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2. 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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3. Outcome of Children and Adolescents with Recurrent Classical Hodgkin Lymphoma: The Italian Experience

Author

Garaventa, Alberto, primary, Parodi, Stefano, additional, Guerrini, Giulia, additional, Farruggia, Piero, additional, Sala, Alessandra, additional, Pillon, Marta, additional, Buffardi, Salvatore, additional, Rossi, Francesca, additional, Bianchi, Maurizio, additional, Zecca, Marco, additional, Vinti, Luciana, additional, Facchini, Elena, additional, Casini, Tommaso, additional, Bernasconi, Sayla, additional, Amoroso, Loredana, additional, D’Amico, Salvatore, additional, Provenzi, Massimo, additional, De Santis, Raffaela, additional, Sau, Antonella, additional, Muggeo, Paola, additional, Mura, Rosa Maria, additional, Haupt, Riccardo, additional, Mascarin, Maurizio, additional, and Burnelli, Roberta, additional

Published
2022
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4. Risk factors for endocrine complications in transfusion-dependent thalassemia patients on chelation therapy with deferasirox: a risk assessment study from a multicentre nation-wide cohort

Author

Casale, M, Forni, G, Cassinerio, E, Pasquali, D, Origa, R, Serra, M, Campisi, S, Peluso, A, Renni, R, Cattoni, A, De Michele, E, Allò, M, Poggi, M, Ferrara, F, Di Concilio, R, Sportelli, F, Quarta, A, Putti, M, Notarangelo, L, Sau, A, Ladogana, S, Tartaglione, I, Picariello, S, Marcon, A, Sturiale, P, Roberti, D, Lazzarino, A, Perrotta, S, Casale, Maddalena, Forni, Gian Luca, Cassinerio, Elena, Pasquali, Daniela, Origa, Raffaella, Serra, Marilena, Campisi, Saveria, Peluso, Angelo, Renni, Roberta, Cattoni, Alessandro, De Michele, Elisa, Allò, Massimo, Poggi, Maurizio, Ferrara, Francesca, Di Concilio, Rosanna, Sportelli, Filomena, Quarta, Antonella, Putti, Maria Caterina, Notarangelo, Lucia Dora, Sau, Antonella, Ladogana, Saverio, Tartaglione, Immacolata, Picariello, Stefania, Marcon, Alessia, Sturiale, Patrizia, Roberti, Domenico, Lazzarino, Antonio Ivan, Perrotta, Silverio, Casale, M, Forni, G, Cassinerio, E, Pasquali, D, Origa, R, Serra, M, Campisi, S, Peluso, A, Renni, R, Cattoni, A, De Michele, E, Allò, M, Poggi, M, Ferrara, F, Di Concilio, R, Sportelli, F, Quarta, A, Putti, M, Notarangelo, L, Sau, A, Ladogana, S, Tartaglione, I, Picariello, S, Marcon, A, Sturiale, P, Roberti, D, Lazzarino, A, Perrotta, S, Casale, Maddalena, Forni, Gian Luca, Cassinerio, Elena, Pasquali, Daniela, Origa, Raffaella, Serra, Marilena, Campisi, Saveria, Peluso, Angelo, Renni, Roberta, Cattoni, Alessandro, De Michele, Elisa, Allò, Massimo, Poggi, Maurizio, Ferrara, Francesca, Di Concilio, Rosanna, Sportelli, Filomena, Quarta, Antonella, Putti, Maria Caterina, Notarangelo, Lucia Dora, Sau, Antonella, Ladogana, Saverio, Tartaglione, Immacolata, Picariello, Stefania, Marcon, Alessia, Sturiale, Patrizia, Roberti, Domenico, Lazzarino, Antonio Ivan, and Perrotta, Silverio

