Treatment Options for Sickle Cell Disease

Pediatr Clin North Am. 2018 Jun;65(3):427-443. doi: 10.1016/j.pcl.2018.01.005.


Sickle cell disease (SCD) complications begin with the polymerization of sickle hemoglobin (HbS). Thus, SCD therapies are focused on preventing HbS production or reducing the circulating amount of HbS. Hydroxyurea treatment has become more widespread, whereas the number of evidence-based indications for erythrocyte transfusion is small. Hematopoietic stem cell transplant is a curative option for SCD but less than 25% of patients have a suitable donor. This article focuses on supportive and preventive care improvements and the benefits of hydroxyurea. Indications for erythrocyte transfusion, hematopoietic stem cell transplant, and gene therapy trials are also summarized.

Keywords: Fetal hemoglobin; Hematopoietic stem cell transplant; Hydroxyurea; Sickle cell disease; Transfusion.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Anemia, Sickle Cell / complications
  • Anemia, Sickle Cell / mortality
  • Anemia, Sickle Cell / therapy*
  • Antisickling Agents / therapeutic use
  • Erythrocyte Transfusion
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Hydroxyurea / therapeutic use


  • Antisickling Agents
  • Hydroxyurea