{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,29]],"date-time":"2026-10-29T12:07:35Z","timestamp":1793275655499,"version":"4.5.0"},"reference-count":22,"publisher":"American Society of Clinical Oncology (ASCO)","issue":"6","content-domain":{"domain":["ascopubs.org"],"crossmark-restriction":true},"short-container-title":["JCO Precision Oncology"],"published-print":{"date-parts":[[2022,5]]},"abstract":"<jats:sec><jats:title>Purpose<\/jats:title><jats:p> Clinical-pathologic features and natural history of patients with isocitrate dehydrogenase ( IDH)-mutant intrahepatic cholangiocarcinoma (CCA) are not well characterized. Here, we sought to describe the natural history, clinical phenotype, and prognostic impact of advanced, IDH-mutated CCA. <\/jats:p><\/jats:sec><jats:sec><jats:title>METHODS<\/jats:title><jats:p> We conducted a multicentric, retrospective analysis of patients with IDH-mutated (IDH1 or IDH2) CCA between 2010 and 2020. Median overall survival (OS) and progression-free survival (PFS) analyses were performed using the Kaplan-Meier method. Chi-square test was used to analyze disease control rate (DCR) and overall response rate (ORR). Matched controls were used for comparing survival between patients with and without IDH mutations (mIDH). <\/jats:p><\/jats:sec><jats:sec><jats:title>RESULTS<\/jats:title><jats:p> Sixty-five patients with IDH-mutated CCA were included. All patients had intrahepatic CCA. On first-line chemotherapy, median OS and median PFS were 21.2 months and 8.3 months, respectively. Notably, median OS (32.4 v 19.5 months, P = .12) and PFS (18.0 v 8.0 months, P = .12) were not significantly affected by disease status at presentation (locally advanced v metastatic, respectively). Median OS was significantly longer in patients with mIDH (21.2 v 10.5 months; P &lt; .01). First-line gemcitabine-containing regimens had a significantly higher DCR and ORR than non\u2013gemcitabine-containing regimens (DCR: 75% v 33%, P = .01; ORR: 39% v 0%, P = .02). In patients receiving IDH inhibitor therapy, median PFS was 4.6 months with a DCR of 29%. <\/jats:p><\/jats:sec><jats:sec><jats:title>CONCLUSION<\/jats:title><jats:p> CCA with m IDH confers a unique subtype resulting in a better survival compared with that of counterparts. IDH inhibitors represent a promising therapeutic option in later lines of therapy in this subgroup. <\/jats:p><\/jats:sec>","DOI":"10.1200\/po.21.00156","type":"journal-article","created":{"date-parts":[[2022,1,5]],"date-time":"2022-01-05T20:59:37Z","timestamp":1641416377000},"update-policy":"https:\/\/doi.org\/10.1200\/crossmark","source":"Crossref","is-referenced-by-count":17,"title":["Isocitrate Dehydrogenase\u2013Mutated Cholangiocarcinoma: Natural History and Clinical Outcomes"],"prefix":"10.1200","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4492-6342","authenticated-orcid":false,"given":"Grant","family":"Wintheiser","sequence":"first","affiliation":[{"name":"Division of Internal Medicine, Mayo Clinic, Rochester, MN"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tyler","family":"Zemla","sequence":"additional","affiliation":[{"name":"Division of Biostatistics, Mayo Clinic, Rochester, MN"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Qian","family":"Shi","sequence":"additional","affiliation":[{"name":"Department of Quantitative Health Sciences, Mayo Clinic, Rochester, MN"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6104-6489","authenticated-orcid":false,"given":"Nguyen","family":"Tran","sequence":"additional","affiliation":[{"name":"Department of Medical Oncology, Mayo Clinic, Rochester, MN"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kritika","family":"Prasai","sequence":"additional","affiliation":[{"name":"Division of Gastroenterology and Hepatology, Mayo Clinic, Rochester, MN"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sri Harsha","family":"Tella","sequence":"additional","affiliation":[{"name":"Department of Medical Oncology, Mayo Clinic, Rochester, MN"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3254-2990","authenticated-orcid":false,"given":"Kabir","family":"Mody","sequence":"additional","affiliation":[{"name":"Division