{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,15]],"date-time":"2026-10-15T18:12:23Z","timestamp":1792087943310,"version":"4.4.0"},"reference-count":24,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2025,9,1]],"date-time":"2025-09-01T00:00:00Z","timestamp":1756684800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2025,9,1]],"date-time":"2025-09-01T00:00:00Z","timestamp":1756684800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,9,1]],"date-time":"2025-09-01T00:00:00Z","timestamp":1756684800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-017"},{"start":{"date-parts":[[2025,9,1]],"date-time":"2025-09-01T00:00:00Z","timestamp":1756684800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-037"},{"start":{"date-parts":[[2025,9,1]],"date-time":"2025-09-01T00:00:00Z","timestamp":1756684800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-012"},{"start":{"date-parts":[[2025,9,1]],"date-time":"2025-09-01T00:00:00Z","timestamp":1756684800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-029"},{"start":{"date-parts":[[2025,9,1]],"date-time":"2025-09-01T00:00:00Z","timestamp":1756684800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-004"}],"content-domain":{"domain":["clinicalkey.com","clinicalkey.com.au","clinicalkey.es","clinicalkey.fr","clinicalkey.jp","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Leukemia Research"],"published-print":{"date-parts":[[2025,9]]},"DOI":"10.1016\/j.leukres.2025.107735","type":"journal-article","created":{"date-parts":[[2025,6,9]],"date-time":"2025-06-09T19:25:54Z","timestamp":1749497154000},"page":"107735","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":11,"special_numbering":"C","title":["Phase 1 first-in-human dose-expansion study of the oral SF3B1 modulator H3B\u20118800 in lower-risk myelodysplastic syndrome"],"prefix":"10.1016","volume":"156","author":[{"given":"James M.","family":"Foran","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Guillermo F.","family":"Sanz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5213-5482","authenticated-orcid":false,"given":"Justin M.","family":"Watts","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2169-4115","authenticated-orcid":false,"given":"Andrew M.","family":"Brunner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gaelle","family":"Fossard","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matteo Giovanni","family":"Della Porta","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4174-8766","authenticated-orcid":false,"given":"Xavier Cheng-Hong","family":"Tsai","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3631-2482","authenticated-orcid":false,"given":"Guillermo","family":"Garcia-Manero","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1772-4545","authenticated-orcid":false,"given":"Sophie","family":"Dimicoli-Salazar","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Luke","family":"Fletcher","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6653-2956","authenticated-orcid":false,"given":"Yoo Jin","family":"Kim","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patricia","family":"Font","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2478-5354","authenticated-orcid":false,"given":"Ana","family":"Alfonso-Pi\u00e9rola","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6233-5912","authenticated-orcid":false,"given":"Juan Manuel","family":"Alonso-Dominguez","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3645-148X","authenticated-orcid":false,"given":"Christopher","family":"Benton","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7829-397X","authenticated-orcid":false,"given":"Junshik","family":"Hong","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Luca","family":"Malcovati","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dominiek","family":"Mazure","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7060-1675","authenticated-orcid":false,"given":"Je-Hwan","family":"Lee","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6981-8677","authenticated-orcid":false,"given":"Su-Peng","family":"Yeh","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1915-9639","authenticated-orcid":false,"given":"Laure","family":"Goursaud","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wilma","family":"Barcellini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0003-1098-0153","authenticated-orcid":false,"given":"Elaine","family":"Wu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0002-4075-4731","authenticated-orcid":false,"given":"Deyanira","family":"Corzo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0009-7434-5603","authenticated-orcid":false,"given":"Keisuke","family":"Kuida","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7526-2633","authenticated-orcid":false,"given":"Richard