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2. The proportion of different BCR-ABL1 transcript types in chronic myeloid leukemia. An international overview

Author

Baccarani, M, Castagnetti, F, Gugliotta, G, Rosti, G, Soverini, S, Albeer, A, Pfirrmann, M, Bekadja, Ma, Entasoltan, B, Nachi, M, Elghandour, A, El Sorady, M, Abdelfattah, R, El Nahass, Y, Samra, M, Azzazi, M, Elsobki, E, Moussa, M, Fahmy, O, Mattar, M, Shehata, Azmy, Se, (Azmy, E, 9 ), Emad), Bolarinwa, (Bolarinwa, Ra, ( 10 ), Rahman A., Eid, (Eid, S, Samir)( 11, ), Khelif, (Khelif, A, Abderrhaim)( 11, ), Hached, (Hached, F, Farhat)( 11, ), Menif, (Menif, S, Samia)( 12, ), Rahman, (Rahman, H, Hafizur)( 13, ), Huang, (Huang, Xj, Xiaojun)(, 14, 15, ), Jiang, (Jiang, Q, Qian)(, 14, (Ye, Yx, Yuanxin)( 16, ), Zhu, (Zhu, Hl, Huanling)( 16, ), Chen, (Chen, Sn, Suning)( 17, ), Varma, (Varma, N, Neelam)( 18, ), Ganesan, (Ganesan, P, Prasanth)( 19, ), Gundeti, (Gundeti, S, Sadashivudu)( 20, ), Malhotra, (Malhotra, H, Hemant)( 21, ), Radhakrishnan, (Radhakrishnan, Vs, ( 22 ), Vivek S., Kumar, (Kumar, L, Lalit)( 23, ), Sharawat, (Sharawat, Sk, Surender Kumar)( 23, ), Seth, (Seth, T, Tulika)( 24, ), Ausekar, (Ausekar, Bv, ( 25 ), B. V., Balasubramanian, (Balasubramanian, P, Poonkuzhali)( 26, ), Poopak, (Poopak, B, Behzad)(, 27, 28, ), Inokuchi, (Inokuchi, K, Koiti)( 29, ), Kim, (Kim, Dw, Dong-Wook)( 30, ), Kindi, Al, S (Al Kindi, Salam)( 31, ), Mirasol, (Mirasol, A, Angelina)( 32, ), Qari, (Qari, M, Mohammed)( 33, ), Goh, (Goh, Yt, Yeow Tee)( 34, ), Shih, (Shih, Ly, Lee-Yung)(, 35, 36, ), Branford, (Branford, S, Susan)(, 37, 38, ), Lion, (Lion, T, Thomas)( 39, ), Valent, (Valent, P, Peter)( 40, ), Burgstaller, (Burgstaller, S, Sonja)( 41, ), Thaler, (Thaler, J, Joseph)( 41, ), Labar, (Labar, B, Boris)( 42, ), Zadro, (Zadro, R, Renata)( 42, ), Mayer, (Mayer, J, Jiri)(, 43, 44, ), Zackova, (Zackova, D, Daniela)(, 43, Faber, (Faber, E, Edgar)( 45, ), Pallisgaard, (Pallisgaard, N, Niels)( 46, ), Xavier-Mahon, (Xavier-Mahon, F, Francois)( 47, ), Lippert, (Lippert, E, Eric)( 48, ), Cayuela, (Cayuela, Jm, Jean Michel)( 49, ), Rea, (Rea, D, Delphine)( 49, ), Millot, (Millot, F, Frederic)( 50, ), Suttorp, (Suttorp, M, Meinolf)( 51, ), Hochhaus, (Hochhaus, A, Andreas)( 52, ), Niederwieser, (Niederwieser, D, Dietger)( 53, ), Saussele, (Saussele, S, Susanne)( 54, ), Haferlach, (Haferlach, T, Torsten)( 55, ), Jeromine, (Jeromine, S, Sabine)( 55, ), Panayiotidis, (Panayiotidis, P, Panayiotis)(, 56, 57, ), Conneally, (Conneally, E, Eibhlin)( 58, ), Langabeer, (Langabeer, S, Steve)( 58, ), Nagler, (Nagler, A, Arnon)(, 59, 60, ), Rupoli, (Rupoli, S, Serena)( 61, ), Santoro, (Santoro, N, Nicola)( 62, ), Albano, (Albano, F, Francesco)( 63, ), Castagnetti, (Castagnetti, F, Fausto), Ottaviani, (Ottaviani, E, Emanuela)(, 64, 65, ), Rambaldi, (Rambaldi, A, Alessandro)(, 66, 67, ), Stagno, (Stagno, F, Fabio)( 68, ), Molica, (Molica, S, Stefano)( 69, ), Biagiotti, (Biagiotti, C, Caterina)( 70, ), Scappini, (Scappini, B, Barbara)( 70, ), Lemoli, (Lemoli, R, Roberto)( 71, ), Iurlo, (Iurlo, A, Alessandra)(, 72, 73, ), Pungolino, (Pungolino, E, Ester)( 74, ), Menna, (Menna, G, Giuseppe), Pane, (Pane, F, Fabrizio)( 76, ), Gottardi, (Gottardi, E, Enrico)(, 77, 78, ), Rege-Cambrin, (Rege-Cambrin, G, Giovanna)(, 77, Binotto, (Binotto, G, Gianni)( 79, ), Putti, (Putti, Mc, Maria Caterina)( 80, ), Falzetti, (Falzetti, F, Franca)( 81, ), Visani, (Visani, G, Giuseppe)( 82, ), Galimberti, (Galimberti, S, Sara)( 83, ), Musto, (Musto, P, Pellegrino)( 84, ), Abruzzese, (Abruzzese, E, Elisabetta)( 85, ), Breccia, (Breccia, M, Massimo)( 86, ), Giona, (Giona, F, Fiorina)( 86, ), Chiusolo, (Chiusolo, P, Patrizia)( 87, ), Sica, (Sica, S, Simona)( 87, ), Fava, (Fava, C, Carmen)( 88, ), Ferrero, (Ferrero, D, Dario)( 88, ), Tiribelli, (Tiribelli, M, Mario)( 89, ), Bonifacio, (Bonifacio, M, Massimiliano)( 90, ), Griskevicius, (Griskevicius, L, Laimonas)( 91, ), Musteata, (Musteata, V, Vasile)( 92, ), Janssen, (Janssen, J, Jeroen)( 93, ), Prejzner, (Prejzner, W, Witold)( 94, ), Sacha, (Sacha, T, Tomasz)( 95, ), Waclaw, (Waclaw, J, Joanna)( 95, ), Almeida, (Almeida, Am, Antonio Medina)( 96, ), Kulikov, (Kulikov, S, Sergei)( 97, ), Turkina, (Turkina, A, Anna)( 97, ), Bogdanovic, (Bogdanovic, A, Andrija)( 98, ), Zupan, (Zupan, I, Irena)( 99, ), Marce, (Marce, S, Silvia)( 100, ), Cervantes, (Cervantes, F, Francisco)( 101, ), Steegmann, (Steegmann, Jl, Juan Luis)( 102, ), Kotlyarchuk, (Kotlyarchuk, K, Konstyantyn)( 103, ), Milner, (Milner, Bj, ( 104 ), Benedict J., Rose, (Rose, S, Susan)( 105, ), Clench, (Clench, T, Tim)( 106, ), Waits, (Waits, P, Paula)( 107, ), Austin, (Austin, S, Steve)( 108, ), Wickham, (Wickham, C, Caroline)( 109, ), Clark, (Clark, R, Richard)( 110, ), Apperley, (Apperley, J, Jane), Claudiani, (Claudiani, S, Simone)( 111, ), Foroni, (Foroni, L, Letizia)( 111, ), Szydlo, (Szydlo, R, Richard)( 111, ), Burt, (Burt, E, Emma)( 112, ), Bescoby, (Bescoby, R, Ruth)( 113, ), Cork, (Cork, L, Leanne)( 113, ), O'Brien, (O'Brien, S, Stephen)( 113, ), Green, (Green, B, Bethaney)( 114, ), Hawtree, (Hawtree, S, Sarah)( 114, ), Watson, (Watson, M, Mark)( 114, ), Bengio, (Bengio, Rm, Raquel Maria)( 115, ), Larripa, (Larripa, I, Irene)( 115, ), Pavlovsky, (Pavlovsky, C, Carolina)( 116, ), Moiraghi, (Moiraghi, B, Beatriz)( 117, ), Pinna, De, CAR (Requiao de Pinna, Cristiane Almeida)( 118, ), Magalhaes, GHR (Romani Magalhaes, Gustavo Henrique)( 119, ), Pagnano, (Pagnano, K, Katia)( 120, ), Funke, (Funke, V, Vaneuza)( 121, ), Tavares, (Tavares, Rs, Renato Sampaio)( 122, ), Prado, (Prado, A, Adriana)( 123, ), Azevedo, (Azevedo, Aa, Alita Andrade)( 124, ), Fogliatto, (Fogliatto, L, Laura)( 125, ), Bonecker, (Bonecker, S, Simone)( 126, ), Centrone, (Centrone, R, Renato)( 127, ), Moellman, (Moellman, A, Artur)( 128, ), Conchon, (Conchon, M, Monika)( 130, ), Centurion, (Centurion, Me, Maria Elida)( 131, ), (Prado, Ai, Ana-Ines)( 132, ), Lopez, (Lopez, Jl, ( 133 ), J. L., Petruzziello, (Petruzziello, F, Fara)( 75, ), Bendit, (Bendit, I, Israel), Baccarani M., Castagnetti F., Gugliotta G., Rosti G., Soverini S., Albeer A., and Pfirrmann M.

