Rare Disease Report

Myeloproliferative Neoplasms

Currently, there is a treatment for patients with intermediate- or high-risk myelofibrosis. They can be prescribed ruxolitinib, and dosing is linked to platelet counts starting at 50,000/Ã
CTI BioPharma and Baxter International announced their phase 3 PERSIST-1 trial met its primary endpoint [patients achieving a 35% or greater reduction in spleen volume when compared with physician-specified best available therapy (BAT)].
The New England Journal of Medicine (NEJM) published results from the pivotal Phase III RESPONSE clinical trial that led to the approval of Jakafi (ruxolitinib) for the treatment of patients with polycythemia vera (PV) who had an inadequate response to or had unacceptable side effects from hydroxyurea.
Jakafi is the first drug approved for this polycythemia vera although the condition in often controlled by phlebotomy and /or off-label use of hydroxyurea.
The American Society of Hematology (ASH) 56th Annual Meeting and Exposition is expected to draw more than 20,000 attendees from around the world.
The US Food and Drug Administration (FDA) issued two new drug approvals for hematologic malignancies just ahead of the 56th ASH Annual Meeting & Exposition.
Jakafi is intended to treat those patients who have an inadequate response to, or cannot tolerate, hydroxyurea.
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