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1. HLA-haploidentical hematopoietic stem cells transplantation with regulatory and conventional T-cell adoptive immunotherapy in pediatric patients with very high-risk acute leukemia

Author

Massei, M. S., Capolsini, I., Mastrodicasa, E., Perruccio, K., Arcioni, F., Cerri, C., Gurdo, G., Sciabolacci, S., Falzetti, F., Zei, T., Iacucci Ostini, R., Brogna, M., Panizza, B. M., Saldi, S., Merluzzi, M., Tognellini, R., Marchesi, M., Minelli, O., Aristei, C., Velardi, A., Pierini, A., Ruggeri, L., Martelli, M. F., Carotti, A., and Caniglia, M.

Published
2023
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2. Musculoskeletal manifestations of childhood cancer and differential diagnosis with juvenile idiopathic arthritis (ONCOREUM): a multicentre, cross-sectional study

Author

Amatruda, M, Atzeni, C, Bertolini, P, Bigucci, B, Caniglia, M, Cappella, M, Cattalini, M, Cefalo, MG, Cellini, M, Cortis, E, Davì, S, De Benedetti, F, Di Cataldo, A, Fabbri, E, Fagioli, F, Fontanili, I, Garaventa, A, Gicchino, MF, Ladogana, S, Locatelli, F, Magnolato, A, Marsili, M, Martino, S, Mascarin, M, Messina, C, Micalizzi, C, Porta, F, Rizzari, C, Civino, Adele, Alighieri, Giovanni, Prete, Eleonora, Caroleo, Anna Maria, Magni-Manzoni, Silvia, Vinti, Luciana, Romano, Micol, Santoro, Nicola, Filocamo, Giovanni, Belotti, Tamara, Santarelli, Francesca, Gorio, Chiara, Ricci, Francesca, Colombini, Antonella, Pastore, Serena, Cesaro, Simone, Barone, Patrizia, Verzegnassi, Federico, Olivieri, Alma Nunzia, Ficara, Monica, Miniaci, Angela, Russo, Giovanna, Gallizzi, Romina, Pericoli, Roberta, Breda, Luciana, Mura, Rossella, Podda, Rosa Anna, Onofrillo, Daniela, Lattanzi, Bianca, Tirtei, Elisa, Maggio, Maria Cristina, De Santis, Raffaela, Consolini, Rita, Arlotta, Annalisa, La Torre, Francesco, Mainardi, Chiara, Pelagatti, Maria Antonietta, Coassin, Elisa, Capolsini, Ilaria, Burnelli, Roberta, Tornesello, Assunta, Soscia, Francesca, De Fanti, Alessandro, Rigante, Donato, Pizzato, Cristina, De Fusco, Carmela, Abate, Massimo Eraldo, Roncadori, Andrea, Rossi, Elisa, Stabile, Giulia, Biondi, Andrea, Lepore, Loredana, Conter, Valentino, Rondelli, Roberto, Pession, Andrea, and Ravelli, Angelo

Published
2021
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3. GAU-PED study for early diagnosis of Gaucher disease in children with splenomegaly and cytopenia

Author

Pession, A., Di Rocco, M., Venturelli, F., Tappino, B., Morello, W., Santoro, N., Giordano, P., Filippini, B., Rinieri, S., Russo, G., Girardi, K., Ruggiero, A., Galea, E., Antonucci, R., Tovaglieri, N., Porta, F., Tartaglione, I., Giona, F., Fagioli, F., Burlina, A., Mura, R., Russo, B., Tornesello, A., Menna, G., Russo, D., Caniglia, M., Schettini, S., Onofrillo, D., Ladogana, S., and Civino, A.

Subjects
Cytopenia, Splenomegaly, Lysosomal storage disease, Gaucher disease, Thrombocytopenia
Published
2023

4. Musculoskeletal manifestations of childhood cancer and differential diagnosis with juvenile idiopathic arthritis (ONCOREUM): a multicentre, cross-sectional study

