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 doxycycline-initiated and prednisolone-initiated treatment for patients with BP. METHODS: 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