Current landscape of paroxysmal nocturnal hemoglobinuria in the era of complement inhibitors and regulators

Julia J. Shi, Yusuf M. Ozcan, Carlos I.Ayala Santos, Hetalkumari Patel, Jamile Shammo, Taha Bat*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

Abstract

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder which is caused by mutations in phosphatidylinositol glycan class A leading to hemolysis of red blood cells via complement inhibition. The first treatment for PNH, eculizumab, was FDA approved in 2007. Since then, many new treatment options for PNH have arisen. This critical review will examine all medications available for PNH on the US market, highlight several major medications in development, and discuss the risks and treatment considerations associated with each option. It is not intended to address PNH clonal dynamics, disease presentation, or discussions on when to initiate treatment.

Original languageEnglish (US)
JournalTherapeutic Advances in Hematology
Volume15
DOIs
StatePublished - Jan 1 2024

Keywords

  • complement cascade
  • complement inhibitors
  • PNH

ASJC Scopus subject areas

  • Hematology

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