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19 results on '"Mura, RM"'

1. Human Fibrinogen Concentrate and Fresh Frozen Plasma in the Management of Severe Acquired Hypofibrinogenemia in Children with Acute Lymphoblastic Leukemia: Results of a Retrospective Survey

Author

Giordano, P, Grassi, M, Saracco, P, Luciani, M, Colombini, A, Testi, A, Micalizzi, C, Petruzziello, F, Putti, M, Casale, F, Consarino, C, Mura, R, Mastrodicasa, E, Notarangelo, L, Onofrillo, D, Pollio, B, Rizzari, C, Tafuri, S, De Leonardis, F, Corallo, P, Santoro, N, Testi, AM, Putti, MC, Mura, RM, Notarangelo, LD, Corallo, PC, Giordano, P, Grassi, M, Saracco, P, Luciani, M, Colombini, A, Testi, A, Micalizzi, C, Petruzziello, F, Putti, M, Casale, F, Consarino, C, Mura, R, Mastrodicasa, E, Notarangelo, L, Onofrillo, D, Pollio, B, Rizzari, C, Tafuri, S, De Leonardis, F, Corallo, P, Santoro, N, Testi, AM, Putti, MC, Mura, RM, Notarangelo, LD, and Corallo, PC

Abstract

Objective of the Study:In this study we aimed to retrospectively evaluate how centers, belonging to the Associazione Italiana Ematologia e Oncologia Pediatrica (AIEOP), manage severe acquired hypofibrinogenemia in children with acute lymphoblastic leukemia, particularly evaluating the therapeutic role of human fibrinogen concentrate (HFC) and fresh frozen plasma (FFP).Methods:We conducted a survey among AIEOP centers; thereafter, we collected and analyzed data with regard to the treatment of episodes of severe acquired hypofibrinogenemia occurring during the induction and reinduction phases of the AIEOP-BFM ALL 2009 protocol.Results:In total, 15 of the 37 AIEOP centers invited to join the survey agreed to collect the data, with 10 and 5 centers declaring to react to severe acquired hypofibrinogenemia (<70 mg/dL) by administering HFC or FFP, respectively. Of the 150 episodes of severe hypofibrinogenemia occurring in 101 patients, 47.3% were treated with HFC and 52.7% with FFP, with a normalization of fibrinogen levels achieved in greater proportion and in a shorter amount of time in the HFC group as compared with the FFP group. None of the patients presented with bleeding or thrombosis during the observation period.Conclusions:Even with the limitations of the retrospective nature of this study, HFC seems to be a safe and effective alternative to FFP for replacement therapy in case of severe hypofibrinogenemia in children with acute lymphoblastic leukemia.

Published
2019

2. A survey on hematology-oncology pediatric AIEOP centres: The challenge of posterior reversible encephalopathy syndrome

Author

Zama D, Gasperini P, Berger M, Petris M, De Pasquale MD, Cesaro S, Guerzoni ME, Mastrodicasa E, Savina F, Ziino O, Kiren V, Muggeo P, Mura RM, Melchionda F, Zanazzo GA, Supportive Therapy Working Group of Italian Pediatric Haematology and Oncology Association (AIEOP)., Zama D, Gasperini P, Berger M, Petris M, De Pasquale MD, Cesaro S, Guerzoni ME, Mastrodicasa E, Savina F, Ziino O, Kiren V, Muggeo P, Mura RM, Melchionda F, Zanazzo GA, and Supportive Therapy Working Group of Italian Pediatric Haematology and Oncology Association (AIEOP).

Subjects
Diagnostic Imaging, Male, Pediatrics, medicine.medical_specialty, posterior reversible encephalopathy syndrome, Adolescent, Neurological complication, medicine.medical_treatment, Hematopoietic stem cell transplantation, 03 medical and health sciences, 0302 clinical medicine, Risk Factors, Internal medicine, Outcome Assessment, Health Care, medicine, Prevalence, Humans, In patient, Child, Chemotherapy, Hematology, business.industry, Incidence (epidemiology), and hematology, Incidence, Disease Management, Infant, Posterior reversible encephalopathy syndrome, General Medicine, pediatric oncology, medicine.disease, Health Surveys, children, hematology-oncology, posterior reversible encephalopathy syndrome, Italy, 030220 oncology & carcinogenesis, Child, Preschool, pediatric oncology, and hematology, Female, Posterior Leukoencephalopathy Syndrome, Symptom Assessment, business, Hematology+Oncology, 030217 neurology & neurosurgery
Abstract

Objectives Posterior reversible encephalopathy syndrome (PRES) is one of the most common neurological complications in hematology-oncology pediatric patients. Despite an increasingly recognized occurrence, no clear consensus exists regarding how best to manage the syndrome, because most cases of PRES have reported in single case reports or small series. Aim of this paper is to identify incidence, clinical features, management and outcome of PRES in a large series of hematology-oncology pediatric patients Methods The cases of PRES occurred in twelve centres of the Italian Association of Pediatric Haematology and Oncology were reported Results 124 cases of PRES in 112 pediatric patients were recorded with an incidence of 2.1% and 4.7% respectively in acute lymphoblastic leukemia in first complete remission and hematopoietic stem cell transplantation (HSCT). The majority of cases occurred after a cycle of chemotherapy rather than after stem cell transplant. PRES after chemotherapy significantly differs from that after HSCT for diagnosis, time of presentation, risk factors, management and outcome Conclusions This study demonstrates that PRES is a common neurological complication and occurring preferentially in course of induction treatment of some hematologic malignancies, as ALL and after HSCT. It also highlights great clinical differences in the management and outcome in patients with PRES occurring after chemotherapy or after HSCT This article is protected by copyright. All rights reserved.

