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1. Acid sphingomyelinase deficiency (ASMD): addressing knowledge gaps in unmet needs and patient journey in Italy—a Delphi consensus

Author

Scarpa, Maurizio, Barbato, Antonio, Bisconti, Annalisa, Burlina, Alberto, Concolino, Daniela, Deodato, Federica, Di Rocco, Maja, Dionisi-Vici, Carlo, Donati, Maria Alice, Fecarotta, Simona, Fiumara, Agata, Galeone, Carlotta, Giona, Fiorina, Giuffrida, Gaetano, Manna, Raffaele, Mariani, Paolo, Pession, Andrea, Scopinaro, Annalisa, Spada, Marco, Spandonaro, Federico, Trifirò, Gianluca, Carubbi, Francesca, and Cappellini, Maria Domenica

Published
2023
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2. Management of children and adolescents with chronic myeloid leukemia in blast phase: International pediatric CML expert panel recommendations

Author

Sembill, Stephanie, Ampatzidou, Maria, Chaudhury, Sonali, Dworzak, Michael, Kalwak, Krzysztof, Karow, Axel, Kiani, Alexander, Krumbholz, Manuela, Luesink, Maaike, Naumann-Bartsch, Nora, De Moerloose, Barbara, Osborn, Michael, Schultz, Kirk R., Sedlacek, Petr, Giona, Fiorina, Zwaan, Christian Michel, Shimada, Hiroyuki, Versluijs, Birgitta, Millot, Frederic, Hijiya, Nobuko, Suttorp, Meinolf, and Metzler, Markus

Published
2023
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5. Recommendations for the management of acute immune thrombocytopenia in children. A Consensus Conference from the Italian Association of Pediatric Hematology and Oncology.

Author

Russo, Giovanna, Parodi, Emilia, Farruggia, Piero, Notarangelo, Lucia D., Perrotta, Silverio, Casale, Maddalena, Cesaro, Simone, Del Borrello, Giovanni, Del Vecchio, Giovanni C., Giona, Fiorina, Gorio, Chiara, Ladogana, Saverio, Lassandro, Giuseppe, Marzollo, Antonio, Maslak, Karolina, Miano, Maurizio, Nardi, Margherita, Palumbo, Giuseppe, Rossi, Francesca, and Spinelli, Marco

Published
2024
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6. Monitoring oral iron therapy in children with iron deficiency anemia: an observational, prospective, multicenter study of AIEOP patients (Associazione Italiana Emato-Oncologia Pediatrica)

Author

Russo, Giovanna, Guardabasso, Vincenzo, Romano, Francesca, Corti, Paola, Samperi, Piera, Condorelli, Annalisa, Sainati, Laura, Maruzzi, Matteo, Facchini, Elena, Fasoli, Silvia, Giona, Fiorina, Caselli, Désirée, Pizzato, Cristina, Marinoni, Maddalena, Boscarol, Gianluca, Bertoni, Elisa, Casciana, Maria Luisa, Tucci, Fabio, Capolsini, Ilaria, Notarangelo, Lucia Dora, Giordano, Paola, Ramenghi, Ugo, and Colombatti, Raffaella

Published
2020
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8. Long term use of eltrombopag in children with chronic immune thrombocytopenia: extended real life retrospective multicenter experience of the Italian Association of Pediatric Hematology and Oncology

Author

Giordano, Paola, primary, Lassandro, Giuseppe, additional, Barone, Angelica, additional, Cesaro, Simone, additional, Fotzi, Ilaria, additional, Giona, Fiorina, additional, Gorio, Chiara, additional, Maggio, Angela, additional, Miano, Maurizio, additional, Marzollo, Antonio, additional, Nardi, Margherita, additional, Pession, Andrea, additional, Ruggiero, Antonio, additional, Russo, Giovanna, additional, Saracco, Paola, additional, Spinelli, Marco, additional, Tolva, Alessandra, additional, Tornesello, Assunta, additional, Palladino, Valentina, additional, and Del Vecchio, Giovanni Carlo, additional

Published
2023
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9. Immune dysregulation in Kabuki syndrome: a case report of Evans syndrome and hypogammaglobulinemia

Author

Leonardi, Lucia, primary, Testa, Alessia, additional, Feleppa, Mariavittoria, additional, Paparella, Roberto, additional, Conti, Francesca, additional, Marzollo, Antonio, additional, Spalice, Alberto, additional, Giona, Fiorina, additional, Gnazzo, Maria, additional, Andreoli, Gian Marco, additional, Costantino, Francesco, additional, and Tarani, Luigi, additional

Published
2023
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10. Pediatric immune thrombocytopenia: a focus on eltrombopag as second-line therapy

Author

Palumbo, Giuseppe, primary, Farruggia, Piero, additional, Ramenghi, Ugo, additional, Russo, Giovanna, additional, Borchiellini, Alessandra, additional, Spinelli, Marco, additional, Dufour, Carlo, additional, Giona, Fiorina, additional, Ladogana, Saverio, additional, Zecca, Marco, additional, Perrotta, Silverio, additional, Pession, Andrea, additional, and Giordano, Paola, additional

