@article {8286, title = {The prognostic value of biological markers in paediatric Hodgkin lymphoma.}, journal = {Eur J Cancer}, volume = {52}, year = {2016}, month = {2016 Jan}, pages = {33-40}, abstract = {

BACKGROUND: Many biological and inflammatory markers have been proposed as having a prognostic value at diagnosis of Hodgkin lymphoma (HL), but very few have been validated in paediatric patients. We explored the significance of these markers in a large population of 769 affected children.

PATIENTS AND METHODS: By using the database of patients enrolled in A.I.E.O.P. (Associazione Italiana di Emato-Oncologia Pediatrica) trial LH2004 for paediatric HL, we identified 769 consecutive patients treated with curative intent from 1st June 2004 to 1st April 2014 with ABVD (doxorubicin, bleomycin, vinblastine, and dacarbazine), or hybrid COPP/ABV (cyclophosphamide, vincristine, prednisone, procarbazine, doxorubicin, bleomycin and vinblastine) regimens.

RESULTS: On multivariate analysis with categorical forms, the 5-year freedom from progression survival was significantly lower in patients with stage IV or elevated value of platelets, eosinophils and ferritin at diagnosis. Furthermore, stage IV and eosinophils seem to maintain their predictive value independently of interim (after IV cycles of chemotherapy) positron emission tomography.

CONCLUSION: Using the combination of four simple markers such as stage IV and elevated levels of platelets, ferritin and eosinophils, it is possible to classify the patients into subgroups with very different outcomes.

}, keywords = {Adolescent, Age Factors, Antineoplastic Combined Chemotherapy Protocols, Biomarkers, Tumor, Blood Platelets, Child, Child, Preschool, Databases, Factual, Disease Progression, Disease-Free Survival, Eosinophils, Female, Ferritins, Hodgkin Disease, Humans, Infant, Infant, Newborn, Italy, Kaplan-Meier Estimate, Leukocyte Count, Male, Multivariate Analysis, Neoplasm Staging, Platelet Count, Predictive Value of Tests, Proportional Hazards Models, Retrospective Studies, Risk Factors, Time Factors, Treatment Outcome}, issn = {1879-0852}, doi = {10.1016/j.ejca.2015.09.003}, author = {Farruggia, Piero and Puccio, Giuseppe and Sala, Alessandra and Todesco, Alessandra and Buffardi, Salvatore and Garaventa, Alberto and Bottigliero, Gaetano and Bianchi, Maurizio and Zecca, Marco and Locatelli, Franco and Pession, Andrea and Pillon, Marta and Favre, Claudio and D{\textquoteright}Amico, Salvatore and Provenzi, Massimo and Trizzino, Angela and Zanazzo, Giulio Andrea and Sau, Antonella and Santoro, Nicola and Murgia, Giulio and Casini, Tommaso and Mascarin, Maurizio and Burnelli, Roberta} } @article {3636, title = {Autoimmune hematological diseases after allogeneic hematopoietic stem cell transplantation in children: an Italian multicenter experience.}, journal = {Biol Blood Marrow Transplant}, volume = {20}, year = {2014}, month = {2014 Feb}, pages = {272-8}, abstract = {

Autoimmune hematological diseases (AHDs) may occur after allogeneic hematopoietic stem cell transplantation (HSCT), but reports on these complications in large cohorts of pediatric patients are lacking. Between 1998 and 2011, 1574 consecutive children underwent allogeneic HSCT in 9 Italian centers. Thirty-three children (2.1\%) developed AHDs: 15 autoimmune hemolytic anemia (45\%), 10 immune thrombocytopenia (30\%), 5 Evans{\textquoteright} syndrome (15\%), 2 pure red cell aplasia (6\%), and 1 immune neutropenia (3\%). The 10-year cumulative incidence of AHDs was 2.5\% (95\% confidence interval, 1.7 to 3.6). In a multivariate analysis, the use of alternative donor and nonmalignant disease was statistically associated with AHDs. Most patients with AHDs (64\%) did not respond to steroids. Sustained complete remission was achieved in 87\% of cases with the anti-CD20 monoclonal antibody (rituximab). Four patients (9\%) (1 autoimmune hemolytic anemia, 1 Evans{\textquoteright} syndrome, 2 immune thrombocytopenia) died at a median of 87~days after AHD diagnosis as a direct or indirect consequence of their disorder. Our data suggest that AHDs are a relatively rare complication occurring after HSCT that usually respond to treatment with rituximab.

