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The safety and efficacy of Gamifant was evaluated in a multicenter, open-label, single-arm study.
evaluated in 34 patients
evaluated in 27 patients
Patients had refractory, recurrent, or progressive disease or were intolerant of conventional therapy.
The most frequent causative mutations were FHL3-UNC13D (MUNC 13-4) (26%), FHL2-PRF1 (19%), and Griscelli Syndrome type 2 (19%).
Patients ranged in age from 0.1 year to 13 years old.
Prior regimens included combinations of dexamethasone, etoposide, cyclosporine A, and antithymocyte globulin.
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.
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
defined as normalization of
≥ 3 HLH abnormalities
defined as ≥ 3 HLH abnormalities improved by at least 50% from baseline
*Soluble CD25 is also referred to as soluble interleukin-2 receptor
