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Gamifant® (emapalumab-lzsg) was evaluated in a multicenter study1
The safety and efficacy of Gamifant was evaluated in a multicenter, open-label, single-arm study.
Safety
evaluated in 34 patients
Efficacy
evaluated in 27 patients
- were treatment-naïve prior to receiving Gamifant
- received Gamifant after conventional treatments attempted
Study population1
Patients had refractory, recurrent, or progressive disease or were intolerant of conventional therapy.
A genetic diagnosis of primary HLH was confirmed in 82% of patients.
The most frequent causative mutations were FHL3-UNC13D (MUNC 13-4) (26%), FHL2-PRF1 (19%), and Griscelli Syndrome type 2 (19%).
The median age of patients in the study was 1 year.
Patients ranged in age from 0.1 year to 13 years old.
Patients had received a median of 3 prior agents before enrollment.
Prior regimens included combinations of dexamethasone, etoposide, cyclosporine A, and antithymocyte globulin.
Primary endpoint1
The primary endpoint was overall response rate (ORR) at end of treatment. ORR was defined as achievement of either complete or partial response or HLH improvement and evaluated using an algorithm of objective clinical and laboratory parameters.
Complete response
defined as normalization of all HLH abnormalities (i.e., no fever, no splenomegaly, neutrophils > 1x109/L, platelets > 100x109/L, ferritin < 2000 μg/L, fibrinogen > 1.50 g/L, D-dimer < 500 μg/L, normal CNS symptoms, no worsening of soluble CD25* > 2-fold baseline)
*Soluble CD25 is also referred to as soluble interleukin-2 receptor
Partial response
defined as normalization of
≥ 3 HLH abnormalities
HLH improvement
defined as ≥ 3 HLH abnormalities improved by at least 50% from baseline
*Soluble CD25 is also referred to as soluble interleukin-2 receptor