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Case series reveals durable restoration of physiological anticoagulant function in pediatric severe protein C deficiency after liver transplantation, indicating long-term curative potential.
Severe congenital protein C (PC) deficiency is a rare inherited coagulation disorder that can be difficult to control with conventional medical treatment alone. We report a single-center case series of three pediatric patients with severe congenital PC deficiency who underwent liver transplantation (LT). In all patients, plasma PC activity levels exceeded 64% after LT and remained stable without anticoagulation treatment or PC supplementation. Two patients were followed for more than 10 years, representing the longest reported outcomes to date. Stable anticoagulant function persisted despite rejection, graft dysfunction, and subsequent surgery, demonstrating durable restoration of physiological anticoagulant function after LT.
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Sakaya et al. (2026) studied this question.
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