J M Mason1, J R Chalmers2, T Godec3, A J Nunn3, G Kirtschig2, F Wojnarowska4, M Childs5, D Whitham5, E Schmidt6, K Harman7, S Walton8, A Chapman9, H C Williams2. 1. Warwick Medical School, University of Warwick, Coventry, CV4 7AL, U.K. 2. Centre of Evidence Based Dermatology, University of Nottingham, Nottingham, NG7 2NR, U.K. 3. MRC Clinical Trials Unit at UCL, Aviation House, 125 Kingsway, London, WC2B 6NH, U.K. 4. Nuffield Department of Clinical Medicine, University of Oxford, Oxford, OX3 7BN, U.K. 5. Nottingham Clinical Trials Unit, Nottingham Health Science Partners, QMC, Nottingham, NG7 2UH, U.K. 6. Department of Dermatology, University of Lübeck, Lübeck, Germany. 7. Dermatology Department, Leicester Royal Infirmary, University Hospitals Leicester, Leicester, LE1 5WW, U.K. 8. Castle Hill Hospital, Castle Road, Cottingham, Hull, HU16 5JQ, U.K. 9. Queen Elizabeth Hospital, Greenwich, London, SE18 4QH, U.K.
Abstract
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 randomized 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-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. RESULTS: In 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 -0·024, 95% CI -0·088 to 0·041]. 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 (-0·090 QALYs, 95% CI -0·22 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 per QALY. CONCLUSIONS: Consistently 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.
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 randomized 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-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. RESULTS: In 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 -0·024, 95% CI -0·088 to 0·041]. 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 (-0·090 QALYs, 95% CI -0·22 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 per QALY. CONCLUSIONS: Consistently 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.
Authors: Joanne R Chalmers; Fenella Wojnarowska; Gudula Kirtschig; James Mason; Margaret Childs; Diane Whitham; Karen Harman; Anna Chapman; Shernaz Walton; Enno Schmidt; Thomas R Godec; Andrew J Nunn; Hywel C Williams Journal: Health Technol Assess Date: 2017-03 Impact factor: 4.014
Authors: Don Husereau; Michael Drummond; Stavros Petrou; Chris Carswell; David Moher; Dan Greenberg; Federico Augustovski; Andrew H Briggs; Josephine Mauskopf; Elizabeth Loder Journal: BMJ Date: 2013-03-25
Authors: John S Barbieri; Ketaki Bhate; Kathleen P Hartnett; Katherine E Fleming-Dutra; David J Margolis Journal: JAMA Dermatol Date: 2019-03-01 Impact factor: 10.282