Pemigatinib in previously treated Chinese patients with locally advanced or metastatic cholangiocarcinoma carrying FGFR2 fusions or rearrangements: A phase II study
Abstract Objective This study evaluated the antitumor activity and safety of pemigatinib in previously treated Chinese patients with advanced cholangiocarcinoma and fibroblast growth factor receptor 2 (FGFR2) fusions or rearrangements. Background Pemigatinib provided clinical benefits for previously...
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| Format: | Article |
| Language: | English |
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Wiley
2023-02-01
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| Series: | Cancer Medicine |
| Subjects: | |
| Online Access: | https://doi.org/10.1002/cam4.5273 |
| _version_ | 1797893208483561472 |
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| author | Guo‐Ming Shi Xiao‐Yong Huang Tian‐Fu Wen Tian‐Qiang Song Ming Kuang Hai‐Bo Mou Le‐Qun Bao Hai‐Tao Zhao Hong Zhao Xie‐Lin Feng Bi‐Xiang Zhang Tao Peng Yu‐Bao Zhang Xiang‐Cheng Li Hong‐Sheng Yu Yu Cao Lian‐Xin Liu Ti Zhang Wei‐Lin Wang Jiang‐Hua Ran Ying‐Bin Liu Wei Gong Ming‐Xia Chen Lian Cao Yang Luo Yan Wang Hui Zhou Guo‐Huan Yang Jia Fan Jian Zhou |
| author_facet | Guo‐Ming Shi Xiao‐Yong Huang Tian‐Fu Wen Tian‐Qiang Song Ming Kuang Hai‐Bo Mou Le‐Qun Bao Hai‐Tao Zhao Hong Zhao Xie‐Lin Feng Bi‐Xiang Zhang Tao Peng Yu‐Bao Zhang Xiang‐Cheng Li Hong‐Sheng Yu Yu Cao Lian‐Xin Liu Ti Zhang Wei‐Lin Wang Jiang‐Hua Ran Ying‐Bin Liu Wei Gong Ming‐Xia Chen Lian Cao Yang Luo Yan Wang Hui Zhou Guo‐Huan Yang Jia Fan Jian Zhou |
| author_sort | Guo‐Ming Shi |
| collection | DOAJ |
| description | Abstract Objective This study evaluated the antitumor activity and safety of pemigatinib in previously treated Chinese patients with advanced cholangiocarcinoma and fibroblast growth factor receptor 2 (FGFR2) fusions or rearrangements. Background Pemigatinib provided clinical benefits for previously treated patients with cholangiocarcinoma carrying FGFR2 fusions or rearrangements and was approved for this indication in multiple countries. Methods In this ongoing, multicenter, single‐arm, phase II study, adult patients with locally advanced or metastatic cholangiocarcinoma carrying centrally confirmed FGFR2 fusions or rearrangements who had progressed on ≥1 systemic therapy received 13.5 mg oral pemigatinib once daily (3‐week cycle; 2 weeks on, 1 week off) until disease progression, unacceptable toxicity, or consent withdrawal. The primary endpoint was objective response rate (ORR) assessed by an independent radiology review committee. Results As of January 29, 2021, 31 patients were enrolled. The median follow‐up was 5.1 months (range, 1.5–9.3). Among 30 patients with FGFR2 fusions or rearrangements evaluated for efficacy, 15 patients achieved partial response (ORR, 50.0%; 95% confidence interval [CI], 31.3–68.7); 15 achieved stable disease, contributing to a disease control rate of 100% (95% CI, 88.4–100). The median time to response was 1.4 months (95% CI, 1.3–1.4), the median duration of response was not reached, and the median progression‐free survival was 6.3 months (95% CI, 4.9–not estimable [NE]). Eight (25.8%) of 31 patients had ≥grade 3 treatment‐emergent adverse events. Hyperphosphatemia, hypophosphatasemia, nail toxicities, and ocular disorders were mostly <grade 3, except for 2 events ≥grade 3. Conclusions The encouraging antitumor activity and favorable safety profile support the use of pemigatinib as a treatment in previously treated Chinese patients with cholangiocarcinoma and FGFR2 rearrangements. |
| first_indexed | 2024-04-10T06:49:14Z |
| format | Article |
| id | doaj.art-33db6e2caae9409c859c394540b3696a |