Published
2023
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11. The Spectrum of Neurological and Sensory Abnormalities in Gaucher Disease Patients: A Multidisciplinary Study (SENOPRO)

Author

Tullo, Maria Giulia, primary, Cerulli Irelli, Emanuele, additional, Caramia, Francesca, additional, Tessari, Gianmarco, additional, Di Bonaventura, Carlo, additional, Turchetta, Rosaria, additional, Giallonardo, Anna Teresa, additional, Palumbo, Giovanna, additional, Bianchi, Simona, additional, Atturo, Francesca, additional, Nebbioso, Marcella, additional, Mancini, Patrizia, additional, Guariglia, Cecilia, additional, and Giona, Fiorina, additional

Published
2023
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13. Management of children and adolescents with chronic myeloid leukemia in blast phase:International pediatric CML expert panel recommendations

Author

Sembill, Stephanie, Ampatzidou, Maria, Chaudhury, Sonali, Dworzak, Michael, Kalwak, Krzysztof, Karow, Axel, Kiani, Alexander, Krumbholz, Manuela, Luesink, Maaike, Naumann-Bartsch, Nora, De Moerloose, Barbara, Osborn, Michael, Schultz, Kirk R., Sedlacek, Petr, Giona, Fiorina, Zwaan, Christian Michel, Shimada, Hiroyuki, Versluijs, Birgitta, Millot, Frederic, Hijiya, Nobuko, Suttorp, Meinolf, Metzler, Markus, Sembill, Stephanie, Ampatzidou, Maria, Chaudhury, Sonali, Dworzak, Michael, Kalwak, Krzysztof, Karow, Axel, Kiani, Alexander, Krumbholz, Manuela, Luesink, Maaike, Naumann-Bartsch, Nora, De Moerloose, Barbara, Osborn, Michael, Schultz, Kirk R., Sedlacek, Petr, Giona, Fiorina, Zwaan, Christian Michel, Shimada, Hiroyuki, Versluijs, Birgitta, Millot, Frederic, Hijiya, Nobuko, Suttorp, Meinolf, and Metzler, Markus

Abstract

Treatment of chronic myeloid leukemia has improved significantly with the introduction of tyrosine kinase inhibitors (TKIs), and treatment guidelines based on numerous clinical trials are available for chronic phase disease. However for CML in the blast phase (CML-BP), prognosis remains poor and treatment options are much more limited. The spectrum of treatment strategies for children and adolescents with CML-BP has largely evolved empirically and includes treatment principles derived from adult CML-BP and pediatric acute leukemia. Given this heterogeneity of treatment approaches, we formed an international panel of pediatric CML experts to develop recommendations for consistent therapy in children and adolescents with this high-risk disease based on the current literature and national standards. Recommendations include detailed information on initial diagnosis and treatment monitoring, differentiation from Philadelphia-positive acute leukemia, subtype-specific selection of induction therapy, and combination with tyrosine kinase inhibitors. Given that allogeneic hematopoietic stem cell transplantation currently remains the primary curative intervention for CML-BP, we also provide recommendations for the timing of transplantation, donor and graft selection, selection of a conditioning regimen and prophylaxis for graft-versus-host disease, post-transplant TKI therapy, and management of molecular relapse. Management according to the treatment recommendations presented here is intended to provide the basis for the design of future prospective clinical trials to improve outcomes for this challenging disease.

Published
2023

14. Acid sphingomyelinase deficiency (ASMD): addressing knowledge gaps in unmet needs and patient journey in Italy-a Delphi consensus

Author

Scarpa, M, Barbato, A, Bisconti, A, Burlina, A, Concolino, D, Deodato, F, Di Rocco, M, Dionisi-Vici, C, Donati, M, Fecarotta, S, Fiumara, A, Galeone, C, Giona, F, Giuffrida, G, Manna, R, Mariani, P, Pession, A, Scopinaro, A, Spada, M, Spandonaro, F, Trifirò, G, Carubbi, F, Cappellini, M, Scarpa, Maurizio, Barbato, Antonio, Bisconti, Annalisa, Burlina, Alberto, Concolino, Daniela, Deodato, Federica, Di Rocco, Maja, Dionisi-Vici, Carlo, Donati, Maria Alice, Fecarotta, Simona, Fiumara, Agata, Galeone, Carlotta, Giona, Fiorina, Giuffrida, Gaetano, Manna, Raffaele, Mariani, Paolo, Pession, Andrea, Scopinaro, Annalisa, Spada, Marco, Spandonaro, Federico, Trifirò, Gianluca, Carubbi, Francesca, Cappellini, Maria Domenica, Scarpa, M, Barbato, A, Bisconti, A, Burlina, A, Concolino, D, Deodato, F, Di Rocco, M, Dionisi-Vici, C, Donati, M, Fecarotta, S, Fiumara, A, Galeone, C, Giona, F, Giuffrida, G, Manna, R, Mariani, P, Pession, A, Scopinaro, A, Spada, M, Spandonaro, F, Trifirò, G, Carubbi, F, Cappellini, M, Scarpa, Maurizio, Barbato, Antonio, Bisconti, Annalisa, Burlina, Alberto, Concolino, Daniela, Deodato, Federica, Di Rocco, Maja, Dionisi-Vici, Carlo, Donati, Maria Alice, Fecarotta, Simona, Fiumara, Agata, Galeone, Carlotta, Giona, Fiorina, Giuffrida, Gaetano, Manna, Raffaele, Mariani, Paolo, Pession, Andrea, Scopinaro, Annalisa, Spada, Marco, Spandonaro, Federico, Trifirò, Gianluca, Carubbi, Francesca, and Cappellini, Maria Domenica

