Randomized Controlled Trial of Rituximab and Cost-Effectiveness Analysis in Treating Fatigue and Oral Dryness in Primary Sjogren's Syndrome



Bowman, Simon J, Everett, Colin C, O'Dwyer, John L, Emery, Paul, Pitzalis, Costantino, Ng, Wan-Fai, Pease, Colin T, Price, Elizabeth J, Sutcliffe, Nurhan, Gendi, Nagui ST
et al (show 13 more authors) (2017) Randomized Controlled Trial of Rituximab and Cost-Effectiveness Analysis in Treating Fatigue and Oral Dryness in Primary Sjogren's Syndrome. ARTHRITIS & RHEUMATOLOGY, 69 (7). pp. 1440-1450.

[thumbnail of art40093.pdf] Text
art40093.pdf - Author Accepted Manuscript

Download (962kB)

Abstract

<h4>Objective</h4>To investigate whether rituximab, an anti-B cell therapy, improves symptoms of fatigue and oral dryness in patients with primary Sjögren's syndrome (SS).<h4>Methods</h4>We conducted a multicenter, randomized, double-blind, placebo-controlled, parallel-group trial that included health economic analysis. Anti-Ro-positive patients with primary SS, symptomatic fatigue, and oral dryness were recruited from 25 UK rheumatology clinics from August 2011 to January 2014. Patients were centrally randomized to receive either intravenous (IV) placebo (250 ml saline) or IV rituximab (1,000 mg in 250 ml saline) in 2 courses at weeks 0, 2, 24, and 26, with pre- and postinfusion medication including corticosteroids. The primary end point was the proportion of patients achieving a 30% reduction in either fatigue or oral dryness at 48 weeks, as measured by visual analog scale. Other outcome measures included salivary and lacrimal flow rates, quality of life, scores on the European League Against Rheumatism (EULAR) Sjögren's Syndrome Patient Reported Index and EULAR Sjögren's Syndrome Disease Activity Index, symptoms of ocular and overall dryness, pain, globally assessed disease activity, and cost-effectiveness.<h4>Results</h4>All 133 patients who were randomized to receive placebo (n = 66) or rituximab (n = 67) were included in the primary analysis. Among patients with complete data, 21 of 56 placebo-treated patients and 24 of 61 rituximab-treated patients achieved the primary end point. After multiple imputation of missing outcomes, response rates in the placebo and rituximab groups were 36.8% and 39.8%, respectively (adjusted odds ratio 1.13 [95% confidence interval 0.50, 2.55]). There were no significant improvements in any outcome measure except for unstimulated salivary flow. The mean ± SD costs per patient for rituximab and placebo were £10,752 ± 264.75 and £2,672 ± 241.71, respectively. There were slightly more adverse events (AEs) reported in total for rituximab, but there was no difference in serious AEs (10 in each group).<h4>Conclusion</h4>The results of this study indicate that rituximab is neither clinically effective nor cost-effective in this patient population.

Item Type: Article
Uncontrolled Keywords: Humans, Sjogren's Syndrome, Xerostomia, Fatigue, Antirheumatic Agents, Treatment Outcome, Odds Ratio, Double-Blind Method, Quality-Adjusted Life Years, Quality of Life, Adult, Aged, Middle Aged, Cost-Benefit Analysis, Female, Male, Visual Analog Scale, Rituximab, United Kingdom, Patient Reported Outcome Measures
Depositing User: Symplectic Admin
Date Deposited: 21 Apr 2017 09:15
Last Modified: 19 Jan 2023 07:06
DOI: 10.1002/art.40093
Related URLs:
URI: https://livrepository.liverpool.ac.uk/id/eprint/3007033