Samborska Magdalena, Skalska-Sadowska Jolanta, Achkar Robert, Wachowiak Jacek, Derwich Katarzyna, Czogała Małgorzata, Balwierz Walentyna, Skoczeń Szymon, Dobaczewski Grzegorz, Chybicka Alicja, Kałwak Krzysztof, Krawczuk-Rybak Maryna, Muszyńska-Rosłan Katarzyna, Adamkiewicz-Drożyńska Elżbieta, Maciejka-Kapuscińska Lucyna, Irga-Jaworska Ninela, Pohorecka Joanna, Chodała-Grzywacz Agnieszka, Karolczyk Grażyna, Wójcik Beata, Kowalczyk Jerzy R, Drabko Katarzyna, Zawitkowska Joanna, Mycko Katarzyna, Badowska Wanda, Ociepa Tomasz, Urasiński Tomasz, Sikorska-Fic Barbara, Matysiak Michał, Laguna Paweł, Dąbrowska-Pawliszyn Anna, Tomaszewska Renata, Szczepański Tomasz, Sobol Grażyna, Mizia-Malarz Agnieszka, Ciebiera Małgorzata, Chaber Radosław, Kołtan Sylwia, Wysocki Mariusz, Styczyński Jan, Woszczyk Mariola, Wieczorek Maria, Karpińska-Derda Irena, Urbańska-Rakus Justyna, Bobeff Katarzyna, Trelińska Joanna, Młynarski Wojciech
Department of Pediatric Hematology, Oncology and Transplantology, University of Medical Sciences, Poznań, Poland.
University of Medical Sciences, Poznań, Poland.
Contemp Oncol (Pozn). 2023;27(4):249-254. doi: 10.5114/wo.2023.135327. Epub 2024 Feb 12.
This study aimed to present the clinical features and results of treatment of patients diagnosed with refractory or relapsed acute myeloid leukaemia (AML) in Polish Paediatric Leukaemia/Lymphoma Study Group (PPL/LSG) institutions, treated in accordance with the Protocol Acute Myeloid Leukaemia Berlin-Frankfurt-Munster 2012, as their first-line therapy.
The outcome data of 10 patients with refractory AML (median age 9.5 years) and 30 with relapsed AML (median age 12 years) were analysed retrospectively. Re-induction was usually based on idarubicin, fludarabine, and cytarabine along with allogeneic haematopoietic stem cell transplant (allo-HSCT) in 5 patients with refractory AML and 7 relapsed AML children.
37.5% (3/8) of refractory AML patients achieved second complete remission second complete remission (CRII). One of ten patients (1/10; 10%) was alive and stayed in complete remission for 34 months after the allo-HSCT. The probability of 3-year event-free survival (pEFS) in this group was 0.125 ±0.11. In the group of relapsed AML patients, the CRII was achieved in 9 patients (34%), and the probability of survival was: pEFS = 0.24 ±0.08; probability overall survival (pOS) = 0.34 ±0.09, with significantly better results achieved in patients who underwent allo-HSCT (pOS = 0.54 ±0.14 vs. 0.08 ±0.08, < 0.0001).
The prognosis of refractory AML and the first AML recurrence in children who were first-line treated in PPL/LSG centres according to Protocol Acute Myeloid Leukaemia Berlin-Frankfurt-Munster 2012 is poor. Failures of re-induction treatment particularly result from difficulties in achieving remission. Allogeneic HSCT improves prognosis in children with refractory and first recurrent AML, under the condition it is performed in complete remission. Novel therapeutic approaches are needed to increase the remission rate and improve the outcomes.
本研究旨在呈现波兰儿童白血病/淋巴瘤研究组（PPL/LSG）机构中被诊断为难治性或复发性急性髓系白血病（AML）的患者的临床特征及治疗结果，这些患者按照《2012年柏林 - 法兰克福 - 明斯特急性髓系白血病方案》接受一线治疗。
回顾性分析10例难治性AML患者（中位年龄9.5岁）和30例复发性AML患者（中位年龄12岁）的结局数据。再诱导治疗通常基于伊达比星、氟达拉滨和阿糖胞苷，5例难治性AML患者和7例复发性AML患儿接受了异基因造血干细胞移植（allo - HSCT）。
37.5%（3/8）的难治性AML患者实现了第二次完全缓解（CRII）。10例患者中有1例（1/10；10%）存活，在allo - HSCT后持续完全缓解34个月。该组3年无事件生存率（pEFS）为0.125±0.11。在复发性AML患者组中，9例患者（34%）实现了CRII，生存率为：pEFS = 0.24±0.08；总生存率（pOS） = 0.34±0.09，接受allo - HSCT的患者结果明显更好（pOS = 0.54±0.14对0.08±0.08，<0.0001）。
在PPL/LSG中心按照《2012年柏林 - 法兰克福 - 明斯特急性髓系白血病方案》进行一线治疗的儿童难治性AML和首次AML复发的预后较差。再诱导治疗失败尤其源于难以实现缓解。异基因HSCT可改善难治性和首次复发AML儿童的预后，条件是在完全缓解时进行。需要新的治疗方法来提高缓解率并改善结局。
