Search

Your search keyword '"Balduzzi, Adriana"' showing total 646 results
Start Over Author "Balduzzi, Adriana"
646 results on '"Balduzzi, Adriana"'

1. Graft-versus-host disease after anti-CD19 chimeric antigen receptor T-cell therapy following allogeneic hematopoietic cell transplantation: a transplant complications and paediatric diseases working parties joint EBMT study

Author

Ortí, Guillermo, Peczynski, Christophe, Boreland, William, O’Reilly, Maeve, von Bonin, Malte, Balduzzi, Adriana, Besley, Caroline, Kalwak, Krzysztof, Ryhänen, Samppa, Güngör, Tayfun, Wynn, Robert F., Bader, Peter, Mielke, Stephan, Blaise, Didier, Amrolia, Persis, Yakoub-Agha, Ibrahim, Calkoen, Friso, Schubert, Maria-Luisa, Potter, Victoria, Pichler, Herbert, Kröger, Nicolaus, Kwon, Mi, Sengeloev, Henrik, Torrent, Anna, Chalandon, Yves, van Gorkom, Gwendolyn, Koenecke, Christian, Graham, Charlotte, Schoemans, Helene, Moiseev, Ivan, Penack, Olaf, and Peric, Zinaida

Published
2024
Full Text
View/download PDF

2. An expert consensus on prevention, diagnosis, and management of hemorrhagic cystitis in pediatric hematopoietic cell transplantation, on behalf of the Infectious Disease and Hematopoietic Cell Transplant Working groups of Italian Pediatric Hematology Oncology Association (AIEOP)

Author

Dell’Orso, Gianluca, Carlucci, Marcello, Cesaro, Simone, Olcese, Evelina, Balduzzi, Adriana, Vendemini, Francesca, Catti, Massimo, Saglio, Francesco, Compagno, Francesca, Maximova, Natalia, Rabusin, Marco, Menconi, Maria Cristina, Perruccio, Katia, Soncini, Elena, Tambaro, Francesco Paolo, Tintori, Veronica, Pagliara, Daria, and Faraci, Maura

Published
2024
Full Text
View/download PDF

3. Safety and efficacy of narsoplimab in pediatric and adult patients with transplant-associated thrombotic microangiopathy: a real-world experience

Author

Castelli, Marta, Micò, Maria Caterina, Grassi, Anna, Algarotti, Alessandra, Lussana, Federico, Finazzi, Maria Chiara, Rambaldi, Benedetta, Pavoni, Chiara, Rizzuto, Giuliana, Tebaldi, Paola, Vendemini, Francesca, Verna, Marta, Bonanomi, Sonia, Biondi, Andrea, Balduzzi, Adriana, Rambaldi, Alessandro, and Gotti, Giacomo

Published
2024
Full Text
View/download PDF

4. Outcomes of allogeneic haematopoietic cell transplantation for myelofibrosis in children and adolescents: the retrospective study of the EBMT Paediatric Diseases WP

Author

Wachowiak, Jacek, Galimard, Jacques-Emmanuel, Dalissier, Arnaud, Rihani, Rawad, AlSaedi, Hawazen, Wynn, Robert F., Dalle, Jean-Hugues, Peffault de Latour, Régis, Sedlacek, Petr, Balduzzi, Adriana, Schroeder, Thomas, Bodova, Ivana, Gonzalez Vicent, Marta, Gruhn, Bernd, Hamladji, Rose-Marie, Krivan, Gergely, Patrick, Katharine, Sobkowiak-Sobierajska, Agnieszka, Stepensky, Polina, Unal, Ali, Amrolia, Persis, Perez Martinez, Antonio, Rialland, Fanny, Aljurf, Mahmoud, Isgro, Antonella, Toren, Amos, Bierings, Marc, Corbacioglu, Selim, and Kałwak, Krzysztof

Published
2024
Full Text
View/download PDF

6. Low rate of nonrelapse mortality in under-4-year-olds with ALL given chemotherapeutic conditioning for HSCT: a phase 3 FORUM study

Author

Bader, Peter, Pötschger, Ulrike, Dalle, Jean-Hugues, Moser, Laura M., Balduzzi, Adriana, Ansari, Marc, Buechner, Jochen, Güngör, Tayfun, Ifversen, Marianne, Krivan, Gergely, Pichler, Herbert, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr, Stein, Jerry, Heusel, Jan Robert, Truong, Tony, Wachowiak, Jacek, Yesilipek, Akif, Locatelli, Franco, and Peters, Christina

Published
2024
Full Text
View/download PDF

8. Essential medicines for childhood cancer in Europe: a pan-European, systematic analysis by SIOPE

Author

Achini, Federica, Balduzzi, Adriana, Beck Popovic, Maja, Beishuizen, Auke, Bergamaschi, Luca, Biondi, Andrea, Bourdeaut, Franck, Braicu, Elena, Brok, Jesper, Brugières, Laurence, Burke, Amos, Calaminus, Gabriele, Casanova, Michela, Choucair, Marie-Louise, Cleirec, Morgane, Corbaciouglu, Selim, Correa Llano, Maria Genoveva, De Rojas, Teresa, Domínguez Pinilla, Nerea, Elmaraghi, Caroline, Ferrari, Andrea, Fossa, Alexander, Gaspar, Nathalie, Herold, Nikolas, Karapiperi, Kyriaki, Karu, Maarja, Kjærsgaar, Mimi, Knörr, Fabian, Koenig, Christa, Kranjcec, Izabela, Krawczyk, Malgorzata, Lehmberg, Kai, Lehrnbecher, Thomas, Lunesink, Maaike, Massano, Davide, Matijasic, Nuša, Merks, Hans, Metzler, Markus, Michalski, Anthony, Minkov, Milen, Morland, Bruce, Niktoreh, Naghmeh, Oltenau, Elena, Orbach, Daniel, Owens, Cormac, Papachristidou, Smaragda, Pasqualini, Claudia, Pavlovic, Maja, Perez Albert, Paula, Poyer, Fiona, Radulovic, Ivana, Reinhardt, Dirk, Rebelo, Joana, Roser, Eva, Russo, Ida, Scheinemann, Katrin, Schindera, Christina, Schrappe, Martin, Sehested, Astrid, Sehouli, Jalid, Spreafico, Filippo, Strauss, Sandra J, Stutterheim, Janine, Svojgr, Karel, Tzotzola, Vasiliki, Van Ewijk, Roelof, Verschuur, Arnauld, Vora, Ajay, Woessmann, Willi, Zajac-Spychala, Olga, Zwaan, Michel, Otth, Maria, Brack, Eva, Kearns, Pamela R, Kozhaeva, Olga, Ocokoljic, Marko, Schoot, Reineke A, and Vassal, Gilles

Published
2022
Full Text
View/download PDF

9. Long-Term Complications after Allogeneic Hematopoietic Stem Cell Transplantation with Treosulfan- or Busulfan-Based Conditioning in Pediatric Patients with Acute Leukemia or Myelodysplastic Syndrome: Results of an Associazione Italiana Ematologia Oncologia Pediatrica Retrospective Study

Author

Saglio, Francesco, Pagliara, Daria, Zecca, Marco, Balduzzi, Adriana, Cattoni, Alessandro, Prete, Arcangelo, Tambaro, Francesco Paolo, Faraci, Maura, Calore, Elisabetta, Locatelli, Franco, and Fagioli, Franca

Published
2024
Full Text
View/download PDF

10. Haematopoietic stem cell transplantation after reduced intensity conditioning in children and adolescents with chronic myeloid leukaemia: A prospective multicentre trial of the I‐BFM Study Group

Author

Pichler, Herbert, primary, Sedlacek, Petr, additional, Meisel, Roland, additional, Beier, Rita, additional, Faraci, Maura, additional, Kalwak, Krzysztof, additional, Ifversen, Marianne, additional, Müller, Ingo, additional, Stein, Jerry, additional, Vettenranta, Kim, additional, Kropshofer, Gabriele, additional, Kolenova, Alexandra, additional, Karlhuber, Susanne, additional, Glogova, Evgenia, additional, Poetschger, Ulrike, additional, Peters, Christina, additional, Suttorp, Meinolf, additional, Matthes‐Leodolter, Susanne, additional, and Balduzzi, Adriana, additional

Published
2024
Full Text
View/download PDF

11. Anti-T-lymphocyte globulin exposure is associated with acute graft-versus-host disease and relapse in pediatric acute lymphoblastic leukemia patients undergoing hematopoietic stem cell transplantation: a multinational prospective study

