Economic Evaluation of Using Daily Prednisolone versus Placebo at the Time of an Upper Respiratory Tract Infection for the Management of Children with Steroid-Sensitive Nephrotic Syndrome: A Model-Based Analysis

PharmacoEconomics - Open - Tập 6 - Trang 605-617 - 2022
Nafsika Afentou1, Emma Frew1, Samir Mehta2, Natalie J. Ives2, Rebecca L. Woolley2, Elizabeth A. Brettell2, Adam R. Khan2, David V. Milford3, Detlef Bockenhauer4,5, Moin A. Saleem6,7, Angela S. Hall8, Ania Koziell9,10, Heather Maxwell11, Shivaram Hegde12, Eric Finlay13, Rodney D. Gilbert14, Caroline Jones15, Karl McKeever16, Wendy Cook17, Nicholas J. A. Webb18,19, Martin T. Christian20
1Health Economics Unit, University of Birmingham, Birmingham, UK
2Birmingham Clinical Trials Unit, University of Birmingham, Birmingham, UK
3Department of Paediatric Nephrology, Birmingham Children’s Hospital, Birmingham, UK
4Department of Renal Medicine, University College London, London, UK
5Department of Paediatric Nephrology, Great Ormond Street Hospital for Children, London, UK
6School of Clinical Sciences, University of Bristol, Bristol, UK
7Department of Paediatric Nephrology, Bristol Royal Hospital for Children, Bristol, UK
8Leicester Children’s Hospital, Leicester, UK
9Child Health Clinical Academic Group, King’s College London, London, UK
10Department of Paediatric Nephrology, Evelina Children’s Hospital, London, UK
11Department of Paediatric Nephrology, Royal Hospital for Sick Children, Glasgow, UK
12Department of Paediatric Nephrology, University Hospital of Wales, Cardiff, UK
13Department of Paediatric Nephrology, Leeds Children’s Hospital, Leeds, UK
14Department of Paediatric Nephrology, Southampton Children’s Hospital, Southampton, UK
15Department of Paediatric Nephrology, Alder Hey Children’s Hospital, Liverpool, UK
16Department of Paediatric Nephrology, Royal Hospital for Sick Children, Belfast, UK
17Nephrotic Syndrome Trust (NeST), Taunton, UK
18Department of Paediatric Nephrology, University of Manchester, Manchester, UK
19Academic Health Science Centre, Royal Manchester Children’s Hospital, Manchester, UK
20Department of Paediatric Nephrology, Nottingham Children’s Hospital, Nottingham, UK

Tóm tắt

Childhood steroid-sensitive nephrotic syndrome is a frequently relapsing disease with significant short- and long-term complications, leading to high healthcare costs and reduced quality of life for patients. The majority of relapses are triggered by upper respiratory tract infections (URTIs) and evidence shows that daily low-dose prednisolone at the time of infection may reduce the risk of relapse. The aim of this study was to assess the cost effectiveness of a 6-day course of low-dose prednisolone at the start of a URTI when compared with placebo. A state-transition Markov model was developed to conduct a cost-utility analysis with the outcome measured in quality-adjusted life-years (QALYs). Resource use and outcome data were derived from the PREDNOS2 trial. The analysis was performed from a UK National Health Service perspective and the results were extrapolated to adulthood. Model parameter and structural uncertainty were assessed using sensitivity analyses. The base-case results showed that administering low-dose prednisolone at the time of a URTI generated more QALYs and a lower mean cost at 1 year compared with placebo. In the long-term, low-dose prednisolone was associated with a cost saving (£176) and increased effectiveness (0.01 QALYs) compared with placebo and thus remained the dominant treatment option. These findings were robust to all sensitivity analyses. A 6-day course of low-dose prednisolone at the time of a URTI in children with steroid-sensitive nephrotic syndrome has the potential to reduce healthcare costs and improve quality of life compared with placebo.

