Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria-Reference-Cited by-同舟云学术

Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria

Published:2024-03-14 Issue:11 Volume:390 Page:994-1008
ISSN:0028-4793
Container-title:New England Journal of Medicine
language:en
Short-container-title:N Engl J Med

Author:

Peffault de Latour Régis¹,Röth Alexander¹,Kulasekararaj Austin G.¹,Han Bing¹,Scheinberg Phillip¹,Maciejewski Jaroslaw P.¹,Ueda Yasutaka¹,de Castro Carlos M.¹,Di Bona Eros¹,Fu Rong¹,Zhang Li¹,Griffin Morag¹,Langemeijer Saskia M.C.¹,Panse Jens¹,Schrezenmeier Hubert¹,Barcellini Wilma¹,Mauad Vitor A.Q.¹,Schafhausen Philippe¹,Tavitian Suzanne¹,Beggiato Eloise¹,Chew Lee Ping¹,Gaya Anna¹,Huang Wei-Han¹,Jang Jun Ho¹,Kitawaki Toshio¹,Kutlar Abdullah¹,Notaro Rosario¹^ORCID,Pullarkat Vinod¹,Schubert Jörg¹,Terriou Louis¹,Uchiyama Michihiro¹,Wong Lee Lee Lily¹,Yap Eng-Soo¹,Sicre de Fontbrune Flore¹,Marano Luana¹,Alashkar Ferras¹,Gandhi Shreyans¹,Trikha Roochi¹,Yang Chen¹,Liu Hui¹,Kelly Richard J.¹,Höchsmann Britta¹,Kerloeguen Cécile¹,Banerjee Partha¹,Levitch Rafael¹,Kumar Rakesh¹,Wang Zhixin¹,Thorburn Christine¹,Maitra Samopriyo¹,Li Shujie¹,Verles Aurelie¹,Dahlke Marion¹,Risitano Antonio M.¹

Affiliation:

1. From the Hematology and Transplant Unit, French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Saint-Louis Hospital (R.P.L., F.S.F.), and Université de Paris (R.P.L.), Paris, Centre Hospitalier Universitaire (CHU) de Toulouse, Institut Universitaire du Cancer de Toulouse–Oncopole, Toulouse (S.T.), and CHU Lille, Université de Lille, Lille (L.T.) — all in France; the Department of Hematology and Stem Cell Transplantation, West German Cancer Center, University Hospital...

Funder

Novartis Pharmaceuticals Corporation

Publisher

Massachusetts Medical Society

Link

http://www.nejm.org/doi/pdf/10.1056/NEJMoa2308695

Reference39 articles.

1. Luzzatto L, De Franceschi L. Hemolytic anemias. In: Loscalzo J, Fauci AS, Kasper DL, Hauser SL, Longo DL, Jameson JL, eds. Harrison’s principles of internal medicine. 21st ed. New York: McGraw Hill, 2022:776-790.

2. Paroxysmal nocturnal haemoglobinuria (PNH) is caused by somatic mutations in the PIG-A gene.

3. Affected erythrocytes of patients with paroxysmal nocturnal hemoglobinuria are deficient in the complement regulatory protein, decay accelerating factor.

4. Isolation and characterization of a membrane protein from normal human erythrocytes that inhibits reactive lysis of the erythrocytes of paroxysmal nocturnal hemoglobinuria.

5. STUDIES ON DESTRUCTION OF RED BLOOD CELLS. II. CHRONIC HEMOLYTIC ANEMIA WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA: CERTAIN IMMUNOLOGICAL ASPECTS OF THE HEMOLYTIC MECHANISM WITH SPECIAL REFERENCE TO SERUM COMPLEMENT 1

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