Liver Transplantation for Acute Liver Failure Following Regorafenib Treatment for Metastatic Gastrointestinal Stromal Tumor: Case Report and Literature Review.

Abstract

Tyrosine kinase inhibitors (TKI) have improved survival in metastatic gastrointestinal stromal tumors (GIST) but can lead to drug-induced liver injury (DILI), rarely progressing to acute liver failure (ALF). We report the first documented case of regorafenib-induced ALF in a 51-year-old patient with metastatic ileal GIST, necessitating emergency liver transplantation (LT). The patient experienced prior hepatotoxicity with imatinib and disease progression under sunitinib. Following LT, sorafenib was initiated to treat peritoneal recurrence, and partial tumor control was achieved 3 years after transplantation. In literature, 6 cases of LT for TKI-induced ALF were reported, all in patients with chronic myeloid leukemia. This report highlights the critical importance of early detection of TKI-induced hepatotoxicity and LT as a life-saving intervention in severe DILI, while emphasizing the complex oncological considerations-including recurrence risk, immunosuppression-related challenges, and long-term survival-that must be carefully evaluated in the context of organ shortage.