{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,25]],"date-time":"2026-08-25T13:23:33Z","timestamp":1787664213155,"version":"build-2736575974"},"reference-count":23,"publisher":"Springer Science and Business Media LLC","license":[{"start":{"date-parts":[[2026,8,25]],"date-time":"2026-08-25T00:00:00Z","timestamp":1787616000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"},{"start":{"date-parts":[[2026,8,25]],"date-time":"2026-08-25T00:00:00Z","timestamp":1787616000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Leukemia"],"DOI":"10.1038\/s41375-026-03100-3","type":"journal-article","created":{"date-parts":[[2026,8,25]],"date-time":"2026-08-25T13:04:24Z","timestamp":1787663064000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Ivosidenib monotherapy in IDH1 mutated myelodysplastic neoplasm\/syndrome"],"prefix":"10.1038","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4376-4097","authenticated-orcid":false,"given":"Marie","family":"S\u00e9bert","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emmanuelle","family":"Clappier","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sylvie","family":"Chevret","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hugo","family":"Bergugnat","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0321-6029","authenticated-orcid":false,"given":"Odile","family":"Rauzy","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aspasia","family":"Stamatoullas","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sophie","family":"Dimicoli-Salazar","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lamya Ait Si","family":"Selmi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cendrine","family":"Chaffaut","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fatiha","family":"Chermat","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9461-0728","authenticated-orcid":false,"given":"Lise","family":"Larcher","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lauriane","family":"Goldwirt","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sylvain","family":"Thepot","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pierre","family":"Peterlin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sophie","family":"Park","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marie-Pierre","family":"Gourin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Norbert","family":"Vey","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C\u00e9cile","family":"Bally","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"S\u00e9bastien","family":"Maury","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gaelle","family":"Fossard","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5909-2742","authenticated-orcid":false,"given":"Maud","family":"D\u2019Aveni","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne-Laure","family":"Taksin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6745-1127","authenticated-orcid":false,"given":"Thomas","family":"Cluzeau","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pierre","family":"Fenaux","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9020-8766","authenticated-orcid":false,"given":"Lionel","family":"Ad\u00e8s","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2026,8,25]]},"reference":[{"key":"3100_CR1","doi-asserted-by":"publisher","first-page":"1358","DOI":"10.1056\/NEJMra1904794","volume":"383","author":"M Cazzola","year":"2020","unstructured":"Cazzola M. Myelodysplastic syndromes. N Engl J Med. 2020;383:1358\u201374.","journal-title":"N Engl J Med"},{"key":"3100_CR2","doi-asserted-by":"publisher","first-page":"59","DOI":"10.1182\/hematology.2023000520","volume":"2023","author":"M S\u00e9bert","year":"2023","unstructured":"S\u00e9bert M. Next-generation therapy for lower-risk MDS. Hematol Am Soc Hematol Educ Program. 