M.","family":"Stone","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"issue":"14","key":"10.1016\/j.leukres.2025.107735_bib1","doi-asserted-by":"crossref","first-page":"1358","DOI":"10.1056\/NEJMra1904794","article-title":"Myelodysplastic syndromes","volume":"383","author":"Cazzola","year":"2020","journal-title":"N. Engl. J. Med"},{"issue":"2","key":"10.1016\/j.leukres.2025.107735_bib2","doi-asserted-by":"crossref","first-page":"106","DOI":"10.6004\/jnccn.2022.0009","article-title":"NCCN guidelines(R) insights: myelodysplastic syndromes, Version 3.2022","volume":"20","author":"Greenberg","year":"2022","journal-title":"J. Natl. Compr. Canc Netw."},{"issue":"1","key":"10.1016\/j.leukres.2025.107735_bib3","doi-asserted-by":"crossref","first-page":"426","DOI":"10.1182\/hematology.2020000127","article-title":"Therapy for lower-risk MDS","volume":"2020","author":"Carraway","year":"2020","journal-title":"Hematol. Am. Soc. Hematol. Educ. Program"},{"issue":"7","key":"10.1016\/j.leukres.2025.107735_bib4","doi-asserted-by":"crossref","DOI":"10.1056\/EVIDoa2200008","article-title":"Molecular international prognostic scoring system for myelodysplastic syndromes","volume":"1","author":"Bernard","year":"2022","journal-title":"NEJM Evid."},{"issue":"12","key":"10.1016\/j.leukres.2025.107735_bib5","doi-asserted-by":"crossref","first-page":"2894","DOI":"10.1038\/s41375-022-01728-5","article-title":"SF3B1 mutated MDS: blast count, genetic co-abnormalities and their impact on classification and prognosis","volume":"36","author":"Huber","year":"2022","journal-title":"Leukemia"},{"key":"10.1016\/j.leukres.2025.107735_bib6","article-title":"A novel prognostic scoring model for myelodysplastic syndrome patients with SF3B1 mutation","volume":"12","author":"Ma","year":"2022","journal-title":"Front Oncol."},{"issue":"2","key":"10.1016\/j.leukres.2025.107735_bib7","doi-asserted-by":"crossref","first-page":"233","DOI":"10.1182\/blood-2015-03-633537","article-title":"SF3B1 mutation identifies a distinct subset of myelodysplastic syndrome with ring sideroblasts","volume":"126","author":"Malcovati","year":"2015","journal-title":"Blood"},{"issue":"2","key":"10.1016\/j.leukres.2025.107735_bib8","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1182\/blood.2020004850","article-title":"SF3B1-mutant MDS as a distinct disease subtype: a proposal from the International Working Group for the Prognosis of MDS","volume":"136","author":"Malcovati","year":"2020","journal-title":"Blood"},{"issue":"13","key":"10.1016\/j.leukres.2025.107735_bib9","doi-asserted-by":"crossref","first-page":"2038","DOI":"10.1182\/blood.2021012652","article-title":"Coordinated missplicing of TMEM14C and ABCB7 causes ring sideroblast formation in SF3B1-mutant myelodysplastic syndrome","volume":"139","author":"Clough","year":"2022","journal-title":"Blood"},{"key":"10.1016\/j.leukres.2025.107735_bib10","doi-asserted-by":"crossref","first-page":"1116438","DOI":"10.3389\/fonc.2023.1116438","article-title":"SF3B1 mutations in myelodysplastic syndromes: a potential therapeutic target for modulating the entire disease process","volume":"13","author":"Jiang","year":"2023","journal-title":"Front Oncol."},{"issue":"2","key":"10.1016\/j.leukres.2025.107735_bib11","doi-asserted-by":"crossref","first-page":"140","DOI":"10.1056\/NEJMoa1908892","article-title":"Luspatercept in Patients with Lower-Risk Myelodysplastic Syndromes","volume":"382","author":"Fenaux","year":"2020","journal-title":"N. Engl. J. Med"},{"issue":"10399","key":"10.1016\/j.leukres.2025.107735_bib12","doi-asserted-by":"crossref","first-page":"348","DOI":"10.1016\/S0140-6736(23)01130-3","article-title":"Are we ready to ring in a new upfront therapy in lower-risk myelodysplastic syndromes?","volume":"402","author":"Carraway","year":"2023","journal-title":"Lancet"},{"issue":"10399","key":"10.1016\/j.leukres.2025.107735_bib13","doi-asserted-by":"crossref","first-page":"373","DOI":"10.1016\/S0140-6736(23)00874-7","article-title":"Efficacy and safety of luspatercept versus epoetin alfa in erythropoiesis-stimulating agent-naive, transfusion-dependent, lower-risk myelodysplastic syndromes (COMMANDS): interim analysis of a phase 3, open-label, randomised controlled trial","volume":"402","author":"Platzbecker","year":"2023","journal-title":"Lancet"},{"issue":"10423","key":"10.1016\/j.leukres.2025.107735_bib14","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1016\/S0140-6736(23)01724-5","article-title":"Imetelstat in patients with lower-risk myelodysplastic syndromes who have relapsed or are refractory to erythropoiesis-stimulating agents (IMerge): a multinational, randomised, double-blind, placebo-controlled, phase 3 trial","volume":"403","author":"Platzbecker","year":"2024","journal-title":"Lancet"},{"key":"10.1016\/j.leukres.2025.107735_bib15","unstructured":"REBLOZYL\u00ae (luspatercept-aamt) for injection, for subcutaneous use Initial U.S. Approval: 2019. Available at: \u3008https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2019\/761136lbl.pdf\u3009. Accessed August 2024."