| institution | Directory Open Access Journal |
| issn | 2045-7634 |
| language | English |
| last_indexed | 2024-04-10T06:49:14Z |
| publishDate | 2023-02-01 |
| publisher | Wiley |
| record_format | Article |
| series | Cancer Medicine |
| spelling | doaj.art-33db6e2caae9409c859c394540b3696a2023-02-28T08:51:57ZengWileyCancer Medicine2045-76342023-02-011244137414610.1002/cam4.5273Pemigatinib in previously treated Chinese patients with locally advanced or metastatic cholangiocarcinoma carrying FGFR2 fusions or rearrangements: A phase II studyGuo‐Ming Shi0Xiao‐Yong Huang1Tian‐Fu Wen2Tian‐Qiang Song3Ming Kuang4Hai‐Bo Mou5Le‐Qun Bao6Hai‐Tao Zhao7Hong Zhao8Xie‐Lin Feng9Bi‐Xiang Zhang10Tao Peng11Yu‐Bao Zhang12Xiang‐Cheng Li13Hong‐Sheng Yu14Yu Cao15Lian‐Xin Liu16Ti Zhang17Wei‐Lin Wang18Jiang‐Hua Ran19Ying‐Bin Liu20Wei Gong21Ming‐Xia Chen22Lian Cao23Yang Luo24Yan Wang25Hui Zhou26Guo‐Huan Yang27Jia Fan28Jian Zhou29Liver Surgery and Transplantation, Zhongshan Hospital Fudan University Shanghai ChinaLiver Surgery and Transplantation, Zhongshan Hospital Fudan University Shanghai ChinaHepatobiliary Surgery, West China Hospital Sichuan University Chengdu ChinaHepatobiliary Surgery Tianjin Cancer Hospital Tian Jin ChinaDepartment of Oncology, Hepatobiliary and Pancreatic Surgery Center The First Affiliated Hospital of Sun Yat‐sen University Guangzhou ChinaMedical Oncology Shulan (Hangzhou) Hospital Hangzhou ChinaHepatobiliary Surgery Hubei Cancer Hospital Wuhan ChinaLiver Surgery Peking Union Medical College Hospital Beijing ChinaHematological Surgery Department Cancer Hospital of Chinese Academy of Medical Science Beijing ChinaHepatobiliary and Pancreatic Surgery Sichuan Cancer Hospital Chengdu ChinaHepatobiliary Surgery, Tongji Hospital Tongji Medical College of HUST Wuhan ChinaHepatological Surgery Department The First Affiliated Hospital of Guangxi Medical University Nanning ChinaHepatobiliary and Pancreatic Surgery Cancer Hospital Affiliated to Harbin Medical University Harbin ChinaLiver Surgery Jiangsu Province Hospital Nanjing ChinaOncology Department Affiliated Hospital of Qingdao University Qingdao ChinaPhase 1 Clinical Research Center Affiliated Hospital of Qingdao University Qingdao ChinaHepatobiliary Surgery Department The First Affiliated Hospital of University of Science and Technology of China Hefei ChinaHepatobiliary Surgery Department Tianjin Cancer Hospital Tianjin ChinaHepatopancreatobiliary Surgery The Second Affiliated Hospital of Zhejiang University School of Medicine Hangzhou ChinaHepatopancreatobiliary Surgery The First Hospital of Kunming Kunming ChinaGeneral Surgery Xin Hua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine Shanghai ChinaGeneral Surgery Xin Hua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine Shanghai ChinaDepartment of Medical Science and Oncological Strategy Innovent Biologics Inc. Suzhou ChinaDepartment of Medical Science and Oncological Strategy Innovent Biologics Inc. Suzhou ChinaDepartment of Medical Science and Oncological Strategy Innovent Biologics Inc. Suzhou ChinaDepartment of Medical Science and Oncological Strategy Innovent Biologics Inc. Suzhou ChinaDepartment of Medical Science and Oncological Strategy Innovent Biologics Inc. Suzhou ChinaLiver Surgery and Transplantation, Zhongshan Hospital Fudan University Shanghai ChinaLiver Surgery and Transplantation, Zhongshan Hospital Fudan University Shanghai ChinaLiver Surgery and Transplantation, Zhongshan Hospital Fudan University Shanghai ChinaAbstract Objective This study evaluated the antitumor activity and safety of pemigatinib in previously treated Chinese patients with advanced cholangiocarcinoma and fibroblast growth factor receptor 2 (FGFR2) fusions or rearrangements. Background Pemigatinib