Subjects
Male, 0301 basic medicine, Cancer Research, bcr-abl, Fusion Proteins, bcr-abl, Global Health, 0302 clinical medicine, hemic and lymphatic diseases, 80 and over, Odds Ratio, Prevalence, Age Factor, Chronic, Young adult, Child, MOLECULAR RESPONSE, Leukemic, Aged, 80 and over, Leukemia, Hematology, Gene Expression Regulation, Leukemic, CHRONIC MYELOGENOUS LEUKEMIA, Age Factors, Myeloid leukemia, Middle Aged, Oncology, Child, Preschool, 030220 oncology & carcinogenesis, Female, Life Sciences & Biomedicine, Human, Adult, Transcriptional Activation, medicine.medical_specialty, Adolescent, Immunology, IMATINIB MESYLATE, DENDRITIC CELLS, CML PATIENTS, Young Adult, 03 medical and health sciences, Myelogenous, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Internal medicine, medicine, Humans, 1112 Oncology and Carcinogenesis, BCR/ABL TRANSCRIPT, Preschool, CYTOGENETIC RESPONSE, Aged, Science & Technology, CHRONIC-PHASE, business.industry, Infant, Newborn, Fusion Proteins, ABL FUSION PROTEINS, P190 BCR-ABL, Infant, 1103 Clinical Sciences, Odds ratio, Newborn, medicine.disease, International BCR-ABL Study Group, Settore MED/15 - MALATTIE DEL SANGUE, 030104 developmental biology, Imatinib mesylate, Gene Expression Regulation, BCR-ABL Positive, business, Chronic myelogenous leukemia
Abstract

There are different BCR-ABL1 fusion genes that are translated into proteins that are different from each other, yet all leukemogenic, causing chronic myeloid leukemia (CML) or acute lymphoblastic leukemia. Their frequency has never been systematically investigated. In a series of 45503 newly diagnosed CML patients reported from 45 countries, it was found that the proportion of e13a2 (also known as b2a2) and of e14a2 (also known as b3a2), including the cases co-expressing e14a2 and e13a2, was 37.9% and 62.1%, respectively. The proportion of these two transcripts was correlated with gender, e13a2 being more frequent in males (39.2%) than in females (36.2%), was correlated with age, decreasing from 39.6% in children and adolescents down to 31.6% in patients ≥ 80 years old, and was not constant worldwide. Other, rare transcripts were reported in 666/34561 patients (1.93%). The proportion of rare transcripts was associatedwith gender (2.27% in females and 1.69% in males) and with age (from 1.79% in children and adolescents up to 3.84% in patients ≥ 80 years old). These data show that the differences in proportion are not by chance. This is important, as the transcript type is a variable that is suspected to be of prognostic importance for response to treatment, outcome of treatment, and rate of treatment-free remission.

Published
2019

7. Corrigendum to: 'Vaccination in immunocompromised host: Recommendations of Italian Primary Immunodeficiency Network Centers (IPINET)' [Vaccine 36 (2018) Pages 3541–3542]

Author

Martire, Baldassarre, Azzari, Chiara, Badolato, Raffaele, Canessa, Clementina, Cirillo, Emilia, Gallo, Vera, Graziani, Simona, Lorenzini, Tiziana, Milito, Cinzia, Panza, Raffaella, Moschese, Viviana, Pignata, Claudio, Martire, B, Lassandro, G, Panza, R, Vacca, A, Marasco, C, Cardinale, F, Sisto, C, Pession, A, Ricci, G, Rondelli, R, Specchia, F, Plebani, A, Badolato, R, Lougaris, V, Soresina, A, Miniero, R, Anastasio, E, Paganelli, R, Dilizia, Sperli, D, Carpino, L, Cirillo, E, Gallo, V, Giardino, G, Spadaro, G, Pecoraro, A ), Putti, Mc, Agostini, C, Cinetto, F, Trizzino, A, Bertolini, P), Arlotta, A, Marseglia, Gl, Bossi, G, Consolini, R, and Et, Al.

