CAR-T(CAR-T)细胞疗法在非肿瘤性疾病中的应用
Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
英文原题:Post-stem cell transplant maintenance for pediatric acute leukemias: insights from a Brazilian institution with a Latin American perspective.
Post-stem cell transplant maintenance for pediatric acute leukemias: insights from a Brazilian institution with a Latin American perspective.
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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。
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。
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.
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