Randomized controlled trial of rituximab and cost-effectiveness analysis in treating fatigue and oral dryness in primary Sjögren'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 S T, Hall, Frances C, Ruddock, Sharon P, Fernandez, Catherine, Reynolds, Catherine, Hulme, Claire T, Davies, Kevin A, Edwards, Christopher J, Lanyon, Peter C, Moots, Robert J, Roussou, Euthalia, Giles, Ian P, Sharples, Linda D and Bombardieri, Michele (2017) Randomized controlled trial of rituximab and cost-effectiveness analysis in treating fatigue and oral dryness in primary Sjögren's Syndrome. Arthritis and Rheumatology, 69 (7). pp. 1440-1450. ISSN 2326-5191

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Abstract

Objective
To investigate whether rituximab, an anti–B cell therapy, improves symptoms of fatigue and oral dryness in patients with primary Sjögren's syndrome (SS).

Methods
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.

Results
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).

Conclusion
The results of this study indicate that rituximab is neither clinically effective nor cost‐effective in this patient population.

Item Type: Article
Schools and Departments: Brighton and Sussex Medical School > Clinical and Experimental Medicine
Subjects: R Medicine
Depositing User: Jessica Stockdale
Date Deposited: 21 Apr 2017 14:43
Last Modified: 03 Feb 2021 16:07
URI: http://sro.sussex.ac.uk/id/eprint/67451

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