Complement in sickle cell disease and targeted therapy: I know one thing, that I know nothing

Blood Rev. 2021 Jul:48:100805. doi: 10.1016/j.blre.2021.100805. Epub 2021 Jan 21.

Abstract

Sickle cell disease (SCD) is a common inherited clinical syndrome, characterized by the presence of hemoglobin S. Anemia, susceptibility to infections and episodes of vaso-occlusive crisis (VOC) are among its features. Since SCD complications (VOC or delayed hemolytic transfusion reaction/DHTR) lead to significant morbidity and mortality, a number of studies have addressed their pathophysiology Although SCD pathophysiology has been mainly attributed to the interaction between sickle cells and neutrophils, platelets or endothelial cells in small vessels leading to hemolysis, the role of complement activation has been increasingly investigated. Importantly, complement inhibition with eculizumab has shown beneficial effects in DHTR. Given the unmet clinical need of novel therapeutics in SCD, our review summarizes current understanding of (a) complement system for the clinician, (b) complement activation in SCD both in asymptomatic state and severe clinical manifestations, (c) probable underlying mechanisms of complement activation in SCD, and (d) new therapeutic perspective of complement inhibition.

Keywords: Complement inhibition; Complement system; Eculizumab; Sickle cell disease.

Publication types

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

MeSH terms

  • Anemia, Sickle Cell / drug therapy*
  • Anemia, Sickle Cell / etiology*
  • Antibodies, Monoclonal, Humanized / administration & dosage
  • Antibodies, Monoclonal, Humanized / adverse effects
  • Antibodies, Monoclonal, Humanized / therapeutic use
  • Complement Activation / drug effects
  • Complement Activation / immunology
  • Complement Inactivating Agents / administration & dosage
  • Complement Inactivating Agents / adverse effects
  • Complement Inactivating Agents / therapeutic use
  • Complement System Proteins / drug effects*
  • Complement System Proteins / immunology*
  • Disease Susceptibility
  • Humans
  • Molecular Targeted Therapy* / methods
  • Transfusion Reaction / immunology
  • Treatment Outcome

Substances

  • Antibodies, Monoclonal, Humanized
  • Complement Inactivating Agents
  • Complement System Proteins
  • eculizumab