Abstract

Transfusion-dependent patients typically develop iron-induced cardiomyopathy, liver disease, and endocrine complications. We aimed to estimate the incidence of endocrine disorders in transfusiondependent thalassemia (TDT) patients during long-term iron-chelation therapy with deferasirox (DFX). We developed a multi-center follow-up study of 426 TDT patients treated with once-daily DFX for a median duration of 8 years, up to 18.5 years. At baseline, 118, 121, and 187 patients had 0, 1, or ≥2 endocrine diseases respectively. 104 additional endocrine diseases were developed during the follow-up. The overall risk of developing a new endocrine complication within 5 years was 9.7% (95% Confidence Interval [CI]: 6.3.13.1). Multiple Cox regression analysis identified three key predictors: Age showed a positive log-linear effect (adjusted hazard ratio [HR] for 50% increase 1.2, 95% CI: 1.1.1.3, P=0.005), the serum concentration of thyrotropin showed a positive linear effect (adjusted HR for 1 mIU/L increase 1.3, 95% CI: 1.1.1.4, P<0.001) regardless the kind of disease incident, while the number of previous endocrine diseases showed a negative linear effect: The higher the number of diseases at baseline the lower the chance of developing further diseasess (adjusted HR for unit increase 0.5, 95% CI: 0.4-0.7, P<0.001). Age and thyrotropin had similar effect sizes across the categories of baseline diseases. The administration of levothyroxine as a covariate did not change the estimates. Although in DFX-treated TDT patients the risk of developing an endocrine complication is generally lower than the previously reported risk, there is considerable risk variation and the burden of these complications remains high. We developed a simple risk score chart enabling clinicians to estimate their patients' risk. Future research will look at increasing the amount of variation explained from our model and testing further clinical and laboratory predictors, including the assessment of d

Published
2021

5. Risk factors for endocrine complications in transfusion-dependent thalassemia patients on chelation therapy with deferasirox: a risk assessment study from a multi-center nation-wide cohort

Author

Casale, Maddalena, primary, Forni, Gian Luca, additional, Cassinerio, Elena, additional, Pasquali, Daniela, additional, Origa, Raffaella, additional, Serra, Marilena, additional, Campisi, Saveria, additional, Peluso, Angelo, additional, Renni, Roberta, additional, Cattoni, Alessandro, additional, De Michele, Elisa, additional, Allò, Massimo, additional, Poggi, Maurizio, additional, Ferrara, Francesca, additional, Di Concilio, Rosanna, additional, Sportelli, Filomena, additional, Quarta, Antonella, additional, Putti, Maria Caterina, additional, Notarangelo, Lucia Dora, additional, Sau, Antonella, additional, Ladogana, Saverio, additional, Tartaglione, Immacolata, additional, Picariello, Stefania, additional, Marcon, Alessia, additional, Sturiale, Patrizia, additional, Roberti, Domenico, additional, Lazzarino, Antonio Ivan, additional, and Perrotta, Silverio, additional

Published
2021
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6. Influenza Vaccination in Asplenia: Improving Quality of Care in Time of Coronavirus

Author

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

Published
2020
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7. Comparison of Hodgkin’s Lymphoma in Children and Adolescents. A Twenty Year Experience with MH’96 and LH2004 AIEOP (Italian Association of Pediatric Hematology and Oncology) Protocols

Author

Burnelli, Roberta, primary, Fiumana, Giulia, additional, Rondelli, Roberto, additional, Pillon, Marta, additional, Sala, Alessandra, additional, Garaventa, Alberto, additional, D’Amore, Emanuele S.G., additional, Sabattini, Elena, additional, Buffardi, Salvatore, additional, Bianchi, Maurizio, additional, Vinti, Luciana, additional, Zecca, Marco, additional, Muggeo, Paola, additional, Provenzi, Massimo, additional, Farruggia, Piero, additional, Rossi, Francesca, additional, D’Amico, Salvatore, additional, Facchini, Elena, additional, Bernasconi, Sayla, additional, De Santis, Raffaela, additional, Casini, Tommaso, additional, Porta, Fulvio, additional, D’Alba, Irene, additional, Mura, Rosamaria, additional, Verzegnassi, Federico, additional, Sau, Antonella, additional, Cesaro, Simone, additional, Perruccio, Katia, additional, Cellini, Monica, additional, Bertolini, Patrizia, additional, Sperlì, Domenico, additional, Pericoli, Roberta, additional, Galimberti, Daniela, additional, Civino, Adele, additional, and Mascarin, Maurizio, additional

Published
2020
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8. 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

9. Retrospective and Prospective Study of Childhood Autoimmune Hemolytic Anemia. a Preliminary Report from the Red Cell Working Group of the Paediatric Hemato-Oncology Italian Associations (AIEOP)

