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Dynamic Imaging of Chimeric Antigen Receptor T Cells with [18F]Tetrafluoroborate Positron Emission Tomography/Computed Tomography
Published on: February 17, 2022
Rituximab/Mycophenolate Combination Therapy in Children with Calcineurin Inhibitor-Resistant FSGS
Saumil Gaur1, Partha P Paul2, Mounika Motamarri2
1Consultant Paediatrician and Transplant Nephrologist, Rainbow Children Hospital, Marathahalli, Bangalore, Karnataka, India.
Insights
Dual therapy with rituximab and mycophenolate offers a promising treatment for children with calcineurin inhibitor-resistant focal segmental glomerulosclerosis (FSGS). This approach successfully maintained remission in most patients, showing efficacy in a challenging pediatric kidney disease.
Area of Science:
- Pediatric Nephrology
- Immunosuppressive Therapy
- Glomerular Diseases
Background:
- Focal segmental glomerulosclerosis (FSGS) in children presents limited therapeutic options, particularly CNI-resistant forms.
- Calcineurin inhibitors (CNIs) like tacrolimus and cyclosporine are standard but not universally effective.
- Novel strategies are needed for pediatric FSGS unresponsive to CNI treatment.
Purpose of the Study:
- To evaluate the efficacy of a dual therapy regimen in children with CNI-resistant FSGS.
- To assess the ability of rituximab and mycophenolate combination therapy to maintain remission.
- To analyze treatment outcomes and safety in this specific pediatric population.
Main Methods:
- Retrospective analysis of 13 children with CNI-resistant FSGS.
- Treatment involved dual therapy with rituximab and mycophenolate for at least 1 year.
- Outcomes measured included sustained remission, renal function, and adverse events.
Main Results:
- 84.6% of children achieved sustained remission at 1 year post-dual therapy.
- The therapy was effective in both steroid-dependent and steroid-resistant FSGS cases.
- No adverse reactions or infections were reported, with preserved renal function.
Conclusions:
- Dual rituximab and mycophenolate therapy is a promising and effective strategy for CNI-resistant pediatric FSGS.
- This combination can achieve sustained remission in a significant majority of treated children.
- The treatment demonstrates a favorable safety profile with preserved kidney function.
Introduction:
There is a paucity of data and therapeutic options nationally and internationally on calcineurin inhibitor (CNI)-resistant forms of focal segmental glomerulosclerosis (FSGS) in children. CNI (tacrolimus or cyclosporine) are proven monotherapy in children with FSGS with a steroid-dependent (SD) or steroid-resistant (SR) course. We analyzed a novel therapeutic option in CNI-resistant FSGS by using the dual therapy of rituximab and mycophenolate to maintain remission.
Methods:
This is a retrospective analysis of clinical, therapeutic profile, and treatment outcomes (sustained remission versus no remission) in subjects with CNI-resistant FSGS who received dual rituximab therapy along with mycophenolate as maintenance therapy for a minimum of 1 year.
Results:
The median age of presentation of 13 recruited children was 7.8 years (range: 2.4-17.6 years); nine (69.2%) were males. Ten (76.9%) of them had an SD course and three (23.1%) had an SR course. Four (30.7%) had evidence of acute/chronic CNI toxicity, and the remaining nine (69.3%) showed no response to CNI therapy despite adequate trough levels. Post dual therapy, 11 (84.6%) had sustained remission for at 1 year and two (15.4%) children did not show remission. None reported adverse reactions or infections, and all had preserved renal functions.
Conclusion:
Dual combination therapy with rituximab and mycophenolate among children with CNI-resistant FSGS can emerge as a promising and efficacious treatment strategy to ensure sustained remission in this subset of patients.
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