Journal reading

Ruxolitinib is effective and safe in Japanese patients with

hydroxyurea-resistant or hydroxyurea-intolerant polycythemia
Vera with splenomegaly

Keita Kirito, Kenshi Suzuki, Koichi Miyamura, Masahiro Takeuchi, Hiroshi Handa,
Shinichiro Okamoto, et al int J Hematol, September 27 2017

Polycythemia Vera is the One of the chronic myeloproliferative disorders

characterized by clonal proliferation of myeloid cells in which the bone marrow
produces too many red blood cells. Preclinical studies identified activating
mutations in the Janus Kinase 2 (JAK2) gene as the underlying molecular cause for
aberrant hematopoiesis and myeloproliferative disease.
Is a randomized, open-label, multicenter, phase 3 study evaluating the
efficacy and safety of ruxolitinib in patient with PV patients were randomized 1 : 1
to receive ruxolitinib 10 mg twice daily or BAT (best available therapy a total of
18 Japanese patients with 6 randomized to ruxolitinib and 12 randomized to BAT.
Primary analysis occurred after all patients completed week 48. The composited
primary endpoint was the percentage of patients of patients who achieved both
hematocrit control and a spleen response at week 32. Secondary endpoints included
the percentage of patients who achieved CHR (complete hematologic remission) at
week 32 and the percentage of patients who maintained the primary response ay
week 48.
The Japanese cohort had a greater proportion of younger patients (median
age, 57 vs 62 years), patient with hydroxyurea resistant (100 vs 94.5%), and patients
carrying a JAK2V17F mutation (100 vs 94.5%). Group had smaller proportion of
man (50% vs 60%) and patients had a shorter duration (4.2 vs 8.2% years), had
fewer occurrence of prior thromboembolic events (16.7 vs 35.5%) and had smaller
spleen (826 vs 1195 cm3) at baseline higher proportions of Japanese patients had
received ≥ 3 phlebotomies 24 weeks prior to randomization (50% vs 30.9%).
In conclusion, Ruxolitinib was superior to BAT in providing hematocrit
control, reducing splenomegaly, and improved symptoms in Japanese patient with
PV. ruxolitinib may be a potential new treatment option in Japanese patients with
PV who have an inadequate response to or have unacceptable side effect from
hydroxyurea

Will be presented by Noveldy Calzoum Bachry

On Friday, January 19th 2018