The efficacy of sequential MMF-rescue-regimen to eradicate inhibitors for refractory severe hemophilia A inhibitor children in China.

Thrombosis research(2023)

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摘要
•Refractoriness was inability to achieve negative inhibitor titer over at least 12 months period taking LD-ITI+Rituximab-IS.•There is therefore a need to explore rescue-regimen(s) for refractory patients.•MMF might exert T-cell immunosuppression not covered by Rituximab which focuses on B-cell suppression.•The sequential MMF-rescue-regimen had efficacy in the patients refractory to our initial LD-ITI+Rituximab-IS regimen.
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