Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association · 2026
Steroid-sparing drugs in children and young adults with nephrotic syndrome: a target trial emulation.
СтатьяРандомизированное исследованиеPragmatic Clinical TrialНазвание и аннотация: Перевод в очереди
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Robinson Cal H, Heath Anna, Greenbaum Larry A, Hladunewich Michelle A, Aman Nowrin, Banh Tonny H M, Brooke Josefina, Canetta Pietro, Cara-Fuentes Gabriel, Dhillon Vaneet, Garner Mackenzie, Helmuth Margaret, Kidd Jason, Larkina Maria, Licht Christoph, McKay Ashlene, Pearl Rachel, Radhakrishnan Seetha, Reidy Kimberly, Selvathesan Nithiakishna, Teoh Chia Wei, Vasilevska-Ristovska Jovanka, Parekh Rulan S
Аннотация
BACKGROUND: Children and young adults with nephrotic syndrome are commonly treated with steroid-sparing immunosuppressive medications. Rituximab is increasingly used in frequently relapsing disease, but the real-world comparative effectiveness of rituximab and calcineurin inhibitors is unclear.
METHODS: Using target trial methods, we emulated a pragmatic randomized controlled trial using observational data from two prospective nephrotic syndrome cohorts. We included children and adults (aged 1-40 years) with steroid-sensitive nephrotic syndrome that first initiated rituximab or calcineurin inhibitor treatment during follow-up. Randomization was emulated using overlap weighting of propensity scores for treatment assignment. The primary outcome was time-to-relapse, analyzed by weighted Cox proportional hazards models. Secondary outcomes were relapse counts, subsequent immunosuppressive treatment, kidney function, hypertension and all-cause mortality.
RESULTS: Of 372 eligible participants, 104 initiated rituximab and 268 initiated calcineurin inhibitors. Baseline characteristics were balanced after propensity score weighting. Mean age at medication initiation was 13.2 years, 61% were male and 69% had received prior steroid-sparing medications. Throughout a median 4.9-year (quartile 1-3, 2.0-7.2) follow-up, relapses occurred in 58 participants (56%) treated with rituximab and 209 (78%) treated with calcineurin inhibitors. There was no significant difference in time-to-relapse after rituximab vs calcineurin inhibitor treatment [weighted hazard ratio (HR) 0.90, 95% confidence interval (CI) 0.59-1.38]. There were also no significant differences in relapse rates or kidney function decline. Participants treated with rituximab were at lower risk of developing hypertension (weighted HR 0.24, 95% CI 0.08-0.79).
CONCLUSIONS: Using real-world data, there was no difference in relapse rates after rituximab vs calcineurin inhibitors among children and young adults with steroid-sensitive nephrotic syndrome. Steroid-sparing immunosuppression selection should be a shared decision, considering medication-specific side effects, costs, access, duration and adherence.
METHODS: Using target trial methods, we emulated a pragmatic randomized controlled trial using observational data from two prospective nephrotic syndrome cohorts. We included children and adults (aged 1-40 years) with steroid-sensitive nephrotic syndrome that first initiated rituximab or calcineurin inhibitor treatment during follow-up. Randomization was emulated using overlap weighting of propensity scores for treatment assignment. The primary outcome was time-to-relapse, analyzed by weighted Cox proportional hazards models. Secondary outcomes were relapse counts, subsequent immunosuppressive treatment, kidney function, hypertension and all-cause mortality.
RESULTS: Of 372 eligible participants, 104 initiated rituximab and 268 initiated calcineurin inhibitors. Baseline characteristics were balanced after propensity score weighting. Mean age at medication initiation was 13.2 years, 61% were male and 69% had received prior steroid-sparing medications. Throughout a median 4.9-year (quartile 1-3, 2.0-7.2) follow-up, relapses occurred in 58 participants (56%) treated with rituximab and 209 (78%) treated with calcineurin inhibitors. There was no significant difference in time-to-relapse after rituximab vs calcineurin inhibitor treatment [weighted hazard ratio (HR) 0.90, 95% confidence interval (CI) 0.59-1.38]. There were also no significant differences in relapse rates or kidney function decline. Participants treated with rituximab were at lower risk of developing hypertension (weighted HR 0.24, 95% CI 0.08-0.79).
CONCLUSIONS: Using real-world data, there was no difference in relapse rates after rituximab vs calcineurin inhibitors among children and young adults with steroid-sensitive nephrotic syndrome. Steroid-sparing immunosuppression selection should be a shared decision, considering medication-specific side effects, costs, access, duration and adherence.