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Simple Summary The outcome of pediatric AML improved considerably worldwide during the past few decades. Hereby, we summarize the therapeutic results of pediatric AML patients registered between 2012 and 2019 in Hungary. As compared to our previous results, improvement was registered in event-free (EFS) and overall (OS) survival, which can be attributed to the application of contemporary diagnostic and therapeutic guidelines, advanced supportation, and higher efficacy of hematopoietic stem cell transplantation. Between 2016 and 2019, a statistically significant increment of 2-year EFS was confirmed over the period between 2012 and 2015. The most prominent progress was observed in acute promyelocytic leukemia (APL). Multidimensional flow cytometry made possible the prompt introduction of ATRA in two cases with M3v, who also represent the first pediatric APL patients in Hungary to be treated with arsenic-trioxide. Besides joining multinational pediatric AML treatment groups, our future aims include the introduction of centralized treatment centers and diagnostic facilities. Abstract Outcome measures of pediatric acute myeloid leukemia (AML) improved considerably between 1990 and 2011 in Hungary. Since 2012, efforts of the Hungarian Pediatric Oncology-Hematology Group (HPOG) included the reduction in the number of treatment centers, contemporary diagnostic procedures, vigorous supportation, enhanced access to hematopoietic stem cell transplantation (HSCT), and to targeted therapies. The major aim of our study was to evaluate AML treatment results of HPOG between 2012 and 2019 with 92 new patients registered (52 males, 40 females, mean age 7.28 years). Two periods were distinguished: 2012–2015 and 2016–2019 (55 and 37 patients, respectively). During these periods, 2 y OS increased from 63.6% to 71.4% (p = 0.057), and the 2 y EFS increased significantly from 56.4% to 68.9% (p = 0.02). HSCT was performed in 37 patients (5 patients received a second HSCT). We demonstrate advances in the diagnosis and treatment of acute promyelocytic leukemia (APL) in two cases. Early diagnosis and follow-up were achieved by multidimensional flow cytometry and advanced molecular methods. Both patients were successfully treated with all-trans retinoic acid and arsenic-trioxide, in addition to chemotherapy. In order to meet international standards of pediatric AML management, HPOG will further centralize treatment centers and diagnostic facilities and join efforts with international study groups.

Abstract: Introduction: Acquired bone marrow failures are rare but fatal diseases in childhood. Since 2013, Hungary has been participating as a full member in the work of the European Working Group on uniform diagnostics and therapy in patients with acquired bone marrow failure syndromes. Hypocellular refractory cytopenia of childhood has been emphasized as a frequent entity, transplanted by reduced intensity conditioning with excellent outcomes. Aim: To analyse and compare the results of treatment before and after our joining. Method: A total of 55 patients have been treated in the 8 centres of the Hungarian Pediatric Oncology Network during 5 years between 2013 and 2017 (severe aplastic anemia: 9, myelodysplastic syndrome: 41, juvenile myelomonocytic leukemia: 5 patients). Allogeneic hematopoietic stem cell transplantation was performed in severe aplastic anemia in 7 cases, while antithymocyte globulin was administered in one case and one patient died before diagnosis. In patients with myelodysplastic syndromes, watch and wait strategy was applied in 4, while transplantation in 37 cases. Reduced intensity conditioning was used in 54 percent of these cases. Transplantation was the treatment of choice in all 5 patients with juvenile myelomonocytic leukemia. Results: In the whole patient cohort, the time from diagnosis to treatment was median 92 (3–393) days, while in severe aplastic anemia median 28 (3–327) days only. Grade II–IV acute graft versus host disease occurred in 22.6%, grade III–IV in 6.8% and chronic in 11.2%. All the patients treated with severe aplastic anemia are alive and in complete remission (100%). The overall estimated survival rate is 85.1% in myelodysplastic syndrome, while 75% in juvenile myelomonocytic leukemia. The median follow-up was 30.4 (1.1–62.5) months. There was a remarkable increase in overall survival comparing the data before (1992–2012) and after (2013) joining the international group, 70% vs. 100% (p = 0.133) in severe aplastic anemia and 31.3% vs. 85.1% (p = 0.000026) in myelodysplastic syndrome. Conclusion: Due to a change in the paradigm of the conditioning regimen in hypocellular refractory cytopenia of childhood, the overall survival rate has significantly increased. Orv Hetil. 2018; 159(42): 1710–1719.

Two rare cases of large cell neuroblastoma (LCN) are reported. Case 1 (8-year-old male) showed the appearance of Neuroblastoma, poorly differentiated subtype with a high MKI (Mitosis-Karyorrhexis Index) and Case 2 (7-year-old male) was Neuroblastoma, undifferentiated subtype with a low MKI. Both cases were classified into the Unfavorable Histology Group according to the International Neuroblastoma Pathology Classification and their tumors were characteristically composed of neuroblastic cells with enlarged and often pale or vacuolated nuclei containing one or few prominent nucleoli. While LCN is a phenotypical variant of MYC-driven neuroblastoma overexpressing MYC-family protein, the two tumors presented in this report had different molecular characteristics: One had n-MYC oncogene amplification with n-MYC protein overexpression (Case 1), and the other had c-MYC protein overexpression without genomic amplification (Case 2). It was also noted that the tumor cells in Case 2 demonstrated “aberrant” desmin expression by immunostaining.