早期死亡仍然是儿童急性早幼粒细胞白血病良好生存率的障碍:一项对62例患者的回顾性研究,时间跨度为17年。

IF 16.4 1区 化学 Q1 CHEMISTRY, MULTIDISCIPLINARY
Pritam Singha Roy, Vinay Munikoty, Amita Trehan, Richa Jain, Prateek Bhatia, Shano Naseem, Neelam Varma, Deepak Bansal
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引用次数: 3

摘要

来自低收入和中等收入国家的儿童急性早幼粒细胞白血病(APL)数据有限。早期死亡是一个令人担忧的问题,但在临床试验中往往没有得到重视。回顾性研究是在印度的一个中心对2003年至2021年的APL患者(≤12岁)进行的。患者接受全反式维甲酸(ATRA)和化疗。入组后3个疗程巩固,随访2年。2015年,该方案更新了以下修改:(a)在诱导结束时而不是在诊断时获得诊断性脑脊液,(b)无论风险类别如何给予鞘内阿糖胞苷,(c)风险分层化疗,(d)在所有巩固周期中纳入ATRA。在17年的时间里,62名患者被确诊。中位年龄为8岁(范围:0.9-12岁)。其中一半患有高危疾病。32%出现分化证,无死亡病例。18例(29%)患者死于出血(83%)或败血症(17%)。13例(21%)早期死亡(≤15天),均因出血。血小板计数9/L预测早期死亡(优势比:4.5;95% CI: 0.9 ~ 22, p = 0.06)。治疗放弃从2003-2015年的23.5%降至2015-2021年的零(p = 0.006)。3例(8%)患者复发。所有患者的4年OS为70.1%,存活>15天的患者为89.6%。更新方案之前和之后的4年EFS(包括遗弃和早期死亡率)分别为61.4%和65.5% (p = 0.77)。早期死亡仍然是儿童APL良好生存的障碍。近年来治疗放弃的显著减少令人欣慰。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Early mortality continues to be a barrier to excellent survival in childhood acute promyelocytic leukemia: a retrospective study of 62 patients spanning 17 years.

Data on childhood acute promyelocytic leukemia (APL) from low-and middle-income countries is limited. Early mortality is a concern and often not highlighted in clinical trials. The retrospective study was conducted on patients (≤12 years) with APL from 2003 to 2021 at a single center in India. Patients were treated with all-trans-retinoic acid (ATRA) and chemotherapy. Induction and three courses of consolidation were followed by maintenance for 2 years. In 2015, the protocol was updated with following modifications: (a) obtaining diagnostic cerebrospinal fluid at end-of-induction rather than at diagnosis, (b) administering intrathecal cytarabine regardless of risk-category, (c) risk-stratified administration of chemotherapy, and (d) inclusion of ATRA in all the cycles of consolidation. Sixty-two patients were diagnosed over the 17 years. The median age was 8 years (range: 0.9-12). Half had high-risk disease. Differentiation syndrome was observed in 32%, none being fatal. Eighteen (29%) patients died due to hemorrhage (83%) or septicemia (17%). Thirteen (21%) had early mortality (≤15 days), all due to hemorrhage. A platelet count <20 × 109/L predicted early mortality (odds ratio: 4.5; 95% CI: 0.9-22, p = 0.06). Treatment abandonment reduced from 23.5% during 2003-2015 to nil during 2015-2021 (p = 0.006). Three (8%) patients relapsed. The 4-year OS of all patients and the patients who survived >15 days was 70.1% and 89.6%, respectively. The 4-year EFS, including abandonment and early mortality, before and following updated protocol, was 61.4% and 65.5%, respectively (p = 0.77). Early mortality continues to be a barrier to an otherwise excellent survival in childhood APL. A significant reduction in treatment abandonment in recent years is gratifying.

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来源期刊
Accounts of Chemical Research
Accounts of Chemical Research 化学-化学综合
CiteScore
31.40
自引率
1.10%
发文量
312
审稿时长
2 months
期刊介绍: Accounts of Chemical Research presents short, concise and critical articles offering easy-to-read overviews of basic research and applications in all areas of chemistry and biochemistry. These short reviews focus on research from the author’s own laboratory and are designed to teach the reader about a research project. In addition, Accounts of Chemical Research publishes commentaries that give an informed opinion on a current research problem. Special Issues online are devoted to a single topic of unusual activity and significance. Accounts of Chemical Research replaces the traditional article abstract with an article "Conspectus." These entries synopsize the research affording the reader a closer look at the content and significance of an article. Through this provision of a more detailed description of the article contents, the Conspectus enhances the article's discoverability by search engines and the exposure for the research.
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