@article {7760,
	title = {Are all cases of paediatric essential thrombocythaemia really myeloproliferative neoplasms? Analysis of a large cohort.},
	journal = {Br J Haematol},
	volume = {169},
	year = {2015},
	month = {2015 May},
	pages = {584-9},
	abstract = {<p>Sporadic essential thrombocythaemia (ET) is rare in paediatrics, and the diagnostic and clinical approach to paediatric cases cannot be simply copied from experience with adults. Here, we assessed 89 children with a clinical diagnosis of ET and found that 23 patients (25{\textperiodcentered}8\%) had a clonal disease. The JAK2 V617F mutation was identified in 14 children, 1 child had the MPL W515L mutation, and 6 had CALR mutations. The monoclonal X-chromosome inactivation pattern was seen in six patients (two with JAK2 V617F and two with CALR mutations). The other 66 patients (74{\textperiodcentered}2\%) had persistent thrombocytosis with no clonality. There were no clinical or haematological differences between the clonal and non-clonal patients. The relative proportion of ET-specific mutations in the clonal children was much the same as in adults. The higher prevalence of non-clonal cases suggests that some patients may not have myeloproliferative neoplasms, with significant implications for their treatment.</p>},
	keywords = {Adolescent, Adult, Amino Acid Substitution, Child, Child, Preschool, Cohort Studies, Female, Hematologic Neoplasms, Humans, Infant, Janus Kinase 2, Male, Mutation, Missense, Neoplasm Proteins, Thrombocythemia, Essential},
	issn = {1365-2141},
	doi = {10.1111/bjh.13329},
	author = {Randi, Maria L and Geranio, Giulia and Bertozzi, Irene and Micalizzi, Concetta and Ramenghi, Ugo and Tucci, Fabio and Notarangelo, Lucia D and Ladogana, Saverio and Menna, Giuseppe and Giordano, Paola and Consarino, Caterina and Farruggia, Piero and Zanazzo, Giulio A and Fiori, Giovanni M and Burnelli, Roberta and Russo, Giovanna and Jankovich, Momcilo and Peroni, Edoardo and Duner, Elena and Basso, Giuseppe and Fabris, Fabrizio and Putti, Maria C}
}
@article {3601,
	title = {Childhood high-risk acute lymphoblastic leukemia in first remission: results after chemotherapy or transplant from the AIEOP ALL 2000 study.},
	journal = {Blood},
	volume = {123},
	year = {2014},
	month = {2014 Mar 6},
	pages = {1470-8},
	abstract = {<p>The outcome of high-risk (HR) acute lymphoblastic leukemia patients enrolled in the AIEOP-BFM ALL 2000 study in Italy is described. HR criteria were minimal residual disease (MRD) levels >=10(-3) at day 78 (MRD-HR), no complete remission (CR) at day 33, t(4;11) translocation, and prednisone poor response (PPR). Treatment (2 years) included protocol I, 3 polychemotherapy blocks, delayed intensification (protocol IIx2 or IIIx3), cranial radiotherapy, and maintenance. A total of 312 HR patients had a 5-year event-free survival (EFS) of 58.9\% (standard error [SE] = 2.8) and an overall survival of 68.9\% (SE = 2.6). In hierarchical order, EFS was 45.9\% (4.4) in 132 MRD-HR patients, 41.2\% (11.9) in 17 patients with no CR at day 33, 36.4\% (14.5) in 11 patients with t(4;11), and 74.0\% (3.6) in 152 HR patients only for PPR. No statistically significant difference was found for disease-free survival in patients with very HR features [MRD-HR, no CR at day 33, t(4;11) translocation], given hematopoietic stem cell transplantation (HSCT) (n = 66) or chemotherapy only (n = 88), after adjusting for waiting time to HSCT (5.7 months). Patients at HR only for PPR have a favorable outcome. MRD-HR is associated with poor outcome despite intensive treatment and/or HSCT and may qualify for innovative therapies. The study was registered at www.clinicaltrials.gov as $\#$NCT00613457.</p>},
	keywords = {Adolescent, Antineoplastic Combined Chemotherapy Protocols, Child, Child, Preschool, Combined Modality Therapy, Female, Hematopoietic Stem Cell Transplantation, Humans, Infant, Male, Neoplasm, Residual, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Radiotherapy, Remission Induction, Treatment Outcome},
	issn = {1528-0020},
	doi = {10.1182/blood-2013-10-532598},
	author = {Conter, Valentino and Valsecchi, Maria Grazia and Parasole, Rosanna and Putti, Maria Caterina and Locatelli, Franco and Barisone, Elena and Lo Nigro, Luca and Santoro, Nicola and Aric{\`o}, Maurizio and Ziino, Ottavio and Pession, Andrea and Testi, Anna Maria and Micalizzi, Concetta and Casale, Fiorina and Zecca, Marco and Casazza, Gabriella and Tamaro, Paolo and La Barba, Gaetano and Notarangelo, Lucia Dora and Silvestri, Daniela and Colombini, Antonella and Rizzari, Carmelo and Biondi, Andrea and Masera, Giuseppe and Basso, Giuseppe}
}