Published
2017

3. Italian cancer figures, report 2012: Cancer in children and adolescents

Author

Pisani, P, Buzzoni, C, Crocetti, E, Maso, L, Rondelli, R, Alessi, D, Aricò, M, Bidoli, E, Ferrari, A, Fusco, M, Gatta, G, Guzzinati, S, Jankovic, M, Locatelli, F, Maule, M, Mosso, M, Pastore, G, Pession, A, Rashid, I, Sacerdote, C, Terracini, B, Tognazzo, S, Trama, A, Ferretti, S, Porta, F, Mazzoleni, G, Tschugguel, B, De Valiere, E, Facchinelli, G, Falk, M, Cappello, T, Giacomin, A, Vercellino, P, Andreone, S, Marzola, L, Migliari, E, Carletti, N, Nenci, I, Caldarella, A, Corbinelli, A, Intrieri, T, Manneschi, G, Nemcova, L, Ocello, C, Sacchettini, C, Zappa, M, Paci, E, Serraino, D, Angelin, T, Birri, S, De Dottori, M, De Santis, E, Forgiarini, O, Zucchetto, A, Zanier, L, Vercelli, M, Orengo, M, Casella, C, Marani, E, Puppo, A, Celesia, M, Cogno, R, Levrieri, I, Manenti, S, Garrone, E, Quaglia, A, Pannozzo, F, Busco, S, Ramazzotti, V, Cercato, M, Battisti, W, Sperduti, I, Macci, L, Bugliarello, E, Valerio, O, Bernazza, E, Tamburo, L, Rossi, M, Curatella, S, Tamburrino, S, Fattoruso, S, Bisanti, L, Autelitano, M, Cuccaro, F, Randi, G, Ghilardi, S, Leone, R, Filipazzi, L, Bonini, A, Giubelli, C, Federico, M, Artioli, M, Valla, K, Braghiroli, B, Cirilli, C, Luminari, S, Pirani, M, Ferrari, L, Bellatalla, C, Panico, M, Perrotta, C, Vassante, B, Vitale, M, Michiara, M, Bozzani, F, Sgargi, P, Tumino, R, Rosa, M, Cascone, G, Frasca, G, Giurdanella, M, Martorana, C, Morana, G, Nicita, C, Rollo, P, Ruggeri, M, Sigona, A, Spata, E, Vacirca, S, Mangone, L, Di Felice, E, Pezzarossi, A, Caroli, S, Pellegri, C, Vicentini, M, D'Angelo, S, Balottari, P, Romagna, R, Falcini, F, Colamartini, A, Bucchi, L, Balducci, C, Ravegnani, M, Vitali, B, Cordaro, C, Caprara, L, Giuliani, O, Giorgetti, S, Palumbo, M, Vattiato, R, Ravaioli, A, Mancini, S, Tonelli, C, Amadori, M, Cremone, L, Iannelli, A, Senatore, G, Zevola, A, Sechi, O, Budroni, M, Cesaraccio, R, Pirino, D, Carboni, D, Fiori, G, Soddu, M, Mameli, G, Mura, F, Contrino, M, Madeddu, A, Tisano, F, Sciacca, S, Muni, A, Mizzi, M, Russo, M, Sacco, G, Aletta, P, Ziino, A, Tessandori, R, Fanetti, A, Maspero, S, Cecconami, L, Annulli, M, Moroni, E, Gonzalez, M, Zanetti, R, Rosso, S, Patriarca, S, Prandi, R, Sobrato, I, Gilardi, F, Busso, P, Piffer, S, Rizzello, R, Gentilini, M, Cappelletti, M, Rosa, F, Stracci, F, D'Alò, D, Scheibel, M, Costarelli, D, Spano, F, Rossini, S, Santucci, C, Petrinelli, A, Solimene, C, Bianconi, F, Brunori, V, Crosignani, P, Tagliabue, G, Contiero, P, Preto, L, Tittarelli, A, Maghini, A, Codazzi, T, Frassoldi, E, Gada, D, Costa, E, Grazia, L, Zambon, P, Baracco, M, Bovo, E, Cin, A, Fiore, A, Greco, A, Monetti, D, Rosano, A, Stocco, C, Donato, F, Limina, R, Adorni, A, Andreis, P, Zani, G, Piovani, F, Salvi, O, Puleio, M, Vitarelli, S, Candela, G, Pappalardo, G, Scuderi, T, Lottero, B, Ribaudo, M, Ricci, P, Guarda, L, Gatti, L, Bozzeda, A, Dall'Acqua, M, Pironi, V, Sardo, A, Mazzei, A, Sirianni, N, Lavecchia, A, Mancuso, P, Vitale, F, Cusimano, R, Amodio, R, Guttadauro, A, Ravazzolo, B, Bucalo, G, Trapani, C, Sunseri, R, Cascio, M, Brucculeri, M, Adamo, M, Mannino, R, Mistretta, A, Costa, A, Rudisi, G, Gola, G, Corti, M, Prosperini, E, Grandi, L, Sciacchitano, S, Fidelbo, M, Benedetto, G, Vasquez, E, Torrisi, A, Pesce, P, Calabretta, L, Bella, F, Leone, A, Ieni, A, Varvarà, M, Castaing, M, Di Prima, A, Benedetto, A, Paderni, F, Usala, M, Pala, F, Sini, G, Pintori, N, Canu, L, Demurtas, G, Doa, N, Lazzarato, F, Magnani, C, Merletti, F, Sarcerdote, C, Macerata, V, de Leon, M, Domati, F, Rossi, G, Goldoni, C, Rossi, F, De Gaetani, C, Benatti, P, Roncucci, L, Di Gregorio, C, Pedroni, M, Pezzi, A, Maffei, S, Mariani, F, Borsi, E, Cocchioni, M, Pascucci, C, Gennaro, V, Lazzarotto, A, Benfatto, L, Mazzucco, G, Montanaro, F, Traina, A, Carruba, G, Dolcemascolo, C, Staiti, R, Zarcone, M, Bisogno, G, Cuttini, M, De Rosa, M, Haupt, R, Soresina, A, Valsecchi, M, Fagioli, F, Bona, G, Dini, G, Conter, V, Biondi, A, Zecca, M, Fedeli, F, Massimino, M, Nespoli, L, Roncarolo, M, Basso, G, Cesaro, S, Memo, L, Bellettato, M, Tamaro, P, Mascarin, M, Rosolen, A, Izzi, G, Paolucci, P, Borgna Pignatti, C, Vecchi, V, Abate, M, D'Ambrosio, A, Favre, C, Caniglia, M, Pierani, P, Felici, L, Visani, G, Fioritoni, G, Foà, R, Riccardi, R, Frega, G, Clerico, A, Casale, F, Poggi, V, Amendola, G, Filosa, A, Ladogana, S, Presta, G, Tornesello, A, De Mattia, D, Consarino, C, Nobile, F, Sperlì, D, D'Angelo, P, Marino, S, Cosmi, C, Mura, R, Battisti, L, Maso, LD, Mosso, ML, Cappello, TD, Vercellino, PC, Orengo, MA, Celesia, MV, Cercato, MC, Artioli, ME, Vitale, MF, Rosa, MG, Giurdanella, MC, Rollo, PC, Ruggeri, MG, Romagna, Rt, Contrino, ML, Ziino, AC, Fanetti, AC, Annulli, ML, Gonzalez, ME, Gentilini, MA, Rosa, FL, Petrinelli, AM, Grazia, Ld, Cin, AD, Fiore, AR, Limina, RM, Sardo, AS, Lavecchia, AM, Cascio, MA, Brucculeri, MA, Adamo, MS, Sini, GM, de Leon, MP, Goldoni, CA, VALSECCHI, MARIA GRAZIA, BIONDI, ANDREA, Roncarolo, MG, Abate, ME, Mura, RM, Battisti, L., Pisani, P, Buzzoni, C, Crocetti, E, Maso, L, Rondelli, R, Alessi, D, Aricò, M, Bidoli, E, Ferrari, A, Fusco, M, Gatta, G, Guzzinati, S, Jankovic, M, Locatelli, F, Maule, M, Mosso, M, Pastore, G, Pession, A, Rashid, I, Sacerdote, C, Terracini, B, Tognazzo, S, Trama, A, Ferretti, S, Porta, F, Mazzoleni, G, Tschugguel, B, De Valiere, E, Facchinelli, G, Falk, M, Cappello, T, Giacomin, A, Vercellino, P, Andreone, S, Marzola, L, Migliari, E, Carletti, N, Nenci, I, Caldarella, A, Corbinelli, A, Intrieri, T, Manneschi, G, Nemcova, L, Ocello, C, Sacchettini, C, Zappa, M, Paci, E, Serraino, D, Angelin, T, Birri, S, De Dottori, M, De Santis, E, Forgiarini, O, Zucchetto, A, Zanier, L, Vercelli, M, Orengo, M, Casella, C, Marani, E, Puppo, A, Celesia, M, Cogno, R, Levrieri, I, Manenti, S, Garrone, E, Quaglia, A, Pannozzo, F, Busco, S, Ramazzotti, V, Cercato, M, Battisti, W, Sperduti, I, Macci, L, Bugliarello, E, Valerio, O, Bernazza, E, Tamburo, L, Rossi, M, Curatella, S, Tamburrino, S, Fattoruso, S, Bisanti, L, Autelitano, M, Cuccaro, F, Randi, G, Ghilardi, S, Leone, R, Filipazzi, L, Bonini, A, Giubelli, C, Federico, M, Artioli, M, Valla, K, Braghiroli, B, Cirilli, C, Luminari, S, Pirani, M, Ferrari, L, Bellatalla, C, Panico, M, Perrotta, C, Vassante, B, Vitale, M, Michiara, M, Bozzani, F, Sgargi, P, Tumino, R, Rosa, M, Cascone, G, Frasca, G, Giurdanella, M, Martorana, C, Morana, G, Nicita, C, Rollo, P, Ruggeri, M, Sigona, A, Spata, E, Vacirca, S, Mangone, L, Di Felice, E, Pezzarossi, A, Caroli, S, Pellegri, C, Vicentini, M, D'Angelo, S, Balottari, P, Romagna, R, Falcini, F, Colamartini, A, Bucchi, L, Balducci, C, Ravegnani, M, Vitali, B, Cordaro, C, Caprara, L, Giuliani, O, Giorgetti, S, Palumbo, M, Vattiato, R, Ravaioli, A, Mancini, S, Tonelli, C, Amadori, M, Cremone, L, Iannelli, A, Senatore, G, Zevola, A, Sechi, O, Budroni, M, Cesaraccio, R, Pirino, D, Carboni, D, Fiori, G, Soddu, M, Mameli, G, Mura, F, Contrino, M, Madeddu, A, Tisano, F, Sciacca, S, Muni, A, Mizzi, M, Russo, M, Sacco, G, Aletta, P, Ziino, A, Tessandori, R, Fanetti, A, Maspero, S, Cecconami, L, Annulli, M, Moroni, E, Gonzalez, M, Zanetti, R, Rosso, S, Patriarca, S, Prandi, R, Sobrato, I, Gilardi, F, Busso, P, Piffer, S, Rizzello, R, Gentilini, M, Cappelletti, M, Rosa, F, Stracci, F, D'Alò, D, Scheibel, M, Costarelli, D, Spano, F, Rossini, S, Santucci, C, Petrinelli, A, Solimene, C, Bianconi, F, Brunori, V, Crosignani, P, Tagliabue, G, Contiero, P, Preto, L, Tittarelli, A, Maghini, A, Codazzi, T, Frassoldi, E, Gada, D, Costa, E, Grazia, L, Zambon, P, Baracco, M, Bovo, E, Cin, A, Fiore, A, Greco, A, Monetti, D, Rosano, A, Stocco, C, Donato, F, Limina, R, Adorni, A, Andreis, P, Zani, G, Piovani, F, Salvi, O, Puleio, M, Vitarelli, S, Candela, G, Pappalardo, G, Scuderi, T, Lottero, B, Ribaudo, M, Ricci, P, Guarda, L, Gatti, L, Bozzeda, A, Dall'Acqua, M, Pironi, V, Sardo, A, Mazzei, A, Sirianni, N, Lavecchia, A, Mancuso, P, Vitale, F, Cusimano, R, Amodio, R, Guttadauro, A, Ravazzolo, B, Bucalo, G, Trapani, C, Sunseri, R, Cascio, M, Brucculeri, M, Adamo, M, Mannino, R, Mistretta, A, Costa, A, Rudisi, G, Gola, G, Corti, M, Prosperini, E, Grandi, L, Sciacchitano, S, Fidelbo, M, Benedetto, G, Vasquez, E, Torrisi, A, Pesce, P, Calabretta, L, Bella, F, Leone, A, Ieni, A, Varvarà, M, Castaing, M, Di Prima, A, Benedetto, A, Paderni, F, Usala, M, Pala, F, Sini, G, Pintori, N, Canu, L, Demurtas, G, Doa, N, Lazzarato, F, Magnani, C, Merletti, F, Sarcerdote, C, Macerata, V, de Leon, M, Domati, F, Rossi, G, Goldoni, C, Rossi, F, De Gaetani, C, Benatti, P, Roncucci, L, Di Gregorio, C, Pedroni, M, Pezzi, A, Maffei, S, Mariani, F, Borsi, E, Cocchioni, M, Pascucci, C, Gennaro, V, Lazzarotto, A, Benfatto, L, Mazzucco, G, Montanaro, F, Traina, A, Carruba, G, Dolcemascolo, C, Staiti, R, Zarcone, M, Bisogno, G, Cuttini, M, De Rosa, M, Haupt, R, Soresina, A, Valsecchi, M, Fagioli, F, Bona, G, Dini, G, Conter, V, Biondi, A, Zecca, M, Fedeli, F, Massimino, M, Nespoli, L, Roncarolo, M, Basso, G, Cesaro, S, Memo, L, Bellettato, M, Tamaro, P, Mascarin, M, Rosolen, A, Izzi, G, Paolucci, P, Borgna Pignatti, C, Vecchi, V, Abate, M, D'Ambrosio, A, Favre, C, Caniglia, M, Pierani, P, Felici, L, Visani, G, Fioritoni, G, Foà, R, Riccardi, R, Frega, G, Clerico, A, Casale, F, Poggi, V, Amendola, G, Filosa, A, Ladogana, S, Presta, G, Tornesello, A, De Mattia, D, Consarino, C, Nobile, F, Sperlì, D, D'Angelo, P, Marino, S, Cosmi, C, Mura, R, Battisti, L, Maso, LD, Mosso, ML, Cappello, TD, Vercellino, PC, Orengo, MA, Celesia, MV, Cercato, MC, Artioli, ME, Vitale, MF, Rosa, MG, Giurdanella, MC, Rollo, PC, Ruggeri, MG, Romagna, Rt, Contrino, ML, Ziino, AC, Fanetti, AC, Annulli, ML, Gonzalez, ME, Gentilini, MA, Rosa, FL, Petrinelli, AM, Grazia, Ld, Cin, AD, Fiore, AR, Limina, RM, Sardo, AS, Lavecchia, AM, Cascio, MA, Brucculeri, MA, Adamo, MS, Sini, GM, de Leon, MP, Goldoni, CA, VALSECCHI, MARIA GRAZIA, BIONDI, ANDREA, Roncarolo, MG, Abate, ME, Mura, RM, and Battisti, L.

