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Lachmann, H. J.; Lauwerys, B.; Miettunen, P.; Kallinich, T.; Jansson, A.; Rosner, I.; Manna, R.; Murias, S.; Savic, S.; Smeets, S.; De Benedetti, F. und Simon, A. (2021): Canakinumab improves patient-reported outcomes in children and adults with autoinflammatory recurrent fever syndromes: results from the CLUSTER trial. In: Clinical and Experimental Rheumatology, Bd. 39, Nr. 5: S51-S58

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Abstract

Objective. To evaluate the effect of canakinumab on health-related quality of life (HRQoL), work/school and social life of patients with autoinflammatory recurrent fever syndromes, including colchicine-resistant familial Mediterranean fever, mevalonate kinase deficiency, and tumour necrosis factor receptor-associated periodic syndrome, in the CLUSTER trial. Methods. HRQoL of patients who received canakinumab 150 mg or 300 mg every four weeks in the CLUSTER trial (n=173) was assessed at baseline and Weeks 17 and 41. For children we used the Child Health Questionnaire - Parent Form 50 (CHQ-PF50), including psychosocial (PsS) and physical (PhS) component summary scores. For adults, the Short-Form-12 (SF-12) Health Survey was used, including physical (PFS) and mental (PCS) component summary scores. The Sheehan Disability Scale (SDS) was used to determine the impact of treatment on work/school, social and family life. Results. The results obtained were remarkably consistent in both paediatric and adult patients across the three disease cohorts. At baseline, median scores for physical components were relatively low (26-29 for PhS and 34-38 for PFS);they improved to values similar to those expected in the general population by Week 17, and this improvement was sustained at Week 41, when median PhS scores were 47-50 and PFS 44-54. Psychosocial and mental scores also improved from baseline to Week 17 and 41, with scores comparable to the general population. Notable improvements were also observed in the SDS scale. Conclusion. Patients with three inherited autoinflammatory syndromes experienced sustained improvements on their HRQoL, work/school, and social life on treatment with canakinumab.

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