2023;2023:59\u201364.","journal-title":"Hematol Am Soc Hematol Educ Program"},{"key":"3100_CR3","doi-asserted-by":"publisher","first-page":"223","DOI":"10.1016\/S1470-2045(09)70003-8","volume":"10","author":"P Fenaux","year":"2009","unstructured":"Fenaux P, Mufti GJ, Hellstrom-Lindberg E, Santini V, Finelli C, Giagounidis A, et al. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol. 2009;10:223\u201332.","journal-title":"Lancet Oncol"},{"key":"3100_CR4","doi-asserted-by":"publisher","DOI":"10.1002\/hem3.70164","volume":"9","author":"DA Sallman","year":"2025","unstructured":"Sallman DA, Komrokji RS, Dezern AE, Sebert M, Garcia-Manero G, Rahm\u00e9 R, et al. Long-term follow-up and combined Phase 2 results of eprenetapopt and azacitidine in patients with TP53 mutant MDS\/AML. Hemasphere. 2025;9:e70164.","journal-title":"Hemasphere"},{"key":"3100_CR5","doi-asserted-by":"publisher","first-page":"5132","DOI":"10.1182\/bloodadvances.2022007334","volume":"6","author":"L Ad\u00e8s","year":"2022","unstructured":"Ad\u00e8s L, Girshova L, Doronin VA, D\u00edez-Campelo M, Valc\u00e1rcel D, Kambhampati S, et al. Pevonedistat plus azacitidine vs azacitidine alone in higher-risk MDS\/chronic myelomonocytic leukemia or low-blast-percentage AML. Blood Adv. 2022;6:5132\u201345.","journal-title":"Blood Adv"},{"key":"3100_CR6","doi-asserted-by":"publisher","first-page":"535","DOI":"10.1111\/bjh.18193","volume":"198","author":"L Ad\u00e8s","year":"2022","unstructured":"Ad\u00e8s L, Duployez N, Guerci-Bresler A, Laribi K, Peterlin P, Vey N, et al. A randomised phase II study of azacitidine (AZA) alone or with Lenalidomide (LEN), Valproic acid (VPA) or Idarubicin (IDA) in higher-Risk MDS or low blast AML: GFM\u2019s \u2018pick a winner\u2019 trial, with the impact of somatic mutations. Br J Haematol. 2022;198:535\u201344.","journal-title":"Br J Haematol"},{"key":"3100_CR7","doi-asserted-by":"publisher","first-page":"e38","DOI":"10.1016\/S2352-3026(23)00333-2","volume":"11","author":"AM Zeidan","year":"2024","unstructured":"Zeidan AM, Ando K, Rauzy O, Turgut M, Wang M-C, Cairoli R, et al. Sabatolimab plus hypomethylating agents in previously untreated patients with higher-risk myelodysplastic syndromes (STIMULUS-MDS1): a randomised, double-blind, placebo-controlled, phase 2 trial. Lancet Haematol. 2024;11:e38\u2013e50.","journal-title":"Lancet Haematol"},{"key":"3100_CR8","doi-asserted-by":"publisher","first-page":"2454","DOI":"10.1182\/blood-2012-03-420489","volume":"120","author":"PL Greenberg","year":"2012","unstructured":"Greenberg PL, Tuechler H, Schanz J, Sanz G, Garcia-Manero G, Sol\u00e9 F, et al. Revised international prognostic scoring system for myelodysplastic syndromes. Blood. 2012;120:2454\u201365.","journal-title":"Blood"},{"key":"3100_CR9","doi-asserted-by":"publisher","unstructured":"Bernard E, Tuechler H, Greenberg PL, Hasserjian RP, Arango Ossa JE, Nannya Y et al. Molecular international prognostic scoring system for myelodysplastic syndromes. NEJM Evidence 2022; 1. https:\/\/doi.org\/10.1056\/EVIDoa2200008.","DOI":"10.1056\/EVIDoa2200008"},{"key":"3100_CR10","doi-asserted-by":"publisher","first-page":"1200","DOI":"10.1182\/blood.2022015850","volume":"140","author":"DA Arber","year":"2022","unstructured":"Arber DA, Orazi A, Hasserjian RP, Borowitz MJ, Calvo KR, Kvasnicka H-M, et al. International Consensus Classification of Myeloid Neoplasms and Acute Leukemias: integrating morphologic, clinical, and genomic data. Blood. 2022;140:1200\u201328.","journal-title":"Blood"},{"key":"3100_CR11","doi-asserted-by":"publisher","first-page":"1703","DOI":"10.1038\/s41375-022-01613-1","volume":"36","author":"JD Khoury","year":"2022","unstructured":"Khoury JD, Solary E, Abla O, Akkari Y, Alaggio R, Apperley JF, et al. The 5th edition of the World Health Organization classification of haematolymphoid tumours: myeloid and histiocytic\/dendritic neoplasms. Leukemia. 