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迈向骨髓瘤精准医学:新证据与挑战

[Towards precision medicine in myeloma: new evidence and challenges].

作者信息

González-Calle Verónica, Fonseca Rafael

机构信息

Clinical Hematology and Medical Oncology, Mayo Clinic, Scottsdale, USA.

Clinical Hematology and Medical Oncology, Mayo Clinic, Scottsdale, USA. E-mail:

出版信息

Medicina (B Aires). 2017;77(3):222-226.

Abstract

In the last few years, next-generation sequencing studies have provided insights into the mutational landscape of multiple myeloma. The identification of actionable mutations might give a precious opportunity for exploring new targeted therapies. Thus, the implementation of promising precision medicine strategies seems to be closer than ever. Throughout this review we describe the main challenges that should to be dealt with in this new era, in order to achieve the main goal of precision medicine, namely matching patients with their right drug. In addition, we provide a review of the most significant preclinical and clinical studies supporting the implementation of precision medicine nowadays. Finally, we highlight the need of clinical trials to evaluate the security and efficacy of these targeted therapies, as well as to validate predictive biomarkers that may allow an appropriate best-candidate selection and improvement of myeloma patients' survival and quality of life.

摘要

在过去几年中,新一代测序研究为多发性骨髓瘤的突变图谱提供了深入见解。可操作突变的识别可能为探索新的靶向治疗提供宝贵机会。因此,实施有前景的精准医学策略似乎比以往任何时候都更接近现实。在这篇综述中,我们描述了在这个新时代应应对的主要挑战,以实现精准医学的主要目标,即将患者与合适的药物相匹配。此外,我们综述了目前支持精准医学实施的最重要的临床前和临床研究。最后,我们强调需要进行临床试验来评估这些靶向治疗的安全性和有效性,以及验证预测性生物标志物,这可能有助于进行适当的最佳候选者选择,并改善骨髓瘤患者的生存率和生活质量。

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