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Background: Autoinflammatory periodic fever syndromes characterized by excessive interleukin(IL)-1b release and severe systemic and organ inflammation have been successfully treated with the anti-IL-1 inhibitor canakinumab (CAN). In clinical trial situations and real life, rapid remission of symptoms and normalization of laboratory parameters were observed in most patients. 1-3
Objectives: The present study explores long-term effectiveness and safety of CAN under routine clinical practice conditions in pediatric and adult patients with CAPS (cryopyrin-associated periodic syndromes), FMF (familial Mediterranean fever), TRAPS (tumor necrosis factor receptor-associated periodic syndrome) and HIDS/MKD (hyperimmunoglobulinemia D syndrome/mevalonate kinase deficiency).
Methods: RELIANCE is a prospective, non-interventional, multi-center, observational study based in Germany with a 3-year follow-up period. Pediatric (age ≥2 years) and adult patients with clinically confirmed diagnoses of CAPS, FMF, TRAPS and HIDS/MKD that routinely receive CAN are enrolled in order to evaluate effectiveness and safety of CAN in clinical routine.
Results: This first interim analysis of patient subgroups diagnosed with FMF, HIDS and TRAPS includes baseline data of 41 patients (10 TRAPS, 2 HIDS, 29 FMF), including preliminary 6-month data of a FMF-subset (N=16).
Evaluation of disease activity and fatigue by patients’ assessment, days absent from school/work, inflammatory markers and physician global assessment (PGA) was performed at baseline and will be further assessed at 6-monthly intervals within the 3-year observation period. Preliminary results of a first subset of 16 patients diagnosed with FMF are available and indicate stable remission and disease control upon long-term CAN treatment: within the first study interval, no major changes were observed regarding the analyzed parameters (
Baseline characteristics of the TRAPS/HIDS/FMF-subgroups and preliminary 6-month data of FMF subset.
| TRAPS | HIDS | FMF | |||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Baseline | Baseline | Baseline
| Baseline
| 6 months
|
|||||||||||
| Number of patients, N | 10 | 2 | 29 | 16 | 16 | ||||||||||
| Mean age, years (SD) | 22 (4; 43) | 11 (5; 18) | 26 (5; 56) | 16 (5; 47) | 16 (5; 47) | ||||||||||
| Female (%), N=9 (1 missing) | 5 (56) | 1 (50) | 15 (52) | 7 (44) | 7 (44) | ||||||||||
| Mean duration of prior canakinumab treatment, years (min; max) | 1 (0; 2) | 3 (2; 4) | n. a. | 2.2 (0; 6) | 2.2 (0; 6) | ||||||||||
| Patient’s assessment of disease activity 0-10, mean (min; max) | 2.1 (0; 5) | 0 (0; 0) | 3 (0; 10) | 2.8 (0; 8) | 2.2 (0; 7) | ||||||||||
| Patient’s assessment of fatigue 0-10 | 3.4 (0; 8) | 0 (0; 0) | 4.4 (0; 9) | 4.6 (0; 9) | 3.9 (0; 8) | ||||||||||
| Number (%) of patients with days absent from school/work due to study indication during last 6 months | 4 (44) | 2 (100) | 5 (17) | 2 (13) | 5 (31) | ||||||||||
| Inflammatory markers, CRP/SAA, mean (mg/dL) | 2.0 | 7.9 | 0.1 | 0.6 | 0.9 | 5.3 | 0.5 | 2.4 | 0.6 | 2.4 | |||||
| PGA of disease activity: no/mild to moderate/severe; N (%)* | 1 (11) | 6 (67) | 0 (0) | 2 (100) | 0 (0) | 0 (0) | 10 (35) | 8 (28) | 2 (7) | 5 (31) | 7 (44) | 1 (6) | 6 (38) | 7 (44) | 0 (0) |
| SAE, N (%) | 0 (0) | 0 (0) | 2 (6.9) | n. a. | 2 (12.5) | ||||||||||
Conclusion: Baseline characteristics of all subgroups and first interim data of FMF patients are available from the RELIANCE study, the longest running real-life CAN registry. Further interval data will be analyzed to assess efficacy and safety of long-term CAN-treatment in patients with autoinflammatory periodic fever syndromes.
REFERENCES:
[1]Lachmann et al. N Engl J Med. 2009;360(23):2416-25
[2]Kuemmerle-Deschner et al. Rheumatology (Oxford). 2016;55(4):689-96
[3]De Benedetti et al. N Engl J Med. 2018;378(20):1908-1919
Disclosure of Interests: Jörg Henes Grant/research support from: Novartis, Roche-Chugai, Consultant of: Novartis, Roche, Celgene, Pfizer, Abbvie, Sanofi, Boehringer-Ingelheim,, J. B. Kuemmerle-Deschner Grant/research support from: Novartis, AbbVie, Sobi, Consultant of: Novartis, AbbVie, Sobi, Michael Borte Grant/research support from: Pfizer, Shire, Ivan Foeldvari Consultant of: Novartis, Gerd Horneff Grant/research support from: AbbVie, Chugai, Merck Sharp & Dohme, Novartis, Pfizer, Roche, Speakers bureau: AbbVie, Bayer, Chugai, Merck Sharp & Dohme, Novartis, Pfizer, Roche, Tilmann Kallinich Grant/research support from: Novartis, Consultant of: Sobi, Roche, Novartis, Birgit Kortus-Goetze Consultant of: Novartis, Frank Weller-Heinemann: None declared, Julia Weber-Arden Employee of: I am employed by Novartis, Norbert Blank Grant/research support from: Novartis, Sobi, Consultant of: Novartis, Sobi, Lilly, Pfizer, Abbvie, BMS, MSD, Actelion, UCB, Boehringer-Ingelheim, Roche