Author

Civino, A, Alighieri, G, Prete, E, Caroleo, A, Magni-Manzoni, S, Vinti, L, Romano, M, Santoro, N, Filocamo, G, Belotti, T, Santarelli, F, Gorio, C, Ricci, F, Colombini, A, Pastore, S, Cesaro, S, Barone, P, Verzegnassi, F, Olivieri, A, Ficara, M, Miniaci, A, Russo, G, Gallizzi, R, Pericoli, R, Breda, L, Mura, R, Podda, R, Onofrillo, D, Lattanzi, B, Tirtei, E, Maggio, M, De Santis, R, Consolini, R, Arlotta, A, La Torre, F, Mainardi, C, Pelagatti, M, Coassin, E, Capolsini, I, Burnelli, R, Tornesello, A, Soscia, F, De Fanti, A, Rigante, D, Pizzato, C, De Fusco, C, Abate, M, Roncadori, A, Rossi, E, Stabile, G, Biondi, A, Lepore, L, Conter, V, Rondelli, R, Pession, A, Ravelli, A, Amatruda, M, Atzeni, C, Bertolini, P, Bigucci, B, Caniglia, M, Cappella, M, Cattalini, M, Cefalo, M, Cellini, M, Cortis, E, Davi, S, De Benedetti, F, Di Cataldo, A, Fabbri, E, Fagioli, F, Fontanili, I, Garaventa, A, Gicchino, M, Ladogana, S, Locatelli, F, Magnolato, A, Marsili, M, Martino, S, Mascarin, M, Messina, C, Micalizzi, C, Porta, F, Rizzari, C, Civino A., Alighieri G., Prete E., Caroleo A. M., Magni-Manzoni S., Vinti L., Romano M., Santoro N., Filocamo G., Belotti T., Santarelli F., Gorio C., Ricci F., Colombini A., Pastore S., Cesaro S., Barone P., Verzegnassi F., Olivieri A. N., Ficara M., Miniaci A., Russo G., Gallizzi R., Pericoli R., Breda L., Mura R., Podda R. A., Onofrillo D., Lattanzi B., Tirtei E., Maggio M. C., De Santis R., Consolini R., Arlotta A., La Torre F., Mainardi C., Pelagatti M. A., Coassin E., Capolsini I., Burnelli R., Tornesello A., Soscia F., De Fanti A., Rigante D., Pizzato C., De Fusco C., Abate M. E., Roncadori A., Rossi E., Stabile G., Biondi A., Lepore L., Conter V., Rondelli R., Pession A., Ravelli A., Amatruda M., Atzeni C., Bertolini P., Bigucci B., Caniglia M., Cappella M., Cattalini M., Cefalo M. G., Cellini M., Cortis E., Davi S., De Benedetti F., Di Cataldo A., Fabbri E., Fagioli F., Fontanili I., Garaventa A., Gicchino M. F., Ladogana S., Locatelli F., Magnolato A., Marsili M., Martino S., Mascarin M., Messina C., Micalizzi C., Porta F., Rizzari C., Civino, A, Alighieri, G, Prete, E, Caroleo, A, Magni-Manzoni, S, Vinti, L, Romano, M, Santoro, N, Filocamo, G, Belotti, T, Santarelli, F, Gorio, C, Ricci, F, Colombini, A, Pastore, S, Cesaro, S, Barone, P, Verzegnassi, F, Olivieri, A, Ficara, M, Miniaci, A, Russo, G, Gallizzi, R, Pericoli, R, Breda, L, Mura, R, Podda, R, Onofrillo, D, Lattanzi, B, Tirtei, E, Maggio, M, De Santis, R, Consolini, R, Arlotta, A, La Torre, F, Mainardi, C, Pelagatti, M, Coassin, E, Capolsini, I, Burnelli, R, Tornesello, A, Soscia, F, De Fanti, A, Rigante, D, Pizzato, C, De Fusco, C, Abate, M, Roncadori, A, Rossi, E, Stabile, G, Biondi, A, Lepore, L, Conter, V, Rondelli, R, Pession, A, Ravelli, A, Amatruda, M, Atzeni, C, Bertolini, P, Bigucci, B, Caniglia, M, Cappella, M, Cattalini, M, Cefalo, M, Cellini, M, Cortis, E, Davi, S, De Benedetti, F, Di Cataldo, A, Fabbri, E, Fagioli, F, Fontanili, I, Garaventa, A, Gicchino, M, Ladogana, S, Locatelli, F, Magnolato, A, Marsili, M, Martino, S, Mascarin, M, Messina, C, Micalizzi, C, Porta, F, Rizzari, C, Civino A., Alighieri G., Prete E., Caroleo A. M., Magni-Manzoni S., Vinti L., Romano M., Santoro N., Filocamo G., Belotti T., Santarelli F., Gorio C., Ricci F., Colombini A., Pastore S., Cesaro S., Barone P., Verzegnassi F., Olivieri A. N., Ficara M., Miniaci A., Russo G., Gallizzi R., Pericoli R., Breda L., Mura R., Podda R. A., Onofrillo D., Lattanzi B., Tirtei E., Maggio M. C., De Santis R., Consolini R., Arlotta A., La Torre F., Mainardi C., Pelagatti M. A., Coassin E., Capolsini I., Burnelli R., Tornesello A., Soscia F., De Fanti A., Rigante D., Pizzato C., De Fusco C., Abate M. E., Roncadori A., Rossi E., Stabile G., Biondi A., Lepore L., Conter V., Rondelli R., Pession A., Ravelli A., Amatruda M., Atzeni C., Bertolini P., Bigucci B., Caniglia M., Cappella M., Cattalini M., Cefalo M. G., Cellini M., Cortis E., Davi S., De Benedetti F., Di Cataldo A., Fabbri E., Fagioli F., Fontanili I., Garaventa A., Gicchino M. F., Ladogana S., Locatelli F., Magnolato A., Marsili M., Martino S., Mascarin M., Messina C., Micalizzi C., Porta F., and Rizzari C.

Abstract

Background: Presenting symptoms of childhood cancers might mimic those of rheumatic diseases. However, the evidence available to guide differential diagnosis remains scarce. Preventing wrong or delayed diagnosis is therefore important to avoid incorrect administration of glucocorticoid or immunosuppressive therapy and worsening of prognosis. As such, we aimed to assess the prevalence and characteristics of presenting musculoskeletal manifestations in patients at cancer onset and to identify the factors that differentiate childhood malignancies with arthropathy from juvenile idiopathic arthritis. Methods: We did a multicentre, cross-sectional study at 25 paediatric haemato-oncology centres and 22 paediatric rheumatology centres in Italy. We prospectively recruited patients who were younger than 16 years that were newly diagnosed with cancer or juvenile idiopathic arthritis. We excluded patients with glucocorticoid pre-treatment (>1 mg/kg per day of oral prednisone or equivalent for ≥2 consecutive weeks). We collected data for patients with a new diagnosis of cancer or juvenile idiopathic arthritis using an electronic case report form on a web-based platform powered by the Cineca Interuniversity Consortium. The primary outcome was to describe the frequency and characteristics of musculoskeletal manifestations at cancer onset; and the secondary outcome was to identify factors that could discriminate malignancies presenting with arthropathy, with or without other musculoskeletal symptoms, from juvenile idiopathic arthritis using multivariable logistic regression analysis. Findings: Between May 1, 2015, and May 31, 2018, 1957 patients were eligible, of which 1277 (65%) had cancer and 680 (35%) had juvenile idiopathic arthritis. Musculoskeletal symptoms occurred in 324 (25% [95% CI 23·0–27·8]) of 1277 patients with cancer, of whom 207 had arthropathy. Patients with malignant bone tumours had the highest frequency of musculoskeletal symptoms (53 [80%] of 66), followed b

Published
2021

5. Musculoskeletal manifestations of childhood cancer and differential diagnosis with juvenile idiopathic arthritis (ONCOREUM): a multicentre, cross-sectional study

