Evaluation of clinical outcomes and prognostic factors in patients with metastatic appendiceal adenocarcinoma treated with first-line chemotherapy: A multi-institutional retrospective study
Abstract
Background Appendiceal adenocarcinoma (AA) is a rare cancer characterized by high incidences of mucinous histology, peritoneal metastasis, and a good prognosis. However, its chemotherapeutic efficacy and prognostic factors are unclear. Objective To evaluate real-world clinical outcomes and explore prognostic factors in patients with metastatic AA treated with chemotherapy. Design Retrospective study. Methods We reviewed the clinical courses of patients with metastatic AA receiving first-line chemotherapy between February 2005 and March 2023. We also explored prognostic factors using multivariate analysis. Results In the 66 identified patients, incidences of mucinous histology and peritoneal metastasis were 41% and 90%, respectively. Forty patients (60.6%) received an oxaliplatin-based regimen combined with bevacizumab. Median progression-free survival (PFS) was 10.9 months, and median overall survival (OS) was 23.0 months. The response rate was 22.0%, and the disease control rate was 87.8% in 41 patients with target lesions. Subsequent chemotherapy was administered to 77.4% of patients. Multivariate analysis showed that presence of the primary tumor, Eastern Cooperative Oncology Group (ECOG) performance status (PS) ≥1, moderate to massive ascites, and elevated pretreatment carcinoembryonic antigen and carbohydrate antigen 19-9 levels were significantly associated with shorter OS. There were significant differences in OS among the three groups classified by the number of poor prognostic factors (none versus [vs.] 1-2 vs. 3-4). Conclusions Patients with metastatic AA showed comparable PFS and OS to those with common colorectal cancer despite a high incidence of peritoneal metastasis and a low response rate. Elevated tumor marker levels, presence of the primary tumor, ECOG PS ≥1, moderate to massive ascites were poor prognostic factors for OS. The number of these risk factors may allow exploratory prognostic stratification; however, this model is hypothesis-generating and requires external validation before clinical application.
Related
- Case of Appendiceal Goblet Cell Adenocarcinoma With Gastric Metastasis.
- Rapidly Progressive Peritoneal Carcinomatosis Originating From Mucinous Appendiceal Adenocarcinoma: A Case Report.
- VEGF-A Immunohistochemical Expression Across Appendiceal Epithelial Lesions: A Retrospective Exploratory Study
- Intensity-modulated radiotherapy combined with albumin-bound paclitaxel and platinum-based chemotherapy for locoregional recurrent cervical cancer: a single-institutional phase II investigator-initiated nonrandomized clinical trial
- Host Determinants of Adjuvant Chemotherapy Initiation and Survival in Upper Gastrointestinal Adenocarcinoma
BibTeX
@article{fukuda2026evaluation,
title = {{Evaluation of clinical outcomes and prognostic factors in patients with metastatic appendiceal adenocarcinoma treated with first-line chemotherapy: A multi-institutional retrospective study}},
author = {Koshiro Fukuda and Hiroki Osumi and Atsuo Takashima and Takahito Awatsu and Akira Ooki and Hidekazu Hirano and Shota Fukuoka and Natsuko Okita and Shohei Udagawa and Hirokazu Shoji and Mariko Ogura and Takeru Wakatsuki and Keisho Chin and Mitsuhiro Fujishiro and Ken Kato and Narikazu Boku and Kensei Yamaguchi and Eiji Shinozaki},
journal = {Therapeutic Advances in Medical Oncology},
year = {2026},
month = oct,
doi = {10.1177/17588359261499142},
url = {https://doi.org/10.1177/17588359261499142},
note = {CC BY},
}
RIS
TY - JOUR
TI - Evaluation of clinical outcomes and prognostic factors in patients with metastatic appendiceal adenocarcinoma treated with first-line chemotherapy: A multi-institutional retrospective study
AU - Koshiro Fukuda
AU - Hiroki Osumi
AU - Atsuo Takashima
AU - Takahito Awatsu
AU - Akira Ooki
AU - Hidekazu Hirano
AU - Shota Fukuoka
AU - Natsuko Okita
AU - Shohei Udagawa
AU - Hirokazu Shoji
AU - Mariko Ogura
AU - Takeru Wakatsuki
AU - Keisho Chin
AU - Mitsuhiro Fujishiro
AU - Ken Kato
AU - Narikazu Boku
AU - Kensei Yamaguchi
AU - Eiji Shinozaki
PY - 2026
DA - 2026/10/23
JO - Therapeutic Advances in Medical Oncology
DO - 10.1177/17588359261499142
UR - https://doi.org/10.1177/17588359261499142
AB - Background Appendiceal adenocarcinoma (AA) is a rare cancer characterized by high incidences of mucinous histology, peritoneal metastasis, and a good prognosis. However, its chemotherapeutic efficacy and prognostic factors are unclear. Objective To evaluate real-world clinical outcomes and explore prognostic factors in patients with metastatic AA treated with chemotherapy. Design Retrospective study. Methods We reviewed the clinical courses of patients with metastatic AA receiving first-line chemotherapy between February 2005 and March 2023. We also explored prognostic factors using multivariate analysis. Results In the 66 identified patients, incidences of mucinous histology and peritoneal metastasis were 41% and 90%, respectively. Forty patients (60.6%) received an oxaliplatin-based regimen combined with bevacizumab. Median progression-free survival (PFS) was 10.9 months, and median overall survival (OS) was 23.0 months. The response rate was 22.0%, and the disease control rate was 87.8% in 41 patients with target lesions. Subsequent chemotherapy was administered to 77.4% of patients. Multivariate analysis showed that presence of the primary tumor, Eastern Cooperative Oncology Group (ECOG) performance status (PS) ≥1, moderate to massive ascites, and elevated pretreatment carcinoembryonic antigen and carbohydrate antigen 19-9 levels were significantly associated with shorter OS. There were significant differences in OS among the three groups classified by the number of poor prognostic factors (none versus [vs.] 1-2 vs. 3-4). Conclusions Patients with metastatic AA showed comparable PFS and OS to those with common colorectal cancer despite a high incidence of peritoneal metastasis and a low response rate. Elevated tumor marker levels, presence of the primary tumor, ECOG PS ≥1, moderate to massive ascites were poor prognostic factors for OS. The number of these risk factors may allow exploratory prognostic stratification; however, this model is hypothesis-generating and requires external validation before clinical application.
SN - 1758-8359
SN - 1758-8340
ER -