학술논문
Evaluating the efficacy and safety of pozelimab in patients with CD55 deficiency with hyperactivation of complement, angiopathic thrombosis, and protein-losing enteropathy disease: an open-label phase 2 and 3 study
Document Type
Article
Author
Ozen, Ahmet; Chongsrisawat, Voranush; Sefer, Asena Pinar; Kolukisa, Burcu; Jalbert, Jessica J; Meagher, Karoline A; Brackin, Taylor; Feldman, Hagit Baris; Baris, Safa; Karakoc-Aydiner, Elif; Ergelen, Rabia; Fuss, Ivan J; Moorman, Heather; Suratannon, Narissara; Suphapeetiporn, Kanya; Perlee, Lorah; Harari, Olivier A; Yancopoulos, George D; Lenardo, Michael J; Miller, Jutta L.; Eshach Adiv, Orly; Bilgic Eltan, Sevgi; Yorgun Altunbas, Melek; Magliocco, Mary; Matthews, Helen; Marciano, Beatriz E.; Chatchatee, Pantipa; Trbovic, Caryn F.; Burczynski, Michael E.; Chaudhari, Umesh; Usta, Yusuf; Altuntaş, Cansu; Yavuz, Sibel; Baştürk, Ahmet; Demirbaş Ar, Fatma; Topal, Erdem; Kalaycı, Ayhan Gazi; Weerapakorn, Wanlapa; Calabi Martínez, Ana Andrea; Bottero, Adriana
Source
The Lancet; February 2024, Vol. 403 Issue: 10427 p645-656, 12p
Subject
Language
ISSN
01406736; 1474547X
Abstract
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.