Abstract

Acid sphingomyelinase deficiency (ASMD) is an ultra-rare disease, and several gaps of knowledge on various issues remain, particularly at a regional/national level. Expert opinions collected through well-defined consensus methodologies are increasingly used to make available reliable information in the context of rare/ultra-rare diseases. With the aim to provide indications on infantile neurovisceral ASMD (also formerly known as Niemann–Pick disease type A), chronic neurovisceral ASMD (formerly known as Niemann–Pick disease type A/B) and chronic visceral ASMD (formerly known as Niemann–Pick disease type B) in Italy, we conducted a Delphi consensus of experts focused on five main areas: (i) patients and disease characteristics; (ii) unmet needs and quality of life; (iii) diagnostic issues; (iv) treatment-related aspects; and (v) patient journey. Pre-specified, objective criteria were used to outline the multidisciplinary panel, based on 19 Italian experts in ASMD in paediatric and adult patients from different Italian Regions, including both clinicians (n = 16) and ASMD patients’ advocacy or payors with expertise in rare diseases (n = 3). During two Delphi rounds, a high ratio of agreement was found on several topics related to ASMD characteristics, diagnosis, management and disease burden. Our findings may provide valuable indications for management of ASMD at a public health level in Italy.

Published
2023

15. Long term use of eltrombopag in children with chronic immune thrombocytopenia: extended real life retrospective multicenter experience of the Italian Association of Pediatric Hematology and Oncology

Author

Giordano, Paola, Lassandro, Giuseppe, Barone, Angelica, Cesaro, Simone, Fotzi, Ilaria, Giona, Fiorina, Gorio, Chiara, Maggio, Angela, Miano, Maurizio, Marzollo, Antonio, Nardi, Margherita, Pession, Andrea, Ruggiero, Antonio, Russo, Giovanna, Saracco, Paola, Spinelli, Marco, Tolva, Alessandra, Tornesello, Assunta, Palladino, Valentina, Del Vecchio, Giovanni Carlo, Ruggiero, Antonio (ORCID:0000-0002-6052-3511), Tornesello, Assunta (ORCID:0000-0002-7485-7440), Giordano, Paola, Lassandro, Giuseppe, Barone, Angelica, Cesaro, Simone, Fotzi, Ilaria, Giona, Fiorina, Gorio, Chiara, Maggio, Angela, Miano, Maurizio, Marzollo, Antonio, Nardi, Margherita, Pession, Andrea, Ruggiero, Antonio, Russo, Giovanna, Saracco, Paola, Spinelli, Marco, Tolva, Alessandra, Tornesello, Assunta, Palladino, Valentina, Del Vecchio, Giovanni Carlo, Ruggiero, Antonio (ORCID:0000-0002-6052-3511), and Tornesello, Assunta (ORCID:0000-0002-7485-7440)

Abstract

BackgroundThe present multicenter retrospective study on eltrombopag administration in Italian children with chronic ITP aims to extend follow-up of our previous study. Materials and methodsThis retrospective multicenter study was conducted in 17 centers affiliated to the Italian Association of Pediatric Hematology and Oncology (AIEOP). Patients were classified into three subgroups: group 1 included patients who discontinued treatment due to a stable platelet count; group 2 included patients who discontinued treatment due to ineffectiveness; group 3 included patients who did not permanently discontinue treatment. Results56 patients were eligible for analysis. The median duration of eltrombopag treatment was 40 months (7-71 months). Twenty patients (36%) discontinued permanently eltrombopag. The reasons of permanent discontinuation were adverse effects (n = 1), inefficacy (n = 10), stable platelet count (n = 9). All patients of group 1 maintained a durable response without additional treatments after eltrombopag discontinuation. We found that patients of group 2 were on treatment for less time (median treatment time: 13.5 months, min: 6.0 - max: 56.0) than patients of group 1 (median treatment time: 34 months, min: 16.0 - max: 62.0) (p < 0.05). Patients of group 2 mostly did not achieve a stable platelet count in the first 6 months of treatment and underwent concomitant therapies during follow-up respect of group 1 and group 3 (p < 0.01). ConclusionOur study found that the benefits of eltrombopag treatment, in terms of platelet count improvement and use of additional therapies, are identifiable from the first 6 months of treatment.

