A systematic review and meta-analysis of pathologic complete response rates for patients with cholangiocarcinoma treated on liver transplant protocols

J Surg Oncol. 2024 Mar;129(3):574-583. doi: 10.1002/jso.27511. Epub 2023 Nov 20.

Abstract

BACKGROUND AND OBJECTIVES: Many heterogenous orthotopic liver transplant (OLT) protocols exist for patients with unresectable cholangiocarcinoma. Little is known about the incidence, predictors for, and the significance of achieving a pathologic complete response (pCR).

Methods: We performed a systematic review through September 2022 of the PubMed, Embase, and Web of Science databases. A random-effect meta-analysis was conducted to pool data across studies with reported pCR rates. Heterogeneity between treatment protocols was assessed via subgroup analysis. The pCR and 1-, 3-, and 5-year recurrence-free survival (RFS) and overall survival (OS) rates were extracted as outcomes of interest.

Results: A total of 15 studies reported pCR rates and were grouped by use of the Mayo protocol (4/15), stereotactic body radiation therapy (2/15), and an Other category (9/15). The pooled pCR rate among all studies was 32%. Both radiation technique and duration of CHT showed no significant association with pCR (p = 0.05 and 0.13, respectively). Pooled 1-year RFS and OS after any neoadjuvant therapy and OLT was 80% (95% confidence interval [CI], 0.61-0.91), and 91% (95% CI, 0.87-0.94), respectively. There was no 1-year OS difference detected among the three groups. pCR was not associated with OS in the meta-regression. Pooled 3- and 5-year OS among all studies was 72% and 61%, respectively.

Conclusions: The pooled incidence of pCR was 32%. Differences in radiation technique did not appear to influence pCR rates and upon meta-regression, pCR was not a surrogate marker for survival.

Keywords: cholangiocarcinoma; neoadjuvant chemoradiation; neoadjuvant therapy; orthotopic liver transplant; pathologic complete response.

MeSH terms

  • Bile Duct Neoplasms* / surgery
  • Bile Ducts, Intrahepatic / pathology
  • Cholangiocarcinoma* / surgery
  • Humans
  • Liver Transplantation*
  • Meta-Analysis as Topic
  • Neoadjuvant Therapy
  • Pathologic Complete Response
  • Systematic Reviews as Topic
  • Treatment Outcome