Rare Disease Report

Myeloproliferative Neoplasms

Ruben Mesa, MD of the Mayo Clinic in Scottsdale Arizona talks about two approaches to treating polycythemia vera, and essential thrombocythemia that use interferons.
Ruben Mesa, MD of the Mayo Clinic in Scottsdale, AZ talks about the data he presented at ASH showing that half of the patients with myeloproliferative neoplasms (MPNs), including myelofibrosis, polycythemia vera, and essential thrombocythemia had to change their employment status (eg, leaving job, medical disability leave, early retirement) due to their rare disease.
Ruben Mesa, MD of the Mayo Clinic in Scottsdale, AZ talks about the data he presented at ASH showing that half of the patients with myeloproliferative neoplasms (MPNs), including myelofibrosis, polycythemia vera, and essential thrombocythemia had to change their employment status (eg, leaving job, medical disability leave, early retirement) due to their rare disease.
The Leukemia & Lymphoma Society (LLS) announced it has committed $40.3 million to 75 new research grants
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.
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