Author

Oostenbrink, Lisa V.E., primary, Von Asmuth, Erik G.J., additional, Jol-van der Zijde, Cornelia M., additional, Jansen-Hoogendijk, Anja M., additional, Vervat, Carly, additional, Bredius, Robbert G.M., additional, Van Tol, Maarten J.D., additional, Schilham, Marco W., additional, Sedlacek, Petr, additional, Ifversen, Marianne, additional, Balduzzi, Adriana, additional, Bader, Peter, additional, Peters, Christina, additional, Moes, Dirk Jan A.R., additional, and Lankester, Arjan C., additional

Published
2024
Full Text
View/download PDF

13. An expert consensus on prevention, diagnosis and management of hemorrhagic cystitis in pediatric hematopoietic cell transplantation, on behalf of the Infectious Disease and Hematopoietic Cell Transplant Working groups of Italian Pediatric Hematology Oncolo-gy Association (AIEOP)

Author

Dell'Orso, Gianluca, primary, Carlucci, Marcello, additional, Cesaro, Simone, additional, Olcese, Evelina, additional, Balduzzi, Adriana, additional, Vendemini, Francesca, additional, Catti, Massimo, additional, Saglio, Francesco, additional, Compagno, Francesca, additional, Maximova, Natalia, additional, Rabusin, Marco, additional, Menconi, Maria Cristina, additional, Perruccio, Katia, additional, Soncini, Elena, additional, Tambaro, Francesco Paolo, additional, tintori, Veronica, additional, Pagliara, Daria, additional, and Faraci, Maura, additional

Published
2024
Full Text
View/download PDF

14. Global characteristics and outcomes of SARS-CoV-2 infection in children and adolescents with cancer (GRCCC): a cohort study

Author

Ribelles, A Juan, Balduzzi, Adriana, Elhaddad, Alaa, Casanovas, Alejandra, Garcia Velazquez, Alejandra, Laptsevich, Aliaksandra, Chang, Alicia, F. Sampaio, Alessandra Lamenha, González Prieto, Almudena, Lassaletta, Alvaro, Suarez M, Amaranto, Alcasabas, Ana Patricia, Colita, Anca, Morales La Madrid, Andres, Samudio, Angélica, Tondo, Annalisa, Colombini, Antonella, Kattamis, Antonis, Lopez Facundo, N Araceli, Bhattacharyya, Arpita, Alimi, Aurélia, Phulpin, Aurélie, Vakrmanova, Barbora, Aksoy, Basak A, Brethon, Benoit, Kobuin, Jator Brian, Nolasco Monteiro, Carla, Paillard, Catherine, Vezina, Catherine, Ceyhun, Bozkurt, Hentea, Cristiana, Meazza, Cristina, Ortiz-Morales, Daniel, Solorzano, Roque Daniel, Arce Cabrera, Daniela, Zama, Daniele, Ghosh, Debjani, Ramírez-Rivera, Diana, Calle Jara, Doris A, Janic, Dragana, Rey Helo, Elianneth, Gouache, Elodie, Guerrero Quiroz, Enmanuel, Lopez, Enrique, Thebault, Eric, Maradiegue, Essy, de Berranger, Eva, Ebeid, Fatma S E, Galaverna, Federica, Antillon-Klussmann, Federico, Espinoza Chacur, Felipe, Negro, Fernando Daniel, Carraro, Francesca, Compagno, Francesca, Barriga, Francisco, Tamayo Pedraza, Gabriela, Sanchez Fernandez, Gissela, Naidu, Gita, Tokuc, Gülnur, Alias, Hamidah, B Segocio, Hannah Grace, Boudiaf, Houda, Asetre Luna, Imelda, Maia, Iris, Astigarraga, Itziar, Maza, Ivan, Montoya Vásquez, Jacqueline E, Jazbec, Janez, Lazic, Jelena, Beck Dean, Jeniffer, Rouger-Gaudichon, Jeremie, Contreras González, Johanny Carolina, Huerta Aragonés, Jorge, Fuster, José L, Quintana, Juan, Palma, Julia, Svojgr, Karel, Quintero, Karina, Malic Tudor, Karolina, Georgantzi, Kleopatra, P Schultz, Kris Ann, Ureña Horno, Laura, Fraquelli, Lidia, Meneghello, Linda, Shalaby, Lobna, Macias Mora, Lola L, A Renner, Lorna, Nunes Silva, Luciana, Sisinni, Luisa, Hammad, Mahmoud, Fernández Sanmartín, M, Zubieta A, C Marcela, Drozdowski, María Constanza, Kourti, Maria, Palladino, Marcela María, Miranda Madrazo, Maria R, Poiree, Marilyne, Popova, Marina, Melgar, Mario, Baragaño, Marta, Avilés-Robles, Martha J, Provenzi, Massimo, Mendes Lins, Mecneide, Fatih Orhan, Mehmet, Villarroel, Milena, Jerónimo, Mónica, Varas Palma, Mónica, Rafie Raza, Muhammad, M Justin, Mulindwa, Shaheen, Najma, Domínguez-Pinilla, Nerea, Whipple, Nicholas S, André, Nicolas, Hrusak, Ondrej, Velasco Puyó, Pablo, Zacasa Vargas, Pamela, Olate Mellado, Paola, Yola Gassant, Pascale, Diaz Romero, Paulina, De Santis, Raffaella, Kebudi, Rejin, Boranbayeva, Riza, Vasquez, Roberto, Segura, Romel A., Rosado, Roy Enrique, Gómez, Sandra, Raimbault, Sandra, Gunasekera, Sanjeeva, Makkeyah, Sara M, Buyukkapu Bay, Sema, M Gómez, Sergio, Bouttefroy, Séverine, Islam, Shahnoor, Abouelnaga, Sherif, Torres, Silvio Fabio, Cesaro, Simone, Nunes, Sofia, Rouxinol, Soraia, Bhaumik, Sucharita, Saliyeva, Symbat, Inostroza, Tamara, Velasquez, Thelma, Hnin, Tint Myo, Norén-Nyström, Ulrika, Baretta, Valentina, Jimenez-Antolinez, Yajaira Valentine, Pérez Alonso, Vanesa, Ayer Miller, Vanessa, Gandemer, Virginie, Lotero, Viviana, Mishkova, Volha, Gómez-García, Wendy, Margaryan, Yeva, Syed, Yumna, Mukkada, Sheena, Bhakta, Nickhill, Chantada, Guillermo L, Chen, Yichen, Vedaraju, Yuvanesh, Faughnan, Lane, Homsi, Maysam R, Muniz-Talavera, Hilmarie, Ranadive, Radhikesh, Metzger, Monika, Friedrich, Paola, Agulnik, Asya, Jeha, Sima, Lam, Catherine, Dalvi, Rashmi, Hessissen, Laila, Moreira, Daniel C, Santana, Victor M, Sullivan, Michael, Bouffet, Eric, Caniza, Miguela A, Devidas, Meenakshi, Pritchard-Jones, Kathy, and Rodriguez-Galindo, Carlos

Published
2021
Full Text
View/download PDF

15. Pelvic ultrasound and pubertal attainment in girls with sexual precocity: the pivotal role of uterine volume in predicting the timing of menarche

Author

Cattoni, A, Russo, G, Capitoli, G, Rodari, G, Nicolosi, M, Molinari, S, Tondelli, D, Pelliccia, C, Radaelli, S, Arosio, A, Fontana, K, Tattesi, G, Passoni, P, Boneschi, A, Giavoli, C, Meroni, S, Stancampiano, M, Garuti, E, Biondi, A, Balduzzi, A, Bizzarri, C, Cattoni, Alessandro, Russo, Gianni, Capitoli, Giulia, Rodari, Giulia, Nicolosi, Maria Laura, Molinari, Silvia, Tondelli, Daniele, Pelliccia, Ciretta, Radaelli, Silvia, Arosio, Andrea Mario Luciano, Fontana, Katia, Tattesi, Giulia, Passoni, Paolo, Boneschi, Annalisa, Giavoli, Claudia, Meroni, Silvia Laura Carla, Stancampiano, Marianna Rita, Garuti, Elda, Biondi, Andrea, Balduzzi, Adriana, Bizzarri, Carla, Cattoni, A, Russo, G, Capitoli, G, Rodari, G, Nicolosi, M, Molinari, S, Tondelli, D, Pelliccia, C, Radaelli, S, Arosio, A, Fontana, K, Tattesi, G, Passoni, P, Boneschi, A, Giavoli, C, Meroni, S, Stancampiano, M, Garuti, E, Biondi, A, Balduzzi, A, Bizzarri, C, Cattoni, Alessandro, Russo, Gianni, Capitoli, Giulia, Rodari, Giulia, Nicolosi, Maria Laura, Molinari, Silvia, Tondelli, Daniele, Pelliccia, Ciretta, Radaelli, Silvia, Arosio, Andrea Mario Luciano, Fontana, Katia, Tattesi, Giulia, Passoni, Paolo, Boneschi, Annalisa, Giavoli, Claudia, Meroni, Silvia Laura Carla, Stancampiano, Marianna Rita, Garuti, Elda, Biondi, Andrea, Balduzzi, Adriana, and Bizzarri, Carla

