| Budget Amount *help |
¥4,810,000 (Direct Cost: ¥3,700,000、Indirect Cost: ¥1,110,000)
Fiscal Year 2016: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2015: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2014: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000)
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| 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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