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Bortezomib in previously treated phosphatase and tension homology‐deficient patients with advanced intrahepatic cholangiocarcinoma: An open‐label, prospective and single‐centre phase II trial.
- Source :
- Clinical & Translational Medicine; May2024, Vol. 14 Issue 5, p1-12, 12p
- Publication Year :
- 2024
-
Abstract
- Introduction: Intrahepatic cholangiocarcinoma (ICC) is characterized by a dismal prognosis with limited therapeutic alternatives. To explore phosphatase and tension homolog (PTEN) as a biomarker for proteasome inhibition in ICC, we conducted a phase II trial to assess the second‐line efficacy of bortezomib in PTEN‐deficient advanced ICC patients. Methods: A total of 130 patients with advanced ICC in our centre were screened by PTEN immunohistochemical staining between 1 July 2017, and 31 December 2021, and 16 patients were ultimately enrolled and treated with single‐agent bortezomib 1.3 mg/m2 on days 1, 4, 8 and 11 of a 21‐day cycle. The primary endpoint was the objective response rate (ORR) according to Response Evaluation Criteria in Solid Tumors v1.1. Results: The median follow‐up was 6.55 months (95% confidence interval [CI]: 0.7–19.9 months). Among the 16 enrolled patients, the ORR was 18.75% (3/16) and the disease control rate was 43.75% (7/16). The median progress‐free survival was 2.95 months (95% CI: 2.1–5.1 months) and the median overall survival (mOS) was 7.2 months (95% CI: 0.7–21.6 months) in the intent‐to‐treat‐patients. Treatment‐related adverse events of any grade were reported in 16 patients, with thrombopenia being the most common toxicity. Patients with PTEN staining scores of 0 were more likely to benefit from bortezomib than those with staining scores > 0. Conclusions: Bortezomib yielded an encouraging objective response and a favourable OS as a second‐line agent in PTEN‐deficient ICC patients. Our findings suggest bortezomib as a promising therapeutic option for patients with PTEN‐deficient ICC. Highlights: There is a limited strategy for the second‐line option of intrahepatic cholangiocarcinoma (ICC).This investigator‐initiated phase 2 study evaluated bortezomib in ICC patients with phosphatase and tension homology deficiency.The overall response rate was 18.75% and the overall survival was 7.2 months in the intent‐to‐treat cohort.These results justify further developing bortezomib in ICC patients with PTEN deficiency. [ABSTRACT FROM AUTHOR]
Details
- Language :
- English
- ISSN :
- 20011326
- Volume :
- 14
- Issue :
- 5
- Database :
- Complementary Index
- Journal :
- Clinical & Translational Medicine
- Publication Type :
- Academic Journal
- Accession number :
- 177482359
- Full Text :
- https://doi.org/10.1002/ctm2.1675