OP0092 LONG-TERM SAFETY AND EFFECTIVENESS OF CANAKINUMAB IN CRYOPYRIN-ASSOCIATED PERIODIC SYNDROMES (CAPS) – 30-MONTH DATA FROM THE RELIANCE REGISTRY
| Autor: | A. Braner, Catharina Schuetz, I. Foeldvari, Prasad T. Oommen, J. B. Kuemmerle-Deschner, Jürgen Rech, G. Horneff, Tilmann Kallinich, A. Janda, B. Kortus-Goetze, Michael Borte, J. Weber-Arden, Frank Weller-Heinemann, Norbert Blank |
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| Rok vydání: | 2021 |
| Předmět: |
Pediatrics
medicine.medical_specialty business.industry Immunology Cryopyrin-associated periodic syndrome medicine.disease Interim analysis General Biochemistry Genetics and Molecular Biology Clinical trial Social life Canakinumab Neonatal onset multisystem inflammatory disease Rheumatology Cohort medicine Immunology and Allergy Long term safety business medicine.drug |
| Zdroj: | Annals of the Rheumatic Diseases. 80:50.2-51 |
| ISSN: | 1468-2060 0003-4967 |
| Popis: | Background:In clinical trials as well as in real-life, the IL-1ß inhibitor canakinumab leads to rapid remission of symptoms in the treatment of CAPS, a monogenic autoinflammatory disease with severe systemic and organ inflammation.Objectives:The RELIANCE registry is designed to explore long-term safety and effectiveness of canakinumab under routine clinical practice conditions in pediatric (≥2 years) and adult patients with CAPS, including Muckle-Wells syndrome (MWS), familial cold autoinflammatory syndrome (FCAS), and neonatal onset multisystem inflammatory disease (NOMID)/chronic infantile neurological cutaneous and articular syndrome (CINCA).Methods:This prospective, non-interventional, observational study with a 3-year follow-up enrolls patients with clinically confirmed diagnoses of CAPS routinely receiving canakinumab. In 6-monthly visits, clinical data, physician assessments and patient-reported outcomes are evaluated starting at baseline with last update at 30 months of follow-up in the total cohort including the cohort with severe CAPS subtypes (NOMID/CINCA).Results:91 CAPS patients (50% female; 14 [15%] NOMID/CINCA subtypes) were enrolled by December 2020 (Table 1). At baseline, median age was 20.5 years and median duration of prior canakinumab treatment was 6 years. 11 drug related severe adverse events per 100 patient years were reported. 68% of patients reached disease remission by physicians´ assessment along with rates of 40-61% absent disease activity in PGA. Patients reported stable low level disease activity, fatigue and Auto-Inflammatory Diseases Activity Index scores (AIDAI, figure 1). CAPS was impairing social life in 50% of patients and another 50% reported days off from school/work. Lab parameters were within normal limits.Table 1.Patient and physician assessment of clinical CAPS disease activity and laboratory markers over time.Baseline12 months30 monthsTotal cohortNOMID/ CINCATotal cohortNOMID/ CINCATotal cohortNOMID/ CINCANumber of patients, N9114678284Number (%) of patients with days absent from work/school during last 6 months30 (34)4 (29)28 (42)2 (25)17 (61)4 (100)Number (%) of patients in disease remission (physician assessment)61 (68.5)11 (78.6)42 (66.7)4 (66.7)19 (67.9)4 (100.0)Physician Global Assessment, percentage of absent/mild-moderate/severe rating, %40 / 53 / 257 / 36 / 033 / 60 / 233 / 50 / 061 / 39 / 075 / 25 / 0Patient assessment of current disease activity; 0–10, median (min; max)2.0 (0; 7)1.0 (0; 6)1.0 (0; 7)1.0 (0; 5)0.0 (0; 7)0.0 (0; 4)Patient assessment of current fatigue; 0–10, median (min; max)3.0 (0; 9)2.0 (0; 6)3.0 (0; 8)2.0 (0; 8)1.0 (0; 8)4.0 (0; 5)Number (%) of patients without impairment of social life by the disease32 (52.5)4 (50.0)31 (62.0)3 (42.9)11 (47.8) 1 (33.3)CRP, median (mg/dl)0.10.20.10.50.00.2SAA, median (mg/dl)0.30.40.50.90.30.1ESR, median (mm/h)5.06.05.03.55.05.5SAENumber of eventsIncidence rate per 100 patient yearsTotal cohortNOMID/CINCATotal cohortNOMID/CINCAAll types of SAE39314.7211.72SADR20#010.690.00# Alport’s syndrome, appendicitis, blister, cardiovascular disorder, chest pain, circulatory collapse, erythema, febrile convulsion, glomerulonephritis, haemophilus test positive, pneumonia, premature delivery, skin discolouration, tonsillectomy, tonsillitis bacterial, tonsillitis streptococcal (all N=1 event), pyrexia (N=3 events), not yet coded (inpatient admission, N=1 event) CRP, c-reactive protein; ESR, erythrocyte sedimentation rate; n. a., not annotated; SAA, serum amyloid A; SADR, serious adverse drug reaction; SAE, serious adverse eventConclusion:The 30-month interim analysis of the RELIANCE study demonstrates that long-term canakinumab treatment is safe and effective in patients with any subtype of CAPS. However, impairment of social life and days off school/work still exists.Disclosure of Interests:J. B. Kuemmerle-Deschner Consultant of: Novartis, AbbVie, Sobi, Grant/research support from: Novartis, AbbVie, Sobi, Birgit Kortus-Goetze Consultant of: Novartis, Prasad Oommen Grant/research support from: Novartis, Ales Janda: None declared, Jürgen Rech Speakers bureau: bbvie, Biogen, BMS, Chugai, GSK, Janssen, Lilly, MSD; Mylan, Novartis, Roche, Sanofi, Sobi, UCB, Consultant of: Abbvie, Biogen, BMS, Chugai, GSK, Janssen, Lilly, MSD, Mylan, Novartis, Roche, Sanofi, Sobi, UCB, Grant/research support from: Novartis, Sobi, Tilmann Kallinich Consultant of: Sobi, Novartis, Roche, Grant/research support from: Novartis, Frank Weller-Heinemann: None declared, Gerd Horneff Speakers bureau: AbbVie, Bayer, Chugai, Merck Sharp & Dohme, Novartis, Pfizer, Roche, Grant/research support from: AbbVie, Chugai, Merck Sharp & Dohme, Novartis, Pfizer, Roche, Ivan Foeldvari Consultant of: Novartis, Catharina Schuetz: None declared, Michael Borte Grant/research support from: Pfizer, Shire, Axel Braner Consultant of: Novartis and SOBI, Julia Weber-Arden Employee of: Novartis, Norbert Blank Consultant of: Novartis, Sobi, Lilly, Pfizer, Abbvie, BMS, MSD, Actelion, UCB, Boehringer-Ingelheim, Roche, Grant/research support from: Novartis, Sobi |
| Databáze: | OpenAIRE |
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