Author

Civino, Adele, primary, Alighieri, Giovanni, additional, Prete, Eleonora, additional, Caroleo, Anna Maria, additional, Magni-Manzoni, Silvia, additional, Vinti, Luciana, additional, Romano, Micol, additional, Santoro, Nicola, additional, Filocamo, Giovanni, additional, Belotti, Tamara, additional, Santarelli, Francesca, additional, Gorio, Chiara, additional, Ricci, Francesca, additional, Colombini, Antonella, additional, Pastore, Serena, additional, Cesaro, Simone, additional, Barone, Patrizia, additional, Verzegnassi, Federico, additional, Olivieri, Alma Nunzia, additional, Ficara, Monica, additional, Miniaci, Angela, additional, Russo, Giovanna, additional, Gallizzi, Romina, additional, Pericoli, Roberta, additional, Breda, Luciana, additional, Mura, Rossella, additional, Podda, Rosa Anna, additional, Onofrillo, Daniela, additional, Lattanzi, Bianca, additional, Tirtei, Elisa, additional, Maggio, Maria Cristina, additional, De Santis, Raffaela, additional, Consolini, Rita, additional, Arlotta, Annalisa, additional, La Torre, Francesco, additional, Mainardi, Chiara, additional, Pelagatti, Maria Antonietta, additional, Coassin, Elisa, additional, Capolsini, Ilaria, additional, Burnelli, Roberta, additional, Tornesello, Assunta, additional, Soscia, Francesca, additional, De Fanti, Alessandro, additional, Rigante, Donato, additional, Pizzato, Cristina, additional, De Fusco, Carmela, additional, Abate, Massimo Eraldo, additional, Roncadori, Andrea, additional, Rossi, Elisa, additional, Stabile, Giulia, additional, Biondi, Andrea, additional, Lepore, Loredana, additional, Conter, Valentino, additional, Rondelli, Roberto, additional, Pession, Andrea, additional, Ravelli, Angelo, additional, Amatruda, M, additional, Atzeni, C, additional, Bertolini, P, additional, Bigucci, B, additional, Caniglia, M, additional, Cappella, M, additional, Cattalini, M, additional, Cefalo, MG, additional, Cellini, M, additional, Cortis, E, additional, Davì, S, additional, De Benedetti, F, additional, Di Cataldo, A, additional, Fabbri, E, additional, Fagioli, F, additional, Fontanili, I, additional, Garaventa, A, additional, Gicchino, MF, additional, Ladogana, S, additional, Locatelli, F, additional, Magnolato, A, additional, Marsili, M, additional, Martino, S, additional, Mascarin, M, additional, Messina, C, additional, Micalizzi, C, additional, Porta, F, additional, and Rizzari, C, additional

Published
2021
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6. Safety and efficacy of brincidofovir for Adenovirus infection in children receiving allogeneic stem cell transplantation: an AIEOP retrospective analyses

Author

Perruccio, K., Menconi, M., Galaverna, F., Pagliara, D., Carraro, F., Fagioli, F., Calore, E., Biffi, A., Baretta, V., Massei, M. S., Capolsini, I., Faraci, M., Verna, M., Soncini, E., Caniglia, M., Locatelli, Franco, and Cesaro, S.

Subjects
Oncology, medicine.medical_specialty, allogeneic stem cell transpantaiton, Adenoviridae Infections, MEDLINE, Organophosphonates, Brincidofovir, allogeneic stem cell transpantaiton, adenovirus infection, Antiviral Agents, ADENOVIRUS, adenovirus infection, Cytosine, Internal medicine, medicine, Humans, Adenovirus infection, Retrospective Studies, Transplantation, business.industry, Hematopoietic Stem Cell Transplantation, Hematology, medicine.disease, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, HSCT, Stem cell, brincidofovir, business, medicine.drug
Published
2021

8. Normative growth charts for Shwachman-Diamond syndrome from Italian cohort of 0-8 years old

Author

Cipolli, Marco, Tridello, Gloria, Micheletto, Alessio, Perobelli, Sandra, Pintani, Emily, Cesaro, Simone, Maserati, Emanuela, Nicolis, Elena, Danesino, Cesare, Ambroni, M, Caniglia, M, Cantarini, Me, Corti, P, Farrugia, P, Frau, Mr, Fuoti, M, Indolfi, G, Ladogana, S, Lucidi, V, Rosaria Matarese SM, Menna, G, Montemitro, E, Nardi, M, Nasi, C, Nocerino, A, Pericoli, R, Raia, V, Ramenghi, U, Sainati, L, Tucci, F, Verzegnassi, F, Zecca, M, Zucchini, A., Cipolli, M, Tridello, G, Micheletto, A, Perobelli, S, Pintani, E, Cesaro, S, Maserati, E, Nicoli, Massimiliano, Danesino CAmbroni, M, Caniglia, M, Cantarini, Me, Corti, P, Farrugia, P, Frau, Mr, Fuoti, M, Indolfi, G, Ladogana, S, Lucidi, V, Rosaria Matarese, Sm, Menna, G, Montemitro, E, Nardi, M, Nasi, C, Nocerino, A, Pericoli, MARIO ANDREA, Raia, V, Ramenghi, U, Sainati, L, Tucci, F, Verzegnassi, F, Zecca, M, and Zucchini, A.

Subjects
Male, Percentile, Pediatrics, medicine.medical_specialty, Birth weight, Population, Shwachman-diamond syndrome, Shwachman–diamond syndrome, genetics, growth charts, Short stature, Body Mass Index, Medicine (all), 03 medical and health sciences, 0302 clinical medicine, Reference Values, 030225 pediatrics, medicine, Humans, Registries, Child, education, Retrospective Studies, 030304 developmental biology, 0303 health sciences, education.field_of_study, business.industry, Research, Body Weight, Infant, Newborn, Infant, Genetics and Genomics, Retrospective cohort study, General Medicine, Body Height, Italy, Child, Preschool, Mutation, Failure to thrive, Cohort, Female, medicine.symptom, business, Body mass index
Abstract

