Ahmet Ozen, Voranush Chongsrisawat, Asena Pinar Sefer, Burcu Kolukisa, Jessica J Jalbert, Karoline A Meagher, Taylor Brackin, Hagit Baris Feldman, Safa Baris, Elif Karakoc-Aydiner, Rabia Ergelen, Ivan J Fuss, Heather Moorman, Narissara Suratannon, Kanya Suphapeetiporn, Lorah Perlee, Olivier A Harari, George D Yancopoulos, Michael J Lenardo
BACKGROUND: CD55 deficiency with hyperactivation of complement, angiopathic thrombosis, and protein-losing enteropathy (CHAPLE) is an ultra-rare genetic disorder characterised by intestinal lymphatic damage, lymphangiectasia, and protein-losing enteropathy caused by overactivation of the complement system. We assessed the efficacy and safety of pozelimab, an antibody blocking complement component 5. METHODS: This open-label, single-arm, historically controlled, multicentre phase 2 and 3 study evaluated ten patients with CHAPLE disease...
January 23, 2024: Lancet