Summary

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• Emapalumab-lzsg (Gamifant) is indicated for the treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent, or progressive disease or intolerance to conventional HLH therapy.
• Two studies from systematic reviews/meta-analyses were reviewed.
• The rate of partial response (PR) with emapalumab ranged from 14.2% to 100%, while the complete response (CR) varied significantly based on the medication used. Most patients could proceed to hematopoietic stem cell transplantation (HSCT), achieving a survival rate ranging from 55.9% to 100%.
• In phase II/III studies, emapalumab was effective in treating primary HLH pediatric patients with refractory or relapsing disease, allowing most patients to proceed successfully to HSCT.
• Emapalumab has demonstrated effectiveness primarily in pediatric populations but also includes mixed-age groups encompassing both children and adults; specific data for only adult populations are limited.
• Safety profiles indicate that emapalumab is relatively well tolerated, with few mild side effects reported across studies, similar in safety profile comparisons made alongside ruxolitinib and alemtuzumab.