ObjectivesShwachman-Diamond syndrome (SDS) is a rare autosomal recessive disorder. Its predominant manifestations include exocrine pancreatic insufficiency, bone marrow failure and skeletal abnormalities. Patients frequently present failure to thrive and susceptibility to short stature. Average birth weight is at the 25th percentile; by the first birthday, >50% of patients drop below the third percentile for height and weight.The study aims at estimating the growth charts for patients affected by SDS in order to give a reference tool helpful for medical care and growth surveillance through the first 8 years of patient’s life.Setting and participantsThis retrospective observational study includes 106 patients (64 M) with available information from birth to 8 years, selected among the 122 patients included in the Italian National Registry of SDS and born between 1975 and 2016. Gender, birth date and auxological parameters at repeated assessment times were collected. The General Additive Model for Location Scale and Shape method was applied to build the growth charts. A set of different distributions was used, and the more appropriate were selected in accordance with the smallest Akaike information criterion.ResultsA total of 408 measurements was collected and analysed. The median number of observations per patient amounted to 3, range 1–11. In accordance with the methods described, specific SDS growth charts were built for weight, height and body mass index (BMI), separately for boys and girls.The 50th and 3rd percentiles of weight and height of the healthy population (WHO standard references) respectively correspond to the 97th and 50th percentiles of the SDS population (SDS specific growth charts), while the difference is less evident for the BMI.ConclusionsSpecific SDS growth charts obtained through our analysis enable a more appropriate classification of patients based on auxological parameters, representing a useful reference tool for evaluating their growth during childhood.

Published
2019

12. Rome Transplant Network, a metropolitan haematopoietic stem cell transplant programme: P824

Author

Arcese, W, Picardi, A., Mauroni, M. R., Adorno, G., Lanti, A., Locatelli, F., Caniglia, M., Ciscato, C., Isacchi, G., Zinno, F., De Fabritiis, P., Dentamaro, T., Delogu, S., Avvisati, G., Tirindelli, M. C., Mazzaroni, M., Petti, C., Mengarelli, A., Pignatelli, A., Monarca, B., Montefusco, E., Grigora, D., Annino, L., Chierichini, A., Pilozzi, V., Angelini, S., Mangione, I., Papa, C., and Miccichè, S.

Published
2011

17. Incidence and risk factors for secondary autoimmune diseases after cord blood transplantation. Retrospective analysis on behalf of Eurocord and the EBMT Working Party Autoimmune Diseases: O179

Author

Daikeler, T., Labopin, M., Socie, G., Faraci, M., Abdel-Rahman, F., Yesilipek, A., Arcese, W., Caniglia, M., OʼMeara, A., Sirvent, A., Voswinkel, J., Verdeguer, A., Gandemer, V., Schoenmans, H., Cant, A., Messina, C., Sedlacek, P., Vora, A., Mialou, V., Martín, Díez J.L., Sora, F., Abunin, M., Herr, A., Crotta, A., Gluckman, E., Farge-Bancel, D., and Rocha, V.

Published
2011

30. Hematopoietic stem cell transplantation for isolated extramedullary relapse of acute lymphoblastic leukemia in children

Author

Gabelli, M., Zecca, M., Messina, C., Carraro, E., Buldini, B., Rovelli, A. M., Fagioli, F., Bertaina, A., Lanino, E., Favre, C., Rabusin, M., Prete, A., Ripaldi, M., Barberi, W., Porta, F., Caniglia, M., Santarone, S., D'Angelo, P., Basso, G., Locatelli, Franco, Locatelli F. (ORCID:0000-0002-7976-3654), Gabelli, M., Zecca, M., Messina, C., Carraro, E., Buldini, B., Rovelli, A. M., Fagioli, F., Bertaina, A., Lanino, E., Favre, C., Rabusin, M., Prete, A., Ripaldi, M., Barberi, W., Porta, F., Caniglia, M., Santarone, S., D'Angelo, P., Basso, G., Locatelli, Franco, and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

Relapse of acute lymphoblastic leukemia (ALL) may occur in extramedullary sites, mainly central nervous system (CNS) and testis. Optimal post-remissional treatment for isolated extramedullary relapse (IEMR) is still controversial. We collected data of children treated with hematopoietic stem cell transplantation (HSCT) for ALL IEMR from 1990 to 2015 in Italy. Among 281 patients, 167 had a relapse confined to CNS, 73 to testis, 14 to mediastinum, and 27 to other organs. Ninety-seven patients underwent autologous HSCT, 79 received allogeneic HSCT from a matched family donor, 75 from a matched unrelated donor, and 30 from an HLA-haploidentical donor. The 10-year overall survival was 56% and was not influenced by gender, ALL blast immune-phenotype, age, site of relapse, duration of first remission, and type of HSCT. In multivariable analysis, the only prognostic factors were disease status at HSCT and year of transplantation. Patients transplanted in third or subsequent complete remission (CR) had a risk of death 2.3 times greater than those in CR2. Children treated after 2000 had half the risk of death than those treated before that year. Our results suggest that both autologous and allogeneic HSCT may be considered for the treatment of pediatric ALL IEMR after the achievement of CR2.

Published
2019

31. Late mortality and causes of death among 5-year survivors of childhood cancer diagnosed in the period 1960–1999 and registered in the Italian Off-Therapy Registry

Author

Bagnasco, F., Caruso, S., Andreano, A., Valsecchi, M. G., Jankovic, M., Biondi, A., Miligi, L., Casella, C., Terenziani, M., Massimino, M., Sacerdote, C., Morsellino, V., Erminio, G., Garaventa, A., Faraci, M., Micalizzi, C., Garre, M. L., Pillon, M., Basso, G., Biasin, E., Fagioli, F., Rondelli, R., Pession, A., Locatelli, Franco, Santoro, N., Indolfi, P., Palumbo, G., Russo, G., Verzegnassi, F., Favre, C., Zecca, M., Mura, R., D'Angelo, P., Cano, C., Byrne, J., Haupt, R., Pierani, P., Porta, F., Consarino, C., Burnelli, R., Fedeli, F., Cellini, M., Casale, F., Menna, G., Bertolini, P., Caniglia, M., Cecinati, V., Casazza, G., Foa, R., Clerico, A., Ladogana, S., Galimberti, D., Rabusin, M., Nespoli, L., Cesaro, S., Locatelli F. (ORCID:0000-0002-7976-3654), Bagnasco, F., Caruso, S., Andreano, A., Valsecchi, M. G., Jankovic, M., Biondi, A., Miligi, L., Casella, C., Terenziani, M., Massimino, M., Sacerdote, C., Morsellino, V., Erminio, G., Garaventa, A., Faraci, M., Micalizzi, C., Garre, M. L., Pillon, M., Basso, G., Biasin, E., Fagioli, F., Rondelli, R., Pession, A., Locatelli, Franco, Santoro, N., Indolfi, P., Palumbo, G., Russo, G., Verzegnassi, F., Favre, C., Zecca, M., Mura, R., D'Angelo, P., Cano, C., Byrne, J., Haupt, R., Pierani, P., Porta, F., Consarino, C., Burnelli, R., Fedeli, F., Cellini, M., Casale, F., Menna, G., Bertolini, P., Caniglia, M., Cecinati, V., Casazza, G., Foa, R., Clerico, A., Ladogana, S., Galimberti, D., Rabusin, M., Nespoli, L., Cesaro, S., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