2022;36:1703\u201319.","journal-title":"Leukemia"},{"key":"3100_CR12","doi-asserted-by":"publisher","first-page":"1617","DOI":"10.1182\/blood.2023023727","volume":"144","author":"E Bernard","year":"2024","unstructured":"Bernard E, Hasserjian RP, Greenberg PL, Arango Ossa JE, Creignou M, Tuechler H, et al. Molecular taxonomy of myelodysplastic syndromes and its clinical implications. Blood. 2024;144:1617\u201332.","journal-title":"Blood"},{"key":"3100_CR13","doi-asserted-by":"publisher","first-page":"225","DOI":"10.1016\/j.ccr.2010.01.020","volume":"17","author":"PS Ward","year":"2010","unstructured":"Ward PS, Patel J, Wise DR, Abdel-Wahab O, Bennett BD, Coller HA, et al. The common feature of leukemia-associated IDH1 and IDH2 mutations is a neomorphic enzyme activity converting alpha-ketoglutarate to 2-hydroxyglutarate. Cancer Cell. 2010;17:225\u201334.","journal-title":"Cancer Cell"},{"key":"3100_CR14","doi-asserted-by":"publisher","first-page":"2386","DOI":"10.1056\/NEJMoa1716984","volume":"378","author":"CD DiNardo","year":"2018","unstructured":"DiNardo CD, Stein EM, de Botton S, Roboz GJ, Altman JK, Mims AS, et al. Durable remissions with ivosidenib in IDH1-mutated relapsed or refractory AML. N Engl J Med. 2018;378:2386\u201398.","journal-title":"N Engl J Med"},{"key":"3100_CR15","doi-asserted-by":"publisher","first-page":"463","DOI":"10.1182\/blood.2019002140","volume":"135","author":"GJ Roboz","year":"2020","unstructured":"Roboz GJ, DiNardo CD, Stein EM, de Botton S, Mims AS, Prince GT, et al. Ivosidenib induces deep durable remissions in patients with newly diagnosed IDH1-mutant acute myeloid leukemia. Blood. 2020;135:463\u201371.","journal-title":"Blood"},{"key":"3100_CR16","doi-asserted-by":"publisher","first-page":"1519","DOI":"10.1056\/NEJMoa2117344","volume":"386","author":"P Montesinos","year":"2022","unstructured":"Montesinos P, Recher C, Vives S, Zarzycka E, Wang J, Bertani G, et al. Ivosidenib and azacitidine in IDH1-mutated acute myeloid leukemia. N Engl J Med. 2022;386:1519\u201331.","journal-title":"N Engl J Med"},{"key":"3100_CR17","doi-asserted-by":"publisher","first-page":"937","DOI":"10.1182\/blood-2009-03-209262","volume":"114","author":"JW Vardiman","year":"2009","unstructured":"Vardiman JW, Thiele J, Arber DA, Brunning RD, Borowitz MJ, Porwit A, et al. The 2008 revision of the World Health Organization (WHO) classification of myeloid neoplasms and acute leukemia: rationale and important changes. Blood. 2009;114:937\u201351.","journal-title":"Blood"},{"key":"3100_CR18","doi-asserted-by":"publisher","DOI":"10.1038\/s41467-023-36193-w","volume":"14","author":"M Boy","year":"2023","unstructured":"Boy M, Bisio V, Zhao L-P, Guidez F, Schell B, Lereclus E, et al. Myelodysplastic Syndrome associated TET2 mutations affect NK cell function and genome methylation. Nat Commun. 2023;14:588.","journal-title":"Nat Commun"},{"key":"3100_CR19","doi-asserted-by":"publisher","DOI":"10.1038\/s41467-019-13161-x","volume":"10","author":"P Gopal","year":"2019","unstructured":"Gopal P, Sarihan EI, Chie EK, Kuzmishin G, Doken S, Pennell NA, et al. Clonal selection confers distinct evolutionary trajectories in BRAF-driven cancers. Nat Commun. 