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Favourable renal survival in paediatric microscopic polyangiitis: efficacy of a novel treatment algorithm
Biswanath Basu1, T K S Mahapatra2, Nirmal Mondal3
1Division of Pediatric Nephrology, Department of Pediatrics, NRS Medical College & Hospital, Kolkata, West Bengal 700014, India.
Background:
Microscopic polyangiitis (MPA) is one of the most common forms of antineutrophil cytoplasm autoantibodies (ANCA)-associated vasculitis in children. Cyclophospamide and glucocorticoid-based treatment protocols are still considered gold standard in managing this multi-system disorder. But treatment-related toxicity is a major cause of chronic morbidity and early mortality in MPA. Hence, the search for an effective and safe alternative immunosuppressant is essential.
Methods:
A retrospective analysis of baseline clinico-pathological presentation and treatment-outcome was performed among 11 paediatric MPA patients. All of whom were treated with a pre-specified cyclophosphamide free, rituximab- and mycophenolate mofetil (MMF)-based management protocol as per centre practice.
Results:
We describe the clinical course of 11 children with MPA over a median follow-up period of 20.9 months. Both patient survival and renal survival at 1 year follow-up were 100%. In spite of the varying degree of renal involvement at presentation, kidney function was recovered in all patients with a median estimated glomerular filtration rate (eGFR) of 79.5 mL/min/1.73 m(2). At last follow-up, 91% (10/11) of patients were in complete remission and one (9%) child continued partial remission state. There was no treatment failure. In total, 73% (8/11) of patients were off steroids at last follow-up and 82% (9/11) of patients never relapsed during follow-up period.
Conclusions:
Efficacy and medium-term safety of rituximab- and MMF-based protocol in managing children with MPA was evident in this study.
Insights
A new treatment protocol using rituximab and mycophenolate mofetil (MMF) shows promising results for pediatric microscopic polyangiitis (MPA). This cyclophosphamide-free approach achieved high remission and survival rates in children with ANCA-associated vasculitis.
Area of Science:
- Pediatric Rheumatology
- Immunology
- Nephrology
Background:
- Microscopic polyangiitis (MPA) is a common pediatric ANCA-associated vasculitis.
- Current gold standard treatments (cyclophosphamide, glucocorticoids) carry significant toxicity risks.
- There is a critical need for safer, effective immunosuppressive alternatives in pediatric MPA.
Purpose of the Study:
- To evaluate the efficacy and safety of a rituximab and mycophenolate mofetil (MMF)-based protocol in pediatric MPA.
- To assess outcomes in children with MPA treated with a cyclophosphamide-free regimen.
Main Methods:
- Retrospective analysis of 11 pediatric MPA patients.
- Treatment utilized a pre-specified cyclophosphamide-free protocol with rituximab and MMF.
- Evaluated clinico-pathological presentation and treatment outcomes.
Main Results:
- 100% patient and renal survival at 1-year follow-up.
- Complete kidney function recovery in all patients (median eGFR 79.5 mL/min/1.73 m²).
- 91% achieved complete remission, 73% were off steroids, and 82% had no relapses at last follow-up.
Conclusions:
- The rituximab and MMF protocol demonstrated significant efficacy in managing pediatric MPA.
- This cyclophosphamide-free regimen showed favorable medium-term safety and effectiveness.
- The findings support rituximab and MMF as a viable alternative for pediatric MPA treatment.
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