Introduction: Advances in paediatric oncology led to the increase in long-term survival, revealing the burden of therapy-related long-term side effects. We evaluated overall and cause-specific mortality in a large cohort of Italian childhood cancer survivors (CCSs) and adolescent cancer survivors identified through the off-therapy registry. Materials and methods: CCSs alive 5 years after cancer diagnosis occurring between 1960 and 1999 were eligible; the last follow-up was between 2011 and 2014. Outcomes were reported as standardised mortality ratios (SMRs) and absolute excess risks (AERs). Results: Among 12,214 CCSs, 1113 (9.1%) deaths occurred. Survival at 35 years since diagnosis was 87% (95% confidence interval [CI]: 86–88) and at 45 years was 81% (95% CI: 77–84). CCSs had an 11-fold increased risk of death (SMR 95% CI: 10.7–12), corresponding to an AER of 48 (95% CI: 45–51). Mortality decreased by 60% for survivors treated most recently (1990–1999). The most frequent causes of death were recurrence of the original cancer (56%), a subsequent neoplasm (19%) and cardiovascular diseases (5.8%). Among those who survived at least 15 years after diagnosis, a secondary malignancy was the leading cause of death. Conclusions: This study confirms the impact of recent advances in anticancer therapy in reducing mortality, mainly attributable to recurrence but also to other causes. However, overall mortality continues to be higher than in the general population. A long-term follow-up is needed to prevent late mortality due to secondary neoplasms and non-neoplastic causes in CCSs.

Published
2019

32. Late mortality and causes of death among 5-year survivors of childhood cancer diagnosed in the period 1960–1999 and registered in the Italian Off-Therapy Registry

Author

Bagnasco, F, Caruso, S, Andreano, A, Valsecchi, M, Jankovic, M, Biondi, A, Miligi, L, Casella, C, Terenziani, M, Massimino, M, Sacerdote, C, Morsellino, V, Erminio, G, Garaventa, A, Faraci, M, Micalizzi, C, Garrè, M, Pillon, M, Basso, G, Biasin, E, Fagioli, F, Rondelli, R, Pession, A, Locatelli, F, Santoro, N, Indolfi, P, Palumbo, G, Russo, G, Verzegnassi, F, Favre, C, Zecca, M, Mura, R, D'Angelo, P, Cano, C, Byrne, J, Haupt, R, Pierani, P, Porta, F, Consarino, C, Burnelli, R, Fedeli, F, Cellini, M, Casale, F, Menna, G, Bertolini, P, Caniglia, M, Cecinati, V, Casazza, G, Foà, R, Clerico, A, Ladogana, S, Galimberti, D, Rabusin, M, Nespoli, L, Cesaro, S, Bagnasco, Francesca, Caruso, Silvia, Andreano, Anita, Valsecchi, Maria Grazia, Jankovic, Momcilo, Biondi, Andrea, Miligi, Lucia, Casella, Claudia, Terenziani, Monica, Massimino, Maura, Sacerdote, Carlotta, Morsellino, Vera, Erminio, Giovanni, Garaventa, Alberto, Faraci, Maura, Micalizzi, Concetta, Garrè, Maria Luisa, Pillon, Marta, Basso, Giuseppe, Biasin, Eleonora, Fagioli, Franca, Rondelli, Roberto, Pession, Andrea, Locatelli, Franco, Santoro, Nicola, Indolfi, Paolo, Palumbo, Giovanna, Russo, Giovanna, Verzegnassi, Federico, Favre, Claudio, Zecca, Marco, Mura, Rossella, D'Angelo, Paolo, Cano, Carmen, Byrne, Julianne, Haupt, Riccardo, Pierani, Paolo, Pession, Andreea, Porta, Fulvio, Consarino, Caterina, Burnelli, Roberta, Fedeli, Fausto, Cellini, Monica, Casale, Fiorina, Menna, Giuseppe, Bertolini, Patrizia, Caniglia, Maurizio, Cecinati, Valerio, Casazza, Gabriella, Foà, Roberto, Clerico, Anna, Ladogana, Saverio, Galimberti, Daniela, Rabusin, Marco, Nespoli, Luigi, Cesaro, Simone, Bagnasco, F, Caruso, S, Andreano, A, Valsecchi, M, Jankovic, M, Biondi, A, Miligi, L, Casella, C, Terenziani, M, Massimino, M, Sacerdote, C, Morsellino, V, Erminio, G, Garaventa, A, Faraci, M, Micalizzi, C, Garrè, M, Pillon, M, Basso, G, Biasin, E, Fagioli, F, Rondelli, R, Pession, A, Locatelli, F, Santoro, N, Indolfi, P, Palumbo, G, Russo, G, Verzegnassi, F, Favre, C, Zecca, M, Mura, R, D'Angelo, P, Cano, C, Byrne, J, Haupt, R, Pierani, P, Porta, F, Consarino, C, Burnelli, R, Fedeli, F, Cellini, M, Casale, F, Menna, G, Bertolini, P, Caniglia, M, Cecinati, V, Casazza, G, Foà, R, Clerico, A, Ladogana, S, Galimberti, D, Rabusin, M, Nespoli, L, Cesaro, S, Bagnasco, Francesca, Caruso, Silvia, Andreano, Anita, Valsecchi, Maria Grazia, Jankovic, Momcilo, Biondi, Andrea, Miligi, Lucia, Casella, Claudia, Terenziani, Monica, Massimino, Maura, Sacerdote, Carlotta, Morsellino, Vera, Erminio, Giovanni, Garaventa, Alberto, Faraci, Maura, Micalizzi, Concetta, Garrè, Maria Luisa, Pillon, Marta, Basso, Giuseppe, Biasin, Eleonora, Fagioli, Franca, Rondelli, Roberto, Pession, Andrea, Locatelli, Franco, Santoro, Nicola, Indolfi, Paolo, Palumbo, Giovanna, Russo, Giovanna, Verzegnassi, Federico, Favre, Claudio, Zecca, Marco, Mura, Rossella, D'Angelo, Paolo, Cano, Carmen, Byrne, Julianne, Haupt, Riccardo, Pierani, Paolo, Pession, Andreea, Porta, Fulvio, Consarino, Caterina, Burnelli, Roberta, Fedeli, Fausto, Cellini, Monica, Casale, Fiorina, Menna, Giuseppe, Bertolini, Patrizia, Caniglia, Maurizio, Cecinati, Valerio, Casazza, Gabriella, Foà, Roberto, Clerico, Anna, Ladogana, Saverio, Galimberti, Daniela, Rabusin, Marco, Nespoli, Luigi, and Cesaro, Simone

