Pediatric Rheumatology Online Journal → July 2003 → Childhood Lupus→ Neonatal Lupus → Abstract #104

THREE YEAR FOLLOW-UP OF MYCOPHENOLATE MOFETIL THERAPY IN CHILDHOOD SLE CONTINUED EFFECTIVENESS AS A MAINTENANCE AGENT

L. F. Imundo,¹ B. Johnson,¹ L. Ebner-Lyon,² S. C. Li,² Y. Kimura.²

¹Pediatric Rheumatology, College of Physicians and Surgeons, Columbia University, New York, NY; ²Pediatric Rheumatology, Hackensack University Medical Center, University of Medicine and Dentistry of New Jersey, Hackensack, NJ

OBJECTIVES: To determine the long-term tolerability and continued effectiveness of Mycophenolate Mofetil (MMF) in a cohort of pediatric SLE patients.
METHODS: A retrospective chart review of all SLE patients who initiated MMF prior to May 2001.
RESULTS: Eighteen patients were identified (2 had incomplete SLE). At the start of therapy, the average age was 15.3 yrs (5-20), disease duration 3.5 yrs (0.4-11). Active disease manifestations included lupus nephritis (11), CNS symptoms (3), and vasculitis (3). Thirteen patients had received prolonged Cytoxan (CYC) therapy, prior to MMF on average: 17 grams/m. Our first report at 1 year of follow-up indicated stabilization or improvement with MMF in 8/9 patients with improved SLEDAI scores, and urine protein excretion. However, on continued follow-up, 31m (23-40m) 4 patients flared and discontinued MMF, three of these had a lupus nephritis flare, one had new onset of pneumonitis and vasculitis. The average time to flare was 11.2m (5-17). One additional patient failed to respond to MMF. Nine patients continued treatment with improvement in SLEDAI score from 11.6 to 4.3. They were also able to decrease or stop steroids, on average the Prednisone dose dropped from 1.6 to 0.04mg/kg/d. Proteinuria also improved, the protein/creatinine ratio fell from 4.6 to 1.3. Two patients did not tolerate MMF and withdrew at 2.0m (0.5-5); two additional patients were noncompliant and stopped MMF at 17m (12-23) without flaring. Adverse events occurred in the 12/18 patients treated including mild leukopenia (4), uncomplicated herpes zoster (5), abdominal pain (3).
CONCLUSION: Initial experience with MMF in childhood SLE indicates it is well tolerated and appears to be effective as an alternative long-term maintenance agent. Follow-up of 18 patients demonstrates an excellent clinical response in 43% of those treated and treatment failures in 31%. . The therapeutic indications for MMF should be established through further study.