title: Efficacy of mycophenolate mofetil in adolescent patients with lupus nephritis: evidence from a two-phase, prospective randomized trial.
abstract: The safety and efficacy of mycophenolate mofetil (MMF) were evaluated in adolescent patients with systemic lupus erythematosus and active or active/chronic class III-V lupus nephritis. During the 24-week induction phase, patients were randomized to oral MMF (target dose 3.0 g/day) or intravenous cyclophosphamide (IVC) (0.5-1.0 g/m(2)/month), plus prednisone. Response was defined as a decrease in 24-hour urine protein:creatinine ratio (P:Cr) to < 3 in patients with baseline nephrotic range proteinuria, or by ≥ 50% if subnephrotic baseline proteinuria, and stabilization (± 25%) or improvement in serum creatinine. In the 36-month maintenance phase, induction therapy responders were randomized 1:1 to MMF (1.0 g twice daily) or oral azathioprine (AZA) (2 mg/kg/day), plus prednisone. In the induction phase, 10 patients received MMF and 14 received IVC; 15 (62.5%) achieved treatment response (MMF, 7 (70%); IVC, 8/15 (57.1%); p = 0.53, odds ratio (95% confidence interval) 2.0 (0.2, 15.5)). There was a non-statistically significant difference in maintenance of response to MMF (7/8; 87.5%) versus AZA (3/8; 37.5%). Seven patients withdrew (MMF, 2; AZA, 5). During both phases, rates of serious adverse events were similar in both arms. During both phases treatment response with MMF was as effective as the comparator.
authors.affiliation: Boston Children's Hospital, Boston, MA 02115, USA.
authors.forename: R
authors.initials: R
authors.lastname: Sundel
authors.forename: N
authors.initials: N
authors.lastname: Solomons
authors.forename: L
authors.initials: L
authors.lastname: Lisk
mesh_terms: D000284:Administration, Oral; D000293:Adolescent; D000367:Age Factors; D001208:Asia; D001379:Azathioprine / Q000627:therapeutic use; D015415:Biomarkers / Q000097:blood; D003404:Creatinine / Q000097:blood; D003520:Cyclophosphamide / Q000627:therapeutic use; D004311:Double-Blind Method; D004359:Drug Therapy, Combination; D005060:Europe; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D008181:Lupus Nephritis / Q000097:blood / Q000150:complications / Q000175:diagnosis / Q000188:drug therapy*; D008297:Male; D009173:Mycophenolic Acid / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D016017:Odds Ratio; D011241:Prednisone / Q000627:therapeutic use; D011446:Prospective Studies; D011507:Proteinuria / Q000188:drug therapy / Q000209:etiology; D013020:South America; D013997:Time Factors; D016896:Treatment Outcome; D014481:United States
chemical_list: D015415:Biomarkers; D007166:Immunosuppressive Agents; D003520:Cyclophosphamide; D003404:Creatinine; D009173:Mycophenolic Acid; D001379:Azathioprine; D011241:Prednisone