Abstract

Introduction: Advances in paediatric oncology led to the increase in long-term survival, revealing the burden of therapy-related long-term side effects. We evaluated overall and cause-specific mortality in a large cohort of Italian childhood cancer survivors (CCSs) and adolescent cancer survivors identified through the off-therapy registry. Materials and methods: CCSs alive 5 years after cancer diagnosis occurring between 1960 and 1999 were eligible; the last follow-up was between 2011 and 2014. Outcomes were reported as standardised mortality ratios (SMRs) and absolute excess risks (AERs). Results: Among 12,214 CCSs, 1113 (9.1%) deaths occurred. Survival at 35 years since diagnosis was 87% (95% confidence interval [CI]: 86–88) and at 45 years was 81% (95% CI: 77–84). CCSs had an 11-fold increased risk of death (SMR 95% CI: 10.7–12), corresponding to an AER of 48 (95% CI: 45–51). Mortality decreased by 60% for survivors treated most recently (1990–1999). The most frequent causes of death were recurrence of the original cancer (56%), a subsequent neoplasm (19%) and cardiovascular diseases (5.8%). Among those who survived at least 15 years after diagnosis, a secondary malignancy was the leading cause of death. Conclusions: This study confirms the impact of recent advances in anticancer therapy in reducing mortality, mainly attributable to recurrence but also to other causes. However, overall mortality continues to be higher than in the general population. A long-term follow-up is needed to prevent late mortality due to secondary neoplasms and non-neoplastic causes in CCSs.

Published
2019

38. PS1573 B0 VS. NON-B0 GENOTYPE: DIFFERENCES IN TDT PATIENTS

Author

Meloni, A., primary, Pistoia, L., additional, Massa, A., additional, Rigoli, L., additional, Paci, C., additional, Argento, C., additional, Caniglia, M., additional, Palazzi, G., additional, Tedesco, L., additional, Missere, M., additional, Positano, V., additional, and Pepe, A., additional

Published
2019
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39. Outcomes of Children with Hemophagocytic Lymphohistiocytosis Given Allogeneic Hematopoietic Stem Cell Transplantation in Italy

Author

Messina, C., Zecca, M., Fagioli, F., Rovelli, A., Giardino, S., Merli, P., Porta, F., Arico, M., Sieni, E., Basso, G., Ripaldi, M., Favre, C., Pillon, M., Marzollo, A., Rabusin, M., Cesaro, S., Algeri, M., Caniglia, M., Di Bartolomeo, P., Ziino, O., Saglio, F., Prete, A., Locatelli, Franco, Locatelli F. (ORCID:0000-0002-7976-3654), Messina, C., Zecca, M., Fagioli, F., Rovelli, A., Giardino, S., Merli, P., Porta, F., Arico, M., Sieni, E., Basso, G., Ripaldi, M., Favre, C., Pillon, M., Marzollo, A., Rabusin, M., Cesaro, S., Algeri, M., Caniglia, M., Di Bartolomeo, P., Ziino, O., Saglio, F., Prete, A., Locatelli, Franco, and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

We report on 109 patients with hemophagocytic lymphohistiocytosis (HLH) undergoing 126 procedures of allogeneic hematopoietic stem cell transplantation (HSCT) between 2000 and 2014 in centers associated with the Italian Pediatric Hematology Oncology Association. Genetic diagnosis was FHL2 (32%), FHL3 (33%), or other defined disorders known to cause HLH (15%); in the remaining patients no genetic abnormality was found. Donor for first transplant was an HLA-matched sibling for 25 patients (23%), an unrelated donor for 73 (67%), and an HLA-partially matched family donor for 11 children (10%). Conditioning regimen was busulfan-based for 61 patients (56%), treosulfan-based for 21 (20%), and fludarabine-based for 26 children (24%). The 5-year probabilities of overall survival (OS) and event-free survival (EFS) were 71% and 60%, respectively. Twenty-six patients (24%) died due to transplant-related causes, whereas 14 (13%) and 10 (9%) patients experienced graft rejection and/or relapse, respectively. Twelve of 14 children given a second HSCT after graft failure/relapse are alive and disease-free. Use of HLA-partially matched family donors was associated with higher risk of graft failure and thus with lower EFS (but not with lower OS) in multivariable analysis. Active disease at transplantation did not significantly affect prognosis. These data confirm that HSCT can cure most HLH patients, active disease not precluding successful transplantation. Because in HLH patients HLA-haploidentical HSCT performed through CD34 + cell positive selection was found to be associated with poor sustained engraftment of donor cells, innovative approaches able to guarantee a more robust engraftment are warranted in patients given this type of allograft.

