A Systematic Review of Efficacy and Safety of Ofatumumab Therapy in Children With Difficult-to-treat Nephrotic Syndrome

Context: Different studies have been done so far on drug safety and efficacy in children with refractory nephrotic syndrome (NS). Ofatumumab might be an effective drug for this syndrome; however, the long-term effects and cost-effectiveness of ofatumumab treatment have not been comprehensively assessed.  Objectives: This study aims to perform a systematic review of the efficacy and safety of ofatumumab in children with difficult-to-treat NS. Study Selection: An electronic literature search was conducted to identify appropriate studies. The search key terms were as follows: (“nephrotic syndrome” OR “minimal change disease” OR “focal segmental glomerulosclerosis” OR “membranous”) AND (“Ofatumumab” or “CD۲۰” or “Arzerra” or “HuMax-CD۲۰”).  Data Extraction: Data were extracted from the articles according to the selection criteria by two independent reviewers. Results: A total of ۸۳ potentially relevant articles were identified. Thirty-two articles were removed due to duplication. Then ۲۶ more articles were excluded because they were book sections and review papers and therefore not relevant. Another ۱۴ items were removed after reviewing the full text of selected papers because the topics did not fit our study subject. Finally, ۱۱ studies were selected in our systematic review. The benchmark considered to assess the efficacy of ofatumumab in children with nephrotic syndrome in most of the studies was a complete remission rate. Conclusions: In conclusion, our systematic review showed that ofatumumab may be an effective drug in refractory NS treatment in children and could bring down the use of steroids and immunosuppressants. However, further large randomized trials are suggested.