Neoadjuvant therapy leads to objective response in intrahepatic cholangiocarcinoma

Benjamin J Van Treeck; Maria C Olave; Ryan D Watkins; Haiyan Lu; Roger K Moreira; Taofic Mounajjed; Michael J Johnson; Carin Y Smith; Sumera I Ilyas; Nguyen H Tran; Sarah M Jenkins; Katelyn A Reed; Rory Smoot; Amit Mahipal; Daniela Allende; Rondell P Graham

doi:10.1016/j.hpb.2024.04.003

Neoadjuvant therapy leads to objective response in intrahepatic cholangiocarcinoma

HPB (Oxford). 2024 Apr 23:S1365-182X(24)01259-0. doi: 10.1016/j.hpb.2024.04.003. Online ahead of print.

Authors

Benjamin J Van Treeck¹, Maria C Olave¹, Ryan D Watkins², Haiyan Lu³, Roger K Moreira¹, Taofic Mounajjed¹, Michael J Johnson¹, Carin Y Smith⁴, Sumera I Ilyas⁵, Nguyen H Tran⁶, Sarah M Jenkins⁴, Katelyn A Reed¹, Rory Smoot², Amit Mahipal⁷, Daniela Allende³, Rondell P Graham⁸

Affiliations

¹ Mayo Clinic, Department of Laboratory Medicine and Pathology, Rochester, MN, USA.
² Mayo Clinic, Department of Surgery, Department of Biochemistry and Molecular Biology, Rochester, MN, USA.
³ Cleveland Clinic, Department of Pathology, Cleveland, OH, USA.
⁴ Mayo Clinic, Department of Quantitative Health Sciences, Rochester, MN, USA.
⁵ Mayo Clinic, Division of Gastroenterology and Hepatology, Department of Immunology, Rochester, MN, USA.
⁶ Mayo Clinic, Department of Oncology, Rochester, MN, USA.
⁷ Mayo Clinic, Department of Oncology, Rochester, MN, USA; University Hospitals Seidman Cancer Center, Case Western Reserve University, Cleveland OH, USA.
⁸ Mayo Clinic, Department of Laboratory Medicine and Pathology, Rochester, MN, USA. Electronic address: graham.rondell@mayo.edu.

PMID: 38705793
DOI: 10.1016/j.hpb.2024.04.003

Abstract

Background: Intrahepatic cholangiocarcinoma (iCCA) is the second most common hepatic malignancy and has a poor prognosis. Surgical resection is the standard of care for patients with resectable disease, representing 30-40% of cases. Increasingly, neoadjuvant systemic therapy is being utilized in patients due to high-risk anatomic or biologic considerations. However, data on the clinical effect of this approach are limited. We performed a cohort study to evaluate the effect of neoadjuvant therapy in patients with oncologically high-risk iCCA.

Methods: iCCA patients (n = 181) between the years 2014-2020 were reviewed for clinical, histopathologic, treatment, and outcome-related data. Tumor regression grade was scored per CAP criteria for gastrointestinal carcinomas.

Results: 47 iCCA patients received neoadjuvant therapy and 72 did not. Neoadjuvant treatment led to objective response and tumor regression by CAP score. After adjustment for age, clinical stage, and tumor size, the outcomes of patients who had neoadjuvant therapy followed by surgery were not significantly different from those patients who had surgery first.

Discussion: In conclusion, neoadjuvant therapy in iCCA facilitated surgical care. The progression-free and overall survival for surgical patients with and without neoadjuvant therapy were not significantly different suggesting this approach needs further exploration as an effective treatment paradigm.