Targeted Drug Can ‘Diminish the Suffering’ of Myelofibrosis

Mayo Clinic: Use of the targeted agent pacritinib significantly reduced the symptoms and burden of advanced myelofibrosis in patients, says a Mayo Clinic researcher who co-led PERSIST-1, the worldwide phase 3 clinical trial that tested the therapy. Specifically, pacritinib substantially reduced severe enlargement of the spleen, a typical feature of advanced myelofibrosis, in more than 20 percent of patients and alleviated debilitating side effects in more than 46 percent. Investigators further found that pacritinib could be used safely in patients with myelofibrosis who have thrombocytopenia, a life-threating loss of blood platelets that can lead to deadly bleeding. The only currently approved therapy for myelofibrosis — ruxolitinib — is not recommended in patients who have severe thrombocytopenia.