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采用MASPORE 2006方案治疗的急性髓系白血病患儿生存结局良好。

Excellent Survival Outcomes of Pediatric Patients With Acute Myeloid Leukemia Treated With the MASPORE 2006 Protocol.

作者信息

Sutiman Natalia, Nwe Mya Soe, Ni Lai Eunice En, Lee Denyse Kawai, Chan Mei Yoke, Eng-Juh Yeoh Allen, Soh Shui Yen, Leung Wing, Tan Ah Moy

机构信息

Duke-NUS Medical School, Singapore.

Haematology/Oncology Service, Department of Paediatric Subspecialties, KK Women's and Children's Hospital, Singapore.

出版信息

Clin Lymphoma Myeloma Leuk. 2021 Mar;21(3):e290-e300. doi: 10.1016/j.clml.2020.11.016. Epub 2020 Dec 1.

DOI:10.1016/j.clml.2020.11.016
PMID:33384264
Abstract

PURPOSE

To determine the prognostic factors in pediatric patients with acute myeloid leukemia (AML) and to assess whether their outcomes have improved over time.

PATIENTS AND METHODS

Sixty-two patients with AML excluding acute promyelocytic leukemia were retrospectively analyzed. Patients in the earlier cohort (n = 36) were treated on the Medical Research Council (MRC) AML12 protocol, whereas those in the recent cohort (n = 26) were treated on the Malaysia-Singapore AML protocol (MASPORE 2006), which differed in terms of risk group stratification, cumulative anthracycline dose, and timing of hematopoietic stem-cell transplantation for high-risk patients.

RESULTS

Significant improvements in 10-year overall survival and event-free survival were observed in patients treated with the recent MASPORE 2006 protocol compared to the earlier MRC AML12 protocol (overall survival: 88.0% ± 6.5% vs 50.1% ± 8.6%, P = .002; event-free survival: 72.1% ± 9.0 vs 50.1% ± 8.6%, P = .045). In univariate analysis, patients in the recent cohort had significantly lower intensive care unit admission rate (11.5% vs 47.2%, P = .005) and numerically lower relapse rate (26.9% vs 50.0%, P = .068) compared to the earlier cohort. Multivariate analysis showed that treatment protocol was the only independent predictive factor for overall survival (hazard ratio = 0.21; 95% confidence interval, 0.06-0.73, P = .014).

CONCLUSION

Outcomes of pediatric AML patients have improved over time. The more recent MASPORE 2006 protocol led to significant improvement in long-term survival rates and reduction in intensive care unit admission rate.

摘要

目的

确定小儿急性髓系白血病(AML)患者的预后因素,并评估其预后是否随时间推移有所改善。

患者与方法

对62例排除急性早幼粒细胞白血病的AML患者进行回顾性分析。早期队列(n = 36)的患者按照医学研究委员会(MRC)AML12方案进行治疗,而近期队列(n = 26)的患者按照马来西亚-新加坡AML方案(MASPORE 2006)进行治疗,该方案在风险组分层、蒽环类药物累积剂量以及高危患者造血干细胞移植时机方面存在差异。

结果

与早期的MRC AML12方案相比,采用近期的MASPORE 2006方案治疗的患者10年总生存率和无事件生存率有显著提高(总生存率:88.0%±6.5%对50.1%±8.6%,P = 0.002;无事件生存率:72.1%±9.0对50.1%±8.6%,P = 0.045)。单因素分析显示,与早期队列相比,近期队列患者的重症监护病房入住率显著降低(11.5%对47.2%,P = 0.005),复发率在数值上也较低(26.9%对50.0%,P = 0.068)。多因素分析表明,治疗方案是总生存率的唯一独立预测因素(风险比=0.21;95%置信区间,0.06 - 0.73,P = 0.014)。

结论

小儿AML患者的预后随时间推移有所改善。近期的MASPORE 2006方案使长期生存率显著提高,重症监护病房入住率降低。

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