Author

Ladogana, Saverio, primary, Colombatti, Raffaella, primary, Perrotta, Silverio, primary, Maggio, Angela, primary, Maruzzi, Matteo, primary, Ciliberti, Andrea, primary, Samperi, Piera, primary, Casale, Maddalena, primary, Giordano, Paola, primary, Del Vecchio, Giovanni Carlo, primary, Perillo, Teresa, primary, Boscarol, Gianluca, primary, Notarangelo, Lucia Dora, primary, Casini, Tommaso, primary, Miano, Maurizio, primary, Fasoli, Silvia, primary, Paola, Corti, primary, Guarina, Angela, primary, Arcioni, Francesco, primary, Sau, Antonella, primary, Giona, Fiorina, primary, Palumbo, Giuseppe, primary, Saracco, Paola, primary, Petrone, Angela, primary, Verzegnassi, Federico, primary, Piccolo, Chiara, primary, Cesaro, Simone, primary, and Russo, Giovanna, primary

Published
2019
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10. Transfusion Therapy in a Multi-Ethnic Sickle Cell Population Real-World Practice. a Preliminary Data Analysis of Multicentre Survey

Author

Graziadei, Giovanna, primary, Sainati, Laura, additional, Bonomo, Pietro, additional, Venturelli, Donatella, additional, Masera, Nicoletta, additional, Casale, Maddalena, additional, Vassanelli, Aurora, additional, Lodi, Gianluca, additional, Piel, Frédéric B, additional, Voi, Vincenzo, additional, De Franceschi, Lucia, additional, Rigano, Paolo, additional, Quota, Alessandra, additional, Notarangelo, Lucia Dora, additional, Russo, Giovanna, additional, Rosso, Rosamaria, additional, Allò, Massimo, additional, D'Ascola, Domenico, additional, Facchini, Elena, additional, Macchi, Silvia, additional, Arcioni, Francesco, additional, Piperno, Alberto, additional, Bonetti, Federico, additional, Palazzi, Giovanni, additional, Bisconte, Maria Grazia, additional, Sau, Antonella, additional, Lisi, Roberto, additional, Giona, Fiorina, additional, Campisi, Saveria, additional, Colarusso, Gloria, additional, Giordano, Paola, additional, Boscariol, Gianluca, additional, Marktel, Sarah, additional, Filosa, Aldo, additional, Origa, Raffaella, additional, Murgia, Mauro, additional, Maroni, Paola, additional, Gianesin, Barbara, additional, Badalamenti, Luca, additional, and Forni, Gian Luca, additional

Published
2018
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11. Real-Life Management of Children and Adolescents with Chronic Myeloid Leukemia: The Italian Experience

Author

Giona, F, Santopietro, M, Menna, G, Putti, M, Micalizzi, C, Santoro, N, Ziino, O, Mura, R, Ladogana, S, Iaria, G, Sau, A, Burnelli, R, Vacca, N, Bernasconi, S, Consarino, C, Petruzziello, F, Moleti, M, Biondi, A, Locatelli, F, Foà, R, Giona, Fiorina, Santopietro, Michelina, Menna, Giuseppe, Putti, Maria Caterina, Micalizzi, Concetta, Santoro, Nicola, Ziino, Ottavio, Mura, Rosamaria, Ladogana, Saverio, Iaria, Grazia, Sau, Antonella, Burnelli, Roberta, Vacca, Nadia, Bernasconi, Sayla, Consarino, Caterina, Petruzziello, Fara, Moleti, Maria Luisa, Biondi, Andrea, Locatelli, Franco, Foà, Robin, Giona, F, Santopietro, M, Menna, G, Putti, M, Micalizzi, C, Santoro, N, Ziino, O, Mura, R, Ladogana, S, Iaria, G, Sau, A, Burnelli, R, Vacca, N, Bernasconi, S, Consarino, C, Petruzziello, F, Moleti, M, Biondi, A, Locatelli, F, Foà, R, Giona, Fiorina, Santopietro, Michelina, Menna, Giuseppe, Putti, Maria Caterina, Micalizzi, Concetta, Santoro, Nicola, Ziino, Ottavio, Mura, Rosamaria, Ladogana, Saverio, Iaria, Grazia, Sau, Antonella, Burnelli, Roberta, Vacca, Nadia, Bernasconi, Sayla, Consarino, Caterina, Petruzziello, Fara, Moleti, Maria Luisa, Biondi, Andrea, Locatelli, Franco, and Foà, Robin

