Studies on the economic impact of this study on the social communities of developing countries. In the national project entitled: TREATMENT OF BETA-THALASSEMIA PATIENTS WITH RAPAMYCIN (SIROLIMUS): FROM PRE-CLINICAL RESEARCH TO A CLINICAL TRIAL

Objectives. The overall aim of the project is a reduction of transfusions in beta-thalassemia patients, through a pharmacologically mediated increased levels of HbF. There is strong evidence that Sirolimus is able to induce HbF production in vitro in erythroid precursor cells (ErPCs) from beta-thalassemia and sickle-cell patients and in vivo in transgenic mice. Therefore, the THALA-RAP objective is to verify whether HbF increases occur in beta-thalassemia patients treated with Sirolimus in peripheral blood and isolated ErPCs. A second list of parameters is the lifespan and the oxidative stress status of the beta-thalassemia red blood cells. Both outcomes support the possible clinical relevance of in vivo effects of Sirolimus.Thus, a cost-effectiveness analysis of the patients’treatment toward the conventional one is conducted.