Pexidartinib (PLX3397) is a small molecule tyrosine kinase and colony-stimulating factor-1 inhibitor with FDA breakthrough therapy designation for tenosynovial giant-cell tumor, and currently under study in several other tumor types, including breast cancer, non-Hodgkin’s lymphoma, and glioblastoma. Here, we report a case of severe drug-induced liver injury requiring liver transplantation due to vanishing bile duct syndrome (VBDS) after exposure to pexidartinib in the I-SPY 2 Trial, a phase 2 multicenter randomized neoadjuvant chemotherapy trial in patients with Stage II–III breast cancer. We also review the current literature on this rare, idiosyncratic, and potentially life-threatening entity.

Pexidartinib（PLX3397）是小分子酪氨酸激酶和集落刺激因子1抑制剂，具有FDA突破性疗法，用于腱鞘巨细胞瘤，目前正在研究其他几种肿瘤类型，包括乳腺癌，非霍奇金淋巴瘤和胶质母细胞瘤。在此，我们报告了I-SPY 2试验（II期患者的2期多中心随机新辅助化疗试验2期临床试验）中暴露于pexidartinib后因胆管综合征（VBDS）消失而导致严重药物诱发的肝损伤，需要肝移植的情况。 –III乳腺癌。我们还回顾了有关这种稀有，特质和可能威胁生命的实体的最新文献。