Published
2023

16. GAU-PED study for early diagnosis of Gaucher disease in children with splenomegaly and cytopenia

Author

Pession, Andrea, Di Rocco, Maja, Venturelli, Francesco, Tappino, Barbara, Morello, William, Santoro, Nicola, Giordano, Paola, Filippini, Beatrice, Rinieri, Simona, Russo, Giovanna, Girardi, Katia, Ruggiero, Antonio, Galea, Eulalia, Antonucci, Roberto, Tovaglieri, Nicola, Porta, Fulvio, Tartaglione, Immacolata, Giona, Fiorina, Fagioli, Franca, Burlina, Alberto, Ruggiero, Antonio (ORCID:0000-0002-6052-3511), Pession, Andrea, Di Rocco, Maja, Venturelli, Francesco, Tappino, Barbara, Morello, William, Santoro, Nicola, Giordano, Paola, Filippini, Beatrice, Rinieri, Simona, Russo, Giovanna, Girardi, Katia, Ruggiero, Antonio, Galea, Eulalia, Antonucci, Roberto, Tovaglieri, Nicola, Porta, Fulvio, Tartaglione, Immacolata, Giona, Fiorina, Fagioli, Franca, Burlina, Alberto, and Ruggiero, Antonio (ORCID:0000-0002-6052-3511)

Abstract

BackgroundGaucher disease (GD) diagnosis can be delayed due to non-specific symptoms and lack of awareness, leading to unnecessary procedures and irreversible complications. GAU-PED study aims to assess GD prevalence in a high-risk pediatric population and the presence, if any, of novel clinical or biochemical markers associated with GD.Materials and methodsDBS samples were collected and tested for beta-glucocerebrosidase enzyme activity for 154 patients selected through the algorithm proposed by Di Rocco et al. Patients showing beta-glucocerebrosidase activity below normal values were recalled to confirm the enzyme deficiency with the gold standard essay on cellular homogenate. Patients tested positive at the gold standard analysis were evaluated through GBA1 gene sequencing.Results14 out of 154 patients were diagnosed with GD, with a prevalence of 9.09% (5.06-14.78%, CI 95%). Hepatomegaly, thrombocytopenia, anemia, growth delay/deceleration, elevated serum ferritin, elevated Lyso-Gb1 and chitotriosidase were significantly associated with GD.ConclusionsGD prevalence in a pediatric population at high-risk appeared to be higher compared to high-risk adults. Lyso-Gb1 was associated with GD diagnosis. The algorithm proposed by Di Rocco et al. can potentially improve the diagnostic accuracy of pediatric GD, allowing the prompt start of therapy, aiming to reduce irreversible complications.

Published
2023

17. “CHildren with Inherited Platelet disorders Surveillance” (CHIPS) retrospective and prospective observational cohort study by Italian Association of Pediatric Hematology and Oncology (AIEOP)

Author

Lassandro, Giuseppe, primary, Palladino, Valentina, additional, Faleschini, Michela, additional, Barone, Angelica, additional, Boscarol, Gianluca, additional, Cesaro, Simone, additional, Chiocca, Elena, additional, Farruggia, Piero, additional, Giona, Fiorina, additional, Gorio, Chiara, additional, Maggio, Angela, additional, Marinoni, Maddalena, additional, Marzollo, Antonio, additional, Palumbo, Giuseppe, additional, Russo, Giovanna, additional, Saracco, Paola, additional, Spinelli, Marco, additional, Verzegnassi, Federico, additional, Morga, Francesca, additional, Savoia, Anna, additional, and Giordano, Paola, additional

Published
2022
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18. Real-World Management of Advanced Systemic Mastocytosis Treated with Midostaurin: Analysis of Patients Who Completed 12 Months of Follow-up from an Italian Observational Study (OVIDIO)

Author

Papayannidis, Cristina, primary, Mannelli, Francesco, additional, Crosera, Lara, additional, Parente, Roberta, additional, Romano, Alessandra, additional, Rondoni, Michela, additional, Elena, Chiara, additional, Criscuolo, Marianna, additional, Di Renzo, Nicola, additional, Giona, Fiorina, additional, Pane, Fabrizio, additional, Cilloni, Daniela, additional, Elli, Elena Maria, additional, Miglino, Maurizio, additional, Mazza, Patrizio, additional, Malato, Alessandra, additional, Pochintesta, Lara, additional, Bini, Roberta, additional, Valsecchi, Diletta, additional, and Grifoni, Federica Irene, additional

Published
2022
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19. Is Gaucher Disease Type 1 a Neuronopathic Disorder? Neuroradiological and Clinical Findings from Senopro_Gaucher Study

Author

Bianchi, Simona, primary, Tullo, Maria Giulia, additional, Caramia, Francesca, additional, Cerulli Irelli, Emanuele, additional, Tessari, Gianmarco, additional, Di Bonaventura, Carlo, additional, Turchetta, Rosaria, additional, Palumbo, Giovanna, additional, Nebbioso, Marcella, additional, Guariglia, Cecilia, additional, and Giona, Fiorina, additional

Published
2022
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22. Peripherally Inserted Central Venous Catheter for Pediatric Hematologic Diseases; A Retrospective 11-year Single-center Experience.

