Efficacy and safety of ruxolitinib in patients with newly-diagnosed polycythemia vera: futility analysis of the RuxoBEAT clinical trial of the GSG-MPN study group

TY - JOUR

T1 - Efficacy and safety of ruxolitinib in patients with newly-diagnosed polycythemia vera: futility analysis of the RuxoBEAT clinical trial of the GSG-MPN study group

AU - Koschmieder, Steffen

AU - Isfort, Susanne

AU - Wolf, Dominik

AU - Heidel, Florian H

AU - Hochhaus, Andreas

AU - Schafhausen, Philippe

AU - Griesshammer, Martin

AU - Wolleschak, Denise

AU - Platzbecker, Uwe

AU - Döhner, Konstanze

AU - Jost, Philipp J

AU - Parmentier, Stefani

AU - Schaich, Markus

AU - von Bubnoff, Nikolas

AU - Stegelmann, Frank

AU - Maurer, Angela

AU - Crysandt, Martina

AU - Gezer, Deniz

AU - Kortmann, Maike

AU - Franklin, Jeremy

AU - Frank, Julia

AU - Hellmich, Martin

AU - Brümmendorf, Tim H

AU - German Study Group for Myeloproliferative Neoplasms (GSG-MPN)

N1 - © 2022. The Author(s).

PY - 2023/2

Y1 - 2023/2

N2 - Patients (pts) with polycythemia vera (PV) suffer from pruritus, night sweats, and other symptoms, as well as from thromboembolic complications and progression to post-PV myelofibrosis. Ruxolitinib (RUX) is approved for second-line therapy in high-risk PV pts with hydroxyurea intolerance or resistance. The RuxoBEAT trial (NCT02577926, registered on October 1, 2015, at clinicaltrials.gov) is a multicenter, open-label, two-arm phase-IIb trial with a target population of 380 pts with PV or ET, randomized to receive RUX or best available therapy. This pre-specified futility analysis assesses the early clinical benefit and tolerability of RUX in previously untreated PV pts (6-week cytoreduction was allowed). Twenty-eight patients were randomly assigned to receive RUX. Compared to baseline, after 6 months of treatment, there was a significant reduction of median hematocrit (46 to 41%), the median number of phlebotomies per year (4.0 to 0), and median patient-reported pruritus scores (2 to 1), and a trend for reduced night sweat scores (1.5 to 0). JAK2V617F allele burden, as part of the scientific research program, also significantly decreased. One hundred nine adverse events (AEs) occurred in 24/28 patients (all grade 1 to 3), and no pt permanently discontinued treatment because of AEs. Thus, treatment with ruxolitinib in untreated PV pts is feasible, well-tolerated, and efficient regarding the above-mentioned endpoints.

AB - Patients (pts) with polycythemia vera (PV) suffer from pruritus, night sweats, and other symptoms, as well as from thromboembolic complications and progression to post-PV myelofibrosis. Ruxolitinib (RUX) is approved for second-line therapy in high-risk PV pts with hydroxyurea intolerance or resistance. The RuxoBEAT trial (NCT02577926, registered on October 1, 2015, at clinicaltrials.gov) is a multicenter, open-label, two-arm phase-IIb trial with a target population of 380 pts with PV or ET, randomized to receive RUX or best available therapy. This pre-specified futility analysis assesses the early clinical benefit and tolerability of RUX in previously untreated PV pts (6-week cytoreduction was allowed). Twenty-eight patients were randomly assigned to receive RUX. Compared to baseline, after 6 months of treatment, there was a significant reduction of median hematocrit (46 to 41%), the median number of phlebotomies per year (4.0 to 0), and median patient-reported pruritus scores (2 to 1), and a trend for reduced night sweat scores (1.5 to 0). JAK2V617F allele burden, as part of the scientific research program, also significantly decreased. One hundred nine adverse events (AEs) occurred in 24/28 patients (all grade 1 to 3), and no pt permanently discontinued treatment because of AEs. Thus, treatment with ruxolitinib in untreated PV pts is feasible, well-tolerated, and efficient regarding the above-mentioned endpoints.

U2 - 10.1007/s00277-022-05080-7

DO - 10.1007/s00277-022-05080-7

M3 - SCORING: Journal article

C2 - 36564535

VL - 102

SP - 349

EP - 358

JO - ANN HEMATOL

JF - ANN HEMATOL

SN - 0939-5555

IS - 2

ER -