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Experience with mycophenolate mofetil as maintenance therapy in five pediatric patients with severe systemic lupus
1Department of Pediatric Nephrology, University of Erlangen-Nürnberg, Germany.
Abstract:
Maintenance therapy of severe pediatric systemic lupus erythematosus (SLE) usually consists of azathioprine and prednisone . In adult SLE patients mycophenolate mofetil (MMF) is successfully used, superiority to azathioprine has not been shown yet. We hypothesized that a maintenance therapy with MMF is able to decrease disease activity as well as the dose of glucocorticoid needed in children and adolescents with SLE. Five girls with a mean age of 13.9 (range 12-15) years were treated with 1.2+/-0.20 g/m (2) MMF daily on individual medical decision. Three patients had severe renal (WHO IV) and one severe cerebral involvement. Three patients with frequent flares on azathioprine maintenance therapy were switched to MMF, two patients with severe renal and cerebral manifestation received MMF additionally after induction therapy. Flares, steroid dosage, and disease activity (SLEDAI) were monthly registered in all patients. The number of flares decreased from 1.28 to 0.25 episodes per patient year during a mean follow-up period of 39 (range 36-42) months after MMF initiation. In parallel prednisone dose could be reduced from 10.80+/-5.25 to 3.25+1.18 mg/d (p<0.01). SLEDAI score dropped from 15.20+/-2.8 before MMF to 3.60+/- 0.9 at the last visit under MMF (p<0.001). No severe adverse event occurred. In our cohort of five pediatric patients MMF was effective and safe for maintenance therapy of SLE over a period of 3.5 years. MMF seems to be successful in preventing flares even in adolescents having unfavorable course on azathioprine treatment before. This observation should be confirmed by a randomized multicenter clinical trial.
Insights
Mycophenolate mofetil (MMF) effectively reduced disease activity and prednisone dosage in pediatric patients with severe systemic lupus erythematosus (SLE). MMF proved safe and beneficial for maintenance therapy, decreasing flares in children and adolescents.
Area of Science:
- Pediatric Rheumatology
- Immunology
- Clinical Pharmacology
Background:
- Systemic lupus erythematosus (SLE) in children often requires aggressive maintenance therapy.
- Current standard maintenance therapy involves azathioprine and prednisone.
- Mycophenolate mofetil (MMF) is used in adults, but its efficacy and safety in pediatric SLE maintenance are less established.
Purpose of the Study:
- To evaluate the efficacy of MMF as maintenance therapy in pediatric SLE.
- To assess MMF's ability to decrease disease activity and glucocorticoid requirements.
- To investigate MMF's safety profile in children and adolescents with SLE.
Main Methods:
- A cohort of five pediatric SLE patients received daily MMF (1.2+/-0.20 g/m^2).
- Patients included those with severe renal or cerebral involvement, or frequent flares on azathioprine.
- Disease activity (SLEDAI), flares, and prednisone dosage were monitored monthly over a mean follow-up of 39 months.
Main Results:
- The number of flares decreased significantly from 1.28 to 0.25 episodes per patient-year.
- Prednisone dosage was reduced from 10.80+/-5.25 mg/d to 3.25+/-1.18 mg/d (p<0.01).
- Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) scores dropped from 15.20+/-2.8 to 3.60+/-0.9 (p<0.001).
Conclusions:
- MMF demonstrated effectiveness and safety as maintenance therapy for severe pediatric SLE over 3.5 years.
- MMF successfully prevented flares, even in patients with a history of azathioprine treatment failure.
- Further confirmation through randomized multicenter clinical trials is warranted.
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