Emapalumab in children with primary hemophagocytic lymphohistiocytosis
New England Journal of Medicine May 11, 2020
Locatelli F, Jordan MB, Allen C, et al. - In an open-label, single-group, phase 2–3 study including individuals who had received conventional therapy before enrollment (previously treated patients) and previously untreated patients who were 18 years of age or younger and had primary hemophagocytic lymphohistiocytosis, researchers tested the safety and effectiveness of emapalumab (a human anti–interferon-γ antibody) administered with dexamethasone. A total of 34 patients (27 previously treated patients and 7 previously untreated patients) had received emapalumab at the cutoff date of July 20, 2017; 26 individuals finished the analysis. For patients with primary hemophagocytic lymphohistiocytosis, emapalumab was an efficacious targeted therapy.
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