Czogała Małgorzata, Pawińska-Wa Sikowska Katarzyna, Ksia Żek Teofila, Sikorska-Fic Barbara, Matysiak Michał, Rodziewicz-Konarska Anna, Chybicka Alicja, Skalska-Sadowska Jolanta, Wachowiak Jacek, Muszyńska-Rosłan Katarzyna, Krawczuk-Rybak Maryna, Grabowski Dominik, Kowalczyk Jerzy, Zielezińska Karolina, Urasiński Tomasz, Tomaszewska Renata, Szczepański Tomasz, Karpińska-Derda Irena, Woszczyk Mariola, Pohorecka Joanna, Karolczyk Grażyna, Młynarski Wojciech, Mycko Katarzyna, Badowska Wanda, Skoczeń Szymon, Balwierz Walentyna
Department of Pediatric Oncology and Hematology, Institute of Pediatrics, Jagiellonian University Medical College, Krakow, Poland.
Department of Pediatric Oncology and Hematology, University Children Hospital, Krakow, Poland.
Front Pediatr. 2020 Mar 20;8:86. doi: 10.3389/fped.2020.00086. eCollection 2020.
The aim of the study was to analyze the treatment outcome and genetic characteristics of acute promyelocytic leukemia (APL) in children in Poland from 2005 to 2018. All 41 patients diagnosed with APL in Poland during the analysis period were eligible for the study. In period I (2005-2015), 33 patients were treated with chemotherapy and all-trans retinoic acid (ATRA), and in period II (2015-2018), 3 patients (high risk) received induction chemotherapy with ATRA and arsenic trioxide (ATO), and 5 patients (standard risk) received ATRA and ATO without chemotherapy. Probability of 5-years overall survival (OS), event-free survival (EFS), and relapse-free survival (RFS) was 0.819 ± 0.069, 0.831 ± 0.063, and 0.961 ± 0.037, respectively, in the whole cohort. Four (11%) early deaths were observed. One patient died of severe infection in the course of disease progression. Relapse occurred in one patient, who died finally because of disease progression. All events occurred in the patients from period I. Variant APL was identified in one patient (successfully treated with chemotherapy with ATRA) and complex translocation in one patient (the only patient with relapse). Additional chromosomal aberrations were found in 26% of patients and FLT3-ITD mutation was detected in 44% of patients; none of those changes influenced clinical outcome. Treatment outcome in the analyzed group is similar to the results reported by other study groups. The main cause of death was coagulation disorders in the early stage of disease. Early, accurate diagnosis followed by specific treatment enables the reduction in the number of early deaths.
该研究的目的是分析2005年至2018年波兰儿童急性早幼粒细胞白血病（APL）的治疗结果和基因特征。分析期间在波兰诊断为APL的所有41例患者均符合该研究的条件。在第一阶段（2005 - 2015年），33例患者接受了化疗和全反式维甲酸（ATRA）治疗，在第二阶段（2015 - 2018年），3例（高危）患者接受了ATRA和三氧化二砷（ATO）诱导化疗，5例（标危）患者接受了ATRA和ATO且未进行化疗。整个队列中5年总生存率（OS）、无事件生存率（EFS）和无复发生存率（RFS）分别为0.819±0.069、0.831±0.063和0.961±0.037。观察到4例（11%）早期死亡。1例患者在疾病进展过程中死于严重感染。1例患者复发，最终因疾病进展死亡。所有事件均发生在第一阶段的患者中。1例患者被鉴定为变异型APL（成功接受ATRA化疗治疗），1例患者存在复杂易位（唯一复发的患者）。26%的患者发现了额外的染色体畸变，44%的患者检测到FLT3 - ITD突变；这些改变均未影响临床结果。分析组的治疗结果与其他研究组报告的结果相似。死亡的主要原因是疾病早期的凝血障碍。早期、准确的诊断并随后进行特异性治疗能够减少早期死亡的数量。
