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When would you use proximal complement inhibitors like pegcetacoplan over terminal complement inhibitors for initial treatment of paroxysmal nocturnal hemoglobinuria?

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What is your preferred first line treatment for PNH?

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1 Answer
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Hematology · Dana-Farber Cancer Institute
Answered on

I use either iptacopan or pegcetacoplan as primary therapy for PNH these days. Since its release, I have used mainly iptacopan. Its advantages are 1) it is oral, 2) the side effect profile is very tolerable, 3) it only inhibits the alternative pathway, thus is likely to be associated with less risk ...

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