Division of Hematology, Department of Internal Medicine and Comprehensive Cancer Center.
Medical Scientist Training Program.
JCI Insight. 2020 Jun 18;5(12):134973. doi: 10.1172/jci.insight.134973.
Detecting, characterizing, and monitoring rare populations of cells can increase testing sensitivity, give insight into disease mechanism, and inform clinical decision making. One area that can benefit from increased resolution is management of cancers in clinical remission but with measurable residual disease (MRD) by multicolor FACS. Detecting and monitoring genomic clonal resistance to treatment in the setting of MRD is technically difficult and resource intensive due to the limited amounts of disease cells. Here, we describe limited-cell FACS sequencing (LC-FACSeq), a reproducible, highly sensitive method of characterizing clonal evolution in rare cells relevant to different types of acute and chronic leukemias. We demonstrate the utility of LC-FACSeq for broad multigene gene panels and its application for monitoring sequential acquisition of mutations conferring therapy resistance and clonal evolution in long-term ibrutinib treatment of patients with chronic lymphocytic leukemia. This technique is generalizable for monitoring of other blood and marrow infiltrating cancers.
检测、表征和监测稀有细胞群体可以提高检测灵敏度,深入了解疾病机制,并为临床决策提供信息。一个可以从提高分辨率中受益的领域是通过多色 FACS 对临床缓解但有可测量残留疾病 (MRD) 的癌症进行管理。在 MRD 中,由于疾病细胞数量有限,检测和监测对治疗的基因组克隆耐药性在技术上具有挑战性并且资源密集。在这里,我们描述了有限细胞流式细胞术测序 (LC-FACSeq),这是一种可重现的、高度敏感的方法,用于表征与不同类型的急性和慢性白血病相关的稀有细胞中的克隆进化。我们展示了 LC-FACSeq 在广泛的多基因基因面板中的实用性及其在监测长期伊布替尼治疗慢性淋巴细胞白血病患者中获得耐药性和克隆进化相关的突变方面的应用。该技术可用于监测其他血液和骨髓浸润性癌症。