In children, idiopathic nephrotic syndrome (NS) generally responds well to treatment with corticosteroids, but long-term use of steroids in children can produce adverse effects (AEs) such as growth impairment and hypertension. Calcineurin inhibitors, too, are associated with AEs, including toxicity and diabetes. Rituximab has been proposed as an option for difficult-to-treat childhood-onset NS, and a recent study evaluated the efficacy and safety of a single dose of rituximab in this setting.
In children, idiopathic nephrotic syndrome (NS) generally responds well to treatment with corticosteroids, but long-term use of steroids in children can produce adverse effects (AEs) such as growth impairment and hypertension. Calcineurin inhibitors, too, are associated with AEs, including toxicity and diabetes. Rituximab has been proposed as an option for difficult-to-treat childhood-onset NS, and a recent study evaluated the efficacy and safety of a single dose of rituximab in this setting.
The study, a multicenter, open-label trial conducted in Korean patients with childhood-onset NS, involved patients aged under 24 years who were either drug-dependent for more than 2 years or who were intolerant to standard treatment.
The study’s design involved a randomized controlled trial (RCT) for drug-dependent patients and a single-arm study for patients whose disease was resistant to conventional treatment. In the RCT, patients were randomly assigned to receive either rituximab (n = 40) or standard treatment (n = 21), and in the single-arm study, all patients (n = 23) received rituximab in addition to their pre-enrollment treatment.
The primary end points of the study were the rate of retaining remission at 6 months in the RCT and the rate of remission within 6 months of starting rituximab in the single-arm study.
In the RCT, at 6 months, 74.3% of rituximab-treated patients were at remission, while 68.7% of standard therapy patients in the control group were in relapse (P = .003). The median duration of remission in the rituximab group was 9 months versus 2.9 months in the standard therapy group.
In the single-arm study, within 6 months of starting rituximab, 9 patients achieved either partial (n = 2) or complete (n = 7) remission.
In all patients treated with rituximab, CD19-positive B cells were depleted within 2 weeks of rituximab treatment. However, relapse after rituximab was not always linked with B-cell recovery; 4 patients had depleted B cells at the time of NS relapse.
In total, 50.8% of patients had infusion reactions to rituximab, and 5 of these patients discontinued infusion (all 5 patients were able to tolerate subsequent rituximab administrations, however). A total of 21 patients reported infections, and 45 experienced any AE. One patient died during the study, but the death was not attributable to rituximab therapy, the investigators concluded.
“Our study laid one more piece of evidence that [rituximab] can be safe and effective in patients with childhood-onset, medication-dependent NS,” wrote the authors, adding that controlled studies are warranted to determine the optimal dosing and interval for rituximab administration in patients with hard-to-treat NS.
Reference
Anh YH, Kim SH, Han KH, et al. Efficacy and safety of rituximab in childhood-onset, difficult-to-treat nephrotic syndrome: a multicenter open-label trial in Korea. Medicine (Baltimore). 2018;97:e13158. doi: 10.1097/MD.0000000000013157.
Biosimilars in America: Overcoming Barriers and Maximizing Impact
July 21st 2024Join us as we explore the complexities of the US biosimilars market, discussing legislative influences, payer and provider adoption factors, and strategies to overcome industry challenges with expert insights from Kyle Noonan, PharmD, MS, value & access strategy manager at Cencora.
Breaking Down Biosimilar Barriers: Payer and PBM Policies
November 13th 2024Part 2 of this series for Global Biosimilars Week dives into the complexities of payer and pharmacy benefit manager (PBM) policies, how they impact biosimilar accessibility, and how addressing these issues may look under a second Trump term.
What AmerisourceBergen's Report Reveals About Payers, Biosimilar Pricing Trends
May 28th 2023On this episode of Not So Different, Tasmina Hydery and Brian Biehn from AmerisourceBergen discussed results from a recent survey, that were also presented at Asembia 2023, diving into the payer perspective on biosimilars and current pricing trends across the US biosimilar industry.
Panelists Stress Stakeholder Education to Build Confidence in Biosimilars
October 31st 2024By expanding educational initiatives to clarify biosimilar safety, efficacy, and interchangeability, stakeholders can foster trust, improve access, and ensure that biosimilars are widely accepted as high-quality, cost-effective alternatives to originator biologics.
Competitive Pricing in Biosimilars: How Adalimumab Could Shape the Industry
Published: October 29th 2024 | Updated: October 29th 2024Sophia Humphreys, PharmD, MHA, BCBBS, of Sutter Health notes that although initial adoption of adalimumab biosimilars remained low in 2023, competitive pricing pressures have already benefited patients and the health care sector.