Appropriate tools for monitoring sarcoma progression are still limited. The aim of the present study was to investigate the value of miR-34a-5p (miR34a) as a circulating biomarker to follow disease progression and measure the therapeutic response. Stable forced re-expression of miR34a in Ewing sarcoma (EWS) cells significantly limited tumor growth in mice. Absolute quantification of miR34a in the plasma of mice and 31 patients showed that high levels of this miRNA inversely correlated with tumor volume. In addition, miR34a expression was higher in the blood of localized EWS patients than in the blood of metastatic EWS patients. In 12 patients, we followed miR34a expression during preoperative chemotherapy. While there was no variation in the blood miR34a levels in metastatic patients at the time of diagnosis or after the last cycle of preoperative chemotherapy, there was an increase in the circulating miR34a levels in patients with localized tumors. The three patients with the highest fold-increase in the miR levels did not show evidence of metastasis. Although this analysis should be extended to a larger cohort of patients, these findings imply that detection of the miR34a levels in the blood of EWS patients may assist with the clinical management of EWS.

用于监测肉瘤进展的适当工具仍然有限。本研究的目的是调查 miR-34a-5p (miR34a) 作为循环生物标志物的价值，以跟踪疾病进展和测量治疗反应。尤文肉瘤 (EWS) 细胞中 miR34a 的稳定强制重新表达显着限制了小鼠肿瘤的生长。对小鼠和 31 名患者血浆中 miR34a 的绝对定量表明，该 miRNA 的高水平与肿瘤体积呈负相关。此外，局部EWS患者血液中的miR34a表达高于转移性EWS患者血液中的表达。在 12 名患者中，我们在术前化疗期间追踪了 miR34a 的表达。虽然转移性患者在诊断时或最后一个周期的术前化疗后血液 miR34a 水平没有变化，但局部肿瘤患者的循环 miR34a 水平有所增加。miR 水平增加倍数最高的三名患者没有显示出转移的证据。尽管该分析应扩展到更大的患者群体，但这些发现表明，检测 EWS 患者血液中的 miR34a 水平可能有助于 EWS 的临床管理。