairia, Antonella Vaccarino, Davide Griguolo, Stefania Paolini, Martina Quintini, Mariarosaria Sessa, Mauro Turrini, Monica Bocchia, Nicola Di Renzo, Elisa Diral, Cristina Foli, Alfredo Molteni, Ubaldo Occhini, Giulia Rivoli, Carmine Selleri, Roberto Bono, Anna Calvisi, Andrea Castelli, Eros Di Bona, Ambra Di Veroli, Luana Fianchi, Sara Galimberti, Daniele Grimaldi, Monia Marchetti, Marianna Norata, Alessandro Rambaldi, Ilaria Tanasi, Patrizia Tosi, Ilaria Naldi, Valeria Santini, and Matteo G. Della Porta

Subjects
Immunology, Cell Biology, Hematology, Biochemistry
Published
2022
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53. Paediatric appendicitis

Author

van Amstel, Paul, el Ghazzaoui, Ali, Hall, Nigel J., Wester, Tomas, Morini, Francesco, van der Lee, Johanna H., Singer, Georg, Pierro, Agostino, Zani, Augusto, Gorter, Ramon R., Metzelder, Martin L., Langer, Sophie, Mitul, Ashrarur R., Karim, Sabbir, Islam, Nazmul, Poupalou, Anna, Miserez, Marc, Willems, Edward, Ortolan, Erika V. P., de Arruda Lourenção, Pedro Luiz Toledo, Ellebæk, Mark Bremholm, Petersen, Susanna, Suominen, Janne, Pakarinen, Mikko, Schmitt, Françoise, Bonnard, Arnaud, Montalva, Louise, Martin, Garance, di Veroli, Antonella Nahom, Assi, Zaki, Pini-Prato, Alessio, Falconi, Ilaria, Alberti, Daniele, Boroni, Giovanni, Montanaro, Beatrice, Morabito, Antonino, Zulli, Andrea, Coletta, Riccardo, Romeo, Carmelo, Antonelli, Enrica, Nascimben, Francesca, Gamba, Piergiorgio, Sgro, Alberto, Raffaele, Alessandro, Ruffoli, Maria, Aloi, Ivan, Frediani, Simone, Gasparella, Marco, Midrio, Paola, Kakar, Mohit, Nah, Shireen A., Gunarasa, Yohesuwary, Risteski, Toni, Cvetanoska, Vesna Naunova, Jovcheski, Lazo, Stensrud, Kjetil Juul, Røkkum, Henrik, Næss, P. l Aksel, Vaz-Silva, Aline, Forte, Joana Patena, Morais, Pedro F., Pereira, Joana Queirós, Antunovic, Sanja Sindjic, Lukac, Marija, Arnold, Marion, Ichino, Martina, Bernstein, Andrew Victor, le Roux, Hettie, Martinez, Leopoldo, Delgado-Miguel, Carlos, Rodriguez, Paolo Bragagnini, Arroyo, Paula Salcedo, Ruiz, Yurema Gonzalez, Svenningsson, Anna, Derikx, Joep P. M., Bakx, Roel, Wijnen, Rene M. H., Keyzer, Claudia M. G., Zijp, Gerda W., Huurman, E. A., van Gemert, Wim, Theeuws, Olivier, de Blaauw, Ivo, Botden, Sanne M. B. I., Joosten, Maja, Boerma, Evert-Jan, Schweitzer, Donald, Uzunlu, Osman, Jester, Ingo, Martin, Ben, Patel, Hetal N., Fouad, Dina, Lam, Christine, Cappiello, Clint D., Lopez, Carla, Natale, Veronica, Lee, Emily, Graduate School, AGEM - Endocrinology, metabolism and nutrition, ARD - Amsterdam Reproduction and Development, General Paediatrics, APH - Methodology, APH - Quality of Care, AGEM - Amsterdam Gastroenterology Endocrinology Metabolism, Paediatric Surgery, Pediatric surgery, Amsterdam Reproduction & Development (AR&D), and Other Research

Subjects
Appendicitis/surgery, radiodiagnosis, retrospective study, Article, computer assisted tomography, coronavirus disease 2019, length of stay, male, image analysis, Appendectomy, Humans, controlled study, postoperative complication, human, nuclear magnetic resonance imaging, Child, outcome assessment, Pandemics, COVID-19/epidemiology, Retrospective Studies, pandemic, health care facility, echography, COVID-19, Appendicitis, health survey, female, multicenter study, adolescent, histopathology, disease severity, Surgery, abdominal abscess, hospitalization
Abstract

[No abstract available]

Published
2022
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54. Pulmonary infections in patients with acute myeloid leukemia receiving frontline treatment with hypomethylating agents

Author

Scamuffa, Maria Cristina, primary, Latagliata, Roberto, additional, Carmosino, Ida, additional, Di Veroli, Ambra, additional, Scalzulli, Emilia, additional, Trapè, Giulio, additional, Ciotti, Giulia, additional, De Angelis, Gioia, additional, Tartaglia, Germana, additional, Tarnani, Michela, additional, Breccia, Massimo, additional, and Girmenia, Corrado, additional

Published
2023
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56. P686: REAL-WORLD EFFICACY PROFILE OF ASCIMINIB IN AN ITALIAN, MULTI-RESISTANT CHRONIC-PHASE CHRONIC MYELOID LEUKEMIA (CP-CML) PATIENT POPULATION

Author

Breccia, Massimo, primary, Russo Rossi, Antonella, additional, Giai, Valentina, additional, Martino, Bruno, additional, Fava, Carmen, additional, Annunziata, Mario, additional, Abruzzese, Elisabetta, additional, Binotto, Gianni, additional, Baratè, Claudia, additional, Pio Nardozza, Aurelio, additional, Misto, Alessandra, additional, Coco, Paola, additional, Calafiore, Valeria, additional, Cristina Carraro, Maria, additional, Cattina, Federica, additional, Cavazzini, Francesco, additional, Teresa Corsetti, Maria, additional, Crucitti, Lara, additional, Crugnola, Monica, additional, Ditonno, Paolo, additional, DI Veroli, Ambra, additional, Ermacora, Anna, additional, Ferrara, Felicetto, additional, Genua, Angelo, additional, Gozzini, Antonella, additional, Impera, Stefana, additional, Iurlo, Alessandra, additional, Levato, Luciano, additional, Luciano, Luigiana, additional, Cristina Miggiano, Maria, additional, De Gobbi, Marco, additional, Santoro, Marco, additional, Scappini, Barbara, additional, Rita Scortechini, Anna, additional, Patriarca, Andrea, additional, Rosati, Serena, additional, Russo, Sabina, additional, Sancetta, Rosaria, additional, Sanpaolo, Grazia, additional, Maria Santeramo, Teresa, additional, Sibilla, Silvia, additional, Sorà, Federica, additional, Sportoletti, Paolo, additional, Stagno, Fabio, additional, Trabacchi, Elena, additional, and Castagnetti, Fausto, additional

