Improved outcomes of the refined risk-stratification and risk-adapted therapy in children with acute myeloid leukemia: final results of the AIEOP AML 2013 Franco Locatelli, Barbara Buldini, Martina Pigazzi, Carmelo Rizzari, Giuseppe Menna, Franca Fagioli, Manuela Tumino, Luca Lo Nigro, Paolo D’Angelo, Maria Giuseppina Cefalo, Valeria Paganelli, Luisa Strocchio, Marco Becilli, Marco Spinelli, Giovanna Giagnuolo, Francesco Saglio, Nicola Santoro, Concetta Micalizzi, Marco Zecca, Riccardo Masetti, Pietro Merli Journal of Hematology and Oncology, 2026 BACKGROUND: The previous Italian national trial (AIEOP-AML-2002/01) on children with acute myeloid leukemia (AML) achieved a 3-year overall (OS) and event-free survival (EFS) of 72.3% and 59.1%, respectively. In this study (AIEOP-AML-2013), we evaluated if refined patient's stratification in 3 groups and a second induction with randomization could improve outcomes. METHODS: In the new AIEOP-AML-2013, patients were stratified into 3 groups [standard-(SR), intermediate-(IR) and high-risk (HR)] mainly according to genetic criteria and centralized assessment of multiparametric flow-cytometry measurable residual disease (MFC-MRD). The 1st induction course was common for all patients [idarubicin-cytarabine-etoposide (ICE)]. IR and HR patients were then randomized to receive either a 2nd ICE or the fludarabine-cytarabine- liposomal-doxorubicin (FLA-My) scheme as second induction. IR patients with HLA-compatible sibling, and all HR patients were consolidated with allogeneic hematopoietic stem cell transplantation (allo-HSCT), while the remaining IR and SR patients were consolidated with chemotherapy only. Data cut-off was February 1, 2024. RESULTS: From June/2015 to June/2022, 371 patients were enrolled in the trial. Twenty-six children (7%) experienced primary induction failure, while only 3 patients died during the 2 induction courses. The cumulative incidence of 3-year non-relapse mortality in continuous complete remission was 6.8%. The proportion of patients allocated to the SR, IR, and HR groups were 19.5%, 22%, and 58.5%, respectively. Three-year cumulative incidence of relapse was 18.9%. The 3-year probabilities of OS and EFS were 83.9% and 68.5%, respectively, both values being significantly better (p = 0.001) than those of the AIEOP-AML-2002/01 study. The 3-year OS of SR, IR, and HR patients were 97.0%, 84.2%, and 79.4%, respectively, (p = 0.01). The probability of EFS did not differ in IR and HR randomized to receive either a 2nd ICE or the FLA-My scheme course. Levels of MRD after the 1st and 2nd induction course strongly influenced the EFS probability. CONCLUSIONS: A significant improvement in the outcomes of children with de novo AML was obtained with refined risk-stratification and risk-adapted therapy. FLA-My did not offer any advantage over repeating a 2nd ICE as 2nd induction course. TRIAL REGISTRATION: This study was registered in the European Clinical Trials Database (EudraCT 2014-000652-28).
The interplay of inborn errors of immunity with hematology – Report of the joint focus meeting of ESID, EHA, and SIOPE, Vienna, Austria Markus G. Seidel, Michael H. Albert, Andishe Attarbaschi, Siobhan O. Burns, Eleonora Gambineri, Antonis Kattamis, Antonio Marzollo, Maria Ester Bernardo, Fabio Candotti, Carmelo Rizzari Ejc Paediatric Oncology, 2026 The three scientific societies ESID, EHA, and SIOPe jointly organized their first focus meeting entitled "The Interplay of Inborn Errors of Immunity with Hematology," which took place in Vienna, Austria, in November 2025 and was met with great success. The sessions addressed a wide range of topics at the interface of these overlapping fields, such as interpretation and integration of histopathological and genetics results, mechanisms of immune dysregulation and tumor predisposition, current diagnostic and therapeutic measures, and combined novel insights from biological mechanisms with clinical challenges. This brief report highlights and summarizes key aspects of the scientific program.
Is creatine kinase a valid aid in early cardiac muscle damage detection during treatment for childhood acute lymphoblastic leukemia? A case report Grazia Fazio, Jari Intra, Orsola Montini, Laura Rachele Bettini, Giacomo Gotti, Alessandra Sala, Silvia Bungaro, Giovanni Cazzaniga, Carmelo Rizzari, Adriana Balduzzi, Marco Casati Frontiers in Oncology, 2026 Creatine kinase (CK) is an enzyme that plays a pivotal role in various physiological processes, including metabolism and muscle function. This enzyme is found in high concentrations in skeletal muscle and the myocardium, with lower levels present in the brain. Increases in CK values have been observed in muscle damage and neuromuscular disorders. The potential for cardiotoxicity resulting from the administration of chemotherapeutic agents has been identified in subjects affected by hematological or solid tumors. In the present work, we describe the case story of a child diagnosed with acute lymphoblastic leukemia who exhibited two episodes of asymptomatic, markedly elevated CK values, identified during chemotherapy treatment. Our aim was to investigate the potential of CK as a biomarker for the early detection of cardiac muscle damage during therapy.
Encouraging Outcomes in Pediatric Malignancy-Associated Hemophagocytic Lymphohistiocytosis: Results From the Italian HLH Registry Francesco Pegoraro, Irene Ferri, Aurora Chinnici, Linda Beneforti, Irene Trambusti, Simona Gobbi, Rosa Maria Daniele, Rosamaria Mura, Elena Palmisani, Marco Zecca, Giulia Albrici, Alessandra Todesco, Carmela De Fusco, Samuele Naviglio, Veronica Barat, Fabio Timeus, Marta Pillon, Carmelo Rizzari, Annalisa Tondo, Maria Luisa Coniglio, Elena Sieni American Journal of Hematology, 2026 Hemophagocytic lymphohistiocytosis (HLH) is a life-threatening hyperinflammatory syndrome resulting from uncontrolled activation of cytotoxic T cells and macrophages [1]. HLH can arise from inherited genetic defects affecting the cytotoxic pathway (primary HLH, pHLH) or develop as a secondary disorder triggered by infections, autoimmune diseases, or malignancies (secondary HLH, S-HLH) [1-4]. In adults, malignancy-associated HLH (M-HLH) is the most frequent form of S-HLH, typically occurring in the context of hematological malignancies, especially lymphomas, and portends a poor prognosis [5-7]. Conversely, data on pediatric M-HLH is limited, largely derived from small, heterogeneous case series [8, 9], which hinders the recognition and management of this condition. To address this gap, we conducted a multicenter retrospective study within the Italian Association for Pediatric Hematology and Oncology, aiming to define the clinical spectrum, therapeutic strategies, and outcomes of children with M-HLH, compared with a well-characterized cohort of pediatric S-HLH not related to malignancy diagnosed in the same period (control group). We included patients enrolled in the Italian HLH Registry, a nationwide database that prospectively collects HLH cases. We retrospectively analyzed children (< 18 years) diagnosed with HLH between January 2009 and December 2024. We included patients with an associated hematological malignancy and a minimum follow-up of 6 months. Clinical, laboratory, and histopathological data of patients and controls were extracted from the Registry and validated by treating physicians. Variables collected included demographics, underlying malignancies, HLH diagnostic criteria [10], laboratory values, treatments and responses, and survival. Categorical variables were reported as numbers and percentages, whereas continuous variables were reported as medians and interquartile ranges (IQRs). Comparisons between groups were made using the Mann–Whitney U test for continuous variables and the chi-square test for categorical variables. The survival rates were estimated using a Kaplan–Meier model. The log-rank test was used to compare survival between groups. A two-sided p value ≤ 0.05 was considered to indicate statistical significance. The study was conducted in accordance with the Declaration of Helsinki and its later amendments [11]. Out of 76 screened patients, five were excluded due