A short oral course of vemurafenib was highly effective in patients with relapsed or refractory hairy-cell leukemia, according to 2 studies involving 50 patients with hairy-cell leukemia who relapsed after treatment or were refractory to purine analogue. Researchers found:

• Rates of complete response were 35% and 42% in the 2 trials.

• In one trial, median relapse-free survival was 19 months among patients with a complete response and 6 months in those with a partial response; similarly, median treatment-free survival was 25 months and 18 months in those groups.

• In the other trial, progression-free survival rate was 73% and overall survival was 91%.

• Drug-related AEs were usually grade 1 or 2.

• Secondary cutaneous tumors developed in 7 of 50 patients.

Citation: Tiacci E, Park JH, De Carolis L, et al. Targeting mutant BRAF in relapsed or refractory hairy-cell leukemia. [Published online ahead of print September 9, 2015]. N Engl J Med. doi: 10.1056/NEJMoa1506583.