Author

Ligia, Silvio, primary, Morano, Salvatore Giacomo, additional, Kaiser, Francesca, additional, Micozzi, Alessandra, additional, Moleti, Maria Luisa, additional, Barberi, Walter, additional, Giona, Fiorina, additional, Chistolini, Antonio, additional, Arena, Valentina, additional, Piciocchi, Alfonso, additional, Forgione, Maurizio, additional, Gasperini, Giulia, additional, Fabbri, Marco, additional, and Testi, Anna Maria, additional

Published
2022
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23. Early response does not predict outcome in children and adolescents with chronic myeloid leukaemia treated with high‐dose imatinib

Author

Giona, Fiorina, Saglio, Giuseppe, Santopietro, Michelina, Menna, Giuseppe, Putti, Maria Caterina, Micalizzi, Concetta, Iaria, Grazia, Santoro, Nicola, Ladogana, Saverio, Mura, Rosamaria, Burnelli, Roberta, Consarino, Caterina, Cosmi, Carlo, Moleti, Maria Luisa, Leszl, Anna, Tucci, Francesca, Nanni, Mauro, Diverio, Daniela, Biondi, Andrea, Locatelli, Franco, and Foà, Robin

Published
2018
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24. ETV6-related thrombocytopenia: dominant negative effect of mutations as common pathogenic mechanism

Author

Faleschini, Michela, primary, Ammeti, Daniele, additional, Papa, Nicole, additional, Alfano, Caterina, additional, Bottega, Roberta, additional, Fontana, Giorgia, additional, Capaci, Valeria, additional, Zanchetta, Melania E., additional, Pozzani, Federico, additional, Montanari, Francesca, additional, Petroni, Valeria, additional, Giordano, Paola, additional, Noris, Patrizia, additional, Giona, Fiorina, additional, and Savoia, Anna, additional

Published
2022
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27. Transfusional Approach in Multi-Ethnic Sickle Cell Patients: Real-World Practice Data From a Multicenter Survey in Italy

Author

Graziadei, Giovanna, primary, De Franceschi, Lucia, additional, Sainati, Laura, additional, Venturelli, Donatella, additional, Masera, Nicoletta, additional, Bonomo, Piero, additional, Vassanelli, Aurora, additional, Casale, Maddalena, additional, Lodi, Gianluca, additional, Voi, Vincenzo, additional, Rigano, Paolo, additional, Pinto, Valeria Maria, additional, Quota, Alessandra, additional, Notarangelo, Lucia D., additional, Russo, Giovanna, additional, Allò, Massimo, additional, Rosso, Rosamaria, additional, D'Ascola, Domenico, additional, Facchini, Elena, additional, Macchi, Silvia, additional, Arcioni, Francesco, additional, Bonetti, Federico, additional, Rossi, Enza, additional, Sau, Antonella, additional, Campisi, Saveria, additional, Colarusso, Gloria, additional, Giona, Fiorina, additional, Lisi, Roberto, additional, Giordano, Paola, additional, Boscarol, Gianluca, additional, Filosa, Aldo, additional, Marktel, Sarah, additional, Maroni, Paola, additional, Murgia, Mauro, additional, Origa, Raffaella, additional, Longo, Filomena, additional, Bortolotti, Marta, additional, Colombatti, Raffaella, additional, Di Maggio, Rosario, additional, Mariani, Raffaella, additional, Piperno, Alberto, additional, Corti, Paola, additional, Fidone, Carmelo, additional, Palazzi, Giovanni, additional, Badalamenti, Luca, additional, Gianesin, Barbara, additional, Piel, Frédéric B., additional, and Forni, Gian Luca, additional

Published
2022
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29. Brain Volume Alterations Using 3tesla Magnetic Resonance and Neuropsychological Findings in Gaucher Disease Patients

Author

Tullo, Maria Giulia, primary, Caramia, Francesca, additional, Tessari, Gianmarco, additional, Di Bonaventura, Carlo, additional, Turchetta, Rosaria, additional, Cerulli Irelli, Emanuele, additional, Giallonardo, Anna Teresa, additional, Plateroti, Rocco, additional, Palumbo, Giovanna, additional, Monaco, Nicola, additional, Filipponi, Valeria, additional, Nebbioso, Marcella, additional, Mancini, Patrizia, additional, Guariglia, Cecilia, additional, and Giona, Fiorina, additional

Published
2021
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32. Long-term results of high-dose imatinib in children and adolescents with chronic myeloid leukaemia in chronic phase: the Italian experience

