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儿童急性白血病的干细胞移植后维持治疗:来自巴西某机构的经验与拉丁美洲视角

英文原题:Post-stem cell transplant maintenance for pediatric acute leukemias: insights from a Brazilian institution with a Latin American perspective.

查看英文原题

Post-stem cell transplant maintenance for pediatric acute leukemias: insights from a Brazilian institution with a Latin American perspective.

PubMed 2025/03/05(内容时间) Front Oncol Q2 · IF 3.4(JCR 2025)

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研究概要

HSCT 后维持治疗在资源有限的环境中可行,并可能改善生存结局。

中文摘要

本回顾性研究纳入 2014 至 2024 年接受造血干细胞移植(HSCT)的儿童急性白血病患者。自 2021 年起,HSCT 后维持治疗成为标准实践,使用药物包括维奈克拉、地西他滨、阿扎胞苷、贝林妥欧单抗、供者淋巴细胞输注(DLI)及靶向治疗。主要结局为总生存期(OS)和无病生存期(DFS);次要结局包括复发率和治疗相关毒性。

共纳入 94 例患者,其中急性淋巴细胞白血病(ALL)64 例、急性髓系白血病(AML)30 例。接受维持治疗的 ALL 患者 OS 为 78%,未接受维持治疗者为 47%(p = 0.02);DFS 分别为 64% 和 45%(p = 0.12)。AML 患者中,维持治疗与 OS 88% 相关,而未接受维持治疗者为 27%。维持治疗患者复发率降低,在移植前微小残留病(MRD)阳性的 AML 患者中尤为明显。治疗总体耐受性良好,毒性可控。讨论:资源有限环境中,HSCT 后维持治疗可行,并可能改善生存结局。T-ALL 使用去甲基化药物联合维奈克拉、B-ALL 在 HSCT 后使用贝林妥欧单抗等策略显示潜在获益。挑战包括药物可及性和治疗方案标准化。仍需进一步试验在低收入和中等收入国家验证这些发现。

展开英文摘要原文

This retrospective study included pediatric acute leukemia patients who underwent HSCT between 2014 and 2024. Post-HSCT maintenance therapy became standard practice in 2021, utilizing agents like venetoclax, decitabine, azacitidine, blinatumomab, DLI, and targeted therapies. Primary outcomes were overall survival (OS) and disease-free survival (DFS); secondary outcomes included relapse rate and treatment-related toxicities.

Among 94 patients (64 with ALL, 30 with AML), ALL patients receiving maintenance therapy had an OS of 78% versus 47% without maintenance (p=0.02); DFS was 64% with maintenance and 45% without (p=0.12). In AML patients, maintenance was associated with an OS of 88% compared to 27% without. Relapse rates decreased in maintenance-treated patients, especially among AML patients with pre-transplant MRD positivity. Treatments were generally well-tolerated, with manageable toxicities. DISCUSSION: Post-HSCT maintenance therapy is feasible in resource-limited settings and may improve survival outcomes. Strategies like hypomethylating agents with venetoclax in T-ALL and post-HSCT blinatumomab in B-ALL show potential benefits. Challenges include drug access and standardizing protocols. Further trials are needed to validate these findings in low- and middle-income countries.

论文信息

作者
Breviglieri CNM、Gouveia RV、Ginani VC、Santos CN、de Oliveira MRS、Batalha ABW、de Alencar GS、Goto EH
单位
Department of Pediatric Hematology and Hematopoietic Stem Cell Transplantation, Hospital Samaritano Higienópolis, São Paulo, Brazil.Brazil
期刊
Frontiers in oncology2025
原文标识
PubMed 40110204 · DOI 10.3389/fonc.2025.1540158