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.