Abstract

Introduction: Among girls assessed for pubertal precocity, pelvic ultrasound (pUS) may represent a pivotal tool to predict the time expected to elapse between sonographic assessment and the onset of menarche (TUS-M). Accordingly, the present analysis is meant to define the statistical relationship between sonographic parameters and TUS-M, in order to identify the most reliable predictor of the timing of menarche. Methods: Retrospective, multicenter analysis. Girls assessed for sexual precocity and showing sonographic and clinical findings consistent with pubertal onset upon referral were considered eligible. Patients treated with GnRH analogues were excluded and only those who had subsequently achieved complete and spontaneous pubertal attainment and for whom the exact date of menarche was available were included. Overall, we enrolled 184 girls from five tertiary care Italian Centers. Results: The time elapsed (months) between baseline endocrine assessment and spontaneous achievement of menarche showed a negative statistically significant correlation (p<0.0001) with LH (r:-0.61), FSH (r:-0.59), estradiol (r:-0.52) and stimulated LH values (r:-0.58). Among pUS parameters, ovarian volume (r:-0.17 left, -0.30 right) and uterine body-to-cervix ratio (r:-0.18) poorly correlated with TUS-M, while uterine diameters (r:-0.61 longitudinal, -0.64 anteroposterior) and volume (r:-0.70) achieved a highly statistical significance (p<0.0001). Uterine volume (UV) showed a negative logarithmic relationship with TUS-M and represented the most reliable predictor of the timing of menarche in uni- and multivariable analyses (p <0.001). ROC analyses identified the UV thresholds that best predict the onset of menarche within 18, 12 and 6 months, respectively: 3.76, 6.02 and 8.80 ml. Conclusion: The logarithm of UV shows the best statistical performance in predicting the timing of menarche in girls assessed for pubertal precocity. Accordingly, we developed a user-friendly online a

Published
2024

16. Adolescent gender dysphoria management: position paper from the Italian Academy of Pediatrics, the Italian Society of Pediatrics, the Italian Society for Pediatric Endocrinology and Diabetes, the Italian Society of Adolescent Medicine and the Italian Society of Child and Adolescent Neuropsychiatry

Author

Calcaterra, V, Tornese, G, Zuccotti, G, Staiano, A, Cherubini, V, Gaudino, R, Fazzi, E, Barbi, E, Chiarelli, F, Corsello, G, Esposito, S, Ferrara, P, Iughetti, L, Laforgia, N, Maghnie, M, Marseglia, G, Perilongo, G, Pettoello-Mantovani, M, Ruggieri, M, Russo, G, Salerno, M, Striano, P, Valerio, G, Wasniewska, M, Agosti, M, Agostoni, C, Aiuti, A, Azzari, C, Badolato, R, Balduzzi, A, Baraldi, E, Canani, R, Biffi, A, Biondi, A, Bisogno, G, Pierri, N, Carnielli, V, Cianfarani, S, Cogo, P, Corvaglia, L, Dani, C, Di Salvo, G, Fagioli, F, Fanos, V, Ferrero, G, Francavilla, R, Galli, L, Gazzolo, D, Giaquinto, C, Giordano, P, Gitto, E, Grosso, S, Guarino, A, Indrio, F, Lanari, M, Lionetti, P, Locatelli, F, Lombardo, F, Maffeis, C, Marino, B, Midulla, F, Del Giudice, E, Del Giudice, M, Montini, G, Parenti, G, Parisi, P, Peroni, D, Perrotta, S, Piacentini, G, Pietrobelli, A, Raimondi, F, Ramenghi, U, Ravelli, A, Romano, C, Rossi, F, Rossi, P, Damiano, V, Spalice, A, Suppiej, A, Troncone, R, Verrotti, A, Null, N, Calcaterra, Valeria, Tornese, Gianluca, Zuccotti, Gianvincenzo, Staiano, Annamaria, Cherubini, Valentino, Gaudino, Rossella, Fazzi, Elisa Maria, Barbi, Egidio, Chiarelli, Francesco, Corsello, Giovanni, Esposito, Susanna Maria Roberta, Ferrara, Pietro, Iughetti, Lorenzo, Laforgia, Nicola, Maghnie, Mohamad, Marseglia, Gianluigi, Perilongo, Giorgio, Pettoello-Mantovani, Massimo, Ruggieri, Martino, Russo, Giovanna, Salerno, Mariacarolina, Striano, Pasquale, Valerio, Giuliana, Wasniewska, Malgorzata, Agosti, Massimo, Agostoni, Carlo Virginio, Aiuti, Alessandro, Azzari, Chiara, Badolato, Raffaele, Balduzzi, Adriana, Baraldi, Eugenio, Canani, Roberto Berni, Biffi, Alessandra, Biondi, Andrea, Bisogno, Gianni, Pierri, Nicola Brunetti, Carnielli, Virginio, Cianfarani, Stefano, Cogo, Paola, Corvaglia, Luigi, Dani, Carlo, Di Salvo, Giovanni, Fagioli, Franca, Fanos, Vassilios, Ferrero, Giovanni Battista, Francavilla, Ruggiero, Galli, Luisa, Gazzolo, Diego, Giaquinto, Carlo, Giordano, Paola, Gitto, Eloisa, Grosso, Salvatore, Guarino, Alfredo, Indrio, Flavia, Lanari, Marcello, Lionetti, Paolo, Locatelli, Franco, Lombardo, Fortunato, Maffeis, Claudio, Marino, Bruno, Midulla, Fabio, Del Giudice, Emanuele Miraglia, Del Giudice, Michele Miraglia, Montini, Giovanni, Parenti, Giancarlo, Parisi, Pasquale, Peroni, Diego, Perrotta, Silverio, Piacentini, Giorgio, Pietrobelli, Angelo, Raimondi, Francesco, Ramenghi, Ugo, Ravelli, Angelo, Romano, Claudio, Rossi, Francesca, Rossi, Paolo, Damiano, Vincenzo Salpietro, Spalice, Alberto, Suppiej, Agnese, Troncone, Riccardo, Verrotti, Alberto, null, null, Calcaterra, V, Tornese, G, Zuccotti, G, Staiano, A, Cherubini, V, Gaudino, R, Fazzi, E, Barbi, E, Chiarelli, F, Corsello, G, Esposito, S, Ferrara, P, Iughetti, L, Laforgia, N, Maghnie, M, Marseglia, G, Perilongo, G, Pettoello-Mantovani, M, Ruggieri, M, Russo, G, Salerno, M, Striano, P, Valerio, G, Wasniewska, M, Agosti, M, Agostoni, C, Aiuti, A, Azzari, C, Badolato, R, Balduzzi, A, Baraldi, E, Canani, R, Biffi, A, Biondi, A, Bisogno, G, Pierri, N, Carnielli, V, Cianfarani, S, Cogo, P, Corvaglia, L, Dani, C, Di Salvo, G, Fagioli, F, Fanos, V, Ferrero, G, Francavilla, R, Galli, L, Gazzolo, D, Giaquinto, C, Giordano, P, Gitto, E, Grosso, S, Guarino, A, Indrio, F, Lanari, M, Lionetti, P, Locatelli, F, Lombardo, F, Maffeis, C, Marino, B, Midulla, F, Del Giudice, E, Del Giudice, M, Montini, G, Parenti, G, Parisi, P, Peroni, D, Perrotta, S, Piacentini, G, Pietrobelli, A, Raimondi, F, Ramenghi, U, Ravelli, A, Romano, C, Rossi, F, Rossi, P, Damiano, V, Spalice, A, Suppiej, A, Troncone, R, Verrotti, A, Null, N, Calcaterra, Valeria, Tornese, Gianluca, Zuccotti, Gianvincenzo, Staiano, Annamaria, Cherubini, Valentino, Gaudino, Rossella, Fazzi, Elisa Maria, Barbi, Egidio, Chiarelli, Francesco, Corsello, Giovanni, Esposito, Susanna Maria Roberta, Ferrara, Pietro, Iughetti, Lorenzo, Laforgia, Nicola, Maghnie, Mohamad, Marseglia, Gianluigi, Perilongo, Giorgio, Pettoello-Mantovani, Massimo, Ruggieri, Martino, Russo, Giovanna, Salerno, Mariacarolina, Striano, Pasquale, Valerio, Giuliana, Wasniewska, Malgorzata, Agosti, Massimo, Agostoni, Carlo Virginio, Aiuti, Alessandro, Azzari, Chiara, Badolato, Raffaele, Balduzzi, Adriana, Baraldi, Eugenio, Canani, Roberto Berni, Biffi, Alessandra, Biondi, Andrea, Bisogno, Gianni, Pierri, Nicola Brunetti, Carnielli, Virginio, Cianfarani, Stefano, Cogo, Paola, Corvaglia, Luigi, Dani, Carlo, Di Salvo, Giovanni, Fagioli, Franca, Fanos, Vassilios, Ferrero, Giovanni Battista, Francavilla, Ruggiero, Galli, Luisa, Gazzolo, Diego, Giaquinto, Carlo, Giordano, Paola, Gitto, Eloisa, Grosso, Salvatore, Guarino, Alfredo, Indrio, Flavia, Lanari, Marcello, Lionetti, Paolo, Locatelli, Franco, Lombardo, Fortunato, Maffeis, Claudio, Marino, Bruno, Midulla, Fabio, Del Giudice, Emanuele Miraglia, Del Giudice, Michele Miraglia, Montini, Giovanni, Parenti, Giancarlo, Parisi, Pasquale, Peroni, Diego, Perrotta, Silverio, Piacentini, Giorgio, Pietrobelli, Angelo, Raimondi, Francesco, Ramenghi, Ugo, Ravelli, Angelo, Romano, Claudio, Rossi, Francesca, Rossi, Paolo, Damiano, Vincenzo Salpietro, Spalice, Alberto, Suppiej, Agnese, Troncone, Riccardo, Verrotti, Alberto, and null, null