Published
2018

41. Il linfoma di Hodgkin in età pediatrica: risultati a lungo termine del protocollo AIEOP-MH’89

Author

Robazza, M., Mascarin, M., Elia, C., Barra, S., Bianchi, M., Buffardi, S., Bunkheila, F., Caniglia, M., Casini, T., Cellini, M., Cesaro, Simone, Consarino, C., D’Amico, S., De Santis, R., Farruggia, P., Guerrini, G., Garaventa, A., Indolfi, P., Locatelli, F., Moleti, M. L., Nardi, M., Pession, A., Pierani, P., Porta, F., Provenzi, M., Riccardi, U., Rondelli, R., Sala, A., Santoro, N., Sau, A., Scarzello, G., Todesco, A., Zanazzo, G. A., Zecca, M., and Brunelli, R.

Subjects
chemioterapia, protocollo MH 89, Linfoma di Hodgkin, Linfoma di Hodgkin, protocollo MH 89, chemioterapia
Published
2016

42. Endoscopic endonasal transclival approach to a pontine cavernous malformation: case report

Author

Dallan, I., Battaglia, P., de Notaris, M., Caniglia, M., Turri-Zanoni, M., and TURRI ZANONI, Mario

Subjects
Neurological signs, Male, medicine.medical_specialty, Adolescent, Transclival, Neurological examination, Nose, Nervous System Malformations, Lesion, Acute onset, Cephalalgia, Pons, medicine, Humans, Brainstem hemorrhage, Cavernoma, Endonasal, Endoscopic, Skull Base, book, book.periodical, medicine.diagnostic_test, business.industry, Cranial nerves, Headache, Endoscopy, General Medicine, Cavernous malformations, medicine.disease, Magnetic Resonance Imaging, Cranial Nerve Diseases, Surgery, Otorhinolaryngology, Pediatrics, Perinatology and Child Health, Brainstem, medicine.symptom, business
Abstract

Cavernous malformations of the brainstem are difficult to manage because of their location in eloquent tissue and their high propensity for symptomatic bleeding. Traditional neurosurgical approaches are often associated with significant morbidities. Here we present the case of a 15 year-old male patient with an acute onset of severe cephalalgia associated with neurological signs (right cranial nerve VI, VII and VIII palsies). MRI revealed a ventral pontine cavernous malformation with signs of recent bleeding. The lesion was removed by way of an endoscopic endonasal transclival approach. Post-operative neurological examination showed a dramatic improvement in cranial nerves function. The patient remains stable two years after surgery.

Published
2015

43. Infections by carbapenem-resistant Klebsiella pneumoniae in SCT recipients: a nationwide retrospective survey from Italy

Author

Girmenia, C, Rossolini, Gm, Piciocchi, A, Bertaina, A, Pisapia, G, Pastore, D, Sica, S, Severino, A, Cudillo, L, Ciceri, F, Scimè, R, Lombardini, L, Viscoli, C, Rambaldi, A, the Gruppo Italiano Trapianto Midollo Osseo (GITMO), Frigeni, M, Corti, C, Mometto, G, Annaloro, C, Casari, E, Castagna, L, Rossi, G, Cattaneo, C, Russo, D, Cancelli, V, Alessandrino, Ep, Ripamonti, F, Pavan, F, Rovelli, A, Pecoraro, C, Busca, A, Carraro, F, Fagioli, F, Gallo, S, Caravelli, D, De Gobbi, M, Saglio, G, Castellino, C, Mordini, N, Gaidano, G, Nassi, L, Raimondi, R, Vespignani, M, Scattolin, Am, Panizzolo, Is, Cesaro, S, Candoni, A, Patriarca, F, Bacigalupo, A, Raiola, A, Castagnola, E, Lanino, E, Stanzani, M, Bandini, G, Massaccesi, E, Prete, A, Bassi, S, Vallisa, D, Caramatti, C, Aversa, F, Zuffa, E, Guidi, S, Bosi, A, Tintori, V, Iori, Ap, Capria, S, Arcese, W, Dentamaro, T, Fabritiis, Pd, Anaclerico, B, Chierichini, A, Piedimonte, M, Ferrari, A, Marchesi, F, Mengarelli, A, Cerchiara, E, Tirindelli, Mc, Gaziev, J, Majolino, I, Chiusolo, P, Lucarelli, B, Massei, Ms, Carotti, A, Perruccio, K, Caniglia, M, Santarone, S, Bartolomeo, Pd, Mazzotta, S, Galieni, P, Olivieri, A, Rosa, Gd, Risitano, A, Delia, M, Specchia, G, Palazzo, G, Messina, G, Irrera, G, Angelucci, E, Baronciani, D, Vacca, A, Crescimanno, A, Musso, M, Imbriani, A, Milone, G., Girmenia, C, Rossolini, Gm, Piciocchi, A, Bertaina, A, Pisapia, G, Pastore, D, Sica, S, Severino, A, Cudillo, L, Ciceri, Fabio, Scime, R, Lombardini, L, Viscoli, C, Rambaldi, A, and and the Gruppo Italiano Trapianto Midollo Osseo, (GITMO)

Subjects
Male, Klebsiella pneumoniae, Carbapenem resistant Klebsiella pneumoniae, Drug Resistance, carbapenem-resistant Klebsiella pneumoniae, colonization, sepsis, Allogeneic Hematopoietic Stem Cell Transplantation, Hematologic Diseases, hemic and lymphatic diseases, polycyclic compounds, Medicine, Adolescent, Adult, Aged, Allografts, Autografts, Female, Humans, Italy, Klebsiella Infections, Middle Aged, Retrospective Studies, Carbapenems, Drug Resistance, Bacterial, Stem Cell Transplantation, Hematology, Transplantation, biology, Bacterial, humanities, surgical procedures, operative, medicine.medical_specialty, Retrospective survey, Internal medicine, business.industry, Retrospective cohort study, Klebsiella infections, biology.organism_classification, Settore MED/15, infection, body regions, Settore MED/15 - MALATTIE DEL SANGUE, Immunology, business
Abstract

