Taxane-Based Chemotherapy Is Effective in Metastatic Appendiceal Adenocarcinoma

Authors

Julia Dansby
Aditya More
Mohammad Zeineddine
Abdelrahman Yousef
Alisha Bent
Farshid Dayyani
Robert Wolff
Michael Overman
John Paul Shen

Publication Date

12-11-2023

Journal

The Oncologist

Abstract

Appendiceal cancer is a rare, orphan disease with no therapies currently approved by the FDA for its treatment. Given the limited data regarding drug efficacy, these tumors have historically been treated with chemotherapy designed for colon cancer. However, an overwhelming body of molecular data has demonstrated that appendiceal adenocarcinoma is a distinct entity with key molecular differences from colon cancer, notably rare APC mutation. Recognizing that APC loss-of-function is thought to contribute to taxane resistance and that taxanes are effective in the treatment of other gastrointestinal tumors, including gastric, esophageal, and small bowel adenocarcinoma, we completed a single-center retrospective study to assess efficacy. In a cohort of 13 patients with metastatic appendiceal adenocarcinoma, treated with taxane chemotherapy the median overall survival was 8.8 months. Of 10 evaluable patients, we observed 3 responses, 4 patients with stable disease, and 3 with progression (30% response rate, 70% disease control rate). The results of this study showing activity of taxane-based chemotherapy in appendiceal adenocarcinoma support further clinical investigation of taxane therapy in this orphan disease.

Keywords

Humans, Retrospective Studies, Rare Diseases, Taxoids, Appendiceal Neoplasms, Adenocarcinoma, Colonic Neoplasms, Antineoplastic Combined Chemotherapy Protocols, appendiceal cancer, taxane chemotherapy

Comments

Supplementary Materials

Data Availability Statement

PMID: 37738316