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儿童复发或难治性急性淋巴细胞白血病治疗结果:单中心三级护理中心经验。

Outcomes of children treated for relapsed or refractory acute lymphoblastic leukemia: A single tertiary care center experience.

机构信息

Oncology Department, King Faisal Specialist Hospital and Research Centre, Jeddah, Saudi Arabia.

Department of Diagnostic Radiography Technology, College of Applied Medical Sciences, Jazan University, Jazan, Saudi Arabia.

出版信息

Cancer Rep (Hoboken). 2024 Jul;7(7):e2117. doi: 10.1002/cnr2.2117.

DOI:10.1002/cnr2.2117
PMID:39051567
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11270327/
Abstract

BACKGROUND

Acute lymphoblastic leukemia (ALL) is one of the most common malignancies among children. Despite success in frontline treatment, 20% of children will relapse or show resistance to treatment.

AIM

The aim of this study is to evaluate the clinical characteristics of children diagnosed and treated for refractory or relapsed ALL and determine 3-year overall survival (OS) outcomes.

METHOD

This study involved a retrospective chart review of patients aged 1-14 years diagnosed with ALL during January 2002 to December 2018. Data were extracted for baseline characteristics at diagnosis and at relapse.

RESULTS

A total of 347 newly diagnosed children with ALL were identified, among whom there were three induction failures (IF) and 28 relapses, resulting in a cohort of 31 patients with a relapse rate of 9%. The male-to-female ratio was 4.16:1, and the mean duration of first complete remission (CR1) was 26 months. Fifteen (48%) patients relapsed ≤18 months, 7 (23%) during 18-36 months, and 9 (29%) relapsed >36 months of IF or CR1. Nineteen patients (61%) had isolated bone marrow (BM) relapse, 7 (23%) patients experienced isolated extramedullary relapse (5 isolated CNS relapse and 2 isolated testicular relapse), and 5 (16%) patients experienced BM involvement with other sites (4 BM + CNS and 1 BM + testis). The 3-year OS of the cohort was 62.3%, while in patients with CR post first-salvage therapy, a 3-year OS of 79.5% was observed (p value <.05 compared with patients who did not achieve remission post first-salvage therapy, 3-year OS: 46.4%). The same statistical difference was observed in 3-year OS when comparing the duration of remission of CR prior to relapse: ≤18 months, 33.2%; 18-36 months, 66.7%; and >36 months, 87.5%. The same trend continued when comparing 3-year OS based on risk stratification at relapse: low risk (LR), 83.3%; intermediate risk (IR), 80%; and high risk (HR), 44.8%.

CONCLUSION

The incidence and outcomes reported are comparable to internationally reported data regarding the duration of CR1. Risk stratification at relapse and remission status post-salvage therapy were identified as significant prognostic factors for survival. No survival difference was observed among patients who received hematopoietic stem cell transplantation after induction compared with those who received chemotherapy, which could be attributed to the smaller sample size, warranting a multi-institutional observational study. These findings corroborate the need for novel therapies and treatment approaches.

摘要

背景

急性淋巴细胞白血病(ALL)是儿童中最常见的恶性肿瘤之一。尽管一线治疗取得了成功,但仍有 20%的儿童会复发或对治疗产生耐药性。

目的

本研究旨在评估诊断和治疗难治性或复发性 ALL 儿童的临床特征,并确定 3 年总生存率(OS)结果。

方法

本研究回顾性分析了 2002 年 1 月至 2018 年 12 月期间诊断为 ALL 的 1-14 岁患者的病历。提取基线特征数据,包括诊断时和复发时的特征。

结果

共确定了 347 例新诊断的 ALL 儿童,其中有 3 例诱导失败(IF)和 28 例复发,导致 31 例患者的复发率为 9%。男女比例为 4.16:1,首次完全缓解(CR1)的平均持续时间为 26 个月。15 例(48%)患者在 IF 或 CR1 后≤18 个月复发,7 例(23%)在 18-36 个月复发,9 例(29%)在>36 个月时复发。19 例(61%)患者为单纯骨髓(BM)复发,7 例(23%)患者为单纯髓外复发(5 例 CNS 孤立复发和 2 例睾丸孤立复发),5 例(16%)患者为 BM 合并其他部位受累(4 例 BM+CNS 和 1 例 BM+睾丸)。该队列的 3 年 OS 为 62.3%,而在首次挽救性治疗后获得 CR 的患者中,3 年 OS 为 79.5%(p 值<.05,与首次挽救性治疗后未缓解的患者相比,3 年 OS:46.4%)。在比较缓解持续时间≤18 个月、18-36 个月和>36 个月时,同样观察到 3 年 OS 的统计学差异:分别为 33.2%、66.7%和 87.5%。在比较基于复发时风险分层的 3 年 OS 时也出现了同样的趋势:低危(LR)为 83.3%、中危(IR)为 80%和高危(HR)为 44.8%。

结论

报告的发生率和结果与国际上关于 CR1 持续时间的报道相似。复发时的风险分层和挽救性治疗后的缓解状态被确定为生存的重要预后因素。与接受诱导化疗的患者相比,接受造血干细胞移植的患者之间未观察到生存差异,这可能归因于较小的样本量,需要进行多机构观察性研究。这些发现证实了需要新的治疗方法和治疗策略。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/22dc/11270327/3ff3008f3b1c/CNR2-7-e2117-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/22dc/11270327/2645d4344123/CNR2-7-e2117-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/22dc/11270327/3ff3008f3b1c/CNR2-7-e2117-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/22dc/11270327/2645d4344123/CNR2-7-e2117-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/22dc/11270327/3ff3008f3b1c/CNR2-7-e2117-g001.jpg

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