TY - JOUR
T1 - Ruxolitinib in patients with polycythemia vera resistant and/or intolerant to hydroxyurea
T2 - European observational study
AU - Theocharides, Alexandre
AU - Gisslinger, Heinz
AU - De Stefano, Valerio
AU - Accurso, Vincenzo
AU - Iurlo, Alessandra
AU - Devos, Timothy
AU - Egyed, Miklos
AU - Lippert, Eric
AU - Delgado, Regina Garcia
AU - Cantoni, Nathan
AU - Dahm, Anders E.A.
AU - Sotiropoulos, Damianos
AU - Houtsma, Erik
AU - Smyth, Aoife
AU - Iqbal, Amir
AU - Di Matteo, Paola
AU - Zuurman, Mike
AU - te Boekhorst, Peter A.W.
N1 - Publisher Copyright:
© 2023 The Authors. European Journal of Haematology published by John Wiley & Sons Ltd.
PY - 2024/3
Y1 - 2024/3
N2 - Background: Hydroxyurea (HU) is a commonly used first-line treatment in patients with polycythemia vera (PV). However, approximately 15%–24% of PV patients report intolerance and resistance to HU. Methods: This phase IV, European, real-world, observational study assessed the efficacy and safety of ruxolitinib in PV patients who were resistant and/or intolerant to HU, with a 24-month follow-up. The primary objective was to describe the profile and disease burden of PV patients. Results: In the 350 enrolled patients, 70% were >60 years old. Most patients (59.4%) had received ≥1 phlebotomy in the 12 months prior to the first dose of ruxolitinib. Overall, 68.2% of patients achieved hematocrit control with 92.3% patients having hematocrit <45% and 35.4% achieved hematologic remission at month 24. 85.1% of patients had no phlebotomies during the study. Treatment-related adverse events were reported in 54.3% of patients and the most common event was anemia (22.6%). Of the 10 reported deaths, two were suspected to be study drug-related. Conclusion: This study demonstrates that ruxolitinib treatment in PV maintains durable hematocrit control with a decrease in the number of phlebotomies in the majority of patients and was generally well tolerated.
AB - Background: Hydroxyurea (HU) is a commonly used first-line treatment in patients with polycythemia vera (PV). However, approximately 15%–24% of PV patients report intolerance and resistance to HU. Methods: This phase IV, European, real-world, observational study assessed the efficacy and safety of ruxolitinib in PV patients who were resistant and/or intolerant to HU, with a 24-month follow-up. The primary objective was to describe the profile and disease burden of PV patients. Results: In the 350 enrolled patients, 70% were >60 years old. Most patients (59.4%) had received ≥1 phlebotomy in the 12 months prior to the first dose of ruxolitinib. Overall, 68.2% of patients achieved hematocrit control with 92.3% patients having hematocrit <45% and 35.4% achieved hematologic remission at month 24. 85.1% of patients had no phlebotomies during the study. Treatment-related adverse events were reported in 54.3% of patients and the most common event was anemia (22.6%). Of the 10 reported deaths, two were suspected to be study drug-related. Conclusion: This study demonstrates that ruxolitinib treatment in PV maintains durable hematocrit control with a decrease in the number of phlebotomies in the majority of patients and was generally well tolerated.
UR - https://www.scopus.com/pages/publications/85175376428
U2 - 10.1111/ejh.14124
DO - 10.1111/ejh.14124
M3 - Article
C2 - 37899734
AN - SCOPUS:85175376428
SN - 0902-4441
VL - 112
SP - 379
EP - 391
JO - European Journal of Haematology
JF - European Journal of Haematology
IS - 3
ER -