Abstract

Background In response to the imperative need for standardized support for adolescent Gender Dysphoria (GD), the Italian Academy of Pediatrics, in collaboration with the Italian Society of Pediatrics, the Italian Society for Pediatric Endocrinology and Diabetes, Italian Society of Adolescent Medicine and Italian Society of Child and Adolescent Neuropsychiatry is drafting a position paper. The purpose of this paper is to convey the author's opinion on the topic, offering foundational information on potential aspects of gender-affirming care and emphasizing the care and protection of children and adolescents with GD.Main body Recognizing that adolescents may choose interventions based on their unique needs and goals and understanding that every individual within this group has a distinct trajectory, it is crucial to ensure that each one is welcomed and supported. The approach to managing individuals with GD is a multi-stage process involving a multidisciplinary team throughout all phases. Decisions regarding treatment should be reached collaboratively by healthcare professionals and the family, while considering the unique needs and circumstances of the individual and be guided by scientific evidence rather than biases or ideologies. Politicians and high court judges should address discrimination based on gender identity in legislation and support service development that aligns with the needs of young people. It is essential to establish accredited multidisciplinary centers equipped with the requisite skills and experience to effectively manage adolescents with GD, thereby ensuring the delivery of high-quality care.Conclusion Maintaining an evidence-based approach is essential to safeguard the well-being of transgender and gender diverse adolescents.

Published
2024

17. Anti-T-lymphocyte globulin exposure is associated with acute graft-versus-host disease and relapse in pediatric acute lymphoblastic leukemia patients undergoing hematopoietic stem cell transplantation: a multinational prospective study

Author

Oostenbrink, L, Von Asmuth, E, Jol-van der Zijde, C, Jansen-Hoogendijk, A, Vervat, C, Bredius, R, Van Tol, M, Schilham, M, Sedlacek, P, Ifversen, M, Balduzzi, A, Bader, P, Peters, C, Moes, D, Lankester, A, Oostenbrink, Lisa V. E., Von Asmuth, Erik G. J., Jol-van der Zijde, Cornelia M., Jansen-Hoogendijk, Anja M., Vervat, Carly, Bredius, Robbert G. M., Van Tol, Maarten J. D., Schilham, Marco W., Sedlacek, Petr, Ifversen, Marianne, Balduzzi, Adriana, Bader, Peter, Peters, Christina, Moes, Dirk Jan A. R., Lankester, Arjan C., Oostenbrink, L, Von Asmuth, E, Jol-van der Zijde, C, Jansen-Hoogendijk, A, Vervat, C, Bredius, R, Van Tol, M, Schilham, M, Sedlacek, P, Ifversen, M, Balduzzi, A, Bader, P, Peters, C, Moes, D, Lankester, A, Oostenbrink, Lisa V. E., Von Asmuth, Erik G. J., Jol-van der Zijde, Cornelia M., Jansen-Hoogendijk, Anja M., Vervat, Carly, Bredius, Robbert G. M., Van Tol, Maarten J. D., Schilham, Marco W., Sedlacek, Petr, Ifversen, Marianne, Balduzzi, Adriana, Bader, Peter, Peters, Christina, Moes, Dirk Jan A. R., and Lankester, Arjan C.

Abstract

Anti T-lymphocyte globulin (ATLG) is used in hematopoietic stem cell transplantation (HSCT) to prevent graft-versus-host disease (GvHD) and graft failure. To date, insight in ATLG pharmacokinetics and -dynamics (PK/PD) is limited, and population PK (POPPK) models are lacking. In this prospective study, we describe ATLG POPPK using NONMEM® and the impact of ATLG exposure on clinical outcome and immune reconstitution in a homogeneous cohort of pediatric acute lymphoblastic leukemia (ALL) patients transplanted with a matched unrelated donor and receiving uniform ATLG dosing. Based on 121 patients and 812 samples for POPPK analysis, a two-compartmental model with parallel linear and non-linear clearance and bodyweight as covariate, best described the ATLG concentrationtime data. The level of ATLG exposure (day active ATLG day 16 8.2%, p.

Published
2024

18. Long-term complications after allogeneic hematopoietic stem cell transplantation for pediatric patients with acute leukemia or myelodysplastic syndrome given either a Treosulfan- or a Busulfan-based conditioning regimen: results of an AIEOP (Associazione Italiana Ematologia Oncologia Pediatrica) retrospective study

Author

Saglio, F, Pagliara, D, Zecca, M, Balduzzi, A, Cattoni, A, Prete, A, Tambaro, F, Faraci, M, Calore, E, Locatelli, F, Fagioli, F, Saglio, Francesco, Pagliara, Daria, Zecca, Marco, Balduzzi, Adriana, Cattoni, Alessandro, Prete, Arcangelo, Tambaro, Francesco Paolo, Faraci, Maura, Calore, Elisabetta, Locatelli, Franco, Fagioli, Franca, Saglio, F, Pagliara, D, Zecca, M, Balduzzi, A, Cattoni, A, Prete, A, Tambaro, F, Faraci, M, Calore, E, Locatelli, F, Fagioli, F, Saglio, Francesco, Pagliara, Daria, Zecca, Marco, Balduzzi, Adriana, Cattoni, Alessandro, Prete, Arcangelo, Tambaro, Francesco Paolo, Faraci, Maura, Calore, Elisabetta, Locatelli, Franco, and Fagioli, Franca

Abstract

Patients undergoing hematopoietic stem cell transplantation (HSCT) for hematologic malignancies during childhood have an increased risk of developing long-term sequelae that are in part attributable to the conditioning regimen. The present study aimed to assess the occurrence of long-term toxicities in a population of children who underwent HSCT for hematologic malignancies using either treosulfan or busulfan in the conditioning regimen. The cumulative incidences of growth impairment, altered gonadal function, altered thyroid function, cataracts, secondary malignant neoplasia, and altered pulmonary function were evaluated retrospectively by univariable and multivariable analyses in a population of 521 pediatric patients with acute leukemias or myelodysplastic syndromes treated in 20 Italian transplant centers affiliated with the Associazione Italiana Ematologia ed Oncologia Pediatrica (AIEOP). The median duration of follow-up for the entire study population was 7.1 years (range, 1 to 16 years). Overall, a larger proportion of patients given busulfan developed long-term toxicities compared to patients treated with treosulfan (34% versus 20%; P = .01). In univariable analysis, gonadal toxicity developed in 10% of patients who received treosulfan (95% confidence interval [CI], 3% to 15%), compared with 38% (95% CI, 24% to 39%) of busulfan-treated patients (P = .02), and this finding was confirmed by multivariable analysis (relative risk, .51; 95% CI, .34 to .76; P = .0009). We did not find any statistically significant associations between the occurrence of other long-term toxicities and the use of either busulfan or treosulfan. This study provides evidence that the use of treosulfan is correlated with a reduced incidence of gonadal toxicity in children undergoing HSCT for hematologic malignancies.

