ESID 2020: Sensitivity analysis confirms the efficacy of Emapalumab in treating phage lymphocytosis (HLH).

HLH is a rare disease that causes immune dysfunction in infants and young children.
most patients are unable to identify the primary onset, many patients have primary immunologic diseases.
clinical manifestations may include swollen lymph nodes, enlarged liver and spleen, fever, and neurological abnormalities.
sensitivity analysis of phase II/III key studies (NCT01818492) presented at the 19th meeting of the European Society of Immunodeficiency (ESID), a pharmaceutical company, showed that the total remission rate (ORR) was as high as 63% for HLH patients who did not respond to standard therapies.
despite today's standard care: glucoticoids and etoposides, these patients still have highly unfilled medical needs, whether or not cyclosporine is used.
the use of current standard therapies has not reduced mortality over the past 20 years.
because of the rarity of the disease, no standard method has been established to measure the rate of treatment response.
this critical study used clinical objective mitigation criteria to determine the sensitivity of primary HLH to Emapalumab.
sensitivity analysis supports the use of clinical objective ORR as the primary endpoint in primary HLH studies.
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