to insufficient follow-up, four because HLH developed after hematopoietic cell-stem transplantation (HSCT), and 12 for lack of clinical data. Finally, 55 children with M-HLH were included (Figure 1A). Thirty-three patients were male (60%), and the median age at HLH diagnosis was 107 months (IQR, 48–150; Table 1). The underlying malignancies included acute lymphoblastic leukemia (ALL, n = 31, 56%), acute myeloid leukemia (AML, n = 9, 16%), non-Hodgkin lymphoma (NHL, n = 7, 13%), Hodgkin lymphoma (n = 4, 7%), and multisystem Langerhans cell histiocytosis (n = 4, 7%) (Figure 1B). The median interval between malignancy diagnosis and HLH onset was 25 days (IQR, 1–131). Half the patients (n = 27) developed HLH during chemotherapy, while in 25 patients HLH developed at malignancy onset, and in the remaining three HLH emerged at relapse or disease progression. At HLH diagnosis, most patients had fever (n = 52, 95%), bi-lineage cytopenia (n = 54, 98%), hyperferritinemia (n = 53, 96%), and triglyceride and/or fibrinogen abnormalities (n = 47, 85%). Splenomegaly (n = 37, 67%), liver involvement (n = 33, 60%), and hemophagocytosis (n = 28, 51%) were less frequently observed (Table 1). Functional studies (i.e., PRF expression and/or CD107a expression) showed abnormalities in nine out of 20 tested patients (45%), while of the 34 patients tested for genetic predisposition to HLH, 10 carried monoallelic variants in pHLH-related genes and one a GATA2 mutation (Figure 1C). When compared with patients with S-HLH, those with M-HLH were significantly older (p < 0.001) and more frequently presented with liver involvement (p = 0.014) and bi-lineage cytopenia (p = 0.010) (Table 1). Thirty-six patients (65%) received HLH-directed treatment, including etoposide in eight, anti-IL1 in 5, and corticosteroids in 23. The overall response rate to HLH-directed therapy was 70% (Figure 1D). HLH reactivation occurred in 5 patients (9%), while 13 underwent HSCT, in most cases due to malignancy (85%). At a median follow-up of 20 months (IQR, 6–71) since HLH diagnosis, 16 patients (32%) died. Causes of death included malignancy in seven patients (44%), HLH in six (38%), and HSCT-related complications in three (18%). The estimated 5-year overall survival probability in children with M-HLH was 76% (95% CI 60–86). No significant differences were observed in survival probability between M-HLH and S-HLH patients (p = 0.190; Figure 1E). Of note, the six patients who died from HLH all had hepatic involvement and rapidly died (median time since diagnosis 29 days) from multiorgan failure despite HLH-directed therapy. In this study, we reported the clinical presentation and outcome of a large cohort of children with M-HLH, as compared with a cohort of S-HLH not related to malignancy. The clinical presentation of M-HLH in children was often non-specific and overlapped with the manifestations of the underlying malignancy. Fever, cytopenias, and hepatic dysfunction are not distinctive, frequently leading to delayed recognition, a limitation to effective intervention. When compared with S-HLH, the only differences of M-HLH patients were older age and more frequent hepatic involvement and bi-lineage cytopenia, findings that might reflect the underlying disease. In contrast, the clinical presentation of pediatric M-HLH diverged from that reported for adult M-HLH, which is usually very severe and related to lymphomas. Conversely, pediatric cases were usually associated with acute leukemia, with frequent FHL-associated genetic variants or functional abnormalities. The potential contribution of these factors to HLH development reinforces the current concept of a disease continuum to which different predisposing and environmental factors converge [1]. Current therapeutic strategies for M-HLH are largely adapted from the HLH-94 and -2004 protocols [12, 13]. Malignancy-directed regimens in children usually contain agents active against HLH—such as corticosteroids, etoposide, and cyclophosphamide—and the HLH component may respond favorably to such protocols [8]. Moreover, rituximab may provide additional benefit in EBV-driven or CD20-positive malignancies [9]. Response to therapy in our cohort was heterogeneous. Patients treated with etoposide seemed to portend poorer outcomes than those receiving corticosteroids alone. Although this is likely to reflect the selection of etoposide for more severe cases rather than a true difference in efficacy, we were not able to assess whether the addition of etoposide to already intensively treated patients contributed to their unfavorable evolution. Of note, novel molecules such as ELA026 have shown promising results in treating adults with M-HLH [14], and their use in the pediatric setting is currently under evaluation (NCT05416307). Survival in our cohort was comparable between M-HLH and S-HLH, but pediatric M-HLH outcomes appeared superior to those reported in adults, who have mortality rates exceeding 70% [5-7]. In addition, deaths in pediatric M-HLH were more related to malignancy or treatment toxicity rather than HLH activity. Fatal HLH cases were characterized by a rapid, fulminant course with hepatic involvement, despite intensive treatment. Recently, in adults with M-HLH, the OHI score was shown to identify patients at high mortality risk [15], but we were not able to assess the reproducibility of the score in children due to insufficient data on soluble CD25. In conclusion, M-HLH in children has a slightly distinct presentation from S-HLH and a more favorable outcome as compared to its adult counterpart. Implementation of diagnostic tests and individualized management, also including novel agents, could contribute to improving care in these patients. F.P., M.L.C., and E.S. conceived the study. F.P., I.F., and E.S. drafted the manuscript. F.P., S.G., R.M.D., R.M., E.P., M.Z., G.A., A.T., C.D.F., S.N., V.B., F.T., M.P., C.R., and E.S. followed the patients. A.C., L.B., and M.L.C. performed the cytometry and molecular analyses. F.P. performed the statistical analysis. All authors critically reviewed the manuscript and approved it in its final version. The authors have nothing to report. The authors declare no conflicts of interest. The data that support the findings of this study are available from the corresponding author upon reasonable request.
INTERIM RESULTS OF THE QUESTIONNAIRE ON PREDISPOSITION TO ACUTE LYMPHOBLASTIC LEUKEMIA IN THE AIEOP-BFM ALL 2017 PROTOCOL Giovanni Cazzaniga Haematologica, 2026 About 8–10% of pediatric cancers are associated with pathogenic variants in cancer predisposition genes. In acute lymphoblastic leukemia (ALL), suspicion of predisposition arises from a family history of early-onset tumors, prior malignancies, comorbidities, or known genetic syndromes.Children enrolled in the AIEOP-BFM ALL 2017 protocol completed a short questionnaire (Diagnosis Form 2) to identify clinical features suggestive of cancer predisposition.Among 1,415 patients, 208 (14.7%) met at least one criterion for suspected predisposition. Of these, 64 (31%) had a family history of early-onset cancer (<45 years). A first-degree relative was affected in 41 cases, while 18 reported ≥2 relatives with malignancies and 14 had ≥2 second-degree relatives involved. The most frequent cancers in first-degree relatives included breast (9), thyroid (6), hematologic malignancies (7; 5 ALL, 2 AML), melanoma (5), bladder (2), and brain tumors (2), alongside single cases of other solid tumors.Fifty-two patients (25% of the 208) had a known genetic condition at enrollment. Thirty-seven carried a cancer predisposition syndrome: 30 with Down syndrome, 3 with neurofibromatosis type 1, 2 with Noonan syndrome, 1 with Shwachman-Diamond syndrome, and 1 with a germline RB1 pathogenic variant. Additional comorbidities included congenital malformations (28; 13%) and neurodevelopmental disorders (20; 9.6%).Genetic counseling was recommended for 153 children (11% of all enrolled). Continued collection of consultation outcomes is needed to clarify predisposition conditions, support centralized counseling and genetic testing, and ensure consistent diagnostic approaches and follow-up for affected patients.
