Clinical efficacy of immune checkpoint inhibitors for cancer of unknown primary: a multi-center retrospective study
Abstract Background Patients diagnosed with cancer of unknown primary (CUP) have limited treatment options, resulting in a dismal prognosis. The efficacy of immune checkpoint inhibitors (ICIs) in these patients is still unclear. Methods We collected the clinical data of these patients retrospectivel...
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BMC
2025-08-01
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| Online Access: | https://doi.org/10.1186/s12885-025-14778-6 |
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| author | Haowen Wang Siqi Song Yuzhou Nie Jinting Wu Linyue Pan Qizhen Zou Gaizhen Kuang Chen Chen Xiaochen Zhang Wenfeng Li Shirong Zhang |
| author_facet | Haowen Wang Siqi Song Yuzhou Nie Jinting Wu Linyue Pan Qizhen Zou Gaizhen Kuang Chen Chen Xiaochen Zhang Wenfeng Li Shirong Zhang |
| author_sort | Haowen Wang |
| collection | DOAJ |
| description | Abstract Background Patients diagnosed with cancer of unknown primary (CUP) have limited treatment options, resulting in a dismal prognosis. The efficacy of immune checkpoint inhibitors (ICIs) in these patients is still unclear. Methods We collected the clinical data of these patients retrospectively and multicentrically. In total, 190 patients diagnosed with CUP in 6 hospitals were collected, of which 58 patients received immunotherapy. Results The median overall survival (OS) was 17.3 months. The OS of the ICIs-treated group was significantly longer than that of the non-ICIs group in unfavorable subgroup, 29.27 months and 10.43 months, respectively (HR = 0.435, 95% CI 0.267–0.709; p = 0.0006). In the first-line systemic treatment, the OS and progression-free survival (PFS) in the ICIs group were significantly longer than that of the non-ICIs group. The OS was 45.53 months versus 12.03 months (HR = 0.431, 95% CI 0.259–0.717; p = 0.004), and the PFS was 11.33 months versus 5.43 months (HR = 0.558, 95% CI 0.351–0.889; p = 0.021). The objective response rate (ORR) and disease control rate (DCR) of ICIs group were higher too. Finally, a predictive nomogram model was developed to accurately forecast the response of patients with CUP to ICIs. Conclusions In this cohort of unfavorable subgroup, immunotherapy appears to extend the survival of CUP patients who received chemotherapy or systemic therapy. One model could predict the time to recurrence following immunotherapy in the cohort. Trial registration Retrospective registration of local ethics committees. |
| format | Article |
| id | doaj-art-dc4e2e1e985445dcafaa1f3ab8be78f6 |
| institution | Kabale University |
| issn | 1471-2407 |
| language | English |
| publishDate | 2025-08-01 |
| publisher | BMC |
| record_format | Article |
| series | BMC Cancer |
| spelling | doaj-art-dc4e2e1e985445dcafaa1f3ab8be78f62025-08-20T03:47:10ZengBMCBMC Cancer1471-24072025-08-0125111310.1186/s12885-025-14778-6Clinical efficacy of immune checkpoint inhibitors for cancer of unknown primary: a multi-center retrospective studyHaowen Wang0Siqi Song1Yuzhou Nie2Jinting Wu3Linyue Pan4Qizhen Zou5Gaizhen Kuang6Chen Chen7Xiaochen Zhang8Wenfeng Li9Shirong Zhang10Department of Interventional Medicine, Zhejiang Provincial People’s Hospital, People’s Hospital of Hangzhou Medical CollegeDepartment of Medical Oncology, the First Affiliated Hospital of Wenzhou Medical UniversityDepartment of Translational Medicine Research Center, Key Laboratory of Clinical Cancer Pharmacology and Toxicology Research of Zhejiang Province, Hangzhou First People’s HospitalDepartment of Oncology, Affiliated Jinhua Hospital, Zhejiang University School of MedicineDepartment of Thoracic Oncology, Zhejiang Cancer HospitalDepartment of Translational Medicine Research Center, Key Laboratory of Clinical Cancer Pharmacology and Toxicology Research of Zhejiang Province, Hangzhou First People’s HospitalDepartment of Medical Oncology, the First Affiliated Hospital of Wenzhou Medical UniversityDepartment of Medical Oncology, the First Affiliated Hospital of Wenzhou Medical UniversityDepartment of Medical Oncology, The First Affiliated Hospital, Zhejiang University School of MedicineDepartment of Medical Oncology, the First Affiliated Hospital of Wenzhou Medical UniversityDepartment of Translational Medicine Research Center, Key Laboratory of Clinical Cancer Pharmacology and Toxicology Research of Zhejiang Province, Hangzhou First People’s HospitalAbstract Background Patients diagnosed with cancer of unknown primary (CUP) have limited treatment options, resulting in a dismal prognosis. The efficacy of immune checkpoint inhibitors (ICIs) in these patients is still unclear. Methods We collected the clinical data of these patients retrospectively and multicentrically. In total, 190 patients diagnosed with CUP in 6 hospitals were collected, of which 58 patients received immunotherapy. Results The median overall survival (OS) was 17.3 months. The OS of the ICIs-treated group was significantly longer than that of the non-ICIs group in unfavorable subgroup, 29.27 months and 10.43 months, respectively (HR = 0.435, 95% CI 0.267–0.709; p = 0.0006). In the first-line systemic treatment, the OS and progression-free survival (PFS) in the ICIs group were significantly longer than that of the non-ICIs group. The OS was 45.53 months versus 12.03 months (HR = 0.431, 95% CI 0.259–0.717; p = 0.004), and the PFS was 11.33 months versus 5.43 months (HR = 0.558, 95% CI 0.351–0.889; p = 0.021). The objective response rate (ORR) and disease control rate (DCR) of ICIs group were higher too. Finally, a predictive nomogram model was developed to accurately forecast the response of patients with CUP to ICIs. Conclusions In this cohort of unfavorable subgroup, immunotherapy appears to extend the survival of CUP patients who received chemotherapy or systemic therapy. One model could predict the time to recurrence following immunotherapy in the cohort. Trial registration Retrospective registration of local ethics committees.https://doi.org/10.1186/s12885-025-14778-6CUPICIsUnfavorable subgroupImmunotherapySurvival. |
| spellingShingle | Haowen Wang Siqi Song Yuzhou Nie Jinting Wu Linyue Pan Qizhen Zou Gaizhen Kuang Chen Chen Xiaochen Zhang Wenfeng Li Shirong Zhang Clinical efficacy of immune checkpoint inhibitors for cancer of unknown primary: a multi-center retrospective study BMC Cancer CUP ICIs Unfavorable subgroup Immunotherapy Survival. |
| title | Clinical efficacy of immune checkpoint inhibitors for cancer of unknown primary: a multi-center retrospective study |
| title_full | Clinical efficacy of immune checkpoint inhibitors for cancer of unknown primary: a multi-center retrospective study |
| title_fullStr | Clinical efficacy of immune checkpoint inhibitors for cancer of unknown primary: a multi-center retrospective study |
| title_full_unstemmed | Clinical efficacy of immune checkpoint inhibitors for cancer of unknown primary: a multi-center retrospective study |
| title_short | Clinical efficacy of immune checkpoint inhibitors for cancer of unknown primary: a multi-center retrospective study |
| title_sort | clinical efficacy of immune checkpoint inhibitors for cancer of unknown primary a multi center retrospective study |
| topic | CUP ICIs Unfavorable subgroup Immunotherapy Survival. |
| url | https://doi.org/10.1186/s12885-025-14778-6 |
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