Author

Giona, Fiorina, Putti, Maria C., Micalizzi, Concetta, Menna, Giuseppe, Moleti, Maria L., Santoro, Nicola, Iaria, Grazia, Ladogana, Saverio, Burnelli, Roberta, Consarino, Caterina, Varotto, Stefania, Tucci, Francesca, Messina, Chiara, Nanni, Mauro, Diverio, Daniela, Biondi, Andrea, Pession, Andrea, Locatelli, Franco, Piciocchi, Alfonso, Gottardi, Enrico, Saglio, Giuseppe, and Foà, Robin

Published
2015
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35. Genetic screening of children with marrow failure. The role of primary Immunodeficiencies

Author

Miano, Maurizio, primary, Grossi, Alice, additional, Dell'Orso, Gianluca, additional, Lanciotti, Marina, additional, Fioredda, Francesca, additional, Palmisani, Elena, additional, Lanza, Tiziana, additional, Guardo, Daniela, additional, Beccaria, Andrea, additional, Ravera, Silvia, additional, Cossu, Vanessa, additional, Terranova, Paola, additional, Giona, Fiorina, additional, Santopietro, Michelina, additional, Cappelli, Enrico, additional, Ceccherini, Isabella, additional, and Dufour, Carlo, additional

Published
2021
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37. EHA2021 Virtual Congress Abstract Book

Author

Angela, Guarina, Angelica, Barone, Assunta, Tornesello, Maddalena, Marinoni, Giovanna, Russo, Giuseppe, Lassandro, Giordano, Paola, Milena, Motta, Marco, Spinelli, Ilaria, Fontanili, Giona, Fiorina, Giuseppe, Menna, Elena, Chiocca, Ilaria, Fotzi, Angela, Petrone, Giuseppe, Puccio, Michele, Citrano, and Piero, Farruggia

Subjects
Cytopenia, Acute leukemia, medicine.medical_specialty, ARDS, Chemotherapy, business.industry, medicine.medical_treatment, Myeloid leukemia, Hematology, medicine.disease, Abstract Book, Transplantation, Leukemia, Internal medicine, medicine, Extracorporeal membrane oxygenation, business
Abstract

Background: Patients with hematologic malignancies appear to have a greater risk of SARS-CoV-2 infection and severe disease due to myelosuppression;furthermore, delays in treatment of patients with hematologic malignancies, especially those with acute leukemia planned for chemotherapy or transplantation, are associated with a risk of disease progression. To date, some societies recommend that chemotherapy should generally not be started until COVID-19 symptoms have completely resolved and viral testing becomes negative. However, implications of the aforementioned recommendations remain uncertain in routine clinical practice, and data on COVID-19 in patients with hematologic malignancies are still limited. Aims: We carried out a single-center analysis of acute leukemia inpatients who developed COVID-19 during the last year. Methods: Since February 2020 to February 2021, a total of 310 Hospitalizations,63 adult patients were admitted in our Center for treatment of hematologic malignancies. The indication for admission was acute myeloid leukemia (AML) in 50 (30%) patients, acute lymphoblastic leukemia (ALL) in4 (8%), lymphoma in 54 (33%), myeloma in4 (8%), other 31 (19%). Diagnosis of SARS-CoV-2 infection was based on virus detection by real-time polymerase chain reaction (SARS-CoV-2 E-gene RT-PCR) in respiratory tract specimens. Standard preventive measures for SARS-CoV-2 infection control were applied to all patients care, accordance with National disease control and prevention guidelines. Results: Ten (6%) patients tested positive for SARS-CoV-2 via PCR in a unique Covid-19 outbreak during hospitalization stay, and they were immediately transferred to Covid Infectious Unit. All these patients, were affected by Acute Leukemia (8 pts AML, 2 pts ALL ph negative), the majority of them was in peak of cytopenia at the Covid-19 infection time. Nine patients had been treated with intensive chemotherapy before SARS-CoV-2 confirmation. At SARS-CoV-2 diagnosis, only one patient had untreated, newly diagnosed AML;3 patients had refractory/relapsed AML. One patient was in complete remission with incomplete hematologic recovery (CRi). Deep vein thrombosis complicated by pulmonary embolism and interstitial pneumonia was observed in a patients despite anticoagulation and in thrombocytopenia. After SARS-CoV-2 infection, no leukemia-specific treatment was adjusted. Three patients (30%) died due to severe acute respiratory distress syndrome (ARDS) despite extracorporeal membrane oxygenation (ECMO) in deep aplasia, all of them was in refractory disease. Seven patients delayed in chemotherapy treatment for a media of 34 days;chemotherapy started until COVID-19 symptoms have completely resolved and two viral testing becomes negative. However, these patients are still alive and maintained their complete remission, remaining for long-time negative for SARS-CoV-2. One patient underwent to bone marrow transplantation Summary/Conclusion: Our findings support the vulnerability of patients with hematologic malignancies in the COVID-19 pandemic, and we reported a high COVID-19 infection mortality of 30%, in accordance with other hematological case series. However, deaths owing to Covid-19 were observed in patients in disease leukemia progression;furthermore, our recovered COVID-19 leukemia patients remained negative for SARSCoV- 2 after delivery of chemotherapy, and underwent to their following chemotherapy and allo-BMT program without any other complications.

