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Toxin-Based Drug Moxetumomab Pasudotox May Be New Option for Rare Leukemia


A peripheral blood smear showing several hairy leukemia cells.

Credit: Case Rep Pathol July 2014. doi: 10.1155/2014/497027. CC BY 3.0.

People diagnosed with hairy cell leukemia (HCL), an uncommon form of leukemia, may soon have an effective new treatment option, according to findings from an international phase 3 clinical trial. In the trial, three-quarters of people with HCL that had come back or progressed after earlier treatment responded to treatment with the toxin-based drug moxetumomab pasudotox (Moxe).

The disease disappeared completely during treatment in 33 of the 80 patients (41%), and more than 70% of these patients remained cancer free after 6 months of follow-up. The trial results were presented June 2 at the American Society of Clinical Oncology (ASCO) annual meeting in Chicago.

"This drug offers an option for patients with relapsed or refractory hairy cell leukemia that avoids additional chemotherapy and also has the potential to provide better long-term outcomes," said Robert Kreitman, M.D., of NCI’s Center for Cancer Research (CCR), who led the trial.



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