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.

Autor: Foltz L; St. Paul's Hospital, University of British Columbia, Vancouver, Canada., Pica GM; Service of Hematology, Centre Hospitalier Métropole Savoie, Chambery, France., Zerazhi H; Clinical Hematology, Avignon Hospital, Avignon, France., Van Droogenbroeck J; Department of Hematology, AZ St. Jan AV, Brugge, Belgium., Visanica S; Service Hématologie, Hôpital Belle Isle, HP Metz, France., Báez de la Fuente E; OCA Hospital, Monterrey, México., Leber B; Division of Hematology and Thromboembolism, Department of Medicine, McMaster University, Hamilton, Canada., de Almeida AM; Hospital da Luz, Lisbon, Portugal., Ranta D; Centre Hospitalier Universitaire, Nancy, France., Kiladjian JJ; Centre d'Investigations Cliniques, AP-HP, Hôpital Saint-Louis, Université Paris Diderot, INSERM CIC 1427, Paris, France., Chrit L; Novartis AG, Basel, Switzerland., Kandra A; Novartis AG, Basel, Switzerland., Morando J; Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA., Devos T; Department of Hematology, University Hospitals Leuven, Leuven, Belgium.; Laboratory of Experimental Transplantation, Department of Microbiology and Immunology, KU Leuven, Leuven, Belgium.
Jazyk: angličtina
Zdroj: Leukemia & lymphoma [Leuk Lymphoma] 2019 Dec; Vol. 60 (14), pp. 3493-3502. Date of Electronic Publication: 2019 Jul 30.
DOI: 10.1080/10428194.2019.1636985
Abstrakt: 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.
Databáze: MEDLINE
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