Published
2021

40. Abstracts from the 36th Annual Meeting of the Histiocyte Society

Author

Daniela De Benedittis, Mohamed, Sara, Rizzo, Lorenzo, Santopietro, Michelina, Palumbo, Giovanna, Luisa, Cardarelli, DI PIPPO, Tiziana, Roggini, Mario, and Giona, Fiorina

Subjects
Oncology, Pediatrics, Perinatology and Child Health, Hematology
Published
2020

41. Advanced mast cell disease: an Italian Hematological Multicenter experience

Author

Pagano, Livio, Valentini, Caterina Giovanna, Caira, Morena, Rondoni, Michela, Van Lint, Maria Teresa, Candoni, Anna, Allione, Bernardino, Cattaneo, Chiara, Marbello, Laura, Caramatti, Cecilia, Pogliani, Enrico Maria, Iannitto, Emilio, Giona, Fiorina, Ferrara, Felicetto, Invernizzi, Rosangela, Fanci, Rosa, Lunghi, Monia, Fianchi, Luana, Sanpaolo, Grazia, Stefani, Pietro Maria, Pulsoni, Alessandro, Martinelli, Giovanni, Leone, Giuseppe, and Musto, Pellegrino

Published
2008
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42. Mast cell leukemia: a report of ten cases

Author

Valentini, Caterina Giovanna, Rondoni, Michela, Pogliani, Enrico Maria, Van Lint, Maria Teresa, Cattaneo, Chiara, Marbello, Laura, Pulsoni, Alessandro, Giona, Fiorina, Martinelli, Giovanni, Leone, Giuseppe, and Pagano, Livio

Published
2008
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43. Association of Immune Thrombocytopenia and Inflammatory Bowel Disease in Children

Author

Guarina, Angela, primary, Barone, Angelica, additional, Tornesello, Assunta, additional, Marinoni, Maddalena, additional, Lassandro, Giuseppe, additional, Giordano, Paola, additional, Motta, Milena, additional, Spinelli, Marco, additional, Fontanili, Ilaria, additional, Giona, Fiorina, additional, Menna, Francesco, additional, Chiocca, Elena, additional, Fotzi, Ilaria, additional, Petrone, Angelamaria, additional, Graziano, Francesco, additional, Saracco, Paola, additional, Puccio, Giuseppe, additional, Citrano, Michele, additional, Russo, Giovanna, additional, and Farruggia, Piero, additional

Published
2021
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44. Impact of SARS-CoV-2 infection on Gaucher disease patients in Italy

Author

Cappellini, Maria Domenica, primary, Barbato, Antonio, additional, Carubbi, Francesca, additional, Di Rocco, Maja, additional, Giona, Fiorina, additional, Giuffrida, Gaetano, additional, Linari, Silvia, additional, Quarta, Antonella, additional, Scarpa, Maurizio, additional, and Spada, Marco, additional

Published
2021
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45. Genetica della trombocitopenia cronica nei bambini

Author

Cappellini, MARIA DOMENICA, Francesca, Carubbi, Maja Di Rocco, Giona, Fiorina, and Gaetano, Giuffrida

Subjects
imiglucerase, Gaucher disease, imiglucerase, Gaucher Registry, Gaucher disease, Gaucher Registry
Published
2020

46. Vaccinazione Antinfluenzale in soggetti con Asplenia: warning in tempo di Coronavirus

Author

Di Maio Nicoletta, Barella, Susanna, Graziadei, Giovanna, Forni, Gianluca, Perrotta, Silverio, Allò, Massimo, Sau, Antonella, Balocco, Manuela, Di Concilio Rosanna, Rivellini, Flavia, Facchini, Elena, Peluso, Angelo, Russo, Giovanna, Rigoli, Luciana, Farruggia, Piero, Campisi, Saveria, Casini, Tommaso, Boscarol, Gianluca, Capolsini, Ilaria, Colombatti, Raffaella, Grotto, Paolo, Giona, Fiorina, Lazzareschi, Ilaria, Pugliese, Pellegrina, Fioredda, Francesca, Notarangelo Lucia Dora, Fasoli, Silvia, Putti Maria Caterina, Casciana Maria Luisa, Migliavacca, Maddalena, Corti, Paola, Tripodi, Serena, Saracco, Paola, Ferrero, Simone, Tornesello, Assunta, Serra, Marilena, Ladogana, Saverio, Palazzi, Giovanni, Verzegnassi, Federico, Baronci, Carlo, Palumbo, Giuseppe, Cesaro, Simone, and Casale, Maddalena

Published
2020

49. Management strategies for newly diagnosed immune thrombocytopenia in Italian AIEOP Centres: do we overtreat? Data from a multicentre, prospective cohort study

Author

Parodi, Emilia, Russo, Giovanna, Farruggia, Piero, Notarangelo, Lucia D., Giraudo, Maria T., Nardi, Margherita, Giona, Fiorina, Giordano, Paola, Ramenghi, Ugo, Barone, Angelica, Boscarol, Gianluca, Cesaro, Simone, Fioredda, Francesca, Ladogana, Saverio, Licciardello, Maria, Rossi, Francesca, Rubert, Laura, Spinelli, Marco, and Tucci, Fabio