Abstract

This study describes up-to-date cancer incidence and survival in Italian paediatric and adolescent patients, based on data collected by the network of Italian cancer registries (AIRTUM). It updates the monograph published on the same topic in 2008. The main objective of this monograph is to present the statistics according to standard rigorous epidemiological methods and disseminate them to a wide range of readers, including the lay public. Given the deep impact of the 2008 monograph on the general public, in this update we complement descriptive statistics with additional data and commentaries on issues of importance for public health, in order to provide unambiguous criteria on how to interpret the statistics. The study is the result of the collaboration between AIRTUM and AIEOP (Italian Association of Paediatric Haematology and Oncology) with contributions from interested parties, including representatives of parent associations. The monograph is divided into three parts. The first part presents incidence rates, survival probabilities, and time trends, by sex, age, geographical area, and cancer site or type, by means of tables and graphs as in the previous monograph, to facilitate direct comparisons. Four articles summarize and comment the results. The second part uses data from AIRTUM and AIEOP to outline patient management and health care issues; it includes estimates of the number of new cases in the next decade and of young adults living after a paediatric cancer diagnosis. Health organizational aspects of treatment services for paediatric patients, based on the AIEOP database, are also discussed, along with long-term complications in cured patients. The third section describes the changes in mortality trends due to improving therapies and healthcare services, and discusses risk factors and prevention of childhood cancer, late adverse events in cured patients, and other related issues.

Published
2013

4. Italian cancer figures, report 2012: Cancer in children and adolescents

Author

Pisani, P, Buzzoni, C, Crocetti, E, Maso, Ld, Rondelli, R, Alessi, D, Aricò, M, Bidoli, E, Ferrari, A, Fusco, M, Gatta, G, Guzzinati, S, Jankovic, M, Locatelli, F, Maule, M, Mosso, Ml, Pastore, G, Pession, A, Rashid, I, Sacerdote, C, Terracini, B, Tognazzo, S, Trama, A, Ferretti, S, Porta, F, Mazzoleni, G, Tschugguel, B, De Valiere, E, Facchinelli, G, Falk, M, Cappello, Td, Giacomin, A, Vercellino, Pc, Andreone, S, Marzola, L, Migliari, E, Carletti, N, Nenci, I, Caldarella, A, Corbinelli, A, Intrieri, T, Manneschi, G, Nemcova, L, Ocello, C, Sacchettini, C, Zappa, M, Paci, E, Serraino, D, Angelin, T, Birri, S, De Dottori, M, De Santis, E, Forgiarini, O, Zucchetto, A, Zanier, L, Vercelli, M, Orengo, Ma, Casella, C, Marani, E, Puppo, A, Celesia, Mv, Cogno, R, Levrieri, I, Manenti, S, Garrone, E, Quaglia, A, Pannozzo, F, Busco, S, Ramazzotti, V, Cercato, Mc, Battisti, W, Sperduti, I, Macci, L, Bugliarello, E, Valerio, O, Bernazza, E, Tamburo, L, Rossi, M, Curatella, S, Tamburrino, S, Fattoruso, S, Bisanti, L, Autelitano, M, Cuccaro, F, Randi, G, Ghilardi, S, Leone, R, Filipazzi, L, Bonini, A, Giubelli, C, Federico, M, Artioli, Me, Valla, K, Braghiroli, B, Cirilli, C, Luminari, S, Pirani, M, Ferrari, L, Bellatalla, C, Panico, M, Perrotta, C, Vassante, B, Vitale, Mf, Michiara, M, Bozzani, F, Sgargi, P, Tumino, R, Rosa, Mg, Cascone, G, Frasca, G, Giurdanella, Mc, Martorana, C, Morana, G, Nicita, C, Rollo, Pc, Ruggeri, Mg, Sigona, A, Spata, E, Vacirca, S, Mangone, L, Di Felice, E, Pezzarossi, A, Caroli, S, Pellegri, C, Vicentini, M, D'Angelo, S, Balottari, P, Romagna, Rt, Falcini, F, Colamartini, A, Bucchi, L, Balducci, C, Ravegnani, M, Vitali, B, Cordaro, C, Caprara, L, Giuliani, O, Giorgetti, S, Palumbo, M, Vattiato, R, Ravaioli, A, Mancini, S, Tonelli, C, Amadori, M, Cremone, L, Iannelli, A, Senatore, G, Zevola, A, Sechi, O, Budroni, M, Cesaraccio, R, Pirino, D, Carboni, D, Fiori, G, Soddu, M, Mameli, G, Mura, F, Contrino, Ml, Madeddu, A, Tisano, F, Sciacca, S, Muni, A, Mizzi, M, Russo, M, Sacco, G, Aletta, P, Ziino, Ac, Tessandori, R, Fanetti, Ac, Maspero, S, Cecconami, L, Annulli, Ml, Moroni, E, Gonzalez, Me, Zanetti, R, Rosso, S, Patriarca, S, Prandi, R, Sobrato, I, Gilardi, F, Busso, P, Piffer, S, Rizzello, R, Gentilini, Ma, Cappelletti, M, Rosa, Fl, Stracci, F, D'Alò, D, Scheibel, M, Costarelli, D, Spano, F, Rossini, S, Santucci, C, Petrinelli, Am, Solimene, C, Bianconi, F, Brunori, V, Crosignani, P, Tagliabue, G, Contiero, P, Preto, L, Tittarelli, A, Maghini, A, Codazzi, T, Frassoldi, E, Gada, D, Costa, E, Grazia, Ld, Zambon, P, Baracco, M, Bovo, E, Cin, Ad, Fiore, Ar, Greco, A, Monetti, D, Rosano, A, Stocco, C, Donato, F, Limina, Rm, Adorni, A, Andreis, P, Zani, G, Piovani, F, Salvi, O, Puleio, M, Vitarelli, S, Candela, G, Pappalardo, G, Scuderi, T, Lottero, B, Ribaudo, M, Ricci, P, Guarda, L, Gatti, L, Bozzeda, A, Dall'Acqua, M, Pironi, V, Sardo, As, Mazzei, A, Sirianni, N, Lavecchia, Am, Mancuso, P, Vitale, F, Cusimano, R, Amodio, R, Guttadauro, A, Ravazzolo, B, Bucalo, G, Trapani, C, Sunseri, R, Cascio, Ma, Brucculeri, Ma, Adamo, Ms, Mannino, R, Mistretta, A, Costa, A, Rudisi, G, Gola, G, Corti, M, Prosperini, E, Grandi, L, Sciacchitano, S, Fidelbo, M, Benedetto, G, Vasquez, E, Torrisi, A, Pesce, P, Calabretta, L, Bella, F, Leone, A, Ieni, A, Varvarà, M, Castaing, M, Di Prima, A, Benedetto, A, Paderni, F, Usala, M, Pala, F, Sini, Gm, Pintori, N, Canu, L, Demurtas, G, Doa, N, Lazzarato, F, Magnani, C, Merletti, F, Sarcerdote, C, Macerata, V, de Leon MP, Domati, F, Rossi, G, Goldoni, Ca, Rossi, F, De Gaetani, C, Benatti, P, Roncucci, L, Di