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Ruxolitinib versus best available therapy for polycythemia vera intolerant or resistant to hydroxycarbamide in a randomized trial

Harrison, Claire N., Nangalia, Jyoti, Boucher, Rebecca, Jackson, Aimee, Yap, Christina, O'Sullivan, Jennifer, Fox, Sonia, Ailts, Isaak, Dueck, Amylou C., Geyer, Holly L., Mesa, Ruben A., Dunn, William G., Nadezhdin, Eugene, Curto-Garcia, Natalia, Green, Anna, Wilkins, Bridget, Coppell, Jason, Laurie, John, Garg, Mamta, Ewing, Joanne, Knapper, Steven ORCID:, Crowe, Josephine, Chen, Frederick, Koutsavlis, Ioannis, Godfrey, Anna, Arami, Siamak, Drummond, Mark, Byrne, Jennifer, Clark, Fiona, Mead-Harvey, Carolyn, Baxter, Elizabeth Joanna, McMullin, Mary Frances and Mead, Adam J. 2023. Ruxolitinib versus best available therapy for polycythemia vera intolerant or resistant to hydroxycarbamide in a randomized trial. Journal of Clinical Oncology 41 (19) , pp. 3534-3544. 10.1200/JCO.22.01935

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PURPOSE Polycythemia vera (PV) is characterized by JAK/STAT activation, thrombotic/hemorrhagic events, systemic symptoms, and disease transformation. In high-risk PV, ruxolitinib controls blood counts and improves symptoms. PATIENTS AND METHODS MAJIC-PV is a randomized phase II trial of ruxolitinib versus best available therapy (BAT) in patients resistant/intolerant to hydroxycarbamide (HC-INT/RES). Primary outcome was complete response (CR) within 1 year. Secondary outcomes included duration of response, event-free survival (EFS), symptom, and molecular response. RESULTS One hundred eighty patients were randomly assigned. CR was achieved in 40 (43%) patients on ruxolitinib versus 23 (26%) on BAT (odds ratio, 2.12; 90% CI, 1.25 to 3.60; P = .02). Duration of CR was superior for ruxolitinib (hazard ratio [HR], 0.38; 95% CI, 0.24 to 0.61; P < .001). Symptom responses were better with ruxolitinib and durable. EFS (major thrombosis, hemorrhage, transformation, and death) was superior for patients attaining CR within 1 year (HR, 0.41; 95% CI, 0.21 to 0.78; P = .01); and those on ruxolitinib (HR, 0.58; 95% CI, 0.35 to 0.94; P = .03). Serial analysis of JAK2V617F variant allele fraction revealed molecular response was more frequent with ruxolitinib and was associated with improved outcomes (progression-free survival [PFS] P = .001, EFS P = .001, overall survival P = .01) and clearance of JAK2V617F stem/progenitor cells. ASXL1 mutations predicted for adverse EFS (HR, 3.02; 95% CI, 1.47 to 6.17; P = .003). The safety profile of ruxolitinib was as previously reported. CONCLUSION The MAJIC-PV study demonstrates ruxolitinib treatment benefits HC-INT/RES PV patients with superior CR, and EFS as well as molecular response; importantly also demonstrating for the first time, to our knowledge, that molecular response is linked to EFS, PFS, and OS.

Item Type: Article
Date Type: Publication
Status: Published
Schools: Medicine
Publisher: American Society of Clinical Oncology
ISSN: 0732-183X
Date of First Compliant Deposit: 15 May 2023
Date of Acceptance: 1 May 2023
Last Modified: 11 Jul 2023 16:15

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