Subjects
Male, p&lt, 56 patients had chronic ITP, which was significantly associated with younger age. Chronicity at 12 months was not affected by different therapeutic approaches at diagnosis or response to therapy, thrombocytopenia, 0.001) . At 1 year, 95% confidence interval: 1.09-2.98). In total, Background - The aim of the present study was to assess management strategies for immune thrombocytopenia (ITP) among Italian paediatric haematologists, and to compare these with those of recent international guidelines. Predictors of early remission or disease chronicity were also evaluated. Materials and methods - During a period of 1 year, 205 children (age: 1 month-18 years) with newly diagnosed ITP were prospectively enrolled by 16 centres belonging to the Italian Association of Paediatric Haematology and Oncology (AIEOP). We collected the subjects demographic data, history, clinical symptoms, platelet count and treatment at presentation and at subsequent visits. Results - Of the 205 patients, 47 (23%) were initially managed with a wait-and-see approach. Compared to these patients, children administered platelet-enhancing therapies were significantly younger (median age: 4.75 vs 7.96 years, p<0.001) and had lower platelet counts. At the 3-month follow-up, 92/202 patients (46%) had persistent ITP. Recovery within 3 months was predicted by younger median age (5.3 vs 7.8 years, p<0.001), and recent viral infection (p<0.001) . At 1 year, 56 patients had chronic ITP, which was associated with older median age (7.54 vs 5.35 years, p<0.001), and a family history of autoimmunity (p<0.05, relative risk: 1.81, 95% confidence interval: 1.09-2.98). In total, 357 pharmacological treatments were recorded (216 intravenous immunoglobulins, 80 steroids). Response to intravenous immunoglobulins did not have an effect on remission rate at 12 months. Discussion - Pediatric hematologists in Italian Centre treat over three-quarters of patients with newly diagnosed ITP, despite recent international guidelines. Almost 80% of patients with mild clinical symptoms received pharmacological treatment at diagnosis, which was significantly associated with younger age. Chronicity at 12 months was not affected by different therapeutic approaches at diagnosis or response to therapy, Prospective Studies, Child, Children, clinical symptoms, which was associated with older median age (7.54 vs 5.35 years, treatment, Background - The aim of the present study was to assess management strategies for immune thrombocytopenia (ITP) among Italian paediatric haematologists, Immunoglobulins, Intravenous, Italy, 80 steroids). Response to intravenous immunoglobulins did not have an effect on remission rate at 12 months. Discussion - Pediatric hematologists in Italian Centre treat over three-quarters of patients with newly diagnosed ITP, Child, Preschool, Practice Guidelines as Topic, Female, Steroids, Guideline Adherence, history, thrombocytopenia, pediatric, treatment, and to compare these with those of recent international guidelines. Predictors of early remission or disease chronicity were also evaluated. Materials and methods - During a period of 1 year, 0.001), Adolescent, platelet count and treatment at presentation and at subsequent visits. Results - Of the 205 patients, 205 children (age: 1 month-18 years) with newly diagnosed ITP were prospectively enrolled by 16 centres belonging to the Italian Association of Paediatric Haematology and Oncology (AIEOP). We collected the subjects demographic data, despite recent international guidelines. Almost 80% of patients with mild clinical symptoms received pharmacological treatment at diagnosis, Humans, IVIG, Purpura, Thrombocytopenic, Idiopathic, Platelet Count, 0.05, 357 pharmacological treatments were recorded (216 intravenous immunoglobulins, Immune thrombocytopenia, ITP, Infant, 47 (23%) were initially managed with a wait-and-see approach. Compared to these patients, 92/202 patients (46%) had persistent ITP. Recovery within 3 months was predicted by younger median age (5.3 vs 7.8 years, and recent viral infection (p&lt, pediatric, Others, and a family history of autoimmunity (p&lt, 0.001) and had lower platelet counts. At the 3-month follow-up, Follow-Up Studies, children administered platelet-enhancing therapies were significantly younger (median age: 4.75 vs 7.96 years
Abstract

BACKGROUND: The aim of the present study was to assess management strategies for immune thrombocytopenia (ITP) among Italian paediatric haematologists, and to compare these with those of recent international guidelines. Predictors of early remission or disease chronicity were also evaluated. MATERIALS AND METHODS: During a period of 1 year, 205 children (age: 1 month-18 years) with newly diagnosed ITP were prospectively enrolled by 16 centres belonging to the Italian Association of Paediatric Haematology and Oncology (AIEOP). We collected the subjects demographic data, history, clinical symptoms, platelet count and treatment at presentation and at subsequent visits. RESULTS: Of the 205 patients, 47 (23%) were initially managed with a wait-and-see approach. Compared to these patients, children administered platelet-enhancing therapies were significantly younger (median age: 4.75 vs 7.96 years; p

Published
2020

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