Gregorio, C, Pedroni, M, Pezzi, A, Maffei, S, Mariani, F, Borsi, E, Cocchioni, M, Pascucci, C, Gennaro, V, Lazzarotto, A, Benfatto, L, Mazzucco, G, Montanaro, F, Traina, A, Carruba, G, Dolcemascolo, C, Staiti, R, Zarcone, M, Bisogno, G, Cuttini, M, De Rosa, M, Haupt, R, Soresina, A, Valsecchi, Mg, Fagioli, F, Bona, G, Dini, G, Conter, V, Biondi, A, Zecca, M, Fedeli, F, Massimino, M, Nespoli, L, Roncarolo, Mg, Basso, G, Cesaro, S, Memo, L, Bellettato, M, Tamaro, Paolo, Mascarin, M, Rosolen, A, Izzi, G, Paolucci, P, Borgna Pignatti, C, Vecchi, V, Abate, Me, D'Ambrosio, A, Favre, C, Caniglia, M, Pierani, P, Felici, L, Visani, G, Fioritoni, G, Foà, R, Riccardi, R, Frega, G, Clerico, A, Casale, F, Poggi, V, Amendola, G, Filosa, A, Ladogana, S, Presta, G, Tornesello, A, De Mattia, D, Consarino, C, Nobile, F, Sperlì, D, D'Angelo, P, Marino, S, Cosmi, C, Mura, Rm, Battisti, L., Pisani, P, Buzzoni, C, Crocetti, E, Maso, Ld, Rondelli, R, Alessi, D, Aricò, M, Bidoli, E, Ferrari, A, Fusco, M, Gatta, G, Guzzinati, S, Jankovic, M, Locatelli, F, Maule, M, Mosso, Ml, Pastore, G, Pession, A, Rashid, I, Sacerdote, C, Terracini, B, Tognazzo, S, Trama, A, Ferretti, S, Porta, F, Mazzoleni, G, Tschugguel, B, De Valiere, E, Facchinelli, G, Falk, M, Cappello, Td, Giacomin, A, Vercellino, Pc, Andreone, S, Marzola, L, Migliari, E, Carletti, N, Nenci, I, Caldarella, A, Corbinelli, A, Intrieri, T, Manneschi, G, Nemcova, L, Ocello, C, Sacchettini, C, Zappa, M, Paci, E, Serraino, D, Angelin, T, Birri, S, De Dottori, M, De Santis, E, Forgiarini, O, Zucchetto, A, Zanier, L, Vercelli, M, Orengo, Ma, Casella, C, Marani, E, Puppo, A, Celesia, Mv, Cogno, R, Levrieri, I, Manenti, S, Garrone, E, Quaglia, A, Pannozzo, F, Busco, S, Ramazzotti, V, Cercato, Mc, Battisti, W, Sperduti, I, Macci, L, Bugliarello, E, Valerio, O, Bernazza, E, Tamburo, L, Rossi, M, Curatella, S, Tamburrino, S, Fattoruso, S, Bisanti, L, Autelitano, M, Cuccaro, F, Randi, G, Ghilardi, S, Leone, R, Filipazzi, L, Bonini, A, Giubelli, C, Federico, M, Artioli, Me, Valla, K, Braghiroli, B, Cirilli, C, Luminari, S, Pirani, M, Ferrari, L, Bellatalla, C, Panico, M, Perrotta, C, Vassante, B, Vitale, Mf, Michiara, M, Bozzani, F, Sgargi, P, Tumino, R, Rosa, Mg, Cascone, G, Frasca, G, Giurdanella, Mc, Martorana, C, Morana, G, Nicita, C, Rollo, Pc, Ruggeri, Mg, Sigona, A, Spata, E, Vacirca, S, Mangone, L, Di Felice, E, Pezzarossi, A, Caroli, S, Pellegri, C, Vicentini, M, D'Angelo, S, Balottari, P, Romagna, Rt, Falcini, F, Colamartini, A, Bucchi, L, Balducci, C, Ravegnani, M, Vitali, B, Cordaro, C, Caprara, L, Giuliani, O, Giorgetti, S, Palumbo, M, Vattiato, R, Ravaioli, A, Mancini, S, Tonelli, C, Amadori, M, Cremone, L, Iannelli, A, Senatore, G, Zevola, A, Sechi, O, Budroni, M, Cesaraccio, R, Pirino, D, Carboni, D, Fiori, G, Soddu, M, Mameli, G, Mura, F, Contrino, Ml, Madeddu, A, Tisano, F, Sciacca, S, Muni, A, Mizzi, M, Russo, M, Sacco, G, Aletta, P, Ziino, Ac, Tessandori, R, Fanetti, Ac, Maspero, S, Cecconami, L, Annulli, Ml, Moroni, E, Gonzalez, Me, Zanetti, R, Rosso, S, Patriarca, S, Prandi, R, Sobrato, I, Gilardi, F, Busso, P, Piffer, S, Rizzello, R, Gentilini, Ma, Cappelletti, M, Rosa, Fl, Stracci, F, D'Alò, D, Scheibel, M, Costarelli, D, Spano, F, Rossini, S, Santucci, C, Petrinelli, Am, Solimene, C, Bianconi, F, Brunori, V, Crosignani, P, Tagliabue, G, Contiero, P, Preto, L, Tittarelli, A, Maghini, A, Codazzi, T, Frassoldi, E, Gada, D, Costa, E, Grazia, Ld, Zambon, P, Baracco, M, Bovo, E, Cin, Ad, Fiore, Ar, Greco, A, Monetti, D, Rosano, A, Stocco, C, Donato, F, Limina, Rm, Adorni, A, Andreis, P, Zani, G, Piovani, F, Salvi, O, Puleio, M, Vitarelli, S, Candela, G, Pappalardo, G, Scuderi, T, Lottero, B, Ribaudo, M, Ricci, P, Guarda, L, Gatti, L, Bozzeda, A, Dall'Acqua, M, Pironi, V, Sardo, A, Mazzei, A, Sirianni, N, Lavecchia, Am, Mancuso, P, Vitale, F, Cusimano, R, Amodio, R, Guttadauro, A, Ravazzolo, B, Bucalo, G, Trapani, C, Sunseri, R, Cascio, Ma, Brucculeri, Ma, Adamo, M, Mannino, R, Mistretta, A, Costa, A, Rudisi, G, Gola, G, Corti, M, Prosperini, E, Grandi, L, Sciacchitano, S, Fidelbo, M, Benedetto, G, Vasquez, E, Torrisi, A, Pesce, P, Calabretta, L, Bella, F, Leone, A, Ieni, A, Varvarà, M, Castaing, M, Di Prima, A, Benedetto, A, Paderni, F, Usala, M, Pala, F, Sini, Gm, Pintori, N, Canu, L, Demurtas, G, Doa, N, Lazzarato, F, Magnani, C, Merletti, F, Sarcerdote, C, Macerata, V, de Leon, Mp, Domati, F, Rossi, G, Goldoni, Ca, Rossi, F, De Gaetani, C, Benatti, P, Roncucci, L, Di Gregorio, C, Pedroni, M, Pezzi, A, Maffei, S, Mariani, F, Borsi, E, Cocchioni, M, Pascucci, C, Gennaro, V, Lazzarotto, A, Benfatto, L, Mazzucco, G, Montanaro, F, Traina, A, Carruba, G, Dolcemascolo, C, Staiti, R, Zarcone, M, Bisogno, G, Cuttini, M, De Rosa, M, Haupt, R, Soresina, A, Valsecchi, Mg, Fagioli, F, Bona, G, Dini, G, Conter, V, Biondi, A, Zecca, M, Fedeli, F, Massimino, M, Nespoli, L, Roncarolo, Mg, Basso, G, Cesaro, S, Memo, L, Bellettato, M, Tamaro, Paolo, Mascarin, M, Rosolen, A, Izzi, G, Paolucci, P, Borgna Pignatti, C, Vecchi, V, Abate, Me, D'Ambrosio, A, Favre, C, Caniglia, M, Pierani, P, Felici, L, Visani, G, Fioritoni, G, Foà, R, Riccardi, R, Frega, G, Clerico, A, Casale, F, Poggi, V, Amendola, G, Filosa, A, Ladogana, S, Presta, G, Tornesello, A, De Mattia, D, Consarino, C, Nobile, F, Sperlì, D, D'Angelo, P, Marino, S, Cosmi, C, Mura, Rm, Battisti, L., Maso, L, Mosso, M, Cappello, T, Vercellino, P, Orengo, M, Celesia, M, Cercato, M, Artioli, M, Vitale, M, Rosa, M, Giurdanella, M, Rollo, P, Ruggeri, M, Romagna, R, Contrino, M, Ziino, A, Fanetti, A, Annulli, M, Gonzalez, M, Gentilini, M, Rosa, F, Petrinelli, A, Grazia, L, Cin, A, Fiore, A, Limina, R, Lavecchia, A, Cascio, M, Brucculeri, M, Sini, G, de Leon, M, Goldoni, C, Valsecchi, M, Roncarolo, M, Tamaro, P, Abate, M, Mura, R, Battisti, L, AIRTUM Working Group[.., Enrica Borsi, ], CCM, and AIEOP Working Group

