Canakinumab treatment in hyperimmunoglobulinemia D with periodic fever syndrome

Objective. To evaluate the efficacy and safety of canakinumab treatment in active hyperimmunoglobulinemia D with periodic fever syndrome (HIDS). Methods. This is a 3-part open-label study with a 6-month treatment period (P1; 300 mg or 4 mg/kg q6w) a 6-month withdrawal period (P2) and a 24-month treatment period (P3). The primary endpoint was reduction in frequency of flares during treatment period compared with the historical period (HP; period in which patients did not receive drugs other than NSAIDs and/or steroids). 63 samples total. 7 at visit 1: anakinra treatment (V1_AK) 9 at visit 2: during active disease after anakinra withdrawal (V2_NT) 9 at visit 3: after anakinra withdrawal and on canakinumab (V3) 8 at visit 4: after anakinra withdrawal and on canakinumab (V4) 8 at visit 5: after anakinra withdrawal and on canakinumab (V5) 15 age- and sex-matched healthy controls (HC). Note: V2_NT is BL in the associated manuscript. 7 at visit 6: during active disease after canakinumab withdrawal (ATTACK)