{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,10]],"date-time":"2026-09-10T04:56:03Z","timestamp":1789016163844,"version":"build-2803163510"},"reference-count":28,"publisher":"American Society of Clinical Oncology (ASCO)","issue":"28","content-domain":{"domain":["ascopubs.org"],"crossmark-restriction":true},"short-container-title":["JCO"],"published-print":{"date-parts":[[2023,10,1]]},"abstract":"<jats:sec><jats:title>PURPOSE<\/jats:title><jats:p> In myelodysplastic syndromes (MDS), severe thrombocytopenia is associated with poor prognosis. This multicenter trial presents the second-part long-term efficacy and safety results of eltrombopag in patients with low-risk MDS and severe thrombocytopenia. <\/jats:p><\/jats:sec><jats:sec><jats:title>METHODS<\/jats:title><jats:p> In this single-blind, randomized, placebo-controlled, phase-II trial of adult patients with International Prognostic Scoring System low- or intermediate-1-risk MDS, patients with a stable platelet (PLT) count (&lt;30 \u00d7 10<jats:sup>3<\/jats:sup>\/mm<jats:sup>3<\/jats:sup>) received eltrombopag or placebo until disease progression. Primary end points were duration of PLT response (PLT-R; calculated from the time of PLT-R to date of loss of PLT-R, defined as bleeding\/PLT count &lt;30 \u00d7 10<jats:sup>3<\/jats:sup>\/mm<jats:sup>3<\/jats:sup> or last date in observation) and long-term safety and tolerability. Secondary end points included incidence and severity of bleeding, PLT transfusions, quality of life, leukemia-free survival, progression-free survival, overall survival and pharmacokinetics. <\/jats:p><\/jats:sec><jats:sec><jats:title>RESULTS<\/jats:title><jats:p> From 2011 to 2021, of 325 patients screened, 169 patients were randomly assigned oral eltrombopag (N = 112) or placebo (N = 57) at a starting dose of 50 mg once daily to maximum of 300 mg. PLT-R, with 25-week follow-up (IQR, 14-68) occurred in 47\/111 (42.3%) eltrombopag patients versus 6\/54 (11.1%) in placebo (odds ratio, 5.9; 95% CI, 2.3 to 14.9; P &lt; .001). In eltrombopag patients, 12\/47 (25.5%) lost the PLT-R, with cumulative thrombocytopenia relapse-free survival at 60 months of 63.6% (95% CI, 46.0 to 81.2). Clinically significant bleeding (WHO bleeding score \u2265 2) occurred less frequently in the eltrombopag arm than in the placebo group (incidence rate ratio, 0.54; 95% CI, 0.38 to 0.75; P = .0002). Although no difference in the frequency of grade 1-2 adverse events (AEs) was observed, a higher proportion of eltrombopag patients experienced grade 3-4 AEs (\u03c7<jats:sup>2<\/jats:sup> = 9.5, P = .002). AML evolution and\/or disease progression occurred in 17% (for both) of eltrombopag and placebo patients with no difference in survival times. <\/jats:p><\/jats:sec><jats:sec><jats:title>CONCLUSION<\/jats:title><jats:p> Eltrombopag was effective and relatively safe in low-risk MDS with severe thrombocytopenia. This trial is registered with ClinicalTrials.gov identifier: NCT02912208 and EU Clinical Trials Register: EudraCT No. 2010-022890-33. <\/jats:p><\/jats:sec>","DOI":"10.1200\/jco.22.02699","type":"journal-article","created":{"date-parts":[[2023,6,9]],"date-time":"2023-06-09T19:59:14Z","timestamp":1686340754000},"page":"4486-4496","update-policy":"https:\/\/doi.org\/10.1200\/crossmark","source":"Crossref","is-referenced-by-count":41,"title":["Eltrombopag for Low-Risk Myelodysplastic Syndromes With Thrombocytopenia: Interim Results of a Phase II, Randomized, Placebo-Controlled Clinical Trial (EQOL-MDS)"],"prefix":"10.1200","volume":"41","clinical-trial-number":[{"clinical-trial-number":"nct02912208","registry":"10.18810\/clinical-trials-gov"}],"author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-9238-5734","authenticated-orcid":false,"given":"Esther Natalie","family":"Oliva","sequence":"first","affiliation":[{"name":"U.O.C. 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