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Oral cyclophosphamide therapy in 100 children with steroid-sensitive nephrotic syndrome: experience from a developing
Jasjeet Sandhu1, Deepak Bhat1, Gurdeep Singh Dhooria2
1Department of Pediatrics, Dayanand Medical College and Hospital, Ludhiana, Punjab, India.
Insights
Oral cyclophosphamide (CYP) effectively treats steroid-dependent nephrotic syndrome. Children over 5 years old initiating CYP treatment showed a better response, indicating age is a key factor for treatment success.
Area of Science:
- Pediatric Nephrology
- Immunosuppressive Therapy
Background:
- Steroid-sensitive nephrotic syndrome (NS) with steroid-dependent (SD) or frequent relapsing (FR) courses often requires immunosuppressive agents.
- Oral cyclophosphamide (CYP) is a key therapeutic option for these challenging NS cases.
Purpose of the Study:
- To evaluate the response rate to oral cyclophosphamide (CYP) in children with SD or FR nephrotic syndrome.
- To identify factors associated with a positive treatment response to oral CYP.
Main Methods:
- Retrospective observational study of 100 children with SD or FR NS treated with oral CYP.
- Response defined as steroid cessation for at least 6 months post-CYP.
- Relapse-free survival analyzed using Kaplan-Meier method.
Main Results:
- Fifty percent of patients achieved responder status at 6 months.
- Relapse-free survival was 31% at 1 year and 11% at 2 years post-CYP.
- Age at NS onset >3 years and age at CYP initiation >5 years were significant predictors of good response.
Conclusions:
- Judicious patient selection is crucial for optimizing oral CYP therapy in nephrotic syndrome.
- Initiating CYP after 5 years of age independently predicts a better treatment response.
- Age at onset of NS >3 years also correlates with improved outcomes.
Background:
Oral cyclophosphamide (CYP) is an important therapeutic agent in treatment of steroid-sensitive nephrotic syndrome having a steroid-dependent (SD) or frequent relapsing (FR) course. This retrospective observational study aimed to determine response to oral CYP and factors associated with positive response in these patients.
Methods:
We studied 100 children (male; 75) with FR (19%) and SD (81%) NS treated with CYP in the Pediatric Nephrology clinic. Responders were defined as children in whom steroids were stopped for at least 6 months following CYP and factors affecting response were analysed. Relapse-free survival was estimated by Kaplan-Meier method.
Results:
Median age at onset of NS was 3 years (IQR 2-5.2). Median age at CYP was 5.7 years (IQR 3.7-7.9). Fifty percent of patients were in the responder group at 6 months of CYP. Relapse-free survival post CYP therapy was 31% at 1 year, 11% at 2 years. Factors predicting good response were age at onset of NS > 3 years with 61.2% response at 6 months (p = 0.028) and older age at CYP initiation (> 5 years) with 61% response (p = 0.008). Multivariate regression analysis showed age at start of CYP > 5 years was an independent factor for good response (p = 0.044, OR = 2.903, CI -1.03 to 8.18).
Conclusions:
Judicious selection of patients, especially with age of onset of NS more than 3 years and initiation of CYP after age of 5 years, can predict better response in this group of patents.
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