TY - JOUR
T1 - Efficacy and safety of ruxolitinib in patients with myelofibrosis and low platelet count (50 x 109/L to < 100 x 109/L) at baseline
T2 - the final analysis of EXPAND
AU - Guglielmelli, Paola
AU - Kiladjian, Jean-Jacques
AU - Vannucchi, Alessandro M.
AU - Duan, Minghui
AU - Meng, Haitao
AU - Pan, Ling
AU - He, Guangsheng
AU - Verstovsek, Srdan
AU - Boyer, Francoise
AU - Barraco, Fiorenza
AU - Niederwieser, Dietger
AU - Pungolino, Ester
AU - Liberati, Anna Marina
AU - Harrison, Claire
AU - Roussou, Pantelia
AU - Wroclawska, Monika
AU - Karumanchi, Divyadeep
AU - Sinclair, Karen
AU - Te Boekhorst, Peter A. W.
AU - Gisslinger, Heinz
N1 - Funding
The authors disclosed receipt of the following
financial support for the research, authorship,
and/or publication of this article: This study was
funded by Novartis Pharmaceuticals.
PY - 2022/9/10
Y1 - 2022/9/10
N2 - Background: Thrombocytopenia is a common feature of myelofibrosis (MF), a myeloproliferative neoplasm driven by dysregulated JAK/STAT signaling; however, pivotal trials assessing the efficacy of ruxolitinib (a JAK1/2 inhibitor) excluded MF patients with low platelet counts (Objectives: Determination of the maximum safe starting dose (MSSD) of ruxolitinib was the primary endpoint, with long-term safety and efficacy as secondary and exploratory endpoints, respectively.Design: EXPAND (NCT01317875) was a phase 1b, open-label, ruxolitinib dose-finding study in patients with MF and low platelet counts (50 toMethods: Patients were stratified according to baseline platelet count into stratum 1 (S1, 75 toResults: Of 69 enrolled patients, 38 received ruxolitinib at the MSSD (S1, n = 20; S2, n = 18) and are the focus of this analysis. The incidence of adverse events was consistent with the known safety profile of ruxolitinib, with thrombocytopenia (S1, 50%; S2, 78%) and anemia (S1, 55%; S2, 44%) the most frequently reported adverse events and no new or unexpected safety signals. Substantial clinical benefits were observed for patients in both strata: 50% (10/20) and 67% (12/18) of patients in S1 and S2, respectively, achieved a spleen response (defined as >= 50% reduction in spleen length from baseline) at any time during the study.Conclusion: The final safety and efficacy results from EXPAND support the use of a 10 mg bid starting dose of ruxolitinib in patients with MF and platelet counts 50 to
AB - Background: Thrombocytopenia is a common feature of myelofibrosis (MF), a myeloproliferative neoplasm driven by dysregulated JAK/STAT signaling; however, pivotal trials assessing the efficacy of ruxolitinib (a JAK1/2 inhibitor) excluded MF patients with low platelet counts (Objectives: Determination of the maximum safe starting dose (MSSD) of ruxolitinib was the primary endpoint, with long-term safety and efficacy as secondary and exploratory endpoints, respectively.Design: EXPAND (NCT01317875) was a phase 1b, open-label, ruxolitinib dose-finding study in patients with MF and low platelet counts (50 toMethods: Patients were stratified according to baseline platelet count into stratum 1 (S1, 75 toResults: Of 69 enrolled patients, 38 received ruxolitinib at the MSSD (S1, n = 20; S2, n = 18) and are the focus of this analysis. The incidence of adverse events was consistent with the known safety profile of ruxolitinib, with thrombocytopenia (S1, 50%; S2, 78%) and anemia (S1, 55%; S2, 44%) the most frequently reported adverse events and no new or unexpected safety signals. Substantial clinical benefits were observed for patients in both strata: 50% (10/20) and 67% (12/18) of patients in S1 and S2, respectively, achieved a spleen response (defined as >= 50% reduction in spleen length from baseline) at any time during the study.Conclusion: The final safety and efficacy results from EXPAND support the use of a 10 mg bid starting dose of ruxolitinib in patients with MF and platelet counts 50 to
U2 - 10.1177/20406207221118429
DO - 10.1177/20406207221118429
M3 - Article
VL - 13
JO - Therapeutic Advances in Hematology
JF - Therapeutic Advances in Hematology
SN - 2040-6207
ER -