2016 Fiscal Year Final Research Report
Investigation of eculizumab therapeutic response to patients with PNH
| Project/Area Number |
26461447
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Multi-year Fund |
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| Section | 一般 |
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| Research Field |
Hematology
|
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| Research Institution | Osaka University |
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Principal Investigator |
|
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| Project Period (FY) |
2014-04-01 – 2017-03-31
|
| Keywords | 発作性夜間ヘモグロビン尿症 / 溶血 / エクリズマブ / 補体 / 遺伝子多型 |
|---|
| Outline of Final Research Achievements |
Approximately 550 patients in Japan with PNH were treated with eculizumab and about 3.5% of patients were found to be refractory by C5 gene polymorphism due to heterozygous base substitution (c.2654G>A, p.Arg885His). Similar polymorphisms were identified in Argentinian patient (c.2653C>T, p.Arg885Cys) and Dutch patient (c.2653C>A, p.Arg885Ser). Functional analysis proved that eculizumab could not bind to C5 due to these polymorphisms and hemolysis could not be suppressed. Thus, we have constructed and operated a system for analysis of eculizumab inadequacy.
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| Free Research Field |
血液内科学
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