Published
2023
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58. Retinoic acid-induced 1 gene haploinsufficiency alters lipid metabolism and causes autophagy defects in Smith-Magenis syndrome

Author

Turco, E, Giovenale, A, Sireno, L, Mazzoni, M, Cammareri, A, Marchioretti, C, Goracci, L, Di Veroli, A, Marchesan, E, D'Andrea, D, Falconieri, A, Torres, B, Bernardini, L, Magnifico, M, Paone, A, Rinaldo, S, Della Monica, M, D'Arrigo, S, Postorivo, D, Nardone, A, Zampino, G, Onesimo, R, Leoni, C, Caicci, F, Raimondo, D, Binda, E, Trobiani, L, De Jaco, A, Tata, A, Ferrari, D, Cutruzzola, F, Mazzoccoli, G, Ziviani, E, Pennuto, M, Vescovi, A, Rosati, J, Turco E. M., Giovenale A. M. G., Sireno L., Mazzoni M., Cammareri A., Marchioretti C., Goracci L., Di Veroli A., Marchesan E., D'Andrea D., Falconieri A., Torres B., Bernardini L., Magnifico M. C., Paone A., Rinaldo S., Della Monica M., D'Arrigo S., Postorivo D., Nardone A. M., Zampino G., Onesimo R., Leoni C., Caicci F., Raimondo D., Binda E., Trobiani L., De Jaco A., Tata A. M., Ferrari D., Cutruzzola F., Mazzoccoli G., Ziviani E., Pennuto M., Vescovi A. L., Rosati J., Turco, E, Giovenale, A, Sireno, L, Mazzoni, M, Cammareri, A, Marchioretti, C, Goracci, L, Di Veroli, A, Marchesan, E, D'Andrea, D, Falconieri, A, Torres, B, Bernardini, L, Magnifico, M, Paone, A, Rinaldo, S, Della Monica, M, D'Arrigo, S, Postorivo, D, Nardone, A, Zampino, G, Onesimo, R, Leoni, C, Caicci, F, Raimondo, D, Binda, E, Trobiani, L, De Jaco, A, Tata, A, Ferrari, D, Cutruzzola, F, Mazzoccoli, G, Ziviani, E, Pennuto, M, Vescovi, A, Rosati, J, Turco E. M., Giovenale A. M. G., Sireno L., Mazzoni M., Cammareri A., Marchioretti C., Goracci L., Di Veroli A., Marchesan E., D'Andrea D., Falconieri A., Torres B., Bernardini L., Magnifico M. C., Paone A., Rinaldo S., Della Monica M., D'Arrigo S., Postorivo D., Nardone A. M., Zampino G., Onesimo R., Leoni C., Caicci F., Raimondo D., Binda E., Trobiani L., De Jaco A., Tata A. M., Ferrari D., Cutruzzola F., Mazzoccoli G., Ziviani E., Pennuto M., Vescovi A. L., and Rosati J.

Abstract

Smith-Magenis syndrome (SMS) is a neurodevelopmental disorder characterized by cognitive and behavioral symptoms, obesity, and sleep disturbance, and no therapy has been developed to alleviate its symptoms or delay disease onset. SMS occurs due to haploinsufficiency of the retinoic acid-induced-1 (RAI1) gene caused by either chromosomal deletion (SMS-del) or RAI1 missense/nonsense mutation. The molecular mechanisms underlying SMS are unknown. Here, we generated and characterized primary cells derived from four SMS patients (two with SMS-del and two carrying RAI1 point mutations) and four control subjects to investigate the pathogenetic processes underlying SMS. By combining transcriptomic and lipidomic analyses, we found altered expression of lipid and lysosomal genes, deregulation of lipid metabolism, accumulation of lipid droplets, and blocked autophagic flux. We also found that SMS cells exhibited increased cell death associated with the mitochondrial pathology and the production of reactive oxygen species. Treatment with N-acetylcysteine reduced cell death and lipid accumulation, which suggests a causative link between metabolic dyshomeostasis and cell viability. Our results highlight the pathological processes in human SMS cells involving lipid metabolism, autophagy defects and mitochondrial dysfunction and suggest new potential therapeutic targets for patient treatment.

Published
2022

59. Deferasirox in the management of iron overload in patients with myelofibrosis treated with ruxolitinib: The multicentre retrospective RUX-IOL study

Author

Elli, E, Di Veroli, A, Bartoletti, D, Iurlo, A, Carmosino, I, Benevolo, G, Abruzzese, E, Bonifacio, M, Bergamaschi, M, Polverelli, N, Caramella, M, Cilloni, D, Tiribelli, M, Pugliese, N, Caocci, G, Crisa, E, Porrini, R, Markovic, U, Renso, R, Auteri, G, Cattaneo, D, Trawinska, M, Scaffidi, L, Biale, L, Bucelli, C, Breccia, M, Gambacorti Passerini, C, Palumbo, G, Latagliata, R, Palandri, F, Elli E. M., Di Veroli A., Bartoletti D., Iurlo A., Carmosino I., Benevolo G., Abruzzese E., Bonifacio M., Bergamaschi M., Polverelli N., Caramella M., Cilloni D., Tiribelli M., Pugliese N., Caocci G., Crisa E., Porrini R., Markovic U., Renso R., Auteri G., Cattaneo D., Trawinska M. M., Scaffidi L., Biale L., Bucelli C., Breccia M., Gambacorti Passerini C., Palumbo G. A., Latagliata R., Palandri F., Elli, E, Di Veroli, A, Bartoletti, D, Iurlo, A, Carmosino, I, Benevolo, G, Abruzzese, E, Bonifacio, M, Bergamaschi, M, Polverelli, N, Caramella, M, Cilloni, D, Tiribelli, M, Pugliese, N, Caocci, G, Crisa, E, Porrini, R, Markovic, U, Renso, R, Auteri, G, Cattaneo, D, Trawinska, M, Scaffidi, L, Biale, L, Bucelli, C, Breccia, M, Gambacorti Passerini, C, Palumbo, G, Latagliata, R, Palandri, F, Elli E. M., Di Veroli A., Bartoletti D., Iurlo A., Carmosino I., Benevolo G., Abruzzese E., Bonifacio M., Bergamaschi M., Polverelli N., Caramella M., Cilloni D., Tiribelli M., Pugliese N., Caocci G., Crisa E., Porrini R., Markovic U., Renso R., Auteri G., Cattaneo D., Trawinska M. M., Scaffidi L., Biale L., Bucelli C., Breccia M., Gambacorti Passerini C., Palumbo G. A., Latagliata R., and Palandri F.

