Table 2 COMFORT-I & II trials on ruxolitinib

Design

1:1 randomization, vs. placebo

2:1 randomization, vs. best available therapy

Number of patients

155 (ruxolitib) vs. 154 (placebo)

146 (ruxolitinib) vs. 73 (BAT)

Dose of ruxolitinib

15-20 mg twice daily based on platelet count

15-20 mg twice daily based on platelet count

Primary end point

reduction in spleen size > 35% at 24 week

reduction of spleen size > 35% at 48 week

Results

35% reduction in spleen size

42% vs. 0.7% at 24 week

28% vs. 0% at 48 week

Duration of spleen response *

67% patient maintained the spleen response for 48 weeks

80% maintained the response at 12 month follow up

Major Adverse events:

Anemia (45%), Thrombocytopenia (13%), headache, pyrexia

Anemia, Diarrhea, Arthralgia, fatigue, abdominal pain

Discontinuation Rate

11% vs. 10.6%

8% vs. 5%