Utilization of anti-CD20 antibodies for treatment of childhood nephrotic syndrome, 2010 to 2022.

Background: A growing body of evidence supports the efficacy of the type I anti-CD20 monoclonal antibody, rituximab, in the management of children with frequently relapsing or steroid-dependent nephrotic syndrome. We examined temporal trends and described current patterns in the use of anti-CD20 antibodies and other corticosteroid-sparing drug therapies in a large multi-institutional population of children with nephrotic syndrome.

Methods: Data came from PEDSnet, a clinical research network that aggregates electronic health record data at several children's healthcare organizations in the United States. Patients with at least one inpatient, emergency, or outpatient physician encounter between January 2010 and November 2022 who met our published computable phenotype algorithm for nephrotic conditions were included. Children with systemic lupus erythematosus or congenital/genetic nephrotic diagnoses were excluded. Treatments were measured from nephrotic syndrome diagnosis to kidney transplant or most recent encounter.

Results: Among 6,892,137 patients across 6 centers, 2962 met criteria for nephrotic conditions (0.4 per 1000 patients). 852 (28.8%) had at least one native kidney biopsy. Nearly half of the population was exposed to at least one steroid-sparing agent, most of whom had exposure to multiple agents. 524 (17.7%) patients were exposed to rituximab, and utilization of rituximab increased over the 12-year study period. Similar trends were observed for mycophenolate and tacrolimus. Concurrently, use of cyclosporine and cyclophosphamide decreased.

Conclusion: Use of rituximab to manage nephrotic syndrome has steadily increased, and tacrolimus, mycophenolate, and rituximab are currently the most commonly used steroid-sparing agents for childhood nephrotic syndrome.