Published
2024

19. Low rate of nonrelapse mortality in under 4-year-olds with ALL given chemo-conditioning for HSCT: Phase III FORUM study

Author

Bader, P, Poetschger, U, Dalle, J, Moser, L, Balduzzi, A, Ansari, M, Buechner, J, Güngör, T, Ifversen, M, Kriván, G, Pichler, H, Renard, M, Staciuk, R, Sedlacek, P, Stein, J, Heusel, J, Truong, T, Wachowiak, J, Yeşilipek, M, Locatelli, F, Peters, C, Bader, Peter, Poetschger, Ulrike, Dalle, Jean-Hugues, Moser, Laura M, Balduzzi, Adriana Cristina, Ansari, Marc, Buechner, Jochen, Güngör, Tayfun, Ifversen, Marianne, Kriván, Gergely, Pichler, Herbert, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr, Stein, Jerry, Heusel, Jan Robert, Truong, Tony, Wachowiak, Jacek, Yeşilipek, M Akif, Locatelli, Franco, Peters, Christina, Bader, P, Poetschger, U, Dalle, J, Moser, L, Balduzzi, A, Ansari, M, Buechner, J, Güngör, T, Ifversen, M, Kriván, G, Pichler, H, Renard, M, Staciuk, R, Sedlacek, P, Stein, J, Heusel, J, Truong, T, Wachowiak, J, Yeşilipek, M, Locatelli, F, Peters, C, Bader, Peter, Poetschger, Ulrike, Dalle, Jean-Hugues, Moser, Laura M, Balduzzi, Adriana Cristina, Ansari, Marc, Buechner, Jochen, Güngör, Tayfun, Ifversen, Marianne, Kriván, Gergely, Pichler, Herbert, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr, Stein, Jerry, Heusel, Jan Robert, Truong, Tony, Wachowiak, Jacek, Yeşilipek, M Akif, Locatelli, Franco, and Peters, Christina

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) is highly effective for treating pediatric high-risk or relapsed acute lymphoblastic leukemia (ALL). For young children, total body irradiation (TBI) is associated with severe late sequelae. In the FORUMstudy (NCT01949129), we assessed safety, event-free survival (EFS), and overall survival (OS) of 2 TBIfree conditioning regimens in children aged <4 years with ALL. Patients received fludarabine (Flu), thiotepa (Thio), and either busulfan (Bu) or treosulfan (Treo) before HSCT. From 2013 to 2021, 191 children received transplantation and were observed for ≥6 months (median followup: 3 years). The 3-year OS was 0.63 (95% confidence interval [95% CI], 0.52-0.72) and 0.76 (95% CI, 0.64-0.84) for Flu/Thio/Bu and Flu/Thio/Treo (P = .075), respectively. Three-year EFSwas 0.52 (95% CI, 0.41-0.61) and 0.51 (95% CI, 0.39-0.62), respectively (P = .794). Cumulative incidence of nonrelapse mortality (NRM)and relapse at 3 years were 0.06 (95% CI, 0.02-0.12) vs 0.03 (95%CI: [removed]1 acute graft-versus-host disease (GVHD) occurred in 29% of patients receiving Flu/Thio/ Bu and 17% of those receiving Flu/Thio/Treo (P = .049), whereas grade 3/4 occurred in 10% and 9%, respectively (P = .813). The 3-year incidence of chronic GVHD was 0.07 (95% CI, 0.03-0.13) vs 0.05 (95% CI, 0.02-0.11), respectively (P = .518). In conclusion, both chemotherapeutic conditioning regimens were well tolerated and NRM was low. However, relapse was the major cause of treatment failure.

Published
2024

20. Low rate of nonrelapse mortality in under-4-year-olds with ALL given chemotherapeutic conditioning for HSCT: a phase 3 FORUM study

Author

Bader, Peter; https://orcid.org/0000-0003-4554-0265, Pötschger, Ulrike, Dalle, Jean-Hugues; https://orcid.org/0000-0002-8406-0793, Moser, Laura M, Balduzzi, Adriana; https://orcid.org/0000-0002-5879-0610, Ansari, Marc; https://orcid.org/0000-0002-9649-6498, Buechner, Jochen; https://orcid.org/0000-0001-5848-4501, Güngör, Tayfun; https://orcid.org/0000-0002-3261-1186, Ifversen, Marianne, Krivan, Gergely, Pichler, Herbert; https://orcid.org/0000-0001-5107-5123, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr; https://orcid.org/0000-0001-7895-2886, Stein, Jerry, Heusel, Jan Robert; https://orcid.org/0000-0002-5753-0817, Truong, Tony, Wachowiak, Jacek, Yesilipek, Akif, Locatelli, Franco; https://orcid.org/0000-0002-7976-3654, Peters, Christina; https://orcid.org/0000-0003-0369-8515, Bader, Peter; https://orcid.org/0000-0003-4554-0265, Pötschger, Ulrike, Dalle, Jean-Hugues; https://orcid.org/0000-0002-8406-0793, Moser, Laura M, Balduzzi, Adriana; https://orcid.org/0000-0002-5879-0610, Ansari, Marc; https://orcid.org/0000-0002-9649-6498, Buechner, Jochen; https://orcid.org/0000-0001-5848-4501, Güngör, Tayfun; https://orcid.org/0000-0002-3261-1186, Ifversen, Marianne, Krivan, Gergely, Pichler, Herbert; https://orcid.org/0000-0001-5107-5123, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr; https://orcid.org/0000-0001-7895-2886, Stein, Jerry, Heusel, Jan Robert; https://orcid.org/0000-0002-5753-0817, Truong, Tony, Wachowiak, Jacek, Yesilipek, Akif, Locatelli, Franco; https://orcid.org/0000-0002-7976-3654, and Peters, Christina; https://orcid.org/0000-0003-0369-8515

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) is highly effective for treating pediatric high-risk or relapsed acute lymphoblastic leukemia (ALL). For young children, total body irradiation (TBI) is associated with severe late sequelae. In the FORUM study (NCT01949129), we assessed safety, event-free survival (EFS), and overall survival (OS) of 2 TBI-free conditioning regimens in children aged <4 years with ALL. Patients received fludarabine (Flu), thiotepa (Thio), and either busulfan (Bu) or treosulfan (Treo) before HSCT. From 2013 to 2021, 191 children received transplantation and were observed for ≥6 months (median follow-up: 3 years). The 3-year OS was 0.63 (95% confidence interval [95% CI], 0.52-0.72) and 0.76 (95% CI, 0.64-0.84) for Flu/Thio/Bu and Flu/Thio/Treo (P = .075), respectively. Three-year EFS was 0.52 (95% CI, 0.41-0.61) and 0.51 (95% CI, 0.39-0.62), respectively (P = .794). Cumulative incidence of nonrelapse mortality (NRM) and relapse at 3 years were 0.06 (95% CI, 0.02-0.12) vs 0.03 (95% CI: <0.01-0.09) (P = .406) and 0.42 (95% CI, 0.31-0.52) vs 0.45 (95% CI, 0.34-0.56) (P = .920), respectively. Grade >1 acute graft-versus-host disease (GVHD) occurred in 29% of patients receiving Flu/Thio/Bu and 17% of those receiving Flu/Thio/Treo (P = .049), whereas grade 3/4 occurred in 10% and 9%, respectively (P = .813). The 3-year incidence of chronic GVHD was 0.07 (95% CI, 0.03-0.13) vs 0.05 (95% CI, 0.02-0.11), respectively (P = .518). In conclusion, both chemotherapeutic conditioning regimens were well tolerated and NRM was low. However, relapse was the major cause of treatment failure. This trial was registered at www.clinicaltrials.gov as #NCT01949129.

Published
2024

21. Endocrine, auxological and metabolic profile in children and adolescents with Down syndrome: from infancy to the first steps into adult life

Author

Molinari, S, Fossati, C, Nicolosi, M, Di Marco, S, Faraguna, M, Limido, F, Ocello, L, Pellegrinelli, C, Lattuada, M, Gazzarri, A, Lazzerotti, A, Sala, D, Vimercati, C, Capitoli, G, Daolio, C, Biondi, A, Balduzzi, A, Cattoni, A, Molinari, Silvia, Fossati, Chiara, Nicolosi, Maria Laura, Di Marco, Santo, Faraguna, Martha Caterina, Limido, Francesca, Ocello, Laura, Pellegrinelli, Claudia, Lattuada, Martina, Gazzarri, Alessandra, Lazzerotti, Alessandra, Sala, Debora, Vimercati, Chiara, Capitoli, Giulia, Daolio, Cecilia, Biondi, Andrea, Balduzzi, Adriana, Cattoni, Alessandro, Molinari, S, Fossati, C, Nicolosi, M, Di Marco, S, Faraguna, M, Limido, F, Ocello, L, Pellegrinelli, C, Lattuada, M, Gazzarri, A, Lazzerotti, A, Sala, D, Vimercati, C, Capitoli, G, Daolio, C, Biondi, A, Balduzzi, A, Cattoni, A, Molinari, Silvia, Fossati, Chiara, Nicolosi, Maria Laura, Di Marco, Santo, Faraguna, Martha Caterina, Limido, Francesca, Ocello, Laura, Pellegrinelli, Claudia, Lattuada, Martina, Gazzarri, Alessandra, Lazzerotti, Alessandra, Sala, Debora, Vimercati, Chiara, Capitoli, Giulia, Daolio, Cecilia, Biondi, Andrea, Balduzzi, Adriana, and Cattoni, Alessandro

