Thalassemia Reports 2019-04-23T17:26:47+02:00 Paola Granata Open Journal Systems <p><strong>Thalassemia Reports</strong> is the premier peer-reviewed international medical journal devoted entirely to the study, diagnosis, and treatment of thalassemia. The journal covers the pathophysiology, diagnosis, classification, and treatment of thalassemia and related disorders for physicians and medical scientists. The primary intent of the journal will be to publish clinically relevant information that will directly improve the care of patients with thalassemia. Basic science studies of interest to those treating disorders will also be considered for publication when relevant. A special section of the journal is dedicated to Case Reports: we would like to invite clinicians and researchers to send us their reports.</p> Efficacy of ruxolitinib as inducer of fetal hemoglobin in primary erythroid cultures from sickle cell and beta-thalassemia patients 2019-04-23T17:26:47+02:00 Alice Pecoraro Antonio Troia Aurelio Maggio Rosalba Di Marzo <p>High levels of HbF may ameliorate the clinical course of β-thalassaemia and SCD. Hydroxyurea (HU) is the only HbF inducer approved for the treatment of patients. However not all patients respond to the treatment, for this reason it is noteworthy to identify new HbF inducers. Ruxolitinib is a JAK inhibitor that decreases the phosphorilation of STAT proteins. In particular STAT3 is a repressor of gamma-globin gene. The decrease of STAT3 phosphorilation could derepress gamma-globin gene and reactivate its trascription. In this study we evaluated the efficacy of ruxolitinib as inducer of HbF production. The analyses were performed in cultured erythroid progenitors from 16 beta-thalassemia intermedia (TI) and 4 sickle cell disease (SCD) patients. The use of quantitative RT-PCR technique allowed us to determine the increase of gamma-globin mRNA expression in human erythroid cultured cells treated with ruxolitinib. The results of our study demonstrated an increase <em>in vitro</em> of gamma-globin mRNA expression in almost all patients. These data suggest that ruxolitinib could be a good candidate to be used <em>in vivo</em> for the treatment of hemoglobinopathies.</p> 2019-04-19T10:08:23+02:00 ##submission.copyrightStatement##