Subjects
ANTIBODY-RESPONSES, Secondary immunodeficiency KeyWords Plus:CHRONIC GRANULOMATOUS-DISEASE, ADVISORY-COMMITTEE, ROTAVIRUS VACCINATION, IMMUNIZATION PRACTICES, Medicine, HUMORAL IMMUNITY, Author Keywords:Vaccination, Primary immunodeficiency, Syndromic immunodeficiency, ACUTE LYMPHOBLASTIC-LEUKEMIA, CALMETTE-GUERIN, POLYSACCHARIDE VACCINATION, MENDELIAN SUSCEPTIBILITY, General Veterinary, General Immunology and Microbiology, business.industry, Public Health, Environmental and Occupational Health, medicine.disease, Virology, Vaccination, Infectious Diseases, Molecular Medicine, business, Host (network)
Published
2018

8. Results of the AIEOP AML 2002/01 multicenter prospective trial for the treatment of children with acute myeloid leukemia

Author

Pession A, Masetti R, Rizzari C, Putti MC, Fagioli F, Luciani M, Lo Nigro L, Menna G, Micalizzi C, Santoro N, Testi AM, Zecca M, Biondi A, Pigazzi M, Rutella S, Rondelli R, Basso G, Locatelli F, AIEOP AML Study Group, CASALE, Fiorina, Pession, A, Masetti, R, Rizzari, C, Putti, Mc, Casale, Fiorina, Fagioli, F, Luciani, M, Lo Nigro, L, Menna, G, Micalizzi, C, Santoro, N, Testi, Am, Zecca, M, Biondi, A, Pigazzi, M, Rutella, S, Rondelli, R, Basso, G, Locatelli, F, AIEOP AML Study, Group, Pession A, Masetti R, Rizzari C, Putti MC, Casale F, Fagioli F, Luciani M, Lo Nigro L, Menna G, Micalizzi C, Santoro N, Testi AM, Zecca M, Biondi A, Pigazzi M, Rutella S, Rondelli R, Basso G, Locatelli F, Putti, M, Casale, F, Testi, A, and AIEOP AML Study, G

Subjects
Oncology, Myeloid, Male, Pediatrics, medicine.medical_treatment, Hematopoietic stem cell transplantation, Biochemistry, Recurrence, hemic and lymphatic diseases, Antineoplastic Combined Chemotherapy Protocols, Child, PEDIATRIC AML, Leukemia, ACUTE MYELOGENOUS LEUKEMIA, Hematopoietic Stem Cell Transplantation, Myeloid leukemia, Induction Chemotherapy, Hematology, HIGH-DOSE CYTARABINE, Leukemia, Myeloid, Acute, medicine.anatomical_structure, Treatment Outcome, N/A, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, Child, Preschool, Female, Autologous, Homologous, medicine.medical_specialty, Adolescent, Immunology, Acute, Transplantation, Autologous, AIEOP, children, acute myeloid leukemia, Internal medicine, medicine, Transplantation, Homologous, Autologous transplantation, Humans, Preschool, Transplantation, business.industry, Infant, Newborn, Induction chemotherapy, Infant, Cell Biology, medicine.disease, Newborn, BONE-MARROW-TRANSPLANTATION, INTENSIVE CHEMOTHERAPY, Consolidation Chemotherapy, Follow-Up Studies, Prospective trial, business
Abstract

We evaluated the outcome of 482 children with acute myeloid leukemia (AML) enrolled in the Associazione Italiana di Ematologia e Oncologia Pediatrica AML 2002/01 trial. Treatment was stratified according to risk group; hematopoietic stem cell transplantation (HSCT) was used in high-risk (HR) children. Patients with core binding factor leukemia achieving complete remission (CR) after the first induction course were considered standard risk (SR; 99 patients), whereas the others (n 5 383) were assigned to the HR group. Allogeneic (ALLO) or autologous (AUTO) HSCT was employed, respectively, in 141 and 102 HR patients after consolidation therapy. CR, early death, and induction failure rates were 87%, 3%, and 10%, respectively. Relapse occurred in 24%of patients achieving CR. The 8-year overall survival (OS), event-free survival (EFS), and disease-free survival (DFS) were 68%, 55%, and 63%, respectively. OS, EFS, and DFS for SR and HR patients were 83%, 63%, and 66% and 64%, 53%, and 62%. DFS was 63% and 73% for HR patients given AUTO-HSCT and ALLO-HSCT, respectively. In multivariate analysis, risk group, white blood cell >100 × 109/L at diagnosis, and monosomal karyotype predicted poorer EFS. Risk-oriented treatment and broad use of HSCT result in a long-term EFS comparing favorably with previously published studies on childhood AML. (Blood. 2013; 122(2):170-178) © 2013 by The American Society of Hematology.

Published
2013

12. Paediatric arterial ischaemic stroke and cerebral sinovenous thrombosis: First report from the italian registry of pediatric thrombosis (R. I. T. I., Registro Italiano Trombosi Infantili)

Author

Suppiej, A, Gentilomo, C, Saracco, P, Sartori, S, Agostini, M, Bagna, R, Bassi, B, Giordano, P, Grassi, M, Guzzetta, A, Lasagni, D, Luciani, M, Molinari, Ac, Palmieri, A, Putti, Mc, Ramenghi, La, Rota, Ll, Sperlì, D, Laverda, Am, Simioni, P(1), Collaborators: Angriman M, Stroke working group of the Italian Registry of Pediatric Thrombosis., Aru, Ab, Barisone, E, Bartalena, L, Berta, M, Bertoni, E, Cancarini, P, Cavaliere, E, Celle, Me, Cerbone, Am, Cesaroni, E, Dalla Via, L, Dell'Oro, Mg, Di Rosa, G, Ferrari, Gm, Fiori, S, Gaffuri, M, Gallina, Mr, Gimmillaro, A, Grandone, E, Ladogana, S, Laforgia, N, La Piana, R, Maschio, F, Miniero, R, Nosadini, M, Panzeri, D, Petrucci, A, Piersigilli, F, Sala, D, Sangermani, R, Santoro, N, Tufano, A, Ventura, G, Vittorini, R., Suppiej, A., Gentilomo, C., Saracco, P., Sartori, S., Agostini, M., Bagna, R., Bassi, B., Giordano, P., Grassi, M., Guzzetta, A., Lasagni, D., Luciani, M., Molinari, A. C., Palmieri, A., Putti, M. C., Ramenghi, L. A., Rota, L. L., Sperli, D., Laverda, A. M., Simioni, P., Angriman, M., Aru, A. B., Barisone, E., Bartalena, L., Berta, M., Bertoni, E., Cancarini, P., Cavaliere, E., Celle, M. E., Cerbone, A. M., Cesaroni, E., Via, L. D., Dell'Oro, M. G., Di Rosa, G., Ferrari, G. M., Fiori, S., Gaffuri, M., Gallina, M. R., Gimmillaro, A., Grandone, E., Ladogana, S., Laforgia, N., La Piana, R., Maschio, F., Miniero, R., Nosadini, M., Panzeri, D., Petrucci, A., Piersigilli, F., Sala, D., Sangermani, R., Santoro, N., Tufano, A., Ventura, G., and Vittorini, R.

