TY - JOUR
T1 - 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
AU - Foltz, Lynda
AU - Pica, Gian Matteo
AU - Zerazhi, Hacene
AU - Van Droogenbroeck, Jan
AU - Visanica, Sorin
AU - Báez de la Fuente, Enrique
AU - Leber, Brian
AU - Almeida, António Medina de
AU - Ranta, Dana
AU - Kiladjian, Jean Jacques
AU - Chrit, Linda
AU - Kandra, Albert
AU - Morando, Juliane
AU - Devos, Timothy
N1 - Funding Information:
L. F. reports consultancy and honoraria from Novartis, and research funding from Gilead, Promedior, Novartis, and Incyte. G. M. P., H. Z., J. V. D., S. V., and E. B. F. have no relevant financial relationships to disclose. B. L. reports consultancy, honoraria, and membership on an entity’s board of directors or advisory committees from Novartis Canada. A. M. A. participated in speaker’s bureau for Novartis and Celgene. D. R. reports consultancy from Novartis. J. J. K. reports membership on an entity’s board of directors or advisory committees from Celgene, Novartis and AOP Orphan, and research funding from Novartis and AOP Orphan. L. C., A. K., and J. M. are employees of Novartis and own stocks of company. T. D. reports consultancy from Novartis, Celgene, and Takeda.
Publisher Copyright:
© 2019, © 2019 Informa UK Limited, trading as Taylor & Francis Group.
PY - 2019/12/6
Y1 - 2019/12/6
N2 - 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.
AB - 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.
KW - Myeloproliferative neoplasms
KW - Polycythemia vera
KW - Ruxolitinib
UR - http://www.scopus.com/inward/record.url?scp=85077097704&partnerID=8YFLogxK
U2 - 10.1080/10428194.2019.1636985
DO - 10.1080/10428194.2019.1636985
M3 - Article
C2 - 31359808
AN - SCOPUS:85077097704
SN - 1042-8194
VL - 60
SP - 3493
EP - 3502
JO - Leukemia and Lymphoma
JF - Leukemia and Lymphoma
IS - 14
ER -
