Written in Blood
Ruxolitinib Versus Best Available Therapy for Essential Thrombocythemia
Treatment with the JAK1/2 inhibitor ruxoli-
tinib failed to improve response rates, com-
pared with best-available therapy (BAT) in
patients with essential thrombocythemia
(ET) who were intolerant or resistant to
standard frontline treatment with low-dose
aspirin and hydroxycarbamide, according
to results from a randomized phase II trial
published in Blood. However, ruxolitinib
improved certain disease-related symp-
toms, including pruritus, concentration,
and ability to perform daily activities, noted
Claire N. Harrison, MD, from Guy’s and
St. Thomas’ Hospital in London, United
Kingdom, and co-authors.
The open-label, randomized, con-
trolled MAJIC-ET trial included 116 adult
patients from 31 U.K. centers (median age
= 64.2 years; range = 34.5-90.5 years) with
high-risk ET that was resistant to treat-
ment with hydroxycarbamide. Six patients
were excluded from the final analysis: four
withdrew without treatment (2 refused to
be in the BAT arm, 1 became ineligible,
and 1 had transformed to post-ET myelo-
fibrosis), and two did not start treatment
within one year of randomization.
The remaining 110 patients were
randomized to receive either:
• ruxolitinib at a starting dose of
25 mg twice-daily (or 20 mg
twice-daily if baseline platelets
were 100-200×10 9 /L; n=58)
• BAT chosen by the physician (n=52)