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

作者：Mason J M; Chalmers J R; Godec T; Nunn A J; Kirtschig G; Wojnarowska F; Childs M; Whitham D; Schmidt E; Harman K; Walton S; Chapman A; Williams H C

来源：British Journal of Dermatology, 2018, 178(2): 415-423.

DOI：10.1111/bjd.16006

BackgroundBullous 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 randomized controlled trial (RCT).
ObjectivesTo compare the cost-effectiveness of doxycycline-initiated and prednisolone-initiated treatment for patients with BP.
MethodsQuality-of-life (EuroQoL-5D-3L) and resource data were collected as part of the BLISTER trial: a multicentre, parallel-group, investigator-blinded RCT. Within-trial analysis was performed using bivariate regression of costs and quality-adjusted life-years (QALYs), with multiple imputation of missing data, informing a probabilistic assessment of incremental treatment cost-effectiveness from a health service perspective.
ResultsIn the base case, there was no robust difference in costs or QALYs per patient at 1 year comparing doxycycline- with prednisolone-initiated therapy [net cost 959, 95% confidence interval (CI) -24 to 1941; net QALYs -0024, 95% CI -0088 to 0041]. However, the findings varied by baseline blister severity. For patients with mild or moderate blistering ( 30 blisters) net costs and outcomes were similar. For patients with severe blistering (> 30 blisters) net costs were higher (2558, 95% CI -82 to 5198) and quality of life poorer (-0090 QALYs, 95% CI -022 to 0042) for patients starting on doxycycline. The probability that doxycycline would be cost-effective for those with severe pemphigoid was 15% at a willingness to pay of 20 000 per QALY.
ConclusionsConsistently 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 National Health Service resources. However, prednisolone-initiated treatment may be more cost-effective for patients with severe blistering.
What's already known about this topic?
Bullous pemphigoid is a subepidermal blistering autoimmune skin disease, associated with increased morbidity and mortality. Prednisolone has long been the main systemic treatment. Although tetracyclines have also been used, their effectiveness and safety have not been estimated reliably to date. BLISTER was a pragmatic noninferiority randomized controlled trial starting oral treatment with either doxycycline 200 mg daily or prednisolone 0.5 mg kg(-1) per day. That trial demonstrated that doxycycline was a significantly safer treatment than prednisolone but less effective in terms of blister control at 6 weeks.
What does this study add?
Although doxycycline and prednisolone offered different effectiveness and safety profiles, costs and quality of life were similar when comparing patients presenting with mild-to-moderate blistering ( 30 blisters). For patients with severe blistering (> 30 blisters) at presentation, starting prednisolone resulted in lower cost and higher quality of life, making it a more cost-effective strategy.
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• 出版日期2018-2