Minimal/Measurable Residual Disease Monitoring in NPM1-Mutated Acute Myeloid Leukemia: A Clinical Viewpoint and Perspectives.
Minimal/Measurable Residual Disease Monitoring in NPM1-Mutated Acute Myeloid Leukemia: A Clinical Viewpoint and Perspectives.
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DOI：
10.3390/ijms19113492
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发表时间：
2018-11-06
影响因子：
5.6
通讯作者：
Luppi M
中科院分区：
文献类型：
--
作者：
Forghieri F;Comoli P;Marasca R;Potenza L;Luppi M
具有NPM1基因突变的急性髓系白血病(AML)因其独特的生物学和临床特征，目前被认为是一个独特的实体。我们在此总结已发表的研究结果，研究微小/可测量残留病(MRD)在接受强化化疗或造血干细胞移植的NPM1突变AML患者中的临床应用。到目前为止，几项临床试验已经证明，分子MRD监测对NPM1突变的AML具有显著的独立预后影响，因此，欧洲白血病网络MRD工作组的共识文件最近建议，NPM1突变的AML患者在治疗和随访期间的临床时间点进行MRD评估。然而，仍然存在一些争议，主要是关于需要考虑的最有临床意义的时间点和MRD阈值，也关于要分析的最佳来源，即骨髓或外周血样本，以及MRD与其他已知预后指标的相关性。此外，我们讨论了新的分子技术，如数字液滴聚合酶链式反应和下一代测序与传统的RQ-PCR相比的潜在优势和缺点，以定量NPM1突变的MRD。总之，有必要进行进一步的前瞻性临床试验，以标准化MRD监测策略，并优化NPM1突变AML患者的MRD引导治疗干预措施。
Acute myeloid leukemia (AML) with NPM1 gene mutations is currently recognized as a distinct entity, due to its unique biological and clinical features. We summarize here the results of published studies investigating the clinical application of minimal/measurable residual disease (MRD) in patients with NPM1-mutated AML, receiving either intensive chemotherapy or hematopoietic stem cell transplantation. Several clinical trials have so far demonstrated a significant independent prognostic impact of molecular MRD monitoring in NPM1-mutated AML and, accordingly, the Consensus Document from the European Leukemia Net MRD Working Party has recently recommended that NPM1-mutated AML patients have MRD assessment at informative clinical timepoints during treatment and follow-up. However, several controversies remain, mainly with regard to the most clinically significant timepoints and the MRD thresholds to be considered, but also with respect to the optimal source to be analyzed, namely bone marrow or peripheral blood samples, and the correlation of MRD with other known prognostic indicators. Moreover, we discuss potential advantages, as well as drawbacks, of newer molecular technologies such as digital droplet PCR and next-generation sequencing in comparison to conventional RQ-PCR to quantify NPM1-mutated MRD. In conclusion, further prospective clinical trials are warranted to standardize MRD monitoring strategies and to optimize MRD-guided therapeutic interventions in NPM1-mutated AML patients.
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