Tài liệu tham khảo

McKinney PA, et al. Time trends and ethnic patterns of childhood nephrotic syndrome in Yorkshire, UK. Pediatr Nephrol. 2001;16(12):1040–4. Rees L, et al. Paediatric Nephrology (Oxford Specialist Handbooks in Paediatrics). Oxford: Oxford University Press; 2012. Webb NJA, et al. Long term tapering versus standard prednisolone treatment for first episode of childhood nephrotic syndrome: phase III randomised controlled trial and economic evaluation. BMJ. 2019;365: l1800. Larkins N, et al. Steroid-sensitive nephrotic syndrome: an evidence-based update of immunosuppressive treatment in children. Arch Dis Child. 2016;101(4):404–8. Noone DG, Iijima K, Parekh R. Idiopathic nephrotic syndrome in children. Lancet. 2018;392(10141):61–74. McCaffrey J, Lennon R, Webb NJ. The non-immunosuppressive management of childhood nephrotic syndrome. Pediatr Nephrol. 2016;31(9):1383–402. Alwadhi RK, Mathew JL, Rath B. Clinical profile of children with nephrotic syndrome not on glucorticoid therapy, but presenting with infection. J Paediatr Child Health. 2004;40(1–2):28–32. Arun S, et al. Efficacy of zinc supplements in reducing relapses in steroid-sensitive nephrotic syndrome. Pediatr Nephrol. 2009;24(8):1583–6. MacDonald NE, et al. Role of respiratory viruses in exacerbations of primary nephrotic syndrome. J Pediatr. 1986;108(3):378–82. Mattoo TK, Mahmoud MA. Increased maintenance corticosteroids during upper respiratory infection decrease the risk of relapse in nephrotic syndrome. Nephron. 2000;85(4):343–5. Abeyagunawardena AS, Trompeter RS. Increasing the dose of prednisolone during viral infections reduces the risk of relapse in nephrotic syndrome: a randomised controlled trial. Arch Dis Child. 2008;93(3):226–8. Gulati A, et al. Daily corticosteroids reduce infection-associated relapses in frequently relapsing nephrotic syndrome: a randomized controlled trial. Clin J Am Soc Nephrol. 2011;6(1):63–9. Abeyagunawardena AS, et al. Short courses of daily prednisolone during upper respiratory tract infections reduce relapse frequency in childhood nephrotic syndrome. Pediatr Nephrol. 2017;32(8):1377–82. Christian MT, et al. Evaluation of daily low-dose prednisolone during upper respiratory tract infection to prevent relapse in children with relapsing steroid-sensitive nephrotic syndrome: the PREDNOS 2 Randomized Clinical Trial. JAMA Pediatr. 2022;176(3):236–43. Webb NJ, et al. Short course daily prednisolone therapy during an upper respiratory tract infection in children with relapsing steroid-sensitive nephrotic syndrome (PREDNOS 2): protocol for a randomised controlled trial. Trials. 2014;15:147. Ramsey SD, et al. Cost-effectiveness analysis alongside clinical trials II-An ISPOR Good Research Practices Task Force report. Value Health. 2015;18(2):161–72. Stevens K. Valuation of the Child Health Utility 9D Index. Pharmacoeconomics. 2012;30(8):729–47. Varni JW, et al. The PedsQL 4.0 as a pediatric population health measure: feasibility, reliability, and validity. Ambul Pediatr. 2003;3(6):329-341. Devlin N, Parkin D, Janssen B. Methods for Analysing and Reporting EQ-5D Data. Springer International Publishing; 2020. National Institute for Health Care Excellence. NICE process and methods guides, in guide to the methods of technology appraisal 2013. London: National Institute for Health and Care Excellence; 2013. Curtis L, Burns A. Unit Costs of Health and Social Care 2019. Personal Social Services Research Unit. https://www.pssru.ac.uk/project-pages/unit-costs/unit-costs-2019/. Accessed Feb 2020. National Health Service. National Schedule of NHS costs. 2019. https://www.england.nhs.uk/wp-content/uploads/2020/08/1_-_NCC_Report_FINAL_002.pdf. Accessed Feb 2020. Paediatric Formulary Committee. BNF for Children (online). BMJ Group (ed). Pharmaceutical Press and RCPCH Publications; 2020. www.medicinescomplete.com. Accessed Feb 2020. Lambe T, et al. Mapping The Paediatric Quality Of Life Inventory (PedsQL™) Generic Core Scales onto the Child Health Utility Index-9 Dimension (CHU-9D) score for economic evaluation in children. Pharmacoeconomics. 2018;36(4):451–65. Manca A, Hawkins N, Sculpher MJ. Estimating mean QALYs in trial-based cost-effectiveness analysis: the importance of controlling for baseline utility. Health Econ. 2005;14(5):487–96. Canaway AG, Frew EJ. Measuring preference-based quality of life in children aged 6–7 years: a comparison of the performance of the CHU-9D and EQ-5D-Y–the WAVES pilot study. Qual Life Res. 2013;22(1):173–83. Claxton K. The irrelevance of inference: a decision-making approach to the stochastic evaluation of health care technologies. J Health Econ. 1999;18(3):341–64. Raftery J, et al. ’Not clinically effective but cost-effective’—paradoxical conclusions in randomised controlled trials with “doubly null” results: a cross-sectional study. BMJ Open. 2020;10(1): e029596. O’Brien BJ, Briggs AH. Analysis of uncertainty in health care cost-effectiveness studies: an introduction to statistical issues and methods. Stat Methods Med Res. 2002;11(6):455–68. Briggs A, Sculpher M. An introduction to Markov modelling for economic evaluation. Pharmacoeconomics. 1998;13(4):397–409. Sinha A, et al. Disease course in steroid sensitive nephrotic syndrome. Indian Pediatr. 2012;49(11):881–7.