{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,23]],"date-time":"2026-10-23T03:12:39Z","timestamp":1792725159716,"version":"4.5.0"},"reference-count":24,"publisher":"American Society of Hematology","issue":"7","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2020,2,13]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Ivosidenib (AG-120) is an oral, targeted agent that suppresses production of the oncometabolite 2-hydroxyglutarate via inhibition of the mutant isocitrate dehydrogenase 1 (IDH1; mIDH1) enzyme. From a phase 1 study of 258 patients with\u00a0IDH1-mutant hematologic malignancies, we report results for 34 patients with newly diagnosed acute myeloid leukemia (AML) ineligible for standard therapy who received 500 mg ivosidenib daily. Median age was 76.5 years, 26 patients (76%) had secondary AML, and 16 (47%) had received\u00a0\u22651 hypomethylating agent for an antecedent hematologic disorder. The most common all-grade adverse events were diarrhea (n = 18; 53%), fatigue (n = 16; 47%), nausea (n = 13; 38%), and decreased appetite (n = 12; 35%). Differentiation syndrome was reported in 6 patients (18%) (grade\u00a0\u22653 in 3 [9%]) and did not require treatment discontinuation. Complete remission (CR) plus CR with partial hematologic recovery (CRh) rate was 42.4% (95% confidence interval [CI], 25.5% to 60.8%); CR 30.3% (95% CI, 15.6% to 48.7%). Median durations of CR+CRh and CR were not reached, with 95% CI lower bounds of 4.6 and 4.2 months, respectively; 61.5% and 77.8% of patients remained in remission at 1 year. With median follow-up of 23.5 months (range, 0.6-40.9 months), median overall survival was 12.6 months (95% CI, 4.5-25.7). Of 21 transfusion-dependent patients (63.6%) at baseline, 9 (42.9%) became transfusion independent.\u00a0IDH1\u00a0mutation clearance was seen in 9\/14 patients achieving CR+CRh (5\/10 CR; 4\/4 CRh). Ivosidenib monotherapy was well-tolerated and induced durable remissions and transfusion independence in patients with newly diagnosed AML. This trial was registered at www.clinicaltrials.gov as #NCT02074839.<\/jats:p>","DOI":"10.1182\/blood.2019002140","type":"journal-article","created":{"date-parts":[[2019,12,16]],"date-time":"2019-12-16T15:52:30Z","timestamp":1576511550000},"page":"463-471","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":363,"title":["Ivosidenib induces deep durable remissions in patients with newly diagnosed IDH1-mutant acute myeloid leukemia"],"prefix":"10.1182","volume":"135","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0384-3658","authenticated-orcid":false,"given":"Gail J.","family":"Roboz","sequence":"first","affiliation":[{"name":"Weill Cornell Medicine\/NewYork-Presbyterian Hospital, New York, NY;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9003-0390","authenticated-orcid":false,"given":"Courtney D.","family":"DiNardo","sequence":"first","affiliation":[{"name":"University of Texas MD Anderson Cancer Center, Houston, TX;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eytan M.","family":"Stein","sequence":"first","affiliation":[{"name":"Memorial Sloan Kettering Cancer Center, New York, NY;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8126-4942","authenticated-orcid":false,"given":"St\u00e9phane","family":"de Botton","sequence":"first","affiliation":[{"name":"Institut Gustave Roussy, Villejuif, France;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alice S.","family":"Mims","sequence":"first","affiliation":[{"name":"Ohio State University Wexner Medical Center, Columbus, OH;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gabrielle T.","family":"Prince","sequence":"first","affiliation":[{"name":"The Johns Hopkins Hospital, Baltimore, MD;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jessica K.","family":"Altman","sequence":"first","affiliation":[{"name":"Robert H. 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