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Clinical characteristics and outcome of childhood acute promyelocitic leukemia (APL) in Saudi Arabia: a multicenter SAPHOS leukemia group study.沙特阿拉伯儿童急性早幼粒细胞白血病(APL)的临床特征与转归:一项多中心沙特阿拉伯儿科血液学与肿瘤学学会(SAPHOS)白血病组研究
Hematology. 2018 Jul;23(6):316-323. doi: 10.1080/10245332.2017.1412380. Epub 2017 Dec 7.
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Predictors of thrombohemorrhagic early death in children and adolescents with t(15;17)-positive acute promyelocytic leukemia treated with ATRA and chemotherapy.接受全反式维甲酸(ATRA)和化疗治疗的t(15;17)阳性急性早幼粒细胞白血病儿童及青少年血栓出血性早期死亡的预测因素
Ann Hematol. 2017 Sep;96(9):1449-1456. doi: 10.1007/s00277-017-3042-6. Epub 2017 Jun 8.
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Acute promyelocytic leukemia in children and adolescents.儿童和青少年急性早幼粒细胞白血病。
Acta Haematol. 2014;132(3-4):307-12. doi: 10.1159/000365117. Epub 2014 Sep 10.
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Acute Promyelocytic Leukemia (APL): Comparison Between Children and Adults.急性早幼粒细胞白血病(APL):儿童与成人的比较
Mediterr J Hematol Infect Dis. 2014 Apr 15;6(1):e2014032. doi: 10.4084/MJHID.2014.032. eCollection 2014.
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Retinoic acid and arsenic trioxide for acute promyelocytic leukemia.全反式维甲酸和三氧化二砷联合治疗急性早幼粒细胞白血病。
N Engl J Med. 2013 Jul 11;369(2):111-21. doi: 10.1056/NEJMoa1300874.
6
Improving acute promyelocytic leukemia (APL) outcome in developing countries through networking, results of the International Consortium on APL.通过建立网络改善发展中国家的急性早幼粒细胞白血病(APL)预后:国际 APL 联盟的研究结果。
Blood. 2013 Mar 14;121(11):1935-43. doi: 10.1182/blood-2012-08-449918. Epub 2013 Jan 14.
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Retrospective analysis of 119 cases of pediatric acute promyelocytic leukemia: Comparisons of four treatment regimes.119例儿童急性早幼粒细胞白血病的回顾性分析:四种治疗方案的比较
Exp Ther Med. 2012 Jul;4(1):93-98. doi: 10.3892/etm.2012.546. Epub 2012 Apr 17.
8
Subclinical anthracycline cardiotoxicity in patients with acute promyelocytic leukemia in long-term remission after the AIDA protocol.接受AIDA方案治疗后长期缓解的急性早幼粒细胞白血病患者的亚临床蒽环类药物心脏毒性
Congest Heart Fail. 2012 Jul-Aug;18(4):217-21. doi: 10.1111/j.1751-7133.2011.00278.x. Epub 2012 Jan 9.
9
All-trans-retinoic acid, idarubicin, and IV arsenic trioxide as initial therapy in acute promyelocytic leukemia (APML4).全反式维甲酸、去甲氧柔红霉素和静脉注射三氧化二砷作为急性早幼粒细胞白血病(APML4)的初始治疗。
Blood. 2012 Aug 23;120(8):1570-80; quiz 1752. doi: 10.1182/blood-2012-02-410746. Epub 2012 Jun 19.
10
Outcome of acute promyelocytic leukemia (APL) in children and adolescents: an analysis in two consecutive trials of the European APL Group.儿童和青少年急性早幼粒细胞白血病(APL)的结果:欧洲 APL 组两项连续试验的分析。
J Clin Oncol. 2012 May 10;30(14):1641-6. doi: 10.1200/JCO.2011.38.4560. Epub 2012 Apr 2.

全反式维甲酸、三氧化二砷及低剂量蒽环类药物治疗儿童急性早幼粒细胞白血病的疗效分析

An Outcome Analysis of Childhood Acute Promyelocytic Leukemia Treated with Atra and Arsenic Trioxide, and Limited Dose Anthracycline.

