Therapy with the histone deacetylase inhibitor pracinostat for patients with myelofibrosis
Leukemia Research, 07/27/2012
Clinical Article
Quintas–Cardama A et al. – Pracinostat at the dose tested is reasonably tolerated and has modest activity in patients with myelofibrosis (MF).
Methods- The authors report on a phase II study testing the activity and tolerability of the histone deacetylase inhibitor pracinostat given at 60mg every other day for three weeks per month in 22 patients with intermediate or high risk MF.
- Eight (36%) patients experienced clinical benefit, with 6 (27%) experiencing reductions in splenomegaly (median 3cm, range 1–4cm).
- According to International Working Group criteria, 2 (9%) patients had clinical improvement (anemia response in both cases).
- The most frequent side effect associated to pracinostat therapy was fatigue, which occurred in 20 (91%) patients (grade 2 in 3 patients).
- Grade 3–4 neutropenia, anemia, and thrombocytopenia occurred in 13%, 0%, and 21%, respectively.
- Twenty-one patients permanently discontinued pracinostat, mainly due to lack of efficacy.



