Efficacy and safety of ruxolitinib in patients with myelofibrosis and low platelet count (50 × 10⁹/L to <100 × 10⁹/L) at baseline: the final analysis of EXPAND.

ABSTRACT:

Background

Thrombocytopenia is a common feature of myelofibrosis (MF), a myeloproliferative neoplasm driven by dysregulated JAK/STAT signaling; however, pivotal trials assessing the efficacy of ruxolitinib (a JAK1/2 inhibitor) excluded MF patients with low platelet counts (<100 × 10⁹/L).

Objectives

Determination of the maximum safe starting dose (MSSD) of ruxolitinib was the primary endpoint, with long-term safety and efficacy as secondary and exploratory endpoints, respectively.

Design

EXPAND (NCT01317875) was a phase 1b, open-label, ruxolitinib dose-finding study in patients with MF and low platelet counts (50 to <100 × 10⁹/L).

Methods

Patients were stratified according to baseline platelet count into stratum 1 (S1, 75 to <100 × 10⁹

SUBMITTER: Guglielmelli P 

PROVIDER: S-EPMC9465569 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature