title: Emerging drugs for sickle cell anemia.
abstract: BACKGROUND
  The search for effective therapeutic interventions for sickle cell disease (SCD) has been an ongoing endeavor for over 50 years. During this period, only hydroxyurea (HU), which received US FDA approval in February 1998, was identified as an effective therapeutic agent in preventing or ameliorating the frequency of vaso-occlusive crises, acute chest syndrome and the need for blood transfusion. Approximately 25% of patients with sickle cell anemia (SCA), however, do not respond to HU and some patients experiencing serious side effects of this chemotherapeutic agent. Nevertheless, the success of HU opened the sluice gates to identify other effective drug therapies. The objective of this review is to describe the emerging drug therapies for SCA.
  
  
  METHODS
  In this review, we describe the pathophysiology of SCD and provide an in-depth analysis of the current and new pharmacologic therapies in the field. Literature searches involved multiple databases including Medline In-Process & Other Non-Indexed Citations, MEDLINE, Embase, Cochrane Database of Systematic Reviews, and Scopus.
  
  
  CONCLUSIONS
  SCA is a heterogeneous disease that has caused tremendous global morbidity and early mortality. More effective, individualized and inexpensive therapies are needed. New therapies targeting multiple pathways in its complex pathophysiology are under investigation.
authors.affiliation: Bayhealth Cancer Institute, Hematology/Oncology , Dover, DE , USA.
authors.forename: Priya C
authors.initials: PC
authors.lastname: Singh
authors.forename: Samir K
authors.initials: SK
authors.lastname: Ballas
mesh_terms: D000755:Anemia, Sickle Cell / Q000188:drug therapy* / Q000503:physiopathology; D000818:Animals; D000986:Antisickling Agents / Q000009:adverse effects / Q000627:therapeutic use*; D001803:Blood Transfusion; D015195:Drug Design; D006801:Humans; D006918:Hydroxyurea / Q000009:adverse effects / Q000627:therapeutic use; D058990:Molecular Targeted Therapy
chemical_list: D000986:Antisickling Agents; D006918:Hydroxyurea
keywords: clinical trials; emerging drugs; hydroxyurea; preventative therapy; sickle cell; sickle cell anemia; sickle cell disease; sickle cell therapy; sickle cell treatment