Subjects
Male, Parents, cancer incidence, Adolescent, Epidemiology, Aftercare, cancer in adolescents, Young Adult, children, Neoplasms, cancer in children, Italian population, cancer, Cluster Analysis, Humans, Registries, Survivors, Age of Onset, Mortality, Child, cancer survival, Incidence, Environmental and Occupational Health, Infant, Newborn, Disease Management, Infant, pediatric, Survival Rate, Italy, Child, Preschool, Public Health, Environmental and Occupational Health, Female, Public Health, Morbidity, Forecasting
Abstract

OBJECTIVES: This study describes up-to-date cancer incidence and survival in Italian paediatric and adolescent patients, based on data collected by the network of Italian cancer registries (AIRTUM). It updates the monograph published on the same topic in 2008. The main objective of this monograph is to present the statistics according to standard rigorous epidemiological methods and disseminate them to a wide range of readers, including the lay public. Given the deep impact of the 2008 monograph on the general public, in this update we complement descriptive statistics with additional data and commentaries on issues of importance for public health, in order to provide unambiguous criteria on how to interpret the statistics. The study is the result of the collaboration between AIRTUM and AIEOP (Italian Association of Paediatric Haematology and Oncology) with contributions from interested parties, including representatives of parent associations. The monograph is divided into three parts. The first part presents incidence rates, survival probabilities, and time trends, by sex, age, geographical area, and cancer site or type, by means of tables and graphs as in the previous monograph, to facilitate direct comparisons. Four articles summarize and comment the results. The second part uses data from AIRTUM and AIEOP to outline patient management and health care issues; it includes estimates of the number of new cases in the next decade and of young adults living after a paediatric cancer diagnosis. Health organizational aspects of treatment services for paediatric patients, based on the AIEOP database, are also discussed, along with long-term complications in cured patients. The third section describes the changes in mortality trends due to improving therapies and healthcare services, and discusses risk factors and prevention of childhood cancer, late adverse events in cured patients, and other related issues. MATERIAL AND METHODS: Data herein presented were provided by AIRTUM population-based cancer registries, covering 47%of the Italian population below age 20 years, in the period 2003-2008. Quality of cancer registration in Italy is elevated, with high proportions of microscopically verified diagnoses (91%in the 0-14 years age group and 96% between 15 and 19 years of age) and a very small proportion of cases collected through death certificate only (0.1%).The proportion of cases in diagnostic groups XI (other malignant epithelial neoplasms) and XII (other and unspecified neoplasms) of the International Classification for Childhood Cancer (ICCC), based on the third revision of the International Classification of Diseases for Oncology (ICD-O-3), were 7.0% in the 0-14 years age group and 26.0%in the 15-19 years age group.The ratio between mortality and incidence was 17.7% in both children and adolescents. Detailed results are presented in 24 fact sheets for the 12 major ICCC-3 diagnostic groups and 10 sub-groups of special interest; the series is completed by a sheet on all malignant tumours and one on all tumours including non-malignant neoplasms of the central nervous system. All sheets include results for three age groups (0-14, 15-19, and 0-19 years) and are followed by two commentaries on incidence in the recent period, one on trends and the other on survival. Incidence rates were age-standardized on the European population and presented per million children. Incidence rates are also presented by age group, sex, and geographical area. Incidence trends were evaluated for two periods, 1988-2008 and 1998-2008, using estimated annual percent changes, and survival estimates were calculated by age and period. Indicators and corresponding 95% confidence intervals are shown in forms of graphics and tables at the end of the monograph and online at http://www.registri-tumori.it. Geographical analyses were conducted rearranging cancer registries into four macroareas (North-West, North-East, Centre, and South and Islands). Age groups were the same used in descriptive studies on children worldwide (0, 1-4, 5-9, 10- 14 years for paediatric tumours and 15-19 years for adolescents). Incidence trend analyses included cancer registries with three or more years of registration in the 5-year period, using Poisson regression models. Observed survival was computed according to the Kaplan-Meier method. The estimate of expected cases in the next decade was based on observed incidence rates in the most recent period, extended to the Italian estimated population of children and adolescents in the periods 2011-2015 and 2016-2020. The AIEOP database (Modello 1.01) allowed us to compare the number of patients treated and followed-up in specialized centres with expected cases based on AIRTUM estimates. The AIEOP database also provided information regarding health care migration throughout Italian regions and the number of foreign (immigrated) children treated in Italian AIEOP centres. RESULTS: In the period 2003-2008, 31 cancer registries reported 4,473 incident malignant neoplasms, 2,855 in children and 1,618 in adolescents. Cancer incidence rates were 164 cases per million in children aged 14 years or below and 269 cases per million in patients aged 15-19 years. Limited geographical variations emerged. In children (0-14 years) a significant increase in malignant cancer incidence was observed until 1997 (APC: +3.2%), followed by a plateau (APC: -1.1%not statistically significant).Until the late Nineties, a statistically significant increase was also observed in the incidence of all leukaemias in males (APC: +5.7%), lymphoid leukaemias (APC: +5.6%), representing 80% of all leukaemias, Hodgkin and non- Hodgkin lymphomas (APC: +6.3%). A significant decrease emerged for lymphoid leukaemia starting in 1995 (APC: -1.9%), while no substantial change in cancer incidence rates was observed in the last decade of observation for all malignant neoplasms and lymphomas. In addition, no variation emerged for malignant (according to the most recent classification) central nervous system (CNS) neoplasms, while an annual increase of 1.8% (significant) was observed in the period 1988-2008, when non-malignant tumours were included. Increases in cancer incidence were observed throughout the study period for neuroblastoma (APC: +1.9%) and epithelial tumours or melanoma (APC: +4.1%). In the period 1998-2008, in addition to lymphoid leukaemias, a significant decrease was observed for all malignant neoplasms, lymphomas in girls, CNS tumours (males and females), and renal tumours in girls, while no increases were observed in this age group. In adolescents (15-19 years) between 1988 and 2008, a significant increase in incidence rates was observed (APC: +2.0%) for all malignant neoplasms, all lymphomas (APC: +2.9%; in particular Hodgkin lymphoma, APC: +3.6%), thyroid cancer (APC: +6.1%), and melanoma (APC: +8.1%). Conversely, lymphoid leukaemia is the only neoplasm showing a long-term decrease in adolescents. Recent trends (1998-2008) confirm the long-term increases only for all malignant neoplasms in girls and thyroid cancer (APC: +7.9%, boys and girls), while a decrease in bone tumour incidence emerged in girls, albeit based only on 46 cases. Cancer mortality in children showed a persistent decrease for all neoplasms and even for more frequent cancer sites or types, and mortality rates for cancer were three-fold higher in the early Seventies than in 2008. In addition, five-year survival after cancer diagnosis increased in the last three decades and was still increasing in the period 2003- 2008, reaching 82% in children and 86% in adolescents. In the period 2008-2010, 4,488 children (0-14 years) were treated in one of the AIEOP clinical centres and we estimate, based on the above-presented incidence rates, that they represented 92% of all cancer cases in Italy. However, in adolescents, the proportion of patients treated in AIEOP centres was only 25%. A migration of patients living in the South of Italy to Central and Northern Italy emerged from AIEOP information. The expected number of cancer cases in children aged between 0 and 14 years of age is approximately 7,000 in the period 2016- 2020, while the corresponding figure for adolescents between 15 and 19 years of age is 4,000, with no relevant variation in comparison with the previous five-year period. COMMENTS: The present findings update descriptive cancer epidemiology in children and adolescents in Italy based on data provided by an extensive network of general and specialized population-based cancer registries. Data obtained from cancer registries are supplemented by additional information collected by specialized clinical AIEOP centres and mortality reports collected by the National Institute of Statistics (ISTAT). Incidence rates reported in Italy were slightly higher in comparison to other developed Countries, but relatively consistent between different Italian areas. Our results also showed that the significant increase in cancer incidence observed until the end of Nineties has halted, with the exception solely of thyroid cancer in adolescents. Efficacy of therapeutic protocols has improved constantly since the Seventies, and recent findings confirm this trend in all age groups and, in particular, for rarer tumours and cancer types that have very poor prognosis. Findings derived from cross-analysis with AIEOP data suggest that it is possible to further improve the efficiency of our healthcare system, in particular for adolescents; migration can be reduced with a more rational use of hospitals throughout Italy.

5. Clinical features and follow-up in patients with 22q11.2 deletion syndrome

Author

Cancrini, C, Puliafito, P, Digilio, M, Soresina, A, Martino, S, Rondelli, R, Consolini, R, Ruga, E, Cardinale, F, Finocchi, A, Romiti, Ml, Martire, B, Bacchetta, R, Albano, V, Carotti, A, Specchia, F, Montin, D, Cirillo, E, Cocchi, G, Trizzino, A, Bossi, G, Milanesi, O, Azzari, C, Corsello, G, Pignata, C, Aiuti, A, Pietrogrande, M, Marino, B, Ugazio, A, Plebani, A, Rossi, P, Pierani, P, Gabrielli, A, Danieli, M, De Mattia, D, Sisto, C, Dammacco, F, Ranieri, G, Pession, A, Ricci, G, Minelli, P, Lougaris, V, Badolato, R, Cattaneo, R, Airò, P, Mura, R, Cossu, F, Del Giacco, S, Manconi, P, Consarino, C, Dello Russo, A, Miniero, R, Anastasio, E, Marino, S, Russo, G, Paganelli, R, Sperlì, D, Carpino, L, Aricò, M, Gambineri, E, Lippi, F, Canessa, C, Maggi, E, Romagnani, S, Matucci, A, Vultaggio, A, Gattorno, M, Castagnola, E, Nigro, G, Presta, G, Civino, A, Buzi, F, Gambaretto, G, Fasoli, S, Salpietro, C, Gallizzi, R, Dellepiane, R, Panisi, C, Fabio, G, Carrabba, M, Roncarolo, M, Biondi, A, Vallinoto, C, Poggi, V, Menna, G, Di Nardo, R, Sottile, R, Marone, G, Spadaro, G, Carli, M, Basso, G, Putti, C, Semenzato, G, Agostini, C, D'Angelo, P, Izzi, G, Bertolini, P, Zecca, M, Marseglia, G, Maccario, R, Felici, L, Favre, C, Vecchi, V, Sacchini, P, Rinaldi, G, Livadiotti, S, Simonetti, A, Stabile, A, Duse, M, Iacobini, M, Quinti, I, Fiorilli, M, Moschese, V, Cecere, F, D'Ambrosio, A, De Zan, G, Strafella, S, Tamaro, P, Rabusin, M, Tommasini, A, Tovo, P, De Carli, M, De Carli, S, Nespoli, L, Marinoni, M, Porcellini, A, Lunardi, C, Patuzzo, G, Boner, A, Degani, D, Cancrini, C, Puliafito, P, Digilio, Mc, Soresina, A, Martino, S, Rondelli, R, Consolini, R, Ruga, Em, Cardinale, F, Finocchi, A, Romiti, Ml, Martire, B, Bacchetta, R, Albano, V, Carotti, A, Specchia, F, Montin, D, Cirillo, E, Cocchi, G, Trizzino, A, Bossi, G, Milanesi, O, Azzari, C, Corsello, G, Pignata, C, Aiuti, Alessandro, Pietrogrande, Mc, Marino, B, Ugazio, Ag, Plebani, A, Rossi, P., Cancrini, Caterina, Puliafito, Pamela, Digilio, Maria Cristina, Soresina, Annarosa, Martino, Silvana, Rondelli, Roberto, Consolini, Rita, Ruga, Ezia Maria, Cardinale, Fabio, Finocchi, Andrea, Romiti, Maria Luisa, Martire, Baldassarre, Bacchetta, Rosa, Albano, Veronica, Carotti, Adriano, Specchia, Fernando, Montin, Davide, Cirillo, Emilia, Cocchi, Guido, Trizzino, Antonino, Bossi, Grazia, Milanesi, Ornella, Azzari, Chiara, Corsello, Giovanni, Pignata, Claudio, Pietrogrande, Maria Cristina, Marino, Bruno, Ugazio, Alberto Giovanni, Plebani, Alessandro, Rossi, Paolo, Aiuti, A, Rossi, P, Pierani, P, Gabrielli, A, Danieli, Mg, De Mattia, D, Sisto, C, Dammacco, F, Ranieri, G, Pession, A, Ricci, G, Minelli, P, Lougaris, V, Badolato, R, Cattaneo, R, Airò, P, Mura, Rm, Cossu, F, Del Giacco, S, Manconi, Pe, Consarino, C, Dello Russo, Am, Miniero, R, Anastasio, E, Marino, S, Russo, G, Paganelli, R, Sperlì, D, Carpino, L, Aricò, M, Gambineri, E, Lippi, F, Canessa, C, Maggi, E, Romagnani, S, Matucci, A, Vultaggio, A, Gattorno, M, Castagnola, E, Nigro, G, Presta, G, Civino, A, Buzi, F, Gambaretto, G, Fasoli, S, Salpietro, C, Gallizzi, R, Dellepiane, Rm, Panisi, C, Fabio, G, Carrabba, M, Pietrogrande, M, Roncarolo, Mg, Biondi, A, Vallinoto, C, Poggi, V, Menna, G, Di Nardo, R, Sottile, R, Marone, G, Spadaro, G, Carli, M, Basso, G, Putti, C, Semenzato, G, Agostini, C, D'Angelo, P, Izzi, G, Bertolini, P, Zecca, M, Marseglia, G, Maccario, R, Felici, L, Favre, C, Vecchi, V, Sacchini, P, Rinaldi, G, Livadiotti, S, Simonetti, A, Stabile, A, Duse, M, Iacobini, M, Quinti, I, Fiorilli, M, Moschese, V, Cecere, F, D'Ambrosio, A, De Zan, G, Strafella, S, Tamaro, Paolo, Rabusin, M, Tommasini, A, Tovo, P, De Carli, M, De Carli, S, Nespoli, L, Marinoni, M, Porcellini, A, Lunardi, C, Patuzzo, G, Boner, A, Degani, D., Cancrini, C., Pulisfito, P., Digilio, M. C., Soresina, A., Martino, S., Rondelli, R., Consolini, R., Ruga, E. M., C. a. r. d. i. n. a. l. e., F., Finocchi, A., Romiti, M. L., Martire, B., Bacchetta, R., Albano, V., Carotti, A., Specchia, F., Montin, D., Cocchi, G., Trizzino, A., Bossi, G., Milanesi, O., Azzari, C., Corsello, G., Aiuti, A., Pietrogrande, M. C., Marino, B., Ugazio, A. G., Plebani, A., Digilio, MC, Ruga, EM, Romiti, ML, trizzino, A, Aiuti, Pietrogrande, MC, and Ugazio, AG