}, keywords = {Child, Child, Preschool, Female, Hematologic Diseases, Hematopoietic Stem Cell Transplantation, Humans, Italy, Male, Remission Induction, Risk Factors, Transplantation Conditioning}, issn = {1523-6536}, doi = {10.1016/j.bbmt.2013.11.014}, author = {Faraci, Maura and Zecca, Marco and Pillon, Marta and Rovelli, Attilio and Menconi, Maria Cristina and Ripaldi, Mimmo and Fagioli, Franca and Rabusin, Marco and Ziino, Ottavio and Lanino, Edoardo and Locatelli, Franco and Daikeler, Thomas and Prete, Arcangelo} } @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 = {

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.

}, 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} } @article {1795, title = {Allogeneic hematopoietic stem cell transplantation for Philadelphia-positive acute lymphoblastic leukemia in children and adolescents: a retrospective multicenter study of the Italian Association of Pediatric Hematology and Oncology (AIEOP).}, journal = {Biol Blood Marrow Transplant}, volume = {18}, year = {2012}, month = {2012 Jun}, pages = {852-60}, abstract = {

Philadelphia-positive acute lymphoblastic leukemia (Ph+ ALL) still represents a major challenge. We report the experience of the Italian Association of Pediatric Hematology and Oncology (AIEOP) with allogeneic hematopoietic stem cell transplantation (HSCT) in children with Ph+ ALL from 1990 to 2008. Sixty-nine patients received HSCT from either a related (37, 54\%) or an unrelated (32, 46\%) donor. Twenty-five patients (36\%) underwent transplantation before 2000 and 44 (64\%) after 2000. Twenty-three patients (33\%) received Imatinib mesylate treatment before HSCT and seven (10\%) after HSCT. After a median follow-up of 56 months, the overall survival (OS) probability was 51\% (95\% confidence interval [CI], 38-63), the leukemia-free survival (LFS) was 47\% (95\% CI, 34-59), transplantation-related mortality (TRM) was 17\% (95\% CI, 10-30), and relapse incidence (RI) was 36\% (95\% CI, 26-50). Transplantation in first complete remission, female gender, and lower WBC count at diagnosis were associated with a better LFS in both univariate and multivariate analyses. Patients with p210 transcript had a trend for a worse prognosis compared with those who had the p190 transcript. Our series confirms the role of HSCT in the eradication of Ph+ ALL. Early HSCT is recommended once morphologic remission is obtained.

}, keywords = {Adolescent, Antineoplastic Combined Chemotherapy Protocols, Benzamides, Child, Child, Preschool, Disease-Free Survival, Drug Administration Schedule, Female, Fusion Proteins, bcr-abl, Graft vs Host Disease, Hematopoietic Stem Cell Transplantation, Humans, Infant, Italy, Male, Philadelphia Chromosome, Piperazines, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Pyrimidines, Remission Induction, Retrospective Studies, Secondary Prevention, Transplantation, Homologous, Young Adult}, issn = {1523-6536}, doi = {10.1016/j.bbmt.2011.10.015}, author = {Fagioli, Franca and Zecca, Marco and Rognoni, Carla and Lanino, Edoardo and Balduzzi, Adriana and Berger, Massimo and Messina, Chiara and Favre, Claudio and Rabusin, Marco and Lo Nigro, Luca and Masetti, Riccardo and Prete, Arcangelo and Locatelli, Franco} } @article {1822, title = {Foreign children with cancer in Italy.}, journal = {Ital J Pediatr}, volume = {37}, year = {2011}, month = {2011}, pages = {44}, abstract = {

BACKGROUND: There has been a noticeable annual increase in the number of children coming to Italy for medical treatment, just like it has happened in the rest of the European Union. In Italy, the assistance to children suffering from cancer is assured by the current network of 54 centres members of the Italian Association of Paediatric Haematology and Oncology (AIEOP), which has kept records of all demographic and clinical data in the database of Mod.1.01 Registry since 1989.

METHODS: We used the information stored in the already mentioned database to assess the impact of immigration of foreign children with cancer on centres{\textquoteright} activity, with the scope of drawing a map of the assistance to these cases.