Subjects
Male, Pediatrics, AIS, Children, CSVT, Italy, Registry, Thrombosis, Adolescent, Age of Onset, Brain Ischemia, Cerebral Arterial Diseases, Child, Child, Preschool, Delayed Diagnosis, Female, Humans, Infant, Predictive Value of Tests, Recurrence, Registries, Risk Factors, Sinus Thrombosis, Intracranial, Stroke, Time Factors, Treatment Outcome, Hematology, 030204 cardiovascular system & hematology, Sinus Thrombosis, Lethargy, 0302 clinical medicine, Medicine, Predictive value of tests, medicine.symptom, medicine.medical_specialty, NO, 03 medical and health sciences, Preschool, business.industry, medicine.disease, Intracranial, Clinical trial, Hemiparesis, Etiology, Age of onset, business, 030217 neurology & neurosurgery
Abstract

SummaryData from large case series of children with cerebral thrombotic events are pivotal to improve prevention, early recognition and treatment of these conditions. The Italian Registry of Pediatric Thrombosis (R. I. T. I.) was established in 2007 by a multidisciplinary team, aiming for a better understanding of neonatal and paediatric thrombotic events in Italy and providing a preliminary source of data for the future development of specific clinical trials and diagnostic-therapeutic protocols. We analysed data relative to the paediatric cerebral thrombotic events of the R. I. T. I. which occurred between January 2007 and June 2012. In the study period, 79 arterial ischaemic stroke (AIS) events (49 in males) and 91 cerebral sinovenous thrombosis (CSVT) events (65 in males) were enrolled in the R. I. T. I. Mean age at onset was 4.5 years in AIS, and 7.1 years in CSVT. Most common modes of presentation were hemiparesis, seizures and speech disturbances in AIS, and headache, seizures and lethargy in CSVT. Most common etiologies were underlying chronic diseases, vasculopathy and cardiopathy in AIS, and underlying chronic diseases and infection in CSVT. Time to diagnosis exceeded 24 hours in 46 % AIS and 59 % CSVT. Overall data from the Italian Registry are in substantial agreement with those from the literature, despite small differences. Among these, a longer time to diagnosis compared to other registries and case series poses the accent to the need of an earlier recognition of paediatric cerebrovascular events in Italy, in order to enable prompt and effective treatment strategies.

Published
2015

13. Effectiveness of immunoglobulin replacement therapy on clinical outcome in patients with primary antibody deficiencies: results from a multicenter prospective cohort study

Author

Quinti I, Soresina A, Guerra A, Rondelli R, Spadaro G, Agostini C, Milito C, Trombetta AC, Visentini M, Martini H, Plebani A, Fiorilli M, IPINet Investigators, De Mattia D, Martire B, Cardinale F, Ranieri G, Silvestri F, Masi M, Cossu F, Anastasio E, Schillirò G, Matucci A, Vultaggio A, Aricò M, Pietrogrande MC, Delle Piane RM, Panisi C, Cambiaghi G, Pignata C, Putti MC, Trizzino A, Bertolini P, Consolini R, Ugazio AG, Duse M, Iacobini M, Moschese V, Cancrini C, Finocchi A, Pesce AM, Cagliuso M, Conti V, Granata G, Mitrevski M, Cecere F, Tovo PA, Martino S, Montin D, Nespoli L, Marinoni M, Pellegrini FP, Cazzola G.A., PESSION, ANDREA, Quinti., I, Soresina, A., Guerra, A., Rondelli, R., Spadaro, Giuseppe, Agostini, C., Milito, C., Trombetta, A. C., Visentini, M., Martini, H., Plebani, A., Fiorilli, M., De Mattia, D., Martire, B., Cardinale, F., Ranieri, G., Silvestri, F., Masi, M., Pession, A., Cossu, F., Anastasio, E., Schillirò, G., Matucci, A., Vultaggio, A., Aricò, M., Pietrogrande, M. C., Delle Piane, R. M., Panisi, C., Cambiaghi, G., Pignata, Claudio, Putti, M. C., Trizzino, A., Bertolini, P., Consolini, R., Ugazio, A. G., Duse, M., Iacobini, M., Moschese, V., Cancrini, C., Finocchi, A., Pesce, A. M., Cagliuso, M., Conti, V., Granata, G., Mitrevski, M., Cecere, F., Tovo, P. A., Martino, S., Montin, D., Nespoli, L., Marinoni, M., Pellegrini, F. P., Cazzola, G. A., Quinti I, Soresina A, Guerra A, Rondelli R, Spadaro G, Agostini C, Milito C, Trombetta AC, Visentini M, Martini H, Plebani A, Fiorilli M, IPINet Investigator, De Mattia D, Martire B, Cardinale F, Ranieri G, Silvestri F, Masi M, Pession A, Cossu F, Anastasio E, Schillirò G, Matucci A, Vultaggio A, Aricò M, Pietrogrande MC, Delle Piane RM, Panisi C, Cambiaghi G, Pignata C, Putti MC, Trizzino A, Bertolini P, Consolini R, Ugazio AG, Duse M, Iacobini M, Moschese V, Cancrini C, Finocchi A, Pesce AM, Cagliuso M, Conti V, Granata G, Mitrevski M, Cecere F, Tovo PA, Martino S, Montin D, Nespoli L, Marinoni M, Pellegrini FP, and Cazzola GA.

Subjects
Male, bronchiectasis, X-linked agammaglobulinemia, Comorbidity, Gastroenterology, Immunoglobulin G, 0302 clinical medicine, Agammaglobulinemia, Risk Factors, Immunology and Allergy, Prospective Studies, Child, Prospective cohort study, 0303 health sciences, biology, Incidence, common variable immunodeficiency, Middle Aged, 3. Good health, Treatment Outcome, Italy, Female, Intravenous, Adult, x-linked agammaglobulinemia, medicine.medical_specialty, immunoglobulin replacement, Adolescent, effectiveness, iga, Immunology, Disease-Free Survival, Injections, Databases, 03 medical and health sciences, Immunoglobulin replacement, common variable immunodeficiency, X-linked agammaglobulinemia, bronchiectasis, IgA, effectiveness, Internal medicine, medicine, Humans, Risk factor, Preschool, Factual, Aged, 030304 developmental biology, Settore MED/38 - Pediatria Generale e Specialistica, Bronchiectasis, business.industry, Common variable immunodeficiency, Infant, Pneumonia, X-Linked, medicine.disease, Databases, Factual, Injections, Intravenous, Child, Preschool, Immunoglobulin A, Follow-Up Studies, Common Variable Immunodeficiency, Genes, X-Linked, Genes, biology.protein, Trough level, business, 030215 immunology
Abstract

