The first successful expanded compassionate use of Iptacopan in a patient with paroxysmal nocturnal hemoglobinuria-Reference-Cited by-同舟云学术

The first successful expanded compassionate use of Iptacopan in a patient with paroxysmal nocturnal hemoglobinuria

Published:2022-08-15 Issue:11 Volume:101 Page:2537-2539
ISSN:0939-5555
Container-title:Annals of Hematology
language:en
Short-container-title:Ann Hematol

Author:

Han Bing,Ji Jiang,Zhang Bo,Bai Hua,Zhou Daobin,Feng Feng,Huang Yan,Zhu Huijuan,Chen Limeng,Wu Zhihong,Jiang Xiuchun,Li Xuemei,Jia Qing,Chang Qing,Pan Hui,Peng Hua,Zheng Wenting,Huang Hui,Chen Zheng,Yang Chen,Chen Miao,Du Bin,Zhang Shuyang

Funder

Chinese Academy of Medical Sciences (CAMS) innovation fund for medical sciences

National Natural Science Foundation of China

Publisher

Springer Science and Business Media LLC

Subject

Hematology,General Medicine

Link

https://link.springer.com/content/pdf/10.1007/s00277-022-04933-5.pdf

Reference10 articles.

1. Brodsky RA (2014) Paroxysmal nocturnal hemoglobinuria. Blood 124(18):2804–2811

2. Parker C, Omine M, Richards S et al (2005) Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood 106(12):3699–3709

3. Parker CJ (2016) Update on the diagnosis and management of paroxysmal nocturnal hemoglobinuria. Hematology Am Soc Hematol Educ Program 2016(1):208–216

4. Schubart A, Anderson K, Mainolfi N et al (2019) Small-molecule factor B inhibitor for the treatment of complement-mediated diseases. Proc Natl Acad Sci USA 116(16):7926–7931

5. Risitano AM, Röth A, Soret J et al (2021) Addition of iptacopan, an oral factor B inhibitor, to eculizumab in patients with paroxysmal nocturnal haemoglobinuria and active haemolysis: an open-label, single-arm, phase 2, proof-of-concept trial. The Lancet Haematology 8(5):e344–e354

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