PEDIATRIC PAX5-REARRANGED ACUTE LYMPHOBLASTIC LEUKEMIA (ALL) IS A HIGH-RISK MOLECULAR SUBGROUP OF B-ALL WITH A POOR MRD-DEPENDENT PROGNOSTIC PROFILE N. Peccatori, A. Curto, D. Silvestri, S. Rebellato, Ž. Antić, et al. Haematologica, 2026 Introduction. PAX5-altered acute lymphoblastic leukemia (PAX5-alt ALL) is a recently defined molecular subtype of BCP-ALL, which displays a specific gene expression and is frequently associated with PAX5 aberrations, including rearrangements (PAX5r). Poor/intermediate outcomes were reported for PAX5-alt ALL. However, evidence on clinical features and prognosis of pediatric PAX5r ALL remains limited.Methods: Clinical data of all newly diagnosed PAX5r ALL patients (pts) (<18 years) enrolled in the consecutive AIEOP-BFM ALL trials (ALL 2000, NCT0061345; ALL 2009, NCT01117441; ALL 2017, NCT03643276) or observational studies in Italy (AIEOP), Germany and Austria (BFM) between 2001-2024, were retrospectively collected. PAX5 gene fusions were identified either by cytogenetics, FISH, targeted-RNAseq or whole-RNA-seq (WTS).Results. Overall, 165 pts with a confirmed PAX5r ALL were included in the study. Sixty-five different fusion gene partners were identified, with JAK2 (n=22), NOL4L (N=18), AUTS2 (n=13) and ETV6 (n=12) as the most frequent ones. Median age was 3.4 yrs (0.4-17.8) and 66.1% of the pts were males. Low incidence of CNS involvement (CNS3, 3.8%) and a high rate of hyperleukocytosis (WBC>100x109/L) (22%) was found. According to NCI criteria, 53.3% had high-risk (HR) status. IKZF1plus profile was found in 15.1% of the pts. A tendency to slow treatment response was observed: 13% of pts had prednisone poor response (PPR), 74.7% were PCR-MRD+ at the end of induction (EOI), with 28.6% showing MRD≥5x10-4, and 31.5% were still PCR-MRD+ at the end of consolidation. Per protocol final risk stratification: 25.8%, 42.9% and 31.3% of the pts were standard-risk (SR), medium-risk (MR) and HR, respectively. Further, we restricted the analyses solely to the consecutive PAX5r pts (n=99) enrolled in the AIEOP-BFM ALL 2017 study and compared their clinical features with all non-PAX5r pts (n=1945) enrolled in the same protocol. ETV6::RUNX1 pts were excluded from the analysis. Lower median age at diagnosis (3.4 vs 4.7 yrs), higher hyperleukocytosis rate (23.2% vs 8%, p<0.001), increased rate of IKZF1plus profile (14.4% vs 7.8%, p=0.02) and NCI HR status (57.6% vs 39.1%, p<0.001) were observed in the PAX5r group. No relevant differences in terms of treatment response and risk-stratification distribution were found between the two groups, except for a higher frequency of PPR (14.4% vs 5.7%, p=0.001) in the PAX5r ALL.With a median follow-up of 3.6 yrs, 5-yr EFS and OS for the AIEOP-BFM ALL 2009/2017 PAX5r pts (n=116) were 66.8±5.0% and 95.3±2.1%, respectively; 5-yr EFS were 100%, 59.8±9.3% and 43.8±12.7% for standard-risk (SR), medium-risk (MR) and high-risk (HR) groups, respectively, indicating that the poor prognostic impact of PAX5r applies only when EOI MRD is positive (MR/HR).Conclusions. This multinational retrospective study indicates that PAX5r ALL is frequently associated with HR clinical features and unfavorable outcomes in non-SR pts.
Inequalities in availability of clinical trials for pediatric, adolescent, and young adult patients with cancer in Europe: results from the SIOPE OCEAN project Kerstin K. Rauwolf, Teresa de Rojas, Miguel Martins, Maria Otth, Uta Dirksen, Delphine Heenen, Lejla Kameric, Pamela Kearns, Ruth Ladenstein, Cormac Owens, Caroline Queiroz, Richard Sullivan, Carmelo Rizzari, Gilles Vassal Lancet Regional Health Europe, 2026 There are pronounced inequalities in outcomes of children, adolescents, and young adults (AYA) with cancer across Europe. The OCEAN project aimed at describing the availability of clinical trials for this population, the inequalities between countries, and propose solutions to reduce these disparities. The ClinicalTrials.gov database was searched to identify all European cancer clinical interventional studies including patients <18 years with a start date between 2010 and 2022. The study included 436 cancer-directed trials in 38 European countries. More than half were academic-sponsored (55%), and 49% included exclusively pediatric/AYA patients. Important differences in available trial numbers per country were identified, with more trials observed in Northern and Western Europe as compared to Eastern Europe. There is an urgent need to address differences in clinical trials availability both at European and national levels to advance equity and improve care, research and access to innovation for all pediatric/AYA patients with cancer in Europe. Funding: This work has been performed as part of WP6 on inequalities in cancer research of the 4.UNCAN.eu Coordination and Support Action (#101069496) funded by the European Union and has been supported by Zoé4Life (MO).
High-Dose Methotrexate at All Ages: Safety, Efficacy, and Outcomes from the HDMTX European Registry Bertrand Pourroy, Maria D. Aumente, Christian Koenecke, Martin Stanulla, Andrés J. M. Ferreri, Thais Murciano-Carillo, Madhumita Dandapani, Timothy A. Ritzmann, Pere Barba, Etienne Chatelut, Katrina M. Ingley, Emma Morris, Elisabeth Schorb, Sven Liebig, Stefan Schwartz, Scott C. Howard, Ryan Combs, Nicolás Tentoni, Jennifer Lowe, Gabriela Villanueva, Claudia Sampor, Miriam Hwang, Carmelo Rizzari Cancers, 2026
Reducing Daunorubicin in Induction Therapy in Children With B-Lineage ALL With Favorable Prognosis: Results of Phase III Trial AIEOP-BFM ALL 2009 Hanna Gottschalk, Anja Möricke, Valentino Conter, Martin Schrappe, Jan Starý, Gunnar Cario, Michael Dworzak, Andishe Attarbaschi, Draga Barbaric, Franco Locatelli, Nicole Bodmer, Sarah Elitzur, Daniela Silvestri, Luciano Dalla-Pozza, Anke Katharina Bergmann, Christian Flotho, Barbara Buldini, Martin Stanulla, Shai Izraeli, Carmelo Rizzari, Jean-Pierre Bourquin, Andrea Biondi, Maria Grazia Valsecchi, Martin Zimmermann Journal of Clinical Oncology, 2025
Combined CAR-T/HSCT approach in a patient with refractory acute lymphoblastic leukemia and cystic fibrosis Benedetta E Di Majo, Francesca Vendemini, Sonia Bonanomi, Pietro Casartelli, Sara Napolitano, Giorgio Ottaviano, Giulia Prunotto, Marta Verna, Alessandra Sala, Guglielmo M Migliorino, Francesco Petrella, Lorenzo Rosso, Laura Claut, Paola Rafaniello Raviele, Carmelo Rizzari, Andrea Biondi, Adriana Balduzzi, Giovanna Lucchini Tumori, 2025