A 5-years multicenter prospective study on 201 patients with common variable immunodeficiencies and 101 patients with X-linked agammaglobulinemia over a cumulative follow-up period of 1,365 patient-years was conducted to identify prognostic markers and risk factors for associated clinical co-morbidities, the effects of long-term immunoglobulin treatment and the IgG trough level to be maintained over time required to minimise infection risk. Overall, 21% of the patients with common variable immunodeficiencies and 24% of patients with X-linked agammaglobulinemia remained infection free during the study. A reduction of pneumonia episodes has been observed after initiation of Ig replacement. During the observation time, pneumonia incidence remained low and constant over time. Patients with pneumonia did not have significant lower IgG trough levels than patients without pneumonia, with the exception of patients whose IgG trough levels were persistently < 400 mg/dL. In X-linked agammaglobulinemia, the only co-morbidity risk factor identified for pneumonia by the final multivariable model was the presence of bronchiectasis. In common variable immunodeficiencies, our data allowed us to identify a clinical phenotype characterised by a high pneumonia risk: patients with low IgG and IgA levels at diagnosis; patients who had IgA level < 7 mg/dL and who had bronchiectasis. The effect of therapy with immunoglobulins at replacement dosage for non-infectious co-morbidities (autoimmunity, lymphocytic hyperplasia and enteropathy) remains to be established. A unique general protective trough IgG level in antibody deficiency patients will remain undefined because of the major role played by the progression of lung disease in X-linked agammaglobulinemia and in a subset of patients with common variable immunodeficiencies.

Published
2011

25. Clinical features, long-term follow-up and outcome of a large cohort of patients with chronic granulomatous disease: an Italian multicelter study

Author

Martire, B, Rondelli, R, Soresina, A, Pignata, C, Broccoletti, T, Finocchi, A, Rossi, P, Gattorno, M, Rabusin, M, Azzari, C, Dellepiane, Rm, Pietrogrande, Mc, Trizzino, A, Di Bartolomeo, P, Martino, Silvana, Carpino, L, Cossu, F, Locatelli, F, Maccario, R, Pierani, P, Putti, Mc, Stabile, A, Notarangelo, Ld, Ugazio, Ag, Plebani, A, De Mattia, D, Ipinet, Martire, B, Rondelli, R, Soresina, A, Pignata, Claudio, Broccoletti, Teresa, Finocchi, A, Rossi, P, Gattorno, M, Rabusin, M, Azzari, C, Dellepiane, R. M., Pietrogrande, M. C., Trizzino, A, DI BARTOLOMEO, P, Martino, S, Carpino, L, Cossu, F, Locatelli, F, Maccario, R, Pievani, P, Putti, M. C., Stabile, A, Notarangelo, L. D., Ugazio, A. G., Plebani, A, and DE MATTIA, D.

Subjects
Male, absence of side effects, Granulomatous Disease, Chronic, cause of death, Chronic granulomatous disease, Anti-Infective Agents, amphotericin B, cotrimoxazole, fluconazole, gamma interferon, itraconazole, voriconazole, adolescent, adult, article, brain abscess, child, chronic granulomatous disease, clinical feature, clinical trial, cohort analysis, conjunctivitis, controlled clinical trial, controlled study, dermatitis, disease course, drug efficacy, drug withdrawal, enteritis, enterocolitis, fatigue, female, fever, follow up, function test, granulocyte function, headache, human, incidence, liver abscess, long term care, lung abscess, lymphadenitis, male, multicenter study, myalgia, osteomyelitis, otitis, pneumonia, priority journal, prophylaxis, prospective study, rash, septicemia, single drug dose, sinusitis, skin abscess, survival rate, treatment duration, Antifungal Agents, Antiviral Agents, Bacterial Infections, Child, Child, Preschool, Cohort Studies, Female, Follow-Up Studies, Humans, Infant, Interferon Type II, Italy, Itraconazole, Kaplan-Meiers Estimate, Retrospective Studies, Treatment Outcome, Trimethoprim-Sulfamethoxazole Combination, Immunopathology, Chronic, Interferon gamma (IFNγ), Cohort, clinica features, Cohort study, medicine.medical_specialty, Cotrimoxazole (CTX), Immunology, Interferon-gamma, Settore MED/38 - Pediatria Generale e Specialistica, Follow up, Itraconazole (ITRA), medicine.disease, Kaplan-Meier Estimate, Skin infection, Immunology and Allergy, Mortality rate, Granulomatous Disease, medicine.drug, Chronic Granulomatous Disease (CGD), Infections, Internal medicine, Trimethoprim, Sulfamethoxazole Drug Combination, medicine, Preschool, business.industry, Retrospective cohort study, Surgery, business
Abstract

A retrospective clinical and immunological survey was conducted in 60 patients with Chronic Granulomatous Disease. A prospective controlled non-randomized study of the efficacy of long-term IFNgamma treatment was carried out. The mean age at the time of diagnosis was 4.4 years; mean duration of follow-up was 10.4 years. Lung and skin infections were the most frequent manifestations both prior to diagnosis and during follow-up. Aspergillus species was the first cause of infection and of death in our cohort. The mortality rate was 13%. Long term prophylaxis with IFNgamma did not significantly change the rate of total infection per patient-year compared to controls (p=0.07). Our data provide clear evidence that protocols of continuing intensive surveillance and monitoring of compliance with anti-infective regimens may significantly improve the quality of life and long-term survival in patients with CGD. No evidence justifying long-term prophylaxis with IFNgamma was obtained.

Published
2008

33. Protocollo AIEOP LNH92 per i linfomi non Hodgkin pediatrici

Author

Pillon, M, Rosolen, Angelo, Putti, Mc, Santoro, N, D’Angelo, P, Izzi, G, Tamaro, P, Lippi, A, Cellinim, Ragusa, L, Favre, C, Arrighini, A, Moleti, Ml, Felici, L, Marcellino, C, Varotto, S, and Zanesco, Luigi

Published
1998

41. Genotypic characterization of common acute lymphoblastic leukemia may improve the phenotypic classification

Author

Foa, R, Migone, Nicola, Fierro, Maria Teresa, Basso, G, Lusso, P, Putti, Mc, Giubellino, Mc, Saitta, M, Miniero, R, and Casorati, G.