Abstract

Background: To date, no data on the adherence to specific guidelines for children with chronic myeloid leukemia (CML) in chronic phase (CP) have been reported. Methods: Since 2001, guidelines for treatment with imatinib mesylate (IM) and monitoring in patients younger than 18 years with CP-CML have been shared with 9 pediatric referral centers (P centers) and 4 reference centers for adults and children/adolescents (AP centers) in Italy. In this study, the adherence to these guidelines was analyzed. Results: Thirty-four patients with a median age of 11.4 years and 23 patients with a median age of 11.0 years were managed at 9 P and at 4 AP centers, respectively. Evaluations of bone marrow (BM) and/or peripheral blood (PB) were available for more than 90% of evaluable patients. Cytogenetics and molecular monitoring of PB were more consistently performed in AP centers, whereas molecular analysis of BM was carried out more frequently in P centers. Before 2009, some patients who responded to IM underwent a transplantation, contrary to the guidelines' recommendations. Conclusions: Our experience shows that having specific guidelines is an important tool for an optimal management of childhood CP-CML, together with exchange of knowledge and proactive discussions within the network

Published
2018

12. Management of Chronic Myeloid Leukemia in Children and Adolescents According to Local Guidelines.the Italian Experience

Author

Giona, Fiorina, primary, Santopietro, Michelina, additional, Menna, Giuseppe, additional, Putti, Maria Caterina, additional, Micalizzi, Concetta, additional, Santoro, Nicola, additional, Ziino, Ottavio, additional, Mura, Rosamaria, additional, Ladogana, Saverio, additional, Iaria, Grazia, additional, Sau, Antonella, additional, Burnelli, Roberta, additional, Vacca, Nadia, additional, Nardi, Margherita, additional, Consarino, Caterina, additional, Moleti, Maria Luisa, additional, Nanni, Mauro, additional, Diverio, Daniela, additional, Leszl, Anna, additional, Biondi, Andrea, additional, Locatelli, Franco, additional, Saglio, Giuseppe, additional, and Foà, Robin, additional

Published
2016
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13. Long-Term Outcome in Children and Adolescents with Chronic Myeloid Leukemia in Chronic Phase Treated with High-Dose Imatinib— the Italian Experience

Author

Giona, Fiorina, Santopietro, Michelina, Menna, Giuseppe, Micalizzi, Concetta, Putti, Maria Caterina, Santoro, Nicola, Ziino, Ottavio, Mura, Rosamaria, Ladogana, Saverio, Iaria, Grazia, Sau, Antonella, Burnelli, Roberta, Vacca, Nadia, Nardi, Margherita, Consarino, Caterina, Miulli, Eleonora, Moleti, Maria Luisa, Petruzziello, Fara, Orlando, Sonia Maria, Biondi, Andrea, Locatelli, Franco, Saglio, Giuseppe, and Foa, Robin

Published
2017
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14. Rapporto tra pianificazione attuativa e generale

Author

Vitale, Carmen, Bonetti, Tommaso, Sau, Antonella, Scarale, Paola, Sabbioni, Paolo, Boscolo, Emanuele, Matassoglio, Francesca, Piperata, Giuseppe, Cabiddu, Maria Agostina, Boschetti, Barbara, Boschetti, B (ORCID:0000-0001-9344-2163), Vitale, Carmen, Bonetti, Tommaso, Sau, Antonella, Scarale, Paola, Sabbioni, Paolo, Boscolo, Emanuele, Matassoglio, Francesca, Piperata, Giuseppe, Cabiddu, Maria Agostina, Boschetti, Barbara, and Boschetti, B (ORCID:0000-0001-9344-2163)

Abstract

Il contributo mira a analizzare il rapport tra pianificazione generale e pianificazione attuativa

Published
2014

15. La pianificazione attuativa

Author

Cabiddu, Maria Agostina, Boscolo, Emanuele, Sau, Antonella e altri, Cabiddu, M.A., Boschetti, Barbara, Boschetti, Barbara (ORCID:0000-0001-9344-2163), Cabiddu, Maria Agostina, Boscolo, Emanuele, Sau, Antonella e altri, Cabiddu, M.A., Boschetti, Barbara, and Boschetti, Barbara (ORCID:0000-0001-9344-2163)

