sirolimus has been researched along with Macrophage-Activation-Syndrome* in 1 studies
1 other study(ies) available for sirolimus and Macrophage-Activation-Syndrome
Article | Year |
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Rapamycin as an Adjunctive Therapy for NLRC4 Associated Macrophage Activation Syndrome.
Gain of function (GOF) mutations affecting the inflammasome component NLRC4 are known to cause early-onset macrophage activation syndrome (MAS) and neonatal enterocolitis. Here we report a patient with a NLRC4 GOF mutation presenting with neonatal MAS efficiently treated with a combination of anakinra and rapamycin. Through Topics: Calcium-Binding Proteins; CARD Signaling Adaptor Proteins; Caspase 1; Female; Gain of Function Mutation; Humans; Interleukin-18; Interleukin-1beta; Macrophage Activation Syndrome; Sirolimus | 2018 |