Abstract

Down syndrome (DS) is the most common chromosomal disorder worldwide. Along with intellectual disability, endocrine disorders represent a remarkable share of the morbidities experienced by children, adolescents and young adults with DS. Auxological parameters are plotted on syndrome-specific charts, as growth rates are reduced compared to healthy age- and gender-matched peers. Furthermore, children with DS are at increased risk for thyroid dysfunctions, diabetes mellitus, osteopenia and obesity compared to general population. Additionally, male individuals with DS often show infertility, while women tend to experience menopause at an overall younger age than healthy controls. Given the recent outstanding improvements in the care of severe DS-related comorbidities, infant mortality has dramatically decreased, with a current average life expectancy exceeding 60 years. Accordingly, the awareness of the specificities of DS in this field is pivotal to timely detect endocrine dysfunctions and to undertake a prompt dedicated treatment. Notably, best practices for the screening and monitoring of pediatric endocrine disorders in DS are still controversial. In addition, specific guidelines for the management of metabolic issues along the challenging period of transitioning from pediatric to adult health care are lacking. By performing a review of published literature, we highlighted the issues specifically involving children and adolescent with DS, aiming at providing clinicians with a detailed up-to-date overview of the endocrine, metabolic and auxological disorders in this selected population, with an additional focus on the management of patients in the critical phase of the transitioning from childhood to adult care.

Published
2024

22. The role of haematopoietic stem cell transplantation for sickle cell disease in the era of targeted disease-modifying therapies and gene editing

Author

Amrolia, Persis, Ansari, Marc, Balduzzi, Adriana, Dalassier, Arnaud, Dalle, Jean-Hugues, Hereda Diaz, Cristina, Feuchtinger, Tobias, Locatelli, Franco, Lucchini, Giovanna, Galimard, Jaques-Emmanuel, Gonzalez Vincent, Marta, Handgretinger, Rupert, Kleinschmidt, Katharina, Lawitschka, Anita, Perez Martinez, Antonio, Peters, Christina, Rocha, Vanderson, Ruggeri, Annalisa, Sedlacek, Petr, Svec, Peter, Toporski, Jacek, Yesilipek, Akif, de la Fuente, Josu, Gluckman, Eliane, Makani, Julie, Telfer, Paul, Faulkner, Lawrence, and Corbacioglu, Selim

Published
2020
Full Text
View/download PDF

23. Pelvic ultrasound and pubertal attainment in girls with sexual precocity: the pivotal role of uterine volume in predicting the timing of menarche.

Author

Cattoni, Alessandro, Russo, Gianni, Capitoli, Giulia, Rodari, Giulia, Nicolosi, Maria Laura, Molinari, Silvia, Tondelli, Daniele, Pelliccia, Ciretta, Radaelli, Silvia, Luciano Arosio, Andrea Mario, Fontana, Katia, Tattesi, Giulia, Passoni, Paolo, Boneschi, Annalisa, Giavoli, Claudia, Carla Meroni, Silvia Laura, Stancampiano, Marianna Rita, Garuti, Elda, Biondi, Andrea, and Balduzzi, Adriana

Subjects
PUBERTY, MENARCHE, ULTRASONIC imaging, HIGH-intensity focused ultrasound, STATISTICAL significance, PRECOCIOUS puberty, GONADOTROPIN releasing hormone, TERTIARY care
Abstract

Introduction: Among girls assessed for pubertal precocity, pelvic ultrasound (pUS) may represent a pivotal tool to predict the time expected to elapse between sonographic assessment and the onset of menarche (TUS-M). Accordingly, the present analysis is meant to define the statistical relationship between sonographic parameters and TUS-M, in order to identify the most reliable predictor of the timing of menarche. Methods: Retrospective, multicenter analysis. Girls assessed for sexual precocity and showing sonographic and clinical findings consistent with pubertal onset upon referral were considered eligible. Patients treated with GnRH analogues were excluded and only those who had subsequently achieved complete and spontaneous pubertal attainment and for whom the exact date of menarche was available were included. Overall, we enrolled 184 girls from five tertiary care Italian Centers. Results: The time elapsed (months) between baseline endocrine assessment and spontaneous achievement of menarche showed a negative statistically significant correlation (p<0.0001) with LH (r:-0.61), FSH (r:-0.59), estradiol (r:- 0.52) and stimulated LH values (r:-0.58). Among pUS parameters, ovarian volume (r:-0.17 left, -0.30 right) and uterine body-to-cervix ratio (r:-0.18) poorly correlated with TUS-M, while uterine diameters (r:-0.61 longitudinal, -0.64 anteroposterior) and volume (r:-0.70) achieved a highly statistical significance (p<0.0001). Uterine volume (UV) showed a negative logarithmic relationship with TUS-M and represented the most reliable predictor of the timing of menarche in uni- and multivariable analyses (p <0.001). ROC analyses identified the UV thresholds that best predict the onset of menarche within 18, 12 and 6 months, respectively: 3.76, 6.02 and 8.80 ml. Conclusion: The logarithm of UV shows the best statistical performance in predicting the timing of menarche in girls assessed for pubertal precocity. Accordingly, we developed a user-friendly online application that provides clinicians with an estimation of the months expected to elapse before menarche, based on the UV recorded upon pUS. [ABSTRACT FROM AUTHOR]

Published
2024
Full Text
View/download PDF

27. Matched unrelated donor transplantation versus haploidentical transplantation with post-transplant cyclophosphamide in children with acute myeloid leukemia: a PDWP-EBMT study

Author

Ruggeri, Annalisa, primary, Santoro, Nicole, additional, Galimard, Jacques-Emmanuel, additional, Kalwak, Krzysztof, additional, Algeri, Mattia, additional, Zubarovskaya, Ludmila, additional, Czyzewski, Krzysztof, additional, Skorobogatova, Elena, additional, Sedlacek, Petr, additional, Besley, Caroline, additional, Balduzzi, Adriana, additional, Bertrand, Yves, additional, Peristeri, Julia, additional, Fagioli, Franca, additional, Ifversen, Mariane, additional, Gozdzik, Jolanta, additional, Peters, Christina, additional, Versluijs, Birgitta, additional, Biffi, Alessandra, additional, Prete, Arcangelo, additional, Faraci, Maura, additional, Ghemlas, Ibrahim, additional, Bodova, Ivana, additional, Aleinikova, Olga, additional, Dalissier, Arnaud, additional, Rocha, Vanderson, additional, and Corbacioglu, Selim, additional

Published
2024
Full Text
View/download PDF

28. The impact of donor type on the outcome of pediatric patients with very high risk acute lymphoblastic leukemia. A study of the ALL SCT 2003 BFM-SG and 2007-BFM-International SG

Author

Dalle, Jean-Hugues, Balduzzi, Adriana, Bader, Peter, Pieczonka, Anna, Yaniv, Isaac, Lankester, Arjan, Bierings, Marc, Yesilipek, Akif, Sedlacek, Petr, Ifversen, Marianne, Svec, Peter, Toporski, Jacek, Gungor, Taifun, Wachowiak, Jacek, Glogova, Evgenia, Poetschger, Ulrike, and Peters, Christina

Published
2021
Full Text
View/download PDF

29. Pubertal attainment and Leydig cell function following pediatric hematopoietic stem cell transplantation: a three-decade longitudinal assessment

Author

Cattoni, Alessandro, primary, Nicolosi, Maria Laura, additional, Capitoli, Giulia, additional, Gadda, Alberto, additional, Molinari, Silvia, additional, Louka, Sotiris, additional, Buonsante, Andrea, additional, Orlandi, Simona, additional, Salierno, Gianluca, additional, Bellani, Iacopo, additional, Vendemini, Francesca, additional, Ottaviano, Giorgio, additional, Gaiero, Alberto, additional, Fichera, Graziella, additional, Biondi, Andrea, additional, and Balduzzi, Adriana, additional

Published
2023
Full Text
View/download PDF

32. Sleeping Beauty-engineered CAR T cells achieve antileukemic activity without severe toxicities

Author

Biondi, Andrea, Magnani, Chiara F., Gaipa, Gissuppe, Lussana, Federico, Belotti, Daniela, Gritti, Giuseppe, Napolitano, Sara, Matera, Giada, Cabiati, Benedetta, Buracchi, Chiara, Borleri, Gianmaria, Fazio, Grazia, Zaninelli, Silvia, Tettamanti, Sarah, Cesana, Stefania, Colombo, Valentina, Quaroni, Michele, Cazzaniga, Giovanni, Rovelli, Attilio, Biagi, Ettore, Galimberti, Stefania, Benedicenti, Fabrizio, Montini, Eugenio, Ferrari, Silvia, Introna, Martino, Balduzzi, Adriana, Valsecchi, Maria Grazia, Dastoli, Giuseppe, Rambaldi, Alessandro, and Calabria, Andrea