Abstract

Analisi delle tipologie di strumenti di pianificazione attuativa

Published
2010

17. Molecular Analysis of the SEC23B Gene In Patients Affected by Congenital Dyserythropoietic Anemia Type II (CDAII)

Author

Bianchi, Paola, primary, Schwarz, Klaus, additional, Fermo, Elisa, additional, Heinrich, Katja, additional, Vercellati, Cristina, additional, Marcello, Anna Paola Maria Luisa, additional, van Wijk, Richard, additional, Zwieten, Rob van, additional, Banov, Laura, additional, Sau, Antonella, additional, Wuillemin, Walter A., additional, Opat, Stephen, additional, Barcellini, Wilma, additional, Heimpel, Hermann, additional, and Zanella, Alberto, additional

Published
2010
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21. Transfusional Approach in Multi-Ethnic Sickle Cell Patients: Real-World Practice Data From a Multicenter Survey in Italy

Author

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

Subjects
multi-ethnicity, transfusion therapy, alloimmunization, hydroxycarbamide, sickle cell disease, General Medicine
Abstract

Sickle cell disease (SCD) is a worldwide distributed hereditary red cell disorder characterized by recurrent acute vaso-occlusive crises (VOCs and anemia). Gold standard treatments are hydroxycarbamide (HC) and/or different red blood cell (RBC) transfusion regimens to limit disease progression. Here, we report a retrospective study on 1,579 SCD patients (median age 23 years; 802 males/777 females), referring to 34 comprehensive Italian centers for hemoglobinopathies. Although we observed a similar proportion of Caucasian (47.9%) and African (48.7%) patients, Italian SCD patients clustered into two distinct overall groups: children of African descent and adults of Caucasian descent. We found a subset of SCD patients requiring more intensive therapy with a combination of HC plus chronic transfusion regimen, due to partial failure of HC treatment alone in preventing or reducing sickle cell-related acute manifestations. Notably, we observed a higher use of acute transfusion approaches for SCD patients of African descent when compared to Caucasian subjects. This might be related to (i) age of starting HC treatment; (ii) patients' low social status; (iii) patients' limited access to family practitioners; or (iv) discrimination. In our cohort, alloimmunization was documented in 135 patients (8.5%) and was more common in Caucasians (10.3%) than in Africans (6.6%). Alloimmunization was similar in male and female and more frequent in adults than in children. Our study reinforces the importance of donor-recipient exact matching for ABO, Rhesus, and Kell antigen systems for RBC compatibility as a winning strategy to avoid or limit alloimmunization events that negatively impact the clinical management of SCD-related severe complications.Clinical Trial RegistrationClinicalTrials.gov, identifier: NCT03397017.

Published
2022
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22. Risk factors for endocrine complications in transfusion-dependent thalassemia patients on chelation therapy with deferasirox: a risk assessment study from a multi-center nation-wide cohort

Author

Raffaella Origa, Antonella Sau, Maria Caterina Putti, Maurizio Poggi, Gian Luca Forni, Antonella Quarta, Alessandro Cattoni, Marilena Serra, Immacolata Tartaglione, Domenico Roberti, Elena Cassinerio, Elisa De Michele, Patrizia Sturiale, Filomena Sportelli, Saveria Campisi, Antonio Ivan Lazzarino, Massimo Allò, Silverio Perrotta, Rosanna Di Concilio, Daniela Pasquali, Alessia Marcon, Angelo Peluso, Stefania Picariello, Maddalena Casale, Francesca Ferrara, Roberta Renni, Saverio Ladogana, Lucia Dora Notarangelo, Casale, Maddalena, Luca Forni, Gian, Cassinerio, Elena, Pasquali, Daniela, Origa, Raffaella, Serra, Marilena, Campisi, Saveria, Peluso, Angelo, Renni, Roberta, Cattoni, Alessandro, De Michele, Elisa, Allò, Massimo, Poggi, Maurizio, Ferrara, Francesca, Di Concilio, Rosanna, Sportelli, Filomena, Quarta, Antonella, Caterina Putti, Maria, Dora Notarangelo, Lucia, Sau, Antonella, Ladogana, Saverio, Tartaglione, Immacolata, Picariello, Stefania, Marcon, Alessia, Sturiale, Patrizia, Roberti, Domenico, Ivan Lazzarino, Antonio, Perrotta, Silverio, Casale, M, Forni, G, Cassinerio, E, Pasquali, D, Origa, R, Serra, M, Campisi, S, Peluso, A, Renni, R, Cattoni, A, De Michele, E, Allò, M, Poggi, M, Ferrara, F, Di Concilio, R, Sportelli, F, Quarta, A, Putti, M, Notarangelo, L, Sau, A, Ladogana, S, Tartaglione, I, Picariello, S, Marcon, A, Sturiale, P, Roberti, D, Lazzarino, A, and Perrotta, S