Results in pediatric T-ALL patients treated in trial AIEOP-BFM ALL 2009: Prognostic factors in the context of modern risk-adapted therapy Gunnar Cario, Maria Grazia Valsecchi, Valentino Conter, Giacomo Gotti, Anja Möricke, Martin Stanulla, Michaela Vossen‐Gajcy, Lennart Lenk, Jan Stary, Ondrej Hrusak, Michael Dworzak, Andishe Attarbaschi, Draga Barbaric, Franco Locatelli, Nicole Bodmer, Sarah Elitzur, Daniela Silvestri, Luciano Dalla‐Pozza, Franca Fagioli, Andreas E. Kulozik, Shai Izraeli, Carmelo Rizzari, Annika Rademacher, Barbara Buldini, Jean‐Pierre Bourquin, Martin Zimmermann, Martin Schrappe, Andrea Biondi Hemasphere, 2025
Population Pharmacokinetics of Inotuzumab Ozogamicin in Pediatric Relapsed/Refractory B-Cell Precursor Acute Lymphoblastic Leukemia: Results of Study ITCC-059 Jen-Hao Wu, Edoardo Pennesi, Francisco Bautista, May Garrett, Kei Fukuhara, Erica Brivio, Anneke C. J. Ammerlaan, Franco Locatelli, Inge M. van der Sluis, Claudia Rossig, Christiane Chen-Santel, Bella Bielorai, Arnaud Petit, Jan Starý, Cristina Díaz-de-Heredia, Susana Rives, Aengus O’Marcaigh, Carmelo Rizzari, Gernot Engstler, Karsten Nysom, Alba Rubio-San-Simón, Benedicte Bruno, Yves Bertrand, Benoît Brethon, Fanny Rialland, Geneviève Plat, Uta Dirksen, Lucie Sramkova, C. Michel Zwaan, Alwin D. R. Huitema Clinical Pharmacokinetics, 2024
Demographic Analysis of Cancer Research Priorities and Treatment Correlations Denis Horgan, Marc Van den Bulcke, Umberto Malapelle, Nicola Normanno, Ettore D. Capoluongo, Arsela Prelaj, Carmelo Rizzari, Aliki Stathopoulou, Jaya Singh, Marta Kozaric, France Dube, Manuel Ottaviano, Stefania Boccia, Gabriella Pravettoni, Ivana Cattaneo, Núria Malats, Reinhard Buettner, Karim Lekadir, Francesco de Lorenzo, Catherine Alix-Panabieres, Sara Badreh, Eric Solary, Ruggero De Maria, Paul Hofman Current Oncology, 2024
Bosutinib in Resistant and Intolerant Pediatric Patients With Chronic Phase Chronic Myeloid Leukemia: Results From the Phase I Part of Study ITCC054/COG AAML1921 Erica Brivio, Edoardo Pennesi, Marieke E. Willemse, Alwin D.R. Huitema, Yilin Jiang, Harm D.R. van Tinteren, Vincent H.J. van der Velden, Berna H. Beverloo, Monique L. den Boer, Lukas A.J. Rammeloo, Chad Hudson, Nyla Heerema, Karey Kowalski, Huadong Zhao, Luke Kuttschreuter, Francisco J. Bautista Sirvent, Andrew Bukowinski, Carmelo Rizzari, Jessica Pollard, Laura Murillo-Sanjuán, Matthew Kutny, Sara Zarnegar-Lumley, Michele Redell, Stacy Cooper, Yves Bertrand, Arnaud Petit, Julie Krystal, Markus Metzler, Donna Lancaster, Jean-Pierre Bourquin, Jayashree Motwani, Inge M. van der Sluis, Franco Locatelli, Michael E. Roth, Nobuko Hijiya, Christian M. Zwaan Journal of Clinical Oncology, 2024
Tackling the implementation gap for the uptake of NGS and advanced molecular diagnostics into healthcare systems Denis Horgan, Marc Van den Bulcke, Umberto Malapelle, Giancarlo Troncone, Nicola Normanno, Ettore D. Capoluongo, Arsela Prelaj, Carmelo Rizzari, Dario Trapani, Jaya Singh, Marta Kozaric, John Longshore, Manuel Ottaviano, Stefania Boccia, Gabriella Pravettoni, Ivana Cattaneo, Núria Malats, Reinhard Buettner, Karim Lekadir, Francesco de Lorenzo, Paul Hofman, Ruggero De Maria Heliyon, 2024
Policy of pediatric oncology drug development Lenneke Schrier, C. Michel Zwaan, Carmelo Rizzari, Nicole Scobie, Gregory Reaman, Andy Pearson Essentials of Translational Pediatric Drug Development from Past Needs to Future Opportunities, 2024
Practice of pediatric oncology drug development Lenneke Schrier, Andy Pearson, Carmelo Rizzari, Alwin Huitema, Nicole Scobie, Gregory Reaman, C. Michel Zwaan Essentials of Translational Pediatric Drug Development from Past Needs to Future Opportunities, 2024
Aligning Cancer Research Priorities in Europe with Recommendations for Conquering Cancer: A Comprehensive Analysis Denis Horgan, Marc Van den Bulcke, Umberto Malapelle, Nicola Normanno, Ettore D. Capoluongo, Arsela Prelaj, Carmelo Rizzari, Aliki Stathopoulou, Jaya Singh, Marta Kozaric, France Dube, Manuel Ottaviano, Stefania Boccia, Gabriella Pravettoni, Ivana Cattaneo, Núria Malats, Reinhard Buettner, Karim Lekadir, Francesco de Lorenzo, Patricia Blanc, Catherine Alix-Panabieres, Sara Badreh, Paul Hofman, Eric Solary, Ruggero De Maria Healthcare Switzerland, 2024
The long-term efficacy and safety of nilotinib in pediatric patients with CML: a 5-year update of the DIALOG study Nobuko Hijiya, Alexey Maschan, Carmelo Rizzari, Hiroyuki Shimada, Carlo Dufour, Hiroaki Goto, Hyoung Jin Kang, Terri Guinipero, Zeynep Karakas, Francisco Bautista, Stéphane Ducassou, Keon Hee Yoo, Christian Michel Zwaan, Frédéric Millot, Briana C. Patterson, Jill Samis, Miguel Izquierdo, Ksenia Titorenko, Sai Li, Darintr Sosothikul Blood Advances, 2023
CD72 is a pan-tumor antigen associated to pediatric acute leukemia Barbara Buldini, Giovanni Faggin, Elena Porcù, Pamela Scarparo, Katia Polato, Claudia Tregnago, Elena Varotto, Paolo Rizzardi, Carmelo Rizzari, Franco Locatelli, Alessandra Biffi, Martina Pigazzi Cytometry Part A, 2023
Mobilising Collaboration among Stakeholders to Optimise the Growing Potential of Data for Tackling Cancer Denis Horgan, Marc Van den Bulcke, Umberto Malapelle, Nicola Normanno, Ettore D. Capoluongo, Arsela Prelaj, Carmelo Rizzari, Aliki Stathopoulou, Jaya Singh, Marta Kozaric, France Dube, Manuel Ottaviano, Stefania Boccia, Gabriella Pravettoni, Ivana Cattaneo, Núria Malats, Reinhard Buettner, Karim Lekadir, Francesco de Lorenzo, Catherine Alix-Panabieres, Sara Badreh, Paul Hofman, Ruggero De Maria, Eric Solary Journal of Molecular Pathology, 2023
Development of a centralised triage centre for children with cancer and blood disorders in response to the humanitarian crisis in Ukraine Marta Salek, Alexandra Mueller, Inna Alanbousi, Zuzanna Cepowska, Malgorzata Dutkiewicz, Julian Earl, Dmitry Evseev, Roman Kizyma, Khrystyna Kliuchkivska, Julia Kolodrubiec, Katarzyna Matczak, Yuliya Nogovitsyna, Aleksandra Oszer, Mikhail Pogorelyy, Anna Raciborska, Suheir Rasul, Igor Sokolowski, Andriy Sopilnyak, Anna Vinitsky, Marcin W Wlodarski, Natalia Wobst, Taisiya Yakimkova, Carlos Rodriguez-Galindo, Paul H Wise, Wojciech Mlynarski, Asya Agulnik, Sarah Weeks Alexander, Anna Apel, Mykhaylo V Adyrov, Meghana Avula, Wioletta Anna Bal, Walentyna Aniela Balwierz, Luisa Basset-Salom, Daniel Bastardo Blanco, Karolina Jadwiga Bauer, Ildar T Bayazitov, Pablo Berlanga, Nickhill Hitesh Bhakta, Katarzyna Anna Bieniek, Ewa Iwona Bien, Christopher Andrew Blackwood, Sally Jane Blair, Khrystyna Ihorivna Bodak, Irina Bordeianu, Eric Bouffet, Joao Maria Braganca, Mihaela Silvia Bucurenci, Elżbieta Beata Budny, Andrii Budzyn, Christopher Carl Bumgardner, Raina Nichole Burditt, Victoria Burnside Clapp, Viacheslav Valeriyovych Bykov, Adela Cañete, Monica Carnelli, Elena Cela, Radoslaw Chaber, Anna Cherner-Drieux, Mariya Chubata, Heidi M Clough, Piotr Stefan Czauderna, Jolanta Czernicka - Siwecka, Krzysztof Czyzewski, Jean-Hugues Dalle, Olha Dashchakovska, Linda A de Koning, Bozenna