Abstract

Deferasirox (DFX) is used for the management of iron overload (IOL) in many haematological malignancies including myelofibrosis (MF). The ‘RUX-IOL’ study retrospectively collected 69 MF patients treated with ruxolitinib (RUX) and DFX for IOL to assess: safety, efficacy in term of iron chelation response (ICR) and erythroid response (ER), and impact on overall survival of the combination therapy. The RUX–DFX therapy was administered for a median time of 12.4 months (interquartile range 3.1–71.2). During treatment, 36 (52.2%) and 34 (49.3%) patients required RUX and DFX dose reductions, while eight (11.6%) and nine (13.1%) patients discontinued due to RUX- or DFX-related adverse events; no unexpected toxicity was reported. ICR and ER were achieved by 33 (47.8%) and 32 patients (46.4%) respectively. Thirteen (18.9%) patients became transfusion-independent. Median time to ICR and ER was 6.2 and 2 months respectively. Patients achieving an ER were more likely to obtain an ICR also (p = 0.04). In multivariable analysis, the absence of leukocytosis at baseline (p = 0.02) and achievement of an ICR at any time (p = 0.02) predicted improved survival. In many MF patients, the RUX–DFX combination provided ICR and ER responses that correlated with improved outcome in the absence of unexpected toxicities. This strategy deserves further clinical investigation.

Published
2022

60. LibRob: An Autonomous Assistive Librarian

Author

Di Veroli, Costanza, primary, Le, Cao An, additional, Lemaire, Thibaud, additional, Makabu, Eliot, additional, Nur, Abdullahi, additional, Ooi, Vincent, additional, Park, Jee Yong, additional, Sanna, Federico, additional, Chacon, Rodrigo, additional, and Demiris, Yiannis, additional

Published
2019
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61. A platelet lipidomics signature in patients with COVID-19.

Author

Goracci, Laura, Petito, Eleonora, Di Veroli, Alessandra, Falcinelli, Emanuela, Bencivenga, Caterina, Giglio, Elisa, Becattini, Cecilia, De Robertis, Edoardo, Vaudo, Gaetano, and Gresele, Paolo

Subjects
COVID-19, LIPIDOMICS, BLOOD platelets, VIRAL transmission, BLOOD platelet activation
Abstract

Ischemic cardiovascular and venous thromboembolic events are a frequent cause of death in severe COVID-19 patients. Platelet activation plays a key role in these complications, however platelet lipidomics have not been studied yet. The aim of our pilot investigation was to perform a preliminary study of platelet lipidomics in COVID-19 patients compared to healthy subjects. Lipid extraction and identification of ultrapurified platelets from eight hospitalized COVID-19 patients and eight age- and sex-matched healthy controls showed a lipidomic pattern almost completely separating COVID-19 patients from healthy controls. In particular, a significant decrease of ether phospholipids and increased levels of ganglioside GM3 were observed in platelets from COVID-19 patients. In conclusion, our study shows for the first time that platelets from COVID-19 patients display a different lipidomics signature distinguishing them from healthy controls, and suggests that altered platelet lipid metabolism may play a role in viral spreading and in the thrombotic complications of COVID-19. [ABSTRACT FROM AUTHOR]

Published
2023
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62. Treatment in patients with acute myeloid leukemia/high‐risk myelodysplastic syndrome with hypomethylating agents: Day‐hospital management compared to home care setting

Author

Trapè, Giulio, primary, De Angelis, Gioia, additional, Morucci, Marco, additional, Tarnani, Michela, additional, De Gregoris, Cinzia, additional, Di Veroli, Ambra, additional, Panichi, Valentina, additional, Topini, Giuseppe, additional, Bassi, Loredana, additional, Isidori, Raffaella, additional, Poscente, Monica, additional, Innocenti, Vincenza, additional, Cippitelli, Elisa Emanueli, additional, Talucci, Roberta, additional, Bertelli, Silvia, additional, Crocicchia, Alessandra, additional, Lippi, Annalisa, additional, Pezzuti, Giulia, additional, Fuschino, Michela, additional, Randi, Raffaella, additional, Mastini, Cristina, additional, Ciambella, Silvia, additional, Pessina, Gloria, additional, Montanaro, Marco, additional, and Latagliata, Roberto, additional

Published
2023
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64. Treatment in patients with acute myeloid leukemia/high‐risk myelodysplastic syndrome with hypomethylating agents: Day‐hospital management compared to home care setting

Author

Giulio Trapè, Gioia De Angelis, Marco Morucci, Michela Tarnani, Cinzia De Gregoris, Ambra Di Veroli, Valentina Panichi, Giuseppe Topini, Loredana Bassi, Raffaella Isidori, Monica Poscente, Vincenza Innocenti, Elisa Emanueli Cippitelli, Roberta Talucci, Silvia Bertelli, Alessandra Crocicchia, Annalisa Lippi, Giulia Pezzuti, Michela Fuschino, Raffaella Randi, Cristina Mastini, Silvia Ciambella, Gloria Pessina, Marco Montanaro, and Roberto Latagliata

Subjects
Hematology, General Medicine
Published
2023
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65. Ruxolitinib in clinical practice for primary and secondary myelofibrosis: an analysis of safety and efficacy of Gruppo Laziale of Ph-negative MPN

Author

Breccia, Massimo, Andriani, Alessandro, Montanaro, Marco, Abruzzese, Elisabetta, Buccisano, Francesco, Cedrone, Michele, Centra, Antonietta, Villivà, Nicoletta, Celesti, Francesca, Trawinska, Malgorzata Monica, Massaro, Fulvio, Di Veroli, Ambra, Anaclerico, Barbara, Colafigli, Gioia, Molica, Matteo, Spadea, Antonio, Petriccione, Luca, Cimino, Giuseppe, and Latagliata, Roberto

Published
2017
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66. Choice of Tyrosine Kinase Inhibitor and Early Events during the First Year of Therapy in Newly Diagnosed Chronic Phase Chronic Myeloid Leukemia (CML) Patients with Concomitant Diabetes: A 'Campus CML' Study

Author

Isabella Capodanno, Mario Tiribelli, Maria Cristina Miggiano, Cristina Bucelli, Francesco Cavazzini, Sabrina Leonetti Crescenzi, Sabina Russo, Emilia Scalzulli, Andrea Bernardelli, Luigiana Luciano, Olga Mulas, Giuseppina Loglisci, Chiara Elena, Umberto Pizzano, Immacolata Attolico, Gianni Binotto, Elena Crisà, Paolo Sportoletti, Ambra Di Veroli, Anna Rita Scortechini, Annapaola Leporace, Maria Basile, Monica Crugnola, Fabio Stagno, Pamela Murgano, Davide Rapezzi, Debora Luzi, Alessandra Iurlo, Monica Bocchia, Carmen Fava, Alessandra Malato, Sara Galimberti, Iolanda Donatella Vincelli, Malgorzata Monika Trawinska, Michele Pizzuti, Giovanni Caocci, Massimiliano Bonifacio, Giuseppe Saglio, Giorgina Specchia, Massimo Breccia, and Roberto Latagliata

