A new type of targeted therapy for the idh-1 mutation of bile duct cancer.

This article is from a new therapy targeting idh-1 mutations for cholangiocarcinoma, a new therapy targeting idh-1 mutations in cholangiocarcinoma. The authors: David H. ilson, MD, phdivosidenib group had significantly longer progression free survival than placebo group.cholangiocarcinoma is a kind of cancer which is sensitive to chemotherapy.although gemcitabine combined with cisplatin is the standard treatment, the efficacy of second-line and posterior line treatment is limited.genomic analysis showed that there are a large number of genes that can be used as therapeutic targets in cholangiocarcinoma, and genomic heterogeneity exists among primary intrahepatic cholangiocarcinoma, primary extrahepatic cholangiocarcinoma and primary gallbladder carcinoma.the results of an international, double-blind, placebo-controlled, randomized phase 3 trial (claridhy) sponsored by the industry recently released the results.this study compared the efficacy of ivosedenib (a small molecule inhibitor of mutant idh-1) and placebo in 185 patients (61% - 65% female) with previously treated cholangiocarcinoma carrying idh-1 mutation.most patients had primary intrahepatic cholangiocarcinoma (90% - 95%) with distant metastasis (92% - 93%).at a median follow-up of 6.9 months, the median progression free survival (PFS; primary efficacy endpoint) of the ivosedenib group was better than that of the placebo group (2.7 months vs. 1.4 months; risk ratio, 0.37; P < 0.0001), and the progression free survival rate at 6 months (32% vs. 22%) and 12 months (22% vs. 0%) was also better than that of the placebo group.this trial allows patients in the placebo group to receive ivosedenib after progression, so the median overall survival time of the patients in the ivosedenib group and the placebo group is similar (10.8 months and 9.7 months, respectively). In thegroup, only 2% of patients achieved objective remission, but 51% achieved stable disease. Treatment related adverse events, including pleural effusion, elevated bilirubin levels and prolonged QT interval, were not common in the ivosedenib group.the decline of quality of life in patients with ivosedenib was less than that in patients in placebo group.comment: ivosedenib is an active drug targeting idh-1 mutation in chemotherapy-resistant cholangiocarcinoma, which can improve PFS and stabilize the disease.genomic analysis of these cancers should be part of the standard treatment program to identify mutations as therapeutic targets. About Alfa GK et al. Ivosidenib in IDH1 mutant, chemotherapy refinery cholangiocarcinoma (claridhy): a multicenter, random, double blind, placebo controlled, phase 3 study. Lancet Oncol 2020 Jun; NEJM journal watch is published by NEJM group. Famous international doctors are invited to comment on important papers in medical field to help doctors understand and apply the latest progress."NEJM medical frontier" translated several articles every week, published on app and official website, and 2-3 selected articles were published on wechat.copyright information this article is translated, compiled or invited by the editorial department of NEJM medical frontier.for the translation and writing of articles originated from the English products of NEJM group, the original English version shall prevail.the full text of the Chinese translation and the charts included are exclusively authorized by the NEJM group of the Massachusetts Medical Association. for reprint, please contact nejmqianyan@nejmqianyan.cn 。 unauthorized translation is an infringement, and the copyright owner reserves the right to investigate the legal liability.