Subjects
medicine.medical_specialty, Iron Overload, Thalassemia, blood transfusion, Iron Chelating Agents, Benzoates, Risk Assessment, 03 medical and health sciences, Liver disease, 0302 clinical medicine, Risk Factors, Internal medicine, Medicine, Endocrine system, Humans, Chelation therapy, iron chelation, business.industry, Incidence (epidemiology), Deferasirox, beta-Thalassemia, Hematology, Triazoles, medicine.disease, Chelation Therapy, 030220 oncology & carcinogenesis, Cohort, business, Complication, Thalassemia, endocrine complications, iron chelation, deferasirox, endocrine function, 030215 immunology, medicine.drug, Follow-Up Studies
Abstract

Transfusion-dependent patients typically develop iron-induced cardiomyopathy, liver disease, and endocrine complications. We aimed to estimate the incidence of endocrine disorders in transfusiondependent thalassemia (TDT) patients during long-term iron-chelation therapy with deferasirox (DFX). We developed a multi-center follow-up study of 426 TDT patients treated with once-daily DFX for a median duration of 8 years, up to 18.5 years. At baseline, 118, 121, and 187 patients had 0, 1, or ≥2 endocrine diseases respectively. 104 additional endocrine diseases were developed during the follow-up. The overall risk of developing a new endocrine complication within 5 years was 9.7% (95% Confidence Interval [CI]: 6.3–13.1). Multiple Cox regression analysis identified three key predictors: age showed a positive log-linear effect (adjusted hazard ratio [HR] for 50% increase 1.2, 95% CI: 1.1–1.3, P=0.005), the serum concentration of thyrotropin showed a positive linear effect (adjusted HR for 1 mIU/L increase 1.3, 95% CI: 1.1–1.4, P

Published
2020

23. The prognostic value of biological markers in paediatric Hodgkin lymphoma

Author

Gaetano Bottigliero, Maurizio Mascarin, Roberta Caruso, Simone Cesaro, Grazia Iaria, Marco Zecca, Ada Zaccaron, Paolo Pierani, Giuseppe Puccio, Claudio Favre, Giulio Murgia, Massimo Provenzi, Franco Locatelli, Fulvio Porta, Eva Passone, Carlo Cosmi, Nadia Mirra, Giulio Andrea Zanazzo, Monica Cellini, Roberta Pericoli, Roberta Burnelli, Raffaela De Santis, Antonella Sau, Fausto Fedeli, Marta Pillon, Alberto Garaventa, Salvatore D'Amico, Katia Perruccio, Alessandra Todesco, Maurizio Caniglia, Caterina Consarino, Domenico Sperlì, Luigi Nespoli, Piero Farruggia, Andrea Pession, Tommaso Casini, Nicola Santoro, Maurizio Bianchi, P Bertolini, Adele Civino, Alessandra Sala, Paolo D'Angelo, Mauro Caini, Giovanni Scarzello, Angela Trizzino, Salvatore Buffardi, Roberto Rondelli, Farruggia, Piero, Puccio, Giuseppe, Sala, Alessandra, Todesco, Alessandra, Buffardi, Salvatore, Garaventa, Alberto, Bottigliero, Gaetano, Bianchi, Maurizio, Zecca, Marco, Locatelli, Franco, Pession, Andrea, Pillon, Marta, Favre, Claudio, D'Amico, Salvatore, Provenzi, Massimo, Trizzino, Angela, Zanazzo, Giulio Andrea, Sau, Antonella, Santoro, Nicola, Murgia, Giulio, Casini, Tommaso, Mascarin, Maurizio, and Burnelli, Roberta

