
Progression of MDS, AML Evolution, LFS Survival, PFS, and OS

AML, MDS, CFS
PROGRESSION, AML, DISEASE PROGRESSION
Progression of MDS occurred in nine eltrombopag (8%) versus five placebo subjects (9%; χOverall, the combined outcome AML evolution and/or disease progression occurred in 19 of 111 (17%) eltrombopag patients versus nine of 54 (17%) placebo patients (χFive-year cumulative LFS, PFS, combined outcome-free survival (CFS), and OS in the eltrombopag arm were 65.0% (95% CI, 51.7 to 78.3), 61.4% (95% CI, 45.3 to 77.5), 54.0% (95% CI, 39.3 to 68.7), and 77.3% (95% CI, 64.4 to 90.2), respectively, compared with 44.9% (95% CI, 4.3 to 85.5), 44.6% (95% CI, 5.8 to 83.4), 34.5% (95% CI, 2.4 to 66.6), and 58.1% (95% CI, 9.7 to 100.0) in the placebo arm, and did not reach statistical significance. Additional results are available in the Data Supplement ([Figs S4 and S5]). The number needed to treat for a PLT-R of at least 4 weeks' duration was 3.2, and the number needed to harm for grade 3-4 AEs was 4.1. The number needed to observe AML evolution was 62. Thus, the likelihood of being helped or harmed, in terms of serious AEs versus efficacy, is 1.3 and AML evolution versus efficacy is 19.4. Therefore, eltrombopag treatment is 1.3-19.4 times more likely to help in terms of PLT-R than to harm.