TY - JOUR AU - Matte, Alessandro AU - Mazzi, Filippo AU - Federti, Enrica AU - Olivieri, Oliviero AU - De Franceschi, Lucia PY - 2019/01/01 Y2 - 2024/03/19 TI - NEW THERAPEUTIC OPTIONS FOR THE TREATMENT OF SICKLE CELL DISEASE: Guest Editor: Raffaella Origa JF - Mediterranean Journal of Hematology and Infectious Diseases JA - Mediterr J Hematol Infect Dis VL - 11 IS - 1 SE - Review Articles DO - 10.4084/mjhid.2019.002 UR - https://mjhid.org/mjhid/article/view/2019.002 SP - e2019002 AB - <p>Sickle cell disease (SCD; ORPHA232; OMIM # 603903) is a chronic and invalidating disorder distributed worldwide, with high morbidity and mortality.&nbsp; Given the disease complexity and the multiplicity of pathophysiological targets, development of new therapeutic options is critical, despite the positive effects of hydroxyurea (HU), for many years the only approved drug for SCD.</p><p>New therapeutic strategies might be divided into (1) pathophysiology-related novel therapies and (2) innovations in curative therapeutic options such as hematopoietic stem cell transplantation and gene therapy. The pathophysiology related novel therapies are: a) Agents which reduce sickling or prevent sickle red cell dehydration; b) Agents targeting SCD vasculopathy and sickle cell-endothelial adhesive events; c) Anti-oxidant agents.</p><p>This review highlights new therapeutic strategies in SCD and discusses future developments, research implications, and possible innovative clinical trials.</p><p>&nbsp;</p> ER -