Subjects
Immunology, Cell Biology, Hematology, Biochemistry
Published
2022
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67. Association of Hypometilating Agents (HMA) + Venetoclax (VEN) in the Treatment of Myeloproliferative Neoplasms in Blastic Phase (MPN-BP) and Acute Leukemias Evolved from Myelodysplastic Syndromes (AML-MDS) Already Treated with Azacytidine

Author

Roberto Latagliata, Gianluca Cristiano, Dorela Lama, Susanna Fenu, Lorenzo Rizzo, Benedetta Cambò, Simone Zoletto, Francesca Spirito, Fabrizio Cavalca, Daniele Cattaneo, Natalia Cenfra, Giulia De Luca, Beatrice Esposito Vangone, Ambra Di Veroli, Raffaele Palmieri, Mario Annunziata, Olga Mulas, Elisabetta Abruzzese, Alfredo Molteni, Monica Piedimonte, Ida Carmosino, Prassede Salutari, Annalisa Biagi, Monica Bocchia, Michelina Santopietro, Gianni Binotto, Alessandra Iurlo, Monica Crugnola, Marta Riva, Anna Lina Piccioni, Elena Maria Elli, Antonio Curti, and Antonella Poloni

Subjects
Immunology, Cell Biology, Hematology, Biochemistry
Published
2022
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76. Pre-transplant persistence of minimal residual disease does not contraindicate allogeneic stem cell transplantation for adult patients with acute myeloid leukemia

Author

Buccisano, F, Maurillo, L, Piciocchi, A, Del Principe, M I, Picardi, A, Cerretti, R, Cudillo, L, De Angelis, G, Sarlo, C, Cefalo, M, Ditto, C, Di Veroli, A, Mariotti, B, Nasso, D, De Bellis, E, Del Poeta, G, Voso, M T, Sconocchia, G, Lo Coco, F, Arcese, W, Amadori, S, and Venditti, A

Published
2017
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77. TREATMENT OF LOW-BLAST COUNT AML USING HYPOMETHYLATING AGENTS

Author

Eleonora De Bellis, Luana Fianchi, Francesco Buccisano, Marianna Criscuolo, Luca Maurillo, Laura Cicconi, Mattia Brescini, Maria Ilaria Del Principe, Ambra Di Veroli, Adriano Venditti, Francesco Lo-Coco, and Maria Teresa Voso

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

In 2002, the WHO classification reduced the proportion of blasts in the bone marrow (BM) necessary for the diagnosis of acute myeloid leukemia (AML) from 30% to 20%, eliminating the RAEB-t subtype of myelodysplastic syndromes (MDS). However, this AML subtype, defined as low-blast count AML (LBC-AML, with 20-30% BM-blasts) is characterized by peculiar features, as increased frequency in elderly individuals and after cytotoxic treatment for a different primary disease (therapy-related), poor-risk cytogenetics, lower white blood cell counts, and less frequent mutations of NPM1 and FLT3 genes. The clinical course of this entity is often similar to MDS with 10-19% BM-blasts. The hypomethylating agents azacitidine and decitabine have been shown to induce responses and prolong survival both in MDS and LBC-AML. The role of these agents has been also demonstrated in AML with >30% BM-blasts, particularly in patients with poor-risk cytogenetics and in AML with myelodysplasia related changes. Most recent studies are evaluating strategies to improve outcome, including combinations of hypomethylating agents with immune-response checkpoint inhibitors, which have a role in cancer immune surveillance. Efforts are also ongoing to identify mutations which may predict response and survival in these patients.

Published
2017
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78. Modelling of the cancer cell cycle as a tool for rational drug development: A systems pharmacology approach to cyclotherapy.

Author

Robert C Jackson, Giovanni Y Di Veroli, Siang-Boon Koh, Ian Goldlust, Frances M Richards, and Duncan I Jodrell

Subjects
Biology (General), QH301-705.5
Abstract

The dynamic of cancer is intimately linked to a dysregulation of the cell cycle and signalling pathways. It has been argued that selectivity of treatments could exploit loss of checkpoint function in cancer cells, a concept termed "cyclotherapy". Quantitative approaches that describe these dysregulations can provide guidance in the design of novel or existing cancer therapies. We describe and illustrate this strategy via a mathematical model of the cell cycle that includes descriptions of the G1-S checkpoint and the spindle assembly checkpoint (SAC), the EGF signalling pathway and apoptosis. We incorporated sites of action of four drugs (palbociclib, gemcitabine, paclitaxel and actinomycin D) to illustrate potential applications of this approach. We show how drug effects on multiple cell populations can be simulated, facilitating simultaneous prediction of effects on normal and transformed cells. The consequences of aberrant signalling pathways or of altered expression of pro- or anti-apoptotic proteins can thus be compared. We suggest that this approach, particularly if used in conjunction with pharmacokinetic modelling, could be used to predict effects of specific oncogene expression patterns on drug response. The strategy could be used to search for synthetic lethality and optimise combination protocol designs.

Published
2017
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79. Identification of Predictive Factors for Overall Survival and Response during Hypomethylating Treatment in Very Elderly (≥75 Years) Acute Myeloid Leukemia Patients: A Multicenter Real-Life Experience

Author

Matteo Molica, Carla Mazzone, Pasquale Niscola, Ida Carmosino, Ambra Di Veroli, Cinzia De Gregoris, Fabrizio Bonanni, Salvatore Perrone, Natalia Cenfra, Luana Fianchi, Anna Lina Piccioni, Antonio Spadea, Giovanni Luzi, Andrea Mengarelli, Laura Cudillo, Luca Maurillo, Livio Pagano, Massimo Breccia, Luigi Rigacci, and Paolo De Fabritiis

Subjects
acute myeloid leukemia, very elderly, real-life experience, Settore MED/15 - MALATTIE DEL SANGUE, Cancer Research, Oncology, Settore MED/15
Abstract

Elderly patients represent the most challenging and hard-to-treat patient population due to dismal characteristics of the disease, such as secondary-acute myeloid leukemia (AML), enrichment of unfavorable molecular genes (TP53) and comorbidities. We conducted a multicentric retrospective study to evaluate activity and safety in a real-life setting of hypomethylating drugs (HMAs) in patients older than 75 years with AML. Between September 2010 and December 2021, 220 patients were treated, 164 (74.5%) received AZAcitidine and 56 DECitabine; most patients (57.8%), received more than four cycles of HMAs. The best response obtained was CR in 51 patients (23.2%), PR in 23 (10.5%) and SD in 45 (20.5%); overall transfusion independence was obtained in 47 patients (34%), after a median of 3.5 months. The median OS (mOs) was 8 months (95% CI 5.9–10.2), with 1- and 2-years OS of 39.4% (95% CI 32.7–46) and 17.4% (95% CI 11.7–23.1), respectively; similar mOS was observed according to HMA treatment (AZA 8.3 vs. DEC 7.8 months, p = 0.810). A subset of 57 long survivors (44 in AZA group and 13 in DEC group) received at least 12 cycles of HMAs, their mOS was 24.3 months. In multivariate analysis, age (≥80), Charlson comorbidity index (≥3), creatinine clearance and the type of best response (≥PR) during treatment maintained independent significance in predicting survival. Infectious complications, most frequently pneumonia (35) and septic shock (12), were lethal in 49 patients (22.2%). Our data show that HMAs have similar efficacy compared to pivotal trials and are well tolerated in a setting of very elderly patients with several co-comorbidities.