provided clinical benefits for previously treated patients with cholangiocarcinoma carrying FGFR2 fusions or rearrangements and was approved for this indication in multiple countries. Methods In this ongoing, multicenter, single‐arm, phase II study, adult patients with locally advanced or metastatic cholangiocarcinoma carrying centrally confirmed FGFR2 fusions or rearrangements who had progressed on ≥1 systemic therapy received 13.5 mg oral pemigatinib once daily (3‐week cycle; 2 weeks on, 1 week off) until disease progression, unacceptable toxicity, or consent withdrawal. The primary endpoint was objective response rate (ORR) assessed by an independent radiology review committee. Results As of January 29, 2021, 31 patients were enrolled. The median follow‐up was 5.1 months (range, 1.5–9.3). Among 30 patients with FGFR2 fusions or rearrangements evaluated for efficacy, 15 patients achieved partial response (ORR, 50.0%; 95% confidence interval [CI], 31.3–68.7); 15 achieved stable disease, contributing to a disease control rate of 100% (95% CI, 88.4–100). The median time to response was 1.4 months (95% CI, 1.3–1.4), the median duration of response was not reached, and the median progression‐free survival was 6.3 months (95% CI, 4.9–not estimable [NE]). Eight (25.8%) of 31 patients had ≥grade 3 treatment‐emergent adverse events. Hyperphosphatemia, hypophosphatasemia, nail toxicities, and ocular disorders were mostly <grade 3, except for 2 events ≥grade 3. Conclusions The encouraging antitumor activity and favorable safety profile support the use of pemigatinib as a treatment in previously treated Chinese patients with cholangiocarcinoma and FGFR2 rearrangements.https://doi.org/10.1002/cam4.5273antitumor activitycholangiocarcinomaFGFR2 fusions or rearrangementspemigatinibphase II |
| spellingShingle | Guo‐Ming Shi Xiao‐Yong Huang Tian‐Fu Wen Tian‐Qiang Song Ming Kuang Hai‐Bo Mou Le‐Qun Bao Hai‐Tao Zhao Hong Zhao Xie‐Lin Feng Bi‐Xiang Zhang Tao Peng Yu‐Bao Zhang Xiang‐Cheng Li Hong‐Sheng Yu Yu Cao Lian‐Xin Liu Ti Zhang Wei‐Lin Wang Jiang‐Hua Ran Ying‐Bin Liu Wei Gong Ming‐Xia Chen Lian Cao Yang Luo Yan Wang Hui Zhou Guo‐Huan Yang Jia Fan Jian Zhou Pemigatinib in previously treated Chinese patients with locally advanced or metastatic cholangiocarcinoma carrying FGFR2 fusions or rearrangements: A phase II study Cancer Medicine antitumor activity cholangiocarcinoma FGFR2 fusions or rearrangements pemigatinib phase II |
| title | Pemigatinib in previously treated Chinese patients with locally advanced or metastatic cholangiocarcinoma carrying FGFR2 fusions or rearrangements: A phase II study |
| title_full | Pemigatinib in previously treated Chinese patients with locally advanced or metastatic cholangiocarcinoma carrying FGFR2 fusions or rearrangements: A phase II study |
| title_fullStr | Pemigatinib in previously treated Chinese patients with locally advanced or metastatic cholangiocarcinoma carrying FGFR2 fusions or rearrangements: A phase II study |
| title_full_unstemmed | Pemigatinib in previously treated Chinese patients with locally advanced or metastatic cholangiocarcinoma carrying FGFR2 fusions or rearrangements: A phase II study |
| title_short | Pemigatinib in previously treated Chinese patients with locally advanced or metastatic cholangiocarcinoma carrying FGFR2 fusions or rearrangements: A phase II study |
| title_sort | pemigatinib in previously treated chinese patients with locally advanced or metastatic cholangiocarcinoma carrying fgfr2 fusions or rearrangements a phase ii study |
| topic | antitumor activity cholangiocarcinoma FGFR2 fusions or rearrangements pemigatinib phase II |
| url | https://doi.org/10.1002/cam4.5273 |
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