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Taxane-Based Chemotherapy Is Effective in Metastatic Appendiceal Adenocarcinoma
Julia Dansby1, Aditya More1, Mohammad Zeineddine1
1Department of Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
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.
Insights
Taxane chemotherapy shows promise for appendiceal adenocarcinoma, a rare cancer. This study found a 30% response rate and 70% disease control in patients treated with taxanes, suggesting further investigation.
Area of Science:
- Oncology
- Gastrointestinal Oncology
Background:
- Appendiceal cancer is a rare orphan disease with no FDA-approved treatments.
- Appendiceal adenocarcinoma exhibits distinct molecular differences from colon cancer, including rare APC mutations.
- Existing treatments often involve chemotherapy designed for colon cancer due to limited appendiceal cancer-specific data.
Purpose of the Study:
- To assess the efficacy of taxane-based chemotherapy in patients with metastatic appendiceal adenocarcinoma.
- To investigate taxane chemotherapy as a potential treatment for this rare gastrointestinal malignancy.
Main Methods:
- A single-center retrospective study was conducted.
- The study included a cohort of 13 patients with metastatic appendiceal adenocarcinoma treated with taxane chemotherapy.
- Efficacy was evaluated based on response rate, disease control rate, and overall survival.
Main Results:
- The median overall survival was 8.8 months in the cohort of 13 patients.
- In 10 evaluable patients, a 30% response rate and a 70% disease control rate were observed.
- Specifically, 3 patients responded, 4 had stable disease, and 3 experienced progression.
Conclusions:
- Taxane-based chemotherapy demonstrated activity in appendiceal adenocarcinoma.
- These findings support further clinical investigation of taxane therapy for appendiceal cancer.
- Taxanes may represent a viable treatment option for this orphan gastrointestinal disease.
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