Infections by carbapenem-resistant Klebsiella pneumoniae (CRKp) represent a challenging problem after SCT. A retrospective survey (January 2010 to July 2013) involving 52 Italian centers was performed to assess the epidemiology and the prognostic factors of CRKp infections in auto- and allo-SCT. Cases of CRKp infection were reported in 53.4% of centers. CRKp infections were documented in 25 auto-SCTs and 87 allo-SCTs, with an incidence of 0.4% (from 0.1% in 2010 to 0.7% in 2013) and 2% (from 0.4% in 2010 to 2.9% in 2013), respectively. A CRKp colonization documented before or after transplant was followed by an infection in 25.8% of auto-SCT and 39.2% of allo-SCT patients. The infection-related mortality rates were 16% and 64.4%, respectively. A pre-transplant CRKp infection (hazard ratio (HR) 0.33, 95% confidence intervals (CIs) 0.15-0.74; P = 0.007) and a not CRKp-targeted first-line treatment (HR 2.67, 95% CI 1.43-4.99; P = 0.002) were independent factors associated with an increased mortality in allo-SCT patients who developed a CRKp infection. Our study shows challenging findings of CRKp infections in SCT patients in Italy particularly after allo-SCT. The detection of carriers and the definition of early therapeutic strategies represent critical aspects of the management of CRKp infections after SCT. OI Aversa, Franco/0000-0002-8871-6817; de Fabritiis, Paolo/0000-0002-1835-0581; TIRINDELLI, MARIA CRISTINA/0000-0003-4645-4465; cudillo, laura/0000-0002-6828-9707

Published
2015

44. STUDIO DELLA RICOSTITUZIONE IMMUNOLOGICAASPERGILLO-SPECIFICA IN TRAPIANTI DICELLULE STAMINALI EMOPOIETICHE T-REPLETI E T-DEPLETI. XL Congresso Nazionale Associazione Italiana Ematologia Oncologia Pediatrica (AIEOP). Lecce 24-26 maggio 2015

Author

Perruccio, K., Topini, F., Tosti, A., Gazzola, M. V., Messina, C., Martelli, M. F., Caniglia, M., Velardi, A., and Cesaro, Simone

Subjects
infezioni fungine, T-deplezione, infezioni fungine, trapianto di cellule staminali emopoietiche, T-deplezione, trapianto di cellule staminali emopoietiche
Published
2015

45. Overview of T-cell depletion in haploidentical stem cell transplantation

Author

Daniele, N., Scerpa, M., Caniglia, M., Ciammetti, C., Rossi, C., Bernardo, M., Locatelli, F., Isacchi, G., Zinno, F., Daniele, N., Scerpa, M., Caniglia, M., Ciammetti, C., Rossi, C., Bernardo, M., Locatelli, F., Isacchi, G., and Zinno, F.

Subjects
Cellular processing, Haploidentical transplantation, T-Lymphocytes, Graft-versus-host disease, Immunomagnetic selection, Graft vs Host Disease, Humans, Transplantation, Homologous, Settore MED/05 - Patologia Clinica, Lymphocyte Depletion, Stem Cell Transplantation
Published
2012

46. indicazioni e risultati del trapianto allogenico in età pediatrica

Author

Prete, A., Rondelli, R., Zecca, M., Fagioli, F., Lanino, E., Messina, C., Rovelli, A., Porta, F., Iori, A. P., Favre, C., Rabusin, M., Ripaldi, M., Caselli, D., Lo Nigro, L., Cesaro, Simone, Caniglia, M., Di Bartolomeo, P., Ziino, O., Orofino, M. G., Paolucci, P., Pession, A., and Locatelli, F.

Subjects
RISULTATI, PEDIATRIA, TRAPIANO DI MIDOLLO ALLOGENICO
Published
2014

50. Combined cord blood and bone marrow transplantation from the same human leucocyte antigen-identical sibling donor for children with malignant and non-malignant diseases

Author

Tucunduva, L., Volt, F., Cunha, R., Locatelli, Franco, Zecca, M., Yesilipek, A., Caniglia, M., Gungor, T., Aksoylar, S., Fagioli, F., Bertrand, Y., Addari, M. C., de la Fuente, J., Winiarski, J., Biondi, A., Sengeloev, H., Badell, I., Mellgren, K., de Heredia, C. D., Sedlacek, P., Vora, A., Rocha, V., Ruggeri, A., Gluckman, E., Locatelli F. (ORCID:0000-0002-7976-3654), Tucunduva, L., Volt, F., Cunha, R., Locatelli, Franco, Zecca, M., Yesilipek, A., Caniglia, M., Gungor, T., Aksoylar, S., Fagioli, F., Bertrand, Y., Addari, M. C., de la Fuente, J., Winiarski, J., Biondi, A., Sengeloev, H., Badell, I., Mellgren, K., de Heredia, C. D., Sedlacek, P., Vora, A., Rocha, V., Ruggeri, A., Gluckman, E., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

Summary: Umbilical cord blood (UCB) from an human leucocyte antigen (HLA)-identical sibling can be used for transplantation of patients with malignant and non-malignant diseases. However, the low cellular content of most UCB units represents a limitation to this approach. An option to increase cell dose is to harvest bone marrow (BM) cells from the same donor and infuse them along with the UCB. We studied 156 children who received such a combined graft between 1992 and 2011. Median age was 7 years and 78% of patients (n = 122) were transplanted for non-malignant diseases, mainly haemoglobinopathies. Acute leukaemia (n = 26) was the most frequent malignant diagnosis. Most patients (91%) received myeloablative conditioning. Median donor age was 1·7 years, median infused nucleated cell dose was 24·4 × 107/kg and median follow-up was 41 months. Sixty-days neutrophil recovery occurred in 96% of patients at a median of 17 d. The probabilities of grade-II-IV acute and chronic graft-versus-host disease (GVHD) were 19% and 10%, respectively. Four-year overall survival was 90% (68% malignant; 97% non-malignant diseases) with 3% probability of death. In conclusion, combined UCB and BM transplantation from an HLA-identical sibling donor is an effective treatment for children with malignant and non-malignant disorders with high overall survival and low incidence of GVHD.

Published
2015

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