A Refractory Case of CDKN2A/B Loss Metastatic Intrahepatic Cholangiocarcinoma Achieving a Partial Response After First-Line Treatment with Palbociclib

Abstract

Weiming Fan,1,* Chuan Wang,1,* Xuefeng Zhong,2 Yating Zheng,2 Tingting Chen,2 Mengli Huang,2 Shuying Su1 1Department of Biliary-Pancreatic Surgery, First People’s Hospital of Foshan, Foshan, People’s Republic of China; 2The Medical Department, 3D Medicines, Inc., Shanghai, People’s Republic of China*These authors contributed equally to this workCorrespondence: Shuying Su, Department of Biliary-Pancreatic Surgery, First People’s Hospital of Foshan, No. 81 North Lingnan Avenue, Chancheng District, Foshan, Guangdong Province, People’s Republic of China, Email [email protected]: Intrahepatic cholangiocarcinoma (ICC) is a highly aggressive and malignant subtype of biliary duct tumors. The poor prognosis of advanced ICC brings great challenges to clinical treatment, and chemotherapy-based therapy remains the standard first-line regimen. In recent years, the development of clinical research on targeted therapy for biliary duct tumors has brought new strategies for clinical treatment, but the targets are limited. Herein, we reported a 68-year-old patient with metastasis ICC harboring CDKN2A/B loss, who achieved a partial response (PR) after the first-line treatment with a cyclin-dependent kinases 4 and 6 (CDK4/6) inhibitor called palbociclib, and no obvious side effects were observed. As of the latest follow-up time, the progression-free survival (PFS) had lasted for 20 months. This case reveals the molecular characteristic of ICC patients who respond to palbociclib treatment and illustrates the importance of performing a multiple-gene panel test in ICC patients.Keywords: intrahepatic cholangiocarcinoma, ICC, CDKN2A/B loss, palbociclib, CDK4/6 inhibitor, next-generation sequencing, NGS

Keywords

• intrahepatic cholangiocarcinoma (icc)
• cdkn2a/b loss
• palbociclib
• cdk4/6 inhibitor
• next generation sequencing (ngs)