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

Enrico Tiacci, MD, 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%, respectively. The rates of complete response in the two trials were 35 and 42%, 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% and overall survival was 91% 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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