The following is a summary of “Eltrombopag for Low-Risk Myelodysplastic Syndromes With Thrombocytopenia: Interim Results of a Phase II, Randomized, Placebo-Controlled Clinical Trial (EQOL-MDS),” published in the October 2023 issue of Oncology by Oliva, et al.
For a multicenter trial, researchers sought to present eltrombopag’s long-term efficacy and safety results in patients with low-risk myelodysplastic syndromes (MDS) who also had severe thrombocytopenia, a condition linked to poor prognosis.
In the single-blind, randomized, placebo-controlled, phase-II trial involving adult patients with low- or intermediate-1-risk MDS according to the International Prognostic Scoring System, individuals with a stable platelet (PLT) count of less than 30 × 103/mm3 received either eltrombopag or a placebo until their disease progressed. The primary endpoints of the study were the duration of platelet response (PLT-R), defined as the time from achieving PLT-R to the date of PLT-R loss (defined as bleeding or a PLT count below 30 × 103/mm3, or the last observation date), and the long-term safety and tolerability of the treatment. Secondary endpoints included the incidence and severity of bleeding, PLT transfusions, quality of life, leukemia-free survival, progression-free survival, overall survival, and pharmacokinetics.
Between 2011 and 2021, out of 325 screened patients, 169 were randomly assigned to receive either oral eltrombopag (N = 112) or a placebo (N = 57), starting at a dose of 50 mg once daily, up to a maximum of 300 mg. PLT-R was achieved in 47 out of 111 (42.3%) eltrombopag patients versus 6 out of 54 (11.1%) in the placebo group (odds ratio, 5.9; 95% CI, 2.3 to 14.9; P < .001). Among the eltrombopag patients, 12 out of 47 (25.5%) eventually lost the PLT-R, with a cumulative thrombocytopenia relapse-free survival at 60 months of 63.6% (95% CI, 46.0 to 81.2). Clinically significant bleeding (defined as a WHO bleeding score ≥ 2) occurred less frequently in the eltrombopag group than in the placebo group (incidence rate ratio, 0.54; 95% CI, 0.38 to 0.75; P = .0002). Although no difference was observed in the frequency of grade 1-2 adverse events (AEs), a higher proportion of eltrombopag patients experienced grade 3-4 AEs (χ2 = 9.5, P = .002). The evolution to acute myeloid leukemia (AML) and/or disease progression occurred in 17% of eltrombopag and placebo patients, with no difference in survival times.
Eltrombopag demonstrated effectiveness and relative safety in patients with low-risk MDS who had severe thrombocytopenia.
Source: ascopubs.org/doi/full/10.1200/JCO.22.02699
