Efficacy and Safety Outcomes With Pacritinib Versus Ruxolitinib in Patients With Myelofibrosis and Thrombocytopenia: Analysis of Data From the Phase III PERSIST-2 Study
Year: 2026
Type: article
Abstract: PURPOSE Pacritinib is a treatment option for patients with myelofibrosis (MF) and cytopenias. Ruxolitinib has been used in patients with cytopenic MF, but often at lower doses associated with suboptimal responses. No prospective head-to-head efficacy data are available. This post hoc analysis of data from the phase III PERSIST-2 study compared outcomes in patients with MF and platelet counts ≤100 × 10 9 /L who received pacritinib 200 mg twice daily versus the subset of patients who received ruxolitinib as best available therapy (BAT). METHODS A post hoc subgroup analysis of efficacy and safety outcomes was performed. The Intention-to-treat Efficacy population included 106 patients (74 randomly assigned to pacritinib and 32 treated with ruxolitinib as BAT). The safety population included 150 patients (106 treated with pacritinib and 44 treated with ruxolitinib). RESULTS Most patients receiving pacritinib maintained full doses over time (total daily dose [TDD] 400 mg), whereas most patients treated with ruxolitinib received low-dose ruxolitinib (TDD ≤ 10 mg). Patients achieved higher rates of spleen volume response (≥35% reduction) with pacritinib versus ruxolitinib (22% v 3%, P = .02 unadjusted; P = .06 adjusted by multivariable regression analysis) and numerically higher rates of symptom response (≥50% reduction in modified Total Symptom Score, excluding tiredness; 35% v 19%, P = .11 unadjusted; P = .13 adjusted), and a greater percentage reported much or very much improved symptoms at week 24 (35% v 16%, P = .06). Pacritinib also demonstrated greater anemia benefit (achievement of transfusion independence) compared with ruxolitinib. The safety profiles of the two therapies were generally comparable, except for gastrointestinal events and infections, which were more frequent with pacritinib. CONCLUSION These results suggest pacritinib may provide improved clinical outcomes compared with low-dose ruxolitinib in patients with cytopenic MF.
Source: JCO oncology advances.
DOI: 10.1200/oa-25-00222
Authors: Claire N. Harrison, Prithviraj Bose, Ruben Mesa, Aaron T. Gerds, Stephen T. Oh, Marta Sobas, Valentín Garcia-Gutierrez, Christof Scheid, Alessandro Maria Vannucchi, Jean-Jacques Kiladjian, Vikas Gupta, Abdulraheem Yacoub, David Lavie, Purvi Suthar, Karisse Roman-Torres, Sarah A. Buckley, John Mascarenhas
Institutions: Guy's and St Thomas' NHS Foundation Trust, The University of Texas MD Anderson Cancer Center, Wake Forest University Health Sciences, Wake Forest University, Cleveland Clinic, Washington University in St. Louis, Nicolaus Copernicus University, Collegium Medicum in Bydgoszcz, Universidad de Alcalá, Instituto Ramón y Cajal de Investigación Sanitaria, Hospital Universitario Ramón y Cajal, University Hospital Cologne, University of Florence, Université Paris Cité, Hôpital Saint-Louis, University Health Network, Princess Margaret Cancer Centre, The University of Kansas Cancer Center, Hadassah Medical Center, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai
Language: English
Subfield: Genetics
Field: Medicine
Domain: Health Sciences
Open Access status: diamond
OpenAlex ID: W7213431555