Subjects
B-Lymphocytes, Genes, Immunoglobulin, Genotype, Antibodies, Neoplasm, Antibodies, Monoclonal, Cell Differentiation, DNA, Neoplasm, Leukemia, Lymphoid, Neoplasm Proteins, Antigens, Differentiation, B-Lymphocyte, Phenotype, Antigens, Neoplasm, Humans, Immunoglobulin Light Chains, Immunoglobulin Heavy Chains, Alleles
Abstract

The reactivity of five anti-B monoclonal antibodies (McAb)-OKB2 (CD24), B4 (CD19), Leu12 (CD19), BA1 (CD24), B1 (CD20)--as well as the presence of cytoplasmic immunoglobulins (CyIg) were assessed in 100 cases of common acute lymphoblastic leukemia (cALL) at presentation (TdT+, J5 [CD10]+, HLA-Dr+). All cases studied revealed one or more B-cell related markers and a hierarchy in their expression was documented: OKB2 was positive in all cases tested (100%), B4 was expressed in 96.4% of cases, Leu12 in 95.8%, BA1 in 94.9%, B1 in 18.3%, and CyIg in 23%. Further evidence of the B-cell origin of cALL was obtained by molecular analyses at the DNA level which demonstrated the presence of an Ig heavy chain gene rearrangement in all 37 cases assessed, while 37.8% showed a light chain gene reorganization. A genomic subclassification of cALL demonstrated that the majority of cases showed an immature molecular configuration with one (8.1%) or both (54.1%) Ig heavy chain alleles rearranged and a germ-line configuration of the light chain genes; 27% revealed a heavy chain gene involvement and one k allele rearranged. Only four cases (10.8%) showed a more mature configuration with both k alleles rearranged or a gamma chain gene involvement. This study confirms that cALL is characterized by the proliferation of immature B-lineage-committed elements and indicates that the leukemic cells are blocked at different levels of B-differentiation which may be recognized with the use of multiple phenotypic or genotypic B-cell-related markers.

Published
1987

44. Systemic Catheter-Related Venous Thromboembolism in Children: Data From the Italian Registry of Pediatric Thrombosis

Author

Lasagni, D., Nosadini, M., Molinari, A. C., Saracco, P., Pelizza, M. F., Piersigilli, F., Putti, M. C., Gaffuri, M., Giordano, P., Lorenzoni, G., Francavilla, A., Trapani, S., Luciani, M., Suppiej, A., Tufano, A., Tormene, D., Martinato, M., Gregori, D., Sartori, S., Simioni, P., Agostini, M., Bassi, B., Bertoni, E., Casani, A., Farinasso, D., Gallo, E., Gentilomo, C., Grassi, M., Lunetta, F., Magarotto, M., Maschio, F., Palmieri, A., Pettenazzo, A., Sangermani, R., Laverda, A., Lasagni, D, Nosadini, M, Molinari, Ac, Saracco, P, Pelizza, Mf, Piersigilli, F, Putti, Mc, Gaffuri, M, Giordano, P, Lorenzoni, G, Francavilla, A, Trapani, S, Luciani, M, Suppiej, A, Tufano, A, Tormene, D, Martinato, M, Gregori, D, Sartori, S, Simioni, P, Systemic Thromboses Working Group of the Italian Registry of Pediatric Thrombosis (RITI Registro Italiano Trombosi, Infantili)., UCL - SSS/IREC/SLUC - Pôle St.-Luc, and UCL - (SLuc) Service de néonatologie

Subjects
central venous catheter (CVC), pediatric, children, Pediatrics, Perinatology and Child Health, catheter-complications, heparin, registry, thrombosis, Socio-culturale, equipment and supplies
Abstract

BackgroundCentral venous catheters (CVCs) represent one of the main risk factors for venous thrombotic events (VTEs) in children.MethodsWe studied the Italian Registry of Pediatric Thrombosis (RITI) with regard to systemic radiologically confirmed CVC-related VTEs (CVC-VTEs) occurred during 6.5 years in children aged 29 days to 18 years.ResultsA total of 78 CVC-VTEs were included, which occurred in 76 patients (40/76, 53% males). CVC-VTEs comprised 67 non-cardiac VTEs (86%) and 11 intracardiac thrombotic events (ICTEs) (14%); the median age at onset was 19 and 17 months, respectively. The most frequent reason for CVC insertion was supportive therapy. The catheters were placed percutaneously in 85% of cases (56/66) and surgically in the remaining 15% (10/66). Peripherally inserted central catheters (PICCs) were used in 47% (31/66) cases, partially implanted catheters in 42% (28/66), non-implantable catheters in 7% (5/66), and totally implanted catheters (Port) in 2% (1/66). CVC-VTEs were symptomatic in 77% of cases (60/78), while in the remaining 23%, they were incidentally detected on the imaging performed for the underlying condition. The median time between CVC insertion and the onset of symptoms was 10 days in non-cardiac VTEs and 39 days in ICTEs. Doppler ultrasound was the diagnostic technique most frequently used. The venous compartment most frequently affected was the veins of the lower extremities (52%, 43/73). Anti-thrombotic treatment was administered in 96% of CVC-VTEs (75/78). About 2.6% (2/76) of patients experienced a second thrombotic event. At discharge, post-thrombotic syndrome was reported in 13.5% (5/37) events with available data, CVC replacement in 10.8% (4/47), and ischemic necrosis with toe finger amputation in 2.7% (1/37). Three patients died due to an underlying condition; no CVC-VTE-related deaths were reported.ConclusionsWe have carried out a registry-based study on CVC-VTEs in the children in Italy, providing the data that may help improve the detection and management of this CVC-related complication.

Published
2022

45. Access to emergency departments for acute events and identification of sickle cell disease in refugees

Author

Filippo Mazzi, Rossellina Rosso, Paolo Rigano, Giovanna Graziadei, Giovanna Russo, Claudio Pulvirenti, Monica Rizzi, Federico Bonetti, Vincenzo Spadola, Gian Luca Forni, Valeria Pinto, Maria Caterina Putti, Giovanbattista Ruffo, Barbara Gianesin, Caterina Lux, Roberto Lisi, Maddalena Casale, Lucia De Franceschi, Frédéric B. Piel, De Franceschi, L, Lux, C, Piel, Fb, Gianesin, B, Bonetti, F, Casale, M, Graziadei, G, Lisi, R, Pinto, V, Putti, Mc, Rigano, P, Rosso, R, Russo, G, Spadola, V, Pulvirenti, C, Rizzi, M, Mazzi, F, Ruffo, G, and Forni, Gl.

Subjects
Male, Sickle cell disease, refugees, emergency, anemia, screening, Pilot Projects, Disease, Biochemistry, Health Services Accessibility, sickle cell disease, emergency department, acute vaso-occlusive crisis, Epidemiology, EPIDEMIOLOGY, Mass Screening, Child, 1102 Cardiorespiratory Medicine and Haematology, RISK, Refugees, Emergency Service, emergency, Anemia, Hematology, Sickle cell anemia, Sickle Cell, Italy, Child, Preschool, Female, Medical emergency, Emergency Service, Hospital, Life Sciences & Biomedicine, medicine.medical_specialty, emergency department, Adolescent, Refugee, Immunology, Anemia, Sickle Cell, HEMOGLOBIN DISORDERS, Young Adult, Hospital, acute vaso-occlusive crisis, medicine, Anemia sickle-cell, Humans, Preschool, Mass screening, Science & Technology, business.industry, screening, Infant, 1103 Clinical Sciences, Cell Biology, Emergency department, medicine.disease, Hemoglobin disorders, IMMIGRATION, 1114 Paediatrics and Reproductive Medicine, sickle cell disease, business
Published
2019

46. Childhood high-risk acute lymphoblastic leukemia in first remission: results after chemotherapy or transplant from the AIEOP ALL 2000 study

Author

Fiorina Casale, Elena Barisone, Gaetano La Barba, Anna Maria Testi, Daniela Silvestri, Maurizio Aricò, Concetta Micalizzi, Paolo Tamaro, Giuseppe Basso, Rosanna Parasole, Lucia Dora Notarangelo, Carmelo Rizzari, Maria Grazia Valsecchi, Ottavio Ziino, Valentino Conter, Antonella Colombini, Maria Caterina Putti, Marco Zecca, Luca Lo Nigro, Franco Locatelli, Giuseppe Masera, Andrea Biondi, Nicola Santoro, Gabriella Casazza, Andrea Pession, Conter, V, Valsecchi, M, Parasole, R, Putti, M, Locatelli, F, Barisone, E, Lo Nigro, L, Santoro, N, Aricò, M, Ziino, O, Pession, A, Testi, A, Micalizzi, C, Casale, F, Zecca, M, Casazza, G, Tamaro, P, La Barba, G, Notarangelo, L, Silvestri, D, Colombini, A, Rizzari, C, Biondi, A, Masera, G, Basso, G, Valsecchi, Mg, Putti, Mc, Testi, Am, Tamaro, Paolo, Notarangelo, Ld, Basso, G., Conter, V1, and Casale, Fiorina

Subjects
Male, medicine.medical_specialty, Neoplasm, Residual, Adolescent, medicine.medical_treatment, Immunology, Chromosomal translocation, high risk, acute lymphoblastic leukemia, Hematopoietic stem cell transplantation, Biochemistry, Gastroenterology, Antineoplastic Combined Chemotherapy Protocols, Child, Child, Preschool, Combined Modality Therapy, Female, Hematopoietic Stem Cell Transplantation, Humans, Infant, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Radiotherapy, Remission Induction, Treatment Outcome, Hematology, Cell Biology, Prednisone, hemic and lymphatic diseases, Internal medicine, Medicine, Neoplasm, Preschool, Chemotherapy, Antineoplastic Combined Chemotherapy Protocol, business.industry, medicine.disease, Minimal residual disease, Surgery, Clinical trial, Radiation therapy, N/A, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, Residual, business, Human, medicine.drug
Abstract

The outcome of high-risk (HR) Acute Lymphoblastic Leukemia (ALL) patients enrolled in AIEOP-BFM ALL 2000 study (NCT00613457) in Italy is described. Overall, 1999 Philadelphia negative ALL patients entered the study. HR criteria were: minimal residual disease (MRD) levels ≥10-3 at day 78 (HR-MRD), no complete remission (no-CR) at day 33, t(4;11) translocation, Prednisone Poor Response (PPR). Treatment (2 years) included protocol I, 3 polychemotherapy blocks, delayed intensification (protocol IIx2 or IIIx3), cranial radiotherapy, maintenance. 312 HR patients (15.6% of the total) had 5-year event-free survival (EFS) and overall survival (OS) of 58.9%(SE 2.8) and 68.9%(2.6). In hierarchical order, EFS was 45.9%(4.4) in 132 HR-MRD patients, 41.2%(11.9) in 17 patients no-CR at day 33, 36.4%(14.5) in 11 patients with t(4;11), 74.0%(3.6) in 152 HR patients only for PPR. No statistically significant difference was found for disease-free survival (DFS) in patients with very high risk features (HR-MRD, no-CR at day 33, t(4;11) translocation), given HSCT (n=66) or chemotherapy only (n=88), after adjusting for waiting time to hematopoietic stem cell transplantation (HSCT) (5.7 months). Patients at HR only for PPR have favorable outcome. High risk MRD is associated with poor outcome despite intensive treatment and/or HSCT and may qualify for innovative therapies. The study is registered at the US National Institutes of Health website http://clinicaltrials.gov as "Combination Chemotherapy Based on Risk of Relapse in Treating Young Patients With Acute Lymphoblastic Leukaemia" with the protocol identification number NCT00613457.

Published
2014

47. Multiparametric Cardiac Magnetic Resonance Survey in Children With Thalassemia Major: A Multicenter Study

Author

Maria Rita Gamberini, Gianluca Valeri, Tommaso Casini, Giovanni Palazzi, Domenico Giuseppe D'Ascola, Cristina Salvatori, Maria Giovanna Neri, Patrizia Toia, Liana Cuccia, Maddalena Casale, Aurelio Maggio, Antonella Meloni, Massimo Midiri, Antonella Quarta, Vincenzo Caruso, Vincenzo Positano, Maria Caterina Putti, Alessia Pepe, Lorella Pitrolo, Aldo Filosa, Casale, Maddalena, Meloni, A, Filosa, A, Cuccia, L, Caruso, V, Palazzi, G, Gamberini, Mr, Pitrolo, L, Putti, Mc, D'Ascola, Dg, Casini, T, Quarta, A, Maggio, A, Neri, Mg, Positano, V, Salvatori, C, Toia, P, Valeri, G, Midiri, M, and Pepe, A.

Subjects
Gadolinium DTPA, Male, medicine.medical_specialty, Liver Iron Concentration, Hemosiderosis, Adolescent, Iron, Thalassemia, Contrast Media, Magnetic Resonance Imaging, Cine, Management of thalassemia, Iron Chelating Agents, Ventricular Function, Left, Medication Adherence, Predictive Value of Tests, Internal medicine, medicine, Humans, Radiology, Nuclear Medicine and imaging, Child, Ventricular remodeling, Retrospective Studies, Ventricular Remodeling, medicine.diagnostic_test, business.industry, Myocardium, beta-Thalassemia, Age Factors, Beta thalassemia, Magnetic resonance imaging, medicine.disease, Fibrosis, Italy, Liver, Ventricular Function, Right, Cardiology, Female, Myocardial fibrosis, Cardiomyopathies, Cardiology and Cardiovascular Medicine, Nuclear medicine, business
Abstract

Background— Cardiovascular magnetic resonance (CMR) plays a key role in the management of thalassemia major patients, but few data are available in pediatric population. This study aims at a retrospective multiparametric CMR assessment of myocardial iron overload, function, and fibrosis in a cohort of pediatric thalassemia major patients. Methods and Results— We studied 107 pediatric thalassemia major patients (61 boys, median age 14.4 years). Myocardial and liver iron overload were measured by T2* multiecho technique. Atrial dimensions and biventricular function were quantified by cine images. Late gadolinium enhancement images were acquired to detect myocardial fibrosis. All scans were performed without sedation. The 21.4% of the patients showed a significant myocardial iron overload correlated with lower compliance to chelation therapy ( P P =0.001 and P P P =0.022) and negative cardiac remodeling indexes. A pathological magnetic resonance imaging liver iron concentration was found in the 77.6% of the patients. Conclusions— Cardiac damage detectable by a multiparametric CMR approach can occur early in thalassemia major patients. So, the first T2* CMR assessment should be performed as early as feasible without sedation to tailor the chelation treatment. Conversely, late gadolinium enhancement CMR should be postponed in the teenager age.