Subjects
Male, Pediatrics, 22q11.2 deletion, Delayed Diagnosis, Time Factors, Chromosomes, Human, Pair 22, Developmental Disabilities, digeorge syndrome, Sex Factor, Severity of Illness Index, Retrospective Studie, DiGeorge syndrome, Early Diagnosi, Age Factor, Prospective Studies, Neonatal hypocalcemia, Prospective cohort study, Child, medicine.diagnostic_test, Delayed Diagnosi, Primary immune disorders, Age Factors, del 22q, MIM, Abnormalities, Multiple, Adolescent, Adult, Child, Preschool, DiGeorge Syndrome, Early Diagnosis, Female, Follow-Up Studies, Genetic Testing, Humans, Infant, Infant, Newborn, Monitoring, Physiologic, Retrospective Studies, Risk Assessment, Sex Factors, Young Adult, Disease Progression, Cohort, Abnormalities, Multiple, Pediatrics, Perinatology and Child Health, Human, medicine.medical_specialty, Time Factor, Monitoring, Developmental Disabilitie, Italian Association of Pediatric Haematology and Oncology, Context (language use), Chromosomes, Follow-Up Studie, Severity of illness, medicine, 22q11DS, 22q11.2 deletion syndrome, AIEOP, Mendelian Inheritance in Man, Preschool, Physiologic, Genetic testing, Settore MED/38 - Pediatria Generale e Specialistica, business.industry, Retrospective cohort study, medicine.disease, Newborn, Prospective Studie, Pair 22, business
Abstract

Objective To investigate the clinical manifestations at diagnosis and during follow-up in patients with 22q11.2 deletion syndrome to better define the natural history of the disease. Study design A retrospective and prospective multicenter study was conducted with 228 patients in the context of the Italian Network for Primary Immunodeficiencies. Clinical diagnosis was confirmed by cytogenetic or molecular analysis. Results The cohort consisted of 112 males and 116 females; median age at diagnosis was 4 months (range 0 to 36 years 10 months). The diagnosis was made before 2 years of age in 71% of patients, predominantly related to the presence of heart anomalies and neonatal hypocalcemia. In patients diagnosed after 2 years of age, clinical features such as speech and language impairment, developmental delay, minor cardiac defects, recurrent infections, and facial features were the main elements leading to diagnosis. During follow-up (available for 172 patients), the frequency of autoimmune manifestations ( P = .015) and speech disorders ( P = .002) increased. After a median follow-up of 43 months, the survival probability was 0.92 at 15 years from diagnosis. Conclusions Our data show a delay in the diagnosis of 22q11.2 deletion syndrome with noncardiac symptoms. This study provides guidelines for pediatricians and specialists for early identification of cases that can be confirmed by genetic testing, which would permit the provision of appropriate clinical management.

Published
2014

6. Epilepsy after acute central nervous system complications of pediatric hematopoietic cell transplantation: A retrospective, multicenter study.

Author

Bergonzini L, Leardini D, Rao R, Foiadelli T, Faraci M, Mancardi MM, Nobile G, Orsini A, Savasta S, Gottardi F, Fetta A, Mina T, Casazza G, Menconi MC, Pruna D, Mura RM, Piroddi A, Rucci P, Masetti R, and Cordelli DM

Subjects
Humans, Male, Female, Child, Adolescent, Retrospective Studies, Child, Preschool, Infant, Young Adult, Incidence, Posterior Leukoencephalopathy Syndrome etiology, Posterior Leukoencephalopathy Syndrome epidemiology, Risk Factors, Central Nervous System Diseases etiology, Central Nervous System Diseases epidemiology, Hematopoietic Stem Cell Transplantation adverse effects, Epilepsy etiology, Epilepsy epidemiology, Epilepsy therapy
Abstract

Background: Acute central nervous system (CNS) complications are common and well described among pediatric patients undergoing haematopoietic cell transplantation (HCT). However, their long-term outcomes are not known. The aim of this study is to describe the incidence, characteristics, and risk factors of long-term epilepsy in pediatric patients with acute CNS complications of HCT., Methods: This retrospective study included pediatric patients who developed acute CNS complications from autologous or allogeneic HCT between 2000 and 2022. Clinical, therapeutic and prognostic data including long-term outcomes were analyzed. A diagnosis of epilepsy was provided if unprovoked seizures occurred during follow-up., Results: Ninety-four patients (63 males, 31 females, median age 10 years, range 1-21 years) were included. The most common acute CNS complications were posterior reversible encephalopathy syndrome (n = 43, 46 %) and infections (n = 15, 16 %). Sixty-five patients (69 %) had acute symptomatic seizures, with 14 (16 %) having one or more episodes of status epilepticus (SE). Nine patients (9.6 %) were diagnosed with long-term focal epilepsy during the follow-up (5-year cumulative incidence from the acute complication, 13.3 %). Acute symptomatic SE during neurological complications of HCT was associated with an increased risk of long-term epilepsy (OR=14, 95 % CI 2.87-68.97)., Conclusions: A higher occurrence of epilepsy has been observed in our cohort compared to the general population. Acute symptomatic SE during HCT was associated with a higher risk of long-term epilepsy. Pediatric patients with CNS complications during HCT could benefit from specific neurological follow-up. Further studies are needed to characterize mechanisms of epileptogenesis in pediatric patients undergoing HCT., Competing Interests: Declaration of competing interest The Authors declare no potential conflicts of interest with respect to the research, authorship, and/or publication of this article., (Copyright © 2024 British Epilepsy Association. Published by Elsevier Ltd. All rights reserved.)

Published
2024
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7. TP53 DNA binding domain mutational status and rituximabbased treatment are independent prognostic factors for pediatric Burkitt lymphoma patients stratification.

Author

Martire G, Lovisa F, Carraro E, Rizzato D, Cesaro S, Mura RM, Tondo A, Bertolin C, Boaretto F, Salviati L, Biffi A, Pillon M, and Mussolin L

Subjects
Humans, Child, Prognosis, Male, Female, Child, Preschool, Adolescent, Antineoplastic Combined Chemotherapy Protocols therapeutic use, Rituximab therapeutic use, Burkitt Lymphoma genetics, Burkitt Lymphoma drug therapy, Burkitt Lymphoma mortality, Burkitt Lymphoma diagnosis, Mutation, Tumor Suppressor Protein p53 genetics
Published
2024
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8. Spindle Cell Lesions with Oncogenic EGFR Kinase Domain Aberrations: Expanding the Spectrum of Protein Kinase-Related Mesenchymal Tumors.

Author

Vallese S, Barresi S, Hiemcke-Jiwa L, Patrizi S, Kester L, Giovannoni I, Cardoni A, Pedace L, Nardini C, Tancredi C, Desideri M, von Deimling A, Mura RM, Piga M, Errico ME, Stracuzzi A, Alaggio R, Miele E, and Flucke U

Subjects
Humans, Male, Female, Child, Child, Preschool, Adolescent, Infant, Retrospective Studies, Mutation, Soft Tissue Neoplasms genetics, Soft Tissue Neoplasms pathology, Soft Tissue Neoplasms enzymology, Biomarkers, Tumor genetics, Biomarkers, Tumor analysis, DNA Methylation, Immunohistochemistry, ErbB Receptors genetics
Abstract

EGFR aberrations are reported in a subset of myofibroblastic lesions with kinase domain duplication (EGFR-KDD) and exon 20 mutations being assigned to infantile fibrosarcomas (IFS), mesoblastic nephroma, and fibrous hamartoma of infancy (FHI), respectively. In this retrospective study, we correlated molecular findings with the histomorphology of 14 myofibroblastic lesions harboring such genetic changes identified by NGS. We additionally performed DNA methylation profiling (DNAmp) and immunohistochemistry. Lesions were from 10 males and 4 females with a mean age of 3 years (range, 0.3-14) and occurred subcutaneously in the upper limbs (n = 5), lower limbs (n = 3), back/thorax (n = 5), and the nasal cavity (n = 1). Eleven were cured by surgery, including 1 relapsed case. Two patients were lost to follow-up. One case was very recent, and the patient was biopsied. Histologically, the lesions showed a wide spectrum varying from classic FHI (n = 9) to IFS (n = 1) or lipofibromatosis-like tumors (LFT-like) (n = 2) or dermatofibrosarcoma protuberans-like (DFSP-like) (n = 1) to a predominantly myxoid spindle cell lesion (n = 1). Immunohistochemically, all neoplasms stained with CD34, whereas S100 was positive in 2/14. EGFR expression was observed in 9/10 cases. Molecularly, the IFS and 1 LFT-like harbored EGFR-KDD, whereas an exon 20 mutation was identified in all FHI, 1 LFT-like, the DFSP-like, and in predominant myxoid spindle cell lesion. By DNAmp, all but 2 cases formed a well-defined cluster, demonstrating that these lesions are also epigenetically related. In conclusion, EGFR kinase domain aberrations found in FHI, IFS, LFT-like, DFSP-like, and a spindle cell lesion with a predominant myxoid stroma of children and adolescents showed that these neoplasms with a broad morphologic spectrum belong to the group of protein kinase-related lesions with a distinct epigenetic signature. Molecular analyses, including DNAmp, help to identify and characterize this emerging category and become mandatory when targeted treatment is considered., (Copyright © 2024 United States & Canadian Academy of Pathology. Published by Elsevier Inc. All rights reserved.)

Published
2024
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9. Phase II Study of Allogeneic Hematopoietic Stem Cell Transplantation for Children with High-Risk Neuroblastoma Using a Reduced-Intensity Conditioning Regimen: Results from the AIEOP Trial.

