Tesidolumab (LFG316) for treatment of C5-variant patients with paroxysmal nocturnal hemoglobinuria

• Authors: NISHIMURA Jun-ichi; ANDO Kiyoshi; MASUKO Masayoshi; NOJI Hideyoshi; ITO Yoshikazu; MAYER Jiří; GRISKEVICIUS Laimonas; BUCHER Christoph; MULLERSHAUSEN Florian; GERGELY Peter; ROZENBERG Izabela; SCHUBART Anna; CHAWLA Raghav; RONDEAU Jean-Michel; ROGUSKA Michael; SPLAWSKI Igor; KEATING Mark T; JOHNSON Leslie; DANEKULA Rambabu; BAGGER Morten; WATANABE Yoko; HARALDSSON Borje; KANAKURA Yuzuru
• Year of publication: 2022
• Type: Article in Periodical
• Magazine / Source: Haematologica
• MU Faculty or unit: Faculty of Medicine
• Web: https://haematologica.org/article/view/haematol.2020.265868
• Doi: http://dx.doi.org/10.3324/haematol.2020.265868
• Keywords: Tesidolumab; paroxysmal nocturnal hemoglobinuria; C5-variant patients
• Description: Paroxysmal nocturnal hemoglobinuria (PNH) is caused by clonal expansion of hematopoietic stem cells that carry a somatic mutation in the X-linked gene PIG-A (phosphatidylinositol glycan anchor biosynthesis class A). The mutation leads to a deficiency of glycosylphosphatidylinositol-anchored membrane proteins. The loss of membrane-associated complement regulatory proteins CD55 and CD59 increases susceptibility of red blood cells and platelets to complement-mediated lysis, leading to hemolytic anemia, thrombophilia and reduced life expectancy in untreated patients.