AB1496 LONG-TERM EFFICACY AND SAFETY OF CANAKINUMAB IN PATIENTS WITH HIDS (HYPER-IGD SYNDROME) − INTERIM ANALYSIS OF THE RELIANCE REGISTRY

Background Hyper-IgD Syndrome/Mevalonate Kinase Deficiency (HIDS/MKD) is a rare autoinflammatory disease caused by defect in the gene encoding mevalonate kinase. Treatment with interleukin-1β inhibitor canakinumab (CAN, approved since 2017 for HIDS/MKD) resulted rapid remission most patients, both controlled trials and clinical practice. Objectives The present study investigates long-term efficacy safety of CAN under routine conditions pediatric (age ≥2 years) adult patients HIDS/MKD. Methods RELIANCE prospective, non-interventional, multi-center, observational based Germany. Patients clinically confirmed HIDS/MKD diagnosis who routinely receive are enrolled study. Disease remission, activity, fatigue, days absent from school/work due to indication, inflammatory markers assessed at baseline 6-monthly intervals. Results interim analysis includes (N=8) as well preliminary 18-month (N=6) 30-month data (N=5) December 2022. Of these 63% were female median age was 8 years (2−39 years). duration prior treatment entry 1.5 (0−5 majority has been treated greater than standard dose (5/8 baseline, 4/6 month 18, 3/3 30). Preliminary results indicate stable control physicians according laboratory parameters ( table 1 ). Even though outcomes HIDS cohort on small patient numbers, resemble larger cohorts within registry (CAPS, TRAPS, FMF). Serious adverse events occurred N=2 but none considered drug related. Conclusion suggest good participating In addition, 18 30 months revealed no unexpected concerns. Further end-of-study will be analyzed assess course Table 1. Baseline characteristics Number N 6 5 (consecutive number #1-8) (physician assessment) 4 (#1,3,5,6) (#2,5,6,7) (#1,2,3,7) Patient´s assessment current activity; 0–10, (min; max) 0.0 (0; 7) 4) 1) fatigue; 2.5 1.0 2) (%*) without impairment social life 3 (50) 2 work/school during last (25) (17) (40) Current influence mood; %* negative | positive 0 33 67 100 50 CRP, (mg/dl) 0.2 0.1 SAA, 0.6 0.4 1.7 ESR, (mm/h) 10.0 10.5 6.0 SAE Incidence rate ‡ per patient-years All types 8.95 SADR 0.00 c-reactive protein; erythrocyte sedimentation rate; serum amyloid A; SADR, serious reaction; SAE, event *not reported all = * 36525/ sum observation (=8159) REFERENCES: NIL. Acknowledgements: Disclosure Interests Prasad Oommen Grant/research support from: Novartis, Tilmann Kallinich Speakers bureau: Roche, Jürgen Rech AbbVie, Biogen, BMS, Chugai, GSK, Janssen, Lilly, MSD; Mylan, Sanofi, Sobi, UCB, Consultant of: MSD, Norbert Blank Pfizer, Abbvie, Actelion, Boehringer-Ingelheim, Julia Weber-Arden Employee J. B. Kuemmerle-Deschner Sobi.