Efficacy of a tight-control and treat-to-target strategy in axial spondyloarthritis: results of the open-label, pragmatic, cluster-randomised TICOSPA trial.

OBJECTIVES: To compare the benefits of a tight-control/treat-to-target strategy (TC/T2T) in axial spondyloarthritis (axSpA) with those of usual care (UC). METHODS: Pragmatic, prospective, cluster-randomised, controlled, open, 1-year trial (NCT03043846). 18 centres were randomised (1:1). Patients met Axial Spondylo Arthritis International Society (ASAS) criteria for axSpA, had an Ankylosing Spondylitis Disease Activity Score (ASDAS) ≥2.1, received non-optimal treatment by non-steroidal anti-inflammatory drugs and were biologic-naive. INTERVENTIONS: (1) TC/T2T: visits every 4 weeks and prespecified strategy based on treatment intensification until achieving target (ie, ASDAS <2.1); (2) UC: visits every 12 weeks and treatment at the rheumatologist's discretion. MAIN OUTCOME: Percentage of patients with a ≥30% improvement on the ASAS-Health Index (ASAS-HI). Other efficacy outcomes and adverse events were recorded. A health economic evaluation was performed. STATISTICAL ANALYSIS: Two-level mixed models were used to estimate efficacy outcomes. Cost-effectiveness was assessed by the incremental cost per quality-adjusted life-year (QALY) gained for TC/T2T versus UC. RESULTS: 160 patients were included (80/group). Mean (SD) age was 37.9 (11.0) years and disease duration was 3.7 (6.2) years; 51.2% were men. ASDAS at inclusion was 3.0 (0.7), and ASAS-HI was 8.6 (3.7). ASAS-HI improved by ≥30% in 47.3% of the TC/T2T arm and in 36.1% of those receiving UC (non-significant). All secondary efficacy outcomes were more frequent in the TC/T2T arm, although not all statistically significant. Safety was similar in both arms. From a societal perspective, TC/T2T resulted in an additional 0.04 QALY, and saved €472 compared with UC. CONCLUSION: TC/T2T was not significantly superior to UC for the primary outcome, while many secondary efficacy outcomes favoured it, had a similar safety profile and was favourable from a societal health economic perspective. TRIAL REGISTRATION NUMBER: NCT03043846.

Valuing Treatment With Infliximab for Ankylosing Spondylitis Using a Willingness-to-Pay Approach.

OBJECTIVE: To investigate willingness to pay (WTP) for treatment with infliximab by patients with ankylosing spondylitis (AS) and explore factors associated with WTP. METHODS: Data from 85 patients participating in the European AS Infliximab Cohort (EASIC) open-label extension of the AS Study for the Evaluation of Recombinant Infliximab Therapy (ASSERT) were used. WTP was included at baseline in EASIC and comprised a hypothetical scenario exploring whether the patient would be willing to pay for beneficial effects of infliximab and, if so, what amount they would be willing to pay per administration. Factors associated with WTP were explored using zero-inflated negative binomial (ZINB) regressions. RESULTS: Of the 85 patients, 63 (74.1%) were willing to pay, and among these, the mean amount they were willing to pay per administration was €275 (median €100 [interquartile range €50-200]). Multivariable ZINB analysis showed that Assessment of SpondyloArthritis international Society criteria for 20% improvement (ASAS20) response was associated with a 7-fold lower likelihood to pay 0 euros (odds ratio [OR] 0.14 [95% confidence interval (95% CI) 0.03-0.71]) and a 3-fold increase in the amount willing to pay (exp(ß) = 3.32 [95% CI 1.44-7.69]). In addition, the country of residence was associated with a lower likelihood to pay 0 euros (OR 0.07 [95% CI 0.02-0.36]), while increased age was associated with the amount willing to pay (exp(ß) = 1.05 [95% CI 1.01-1.09]). CONCLUSION: In a hypothetical scenario, three-quarters of patients with AS receiving long-term infliximab stated that they were willing to pay an out-of-pocket contribution for this treatment. Treatment response contributed to the willingness as well as to the amount patients were willing to pay.