An Adaptive Design Study To Evaluate The Efficacy, Safety And Pharmacokinetics Of Emapalumab In Adult Patients With Non-Primary Hemophagocytic Lymphohistiocytosis

Background: Secondary hemophagocytic lymphohistiocytosis (sHLH) is a rare, life-threatening syndrome which may develop on the background of several clinical conditions (e.g., autoimmune disease, infection or malignancy) and is characterized by uncontrolled hyperinflammation. Preclinical data have shown the central role of interferon-gamma (IFNγ) in the pathogenesis of this disease (Strippoli et al. 2012; Prencipe et al. 2018), and observational studies confirm the presence of an IFNγ signature in sHLH (data on file, Buatois et al. 2017, Bracaglia et al. 2017, Akashi et al. 1994, Henter et al. 1991; Ohga et al. 1993; Maruoka et al. 2014).