Published
2022
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80. Deferasirox in the management of iron overload in patients with myelofibrosis treated with ruxolitinib: The multicentre retrospective RUX-IOL study

Author

Elena Maria Elli, Ambra Di Veroli, Daniela Bartoletti, Alessandra Iurlo, Ida Carmosino, Giulia Benevolo, Elisabetta Abruzzese, Massimiliano Bonifacio, Micaela Bergamaschi, Nicola Polverelli, Marianna Caramella, Daniela Cilloni, Mario Tiribelli, Novella Pugliese, Giovanni Caocci, Elena Crisà, Raffaele Porrini, Uros Markovic, Rossella Renso, Giuseppe Auteri, Daniele Cattaneo, Malgorzata Monika Trawinska, Luigi Scaffidi, Lucia Biale, Cristina Bucelli, Massimo Breccia, Carlo Gambacorti‐Passerini, Giuseppe Alberto Palumbo, Roberto Latagliata, Francesca Palandri, Elli, E, Di Veroli, A, Bartoletti, D, Iurlo, A, Carmosino, I, Benevolo, G, Abruzzese, E, Bonifacio, M, Bergamaschi, M, Polverelli, N, Caramella, M, Cilloni, D, Tiribelli, M, Pugliese, N, Caocci, G, Crisa, E, Porrini, R, Markovic, U, Renso, R, Auteri, G, Cattaneo, D, Trawinska, M, Scaffidi, L, Biale, L, Bucelli, C, Breccia, M, Gambacorti Passerini, C, Palumbo, G, Latagliata, R, and Palandri, F

Subjects
ruxolitinib, myelofibrosis, Hematology, Iron Chelating Agents, Benzoates, cancer, deferasirox, iron overload, Pyrimidines, myelofibrosi, MED/15 - MALATTIE DEL SANGUE, Primary Myelofibrosis, Nitriles, Humans, Pyrazoles, Retrospective Studies
Abstract

Deferasirox (DFX) is used for the management of iron overload (IOL) in many haematological malignancies including myelofibrosis (MF). The ‘RUX-IOL’ study retrospectively collected 69 MF patients treated with ruxolitinib (RUX) and DFX for IOL to assess: safety, efficacy in term of iron chelation response (ICR) and erythroid response (ER), and impact on overall survival of the combination therapy. The RUX–DFX therapy was administered for a median time of 12.4months (interquartile range 3.1–71.2). During treatment, 36 (52.2%) and 34 (49.3%) patients required RUX and DFX dose reductions, while eight (11.6%) and nine (13.1%) patients discontinued due to RUX- or DFX-related adverse events; no unexpected toxicity was reported. ICR and ER were achieved by 33 (47.8%) and 32 patients (46.4%) respectively. Thirteen (18.9%) patients became transfusion-independent. Median time to ICR and ER was 6.2 and 2months respectively. Patients achieving an ER were more likely to obtain an ICR also (p=0.04). In multivariable analysis, the absence of leukocytosis at baseline (p=0.02) and achievement of an ICR at any time (p=0.02) predicted improved survival. In many MF patients, the RUX–DFX combination provided ICR and ER responses that correlated with improved outcome in the absence of unexpected toxicities. This strategy deserves further clinical investigation.

Published
2022

81. Retinoic acid-induced 1 gene haploinsufficiency alters lipid metabolism and causes autophagy defects in Smith-Magenis syndrome

Author

Turco, Elisa Maria, primary, Giovenale, Angela Maria Giada, additional, Sireno, Laura, additional, Mazzoni, Martina, additional, Cammareri, Alessandra, additional, Marchioretti, Caterina, additional, Goracci, Laura, additional, Di Veroli, Alessandra, additional, Marchesan, Elena, additional, D’Andrea, Daniel, additional, Falconieri, Antonella, additional, Torres, Barbara, additional, Bernardini, Laura, additional, Magnifico, Maria Chiara, additional, Paone, Alessio, additional, Rinaldo, Serena, additional, Della Monica, Matteo, additional, D’Arrigo, Stefano, additional, Postorivo, Diana, additional, Nardone, Anna Maria, additional, Zampino, Giuseppe, additional, Onesimo, Roberta, additional, Leoni, Chiara, additional, Caicci, Federico, additional, Raimondo, Domenico, additional, Binda, Elena, additional, Trobiani, Laura, additional, De Jaco, Antonella, additional, Tata, Ada Maria, additional, Ferrari, Daniela, additional, Cutruzzolà, Francesca, additional, Mazzoccoli, Gianluigi, additional, Ziviani, Elena, additional, Pennuto, Maria, additional, Vescovi, Angelo Luigi, additional, and Rosati, Jessica, additional

Published
2022
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82. Risk Factors for Thrombosis in Patients Aged > 60 Years with Essential Thrombocythemia without Previous Thrombotic Events

Author

Latagliata, Roberto, primary, Andriani, Alessandro, additional, Breccia, Massimo, additional, Carmosino, Ida, additional, Vozella, Federico, additional, Romano, Atelda, additional, Biagi, Annalisa, additional, Maurillo, Luca, additional, Trawinska, Malgorzata Monika, additional, Paciaroni, Katia, additional, Santopietro, Michelina, additional, Leonetti Crescenzi, Sabrina, additional, D'Addosio, Ada, additional, Tatarelli, Caterina, additional, Abruzzese, Elisabetta, additional, Di Veroli, Ambra, additional, Rossi, Elena, additional, De Stefano, Valerio, additional, and Montanaro, Marco, additional

Published
2022
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83. Efficacy and Safety of Luspatercept in Adult Patients with Transfusion-Dependent Anemia Due to Very Low, Low and Intermediate Risk Myelodysplastic Syndromes (MDS) with Ring Sideroblasts, Who Had an Unsatisfactory Response to or Are Ineligible for Erythropoietin-Based Therapy: A Retrospective Multicenter Study By Fondazione Italiana Sindromi Mielodisplastiche (FiSiM ETS)

