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Novel therapeutic agents provide hope for patients with hard-to-treat blood disorders
Date:12/9/2012

percent CRc rate (3% CR, 3% CRp, and 29% CRi) with a median duration of response of five weeks and median overall survival of 25.6 weeks. Of those patients in both cohorts who did not respond to their last AML therapy, 47 percent of those with FLT3-ITD and 31 percent of those without achieved a CRc with quizartinib. In both cohorts, 34 percent of patients were successfully bridged to a potentially curative allogeneic transplant.

Common adverse effects of treatment with quizartinib (observed in more than 20% of patients) included QT prolongation (26%), a heart complication associated with some medications and managed by reducing dosage, as well as nausea (38%), vomiting (26%), anemia (29%), fever (25%), diarrhea (20%), and fatigue (20%). These results demonstrate that quizartinib can produce a high treatment response rate in a group of very poor-prognosis AML patients with the FLT3-ITD mutation with manageable toxicity.

"Quizartinib is the first and only single-agent drug that has produced a clinical benefit in AML patients with this deadly mutation who have failed previous therapy," said Dr. Levis. "The number of patients bridged to a stem cell transplant was very significant. We plan on using these encouraging results to design and conduct additional randomized trials that will hopefully lead to the approval of quizartinib to make it accessible to those patients who previously had no hope for a cure."

Dr. Levis will present this study in an oral presentation on Monday, December 10, at 4:30 p.m. EST at the Georgia World Congress Center in Room A101, Level 1, Building A.

Combination Therapy Using JAK2 and HSP90 Inhibitors Increased Efficacy in Myelofibrosis in Vivo [Abstract 805]

Researchers have demonstrated that combination therapy with PU-H71 and ruxolitinib increases the durability and effectiveness of a treatment t
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Contact: Andrea Slesinski
aslesinski@hematology.org
614-352-5096
American Society of Hematology
Source:Eurekalert

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