Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis

Case series of 34 patients found a response rate of 63% and 65% for emapalumab infusion in those previously treated with other agents, and treatment naïve respectively. This was significantly, more than the prespecified null hypothesis of 40% (p<0.05 for both).


New England Journal of Medicine