Author

Lanino, Luca, primary, Salutari, Prassede, additional, Perego, Alessandra, additional, Fattizzo, Bruno, additional, Riva, Marta, additional, Ubezio, Marta, additional, Musto, Pellegrino, additional, Cilloni, Daniela, additional, Oliva, Esther Natalie, additional, Voso, Maria Teresa, additional, Pelizzari, Anna Maria, additional, Poloni, Antonella, additional, Capodanno, Isabella, additional, Elena, Chiara, additional, Fozza, Claudio, additional, Pane, Fabrizio, additional, Breccia, Massimo, additional, De Gobbi, Marco, additional, Di Bassiano, Francesco, additional, Barraco, Daniela, additional, Crisà, Elena, additional, Ferrero, Dario, additional, Frairia, Chiara, additional, Vaccarino, Antonella, additional, Griguolo, Davide, additional, Paolini, Stefania, additional, Quintini, Martina, additional, Sessa, Mariarosaria, additional, Turrini, Mauro, additional, Bocchia, Monica, additional, Di Renzo, Nicola, additional, Diral, Elisa, additional, Foli, Cristina, additional, Molteni, Alfredo, additional, Occhini, Ubaldo, additional, Rivoli, Giulia, additional, Selleri, Carmine, additional, Bono, Roberto, additional, Calvisi, Anna, additional, Castelli, Andrea, additional, Di Bona, Eros, additional, Di Veroli, Ambra, additional, Fianchi, Luana, additional, Galimberti, Sara, additional, Grimaldi, Daniele, additional, Marchetti, Monia, additional, Norata, Marianna, additional, Rambaldi, Alessandro, additional, Tanasi, Ilaria, additional, Tosi, Patrizia, additional, Naldi, Ilaria, additional, Santini, Valeria, additional, and Della Porta, Matteo G., additional

Published
2022
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84. Permanent Discontinuation of Tyrosine Kinase Inhibitor Frontline Therapy in Patients with Chronic Phase Chronic Myeloid Leukemia Patients during the First 36 Months of Treatment: A "Campus CML" Study

Author

Latagliata, Roberto, primary, Capodanno, Isabella, additional, Miggiano, Maria Cristina, additional, Iurlo, Alessandra, additional, Cavazzini, Francesco, additional, Leonetti Crescenzi, Sabrina, additional, Russo, Sabina, additional, Carmosino, Ida, additional, Bernardelli, Andrea, additional, Mulas, Olga, additional, Elena, Chiara, additional, Binotto, Gianni, additional, Crisà, Elena, additional, Di Veroli, Ambra, additional, Scortechini, Anna Rita, additional, Basile, Maria, additional, Crugnola, Monica, additional, Murgano, Pamela, additional, Pizzuti, Michele, additional, Bucelli, Cristina, additional, Fava, Carmen, additional, Dalmazzo, Matteo, additional, Lunghi, Francesca, additional, Caocci, Giovanni, additional, Bonifacio, Massimiliano, additional, Saglio, Giuseppe, additional, Specchia, Giorgina, additional, Breccia, Massimo, additional, and Tiribelli, Mario, additional

Published
2022
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86. Domiciliary Nursing Management for Patients Affected By Multiple Myeloma during Covid-19 Pandemia in the Viterbo Home Care Unit: Data Analysis from March 2020 to March 2022

Author

Emanueli Cippitelli, Elisa, primary, Innocenti, Vincenza, additional, De Angelis, Gioia, additional, Bertelli, Silvia, additional, Talucci, Roberta, additional, Perazzini, Roberta, additional, Burla, Roberta, additional, Panichi, Valentina, additional, Topini, Giuseppe, additional, Di Veroli, Ambra, additional, Fiorini, Alessia, additional, Mercanti, Caterina, additional, Natalino, Fiammetta, additional, Stefanizzi, Caterina, additional, De Gregoris, Cinzia, additional, Tarnani, Michela, additional, Morucci, Marco, additional, Mastino, Cristina, additional, Trapè, Giulio, additional, Montanaro, Marco, additional, Latagliata, Roberto, additional, and Ciambella, Silvia, additional

Published
2022
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87. Clinical Characteristics at Onset and Treatment Approaches in Young and Elderly Patients with Essential Thrombocitemia (ET) in Lazio Region: Evaluation of Retrospective and Prospective Databases

Author

Andriani, Alessandro, primary, Breccia, Massimo, additional, Scalzulli, Emilia, additional, Cerchiara, Elisabetta, additional, Romano, Atelda, additional, Biagi, Annalisa, additional, Maurillo, Luca, additional, Trawinska, Malgorzata Monika, additional, Paciaroni, Katia, additional, Santopietro, Michelina, additional, Leonetti Crescenzi, Sabrina, additional, D'Addosio, Ada, additional, Tatarelli, Caterina, additional, Abruzzese, Elisabetta, additional, Di Veroli, Ambra, additional, Rossi, Elena, additional, Latagliata, Roberto, additional, and Montanaro, Marco, additional

Published
2022
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88. Choice of Tyrosine Kinase Inhibitor and Early Events during the First Year of Therapy in Newly Diagnosed Chronic Phase Chronic Myeloid Leukemia (CML) Patients with Concomitant Diabetes: A "Campus CML" Study

Author

Capodanno, Isabella, primary, Tiribelli, Mario, additional, Miggiano, Maria Cristina, additional, Bucelli, Cristina, additional, Cavazzini, Francesco, additional, Leonetti Crescenzi, Sabrina, additional, Russo, Sabina, additional, Scalzulli, Emilia, additional, Bernardelli, Andrea, additional, Luciano, Luigiana, additional, Mulas, Olga, additional, Loglisci, Giuseppina, additional, Elena, Chiara, additional, Pizzano, Umberto, additional, Attolico, Immacolata, additional, Binotto, Gianni, additional, Crisà, Elena, additional, Sportoletti, Paolo, additional, Di Veroli, Ambra, additional, Scortechini, Anna Rita, additional, Leporace, Annapaola, additional, Basile, Maria, additional, Crugnola, Monica, additional, Stagno, Fabio, additional, Murgano, Pamela, additional, Rapezzi, Davide, additional, Luzi, Debora, additional, Iurlo, Alessandra, additional, Bocchia, Monica, additional, Fava, Carmen, additional, Malato, Alessandra, additional, Galimberti, Sara, additional, Vincelli, Iolanda Donatella, additional, Trawinska, Malgorzata Monika, additional, Pizzuti, Michele, additional, Caocci, Giovanni, additional, Bonifacio, Massimiliano, additional, Saglio, Giuseppe, additional, Specchia, Giorgina, additional, Breccia, Massimo, additional, and Latagliata, Roberto, additional

