Vemurafenib deemed highly effective in hairy-cell leukemia

(HealthDay)—For patients with relapsed or refractory hairy-cell leukemia, a short oral course of vemurafenib is highly effective, according to a study published online Sept. 9 in the New England Journal of Medicine.

Enrico Tiacci, M.D., from the Institute of Perugia in Italy, and colleagues conducted two phase 2, single-group, multicenter studies of oral vemurafenib in Italy (28 patients enrolled) and the United States (26 of 36 planned patients enrolled). Therapy was administered for a median of 16 and 18 weeks, respectively. The primary end points were the complete response rate and overall response rate, respectively.

The researchers found that after a median of eight weeks in the Italian study and 12 weeks in the U.S. study, the overall response rates were 96 and 100 percent, respectively. The rates of complete response in the two trials were 35 and 42 percent, respectively. The median relapse-free survival was 19 months among patients with a complete response and six months among those with a partial response in the Italian trial; median treatment-free survival was 25 and 18 months, respectively. At one year, the progression-free survival rate was 73 percent and overall survival was 91 percent in the U.S. trial.

"A short oral course of vemurafenib was highly effective in patients with relapsed or refractory hairy-cell leukemia," the authors write.

One author disclosed financial ties to Genentech, the manufacturer of vemurafenib.

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