of Medical Oncology, Mayo Clinic, Jacksonville, FL"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel","family":"Ahn","sequence":"additional","affiliation":[{"name":"Department Medical Oncology, Mayo Clinic, Phoenix, AZ"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2700-2658","authenticated-orcid":false,"given":"Mitesh","family":"Borad","sequence":"additional","affiliation":[{"name":"Department Medical Oncology, Mayo Clinic, Phoenix, AZ"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7721-1699","authenticated-orcid":false,"given":"Tanios","family":"Bekaii-Saab","sequence":"additional","affiliation":[{"name":"Department Medical Oncology, Mayo Clinic, Phoenix, AZ"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1111-8333","authenticated-orcid":false,"given":"Amit","family":"Mahipal","sequence":"additional","affiliation":[{"name":"Department of Medical Oncology, Mayo Clinic, Rochester, MN"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"233","reference":[{"key":"b1","doi-asserted-by":"publisher","DOI":"10.1053\/j.gastro.2009.02.038"},{"key":"b2","doi-asserted-by":"publisher","DOI":"10.1002\/cncr.30254"},{"key":"b3","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0115383"},{"key":"b4","author":"Jain A","journal-title":"JCO Precis Oncol"},{"key":"b5","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pgen.1004135"},{"key":"b6","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1502309"},{"key":"b7","doi-asserted-by":"publisher","DOI":"10.1126\/science.aan6733"},{"key":"b8","doi-asserted-by":"publisher","DOI":"10.1093\/annonc\/mdw013"},{"key":"b9","doi-asserted-by":"publisher","DOI":"10.1016\/S1470-2045(19)30733-8"},{"key":"b10","doi-asserted-by":"publisher","DOI":"10.21037\/jgo.2019.03.10"},{"key":"b11","doi-asserted-by":"publisher","DOI":"10.1158\/1078-0432.CCR-12-1773"},{"key":"b12","doi-asserted-by":"publisher","DOI":"10.1016\/S1470-2045(20)30157-1"},{"key":"b13","doi-asserted-by":"publisher","DOI":"10.1634\/theoncologist.2015-0210"},{"key":"b14","doi-asserted-by":"publisher","DOI":"10.1001\/jamasurg.2013.5137"},{"key":"b15","doi-asserted-by":"publisher","DOI":"10.1002\/cncr.31942"},{"key":"b16","doi-asserted-by":"publisher","DOI":"10.1634\/theoncologist.2011-0386"},{"key":"b17","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa0908721"},{"key":"b18","doi-asserted-by":"publisher","DOI":"10.1038\/sj.bjc.6605779"},{"key":"b19","first-page":"3827","volume":"21","author":"Zhu AX","year":"2014","journal-title":"Ther Targets"},{"key":"b20","doi-asserted-by":"publisher","DOI":"10.1186\/s12885-020-06804-6"},{"key":"b21","doi-asserted-by":"publisher","DOI":"10.1038\/s41598-020-72810-0"},{"key":"b22","doi-asserted-by":"publisher","DOI":"10.1097\/CAD.0b013e3283637292"}],"updated-by":[{"DOI":"10.1200\/po.22.00162","type":"correction","label":"Correction","source":"publisher","updated":{"date-parts":[[2022,4,6]],"date-time":"2022-04-06T00:00:00Z","timestamp":1649203200000}}],"container-title":["JCO Precision Oncology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/ascopubs.org\/doi\/pdfdirect\/10.1200\/PO.21.00156","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2022,5,29]],"date-time":"2022-05-29T11:04:01Z","timestamp":1653822241000},"score":1,"resource":{"primary":{"URL":"https:\/\/ascopubs.org\/doi\/10.1200\/PO.21.00156"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,5]]},"references-count":22,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2022,5]]}},"alternative-id":["10.1200\/PO.21.00156"],"URL":"https:\/\/doi.org\/10.1200\/po.21.00156","relation":{"correction":[{"id-type":"doi","id":"10.1200\/PO.22.00162","asserted-by":"object"}]},"ISSN":["2473-4284"],"issn-type":[{"value":"2473-4284","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,5]]},"assertion":[{"value":"2021-04-10","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2021-09-27","order":1,"name":"revised","label":"Revised","group":{"name":"publication_history","label":"Publication History"}},{"value":"2021-11-23","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2022-01-05","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"e2100156"}}