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The use of the complement inhibitor eculizumab (Soliris(R)) for treating Korean patients with paroxysmal nocturnal hemoglobinuria

Authors
 Jin Seok Kim  ;  Jong Wook Lee  ;  Byoung Kook Kim  ;  Je-Hwan Lee  ;  Jooseop Chung 
Citation
 Korean Journal of Hematology, Vol.45(4) : 269-274, 2010 
Journal Title
 Korean Journal of Hematology 
ISSN
 1738-7949 
Issue Date
2010
Keywords
Eculizumab ; Efficacy ; Paroxysmal nocturnal hemoglobinuria ; Safety
Abstract
BACKGROUND: Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal disorder characterized by chronic complement-mediated hemolysis. Eculizumab, a humanized monoclonal antibody against the terminal complement protein C5, potently reduces chronic intravascular hemolysis. We tested the clinical efficacy and safety of a 24-week treatment with eculizumab in 6 Korean patients with PNH.0aMETHODS: We enrolled 6 patients with PNH who had clinically significant hemolysis. Eculizumab was administered intravenously at 600 mg/week for the first 4 weeks followed by 900 mg at week 5 and 2nd weekly thereafter.0aRESULTS: Three men and 3 women with a median age of 39.5 years (24-61 years) were enrolled. The median duration of PNH was 11 years (6-25 years). Hemolysis occurred in all patients [median lactate dehydrogenase (LDH) level, 7.95 times the upper limit of the reference range of LDH]. All patients treated with eculizumab had a rapid and sustained reduction in the degree of hemolysis. RBC transfusion requirements for 3 months were decreased from 0-12 units (median requirement, 1.5 units) to 0-6 units (median requirement, 0 units). Improvement in fatigue was noted in 4 patients. Further, 5 patients who had been receiving corticosteroids either reduced the dose or discontinued therapy. No significant adverse events related to eculizumab therapy were observed.0aCONCLUSION: These results show that eculizumab reduces the degree of intravascular hemolysis, reduces or eliminates the requirement of RBC transfusion, and improves anemia and fatigue in patients with PNH. Eculizumab is an effective and safe option for treating Korean patients with PNH
Files in This Item:
T201004889.pdf Download
DOI
10.5045/kjh.2010.45.4.269
Appears in Collections:
1. College of Medicine (의과대학) > Dept. of Internal Medicine (내과학교실) > 1. Journal Papers
Yonsei Authors
Kim, Jin Seok(김진석) ORCID logo https://orcid.org/0000-0001-8986-8436
URI
https://ir.ymlib.yonsei.ac.kr/handle/22282913/102770
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