Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria

Iptacopan, an oral inhibitor of factor B, increased hemoglobin levels in patients with persistent anemia despite anti-C5 therapy and in patients without previous complement-inhibitor therapy.

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Bibliographische Detailangaben
Veröffentlicht in:	The New England journal of medicine 2024-03, Vol.390 (11), p.994-1008
Hauptverfasser:	Peffault de Latour, Régis, Röth, Alexander, Kulasekararaj, Austin G., Han, Bing, Scheinberg, Phillip, Maciejewski, Jaroslaw P., Ueda, Yasutaka, de Castro, Carlos M., Di Bona, Eros, Fu, Rong, Zhang, Li, Griffin, Morag, Langemeijer, Saskia M.C., Panse, Jens, Schrezenmeier, Hubert, Barcellini, Wilma, Mauad, Vitor A.Q., Schafhausen, Philippe, Tavitian, Suzanne, Beggiato, Eloise, Chew, Lee Ping, Gaya, Anna, Huang, Wei-Han, Jang, Jun Ho, Kitawaki, Toshio, Kutlar, Abdullah, Notaro, Rosario, Pullarkat, Vinod, Schubert, Jörg, Terriou, Louis, Uchiyama, Michihiro, Wong Lee Lee, Lily, Yap, Eng-Soo, Sicre de Fontbrune, Flore, Marano, Luana, Alashkar, Ferras, Gandhi, Shreyans, Trikha, Roochi, Yang, Chen, Liu, Hui, Kelly, Richard J., Höchsmann, Britta, Kerloeguen, Cécile, Banerjee, Partha, Levitch, Rafael, Kumar, Rakesh, Wang, Zhixin, Thorburn, Christine, Maitra, Samopriyo, Li, Shujie, Verles, Aurelie, Dahlke, Marion, Risitano, Antonio M.
Format:	Artikel
Sprache:	eng
Schlagworte:	Administration, Oral Anemia Bilirubin Bone marrow Clinical trials Complement Inactivating Agents Complement inhibitors Erythrocyte Transfusion Hematology Hemoglobin Hemoglobins Hemoglobinuria, Paroxysmal Hemolytic anemia Humans L-Lactate dehydrogenase Life Sciences Oncology Paroxysmal nocturnal hemoglobinuria Patients
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Beschreibung
Zusammenfassung:	Iptacopan, an oral inhibitor of factor B, increased hemoglobin levels in patients with persistent anemia despite anti-C5 therapy and in patients without previous complement-inhibitor therapy.
ISSN:	0028-4793 1533-4406
DOI:	10.1056/NEJMoa2308695