作者信息

Jabbar Naeem, Khayyam Naema, Arshad Uzma, Maqsood Sidra, Hamid Syed Ahmer, Mansoor Neelum

机构信息

Pediatric Hematology-Oncology Section of Pediatric Department, The Indus Hospital, Plot C-76, Sector 31/5, Opposite Darussalam Society, Korangi Crossing, Karachi, 75190 Pakistan.

Jinnah Medical College Hospital, Karachi, Pakistan.

出版信息

Indian J Hematol Blood Transfus. 2021 Oct;37(4):569-575. doi: 10.1007/s12288-021-01404-1. Epub 2021 Jan 30.

DOI:10.1007/s12288-021-01404-1
PMID:34744341
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8523738/
Abstract

The overall survival of Acute Promyelocytic Leukemia (APL), reported in recent studies, is approaching to 90% wherein, arsenic trioxide (ATO) and all-trans retinoic acid (ATRA) are used as the mainstay of treatment with either limited or no use of anthracycline and cytarabine. This study is aimed to ascertain the outcome of children with APL using similar approach. A total of 30 patients with APL, registered from January 2015 to December 2018, were reviewed. Diagnosis was established on bone marrow aspirate and confirmed by the presence of PML-RARA translocation. Treatment protocol was based on Australian APML 4 study performed by Australian Leukemia Lymphoma Group (ALLG). Lumbar puncture was not performed as it was not part of the protocol due to the risk of bleeding. The mean age in current cohort was 9 years with 53% males. Seven (23.3%) patients died and three (10%) abandoned treatment during induction. Twenty patients completed the intensive phase of chemotherapy and all (100%) of them attained molecular remission (MR). One patient dropped out after MR whereas, 19 remain on follow up with no evidence of disease, reflecting disease free survival (DFS) of 95%. With a median follow up of 2.5 years (range 2.1-4.8 years) the 5 years Kaplan-Meier estimate of OS was 63% and 73%, with and without abandonment, respectively. Analysis of outcome according to risk groups revealed inferior outcome of high risk (HR) group (38% and 50% with and without abandonment, respectively) in contrast to standard risk (SR) group which showed better outcome (82% and 88% with and without abandonment, respectively). The attainment of 100% molecular remission and absence of relapse supports the effectiveness of this regimen. Moreover, it is found to be less toxic and therefore, can be conveniently managed in day-care settings.

摘要

近期研究报道,急性早幼粒细胞白血病(APL)的总生存率接近90%,其中三氧化二砷(ATO)和全反式维甲酸(ATRA)被用作主要治疗药物,蒽环类药物和阿糖胞苷的使用有限或未使用。本研究旨在确定采用类似方法治疗的APL患儿的治疗结果。回顾了2015年1月至2018年12月登记的30例APL患者。通过骨髓穿刺确诊,并经PML-RARA易位证实。治疗方案基于澳大利亚白血病淋巴瘤组(ALLG)进行的澳大利亚APML 4研究。由于存在出血风险,腰椎穿刺未作为方案的一部分进行。当前队列的平均年龄为9岁,男性占53%。7例(23.3%)患者在诱导期死亡,3例(10%)放弃治疗。20例患者完成了强化化疗阶段,所有患者(100%)均达到分子缓解(MR)。1例患者在达到MR后退出,19例仍在随访中,无疾病证据,无病生存率(DFS)为95%。中位随访2.5年(范围2.1 - 4.8年),5年Kaplan-Meier估计的总生存率(OS)分别为63%(未放弃治疗)和73%(放弃治疗)。根据风险组分析结果显示,高危(HR)组的结果较差(放弃治疗和未放弃治疗的分别为38%和50%),而标准风险(SR)组的结果较好(放弃治疗和未放弃治疗的分别为82%和88%)。100%的分子缓解率和无复发支持了该方案的有效性。此外,发现该方案毒性较小,因此可以在日间护理环境中方便地进行管理。