Treatment of Primary Myelofibrosis with Valproic acid

Abstract

Primary myelofibrosis is a group of hematologic malignancies with genetic, chromosomal and epigenetic abnormalities causing myeloproliferation and abnormal cytokine expression. The marrow fibrosis (collagen or reticulin) and extramedullary hematopoiesis results in anemia, thrombocytopenia, splenomegaly, bone marrow failure and may turn to acute myeloid leukemia. The median survival rate is short. Treatment options depend on patients and their economic status including medications, supportive blood component transfusion and symptomatic treatment. This report uses valproic acid acting as a histone deacetylase inhibitor in a 62-year-old female patient. She showed improved complete blood count parameters (CBC), decreased blood transfusion, splenomegaly, bone marrow fibrosis and marrow failure symptoms. After stopping valproic treatment for 6, 7 and 8 years, the hematocrit remained within normal limit. Other laboratories assays such as liver function test, renal function test, fasting blood sugar and lipid levels were also normal. The survival was longer than that predicted by the IPSS score.