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

Nafsika Afentou; Emma Frew; Samir Mehta; Natalie J Ives; Rebecca L Woolley; Elizabeth A Brettell; Adam R Khan; David V Milford; Detlef Bockenhauer; Moin A Saleem; Angela S Hall; Ania Koziell; Heather Maxwell; Shivaram Hegde; Eric Finlay; Rodney D Gilbert; Caroline Jones; Karl McKeever; Wendy Cook; Nicholas J A Webb; Martin T Christian; PREDNOS Collaborative Group, NIHR Medicines for Children Research Network Nephrology Clinical Studies Group

doi:10.1007/s41669-022-00334-6

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

Pharmacoecon Open. 2022 Jul;6(4):605-617. doi: 10.1007/s41669-022-00334-6. Epub 2022 Jun 22.

Authors

Nafsika Afentou¹, Emma Frew², Samir Mehta³, Natalie J Ives³, Rebecca L Woolley³, Elizabeth A Brettell³, Adam R Khan³, David V Milford⁴, Detlef Bockenhauer^{5

6}, Moin A Saleem^{7

8}, Angela S Hall⁹, Ania Koziell^{10

11}, Heather Maxwell¹², Shivaram Hegde¹³, Eric Finlay¹⁴, Rodney D Gilbert¹⁵, Caroline Jones¹⁶, Karl McKeever¹⁷, Wendy Cook¹⁸, Nicholas J A Webb^{19

20}, Martin T Christian²¹; PREDNOS Collaborative Group, NIHR Medicines for Children Research Network Nephrology Clinical Studies Group

Collaborators

PREDNOS Collaborative Group, NIHR Medicines for Children Research Network Nephrology Clinical Studies Group:
Nicholas Webb, Martin Christian, Natalie Ives, Emma Frew, Elizabeth Brettell, Emma Barsoum, Helen Bodenham-Chilton, Adam Khan, Noreen Akhtar, Charmaine Hunt, Carla Galinha, Dominique Smith, Neil Winkles, Natalie Ives, Rebecca Woolley, Samir Mehta, Catherine Moakes, Emma Frew, Nafsika Afentou, Wendy Cook, Sandra Cope, Megan Thomas, Nigel Coad, Sandra Cope, Darren Green, Kate Hillman, Zala Ibrahim, Ly-Mee Yu, Alison Watson, Fatima Bibi Team, Eduardo Moya, Moin Saleem, Alison Kelly, Eilean Crosbie, Rachel Swingler, Susan Kilroy Team, Oyekunle Ayonrinde, Amanda Smith, Andrea Turner, Jonathan Campbell, Aine Turner, Stephen Brearey, Caroline Burchett, Sarah De-Beger, Theo Fenton, Glyn Jones, Nicci Kelsall, Selwyn D'Costa, Dinakaran Jayachandran, Asha Nair Catherine Tarn Nozedar, Dawn Egginton, Oliver Cuthell, Catherine Derry, Kathiresan Natesan, Sarah-Jane Sharman, Rajeeva Singh, Kathryn Deakin, Gail Castle Team, Bukar Wobi, Bemigho Etuwewe, Caroline Burnett Team, Kamal Khoobarry, Graham Whincup, Anne Cowley, Ania Koziell, Manish Sinha, Christopher Reid, Mohammad Ahmad Team, Beena Padmakumar, Talaivirichan Magadevan, Grainne O'Connor, Louise Woodhead, Mireille Formosa, Nayan Peepah Nardeosingh, Kathryn Allison, Markus Hesseling, Annette Bolger, Lucie Hobson Team, Adamu Sambo, Lyda Jadresic, Susan Beames, Detlef Bockenhauer, Daljit Hothi, Elizabeth Vella, Corinne Linton, Shaima Yussuf, Tendai Bazaya, Mahmoud Abou-Rayyah Team, Nick West, Rajiv Sood, Hilarious De Jesus Team, Vikas Gupta, Verghese Mathew, Esi Bentsi-Enchill, Allyson Davison, Janet Craze, Elhussein Rfidah, Janine Musselwhite, Angela Moon Team, Harsha Bilolikar, Sonia White Team, Simon Rhodes, Caroline Moulds, Hitesh Prajapati, Eric Finlay, Pallavi Yadav, Amanda Newnham, Kay Tyerman, Majorie Allen, Lucy Wellings Team, Angela Hall, Jackie Philps Team, Subajini Kaviethasan, Sally Smith Team, David Broodbank, Sourabh Mukhopadhyay, Ruchika Gupta, Amanda Roper, Susie Butler Team, Tomasz Rajkowski, Michael Eisenhut, Karen Duncan, Karen Samm, Samantha Clough Team, David Wright, Krishnakumar Thattakkat, Ignatius Losa, Natalie Keenan Team, Krishnan Balasubramanian, Muhammad Javed, Sharon Kempson, Marie Phipps Team, Janette Cansick, Maines Msiska, Lazarus Anguvaa, Mya Aye, Sally Conway, Natalie Beer, Francesca Wright Team, Jennifer Langlands, Kirsty O'Brien, Nicola Thorne Team, Karen Davies, Sharon Kempson, Marie Phipps Team, Ami Parikh, Nimze Gadong, Bahadur Anjum, Nicolene Plaatjies, Ivone Lancoma-Malcolm, Hilarious De Jesus Team, Vipan Datta, Chris Upton, Louisa Fear, Louise Coke Team, Dermot Dalton, Becky Holbrook Team, Imogen Norton, Martin Christian, Andrew Lunn, Olivia Vincent, Helen Navarra, Neelam Khan Team, Mona Aslam, Paula Goodyear, Steve Wadams, Susan Power, Amy Roff Team, Manish Gupta, Naeem Ayub, Charlotte Owen Team, Judith Scanlan, Sharon McCready, Andrew Gribbin, Mansoor Ahmed, Dominic Muogbo, Heather Carswell, Stephanie Boswell, Claire Backhouse Team, Junaid Solebo, Helen Smith, Alan Webb, Ing-Marie Logie, Sandra Dekker, Sanjay Suri, Janet Shackleton Team, Craig Oxley, Margaret Connon Team, Vineeta Joshi, Nicola Pemberton Team, Heather Maxwell, Amita Sharma, Elizabeth Waxman Team, Karl McKeever, Muriel Millar Team, Ann Gordon, Susan Edees, Susan Hallett Team, Javed Iqbal, Beate von Bremen, Heather Collier, Andrew Lancaster Team, Fiona Watson, Joanne Henry Team, Richard Bowker, Coral Smith, Hannah Cottis, Rebecca Samuel, Caroline Harrill, Suzanne Wilkins Team, Heather Maxwell, Ben Reynolds, David Hughes, Elizabeth, Waxman Team, Ben Reynolds, David Hughes, Tracey McGregor, Maxine Ramsay, Julie Baggott, Naomi Matos Team, Caroline Jones, Henry Morgan, Richard Holt, Louise Oni Theresa Moorcroft, Joanne Shakeshaft Team, Mohan Shenoy, Nicholas Webb, Amrit Kaur, Dean Wallace, Nicholas Plant, Shaila Sukthankar, Angela Branson, Helen Blackburn, Jane Howell, Jess Nichols Team, Furqan Basharat, Saeeda Raja, Marie Phipps, Helen Parker, Joanne Tomlinson, Eric Roe Team, Lynn Diskin, Alison Barratt, Subramanian Mahadevan-Bava, Abigail Weston, Daljit Kaur, Udupa Venkatesh, Emma Temlett, Simon Dyer, Kerry Elliott, Rosie Furness Team, Andrew Lunn, Janet Shackleton, Sarah Shortland, Miranda Murray Team, Rodney Gilbert, Matthew Harmer, Shuman Haq, Lisa Fairhead, Louise Haskell, Victoria Bingham Team, Anupam Shrivastava, Onie Hove, Bernard Hadebe, Christopher Magier, Bettina Harms, Sian Butterworth, Tariq Bhatti, Aisling Parker, Lorna Walding Team, Nicholas Brennan, Nicholas Brennan, Chris Cooper, Sara Bennett Team, Anjali Date, Anjali Petkar, Wendy Hulse Team, Munir Ahmed, Tom Dawson, Connie Rowlands Stephanie Chamberlain, Simon Meyrick, Iain Darwood, Emma Collins, Rajesh Lall, Elizabeth Onifade, Joanna Green Team, Sally Johnson, Heather Lambert, Yincent Tse, Michal Malina, Vijaya Sathyanarayan, Jenny Booth, Kathryn Bell, Stephen Crulley Team, Jackie Buck, Deborah Beeby, Louise Hunt Team, Sundeep Sandhu, Gur Millman, Murray Wheeler, Anna Clayton, David Thompson Team, Bridget Oates, Claire Bell, Joanna Wardrop Team, Vijay Tandle, Carolyn Campbell, Dawn Egginton Team, Shivaram Hegde, Rajesh Krishnan, Zoe Morrison, Jennifer Muller, Louise Yendle Team, Delyth Webb, Natalie Rogers, Nour Elhadi, Dipali Shah, Amrinder Sayan Team, Karine Cesar, Raman Lakshman, Helen Cockerill, Zilla Huma, Thin Thin Saing, Angela Brown, Karen Leitch Team, Gopi Vemuri, Claire Holliday, Jessica Carey, Louise Woodhead TeamMadalitso Kubwalo, Annette Bolger Team