Published
2022
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89. Association of Hypometilating Agents (HMA) + Venetoclax (VEN) in the Treatment of Myeloproliferative Neoplasms in Blastic Phase (MPN-BP) and Acute Leukemias Evolved from Myelodysplastic Syndromes (AML-MDS) Already Treated with Azacytidine

Author

Latagliata, Roberto, primary, Cristiano, Gianluca, additional, Lama, Dorela, additional, Fenu, Susanna, additional, Rizzo, Lorenzo, additional, Cambò, Benedetta, additional, Zoletto, Simone, additional, Spirito, Francesca, additional, Cavalca, Fabrizio, additional, Cattaneo, Daniele, additional, Cenfra, Natalia, additional, De Luca, Giulia, additional, Esposito Vangone, Beatrice, additional, Di Veroli, Ambra, additional, Palmieri, Raffaele, additional, Annunziata, Mario, additional, Mulas, Olga, additional, Abruzzese, Elisabetta, additional, Molteni, Alfredo, additional, Piedimonte, Monica, additional, Carmosino, Ida, additional, Salutari, Prassede, additional, Biagi, Annalisa, additional, Bocchia, Monica, additional, Santopietro, Michelina, additional, Binotto, Gianni, additional, Iurlo, Alessandra, additional, Crugnola, Monica, additional, Riva, Marta, additional, Piccioni, Anna Lina, additional, Elli, Elena Maria, additional, Curti, Antonio, additional, and Poloni, Antonella, additional

Published
2022
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90. Identification of Predictive Factors for Overall Survival and Response during Hypomethylating Treatment in Very Elderly (≥75 Years) Acute Myeloid Leukemia Patients: A Multicenter Real-Life Experience

Author

Molica, Matteo, primary, Mazzone, Carla, additional, Niscola, Pasquale, additional, Carmosino, Ida, additional, Di Veroli, Ambra, additional, De Gregoris, Cinzia, additional, Bonanni, Fabrizio, additional, Perrone, Salvatore, additional, Cenfra, Natalia, additional, Fianchi, Luana, additional, Piccioni, Anna Lina, additional, Spadea, Antonio, additional, Luzi, Giovanni, additional, Mengarelli, Andrea, additional, Cudillo, Laura, additional, Maurillo, Luca, additional, Pagano, Livio, additional, Breccia, Massimo, additional, Rigacci, Luigi, additional, and De Fabritiis, Paolo, additional

Published
2022
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91. Myelodysplastic syndromes with del(5q): A real-life study of determinants of long-term outcomes and response to lenalidomide

Author

Gurnari, Carmelo, primary, Piciocchi, Alfonso, additional, Soddu, Stefano, additional, Bonanni, Fabrizio, additional, Scalzulli, Emilia, additional, Niscola, Pasquale, additional, Di Veroli, Ambra, additional, Piccioni, Anna Lina, additional, Piedimonte, Monica, additional, Maiorana, Gianluca, additional, Salutari, Prassede, additional, Cicconi, Laura, additional, Santopietro, Michelina, additional, Gumenyuk, Svitlana, additional, Sarlo, Chiara, additional, Fenu, Susanna, additional, Tafuri, Agostino, additional, Latagliata, Roberto, additional, Fianchi, Luana, additional, Criscuolo, Marianna, additional, Maciejewski, Jaroslaw P., additional, Maurillo, Luca, additional, Buccisano, Francesco, additional, Breccia, Massimo, additional, and Voso, Maria Teresa, additional

Published
2022
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93. Quantitative Systems Modeling Approaches Towards Model-Informed Drug Development: Perspective Through Case Studies

Author

Meghna Verma, Louis Gall, Jacopo Biasetti, Giovanni Di Veroli, Cesar Pichardo-Almarza, and Holly Kimko

Abstract

Quantitative systems pharmacology modeling has become an increasingly popular approach impacting our understanding of disease mechanisms and helping predict patients’ treatment responses to facilitate study design or development go/no-go decisions. In this paper, we present examples of QSP modeling applications to highlight the notable contributions and opportunities that these approaches are to offer during the drug development process. We also consider the barriers to successful applications; outline the factors required to support the success of these modeling approaches; and provide insights for a strategy to enhance acceptance by the regulatory agencies.

Published
2022
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96. Infectious disease ward admission positively influences P. jiroveci pneumonia (PjP) outcome: A retrospective analysis of 116 HIV-positive and HIV-negative immunocompromised patients.

Author

Alessandra Ricciardi, Elisa Gentilotti, Luigi Coppola, Gaetano Maffongelli, Carlotta Cerva, Vincenzo Malagnino, Alessia Mari, Ambra Di Veroli, Federica Berrilli, Fabiana Apice, Nicola Toschi, David Di Cave, Saverio Giuseppe Parisi, Massimo Andreoni, and Loredana Sarmati

Subjects
Medicine, Science
Abstract

P. jiroveci (Pj) causes a potentially fatal pneumonia in immunocompromised patients and the factors associated with a bad outcome are poorly understood. A retrospective analysis on Pj pneumonia (PjP) cases occurring in Tor Vergata University Hospital, Italy, during the period 2011-2015. The patients' demographic, clinical and radiological characteristics and the Pj genotypes were considered. The study population included 116 patients, 37.9% of whom had haematological malignancy or underwent haematological stem cell transplantation (HSCT), 22.4% had HIV infection, 16.4% had chronic lung diseases (CLD), 7.8% had a solid cancer, and 3.4% underwent a solid organ transplant (SOT). The remaining 12.1% had a miscellaneous other condition. At univariate analysis, being older than 60 years was significantly correlated with a severe PjP (OR [95%CI] 2.52 [0.10-5.76]; p = 0.031) and death (OR [95%CI] 2.44 [1.05-5.70]; p = 0.036), while a previous trimethoprim/sulfamethoxazole (TMP/SMX) prophylaxis were significantly associated with a less severe pneumonia (OR[95%CI] 0.35 [0.15-0.84], p = 0.023); moreover, death due to PjP was significantly more frequent in patients with CLD (OR[95%CI] 3.26 [1.17-9.05]; p = 0.019) while, admission to the Infectious Diseases Unit was significantly associated with fewer deaths (OR[95%CI] 0.10 [0.03-0.36], p = 0.002). At multivariate analysis, a better PjP outcome was observed in patients taking TMP/SMX prophylaxis and that were admitted to the Infectious Diseases Unit (OR[95%CI] 0.27 [0.07-1.03], p = 0.055, OR[95%CI] 0.16 [0.05-0.55]; p = 0.004, respectively). In conclusion, in our study population, TMP/SMX prophylaxis and infectious disease specialist approach were variables correlated with a better PjP outcome.

