BackgroundSystemic juvenile idiopathic arthritis (SJIA) is a severe autoinflammatory disease characterised by systemic features including high fevers, rash and arthritis. SJIA can impose a high physical, psychosocial, behavioral and financial burden on patients (pts) and their families.ObjectivesTo analyse the impact of the burden of SJIA by evaluating caregiver perspectives of disease burden utilising a SJIA-specific questionnaire combined with physician data about disease severity and treatment in an international, real-world study.MethodsSJIA treatment centers in France, Germany, Netherlands, UK and the US participated. Pts (aged 4–18 years) with confirmed SJIA received one of the following biologic treatments for ≥2 months: anakinra (ANA), canakinumab (CAN), or tocilizumab (TOC). SJIA burden in patients on biologics was assessed using a caregiver questionnaire and retrospective chart review. Validated measures included: Child Health Questionnaire Parent-Form 50 (CHQ-PF50), 36-Item Short-Form Health Survey (SF-36v2) and Work Productivity and Activity Impairment questionnaire: Specific Health Problem (WPAI:SHP). Caregivers completed function, treatment satisfaction and resource utilization questions.ResultsSixty-one pts enrolled from June 2015- June 2016: 12 on ANA, 25 on CAN, 24 on TOC; 46% from the US; 48% female; mean age at survey was 11.3 years. Mean age at SJIA diagnosis was 6.4 years, mean age at start of ANA, CAN, and TOC treatment was 9.9, 9.1, and 7.5 years, respectively. Caregivers were 79% female, mean age 41.2 years, and 36% reduced or stopped working due to their child's SJIA. Of the pts enrolled on CAN and TOC, 72% and 46% respectively had previously been on ANA. Baseline CHAQ, CHQ-PF50, and WPAI scores were worse in CAN and TOC than ANA pts. Mean (±SD) CHQ-PF50 physical (PhS) and psychosocial (PsS) summary scores were significantly lower in SJIA patients than a normative population (PhS: 40.0±18.2 vs. 53.0±8.8; PsS: 46.6±11.3 vs. 51.2±9.1) as was caregivers' mean SF-36v2 mental component score (46.2±10.7 vs. 50.0+10). Highest caregiver stressors were worry over long-term SJIA impact on their child (45%) and uncertainty about the future (28%).ConclusionsTreatment sequencing and patient-reported outcome measures indicate ANA is used as 1st line for less severe SJIA while CAN and TOC are used as 2nd/3rd line for severe SJIA. Caregivers expressed stress over the long-term impact of SJIA and fear for the future and had variable treatment satisfaction and resource utilisation levels.Disclosure of InterestS. Shenoi: None declared, G. Horneff Grant/research support from: Abbvie, Chugai, Novartis, Pfizer, Roche, M. Cidon: None declared, A. Ramanan: None declared, Y. Kimura Grant/research support from: Novartis, SOBI, CARRA, Inc (salary support), P. Quartier Grant/research support from: Abbvie, Novartis, Pfizer, Chugai-Roche, Consultant for: Abbvie, Novartis, Pfizer, Roche, Sobi, Speakers bureau: Abbvie, Novartis, Sobi, Roche, I. Foeldvari Speakers bureau: Novartis, Abbvie, Chugai, A. Zeft Shareholder of: ...
BackgroundPeriodic Fever Syndromes (PFS) are rare autoinflammatory conditions including Familial Mediterranean Fever (FMF), Hyper-IgD Syndrome/ Mevalonate Kinase Deficiency (HIDS/MKD), and TNF-Receptor Associated Periodic Syndrome (TRAPS).1 It has been shown that colchicine-resistant FMF (crFMF), HIDS/MKD and TRAPS considerably impact physical and emotional aspects of patients' lives.2–4 Open label studies suggested that canakinumab (CAN), a fully human and highly specific anti-IL-1β monoclonal antibody, is efficacious in crFMF, HIDS/MKD and TRAPS.5–7 To date, there is no data showing the effect of CAN on Health-Related Quality of Life (HRQoL) in PFS patients.ObjectivesTo evaluate the effect of CAN on HRQoL using Child Health Questionnaire – Parent Form 50 (CHQ-PF50) and SF-12 Health Survey (SF-12) in PFS patients.MethodsIn a Phase 3 randomised placebo controlled study of CAN in PFS (NCT02059291), SF-12 Physical Component Summary (PCS) and Mental Component Summary (MCS) were assessed in adults. For children (>5–<18 years), CHQ-PF50 Physical (PhS) and Psychosocial (PsS) Summary scores were assessed.Results181 patients were randomised to CAN or placebo in 3 cohorts (63 crFMF, 72 MKD/HIDS, 46 TRAPS). 71 adults ≥18 years and 110 children (age range ≥2–<18 years). Patients reported early clinically meaningful improvement in SF-12 PCS scores reported at Week (Wk) 5 which were sustained and increased to a large effect size by Wk 16 for all indications (Table). Similarly, clinically meaningful improvements in SF-12 MCS, CHQ-PF50 PhS and PsS was observed in all indications, with the exception of PsS in HIDS/MKD and TRAPS patients (Table).Table 1.Patient reported outcomesMean change from baseline (n/N)crFMFHIDS/MKDTRAPSWeek 5Week 16Week 5Week 16Week 5Week 16SF-12 PCS7.9 (29/30)9.55 (30/31)13.81 (15/15)13.81 (14/14)9.63 (16/17)11.64 (13/14)SF-12 MCS4.83 (29/30)4.27 (30/31)6.41 (15/15)8.14 (14/14)5.65 (16/17)5.51 (13/14)CHQ-PF50 PhS13.2 (21/24)20.1 (18/21)5.5 (32/34)9.9 (27/29)7.4 (16/18)14.9 (13/14)CHQ-PF50 PsS4.1 (21/24)7.2 (18/21)1.8* (32/34)5.2 (27/29)0.9* (16/18)1.2* (13/14)N = total number of patients; n = patients who received at least one dose of canakinumab. *Minimal important difference8,9from baseline was not achieved.ConclusionsCanakinumab showed rapid improvement by Week 5 in patient reported outcomes in adults and children with PFS, which was sustained through Week 16.ReferencesSavic S. and Wood P. Clin. Med. 2011;11(4):396–401Dandekar P, et al. Pediatr. Rheumatol. 13(S1):P22Dandekar P, et al. Pediatr. Rheumatol. 13(S1):P23Dandekar P, et al. Pediatr. Rheumatol. 13(S1):P24Brik R, et al. Arthritis Rheumatol. 2014;66(11):3241–3Arostegui, J.I, et al. Arthritis Rheumatol. 2015; 67 (S10)Gattorno M, et al. Arthritis Rheumatol. 2015; 67 (S10)User's manual for the SF-12v2 Health Survey, 3rd ed. 2012Cohen J, et. al. Statistical Power Analysis for the Behavioural Sciences,2nd ed. 1988Disclosure of InterestH. Lachmann Consultant for: Novartis, SOBI, Takeda, GSK, Speakers bureau: Novartis, SOBI, A. Simon Grant/research suppo...
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