biostudies-literatureUnknown41(28)Oliva EN<h4>Purpose</h4>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.<h4>Methods</h4>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 (<30 × 10³/mm³) 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 <30 × 10³/mm³ or last date in observatiJournal of clinical oncology : official journal of the American Society of Clinical Oncology4486-4496https://www.ebi.ac.uk/biostudies/studies/S-EPMC10552995biostudies-literatureEltrombopag for Low-Risk Myelodysplastic Syndromes With Thrombocytopenia: Interim Results of a Phase II, Randomized, Placebo-Controlled Clinical Trial (EQOL-MDS).PMC10552995Russo EGuarini ARiva MOliva ENCandoni ABreccia MBeltrami GFenaux PLatagliata RCascavilla NPatriarca AFattizzo BReda GDelfino IMBuccisano FStamatoullas-Bastard ALiberati AMCapodanno IFerrero DNiscola PCrisa EBocchia MTripepi GPalumbo GAGerming URivellini FPoloni ABova ID'Errigo MGRicco AArcadi MTIanni GCufari PRizzo LSantini VCastelli AMolteni AImpera SDi Renzo NGiai VZini GSalutari PfalseEltrombopag for Low-Risk Myelodysplastic Syndromes With Thrombocytopenia: Interim Results of a Phase II, Randomized, Placebo-Controlled Clinical Trial (EQOL-MDS).<h4>Purpose</h4>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.<h4>Methods</h4>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 (<30 × 10³/mm³) 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 <30 × 10³/mm³ or last date in observati2023-01-01T00:00:00Z2023 Oct2025-04-26T01:58:59.633Z2025-04-06T10:18:13.888ZS-EPMC105529953729491410.1200/JCO.22.02699