Affiliations

¹ Health Economics Unit, University of Birmingham, Birmingham, UK.
² Health Economics Unit, University of Birmingham, Birmingham, UK. [email protected].
³ Birmingham Clinical Trials Unit, University of Birmingham, Birmingham, UK.
⁴ Department of Paediatric Nephrology, Birmingham Children's Hospital, Birmingham, UK.
⁵ Department of Renal Medicine, University College London, London, UK.
⁶ Department of Paediatric Nephrology, Great Ormond Street Hospital for Children, London, UK.
⁷ School of Clinical Sciences, University of Bristol, Bristol, UK.
⁸ Department of Paediatric Nephrology, Bristol Royal Hospital for Children, Bristol, UK.
⁹ Leicester Children's Hospital, Leicester, UK.
¹⁰ Child Health Clinical Academic Group, King's College London, London, UK.
¹¹ Department of Paediatric Nephrology, Evelina Children's Hospital, London, UK.
¹² Department of Paediatric Nephrology, Royal Hospital for Sick Children, Glasgow, UK.
¹³ Department of Paediatric Nephrology, University Hospital of Wales, Cardiff, UK.
¹⁴ Department of Paediatric Nephrology, Leeds Children's Hospital, Leeds, UK.
¹⁵ Department of Paediatric Nephrology, Southampton Children's Hospital, Southampton, UK.
¹⁶ Department of Paediatric Nephrology, Alder Hey Children's Hospital, Liverpool, UK.
¹⁷ Department of Paediatric Nephrology, Royal Hospital for Sick Children, Belfast, UK.
¹⁸ Nephrotic Syndrome Trust (NeST), Taunton, UK.
¹⁹ Department of Paediatric Nephrology, University of Manchester, Manchester, UK.
²⁰ Academic Health Science Centre, Royal Manchester Children's Hospital, Manchester, UK.
²¹ Department of Paediatric Nephrology, Nottingham Children's Hospital, Nottingham, UK.

Abstract

Background: 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.

Objective: 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.

Methods: 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.

Results: 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.

Conclusion: 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.