Author

Prete A, Lanino E, Saglio F, Biffi A, Calore E, Faraci M, Rondelli R, Favre C, Zecca M, Casazza G, Porta F, Luksch R, Cesaro S, Rabusin M, Parasole R, Mura RM, Lo Nigro L, Leardini D, Pagliara D, Locatelli F, and Fagioli F

Subjects
Adolescent, Child, Child, Preschool, Female, Humans, Infant, Male, Disease-Free Survival, Prospective Studies, Transplantation, Homologous, Feasibility Studies, Graft vs Host Disease, Hematopoietic Stem Cell Transplantation methods, Neuroblastoma therapy, Transplantation Conditioning methods
Abstract

Despite aggressive multimodal treatment, the outcomes of pediatric patients with high-risk (HR) neuroblastoma (NB) remain poor. The rationale for allogeneic hematopoietic stem cell transplantation (allo-HCT) to treat NB was based on the possible graft-versus-tumor effect; however, toxicity limits its efficacy. We sought to prospectively assess the feasibility and efficacy of allo-HCT using a reduced-intensity conditioning regimen in pediatric patients with HR NB in a multicenter phase II trial. Primary endpoints were the rate of neutrophil and platelet engraftment, 5-year transplantation-related mortality (TRM), and disease-free survival (DFS). Secondary endpoint measures included the incidence of acute graft-versus-host disease (aGVHD) and chronic GVHD. Fifty-one patients were enrolled in the study. The 5-year cumulative incidence (CuI) of TRM was 29.4 ± 6.4%, and that of DFS was 11.8 ± 4.5%. Patients undergoing allo-HCT within 1 year of diagnosis or with bone marrow as their stem cell source had a higher DFS probability. The CuI of neutrophil engraftment, platelet engraftment, and grade II-IV aGVHD was 97.9 ± 2.1%, 93.8 ± 3.5%, and 47.1 ± 7.0%, respectively. The development of new therapeutic strategies could further improve disease control., (Copyright © 2024. Published by Elsevier Inc.)

Published
2024
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10. Brentuximab vedotin in the treatment of paediatric patients with relapsed or refractory Hodgkin's lymphoma: Results of a real-life study.

Author

Massano D, Carraro E, Mussolin L, Buffardi S, Barat V, Zama D, Muggeo P, Vendemini F, Sau A, Moleti ML, Verzegnassi F, D'Amico S, Casini T, Garaventa A, Schiavello E, Cellini M, Vinti L, Farruggia P, Perruccio K, Cesaro S, De Santis R, Marinoni M, D'Alba I, Mura RM, Burnelli R, Mascarin M, and Pillon M

Subjects
Adult, Brentuximab Vedotin, Child, Humans, Neoplasm Recurrence, Local drug therapy, Retrospective Studies, Treatment Outcome, Hodgkin Disease therapy, Immunoconjugates adverse effects
Abstract

Background: Brentuximab vedotin (BV) is an antibody drug-conjugated anti-CD30 approved for the treatment of adult classical Hodgkin's lymphoma (HL), whereas it is considered as off-label indication in paediatrics. The aim of the study was to evaluate the safety and efficacy of BV to treat patients aged less than 18 years with refractory/relapsed HL., Materials and Methods: In this multicentre, retrospective study, 68 paediatric patients who received at least one dose of BV between November 2011 and August 2020 were enrolled. A median of nine doses of BV were administered as monotherapy (n = 31) or combined with other therapies (n = 37). BV was administrated alone as consolidation therapy after stem cell transplantation (SCT) in 12 patients, before SCT in 18 patients, whereas in 15 patients it was used before and after SCT as consolidation therapy. Median follow-up was 2.8 years (range: 0.6-8.9 years)., Results: The best response was observed in the 86% of patients; the overall response rate was 66%. The 3-year progression-free survival was 58%, whereas the overall survival was 75%. No statistically significant differences between patients treated with BV monotherapy or combination were highlighted. In multivariate analysis, patients with non-nodular sclerosis HL and not transplanted had an increased risk of failure. Overall, 46% of patients had grade 3-4 adverse events that led to BV discontinuation in five of them., Conclusion: In conclusion, our study confirms that BV was a safe and effective drug, able to induce complete remission, either as monotherapy or in association with standard therapy., (© 2022 Wiley Periodicals LLC.)

Published
2022
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11. Favorable outcome of SARS-CoV-2 infection in pediatric hematology oncology patients during the second and third pandemic waves in Italy: a multicenter analysis from the Infectious Diseases Working Group of the Associazione Italiana di Ematologia e Oncologia Pediatrica (AIEOP).

Author

Zama D, Baccelli F, Colombini A, Contino A, Calore E, Petris MG, Meneghello L, Mercolini F, Lo Vecchio A, Montalto S, Meazza C, Barone A, Compagno F, Muggeo P, Soncini E, Brescia L, Giraldi E, Giurici N, Mura RM, Cellini M, Perruccio K, Petroni V, La Spina M, Ziino O, Burnelli R, De Santis R, Mascarin M, Barretta V, Tridello G, and Cesaro S

Subjects
Child, Humans, Italy epidemiology, Pandemics, RNA, Viral, SARS-CoV-2, COVID-19 epidemiology, Communicable Diseases epidemiology, Hematology, Neoplasms epidemiology
Abstract

COVID-19 has a mild clinical course with low mortality rate in general pediatric population, while variable outcomes have been described in children with cancer. Infectious diseases working party of the AIEOP collected data on the clinical characteristics and outcomes of SARS-CoV-2 infections in pediatric oncology/hematology patients from April 2020 to May 2021, including the second and the third waves of the pandemic in Italy. Factors potentially associated with moderate, severe, or critical COVID-19 were analyzed. Of the 153 SARS-Cov2 infections recorded, 100 were asymptomatic and 53 symptomatic. The course of COVID-19 was mild in 41, moderate in 2, severe in 5, and critical in 5 children. A total of 40.5% of patients were hospitalized, ten requiring oxygen support and 5 admitted to the intensive care unit. Antibiotics and steroids were the most used therapies. No patient died due to SARS-CoV-2 infection. Infections occurring early (< 60 days) after the diagnosis of the underlying disease or after SCT were associated to moderate, severe, and critical disease compared to infections occurring late (> 60 days) or during maintenance therapy. In the patients on active chemotherapy, 59% withdrew the treatment for a median of 15 days. SARS-CoV-2 presented a favorable outcome in children with cancer in Italy during the pandemic. Modification of therapy represents a major concern in this population. Our findings suggest considering regular chemotherapy continuation, particularly in patients on maintenance therapy or infected late after the diagnosis., (© 2022. The Author(s).)

Published
2022
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12. Outcome of Children and Adolescents with Recurrent Classical Hodgkin Lymphoma: The Italian Experience.

Author

Garaventa A, Parodi S, Guerrini G, Farruggia P, Sala A, Pillon M, Buffardi S, Rossi F, Bianchi M, Zecca M, Vinti L, Facchini E, Casini T, Bernasconi S, Amoroso L, D'Amico S, Provenzi M, De Santis R, Sau A, Muggeo P, Mura RM, Haupt R, Mascarin M, and Burnelli R

Abstract

The objective of this study was to identify prognostic factors for children and adolescents with relapsed or progressive classical Hodgkin’s lymphoma (cHL) to design salvage therapy tailored to them. We analyzed a homogeneous pediatric population, diagnosed with progressive/relapsed cHL previously enrolled in two subsequent protocols of the Italian Association of Pediatric Hematology and Oncology in the period 1996−2016. There were 272 eligible patients, 17.5% of treated patients with cHL. Overall survival (OS) and event-free survival (EFS) after a 10-year follow-up were 65.3% and 53.3%, respectively. Patients with progressive disease (PD), advanced stage at recurrence, and ≥5 involved sites showed a significantly worse OS. PD, advanced stage, and extra-nodal involvement at recurrence were significantly associated with a poorer EFS. Multivariable analysis identified three categories for OS based on the type of recurrence and number of localizations: PD and ≥5 sites: OS 34%; PD and <5 sites: OS 56.5%; relapses: OS 73.6%. Four categories were obtained for EFS based on the type of recurrence and stage: PD and stage 3−4: EFS 25.5%; PD and stage 1−2: EFS 43%; relapse and stage 3−4: EFS 55.4%; relapse and stage 1−2: EFS 72.1%. Patients with PD, in advanced stage, or with ≥5 involved sites had a very poor survival and they should be considered refractory to first- and second-line standard chemotherapy. Probably, they should be considered for more innovative approaches since the first progression. Conversely, patients who relapsed later with localized disease had a better prognosis, and they could be considered for a conservative approach.

Published
2022
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13. Treatment and outcome of intracranial ependymoma after first relapse in the 2nd AIEOP protocol.

Author

Massimino M, Barretta F, Modena P, Johann P, Ferroli P, Antonelli M, Gandola L, Garrè ML, Bertin D, Mastronuzzi A, Mascarin M, Quaglietta L, Viscardi E, Sardi I, Ruggiero A, Boschetti L, Giagnacovo M, Biassoni V, Schiavello E, Chiapparini L, Erbetta A, Mussano A, Giussani C, Mura RM, Barra S, Scarzello G, Scimone G, Carai A, Giangaspero F, and Buttarelli FR

Subjects
Child, Preschool, Female, Humans, Neoplasm Recurrence, Local therapy, Prognosis, Treatment Outcome, Brain Neoplasms pathology, Ependymoma pathology
Abstract

Background: More than 40% of patients with intracranial ependymoma need a salvage treatment within 5 years after diagnosis, and no standard treatment is available as yet. We report the outcome after first relapse of 64 patients treated within the 2nd AIEOP protocol., Methods: We considered relapse sites and treatments, that is, various combinations of complete/incomplete surgery, if followed by standard or hypofractionated radiotherapy (RT) ± chemotherapy (CT). Molecular analyses were available for 38/64 samples obtained at first diagnosis. Of the 64 cases, 55 were suitable for subsequent analyses., Results: The median follow-up was 147 months after diagnosis, 84 months after first relapse, 5-year EFS/OS were 26.2%/30.8% (median EFS/OS 13/32 months) after relapse. For patients with a local relapse (LR), the 5-year cumulative incidence of second LRs was 51.6%, with a 5-year event-specific probability of being LR-free of 40.0%. Tumor site/grade, need for shunting, age above/below 3 years, molecular subgroup at diagnosis, had no influence on outcomes. Due to variation in the RT dose/fractionation used and the subgroup sizes, it was not possible to assess the impact of the different RT modalities. Multivariable analyses identified completion of surgery, the absence of symptoms at relapse, and female sex as prognostically favorable. Tumors with a 1q gain carried a higher cumulative incidence of dissemination after first relapse., Conclusions: Survival after recurrence was significantly influenced by symptoms and completeness of surgery. Only a homogeneous protocol with well-posed, randomized questions could clarify the numerous issues, orient salvage treatment, and ameliorate prognosis for this group of patients., (© The Author(s) 2021. Published by Oxford University Press on behalf of the Society for Neuro-Oncology. All rights reserved. For permissions, please e-mail: journals.permissions@oup.com.)