Malgorzata Dembowska-Baginska, Katarzyna Derwich, Uta Dirksen, Rachel Dommett, Olha Ihorivna Dorosh, Nuno Jorge dos Reis Farinha, Katarzyna Anna Drabko, Monica Desiree Dragomir, Michael Dworzak, Sergii Vitaliiovych Dyma, Angelika Eggert, Martin William English, Becky S Farren, Nataliia Yuriina Fedyk, Ana Fernández-Teijeiro, Severyn Romanovych Ferneza, Whitney Baer Foster, Leeanna Elizabeth Fox Irwin, Robert Maciej Gałązkowski, Galyna Ganieva, Vasylyna Andriivna Garanzha, Marina S Gelman, Jan Krzysztof Godzinski, Anne Françoise Goeres, Rodica Golban, Dylan Elizabeth Graetz, Jeanette Greiner, Michael J Griksaitis, Sumit Gupta, Michal Andrzej Hampel, Sara Grace Hastings, Delphine Liliane Heenen, Marcela C Hill, Ihor Holiuk, Wolfgang Holter, Rachael Emma Hough, Lukasz Marek Hutnik, Ninela Irga-Jaworska, Oleksandr Andriyovych Istomin, Anna Ignatova, Szymon Lech Janczar, Arman Kacharian, Krzysztof Kalwak, Grażyna Małgorzata Karolczyk, Nataliia Mikolaivna Karpenko, Halyna Oleksandrivna Katsubo, Antonis Kattamis, Bernarda Jadwiga Kazanowska, Alex Kentsis, Petra Ketteler, Anita Kienesberger, Roman Kiselev, Zoryana Petrivna Kizyma, Hryhorii Ivanovych Klymniuk, Alexandra Kolenova, Yuliia Kostiuk, Tomasz Kowalik, Olena Igorivna Kozlova, Vladyslav Kozubenko, Kathelijne Kraal, Tetyana Oleksandrivna Kramar, Maryna Maryna Krawczuk-Rybak, Irina Kulemzina, Paulina Kurkowska, Andriy S. Kuzyk, Ruth Lydia Ladenstein, Pawel Jozef Laguna, Alvaro Lassaletta, Kai Lehmberg, Oksana Leontieva, Serhii Liashenko, Loizos G. Loizou, Sonia Anna Lucchetta, Matthew William Lupo, Lesya Lysytsia, Oleksandr Lysytsia, Katarzyna Anna Machnik, Maura Massimino, Jeff A Mainland, Michal Jacek Matysiak, Pierre Mayeur, Beth Anne Miller, Anastasia A Minervina, Volha Mishkova, Agnieszka Joanna Mizia-Malarz, Andres Morales La Madrid, Daniel C Moreira, Lucas Moreno, Vadim P Moskvin, Sheena Teresa Mukkada, Katarzyna Maria Muszyńska-Rosłan, Iryna Volodymyrivna Mykychak, Charlotte Niemeyer, Akoya Janae' Nelson, Tomasz Ociepa, Stefano Oltolini, Nataliia Onipko, Andrew Pappas, Amit B Patel, Alina Alina Patrahau, Jennifer L. Pauley, Yehor Mikhailovich Pavlenko, Andrij Oleksandrovych Pavlovych, Jarosław Władysław Peregud-Pogorzelski, Marta Perek-Polnik, Vanesa Pérez, Antonio Pérez-Martínez, Yana Pikman, Graziano Pitozzi Pitozzi, Rui Gentil Portugal, Victoria Vita Posternak, Viktoriya Pleshkan, Arcangelo Prete, Kathy Pritchard-Jones, Alessandra Radaelli, Tegan Jemma Reeves, Dirk Reinhardt, Andrey V Reshetnyak, Andrew Jacob Rider, Carmelo Rizzari, Damiano Damiano Rizzi, Karen Gabriela Rodriguez Hermosillo, Olena Volodymyrivna Ronenko, Aneta Olga Rostkowska, Liudmyla Yaroslavivna Rudko, Firas Mohamed Sakaan, Nadezhda Aleksandrovna Sakhar, Zeena S Salman, Natallia N Savva, Davide Scaccaglia, Elizabeth Hawthorne Schaeffer, Carina Ursula Schneider, Nicole Scobie, Olena Volodymyrivna Semeniuk, Roksoliana Shevchyk, Ana Shuler, Stanislav Shvets, Liz Sniderman, Szymon Pawel Skoczen, William John Smeal, Anna Alexandra Sonkin, Andrea Spota, Lucie Sramkova, Alla Ivanivna Stepanjuk, Jaroslav Sterba, Brigitte Strahm, Jan Styczynski, Olha Svintsova, Andriy V Synyuta, Tomasz Szczepanski, Paweł Kukiz Szczucinski, Bartosz Miroslaw Szmyd, Maria Tasso Cereceda, Alina Teliuk, Iwona Tomanek, Phoebe Topping, Montserrat Torrent, Joanna Trelińska, Olha Orestivna Troyanovska, Lyudmila G. Tsurkan, Iryna Tsymbalyuk-Voloshyn, Sergiy Ihorovych Tyupa, Tomasz Franciszek Urasinski, Agnieszka Urbanek Dądela, Nataliia Jroslavivna Vasilieva, Aksana Vasilyeva, Jaime Verdú-Amorós, Natalia Vilcu-Bajurean, Leo Vinitsky, Victoria Vivtcharenko, Nelia Vovk, Giovanni Volpe, Oksana Ivanivna Vorobel, Jacek Tadeusz Wachowiak, Marcin Sławomir Wasiak, Lance Allan Wiedower, Lena Isolde Wuenschel, Mariusz Stanislaw Wysocki, Marina Yurieva, Anastasiia Zagurska, Stanislav S Zakharenko, Aelita V Zakharenko, Khrystyna Zapotochna, Joanna Emilia Zawitkowska, Marco Zecca Lancet Oncology, 2023
Improved survival and MRD remission with blinatumomab vs. chemotherapy in children with first high-risk relapse B-ALL Franco Locatelli, Gerhard Zugmaier, Carmelo Rizzari, Joan D. Morris, Bernd Gruhn, Thomas Klingebiel, Rosanna Parasole, Christin Linderkamp, Christian Flotho, Arnaud Petit, Concetta Micalizzi, Yi Zeng, Rajendra Desai, William N. Kormany, Cornelia Eckert, Anja Möricke, Mary Sartor, Ondrej Hrusak, Christina Peters, Vaskar Saha, Luciana Vinti, Arend von Stackelberg Leukemia, 2023
NUP214–ABL1 fusion in childhood T-ALL Giulia Veltri, Max Sandei, Daniela Silvestri, Silvia Bresolin, Andrea Pession, Nicola Santoro, Ottavio Ziino, Marinella Veltroni, Carmelo Rizzari, Alessandra Biffi, Maria Grazia Valsecchi, Valentino Conter, Barbara Buldini, Benedetta Accordi, Valentina Serafin Pediatric Blood and Cancer, 2022
PAX5 fusion genes are frequent in poor risk childhood acute lymphoblastic leukaemia and can be targeted with BIBF1120 Grazia Fazio, Silvia Bresolin, Daniela Silvestri, Manuel Quadri, Claudia Saitta, Elena Vendramini, Barbara Buldini, Chiara Palmi, Michela Bardini, Andrea Grioni, Silvia Rigamonti, Marta Galbiati, Stefano Mecca, Angela Maria Savino, Alberto Peloso, Jia-Wey Tu, Sanil Bhatia, Arndt Borkhardt, Concetta Micalizzi, Luca Lo Nigro, Franco Locatelli, Valentino Conter, Carmelo Rizzari, Maria Grazia Valsecchi, Geertruij te Kronnie, Andrea Biondi, Giovanni Cazzaniga Ebiomedicine, 2022
Global effort to evacuate Ukrainian children with cancer and blood disorders who have been affected by war Asya Agulnik, Roman Kizyma, Marta Salek, Marcin W Wlodarski, Mikhail Pogorelyy, Aleksandra Oszer, Taisiya Yakimkova, Yuliya Nogovitsyna, Malgorzata Dutkiewicz, Jean-Hugues Dalle, Uta Dirksen, Angelika Eggert, Ana Fernández-Teijeiro, Jeanette Greiner, Kathelijne Kraal, Alexandra Mueller, Lucie Sramkova, Marco Zecca, Paul H Wise, Wojciech Mlynarski, Meghana Avula, Mykhaylo V Adyrov, Pablo Berlanga, Christopher Andrew Blackwood, Eric Bouffet, Piotr Stefan Czauderna, Linda A de Koning, Nuno Jorge dos Reis Farinha, Whitney Baer Foster, Dylan Elizabeth Graetz, Sumit Gupta, Wolfgang Holter, Rachael Emma Hough, Khrystyna Kliuchkivska, Alexandra Kolenova, Julia Kołodrubiec, Daniel C Moreira, Sheena Teresa Mukkada, Iryna Mykychak, Anna Raciborska, Zeena S Salman, Andriy Sopilnyak, Sergiy Tyupa, Anna Vinitsky, Natalia Margarete Wobst, Beth Anne Miller, Suheir Subhi Rasul, Carlos Rodriguez-Galindo, Inna Alanbousi, Sarah Weeks Alexander, Anna Apel, Wioletta Anna Bal, Walentyna Aniela Balwierz, Luisa Basset-Salom, Daniel Bastardo Blanco, Karolina Jadwiga Bauer, Ildar T Bayazitov, Nickhill Hitesh Bhakta, Ewa Iwona Bien, Katarzyna Anna Bieniek, Sally Jane Blair, Khrystyna Ihorivna Bodak, Irina Michael Bordeianu, Joao Maria Braganca, Mihaela Silvia Bucurenci, Elżbieta