Real-World Outcomes of Ivosidenib in IDH1-Mutant Biliary Tract Cancer: A Multicenter Retrospective Analysis and Comparison with the TrinetX Database.

IF 4 3区医学 Q2 ONCOLOGY

Targeted Oncology Pub Date : 2026-04-20 DOI:10.1007/s11523-026-01211-6

Lena Dreikhausen, Alexander Olkus, Michael Dill, Christoph Springfeld, Philipp Heumann, Nadine Schulte, Konstantina Atanasova, Matthias P Ebert, Anne Letsch, Anna M Wandmacher, Tianzuo Zhan

{"title":"Real-World Outcomes of Ivosidenib in IDH1-Mutant Biliary Tract Cancer: A Multicenter Retrospective Analysis and Comparison with the TrinetX Database.","authors":"Lena Dreikhausen, Alexander Olkus, Michael Dill, Christoph Springfeld, Philipp Heumann, Nadine Schulte, Konstantina Atanasova, Matthias P Ebert, Anne Letsch, Anna M Wandmacher, Tianzuo Zhan","doi":"10.1007/s11523-026-01211-6","DOIUrl":null,"url":null,"abstract":"Background: Ivosidenib is established as targeted therapy in patients with isocitrate dehydrogenase 1 (IDH1)-mutant advanced biliary tract cancer based on results of the phase III ClarIDHy trial. However, data on the efficacy of ivosidenib in real-world cohorts are limited.Objective: This retrospective analysis aims to provide data on the efficacy and safety of ivosidenib in biliary tract cancer in a real-world cohort.Methods: Patients with IDH1 mutant biliary tract cancer treated with ivosidenib at four German university medical centers from 2020 to 2024 were retrospectively identified. Clinicopathological parameters and treatment courses were collected. Furthermore, clinical outcomes of patients with biliary tract cancer who received ivosidenib were retrospectively retrieved from the global health network database TriNetX.Results: In total, 14 patients who received ivosidenib were identified. Mean age at diagnosis was 61 years and 50% of patients were female. Nine patients had an IDH1 R132C, four an IHD1 R132L, and one an IDH1 R132S mutation. Ten patients received ivosidenib as second-line therapy and four in later lines. Partial response was observed in two patients, stable disease in three, and progressive disease in nine patients. Median progression-free survival was 5.6 months (95% confidence interval 4.3, not estimated) and median overall survival was 12.7 months (95% confidence interval 8.2, not estimated). No discontinuation of ivosidenib because of toxicity was observed. A total of 228 patients were identified in the TriNetX database. Mean age was 64.4 years and 64% of patients were female. Median overall survival in this cohort was 9.6 months (95% confidence interval 8.07, 11.19).Conclusions: Real-world data from our cohort and the TriNetX database support the oncological outcomes reported in the ClarIDHy trial and other real-world cohorts.","PeriodicalId":22195,"journal":{"name":"Targeted Oncology","volume":" ","pages":""},"PeriodicalIF":4.0000,"publicationDate":"2026-04-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Targeted Oncology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s11523-026-01211-6","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"ONCOLOGY","Score":null,"Total":0}

引用次数: 0

Abstract

Background: Ivosidenib is established as targeted therapy in patients with isocitrate dehydrogenase 1 (IDH1)-mutant advanced biliary tract cancer based on results of the phase III ClarIDHy trial. However, data on the efficacy of ivosidenib in real-world cohorts are limited.

Objective: This retrospective analysis aims to provide data on the efficacy and safety of ivosidenib in biliary tract cancer in a real-world cohort.

Methods: Patients with IDH1 mutant biliary tract cancer treated with ivosidenib at four German university medical centers from 2020 to 2024 were retrospectively identified. Clinicopathological parameters and treatment courses were collected. Furthermore, clinical outcomes of patients with biliary tract cancer who received ivosidenib were retrospectively retrieved from the global health network database TriNetX.

Results: In total, 14 patients who received ivosidenib were identified. Mean age at diagnosis was 61 years and 50% of patients were female. Nine patients had an IDH1 R132C, four an IHD1 R132L, and one an IDH1 R132S mutation. Ten patients received ivosidenib as second-line therapy and four in later lines. Partial response was observed in two patients, stable disease in three, and progressive disease in nine patients. Median progression-free survival was 5.6 months (95% confidence interval 4.3, not estimated) and median overall survival was 12.7 months (95% confidence interval 8.2, not estimated). No discontinuation of ivosidenib because of toxicity was observed. A total of 228 patients were identified in the TriNetX database. Mean age was 64.4 years and 64% of patients were female. Median overall survival in this cohort was 9.6 months (95% confidence interval 8.07, 11.19).

Conclusions: Real-world data from our cohort and the TriNetX database support the oncological outcomes reported in the ClarIDHy trial and other real-world cohorts.

查看原文本刊更多论文

Ivosidenib治疗idh1突变型胆道癌的实际结果：多中心回顾性分析和与TrinetX数据库的比较

背景：基于III期ClarIDHy试验的结果，Ivosidenib被确定为异柠檬酸脱氢酶1 （IDH1）突变的晚期胆道癌患者的靶向治疗。然而，关于ivosidenib在真实人群中的疗效的数据是有限的。目的：本回顾性分析旨在提供伊沃sidenib治疗胆道癌的有效性和安全性数据。方法：回顾性分析2020年至2024年在德国四所大学医学中心接受伊沃西地尼治疗的IDH1突变型胆道癌患者。收集临床病理参数及疗程。此外，从全球卫生网络数据库TriNetX中回顾性检索了接受伊沃西迪尼治疗的胆道癌患者的临床结果。结果：共确定14例接受伊沃西替尼治疗的患者。诊断时的平均年龄为61岁，50%的患者为女性。9名患者有IDH1 R132C突变，4名患者有IHD1 R132L突变，1名患者有IDH1 R132S突变。10例患者接受伊沃西迪尼作为二线治疗，4例接受后续治疗。2例患者部分缓解，3例病情稳定，9例病情进展。中位无进展生存期为5.6个月（95%可信区间为4.3，未估计），中位总生存期为12.7个月（95%可信区间为8.2，未估计）。没有观察到因毒性而停用伊沃西地尼。TriNetX数据库共确定了228例患者。平均年龄64.4岁，女性占64%。该队列的中位总生存期为9.6个月（95%可信区间为8.07,11.19）。结论：来自我们的队列和TriNetX数据库的真实数据支持ClarIDHy试验和其他真实队列中报告的肿瘤结果。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Targeted Oncology 医学-肿瘤学

CiteScore

8.40

自引率

3.70%

发文量

审稿时长

>12 weeks

期刊介绍： Targeted Oncology addresses physicians and scientists committed to oncology and cancer research by providing a programme of articles on molecularly targeted pharmacotherapy in oncology. The journal includes: Original Research Articles on all aspects of molecularly targeted agents for the treatment of cancer, including immune checkpoint inhibitors and related approaches. Comprehensive narrative Review Articles and shorter Leading Articles discussing relevant clinically established as well as emerging agents and pathways. Current Opinion articles that place interesting areas in perspective. Therapy in Practice articles that provide a guide to the optimum management of a condition and highlight practical, clinically relevant considerations and recommendations. Systematic Reviews that use explicit, systematic methods as outlined by the PRISMA statement. Adis Drug Reviews of the properties and place in therapy of both newer and established targeted drugs in oncology.