Diamond-Blackfan anemia responding to valproic acid

study of 4 adults with sickle cell disease treated with valproic acid reported a 3-fold increase in HbF in 3 patients over 2 to 13 weeks. 6 In another study, valproic acid was as effective as hydroxyurea in increasing the concentration of HbF in patients with sickle cell disease. 7 Furthermore, in patients with sickle cell disease there was a modest increase in both mean HbF concentration and the number of cells containing increased HbF after valproic acid treatment. 8 The mechanism of induction of fetal hemoglobin synthesis by valproate is possibly through modulation of the mitogen-activated protein kinase (MAPK) signal transduction system. 9 In our patient, initiation of valproic acid therapy was temporally associated with an improvement in the hemoglobin concentration, without an increase in HbF. A search of the literature did not reveal any previous use of valproic acid in the treatment of DBA. We recommend considering the use of valproic acid in treating DBA if there is no response to other therapies. Further studies are needed to confirm the potential benefits of this drug in DBA.