Subjects
Transposons -- Research, Immunological research, T cells -- Health aspects, Biological products -- Usage, Cellular therapy -- Methods, Immunotherapy -- Methods, Acute lymphocytic leukemia -- Care and treatment, Biomedical engineering -- Research, Health care industry
Abstract

BACKGROUND. Chimeric antigen receptor (CAR) T cell immunotherapy has resulted in complete remission (CR) and durable response in highly refractory patients. However, logistical complexity and high costs of manufacturing autologous viral products limit CAR T cell availability. METHODS. We report the early results of a phase I/II trial in B cell acute lymphoblastic leukemia (B-ALL) patients relapsed after allogeneic hematopoietic stem cell transplantation (HSCT) using donor-derived CD19 CAR T cells generated with the Sleeping Beauty (SB) transposon and differentiated into cytokine-induced killer (CIK) cells. RESULTS. The cellular product was produced successfully for all patients from the donor peripheral blood (PB) and consisted mostly of [CD3.sup.+] lymphocytes with 43% CAR expression. Four pediatric and 9 adult patients were infused with a single dose of CAR T cells. Toxicities reported were 2 grade I and 1 grade II cytokine-release syndrome (CRS) cases at the highest dose in the absence of graft-versus-host disease (GVHD), neurotoxicity, or dose-limiting toxicities. Six out of 7 patients receiving the highest doses achieved CR and CR with incomplete blood count recovery (CRi) at day 28. Five out of 6 patients in CR were also minimal residual disease negative (MRD). Robust expansion was achieved in the majority of the patients. CAR T cells were measurable by transgene copy PCR up to 10 months. Integration site analysis showed a positive safety profile and highly polyclonal repertoire in vitro and at early time points after infusion. CONCLUSION. SB-engineered CAR T cells expand and persist in pediatric and adult B-ALL patients relapsed after HSCT. Antileukemic activity was achieved without severe toxicities. TRIAL REGISTRATION. ClinicalTrials.gov NCT03389035. FUNDING. This study was supported by grants from the Fondazione AIRC per la Ricerca sul Cancro (AIRC); Cancer Research UK (CRUK); the Fundacion Cientifica de la Asociacion Espanola Contra el Cancer (FC AECC); Ministero Della Salute; Fondazione Regionale per la Ricerca Biomedica (FRRB)., Introduction Substantial efforts over the past few years have led chimeric antigen receptor (CAR) T cell therapy to success in relapsed and refractory (r/r) B cell malignancies. The early concept [...]

Published
2020
Full Text
View/download PDF

34. Occurrence of long-term effects after hematopoietic stem cell transplantation in children affected by acute leukemia receiving either busulfan or total body irradiation: results of an AIEOP (Associazione Italiana Ematologia Oncologia Pediatrica) retrospective study

Author

Saglio, Francesco, Zecca, Marco, Pagliara, Daria, Giorgiani, Giovanna, Balduzzi, Adriana, Calore, Elisabetta, Favre, Claudio, Faraci, Maura, Prete, Arcangelo, Tambaro, Francesco Paolo, Quarello, Paola, Locatelli, Franco, and Fagioli, Franca

Published
2020
Full Text
View/download PDF

35. Supportive care during pediatric hematopoietic stem cell transplantation: beyond infectious diseases. A report from workshops on supportive care of the Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT)

Author

Nava, Tiago, Ansari, Marc, Dalle, Jean-Hugues, de Heredia, Christina Diaz, Güngör, Tayfun, Trigoso, Eugenia, Falkenberg, Ulrike, Bertaina, Alice, Gibson, Brenda, Jarisch, Andrea, Balduzzi, Adriana, Boenig, Halvard, Krivan, Gergely, Vettenranta, Kim, Matic, Toni, Buechner, Jochen, Kalwak, Krzysztof, Lawitschka, Anita, Yesilipek, Akif, Lucchini, Giovanna, Peters, Christina, Turkiewicz, Dominik, Niinimäki, Riitta, Diesch, Tamara, Lehrnbecher, Thomas, Sedlacek, Petr, Hutt, Daphna, Dalissier, Arnaud, Wachowiak, Jacek, Yaniv, Isaac, Stein, Jerry, Yalçin, Koray, Sisinni, Luisa, Deiana, Marco, Ifversen, Marianne, Kuhlen, Michaela, Meisel, Roland, Bakhtiar, Shahrzad, Cesaro, Simone, Willasch, Andre, Corbacioglu, Selim, and Bader, Peter

Published
2020
Full Text
View/download PDF

36. More precisely defining risk peri-HCT in pediatric ALL: pre- vs post-MRD measures, serial positivity, and risk modeling

Author

Bader, Peter, Salzmann-Manrique, Emilia, Balduzzi, Adriana, Dalle, Jean-Hugues, Woolfrey, Ann E., Bar, Merav, Verneris, Michael R., Borowitz, Michael J., Shah, Nirali N., Gossai, Nathan, Shaw, Peter J., Chen, Allen R., Schultz, Kirk R., Kreyenberg, Hermann, Di Maio, Lucia, Cazzaniga, Gianni, Eckert, Cornelia, van der Velden, Vincent H.J., Sutton, Rosemary, Lankester, Arjan, Peters, Christina, Klingebiel, Thomas E., Willasch, Andre M., Grupp, Stephan A., and Pulsipher, Michael A.

Published
2019
Full Text
View/download PDF

37. Transplantation in Children and Adolescents with Acute Lymphoblastic Leukemia from a Matched Donor versus an HLA-Identical Sibling: Is the Outcome Comparable? Results from the International BFM ALL SCT 2007 Study

Author

Balduzzi, Adriana, Dalle, Jean-Hugues, Wachowiak, Jacek, Yaniv, Isaac, Yesilipek, Akif, Sedlacek, Petr, Bierings, Marc, Ifversen, Marianne, Sufliarska, Sabina, Kalwak, Krzysztof, Lankester, Arjan, Toporski, Jacek, Di Maio, Lucia, Glogova, Evgenia, Poetschger, Ulrike, and Peters, Christina

Published
2019
Full Text
View/download PDF

38. Unrelated donor vs HLA-haploidentical α/β T-cell– and B-cell–depleted HSCT in children with acute leukemia

Author

Bertaina, Alice, Zecca, Marco, Buldini, Barbara, Sacchi, Nicoletta, Algeri, Mattia, Saglio, Francesco, Perotti, Cesare, Gallina, Anna Maria, Bertaina, Valentina, Lanino, Edoardo, Prete, Arcangelo, Barberi, Walter, Tumino, Manuela, Favre, Claudio, Cesaro, Simone, Del Bufalo, Francesca, Ripaldi, Mimmo, Boghen, Stella, Casazza, Gabriella, Rabusin, Marco, Balduzzi, Adriana, Fagioli, Franca, Pagliara, Daria, and Locatelli, Franco

Published
2018
Full Text
View/download PDF

39. Endocrine, auxological and metabolic profile in children and adolescents with Down syndrome: from infancy to the first steps into adult life.

Author

Molinari, Silvia, Fossati, Chiara, Nicolosi, Maria Laura, Di Marco, Santo, Faraguna, Martha Caterina, Limido, Francesca, Ocello, Laura, Pellegrinelli, Claudia, Lattuada, Martina, Gazzarri, Alessandra, Lazzerotti, Alessandra, Sala, Debora, Vimercati, Chiara, Capitoli, Giulia, Daolio, Cecilia, Biondi, Andrea, Balduzzi, Adriana, and Cattoni, Alessandro

Subjects
DOWN syndrome, ENDOCRINE diseases, YOUNG adults, ADULTS, THYROID diseases, CHILDREN with intellectual disabilities, YOUNG women
Abstract

Down syndrome (DS) is the most common chromosomal disorder worldwide. Along with intellectual disability, endocrine disorders represent a remarkable share of the morbidities experienced by children, adolescents and young adults with DS. Auxological parameters are plotted on syndrome-specific charts, as growth rates are reduced compared to healthy age- and gender-matched peers. Furthermore, children with DS are at increased risk for thyroid dysfunctions, diabetes mellitus, osteopenia and obesity compared to general population. Additionally, male individuals with DS often show infertility, while women tend to experience menopause at an overall younger age than healthy controls. Given the recent outstanding improvements in the care of severe DS-related comorbidities, infant mortality has dramatically decreased, with a current average life expectancy exceeding 60 years. Accordingly, the awareness of the specificities of DS in this field is pivotal to timely detect endocrine dysfunctions and to undertake a prompt dedicated treatment. Notably, best practices for the screening and monitoring of pediatric endocrine disorders in DS are still controversial. In addition, specific guidelines for the management of metabolic issues along the challenging period of transitioning from pediatric to adult health care are lacking. By performing a review of published literature, we highlighted the issues specifically involving children and adolescent with DS, aiming at providing clinicians with a detailed up-to-date overview of the endocrine, metabolic and auxological disorders in this selected population, with an additional focus on the management of patients in the critical phase of the transitioning from childhood to adult care. [ABSTRACT FROM AUTHOR]

