TRANSFORM-1: A randomized, double-blind, placebo-controlled, multicenter, international phase 3 study of navitoclax in combination with ruxolitinib versus ruxolitinib plus placebo in patients with untreated myelofibrosis

Data from the first randomized trial in JAK inhibitor–naïve intermediate-2 or high-risk myelofibrosis with navitoclax (NAV) plus ruxolitinib (RUX)—presented during the 2023 American Society of Hematology Annual Meeting—showed that the rate of spleen volume reduction of ≥35% at Week 24 was twice as high in the NAV + RUX group (63.2%) as the group receiving placebo plus RUX (31.5.%; P<0.0001). These responses were durable with median duration of response in the NAV + RUX group not reached compared with 19.4 months in patients on placebo plus RUX (95% CI: 16.8, NR). Hematologic adverse events were common but manageable with dose modifications.