Mr Shuang-jiang Andrew Tan1, Dr Hugh McCarthy2,3,4, Mr Tom Veitch1, Dr Siah Kim2,3,5, Dr Diedre Hahn2,3,4, Professor Stephen Alexander2,3,4
1Sydney Medical School, Faculty of Medicine and Health, University Of Sydney, Camperdown, Australia, 2Department of Nephrology, The Children’s Hospital at Westmead, Westmead , Australia, 3Centre for Kidney Research, The Children’s Hospital at Westmead, Westmead, Australia, 4Child and Adolescent Health, Sydney Medical School, Faculty of Medicine and Heath, University of Sydney, Camperdown, Australia, 5School of Public Health, Faculty of Medicine and Health, University of Sydney, Camperdown, Australia
Biography:
Dr Hugh McCarthy (PhD FRCPCH FRACP) is a full time paediatric nephrologist and clinical researcher within the University Of Sydney and Sydney Children’s Hospitals Network. Hugh established and runs the NSW paediatric renal genetic clinical service. His research focuses on the care of children living with rare and genetic renal disease and the study of the pathogenesis of nephrotic syndrome both genetic and immune mediated. Hugh developed the UK National Registry of Rare Renal Disease (RADAR) as part of his PhD.
Background:
Limited evidence exists around the relative efficacies of second-line agents (SLAs) for steroid sensitive nephrotic syndrome (SSNS) resulting in ambiguous international guidelines.
Aims:
To study the use of second line agents for childhood steroid sensitive nephrotic syndrome in New South Wales (NSW) and identify determinants of choice with variation over time.
Methods:
A retrospective medical record review of children with SSNS at Sydney Children’s Hospital, Randwick (SCH) or The Children’s Hospital at Westmead (CHW) was conducted from 2008 to 2022. Logistic regression was used to identify variables associated with choice of SLA.
Results:
143 children from a total of 173 with SSNS (83%) received an SLA. Median age was 11 years, median length of follow-up 4.8 years and median number of second line agents or combination of agents was 2 (IQR 1-4). Calcineurin inhibitors (CNIs) were the most common first choice in 84/143 (59%). Levamisole was the main alternative first choice, used first in 29/143 (20%). Use of cyclophosphamide, ciclosporin A decreased and rituximab correspondingly increased from 2008 to 2022. Mycophenolate was rarely first choice (n=4, 3%). Overall, 124 children (87%) received a CNI at some point and 67 (47%) received rituximab (although total dose and combination immunosuppression varied widely). Female gender (OR, 95% CI) (3.12, 1.03-9.46, 0.05) and hospital site (0.06, 0.02-0.21, <0.01) were associated with an alternative to CNI as first choice.
Conclusions:
Calcineurin inhibitors are the mainstay of therapy in NSW. Overall choice of therapy has changed over time with increasing, and earlier, use of rituximab. There remains significant variation confirming the need for future quantitative and qualitative study of treatment efficacy and tolerability.
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