A Scientific Renaissance: Novel Drugs in Sickle Cell Disease

Ahmar U Zaidi; Matthew M Heeney

doi:10.1016/j.pcl.2018.01.006

A Scientific Renaissance: Novel Drugs in Sickle Cell Disease

Pediatr Clin North Am. 2018 Jun;65(3):445-464. doi: 10.1016/j.pcl.2018.01.006.

Authors

Ahmar U Zaidi¹, Matthew M Heeney²

Affiliations

¹ Children's Hospital of Michigan, Wayne State University School of Medicine, Department of Pediatrics, Division of Hematology/Oncology, 3901 Beaubien, Detroit, MI 48201, USA. Electronic address: ahmar@wayne.edu.
² Boston Children's Hospital, Harvard Medical School, 300 Longwood Avenue, Boston, MA 02115, USA.

PMID: 29803276
DOI: 10.1016/j.pcl.2018.01.006

Abstract

We have entered an era of exploding interest in therapeutics for sickle cell disease. The expansion in our understanding of sickle cell disease pathophysiology has enhanced the range of potential therapeutic targets. From induction of fetal hemoglobin to antiadhesion molecules, we are potentially on the cusp of making life-altering modifications for individuals with sickle cell disease. This disease population cannot afford to let the current momentum wane. Studies exploring combinations of therapies affecting multiple steps in the pathophysiology and exploring novel and clinically relevant outcomes are incumbent.

Keywords: Antiadhesion molecules; Induction of fetal hemoglobin; Pathophysiology; Sickle cell disease; Therapeutic targets.

Publication types

Review

MeSH terms

Anemia, Sickle Cell / drug therapy*
Antisickling Agents / therapeutic use*
Drugs, Investigational / therapeutic use*
Fetal Hemoglobin / drug effects
Humans

Substances

Antisickling Agents
Drugs, Investigational
Fetal Hemoglobin