Published
2015

48. Infants with acute myeloid leukemia treated according to the Associazione Italiana di Ematologia e Oncologia Pediatrica 2002/01 protocol have an outcome comparable to that of older children

Author

Paolo Pierani, Andrea Pession, Riccardo Masetti, Giuseppe Basso, Marco Togni, Franca Fagioli, Angela Mastronuzzi, Franco Locatelli, Roberto Rondelli, Martina Pigazzi, Maria Caterina Putti, Giuseppe Menna, Masetti R, Rondelli R, Fagioli F, Mastronuzzi A, Pierani P, Togni M, Menna G, Pigazzi M, Putti MC, Basso G, Pession A, and Locatelli F

Subjects
Myeloid, Male, medicine.medical_specialty, Pediatrics, medicine.medical_treatment, Treatment outcome, Intensive chemotherapy, Hematopoietic stem cell transplantation, Acute, hemic and lymphatic diseases, Internal medicine, Medical, Medicine, Humans, Online Only Articles, Acute myeloid leukemia, Infants, Child, Female, Follow-Up Studies, Hematopoietic Stem Cell Transplantation, Induction Chemotherapy, Infant, Infant, Newborn, Italy, Leukemia, Myeloid, Acute, Societies, Medical, Survival Rate, Treatment Outcome, Hematology, Survival rate, Leukemia, business.industry, Follow up studies, Induction chemotherapy, Myeloid leukemia, Newborn, infants, acute myeloid leukemia, intensive chemotherapy, hematopoietic stem cell transplantation, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, Immunology, business, Societies
Abstract

Children under the age of one year (i.e. infants) with acute myeloid leukemia (AML) represent a distinct subgroup of patients with peculiar clinical and biological characteristics.[1][1] As compared to older children, a higher prevalence of unfavorable clinical and cytogenetic/molecular features has

Published
2014

49. MLL partner genes drive distinct gene expression profiles and genomic alterations in pediatric acute myeloid leukemia: an AIEOP study

Author

Martina Pigazzi, S. Gelain, Andrea Zangrando, Carmelo Rizzari, Marco Giordan, Andrea Pession, Silvia Bresolin, A Di Meglio, Luca Trentin, Emma Baron, Alessandra Beghin, Giuseppe Basso, Anna Leszl, M. C. Putti, G te Kronnie, Riccardo Masetti, Barbara Buldini, Francesco Locatelli, Pigazzi M, Masetti R, Bresolin S, Beghin A, Di Meglio A, Gelain S, Trentin L, Baron E, Giordan M, Zangrando A, Buldini B, Leszl A, Putti MC, Rizzari C, Locatelli F, Pession A, Te Kronnie G, and Basso G

Subjects
Male, Cancer Research, medicine.medical_specialty, Kinesins, Myosins, Translocation, Genetic, Fusion gene, hemic and lymphatic diseases, Internal medicine, Gene expression, medicine, Humans, granulocytic leukemia, Child, neoplasms, Gene, Genetics, Hematology, business.industry, Chromosomes, Human, Pair 11, Gene Expression Profiling, pediatric acute myeloid leukemia, Genomics, Histone-Lysine N-Methyltransferase, medicine.disease, Gene expression profiling, Haematopoiesis, Leukemia, Leukemia, Myeloid, Acute, Oncology, Myeloid-Lymphoid Leukemia Protein, Chromosomes, Human, Pair 6, Female, business
Abstract

MLL partner genes drive distinct gene expression profiles and genomic alterations in pediatric acute myeloid leukemia: an AIEOP study

Published
2011

50. Rationale for a Pediatric-Inspired Approach in the Adolescent and Young Adult Population with Acute Lymphoblastic Leukemia, with a Focus on Asparaginase Treatment

Author

Sara Casagranda, Ilaria Iacobucci, Maria Chiara Abbenante, Maria Caterina Putti, Chiara Sartor, Carmelo Rizzari, Giulia Maria Ferrari, Giovanni Martinelli, Cristina Papayannidis, Antonella Colombini, Rizzari, C, Putti, Mc, Colombini, A, Casagranda, S, Ferrari, Gm, Papayannidis, C, Iacobucci, I, Abbenante, Mc, Sartor, C, Martinelli, G, Putti, M, Ferrari, G, and Abbenante, M

Subjects
Pediatrics, medicine.medical_specialty, Asparaginase, medicine.medical_treatment, Lymphoblastic Leukemia, Population, Review, acute lymphoblastic leukemia, chemistry.chemical_compound, medicine, Young adult, education, Childhood Acute Lymphoblastic Leukemia, Chemotherapy, education.field_of_study, lcsh:RC633-647.5, business.industry, asparaginase, acute lymphoblastic leukemia, adolescent, young adult, Incidence (epidemiology), acute lymphoblastic leukemia, pediatric, asparaginase, lcsh:Diseases of the blood and blood-forming organs, Hematology, asparaginase, humanities, chemistry, adolescent, young adult, Methotrexate, business, medicine.drug
Abstract

In the last two decades great improvements have been made in the treatment of childhood acute lymphoblastic leukemia, with 5-year overall survival rates currently approaching almost 90%. In comparison, results reported in adolescents and young adults (AYAs) are relatively poor. In adults, results have improved, but are still lagging behind those obtained in children. Possible reasons for this different pattern of results include an increased incidence of unfavorable and a decreased incidence of favorable cytogenetic abnormalities in AYAs compared with children. Furthermore, in AYAs less intensive treatments (especially lower cumulative doses of drugs such as asparaginase, corticosteroids and methotrexate) and longer gaps between courses of chemotherapy are planned compared to those in children. However, although favorable results obtained in AYAs receiving pediatric protocols have been consistently reported in several international collaborative trials, physicians must also be aware of the specific toxicity pattern associated with increased success in AYAs, since an excess of toxicity may compromise overall treatment schedule intensity. Cooperative efforts between pediatric and adult hematologists in designing specific protocols for AYAs are warranted.

Published
2014

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