},{"key":"10.1016\/j.leukres.2025.107735_bib16","unstructured":"FDA approves imetelstat for low- to intermediate-1 risk myelodysplastic syndromes with transfusion-dependent anemia. Available at: \u3008https:\/\/www.fda.gov\/drugs\/resources-information-approved-drugs\/fda-approves-imetelstat-low-intermediate-1-risk-myelodysplastic-syndromes-transfusion-dependent\u3009. Accessed August 2024."},{"issue":"12","key":"10.1016\/j.leukres.2025.107735_bib17","doi-asserted-by":"crossref","first-page":"3542","DOI":"10.1038\/s41375-021-01328-9","article-title":"Phase I First-in-Human Dose Escalation Study of the oral SF3B1 modulator H3B-8800 in myeloid neoplasms","volume":"35","author":"Steensma","year":"2021","journal-title":"Leukemia"},{"issue":"8","key":"10.1016\/j.leukres.2025.107735_bib18","doi-asserted-by":"crossref","first-page":"1307","DOI":"10.1002\/ajh.26984","article-title":"Myelodysplastic syndromes: 2023 update on diagnosis, risk-stratification, and management","volume":"98","author":"Garcia-Manero","year":"2023","journal-title":"Am. J. Hematol."},{"issue":"1","key":"10.1016\/j.leukres.2025.107735_bib19","doi-asserted-by":"crossref","first-page":"576","DOI":"10.1182\/asheducation-2018.1.576","article-title":"Drug-associated thrombocytopenia","volume":"2018","author":"Bakchoul","year":"2018","journal-title":"Hematol. Am. Soc. Hematol. Educ. Program"},{"issue":"ement 1","key":"10.1016\/j.leukres.2025.107735_bib20","doi-asserted-by":"crossref","first-page":"34","DOI":"10.1182\/blood-2020-143319","article-title":"Burden of Cardiovascular Events and Bleeding Is High in Myelodysplastic Syndromes","volume":"136","author":"Chhetri","year":"2020","journal-title":"Blood"},{"key":"10.1016\/j.leukres.2025.107735_bib21","doi-asserted-by":"crossref","DOI":"10.1016\/j.phrs.2024.107077","article-title":"Drug-induced atrial fibrillation. A narrative review of a forgotten adverse effect","volume":"200","author":"Tamargo","year":"2024","journal-title":"Pharmacol. Res."},{"key":"10.1016\/j.leukres.2025.107735_bib22","doi-asserted-by":"crossref","first-page":"497","DOI":"10.1038\/nm.4493","article-title":"H3B-8800, an orally available small-molecule splicing modulator, induces lethality in spliceosome-mutant cancers","volume":"24","author":"Seiler","year":"2018","journal-title":"Nat. Med."},{"key":"10.1016\/j.leukres.2025.107735_bib23","first-page":"1217","article-title":"Cardiomyopathy and Arrhythmia Caused by Aberrant RNA Splicing","volume":"126","author":"Guo","year":"2020","journal-title":"Circ. Res."},{"key":"10.1016\/j.leukres.2025.107735_bib24","first-page":"366","article-title":"Translating Atrial Fibrillation Genetics Into Precision Medicine","volume":"9","author":"Deo","year":"2024","journal-title":"JACC Basic Translational Science"}],"container-title":["Leukemia Research"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0145212625000955?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0145212625000955?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,4]],"date-time":"2025-11-04T08:31:26Z","timestamp":1762245086000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0145212625000955"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,9]]},"references-count":24,"alternative-id":["S0145212625000955"],"URL":"https:\/\/doi.org\/10.1016\/j.leukres.2025.107735","relation":{},"ISSN":["0145-2126"],"issn-type":[{"value":"0145-2126","type":"print"}],"subject":[],"published":{"date-parts":[[2025,9]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Phase 1 first-in-human dose-expansion study of the oral SF3B1 modulator H3B\u20118800 in lower-risk myelodysplastic syndrome","name":"articletitle","label":"Article Title"},{"value":"Leukemia Research","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.leukres.2025.107735","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2025 Published by Elsevier Ltd.","name":"copyright","label":"Copyright"}],"article-number":"107735"}}