Comparing two treatments in patients with Primary Hemophagocytic Lymphohistiocytosis (HLH)

What is it about?

Primary HLH is a rare, genetic immune-mediated disorder and is fatal if untreated. Conventional treatment (CVT) for newly diagnosed patients comprises off-label use of dexamethasone, etoposide and cyclosporine as per the HLH-94 and HLH-2004 protocol. Emapalumab, a fully human monoclonal antibody that neutralizes IFN-gamma, is approved in the USA for treatment of adult and pediatric patients with primary HLH presenting with refractory, recurrent or progressive disease or intolerance to CVT. In the absence of comparators and head-to-head studies, we conducted a naïve, adjusted treatment comparison of emapalumab versus CVT.

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Why is it important?

Emapalumab may provide an efficacy benefit compared with CVT for the treatment of primary HLH patients who are intolerant or refractory to CVT.