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[Novel therapeutic agents for hemolytic anemia]

Jun-Ichi Nishimura1

  • 1Department of Hematology and Oncology, Graduate School of Medicine, Osaka University.

[Rinsho Ketsueki] the Japanese Journal of Clinical Hematology
|October 4, 2023
PubMed

In recent years, it has become clear that various diseases are caused by complement (related molecule) abnormalities (complementopathies) or are exacerbated by complement (complement-related diseases), and novel therapeutic agents targeting complement (anti-complement agents) are now being developed. Paroxysmal nocturnal hemoglobinuria (PNH) is a hematopoietic stem cell disorder characterized by complement-mediated intravascular hemolysis due to a deficiency of complement regulatory factors, making it a perfect candidate for anti-complement agents. In 2007, the anti-C5 monoclonal antibody eculizumab was approved for PNH, as the first anti-complement agent. The indications for eculizumab are expanding, and aggressive development is underway for new anti-complement agents, not only for PNH but also a variety of other diseases. In addition, the anti-C1s antibody sutimlimab was approved last year for the treatment of cold agglutinin disease, a form of autoimmune hemolytic anemia. This presentation overviews novel anti-complement agents for these hemolytic anemias.

Keywords:
Autoimmune hemolytic anemiaCold agglutinin diseaseCold agglutinin syndromeParoxysmal nocturnal hemoglobinuria

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