Beata Budny, Andrii Budzyn, Christopher Carl Bumgardner, Raina Nichole Burditt, Victoria Grace Burnside Clapp, Viacheslav Bykov, Adela Cañete, Monica Carnelli, Elena Cela, Zuzanna Paulina Cepowska, Radoslaw Chaber, Anna Cherner-Drieux, Mariya Chubata, Heidi M Clough, Jolanta Czernicka - Siwecka, Krzysztof Czyzewski, Olha Dashchakovska, Bozenna Malgorzata Dembowska-Baginska, Katarzyna Derwich, Rachel Dommett, Olha Dorosh, Katarzyna Anna Drabko, Monica Desiree Dragomir, Michael Dworzak, Sergii Dyma, Julian Darocus Earl, Martin William English, Dmitry A Evseev, Becky S Farren, Nataliia Fedyk, Severyn Ferneza, Leeanna Elizabeth Fox Irwin, Robert Maciej Gałązkowski, Galyna Ganieva, Vasylyna Garanzha, Marina S Gelman, Jan Krzysztof Godzinski, Anne Francoise Goeres, Rodica Golban, Michael J Griksaitis, Michal Andrzej Hampel, Sara Grace Hastings, Delphine Liliane Heenen, Marcela C Hill, Igor Holiuk, Lukasz Marek Hutnik, Ninela Irga-Jaworska, Oleksandr Istomin, Szymon Lech Janczar, Arman Kacharian, Krzysztof Kalwak, Grażyna Malgorzata Karolczyk, Nataliia Mikolaivna Karpenko, Halyna Katsubo, Bernarda Jadwiga Kaznowska, Alex Kentsis, Petra Ketteler, Anita Kienesberger, Roman Kiselev, Zoryana Kizyma, Hryhorii Klymniuk, Yuliia Kostiuk, Tomasz Kowalik, Olena Kozlova, Vladyslav Kozubenko, Tetyana Kramar, Maryna Krawczuk-Rybak, Irina Kulemzina, Paulina Kurkowska, Andriy S Kuzyk, Ruth Lydia Ladenstein, Pawel Jozef Laguna, Alvaro Lassaletta, Kai Lehmberg, Oksana Leontieva, Serhii Liashenko, Loizos G Loizou, Sonia Anna Lucchetta, Matthew William Lupo, Lesya Lysytsia, Oleksandr Lysytsia, Katarzyna Anna Machnik, Jeff A Mainland, Katarzyna Ewa Matczak, Michal Jacek Matysiak, Pierre Mayeur, Anastasia A Minervina, Volha Mishkova, Agnieszka Joanna Mizia-Malarz, Andres Morales La Madrid, Lucas Moreno, Vadim P Moskvin, Katarzyna Maria Muszyńska-Rosłan, Akoya Janae Nelson, Tomasz Ociepa, Stefano Oltolini, Nataliia Onipko, Andrew Pappas, Amit B Patel, Alina Patrahau, Jennifer L Pauley, Yehor Pavlenko, Andrij Pavlovych, Jarosław Peregud-Pogorzelski, Marta Perek-Polnik, Vanesa Perez, Antonio Perez-Martinez, Yana Pikman, Graziano Pitozzi, Rui Gentil Portugal, Victoria Vita Posternak, Arcangelo Prete, Kathy Pritchard-Jones, Alessandra Radaelli, Tegan Reeves, Dirk Reinhardt, Andrey V Reshetnyak, Andrew Jacob Rider, Carmelo Rizzari, Damiano Rizzi, Karen Gabriela Rodriguez Hermosillo, Olena Ronenko, Aneta Olga Rostowska, Liudmyla Rudko, Firas Mohamed Sakaan, Nadezhda Sakhar, Natallia N Savva, Davide Scaccaglia, Elizabeth Hawthorne Schaeffer, Carina Ursula Schneider, Nicole Scobie, Olena Semeniuk, Roksoliana Shevchyk, Ana I Shuler, Stanislav Shvets, Szymon Pawel Skoczen, William John Smeal, Igor Sokolowski, Anna A Sonkin, Alla Ivanivna Stepanjuk, Andrea Spota, Jaroslav Sterba, Jan Styczynski, Olha Svintsova, Andriy V Synyuta, Tomasz Szczepanski, Paweł Kukiz Szczucinski, Bartosz Miroslaw Szmyd, Maria Tasso Cereceda, Alina Teliuk, Iwona Tomanek, Phoebe Topping, Montserrat Torrent, Joanna Trelińska, Olha Troyanovska, Elena Trubnikova, Lyudmila G Tsurkan, Iryna Tsymbalyuk-Voloshyn, Tomasz Franciszek Urasinski, Agnieszka Urbanek-Dadela, Nataliia Vasilieva, Aksana Vasilyeva, Jaime Verdú-Amorós, Natalia Vilcu-Bajurean, Leo Vinitsky, Giovanni Volpe, Oksana Vorobel, Jacek Tadeusz Wachowiak, Marcin Slawomir Wasiak, Lance Allan Wiedower, Lena Isolde Wuenschel, Mariusz Stanislaw Wysocki, Marina Yurieva, Anastasiia Zagurska, Stanislav S Zakharenko, Aelita V Zakharenko, Khrystyna Zapotochna, Joanna Emilia Zawitkowska Lancet Haematology, 2022
Inotuzumab ozogamicin as single agent in pediatric patients with relapsed and refractory acute lymphoblastic leukemia: results from a phase II trial Edoardo Pennesi, Naomi Michels, Erica Brivio, Vincent H. J. van der Velden, Yilin Jiang, Adriana Thano, Anneke J. C. Ammerlaan, Judith M. Boer, H. Berna Beverloo, Barbara Sleight, Ying Chen, Britta Vormoor-Bürger, Susana Rives, Bella Bielorai, Claudia Rössig, Arnaud Petit, Carmelo Rizzari, Gernot Engstler, Jan Starý, Francisco J. Bautista Sirvent, Christiane Chen-Santel, Benedicte Bruno, Yves Bertrand, Fanny Rialland, Geneviève Plat, Dirk Reinhardt, Luciana Vinti, Arend Von Stackelberg, Franco Locatelli, Christian M. Zwaan Leukemia, 2022
Daratumumab with or without chemotherapy in relapsed and refractory acute lymphoblastic leukemia. A retrospective observational Campus ALL study Marco Cerrano, Massimiliano Bonifacio, Matteo Olivi, Antonio Curti, Michele Malagola, Michelina Dargenio, Anna Maria Scattolin, Cristina Papayannidis, Fabio Forghieri, Carmela Gurrieri, Ilaria Tanasi, Patrizia Zappasodi, Roberta La Starza, Nicola Stefano Fracchiolla, Patrizia Chiusolo, Luisa Giaccone, Maria Ilaria Del Principe, Fabio Giglio, Marzia Defina, Claudio Favre, Carmelo Rizzari, Barbara Castella, Giovanni Pizzolo, Felicetto Ferrara, Sabina Chiaretti, Robin Foà Haematologica, 2022
CD56, HLA-DR, and CD45 recognize a subtype of childhood AML harboring CBFA2T3-GLIS2 fusion transcript Andrea Zangrando, Francesca Cavagnero, Pamela Scarparo, Elena Varotto, Samuela Francescato, Claudia Tregnago, Rosanna Cuccurullo, Franca Fagioli, Luca Lo Nigro, Riccardo Masetti, Maria Caterina Putti, Carmelo Rizzari, Nicola Santoro, Andrea Pession, Martina Pigazzi, Franco Locatelli, Giuseppe Basso, Barbara Buldini Cytometry Part A, 2021
A phase 2 study of nilotinib in pediatric patients with CML: Long-term update on growth retardation and safety Nobuko Hijiya, Alexey Maschan, Carmelo Rizzari, Hiroyuki Shimada, Carlo Dufour, Hiroaki Goto, Hyoung Jin Kang, Terri Guinipero, Zeynep Karakas, Francisco Bautista, Stéphane Ducassou, Keon Hee Yoo, Christian Michel Zwaan, Frédéric Millot, Briana Patterson, Jill Samis, Paola Aimone, Alex Allepuz, Ksenia Titorenko, Darintr Sosothikul Blood Advances, 2021
Musculoskeletal manifestations of childhood cancer and differential diagnosis with juvenile idiopathic arthritis (ONCOREUM): a multicentre, cross-sectional study Adele Civino, Giovanni Alighieri, Eleonora Prete, Anna Maria Caroleo, Silvia Magni-Manzoni, Luciana Vinti, Micol Romano, Nicola Santoro, Giovanni Filocamo, Tamara Belotti, Francesca Santarelli, Chiara Gorio, Francesca Ricci, Antonella Colombini, Serena Pastore, Simone Cesaro, Patrizia Barone, Federico Verzegnassi, Alma Nunzia Olivieri, Monica Ficara, Angela Miniaci, Giovanna Russo, Romina Gallizzi, Roberta Pericoli, Luciana Breda, Rossella Mura, Rosa Anna Podda, Daniela Onofrillo, Bianca Lattanzi, Elisa Tirtei, Maria Cristina Maggio, Raffaela De Santis, Rita Consolini, Annalisa Arlotta, Francesco La Torre, Chiara Mainardi, Maria Antonietta Pelagatti, Elisa Coassin, Ilaria Capolsini, Roberta Burnelli, Assunta Tornesello, Francesca Soscia, Alessandro De Fanti, Donato Rigante, Cristina Pizzato, Carmela De Fusco, Massimo Eraldo Abate, Andrea Roncadori, Elisa Rossi, Giulia Stabile, Andrea Biondi, Loredana Lepore, Valentino Conter, Roberto Rondelli, Andrea Pession, Angelo Ravelli, M Amatruda, C Atzeni, P Bertolini, B Bigucci, M Caniglia, M Cappella, M Cattalini, MG Cefalo, M Cellini, E Cortis, S Davì, F De Benedetti, A Di Cataldo, E Fabbri, F Fagioli, I Fontanili, A Garaventa, MF Gicchino, S Ladogana, F Locatelli, A Magnolato, M Marsili, S Martino, M Mascarin, C Messina, C Micalizzi, F Porta, C Rizzari Lancet Rheumatology, 2021