Subjects
Male, Oncology, Cancer Research, Pathology, Time Factors, Databases, Factual, medicine.medical_treatment, hodgkin lymphoma, paediatric, prognostic factor, Hodgkin lymphoma, Paediatric, Prognostic factor, Adolescent, Age Factors, Antineoplastic Combined Chemotherapy Protocols, Biomarkers, Tumor, Child, Child, Preschool, Disease Progression, Disease-Free Survival, Female, Ferritins, Hodgkin Disease, Humans, Infant, Infant, Newborn, Italy, Kaplan-Meier Estimate, Leukocyte Count, Multivariate Analysis, Neoplasm Staging, Platelet Count, Predictive Value of Tests, Proportional Hazards Models, Retrospective Studies, Risk Factors, Treatment Outcome, Blood Platelets, Eosinophils, Procarbazine, chemistry.chemical_compound, 0302 clinical medicine, Prednisone, Tumor, Vinblastine, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, 030220 oncology & carcinogenesis, medicine.drug, medicine.medical_specialty, Vincristine, Dacarbazine, Bleomycin, Databases, 03 medical and health sciences, Internal medicine, medicine, Preschool, Factual, Chemotherapy, business.industry, Newborn, chemistry, ABVD, business, Biomarkers, 030215 immunology
Abstract

Background Many biological and inflammatory markers have been proposed as having a prognostic value at diagnosis of Hodgkin lymphoma (HL), but very few have been validated in paediatric patients. We explored the significance of these markers in a large population of 769 affected children. Patients and methods By using the database of patients enrolled in A.I.E.O.P. (Associazione Italiana di Emato-Oncologia Pediatrica) trial LH2004 for paediatric HL, we identified 769 consecutive patients treated with curative intent from 1st June 2004 to 1st April 2014 with ABVD (doxorubicin, bleomycin, vinblastine, and dacarbazine), or hybrid COPP/ABV (cyclophosphamide, vincristine, prednisone, procarbazine, doxorubicin, bleomycin and vinblastine) regimens. Results On multivariate analysis with categorical forms, the 5-year freedom from progression survival was significantly lower in patients with stage IV or elevated value of platelets, eosinophils and ferritin at diagnosis. Furthermore, stage IV and eosinophils seem to maintain their predictive value independently of interim (after IV cycles of chemotherapy) positron emission tomography. Conclusion Using the combination of four simple markers such as stage IV and elevated levels of platelets, ferritin and eosinophils, it is possible to classify the patients into subgroups with very different outcomes.

Published
2016
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24. Second-line therapy in paediatric warm autoimmune haemolytic anaemia. Guidelines from the Associazione Italiana Onco-Ematologia Pediatrica (AIEOP)

Author

Ladogana S., Maruzzi M., Samperi P., Condorelli A., Casale M., Giordano P., Notarangelo L. D., Farruggia P., Giona F., Nocerino A., Fasoli S., Casciana M. L., Miano M., Tucci F., Casini T., Saracco P., Barcellini W., Zanella A., Perrotta S., Russo G., Baronci C., Casadei A. M., Cazzaniga G., Coluzzi S., Ciliberti A., Del Vecchio G. C., Facchini E., Girelli G., Lazzareschi I., Masera N., Perseghin P., Peruccio D., Petrone A., Sau A., Schiro R., Tumino M., Vasta I., Verzegnassi F., Ladogana, Saverio, Maruzzi, Matteo, Samperi, Piera, Condorelli, Annalisa, Casale, Maddalena, Giordano, Paola, Notarangelo, Lucia D., Farruggia, Piero, Giona, Fiorina, Nocerino, Agostino, Fasoli, Silvia, Casciana, Maria L., Miano, Maurizio, Tucci, Fabio, Casini, Tommaso, Saracco, Paola, Barcellini, Wilma, Zanella, Alberto, Perrotta, Silverio, Russo, Giovanna, Baronci, Carlo, Casadei, Anna Maria, Cazzaniga, Giovanni, Coluzzi, Serelina, Corti, Paola, Del Vecchio, Giovanni C., Facchini, Elena, Girelli, Gabriella, Lazzareschi, Ilaria, Masera, Nicoletta, Perseghin, Paolo, Peruccio, Daniela, Petrone, Angelamaria, Sau, Antonella, Schirò, Raffaella, Tumino, Manuela, Vasta, Isabella, Verzegnassi, Federico, Ladogana, S, Maruzzi, M, Samperi, P, Condorelli, A, Casale, M, Giordano, P, Notarangelo, L, Farruggia, P, Giona, F, Nocerino, A, Fasoli, S, Casciana, M, Miano, M, Tucci, F, Casini, T, Saracco, P, Barcellini, W, Zanella, A, Perrotta, S, Russo, G, Baronci, C, Casadei, A, Cazzaniga, G, Coluzzi, S, Ciliberti, A, Del Vecchio, G, Facchini, E, Girelli, G, Lazzareschi, I, Masera, N, Perseghin, P, Peruccio, D, Petrone, A, Sau, A, Schiro, R, Tumino, M, Vasta, I, and Verzegnassi, F

