由于很少有大型研究确定脊索瘤相关生物标记物，我们的目标是利用我们的大型单中心脊索瘤肿瘤库，发现新的信号通路。 回顾性收集了73例脊索瘤患者的临床和病理数据。从61个存档的脊索瘤标本中建立肿瘤微阵列；进行TOMM20、TIGAR和MCT1的免疫组化检测；并对染色强度和阳性肿瘤细胞百分比进行半定量分析。将TIGAR、TOMM20和MCT1的平均综合得分按疾病状态和解剖位置进行比较。与原发病灶相比，复发性和转移性脊索瘤中的TOMM20表达更高。将仅有原发病的患者和有复发疾病的患者的原发病灶的综合得分进行比较，结果显示TIGAR和TOMM20的表达在原发病灶中显著高于有复发疾病的患者，其次是有复发疾病史的患者。TOMM20的综合得分大于或等于3时，会显著降低总生存率（风险比为5.83）和无复发生存率（风险比为8.95）。发现促进脊索瘤生长和复发的新的信号通路，对于开发针对脊索瘤的靶向治疗至关重要。TOMM20可能是与脊索瘤疾病进展相关的生物标记物。©作者 2023。由牛津大学出版社代表美国临床病理学会出版。保留所有权利。如果需要权限，请发送电子邮件至：journals.permissions@oup.com。

As few large studies identify correlative biomarkers in chordoma, our objective was to use our large, single-center chordoma tumor bank to identify novel signaling pathways.Clinical and pathologic data for 73 patients with chordoma were retrospectively collected. Tumor microarrays were built from 61 archived chordoma specimens; immunohistochemistry for TOMM20, TIGAR, and MCT1 were performed; and semiquantitative analysis of staining intensity and percentage of positive tumor cells was performed. Average composite scores of MCT1, TIGAR, and TOMM20 expression were compared by disease status and anatomic location.Higher expression of TOMM20 was seen in recurrent and metastatic chordomas compared with primary lesions. Comparing composite scores of primary lesions in patients with primary disease only vs those with recurrent disease showed that TIGAR and TOMM20 expressions are significantly higher in primary lesions, followed by a history of recurrence. A TOMM20 composite score of greater than or equal to 3 significantly decreased overall survival (hazard ratio [HR], 5.83) and recurrence-free survival (HR, 8.95).Identifying novel signaling pathways that promote chordoma growth and recurrence is critical for developing targeted therapy for chordoma. TOMM20 may be a biomarker associated with chordoma disease progression.© The Author(s) 2023. Published by Oxford University Press on behalf of American Society for Clinical Pathology. All rights reserved. For permissions, please e-mail: journals.permissions@oup.com.