Recurrent genetic fusions redefine MLL germ line acute lymphoblastic leukemia in infants Grazia Fazio, Michela Bardini, Paola De Lorenzo, Andrea Grioni, Manuel Quadri, Lucia Pedace, Lilia Corral Abascal, Sonia Palamini, Chiara Palmi, Barbara Buldini, Luciana Vinti, Rosanna Parasole, Elena Barisone, Marco Zecca, Claudio Favre, Franco Locatelli, Valentino Conter, Carmelo Rizzari, Maria Grazia Valsecchi, Andrea Biondi, Giovanni Cazzaniga Blood, 2021
Impact of COVID-19 in paediatric early-phase cancer clinical trials in Europe: A report from the Innovative Therapies for Children with Cancer (ITCC) consortium Alba Rubio-San-Simón, Nicolas André, Maria Giuseppina Cefalo, Isabelle Aerts, Alicia Castañeda, Sarah Benezech, Guy Makin, Natasha van Eijkelenburg, Karsten Nysom, Lynley Marshall, Marion Gambart, Raquel Hladun, Claudia Rossig, Luca Bergamaschi, Franca Fagioli, Ben Carpenter, Stephane Ducassou, Cormac Owens, Ingrid Øra, Antonio Juan Ribelles, Bram De Wilde, Pilar Guerra-García, Marion Strullu, Carmelo Rizzari, Torben Ek, Simone Hettmer, Nicolas U. Gerber, Christine Rawlings, Manuel Diezi, Sauli Palmu, Antonio Ruggiero, Jaime Verdú, Teresa de Rojas, Gilles Vassal, Birgit Geoerger, Lucas Moreno, Francisco Bautista European Journal of Cancer, 2020
Children with cancer in the time of COVID-19: An 8-week report from the six pediatric onco-hematology centers in Lombardia, Italy Andrea Ferrari, Marco Zecca, Carmelo Rizzari, Fulvio Porta, Massimo Provenzi, Maddalena Marinoni, Richard Fabian Schumacher, Roberto Luksch, Monica Terenziani, Michela Casanova, Filippo Spreafico, Stefano Chiaravalli, Francesca Compagno, Federica Bruni, Chiara Piccolo, Laura Bettini, Mariella D'Angiò, Giulia Maria Ferrari, Andrea Biondi, Maura Massimino, Adriana Balduzzi Pediatric Blood and Cancer, 2020
Randomized post-induction and delayed intensification therapy in high-risk pediatric acute lymphoblastic leukemia: long-term results of the international AIEOP-BFM ALL 2000 trial on behalf of the AIEOP-BFM (Associazione Italiana di Ematologia e Oncologia Pediatrica & Berlin-Frankfurt-Münster) Study Group, Andishe Attarbaschi, Georg Mann, Martin Zimmermann, Peter Bader, Elena Barisone, Giuseppe Basso, Andrea Biondi, Gunnar Cario, Gianni Cazzaniga, Antonella Colombini, Christian Flotho, Michaela Kuhlen, Peter Lang, Melchior Lauten, Christin Linderkamp, Franco Locatelli, Luca Lo Nigro, Anja Möricke, Felix Niggli, Renate Panzer-Grümayer, Rosanna Parasole, Christina Peters, Maria Caterina Putti, Carmelo Rizzari, Meinolf Suttorp, Maria Grazia Valsecchi, Valentino Conter, Martin Schrappe Leukemia, 2020
Flash survey on severe acute respiratory syndrome coronavirus-2 infections in paediatric patients on anticancer treatment Ondrej Hrusak, Tomas Kalina, Joshua Wolf, Adriana Balduzzi, Massimo Provenzi, Carmelo Rizzari, Susana Rives, María del Pozo Carlavilla, Maria E.V. Alonso, Nerea Domínguez-Pinilla, Jean-Pierre Bourquin, Kjeld Schmiegelow, Andishe Attarbaschi, Pernilla Grillner, Karin Mellgren, Jutte van der Werff ten Bosch, Rob Pieters, Triantafyllia Brozou, Arndt Borkhardt, Gabriele Escherich, Melchior Lauten, Martin Stanulla, Owen Smith, Allen E.J. Yeoh, Sarah Elitzur, Ajay Vora, Chi-Kong Li, Hany Ariffin, Alexandra Kolenova, Luciano Dallapozza, Roula Farah, Jelena Lazic, Atsushi Manabe, Jan Styczynski, Gabor Kovacs, Gabor Ottoffy, Maria S. Felice, Barbara Buldini, Valentino Conter, Jan Stary, Martin Schrappe European Journal of Cancer, 2020
Emapalumab in children with primary hemophagocytic lymphohistiocytosis Franco Locatelli, Michael B. Jordan, Carl Allen, Simone Cesaro, Carmelo Rizzari, Anupama Rao, Barbara Degar, Timothy P. Garrington, Julian Sevilla, Maria-Caterina Putti, Franca Fagioli, Martina Ahlmann, Jose-Luis Dapena Diaz, Michael Henry, Fabrizio De Benedetti, Alexei Grom, Genevieve Lapeyre, Philippe Jacqmin, Maria Ballabio, Cristina de Min New England Journal of Medicine, 2020
Legionellosis in paediatrics Medico E Bambino, 2020
Phase 2 study of nilotinib in pediatric patients with Philadelphia chromosome–positive chronic myeloid leukemia Nobuko Hijiya, Alexey Maschan, Carmelo Rizzari, Hiroyuki Shimada, Carlo Dufour, Hiroaki Goto, Hyoung Jin Kang, Terri Guinipero, Zeynep Karakas, Francisco Bautista, Stéphane Ducassou, Keon Hee Yoo, Christian Michel Zwaan, Frédéric Millot, Paola Aimone, Alex Allepuz, Sara Quenet, Florence Hourcade-Potelleret, Sabine Hertle, Darintr Sosothikul Blood, 2019
Acute myeloid leukaemia niche regulates response to L-asparaginase Ilaria M. Michelozzi, Valentina Granata, Giada De Ponti, Gaia Alberti, Chiara Tomasoni, Laura Antolini, Carlo Gambacorti‐Passerini, Bernhard Gentner, Francesco Dazzi, Andrea Biondi, Tiziana Coliva, Carmelo Rizzari, Alice Pievani, Marta Serafini British Journal of Haematology, 2019
Protocol II vs protocol III given twice during reinduction therapy in children with medium-risk ALL Franco Locatelli, Maria Grazia Valsecchi, Anja Möricke, Martin Zimmermann, Bernd Gruhn, Andrea Biondi, Andreas E. Kulozik, Daniela Silvestri, Nicole Bodmer, Maria Caterina Putti, Stefan Burdach, Concetta Micalizzi, Andrea Teigler-Schlegel, Jörg Ritter, Andrea Pession, Gunnar Cario, Stefan Bielack, Giuseppe Basso, Thomas Klingebiel, Luciana Vinti, Carmelo Rizzari, Andishe Attarbaschi, Nicola Santoro, Rosanna Parasole, Georg Mann, Leonid Karawajew, Oskar A. Haas, Valentino Conter, Martin Schrappe Blood, 2017
Phase I/Phase II study of blinatumomab in pediatric patients with relapsed/refractory acute lymphoblastic leukemia Arend von Stackelberg, Franco Locatelli, Gerhard Zugmaier, Rupert Handgretinger, Tanya M. Trippett, Carmelo Rizzari, Peter Bader, Maureen M. O’Brien, Benoît Brethon, Deepa Bhojwani, Paul Gerhardt Schlegel, Arndt Borkhardt, Susan R. Rheingold, Todd Michael Cooper, Christian M. Zwaan, Phillip Barnette, Chiara Messina, Gérard Michel, Steven G. DuBois, Kuolung Hu, Min Zhu, James A. Whitlock, Lia Gore Journal of Clinical Oncology, 2016
Williams syndrome and mature B-Leukemia: A random association? Valentina Decimi, Grazia Fazio, Fabiola Dell'Acqua, Silvia Maitz, Marta Galbiati, Carmelo Rizzari, Andrea Biondi, Giovanni Cazzaniga, Angelo Selicorni European Journal of Medical Genetics, 2016
