Doxycycline compared to prednisolone therapy for patients with bullous pemphigoid: cost-effectiveness analysis of the BLISTER trial

Background: Bullous pemphigoid (BP) is an autoimmune blistering skin disorder associated with significant morbidity and mortality. Doxycycline and prednisolone to treat bullous pemphigoid were compared within a randomised controlled trial (RCT). Objectives: To compare the cost-effectiveness of d...

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Main Authors: Mason, James M., Chalmers, J.R., Godec, Thomas R., Nunn, Andrew J., Kirtschig, Gudula, Wojnarowska, Fenella, Childs, Margaret, Whitham, Diane, Schmidt, Enno, Harman, Karen, Walton, Shernaz, Chapman, Anna, Williams, Hywel C.
Format: Article
Published: Wiley 2018
Online Access:https://eprints.nottingham.ac.uk/46817/
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Summary:Background: Bullous pemphigoid (BP) is an autoimmune blistering skin disorder associated with significant morbidity and mortality. Doxycycline and prednisolone to treat bullous pemphigoid were compared within a randomised controlled trial (RCT). Objectives: To compare the cost-effectiveness of doxycycline-initiated and prednisolone-initiated treatment for patients with BP. Methods: Quality-of-life (EuroQoL EQ-5D-3L) and resource data were collected as part of the BLISTER trial: a multicentre, parallel-group, investigator-blinded RCT. Within-trial analysis used bivariate regression of costs and QALYs, with multiple imputation of missing data, informing a probabilistic assessment of incremental treatment cost-effectiveness from a health service perspective. Results: In the base case, there was no robust difference in costs or QALYs per patient at 1 year comparing doxycycline-initiated therapy with prednisolone-initiated therapy (net cost: £959, 95% CI –£24 to £1941; net QALYs: –0.024, 95% CI –0.088 to 0.041). However, findings varied by baseline blister severity. For patients with mild or moderate blistering (≤30) net costs and outcomes were similar. For patients with severe blistering (>30) net costs were higher (£2558, 95% CI –£82 to £5198) and quality of life poorer (–0.090 QALYs, 95% CI–0.222 to 0.042) for patients starting on doxycycline. The probability that doxycycline would be cost-effective for those with severe pemphigoid was 1.5% at a willingness to pay of £20,000/QALY. Conclusions: Consistent with the clinical findings of the BLISTER trial, patients with mild or moderate blistering should receive treatment guided by the safety and effectiveness of the drugs and patient preference - neither strategy is clearly a preferred use of NHS resources. However, prednisolone-initiated treatment may be more cost-effective for patients with severe blistering.