Rituximab for troublesome cases of childhood nephrotic syndrome

Abstract

Nephrotic syndrome (NS) is the most common glomerular disease of childhood. Steroid-dependent and steroid-resistant nephrotic syndrome present challenges in their pharmaceutical management; patients may need several immunosuppressive medication for optimum control, each of which medication has its own safety profile. Rituximab (RTX) is a monoclonal antibody that targets B cells and has been used successfully for management of lymphoma and rheumatoid arthritis. Recent clinical studies showed that rituximab may be an efficacious and safe alternative for the treatment of complicated nephrotic syndrome. In this review article, we aim to review the efficacy and safety of RTX therapy in nephrotic syndrome. We reviewed the literature pertaining to this topic by searching for relevant studies on PubMed and Medline using specific keywords. The initial search yielded 452 articles. These articles were then examined to ensure their relevance to the topic of research. We focused on multicenter randomized controlled trials with relatively large numbers of patients. A total of 29 articles were finally identified and will be summarized in this review. The majority of clinical studies of RTX in complicated pediatric NS showed that rituximab is effective in approximately 80% of patients with steroid-dependent NS, as it decreases the number of relapses and steroid dosage. However, RTX is less effective at achieving remission in steroid-resistant NS. RTX use was generally safe, and most side effects were transient and infusion-related. More randomized, double-blinded clinical studies are needed to assess the role of RTX in children with nephrotic syndrome.

Keywords: Rituximab, Pediatric, Nephrotic syndrome, Efficacy, Safety

Core tip: Nephrotic syndrome (NS) is the most common pediatric glomerular disease. Although outcomes are favorable, the treatment of complicated nephrotic syndrome can be challenging. Rituximab (RTX) offers a safe and effective alternative to current immunosuppressive therapies for complicated cases of NS. The best outcomes are seen in patients with steroid-dependent NS who have failed to respond to multiple therapies. However, the benefits of RTX therapy are limited in patients with steroid-resistant nephrotic syndrome. Successful RTX therapy induces prolonged remission and enables discontinuation of other medications without increasing the risk of infection and other adverse events.