Published
2024
Full Text
View/download PDF

40. Allogeneic HSCT for classical BCR::ABL1-negative myeloproliferative neoplasms in children: the retrospective study of the EBMT Pediatric Diseases WP

Author

Wachowiak, Jacek, primary, Galimard, Jacques-Emmanuel, additional, Dalissier, Arnaud, additional, Rihani, Rawad, additional, ALSAEDI, HAWAZEN, additional, Wynn, Robert, additional, Dalle, Jean-Hugues, additional, de Latour, Regis Peffault, additional, Sedlacek, Petr, additional, Balduzzi, Adriana, additional, Schroeder, Thomas, additional, Bodova, Ivana, additional, Vicent, Marta Gonzalez, additional, Gruhn, Bernd, additional, Hamladji, Rose-Marie, additional, Kriván, Gergely, additional, Patrick, Katharine, additional, Sobkowiak-Sobierajska, Agnieszka, additional, Stepensky, Polina, additional, Unal, Ali, additional, Amrolia, Persis, additional, Perez-Martinez, Antonio, additional, Rialland, Fanny, additional, Aljurf, Mahmoud D, additional, Isgro', Antonella, additional, Toren, Amos, additional, Bierings, Marc, additional, Corbacioglu, Selim, additional, and Kalwak, Krzysztof, additional

Published
2023
Full Text
View/download PDF

42. Immune effector cell–associated hematotoxicity: EHA/EBMT consensus grading and best practice recommendations

Author

Rejeski, Kai, primary, Subklewe, Marion, additional, Aljurf, Mahmoud, additional, Bachy, Emmanuel, additional, Balduzzi, Adriana, additional, Barba, Pere, additional, Bruno, Benedetto, additional, Benjamin, Reuben, additional, Carrabba, Matteo G., additional, Chabannon, Christian, additional, Ciceri, Fabio, additional, Corradini, Paolo, additional, Delgado, Julio, additional, Di Blasi, Roberta, additional, Greco, Raffaella, additional, Houot, Roch, additional, Iacoboni, Gloria, additional, Jäger, Ulrich, additional, Kersten, Marie José, additional, Mielke, Stephan, additional, Nagler, Arnon, additional, Onida, Francesco, additional, Peric, Zinaida, additional, Roddie, Claire, additional, Ruggeri, Annalisa, additional, Sánchez-Guijo, Fermín, additional, Sánchez-Ortega, Isabel, additional, Schneidawind, Dominik, additional, Schubert, Maria-Luisa, additional, Snowden, John A., additional, Thieblemont, Catherine, additional, Topp, Max, additional, Zinzani, Pier Luigi, additional, Gribben, John G., additional, Bonini, Chiara, additional, Sureda, Anna, additional, and Yakoub-Agha, Ibrahim, additional

Published
2023
Full Text
View/download PDF

44. Thyroid Disorders Following Hematopoietic Stem Cell Transplantation in Childhood: Impact of Conditioning Regimen on Thyroid Dysfunction, Volume Changes, and Occurrence of Nodules

Author

Cattoni, Alessandro, Molinari, Silvia, Gaiero, Alberto, De Lorenzo, Paola, Fichera, Graziella, Riva, Benedetta, Di Marco, Santo, Tommesani, Chiara, Mariani, Eugenia, Medici, Francesco, Pagni, Fabio, Nicolosi, Maria Laura, Fraschini, Donatella, Napolitano, Sara, Rovelli, Attilio, Biondi, Andrea, Valsecchi, Maria Grazia, and Balduzzi, Adriana

Published
2022
Full Text
View/download PDF

45. Allogeneic Stem Cell Transplantation from HLA-Mismatched Donors for Pediatric Patients with Acute Lymphoblastic Leukemia Treated According to the 2003 BFM and 2007 International BFM Studies: Impact of Disease Risk on Outcomes

Author

Dalle, Jean-Hugues, Balduzzi, Adriana, Bader, Peter, Lankester, Arjan, Yaniv, Isaac, Wachowiak, Jacek, Pieczonka, Anna, Bierings, Marc, Yesilipek, Akif, Sedlaçek, Petr, Ifversen, Marianne, Sufliarska, Sabina, Toporski, Jacek, Glogova, Evgenia, Poetschger, Ulrike, and Peters, Christina

Published
2018
Full Text
View/download PDF

46. Phase II Study of Sequential Infusion of Donor Lymphocyte Infusion and Cytokine-Induced Killer Cells for Patients Relapsed after Allogeneic Hematopoietic Stem Cell Transplantation

Author

Introna, Martino, Lussana, Federico, Algarotti, Alessandra, Gotti, Elisa, Valgardsdottir, Rut, Micò, Caterina, Grassi, Anna, Pavoni, Chiara, Ferrari, Maria Luisa, Delaini, Federica, Todisco, Elisabetta, Cavattoni, Irene, Deola, Sara, Biagi, Ettore, Balduzzi, Adriana, Rovelli, Attilio, Parma, Matteo, Napolitano, Sara, Sgroi, Giusy, Marrocco, Emanuela, Perseghin, Paolo, Belotti, Daniela, Cabiati, Benedetta, Gaipa, Giuseppe, Golay, Josée, Biondi, Andrea, and Rambaldi, Alessandro

Published
2017
Full Text
View/download PDF

47. S111: TREATMENT OF POST-TRANSPLANT RELAPSE IN CHILDREN, ADOLESCENTS AND YOUNG ADULTS WITH BCP ALL USING CD19-CAR-T: A EUROPEAN RETROSPECTIVE ANALYSIS OF REAL-WORLD DATA

Author

Bader, Peter, primary, Alsonso, Anna, additional, Attarbaschi, Andishe, additional, Bodmer, Nicole, additional, Bonig, Halvard, additional, Burridge, Saskia, additional, Bourquin, Jean-Pierre, additional, Bücklein, Veit, additional, Cario, Gunnar, additional, Oporto Espuelas, Macarena, additional, Feuhtinger, Tobias, additional, Fuereder, Anna, additional, Hauer, Julia, additional, Hutter, Martin, additional, Kałwak, Krzysztof, additional, Künkele, Annette, additional, Meisel, Roland, additional, Mielcarek-Siedziuk, Monika, additional, Müller, Ingo, additional, Napolitano, Sara, additional, Penack, Olaf, additional, Rives, Susana, additional, Rossig, Claudia, additional, von Stackelberg, Arend, additional, Jarisch, Andrea, additional, Ghorashian, Sara, additional, and Balduzzi, Adriana, additional

Published
2023
Full Text
View/download PDF

49. Busulfan–fludarabine‐ or treosulfan–fludarabine‐based conditioning before allogeneic HSCT from matched sibling donors in paediatric patients with sickle cell disease: A study on behalf of the EBMT Paediatric Diseases and Inborn Errors Working Parties

Author

Cseh, Annamária, Galimard, Jacques‐Emmanuel, de la Fuente, Josu, Isgro, Antonella, Zecca, Marco, Garwer, Birgit, Biffi, Alexandra, Aljurf, Mahmoud, Sundin, Mikael, Belendez, Cristina, Locatelli, Franco, Balduzzi, Adriana, Lawson, Sarah, Sengeloev, Henrik, Ifversen, Marianne, Saccardi, Riccardo, Wynn, Robert, Lankester, Arjan C., Corbacioglu, Selim, and Peters, Christina

Subjects
SICKLE cell anemia, CHILD patients, HEMATOPOIETIC stem cell transplantation, GONADAL diseases, PEDIATRICS
Abstract

This article summarizes a study published in the British Journal of Haematology that examines the outcomes of hematopoietic stem cell transplantation (HSCT) for sickle cell disease (SCD) using different conditioning regimens. The study found that both busulfan-fludarabine (bu-flu) and treosulfan-fludarabine (treo-flu) regimens resulted in excellent overall survival rates and low rates of acute and chronic graft-versus-host disease (GVHD) and graft failure. However, treo-flu had a higher incidence of chronic GVHD and extensive chronic GVHD, but a lower frequency of gonadal damage compared to bu-flu. The study emphasizes the potential of HSCT with myeloablative conditioning in treating SCD patients. The report provides a culturally sensitive and comprehensive analysis of the topic, making it a valuable resource for library patrons conducting research on this subject. [Extracted from the article]

Published
2024
Full Text
View/download PDF

Catalog

Books, media, physical & digital resources
See catalog results