Objective:
To reduce the risk of relapse in adult and pediatric patients aged 2 years and older with frequently relapsing or steroid-dependent, childhood-onset, idiopathic nephrotic syndrome who are in remission.
Approach:
- Trial Design: The efficacy and safety of obinutuzumab were assessed in INSHORE, a phase 3 randomized open-label, multicenter controlled trial of 85 patients aged 2 years and older.
- Treatment Comparison: Patients were randomly assigned to receive intravenous obinutuzumab or twice-daily oral mycophenolate mofetil.
- Efficacy Assessment: At week 52, the primary efficacy endpoint was assessed using a first-morning urine protein-creatinine ratio threshold of no more than 0.2 g/g.
Key Findings:
- 73% of patients receiving obinutuzumab reached the primary efficacy endpoint compared to a statistically significantly lower percentage of those receiving standard-of-care treatment.
- Obinutuzumab is associated with risks of hepatitis B virus reactivation and progressive multifocal leukoencephalopathy.
- Common side effects include infections, infusion-related reactions, and neutropenia.
Interpretation:
Obinutuzumab has received Breakthrough Therapy, Orphan Drug, and Priority Review designations for this indication.
Limitations:
- Enrollment was limited to patients in complete remission, requiring normal urine protein levels and no swelling.
Conclusion:
Obinutuzumab is now approved for reducing relapse risk in specific pediatric and adult patients with idiopathic nephrotic syndrome.
Sources:
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