Related Experiment Videos
Intensive pulse therapies for focal glomerulosclerosis in South African children
M Adhikari1, R Bhimma, H M Coovadia
1Department of Paediatrics and Child Health, University of Natal, Faculty of Medicine, Congella, South Africa.
Insights
Two treatment regimens for steroid-resistant focal segmental glomerulosclerosis (SR-FGS) in children were compared. Regimen B, a shorter, less intensive protocol, showed comparable efficacy to Regimen A but was more cost-effective and required fewer hospital visits.
Area of Science:
- Pediatric Nephrology
- Immunosuppressive Therapy
- Glomerular Diseases
Background:
- Steroid-resistant focal segmental glomerulosclerosis (SR-FGS) is a challenging kidney disease in children.
- Optimal immunosuppressive treatment protocols for SR-FGS remain under investigation.
Purpose of the Study:
- To compare the efficacy and safety of two different immunosuppressive regimens for pediatric SR-FGS.
- To evaluate the cost-effectiveness and healthcare resource utilization of the two treatment protocols.
Main Methods:
- Retrospective analysis of two treatment regimens (A and B) in children with SR-FGS.
- Regimen A: 16-month protocol with methylprednisolone, oral prednisone, and cyclophosphamide.
- Regimen B: 6-month protocol with intravenous cyclophosphamide, intravenous methylprednisolone, and oral prednisone.
Main Results:
- Both regimens improved edema, reduced proteinuria, resolved hematuria, and increased estimated GFR in responding children.
- Regimen B achieved complete remission in 2/5 patients and partial remission in 1/5, with a shorter follow-up and fewer hospital visits.
- Regimen A resulted in one short remission and improvement in others, with longer treatment duration and higher costs.
Conclusions:
- Shorter, less intensive immunosuppressive protocols like Regimen B may offer comparable benefits to longer protocols for pediatric SR-FGS.
- Regimen B demonstrates significant cost-effectiveness and reduced healthcare burden compared to Regimen A.
- Further multicenter controlled trials are warranted to establish optimal management strategies for SR-FGS.
Abstract:
Seven children with steroid-resistant focal segmental glomerulosclerosis (SR-FGS) were placed on a therapeutic protocol of methylprednisolone (MP), oral prednisone (pred) and oral cyclophosphamide (CYC) given over 16 months (regimen A). Another 5 children with SR-FGS were treated with a shorter course of intravenous CYC (monthly doses over 6 months), intravenous MP (3 consecutive daily doses) and oral pred 2 mg/kg (alternate days) (regimen B). With regimen A, 1 child had a short remission, and in the others, oedema subsided, the urine protein/ creatinine ratio decreased, haematuria disappeared and the estimated glomerular filtration rate (GFR) increased. The observation period was 21-42 months and the drugs were well tolerated. With regimen B, 2 patients went into complete remission, 1 had partial remission, 1 failed to respond and another died because of severe concurrent infections. In the responding children, oedema cleared, the urine protein/ creatinine ratio decreased, haematuria disappeared and the GFR rose. The follow-up was between 3 and 34 months. Minor side effects were alopecia and transient hypertension. Both regimens improved the quality of life of most children. Compared with regimen A, regimen B is six times less costly with a quarter of the number of hospital visits. These observations may be of value in designing appropriate multicentre controlled trials, which have been advocated recently, for the rational and optimum management of SR-FGS.