Subjects
Male, autoimmune haemolytic anaemia, child, therapy, rituximab, mycophenolate mofetil, splenectomy, therapy, Erythrocytes, Adolescent, Incidence, mycophenolate mofetil, Infant, Hematology, Guideline, splenectomy, rituximab, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, Child, Preschool, Humans, Immunology and Allergy, autoimmune haemolytic anaemia, Female, Anemia, Hemolytic, Autoimmune, Child, Autoantibodies
Published
2018

25. Risk factors for endocrine complications in transfusion-dependent thalassemia patients on chelation therapy with deferasirox: a risk assessment study from a multi-center nation-wide cohort.

Author

Casale M, Forni GL, Cassinerio E, Pasquali D, Origa R, Serra M, Campisi S, Peluso A, Renni R, Cattoni A, De Michele E, Allò M, Poggi M, Ferrara F, Di Concilio R, Sportelli F, Quarta A, Putti MC, Notarangelo LD, Sau A, Ladogana S, Tartaglione I, Picariello S, Marcon A, Sturiale P, Roberti D, Lazzarino AI, and Perrotta S

Subjects
Benzoates adverse effects, Chelation Therapy adverse effects, Deferasirox adverse effects, Follow-Up Studies, Humans, Iron Chelating Agents adverse effects, Risk Assessment, Risk Factors, Triazoles adverse effects, Iron Overload drug therapy, Iron Overload epidemiology, Iron Overload etiology, Thalassemia complications, Thalassemia epidemiology, Thalassemia therapy, beta-Thalassemia complications
Abstract

Transfusion-dependent patients typically develop iron-induced cardiomyopathy, liver disease, and endocrine complications. We aimed to estimate the incidence of endocrine disorders in transfusiondependent thalassemia (TDT) patients during long-term iron-chelation therapy with deferasirox (DFX). We developed a multi-center follow-up study of 426 TDT patients treated with once-daily DFX for a median duration of 8 years, up to 18.5 years. At baseline, 118, 121, and 187 patients had 0, 1, or ≥2 endocrine diseases respectively. 104 additional endocrine diseases were developed during the follow-up. The overall risk of developing a new endocrine complication within 5 years was 9.7% (95% Confidence Interval [CI]: 6.3-13.1). Multiple Cox regression analysis identified three key predictors: age showed a positive log-linear effect (adjusted hazard ratio [HR] for 50% increase 1.2, 95% CI: 1.1-1.3, P=0.005), the serum concentration of thyrotropin showed a positive linear effect (adjusted HR for 1 mIU/L increase 1.3, 95% CI: 1.1-1.4, P<0.001) regardless the kind of disease incident, while the number of previous endocrine diseases showed a negative linear effect: the higher the number of diseases at baseline the lower the chance of developing further diseasess (adjusted HR for unit increase 0.5, 95% CI: 0.4-0.7, P<0.001). Age and thyrotropin had similar effect sizes across the categories of baseline diseases. The administration of levothyroxine as a covariate did not change the estimates. Although in DFX-treated TDT patients the risk of developing an endocrine complication is generally lower than the previously reported risk, there is considerable risk variation and the burden of these complications remains high. We developed a simple risk score chart enabling clinicians to estimate their patients' risk. Future research will look at increasing the amount of variation explained from our model and testing further clinical and laboratory predictors, including the assessment of direct endocrine magnetic resonance imaging.

Published
2022
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