Go to JCI Insight
  • About
  • Editors
  • Consulting Editors
  • For authors
  • Publication ethics
  • Alerts
  • Advertising
  • Job board
  • Subscribe
  • Contact
  • Current issue
  • Past issues
  • By specialty
    • COVID-19
    • Cardiology
    • Gastroenterology
    • Immunology
    • Metabolism
    • Nephrology
    • Neuroscience
    • Oncology
    • Pulmonology
    • Vascular biology
    • All ...
  • Videos
    • Conversations with Giants in Medicine
    • Author's Takes
  • Reviews
    • View all reviews ...
    • Lung inflammatory injury and tissue repair (Jul 2023)
    • Immune Environment in Glioblastoma (Feb 2023)
    • Korsmeyer Award 25th Anniversary Collection (Jan 2023)
    • Aging (Jul 2022)
    • Next-Generation Sequencing in Medicine (Jun 2022)
    • New Therapeutic Targets in Cardiovascular Diseases (Mar 2022)
    • Immunometabolism (Jan 2022)
    • View all review series ...
  • Viewpoint
  • Collections
    • In-Press Preview
    • Commentaries
    • Research letters
    • Letters to the editor
    • Editorials
    • Viewpoint
    • Top read articles
  • Clinical Medicine
  • JCI This Month
    • Current issue
    • Past issues

  • Current issue
  • Past issues
  • Specialties
  • Reviews
  • Review series
  • Conversations with Giants in Medicine
  • Author's Takes
  • In-Press Preview
  • Commentaries
  • Research letters
  • Letters to the editor
  • Editorials
  • Viewpoint
  • Top read articles
  • About
  • Editors
  • Consulting Editors
  • For authors
  • Publication ethics
  • Alerts
  • Advertising
  • Job board
  • Subscribe
  • Contact
Complementopathies and precision medicine
Eleni Gavriilaki, Robert A. Brodsky
Eleni Gavriilaki, Robert A. Brodsky
Published April 20, 2020
Citation Information: J Clin Invest. 2020;130(5):2152-2163. https://doi.org/10.1172/JCI136094.
View: Text | PDF
Review

Complementopathies and precision medicine

  • Text
  • PDF
Abstract

The renaissance of complement diagnostics and therapeutics has introduced precision medicine into a widened field of complement-mediated diseases. In particular, complement-mediated diseases (or complementopathies) with ongoing or published clinical trials of complement inhibitors include paroxysmal nocturnal hemoglobinuria, cold agglutinin disease, hemolytic uremic syndrome, nephropathies, HELLP syndrome, transplant-associated thrombotic microangiopathy, antiphospholipid antibody syndrome, myasthenia gravis, and neuromyelitis optica. Recognizing that this field is rapidly expanding, we aim to provide a state-of-the-art review of (a) current understanding of complement biology for the clinician, (b) novel insights into complement with potential applicability to clinical practice, (c) complement in disease across various disciplines (hematology, nephrology, obstetrics, transplantation, rheumatology, and neurology), and (d) the potential future of precision medicine. Better understanding of complement diagnostics and therapeutics will not only facilitate physicians treating patients in clinical practice but also provide the basis for future research toward precision medicine in this field.

Authors

Eleni Gavriilaki, Robert A. Brodsky

×

Figure 1

Targets of complement inhibitors in various stages of clinical development for complement-mediated disorders.

Options: View larger image (or click on image) Download as PowerPoint
Targets of complement inhibitors in various stages of clinical developme...
Complement-targeting compounds are shown in red and indicate the step of the complement pathway they target. From left to right: sutimlimab inhibits C1s of the classical pathway; narsoplimab inhibits mannose-binding protein-associated serine protease 2 (MASP-2) of the lectin pathway; pegcetacoplan (formerly APL-2) and AMY-101 inhibit C3 and C3 convertase activity; IONIS-FB-LRx and LPN023 inhibit factor B; lampalizumab and danicopan inhibit factor D; mini-FH/AMY-201 inhibits alternative pathway C3 convertase; CLG561 inhibits properdin; MicroCept inhibits C3 and C5 convertases; eculizumab, ravulizumab, crovalimab, ABP959, tesidolumab, REGN3918, mubodina, coversin, RA101495, cemdisiran, and zimura inhibit C5; and avacopan inhibits C5a receptor; and IFX-1 inhibits C5a.

Copyright © 2023 American Society for Clinical Investigation
ISSN: 0021-9738 (print), 1558-8238 (online)

Sign up for email alerts