RESULTS: Out of 14,738 cases recorded by all centres in the period from 1999 to 2008, 92.2\% were born and resident in Italy, 4.1\% (608) were born abroad and living abroad and 3.7\% (538) were born abroad and living in Italy. Foreign children cases have increased over the years from 2.5\% in 1999 to. 8.1\% in 2008.Most immigrant children came from Europe (65.7\%), whereas patients who came from America, Asia and Oceania amounted to 13.2\%, 10.1\%, 0.2\%, respectively. The immigrant survival rate was lower compared to that of children who were born in Italy. This is especially true for acute lymphoblastic leukaemia patients entered an AIEOP protocol, who showed a 10-years survival rate of 71.0\% vs. 80.7\% (p < 0.001) for immigrants and patients born in Italy, respectively.

CONCLUSIONS: Children and adolescents are an increasingly important part of the immigration phenomenon, which occurs in many parts of the world. In Italy the vast majority of children affected by malignancies are treated in AIEOP centres. Since immigrant children are predominantly treated in northern Italy, these centres have developed a special expertise in treating immigrant patients, which is certainly very useful for the entire AIEOP network.

}, keywords = {Adolescent, Africa, Asia, Child, Child, Preschool, Databases, Factual, Emigrants and Immigrants, Ethnic Groups, Europe, Eastern, European Union, Female, Humans, Incidence, Infant, Infant, Newborn, Italy, Male, Neoplasms, North America, Oceania, Prevalence, Retrospective Studies, South America, Survival Rate}, issn = {1824-7288}, doi = {10.1186/1824-7288-37-44}, author = {Rondelli, Roberto and Dini, Giorgio and De Rosa, Marisa and Quarello, Paola and Bisogno, Gianni and Aric{\`o}, Maurizio and Vasconcelos, Carivaldo and Tamaro, Paolo and Casazza, Gabriella and Zecca, Marco and De Laurentis, Clementina and Porta, Fulvio and Pession, Andrea} } @article {1736, title = {Mutations in ANKRD26 are responsible for a frequent form of inherited thrombocytopenia: analysis of 78 patients from 21 families.}, journal = {Blood}, volume = {117}, year = {2011}, month = {2011 Jun 16}, pages = {6673-80}, abstract = {

Until recently, thrombocytopenia 2 (THC2) was considered an exceedingly rare form of autosomal dominant thrombocytopenia and only 2 families were known. However, we recently identified mutations in the 5{\textquoteright}-untranslated region of the ANKRD26 gene in 9 THC2 families. Here we report on 12 additional pedigrees with ANKRD26 mutations, 6 of which are new. Because THC2 affected 21 of the 210 families in our database, it has to be considered one of the less rare forms of inherited thrombocytopenia. Analysis of all 21 families with ANKRD26 mutations identified to date revealed that thrombocytopenia and bleeding tendency were usually mild. Nearly all patients had no platelet macrocytosis, and this characteristic distinguishes THC2 from most other forms of inherited thrombocytopenia. In the majority of cases, platelets were deficient in glycoprotein Ia and α-granules, whereas in vitro platelet aggregation was normal. Bone marrow examination and serum thrombopoietin levels suggested that thrombocytopenia was derived from dysmegakaryopoiesis. Unexplained high values of hemoglobin and leukocytes were observed in a few cases. An unexpected finding that warrants further investigation was a high incidence of acute leukemia. Given the scarcity of distinctive characteristics, the ANKRD26-related thrombocytopenia has to be taken into consideration in the differential diagnosis of isolated thrombocytopenias.

}, keywords = {Adolescent, Adult, Aged, Aged, 80 and over, Ankyrin Repeat, Child, Cohort Studies, Family, Female, Gene Frequency, Humans, Inheritance Patterns, Male, Middle Aged, Mutation, Pedigree, Thrombocytopenia, Transcription Factors, Young Adult}, issn = {1528-0020}, doi = {10.1182/blood-2011-02-336537}, author = {Noris, Patrizia and Perrotta, Silverio and Seri, Marco and Pecci, Alessandro and Gnan, Chiara and Loffredo, Giuseppe and Pujol-Moix, N{\'u}ria and Zecca, Marco and Scognamiglio, Francesca and De Rocco, Daniela and Punzo, Francesca and Melazzini, Federica and Scianguetta, Saverio and Casale, Maddalena and Marconi, Caterina and Pippucci, Tommaso and Amendola, Giovanni and Notarangelo, Lucia D and Klersy, Catherine and Civaschi, Elisa and Balduini, Carlo L and Savoia, Anna} }