Safety and efficacy findings from the open-label, multicenter, phase 3b, expanded treatment protocol study of ruxolitinib for treatment of patients with polycythemia vera who are resistant/intolerant to hydroxyurea and for whom no alternative treatments are available. Issue 14 (6th December 2019)

• Record Type: Journal Article
• Authors: Foltz, Lynda; Pica, Gian-Matteo; Zerazhi, Hacene; Van Droogenbroeck, Jan; Visanica, Sorin; Báez de la Fuente, Enrique; Leber, Brian; de Almeida, Antonio Medina; Ranta, Dana; Kiladjian, Jean-Jacques; Chrit, Linda; Kandra, Albert; Morando, Juliane; Devos, Timothy
• Is Part Of: Leukemia & lymphoma. Volume 60:Issue 14(2019)
• Page Start: 3493
• Page End: 3502
• Publication Date: 2019-12-06
• Subjects: Myeloproliferative neoplasms -- polycythemia vera -- ruxolitinib; Leukemia -- Periodicals; Lymphomas -- Periodicals; 616.99419
• DOI: 10.1080/10428194.2019.1636985
• Languages: English
• ISSNs: 1042-8194

Abstract

Abstract: Ruxolitinib was recently approved for the treatment of patients with polycythemia vera who are resistant/intolerant to hydroxyurea based on data from the RESPONSE studies. This phase 3b, Expanded Treatment Protocol study (NCT02292446) of ruxolitinib for hydroxyurea-resistant/intolerant patients with polycythemia vera ( N = 161: median exposure = 25.1 weeks) further evaluated the safety of ruxolitinib. Adverse events (AEs) led to dose adjustment/interruption in 37.9% of patients and study drug discontinuation in 8.7% of patients. The most common hematologic AEs included anemia and thrombocytosis; while headache and diarrhea were the most frequent nonhematologic AEs. At week 24, 45.3% of patients achieved hematocrit control; hematologic remission was seen in 18% of patients. At least, 50% of reduction in spleen length was achieved in 86.7% of patients from baseline at any time. The observed safety profile of ruxolitinib was consistent and the efficacy results were similar to the observed values in the RESPONSE studies.