Published
2017
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97. Sequential occurrence of chronic myeloproliferative and lymphoproliferative neoplasms: a collaborative retrospective study by pH-negative MPN latial group

Author

Breccia, Massimo, primary, Petriccione, Luca, additional, Tatarelli, Caterina, additional, De Muro, Marianna, additional, Trawinska, Malgorzata Monica, additional, Santopietro, Michelina, additional, Spadea, Antonio, additional, Di Veroli, Ambra, additional, Scalzulli, Emilia, additional, Paciaroni, Katia, additional, Tafuri, Agostino, additional, Latagliata, Roberto, additional, Andriani, Alessandro, additional, and Di Napoli, Arianna, additional

Published
2022
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98. Anagrelide in Essential Thrombocythemia (ET): Results from 150 patients over 25 years by the 'Ph1‐negative Myeloproliferative Neoplasms Latium Group'

Author

Enrico Montefusco, Laura Scaramucci, Michele Cedrone, Antonio Spadea, Raffaele Porrini, Francesca Spirito, Giuseppe Cimino, Massimo Breccia, Angela Rago, Nicoletta Villivà, Marco Montanaro, Roberto Latagliata, Francesca Paoloni, Ermina Baldacci, Cristina Santoro, Ambra Di Veroli, Barbara Anaclerico, Alessandro Andriani, and Maria Gabriella Mazzucconi

Subjects
Adult, Male, medicine.medical_specialty, Young Adult, 03 medical and health sciences, 0302 clinical medicine, Pregnancy, Internal medicine, medicine, Palpitations, Humans, In patient, Adverse effect, Case report form, Aged, Retrospective Studies, Aged, 80 and over, Thrombotic risk, Drug Substitution, Essential thrombocythemia, business.industry, Pregnancy Complications, Hematologic, Age Factors, Disease Management, Anemia, Thrombosis, Hematology, General Medicine, Anagrelide, Middle Aged, Prognosis, medicine.disease, Treatment Outcome, Italy, Health Care Surveys, 030220 oncology & carcinogenesis, Retreatment, Quinazolines, Female, Disease Susceptibility, medicine.symptom, business, Follow-Up Studies, Thrombocythemia, Essential, 030215 immunology, medicine.drug
Abstract

Background and aims Anagrelide is a drug effective in reducing platelet counts in essential thrombocythemia (ET) and Ph1-negative myeloproliferative neoplasms. The aim of this study was to evaluate the real-life use of anagrelide in patients with ET followed over 25 years at the Haematological Institutes belonging to "Ph1-negative Myeloproliferative Neoplasms Latium Group." Patients and methods Eligibility criteria were diagnosis of ET and treatment with anagrelide. Data were collected through an ad hoc case report form. Results One hundred and fifty patients received anagrelide for a median time of 7.4 years (0.1-23.2). Anagrelide was administered as first-line therapy in 34.7% of patients, as second-line in 52% and as third-line in 13.3%: 85.4% responded to therapy. Sixty-eight/136 evaluable patients reported side effects: palpitations, peripheral vasodilation, anaemia, diarrhoea and gastric distress. Fourteen thrombotic (arterial 10, venous 4) and 51 bleeding events (minor 48, major 3) occurred. Sixteen/150 (10.6%) patients developed secondary myelofibrosis and 3/150 (2%) an acute myeloid leukaemia. Conclusions In our experience, anagrelide is an effective drug in reducing platelet levels in a high percentage of patients with ET. It is especially addressed to younger people. A careful assessment of the thrombotic risk and monitoring of cardiac function, at diagnosis and during follow-up, is mandatory.

Published
2020
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99. Myelodysplastic Syndromes with Isolated 20q Deletion: A New Clinical-Biological Entity?

Author

Alessia Campagna, Daniela De Benedittis, Luana Fianchi, Emilia Scalzulli, Lorenzo Rizzo, Pasquale Niscola, Anna Lina Piccioni, Ambra Di Veroli, Stefano Mancini, Nicoletta Villivà, Tiziano Martini, Sara Mohamed, Ida Carmosino, Marianna Criscuolo, Susanna Fenu, Maria Antonietta Aloe Spiriti, Francesco Buccisano, Marco Mancini, Agostino Tafuri, Massimo Breccia, Antonella Poloni, and Roberto Latagliata

Subjects
myelodysplastic syndromes, 20q deletion, erythropoietin, thrombocytopenia, General Medicine
Abstract

Aims: To define the peculiar features of patients with the deletion of the chromosome 20 long arm (del20q), data from 69 patients with myelodysplastic syndromes (MDSs) and isolated del20q, followed by the Gruppo Romano-Laziale Sindromi Mielodisplastiche (GROM-L) and Ospedale Torrette of Ancona, were collected and compared with those of 502 MDS patients with normal karyotype (NK-MDS). Results: Compared to the NK-MDS group, patients with del20q at diagnosis were older (p = 0.020) and mainly male (p = 0.006). They also had a higher rate of bone marrow blast < 5% (p = 0.004), a higher proportion of low and int-1 risk according to IPSS score (p = 0.023), and lower median platelet (PLT) count (p < 0.001). To date, in the del20q cohort, 21 patients (30.4%) received no treatment, 42 (61.0%) were treated with erythropoiesis-stimulating agents (ESA), 3 (4.3%) with hypomethylating agents, and 3 (4.3%) with other treatments. Among 34 patients evaluable for response to ESA, 21 (61.7%) achieved stable erythroid response according to IWG 2006 criteria and 13 (38.2%) were resistant. Nine patients (13.0%) progressed to acute myeloid leukaemia (AML) after a median time from diagnosis of 28 months (IR 4.1–51.7). The median overall survival (OS) of the entire cohort was 60.6 months (95% CI 54.7–66.4). the 5-year cumulative OS was 55.9% (95% CI 40.6–71.2). Conclusion: According to our results, we hypothesize that MDSs with isolated del 20q may represent a distinct biological entity, with peculiar clinical and prognostic features. The physio-pathological mechanisms underlying the deletion of the chromosome 20 long arm are still unclear and warrant future molecular analysis.

Published
2022

100. Risk Factors for Thrombosis in Patients Aged > 60 Years with Essential Thrombocythemia without Previous Thrombotic Events

Author

Roberto Latagliata, Alessandro Andriani, Massimo Breccia, Ida Carmosino, Federico Vozella, Atelda Romano, Annalisa Biagi, Luca Maurillo, Malgorzata Monika Trawinska, Katia Paciaroni, Michelina Santopietro, Sabrina Leonetti Crescenzi, Ada D'Addosio, Caterina Tatarelli, Elisabetta Abruzzese, Ambra Di Veroli, Elena Rossi, Valerio De Stefano, and Marco Montanaro

Subjects
Immunology, Cell Biology, Hematology, Biochemistry
Published
2022
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