Investigators from multiple institutions in the United Kingdom conducted a randomized controlled trial to compare the effectiveness of a prolonged tapering regimen of prednisolone to the standard regimen in childhood nephrotic syndrome. Study participants were children 1–14 years old with their first episode of steroid-sensitive nephrotic syndrome. Eligibility requirements included a ratio of first morning urine protein (or albumin) to creatinine >200 mg/mmol, serum or plasma albumin level <25 g/L, and no previous treatment with corticosteroids. All study patients were treated with prednisolone 60 mg/m2 daily for 4 weeks at which point they were randomized to complete a course of prednisolone, including taper, for a total of 8 weeks (standard treatment), or a total of 16 weeks of treatment with a more prolonged taper. Patients were followed up for 24–48 months. The primary study outcome was time to first relapse, with a hazard ratio calculated using a Cox proportional hazards model to assess differences between treatment groups. Secondary outcomes included total number of relapses in study children and need for treatment with immunosuppressive drugs. Incident rate ratios and relative risk were used to compare these outcomes among children in the 2 treatment groups. Data on health care costs were collected in study participants to allow for a cost comparison between treatment groups.
Data were analyzed in 223 children with steroid-sensitive nephrotic syndrome: 114 randomized to the 16-week treatment course and 109 to standard treatment. The mean (standard deviation) age of study participants was 4.9 (3.1) years, and 65% were boys. Baseline characteristics did not differ between the 2 treatment groups. For the primary outcome of time to first relapse, there was no significant difference between the 2 treatment groups. The median time to first relapse was 139 days for those in the 16-week treatment group and 87 days for those receiving standard treatment (hazard ratio, 0.87; 95% CI, 0.65–1.17). Among those in the 16-week treatment group, 80% had at least one relapse versus 81% of patients randomized to standard treatment; the mean number of relapses in children in the 2 groups were 3.98 and 3.61, respectively (incident rate ratio, 1.09; 95% CI, 0.86–1.39). However, 16-week treatment was associated with an estimated cost savings of $2,160 (95% CI, 109–3,455).
The authors conclude that 16 weeks of prednisolone treatment for childhood nephrotic syndrome was not better than 8 weeks of treatment but was associated with some cost savings.
Dr Sanchez-Kazi has disclosed no financial relationship relevant to this commentary. This commentary does not contain a discussion of an unapproved/investigative use of a commercial product/device.
Nephrotic syndrome is a common glomerular disease in childhood. Most children respond to corticosteroids, but the optimal length of initial therapy is poorly defined.1–4 Several combinations...