2019;10:5143.","journal-title":"Nat Commun"},{"key":"3100_CR20","doi-asserted-by":"publisher","first-page":"4209","DOI":"10.1182\/bloodadvances.2023012302","volume":"8","author":"CD DiNardo","year":"2024","unstructured":"DiNardo CD, Roboz GJ, Watts JM, Madanat YF, Prince GT, Baratam P, et al. Final phase 1 substudy results of ivosidenib for patients with mutant IDH1 relapsed\/refractory myelodysplastic syndrome. Blood Adv. 2024;8:4209\u201320.","journal-title":"Blood Adv"},{"key":"3100_CR21","doi-asserted-by":"publisher","first-page":"3322","DOI":"10.1200\/JCO.2011.35.8135","volume":"29","author":"T Pr\u00e9bet","year":"2011","unstructured":"Pr\u00e9bet T, Gore SD, Esterni B, Gardin C, Itzykson R, Thepot S, et al. Outcome of high-risk myelodysplastic syndrome after azacitidine treatment failure. J Clin Oncol. 2011;29:3322\u20137.","journal-title":"J Clin Oncol"},{"key":"3100_CR22","doi-asserted-by":"publisher","first-page":"1126","DOI":"10.1182\/blood.2024025464","volume":"145","author":"JS Garcia","year":"2025","unstructured":"Garcia JS, Platzbecker U, Odenike O, Fleming S, Fong CY, Borate U, et al. Efficacy and safety of venetoclax plus azacitidine for patients with treatment-naive high-risk myelodysplastic syndromes. Blood. 2025;145:1126\u201335.","journal-title":"Blood"},{"key":"3100_CR23","doi-asserted-by":"publisher","first-page":"1102","DOI":"10.1016\/j.stem.2025.04.012","volume":"32","author":"M Sirenko","year":"2025","unstructured":"Sirenko M, Lee S, Sun Z, Chaligne R, Loghavi S, Asimomitis G, et al. Deconvoluting clonal and cellular architecture in IDH-mutant acute myeloid leukemia. Cell Stem Cell. 2025;32:1102\u20131121.e5.","journal-title":"Cell Stem Cell"}],"container-title":["Leukemia"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.nature.com\/articles\/s41375-026-03100-3.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.nature.com\/articles\/s41375-026-03100-3","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.nature.com\/articles\/s41375-026-03100-3.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,8,25]],"date-time":"2026-08-25T13:04:25Z","timestamp":1787663065000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.nature.com\/articles\/s41375-026-03100-3"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,8,25]]},"references-count":23,"alternative-id":["3100"],"URL":"https:\/\/doi.org\/10.1038\/s41375-026-03100-3","relation":{},"ISSN":["0887-6924","1476-5551"],"issn-type":[{"value":"0887-6924","type":"print"},{"value":"1476-5551","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,8,25]]},"assertion":[{"value":"12 April 2026","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"14 July 2026","order":2,"name":"revised","label":"Revised","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"31 July 2026","order":3,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"25 August 2026","order":4,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"MS reports consulting\/advisory fees from AbbVie, Servier, Pierre Fabre, BMS, and research funding from AbbVie and Servier. EC, SC, HB, OR, SD, LASS, CC, FC, LL, LG, ST, MPG, NV, CB, SM, GF, MDA, ALT reported no COI. PP declares consulting\/advisory fees from AbbVie, Astellas, BMS, Da\u00efchi-Sankyo and Servier. SP declares consulting\/advisory fees from BMS, Novartis and Pfizer. TC declares consulting\/advisory fees from BMS, AbbVie, Jazz Pharma, Novartis, Servier, Arog, Amgen, Syros, Takeda, Janssen, Keros, Incyte. LA declares consulting\/advisory fees from Kura, Takeda, Servier, Pierre Fabre, Janssen, Jazz Pharma and research support from BMS, AbbVie and Novartis.","order":1,"name":"Ethics","label":"Competing interests","group":{"name":"EthicsHeading","label":"Ethics"}},{"value":"This study was conducted in accordance with the Declaration of Helsinki. The study protocol and informed consent form were approved by the institutional review board\/ethics committee of the GFM. All patients provided written informed consent.","order":2,"name":"Ethics","label":"Ethics approval and consent to participate","group":{"name":"EthicsHeading","label":"Ethics"}}]}}