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拉罗替尼治疗 TRK 融合型先天性中胚层肾瘤骨转移引起的戏剧性骨重建。

Dramatic bone remodeling following larotrectinib administration for bone metastasis in a patient with TRK fusion congenital mesoblastic nephroma.

机构信息

Department of Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee.

Department of Diagnostic imaging, St. Jude Children's Research Hospital, Memphis, Tennessee.

出版信息

Pediatr Blood Cancer. 2018 Oct;65(10):e27271. doi: 10.1002/pbc.27271. Epub 2018 Jun 12.

DOI:10.1002/pbc.27271
PMID:29893456
Abstract

Mesoblastic nephroma is the most frequent renal tumor in newborns and young infants, and the cellular type is characterized by an ETV6-NTRK fusion, which constitutively activates the tropomyosin-related kinase (TRK) signaling pathway. Larotrectinib is a highly selective TRK inhibitor with activity in adult and pediatric patients who have TRK fusions. We present a rare case of a patient with mesoblastic nephroma metastatic to bone who had a dramatic response to larotrectinib.

摘要

中胚层肾瘤是新生儿和婴幼儿最常见的肾肿瘤，细胞型特征为 ETV6-NTRK 融合，该融合可使原肌球蛋白相关激酶 (TRK) 信号通路持续激活。拉罗替尼是一种高度选择性的 TRK 抑制剂，在具有 TRK 融合的成年和儿科患者中具有活性。我们报告了一例罕见的中胚层肾瘤骨转移患者，其对拉罗替尼有显著反应。

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