Why the study?
Does edoxaban prevent purpura fulminans and bleeding in a pediatric patient with severe congenital protein C deficiency?
Does edoxaban prevent purpura fulminans and bleeding in a pediatric patient with severe congenital protein C deficiency?
Edoxaban may be a promising and safe alternative to warfarin for managing severe congenital protein C deficiency in pediatric patients.
Should not yet change practice in pediatric protein C deficiency; leaves open edoxaban's role in prospective trials.
A male patient diagnosed with severe congenital protein C (PC) deficiency during the neonatal period was treated with long-term warfarin but frequently developed purpura fulminans and bleeding. At four years of age, edoxaban was initiated (direct oral anticoagulant [DOAC]). His d-dimer and fibrin/fibrinogen degradation product levels were closely monitored. His PC activity increased from below the sensitivity range to 17%; this increase was thought to be due to a reduction in PC consumption during edoxaban therapy. After edoxaban introduction, he experienced just one episode of purpura fulminans over two years without any adverse events. Thus, DOAC may be a promising alternative for the management of congenital PC deficiency.
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Watanabe et al. (2019) studied this question.
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