Road Traffic Pollution and Childhood Leukemia: A Nationwide Case-control Study in Italy Corrado Magnani, Alessandra Ranucci, Chiara Badaloni, Giulia Cesaroni, Daniela Ferrante, Lucia Miligi, Stefano Mattioli, Roberto Rondelli, Luigi Bisanti, Paola Zambon, Santina Cannizzaro, Paola Michelozzi, Pierluigi Cocco, Egidio Celentano, Giorgio Assennato, Domenico Franco Merlo, Paola Mosciatti, Liliana Minelli, Marina Cuttini, Maria Valeria Torregrossa, Susanna Lagorio, Riccardo Haupt, Francesco Forastiere, Corrado Magnani, Stefano Mattioli, Andrea Farioli, Lucia Miligi, Alessandra Ranucci, Daniela Ferrante, Roberto Rondelli, Alberto Salvan, Giuseppe Masera, Carmelo Rizzari, Luigi Bisanti, Paola Zambon, Alessandra Greco Veneto, Santina Cannizzaro, Lorenzo Gafà, Lia Lidia Luzzatto, Alessandra Benvenuti, Paola Michelozzi, Ursula Kirchmayer, Pierluigi Cocco, Claudia Galassi, Egidio Celentano, Erni Guarino, Giorgio Assennato, Gigliola de Nichilo, Domenico Franco Merlo, Vittorio Bocchini, Paola Mosciatti, Liliana Minelli, Manuela Chiavarini, Marina Cuttini, Veronica Casotto, Maria Valeria Torregrossa, Rosaria Maria Valenti, Francesco Forastiere, Riccardo Haupt, Susanna Lagorio, Serena Risica, Alessandro Polichetti, Francesco Bochicchio, Cristina Nuccetelli, Pierfranco Biddau, Maurizio Aricò, Gian Luca DeSalvo, Franco Locatelli, Andrea Pession, Stefania Varotto, Vincenzo Poggi, Pia Massaglia, Daniele Monetti, Roberto Targhetta, Gabriella Bernini, Franco Pannelli, Giuseppe Sampietro, Gino Schilirò, Alessandro Pulsoni, Stefano Parodi Archives of Medical Research, 2016
A Case of T-cell Acute Lymphoblastic Leukemia Relapsed As Myeloid Acute Leukemia Maddalena Paganin, Barbara Buldini, Giuseppe Germano, Elena Seganfreddo, Annamaria di Meglio, Elisa Magrin, Francesca Grillo, Martina Pigazzi, Carmelo Rizzari, Giovanni Cazzaniga, Hossein Khiabanian, Teresa Palomero, Raul Rabadan, Adolfo A. Ferrando, Giuseppe Basso Pediatric Blood and Cancer, 2016
Early T-cell precursor acute lymphoblastic leukaemia in children treated in AIEOP centres with AIEOP-BFM protocols: A retrospective analysis Valentino Conter, Maria Grazia Valsecchi, Barbara Buldini, Rosanna Parasole, Franco Locatelli, Antonella Colombini, Carmelo Rizzari, Maria Caterina Putti, Elena Barisone, Luca Lo Nigro, Nicola Santoro, Ottavio Ziino, Andrea Pession, Anna Maria Testi, Concetta Micalizzi, Fiorina Casale, Paolo Pierani, Simone Cesaro, Monica Cellini, Daniela Silvestri, Giovanni Cazzaniga, Andrea Biondi, Giuseppe Basso Lancet Haematology, 2016
Collaborative efforts driving progress in pediatric acute myeloid leukemia C. Michel Zwaan, Edward A. Kolb, Dirk Reinhardt, Jonas Abrahamsson, Souichi Adachi, Richard Aplenc, Eveline S.J.M. De Bont, Barbara De Moerloose, Michael Dworzak, Brenda E.S. Gibson, Henrik Hasle, Guy Leverger, Franco Locatelli, Christine Ragu, Raul C. Ribeiro, Carmelo Rizzari, Jeffrey E. Rubnitz, Owen P. Smith, Lillian Sung, Daisuke Tomizawa, Marry M. van den Heuvel-Eibrink, Ursula Creutzig, Gertjan J.L. Kaspers Journal of Clinical Oncology, 2015
Childhood high-risk acute lymphoblastic leukemia in first remission: Results after chemotherapy or transplant from the AIEOP ALL 2000 study Valentino Conter, Maria Grazia Valsecchi, Rosanna Parasole, Maria Caterina Putti, Franco Locatelli, Elena Barisone, Luca Lo Nigro, Nicola Santoro, Maurizio Aricò, Ottavio Ziino, Andrea Pession, Anna Maria Testi, Concetta Micalizzi, Fiorina Casale, Marco Zecca, Gabriella Casazza, Paolo Tamaro, Gaetano La Barba, Lucia Dora Notarangelo, Daniela Silvestri, Antonella Colombini, Carmelo Rizzari, Andrea Biondi, Giuseppe Masera, Giuseppe Basso Blood, 2014
Clinical relevance of molecular aberrations in paediatric acute myeloid leukaemia at first relapse Costa Bachas, Gerrit Jan Schuurhuis, Dirk Reinhardt, Ursula Creutzig, Zinia J. Kwidama, C. Michel Zwaan, Marry M. van den Heuvel‐Eibrink, Evelina S. J. M. De Bont, Sarah Elitzur, Carmelo Rizzari, Valérie de Haas, Martin Zimmermann, Jacqueline Cloos, Gertjan J. L. Kaspers British Journal of Haematology, 2014
SETIL: Italian multicentric epidemiological case-control study on risk factors for childhood leukaemia, non hodgkin lymphoma and neuroblastoma: study population and prevalence of risk factors in Italy Corrado Magnani, Stefano Mattioli, Lucia Miligi, Alessandra Ranucci, Roberto Rondelli, Alberto Salvan, Luigi Bisanti, Giuseppe Masera, Carmelo Rizzari, Paola Zambon, Santina Cannizzaro, Lorenzo Gafà, Lia Lidia Luzzatto, Alessandra Benvenuti, Paola Michelozzi, Ursula Kirchmayer, Pierluigi Cocco, Pierfranco Biddau, Claudia Galassi, Egidio Celentano, Erni Guarino, Giorgio Assennato, Gigliola de Nichilo, Domenico Franco Merlo, Vittorio Bocchini, Franco Pannelli, Paola Mosciatti, Liliana Minelli, Manuela Chiavarini, Marina Cuttini, Veronica Casotto, Maria Valeria Torregrossa, Rosalia Maria Valenti, Francesco Forastiere, Riccardo Haupt, Susanna Lagorio, Serena Risica, Alessandro Polichetti Italian Journal of Pediatrics, 2014
Detection of PICALM-MLLT10 (CALM-AF10) and outcome in children with T-lineage acute lymphoblastic leukemia L Lo Nigro, E Mirabile, M Tumino, C Caserta, G Cazzaniga, C Rizzari, D Silvestri, B Buldini, E Barisone, F Casale, M Luciani, F Locatelli, C Messina, C Micalizzi, A Pession, R Parasole, N Santoro, G Masera, G Basso, M Aricò, M Valsecchi, A Biondi, V Conter, on behalf of AIEOP—Scientific Committee of ALL Leukemia, 2013
Ancestry and pharmacogenetics of antileukemic drug toxicity Shinji Kishi, Cheng Cheng, Deborah French, Deqing Pei, Soma Das, Edwin H. Cook, Nobuko Hijiya, Carmelo Rizzari, Gary L. Rosner, Tony Frudakis, Ching-Hon Pui, William E. Evans, Mary V. Relling Blood, 2007
T-immunophenotype is associated with an increased prevalence of thrombosis in children with acute lymphoblastic leukemia. A retrospective study Haematologica, 2003
High-dose cytosine arabinoside and fractionated total body irradiation as a preparative regimen for the treatment of children with acute lymphoblastic leukemia and Down syndrome by allogeneic bone marrow transplantation Bone Marrow Transplantation, 1996
Extended intrathecal methotrexate may replace cranial irradiation for prevention of CNS relapse in children with intermediate-risk acute lymphoblastic leukemia treated with Berlin-Frankfurt-Münster-based intensive chemotherapy Journal of Clinical Oncology, 1995
Central venous catheter-related complications after bone marrow transplantation in children with hematological malignancies Bone Marrow Transplantation, 1992
Bone marrow transplantation for childhood leukemia: Five years' experience in a pediatric hematology center Haematologica, 1992
Microgranular variant of acute promyelocytic leukemia in children Journal of Clinical Oncology, 1992
