Abstract
Primary hemophagocytic lymphohistiocytosis (pHLH) is an immune-mediated, hyperinflammatory disorder. Interferon-γ (IFNγ) plays a key role in the pathophysiology of pHLH. Emapalumab, a fully human, anti-IFNγ monoclonal antibody neutralizes both free and receptor-bound IFNγ. However, inhibiting IFNγ-mediated signaling could result in immune dysfunction and immunosuppression. This exploratory exposure-safety analysis investigated the relationship between emapalumab and the incidence of adverse events in patients with pHLH. Increased exposure to emapalumab was not associated with an increased predicted risk of severe adverse events, infection, or infusion-related reactions. Emapalumab was associated with a favorable and manageable safety profile across all assessed doses and treatment durations.
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