Key clinical point: Emapalumab, an interferon gamma-blocking antibody, controls disease activity and has a favorable safety profile in patients with primary hemophagocytic lymphohistiocytosis.
Major finding: The overall response rate was 64.7% for all 34 treated patients (95% CI, 46%-80%; P = .0031), and 63% for the 27 patients who had failed prior therapy (95% CI, 42%-81%; P = .0134).
Study details: In the open-label, single-arm, pivotal study, 34 children with primary HLH were treated: 7 who were treatment naive and 27 who had failed conventional HLH therapy.
Disclosures: The study described by Dr. Locatelli was sponsored by Novimmune. Study authors provided disclosures related to Sobi, Novimmune, Rocket Pharmaceuticals, AB2Bio, Novartis, Eli Lilly, Sanofi, UCB, Pfizer, and Abbvie. Two authors reported employment with Novimmune.
Locatelli F et al. ASH 2018; Abstract LBA-6.