Comparative RNA-Sequencing Analysis Benefits a Pediatric Patient With Relapsed Cancer.

Newton, Yulia; Rassekh, S Rod; Deyell, Rebecca J; Shen, Yaoqing; Jones, Martin R; Dunham, Chris; Yip, Stephen; Leelakumari, Sreeja et al. · JCO Precis Oncol · 2018

case_report · Level V

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Abstract

Clinical detection of sequence and structural variants in known cancer genes points to viable treatment options for a minority of children with cancer.<sup>1</sup> To increase the number of children who benefit from genomic profiling, gene expression information must be considered alongside mutations.<sup>2,3</sup> Although high expression has been used to nominate drug targets for pediatric cancers,<sup>4,5</sup> its utility has not been evaluated in a systematic way.<sup>6</sup> We describe a child with a rare sarcoma that was profiled with whole-genome and RNA sequencing (RNA-Seq) techniques. Although the tumor did not harbor DNA mutations targetable by available therapies, incorporation of gene expression information derived from RNA-Seq analysis led to a therapy that produced a significant clinical response. We use this case to describe a framework for inclusion of gene expression into the clinical genomic evaluation of pediatric tumors.