Current and future pharmacologic complement inhibitors

Hematol Oncol Clin North Am. 2015 Jun;29(3):561-82. doi: 10.1016/j.hoc.2015.01.009. Epub 2015 Apr 4.


The availability of anticomplement therapies has been a major achievement for medicine in the last decade. Indeed, eculizumab has changed the treatment paradigm of paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome and promises to do the same in several other human complement-mediated diseases. Nowadays, a 10-year experience has also taught us that there are some pitfalls that represent a challenge to improve the current anticomplement treatment. Most of these observations come from paroxysmal nocturnal hemoglobinuria, where unmet clinical needs are emerging, triggering the attention of several investigators and pharmaceutical companies.

Keywords: C3; C5; Complement therapeutics; Compstatin; Eculizumab; PNH; aHUS.

Publication types

  • Review

MeSH terms

  • Antibodies, Monoclonal, Humanized / metabolism
  • Antibodies, Monoclonal, Humanized / therapeutic use*
  • Atypical Hemolytic Uremic Syndrome / drug therapy*
  • Complement Activation / drug effects
  • Complement C5 / metabolism
  • Complement Inactivating Agents / metabolism
  • Complement Inactivating Agents / therapeutic use*
  • Drug Therapy / methods
  • Drug Therapy / trends
  • Forecasting
  • Hemoglobinuria, Paroxysmal / drug therapy*
  • Humans
  • Protein Binding


  • Antibodies, Monoclonal, Humanized
  • Complement C5
  • Complement Inactivating Agents
  • eculizumab