Significantly more patients receiving Jakafi achieved
the composite primary* and key secondary end points 1,2†
Components of Primary End Point at Week 32 1
80
Composite
Primary End Point
60%
60
40
Individual Components
of Primary End Point
(n = 66)
20
0
(n = 25)
(n = 44)
19%
<1% b
Hct Control + Spleen
Volume Reduction
<1%
(n = 21)
(n = 1)
BAT (n = 112)
40%
(P < 0.0001)
23% a
Jakafi (n = 110)
(n = 1)
≥35% Spleen
Volume Reduction
Hct Control
Without Phlebotomy
BAT, best available therapy;
CI, confidence interval; Hct, hematocrit.
a
95% CI, 15%-32%
b
95% CI, 0%-5%
* The composite primary end point was defined as hematocrit (Hct) control without phlebotomy and a ≥35% spleen volume reduction as measured by CT or MRI. To achieve the Hct
control end point, patients could not become eligible for phlebotomy between weeks 8 and 32. Phlebotomy eligibility was defined as Hct >45% that is ≥3 percentage points higher
than baseline or Hct >48% (lower value). 1,2
†
The RESPONSE (Randomized Study of Efficacy and Safety in Polycythemia Vera with JAK Inhibitor Ruxolitinib versus Best Available Care) trial was a randomized, open-label, active-controlled phase
3 trial comparing Jakafi with best available therapy in 222 patients with polycythemia vera. Patients enrolled in the study were resistant to or intolerant of hydroxyurea, required phlebotomy for Hct
control, and had splenomegaly. All patients entered into a Hct control period, during which time Hct levels were maintained between 40% and 45% for 28 days before patients were randomized to
Jakafi or best available therapy. Best available therapy included hydroxyurea (60%), interferon/pegylated interferon (12%), anagrelide (7%), pipobroman (2%), lenalidomide/thalidomide (5%), and
observation (15%). Patients had been diagnosed with polycythemia vera for at least 24 weeks, had an inadequate response to or were intolerant of hydroxyurea, required phlebotomy, and exhibited
splenomegaly. After week 32, patients were able to cross over to Jakafi treatment. An updated analysis was performed at week 80 only in patients originally randomized to Jakafi.
Patients Achieving Complete Hematologic Remission 1a
Durable response at week 80 1
Jakafi (n = 110)
BAT (n = 112)
60
(P = 0.0016)
40
20
24% b
(n = 26)
8% c
(n = 9)
0
Complete Hematologic Remission at Week 32
19 of 25 patients (76%) who achieved a primary response at week 32
in the Jakafi arm maintained their response
51 of 66 patients (77%) who achieved Hct control at week 32 in the
Jakafi arm maintained their response
43 of 44 patients (98%) who achieved a ≥35% spleen volume reduction
at week 32 in the Jakafi arm maintained their response
15 of 26 patients (58%) who achieved complete hematologic remission
at week 32 in the Jakafi arm maintained their response
BAT, best available t