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多发性骨髓瘤的精准医学:定义可行方法面临的挑战

Precision Medicine in Myeloma: Challenges in Defining an Actionable Approach.

作者信息

González-Calle Verónica, Keane Niamh, Braggio Esteban, Fonseca Rafael

机构信息

Division of Hematology and Oncology, Mayo Clinic, Scottsdale, AZ.

Division of Hematology and Oncology, Mayo Clinic, Scottsdale, AZ.

出版信息

Clin Lymphoma Myeloma Leuk. 2017 Oct;17(10):621-630. doi: 10.1016/j.clml.2017.06.021. Epub 2017 Jun 23.

DOI:10.1016/j.clml.2017.06.021
PMID:28743429
Abstract

Recently, large sequencing studies have provided insights into the mutational landscape of multiple myeloma (MM), identifying actionable mutations and giving a precious opportunity for exploring new targeted therapies. The main goal of precision medicine, matching patients with the right drug, seems to be closer than ever. However, no targeted therapies in MM are approved yet. Several clinical trials testing targeted drugs and enrolling patients with MM are currently ongoing and will provide predictive biomarkers that might support clinical decision making. In this review, we evaluate the evidence supporting the implementation of precision medicine in MM and we discuss the challenges that should be dealt with in this imminent and promising new era.

摘要

最近,大规模测序研究为多发性骨髓瘤(MM)的突变图谱提供了见解,确定了可采取行动的突变,并为探索新的靶向治疗提供了宝贵机会。精准医学的主要目标,即让患者使用合适的药物,似乎比以往任何时候都更接近实现。然而,MM中尚无获批的靶向治疗药物。目前有几项测试靶向药物并招募MM患者的临床试验正在进行中,这些试验将提供可能支持临床决策的预测性生物标志物。在本综述中,我们评估了支持在MM中实施精准医学的证据,并讨论了在这个即将到来且充满希望的新时代应应对的挑战。

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