Gaál Zsuzsanna, Jakab Zsuzsanna, Kárai Bettina, Ujfalusi Anikó, Petrás Miklós, Kállay Krisztián, Kelemen Ágnes, Simon Réka, Kriván Gergely, Kovács Gábor T, Kiss Csongor, Szegedi István
Department of Pediatric Hematology-Oncology, Institute of Pediatrics, University of Debrecen, 4032 Debrecen, Hungary.
National Childhood Cancer Registry, 1094 Budapest, Hungary.
Cancers (Basel). 2021 Oct 11;13(20):5078. doi: 10.3390/cancers13205078.
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% ( = 0.057), and the 2 y EFS increased significantly from 56.4% to 68.9% ( = 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.
1990年至2011年间，匈牙利小儿急性髓系白血病（AML）的预后指标有了显著改善。自2012年以来，匈牙利小儿肿瘤血液学组（HPOG）的工作包括减少治疗中心数量、采用当代诊断程序、大力支持、增加造血干细胞移植（HSCT）和靶向治疗的可及性。我们研究的主要目的是评估HPOG在2012年至2019年间对92例新登记患者（52例男性，40例女性，平均年龄7.28岁）的AML治疗结果。分为两个时期：2012 - 2015年和2016 - 2019年（分别为55例和37例患者）。在此期间，2年总生存率从63.6%提高到71.4%（P = 0.057），2年无事件生存率从56.4%显著提高到68.9%（P = 0.02）。37例患者接受了HSCT（5例患者接受了第二次HSCT）。我们展示了两例急性早幼粒细胞白血病（APL）诊断和治疗方面的进展。通过多维流式细胞术和先进分子方法实现了早期诊断和随访。除化疗外，两名患者均成功接受了全反式维甲酸和三氧化二砷治疗。为了达到小儿AML管理的国际标准，HPOG将进一步集中治疗中心和诊断设施，并与国际研究组共同努力。