Published
2022
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14. Human Fibrinogen Concentrate and Fresh Frozen Plasma in the Management of Severe Acquired Hypofibrinogenemia in Children With Acute Lymphoblastic Leukemia: Results of a Retrospective Survey.

Author

Giordano P, Grassi M, Saracco P, Luciani M, Colombini A, Testi AM, Micalizzi C, Petruzziello F, Putti MC, Casale F, Consarino C, Mura RM, Mastrodicasa E, Notarangelo LD, Onofrillo D, Pollio B, Rizzari C, Tafuri S, De Leonardis F, Corallo PC, and Santoro N

Subjects
Adolescent, Afibrinogenemia chemically induced, Child, Child, Preschool, Female, Humans, Male, Retrospective Studies, Afibrinogenemia drug therapy, Fibrinogen therapeutic use, Plasma, Precursor Cell Lymphoblastic Leukemia-Lymphoma complications
Abstract

Objective of the Study: In this study we aimed to retrospectively evaluate how centers, belonging to the Associazione Italiana Ematologia e Oncologia Pediatrica (AIEOP), manage severe acquired hypofibrinogenemia in children with acute lymphoblastic leukemia, particularly evaluating the therapeutic role of human fibrinogen concentrate (HFC) and fresh frozen plasma (FFP)., Methods: We conducted a survey among AIEOP centers; thereafter, we collected and analyzed data with regard to the treatment of episodes of severe acquired hypofibrinogenemia occurring during the induction and reinduction phases of the AIEOP-BFM ALL 2009 protocol., Results: In total, 15 of the 37 AIEOP centers invited to join the survey agreed to collect the data, with 10 and 5 centers declaring to react to severe acquired hypofibrinogenemia (<70 mg/dL) by administering HFC or FFP, respectively. Of the 150 episodes of severe hypofibrinogenemia occurring in 101 patients, 47.3% were treated with HFC and 52.7% with FFP, with a normalization of fibrinogen levels achieved in greater proportion and in a shorter amount of time in the HFC group as compared with the FFP group. None of the patients presented with bleeding or thrombosis during the observation period., Conclusions: Even with the limitations of the retrospective nature of this study, HFC seems to be a safe and effective alternative to FFP for replacement therapy in case of severe hypofibrinogenemia in children with acute lymphoblastic leukemia.

Published
2019
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15. Topotecan/carboplatin regimen for refractory/recurrent rhabdomyosarcoma in children: Report from the AIEOP Soft Tissue Sarcoma Committee.

Author

Compostella A, Affinita MC, Casanova M, Milano GM, Scagnellato A, Dall'Igna P, Chiaravalli S, Pierobon M, Manzitti C, Zanetti I, Schiavetti A, Sorbara S, Mura RM, Ruggiero A, Ferrari A, and Bisogno G

Subjects
Adolescent, Child, Child, Preschool, Drug Resistance, Neoplasm drug effects, Female, Humans, Infant, Kaplan-Meier Estimate, Male, Neoplasm Recurrence, Local drug therapy, Rhabdomyosarcoma mortality, Young Adult, Antineoplastic Combined Chemotherapy Protocols therapeutic use, Carboplatin administration & dosage, Rhabdomyosarcoma drug therapy, Topotecan administration & dosage
Abstract

Introduction: From 2002 to 2011, the Italian Soft Tissue Sarcoma Committee explored a combination of topotecan and carboplatin as a second-line strategy for children with resistant or relapsing rhabdomyosarcoma., Methods: Patients received two blocks of topotecan 2 mg/m 2 on days 1, 2, and 3, and carboplatin 250 mg/m 2 on days 4 and 5, followed by alternating blocks of topotecan-cyclophosphamide and carboplatin-etoposide for a total of six courses with 3-week intervals. Tumor response was assessed after two cycles, and local control was implemented when feasible., Results: A total of 38 patients were included in this study: 18/38 had alveolar rhabdomyosarcoma (RMS), 10/38 had metastatic disease at diagnosis, 8/38 had tumor progression during first-line chemotherapy, 21/38 had locoregional relapses, and 9/38 had distant relapses. Thirty-two patients could be assessed for tumor response to topotecan-carboplatin, and 9 (28%) showed a complete or partial response. Twenty-four patients experienced grade IV hematologic toxicity, while transient grade 1 tubulopathy, grade 3 mucositis, transient grade 2 nephrotoxicity, and a grade 2 decline in cardiac function occurred in one patient each. The 5-year overall and progression-free survival rates were 17% and 14%, respectively., Conclusion: the prognosis for children with resistant or relapsing RMS remains unsatisfactory. The topotecan-carboplatin regimen was well-tolerated. Though in case of late relapse the response rate was similar to those reported for other regimes, the result achieved remains unsatisfactory. New approaches, possibly including target agents, seem more attractive for future studies.

Published
2019
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16. A survey on hematology-oncology pediatric AIEOP centres: The challenge of posterior reversible encephalopathy syndrome.

Author

Zama D, Gasperini P, Berger M, Petris M, De Pasquale MD, Cesaro S, Guerzoni ME, Mastrodicasa E, Savina F, Ziino O, Kiren V, Muggeo P, Mura RM, Melchionda F, and Zanazzo GA

Subjects
Adolescent, Child, Child, Preschool, Diagnostic Imaging, Disease Management, Female, Health Surveys, Humans, Incidence, Infant, Italy epidemiology, Male, Outcome Assessment, Health Care, Posterior Leukoencephalopathy Syndrome diagnosis, Posterior Leukoencephalopathy Syndrome etiology, Posterior Leukoencephalopathy Syndrome therapy, Prevalence, Risk Factors, Symptom Assessment, Posterior Leukoencephalopathy Syndrome epidemiology
Abstract

Objectives: Posterior reversible encephalopathy syndrome (PRES) is one of the most common neurological complications in hematology-oncology pediatric patients. Despite an increasingly recognized occurrence, no clear consensus exists regarding how best to manage the syndrome, because most cases of PRES have reported in single-case reports or small series. Aim of this paper is to identify incidence, clinical features, management, and outcome of PRES in a large series of hematology-oncology pediatric patients., Methods: The cases of PRES occurred in twelve centers of the Italian Association of Pediatric Hematology and Oncology were reported., Results: One hundred and twenty-four cases of PRES in 112 pediatric patients were recorded with an incidence of 2.1% and 4.7%, respectively, in acute lymphoblastic leukemia in first complete remission and hematopoietic stem cell transplantation (HSCT). The majority of cases occurred after a cycle of chemotherapy rather than after stem cell transplant. PRES after chemotherapy significantly differs from that after HSCT for diagnosis, time of presentation, risk factors, management, and outcome., Conclusions: This study demonstrates that PRES is a common neurological complication and occurring preferentially in course of induction treatment of some hematologic malignancies, as ALL and after HSCT. It also highlights great clinical differences in the management and outcome in patients with PRES occurring after chemotherapy or after HSCT., (© 2017 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.)

Published
2018
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17. High serum sclerostin levels in children with haemophilia A.

Author

Giordano P, Brunetti G, Lassandro G, Notarangelo LD, Luciani M, Mura RM, Lazzareschi I, Santagostino E, Piacente L, Ventura A, Cavallo L, Grano M, and Faienza MF

Subjects
Adaptor Proteins, Signal Transducing, Adolescent, Bone Density physiology, Case-Control Studies, Child, Child, Preschool, Genetic Markers, Humans, Lumbar Vertebrae physiopathology, Male, Bone Morphogenetic Proteins blood, Hemophilia A blood
Published
2016
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18. Bloodstream infections in children with cancer: a multicentre surveillance study of the Italian Association of Paediatric Haematology and Oncology. Supportive Therapy Group-Infectious Diseases Section.

Author

Viscoli C, Castagnola E, Giacchino M, Cesáro S, Properzi E, Tucci F, Mura RM, Alvisi P, Zanazzo G, Surico G, Bonetti F, De Sio L, Izzi G, Di Cataldo A, Ziino O, Massolo F, Nardi M, Santoro N, and Binda S

Subjects
Bacteremia drug therapy, Bacteremia mortality, Child, Drug Resistance, Microbial, Female, Fungemia drug therapy, Fungemia mortality, Humans, Italy epidemiology, Male, Neoplasms mortality, Neoplasms therapy, Neutropenia complications, Neutropenia mortality, Prospective Studies, Bacteremia microbiology, Fungemia microbiology, Neoplasms complications
Abstract

A one-year prospective, multicentre surveillance study on aetiology, main clinical features and outcome of bloodstream infections in children with cancer was conducted in 18 paediatric haematology centres belonging to the Italian Association for Paediatric Haematology and Oncology. A total of 191 bloodstream infections were reported during the study period. Of them, 123 (64%) occurred in neutropenic and 68 (36%) in non-neutropenic patients. Gram-positive cocci caused 45% (85/191) of the episodes, gram-negative rods 41% (78/191), and fungi 9% (18/191). The remaining 5% (10/191) of the episodes were poly-microbial infections. A total of 204 pathogens were isolated (46% gram-positive cocci; 44% gram-negative rods; and 10% fungi). The aetiologic distribution was similar among neutropenic and non-neutropenic patients. A correlation between the infection and the presence of an indwelling central venous catheter was found in 20% (23/114) of the episodes among neutropenic patients and in 55% (23/62) among non-neutropenic patients. Gram-negative micro-organisms were isolated in an unusually high proportion of catheter-related infections (48%). The overall mortality rate from any cause within 30 days from the first positive blood culture was 11%, and was higher among patients who were neutropenic at the onset of the infection than among those who were not neutropenic (15 versus 4%, P = 0.03). In addition, the mortality was significantly higher in recipients of bone marrow transplantation than in patients with acute leukaemia or solid tumour (21, 11 and 6%, respectively) and was also higher in fungaemias and poly-microbial infections (22 and 30%) than in single gram-positive and gram-negative bacteraemias (11 and 6%).

Published
1999
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19. [Von Willebrand's disease].

Author

Alberti M, Fiori GM, Mura RM, Targhetta R, and Biddau P

Subjects
Child, Female, Humans, Male, Pregnancy, Prenatal Diagnosis, von Willebrand Diseases genetics, von Willebrand Diseases therapy, von Willebrand Diseases diagnosis
Published
1992

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