December 24, 2023
SAN DIEGO — First-line treatment with ruxolitinib led to “clinically meaningful” positive results versus best available therapy in patients with high-risk polycythemia vera, according to data presented at ASH Annual Meeting.
The therapy had a notable improved impact on symptoms, according to a German team headed by Steffen Koschmieder, MD, head of the clinical hematology laboratory at University of Aachen, Germany.
Ruxolitinib (Jakafi, Incyte) is currently approved in the European Union for patients with hydroxyurea-resistant or intolerant polycythemia vera, but hydroxyurea or ropeginterferon-alpha (Besremi, PharmaEssentia) is currently in practice as the first option.
As ruxolitinib had not been examined against best available therapy in patients with previously untreated polycythemia vera, Koschmieder’s team probed the difference, hypothesizing that ruxolitinib may have higher efficacy.
The randomized phase 2B RuxoBEAT trial — a multicenter, two-arm, open-label trial — had a target population of 190 patients in each arm and a primary endpoint of clinicohematologic complete response rate at 6 months.
By the 6-month mark, the RuxoBEAT data showed that patients in the ruxolitinib arm showed lower hematocrit, pruritus and fatigue, as well as fewer headaches, weight loss and abdominal discomfort.
While the best available therapy and ruxolitinib both displayed reduced platelet counts, white blood cell counts, hematocrit and phlebotomy rates, the best available therapy did not have the same effect on symptoms, and it did not show the impact on spleen size, hemoglobin levels or splenomegaly levels that ruxolitinib had.
Koschmieder noted that the trial is continuing and more patients are being enrolled.
Reference:
Koschmieder S, et al. Firstline treatment with ruxolitinib versus best available therapy in patients with polycythemia vera: Pre-specified interim analysis of the randomized phase 2b Ruxobeat clinical trial of the German study group for myeloproliferative neoplasms. Presented at: ASH Annual Meeting and Exposition; Dec. 9-12, 2023; San Diego.