A Targeted Therapy for HLH
The report from the pivotal international trial testing emapalumab for the treatment of primary HLH
Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis. Locatelli F, Jordan MB, Allen C, et al N Engl J Med. 2020 May 7;382(19):1811-1822. doi: 10.1056/NEJMoa1911326.
This clinical trial report describes the pivotal international trial of anti-interferon g monoclonal antibody for treating primary HLH. This pivotal study is notable for being the first clinical trial in HLH to study targeted therapies, with objective responses that were systematically reported. This targeted therapy was based on preclinical models of familial HLH, opening a new era of targeted therapies for primary HLH treatment.