Pediatric Rheumatology Online Journal → June 2003 → Medical Treatment → Miscellaneous Medical Treatment→ Abstract #63

NEORAL REGISTRY IN POLYARTICULAR JUVENILE RHEUMATOID ARTHRITIS

E. H. Giannini,¹ N. Ruperto,² A. L. Tomasi,¹ V. Gerloni,² R. Hafner,² S. Nielsen,² J. Pratsidou,² the PRCSG and PRINTO

¹Rheumatology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH; ²Pediatria II - PRINTO, IRCCS G. Gaslini, Genova, Italy

Objectives: To determine the health outcomes of children given Neoral (oral formulation of cyclosporine) for the management of polyarticular juvenile rheumatoid arthritis (JRA).
Background: Neoral is approved by the US FDA for use in adult RA. Despite the lack of blinded, controlled studies in children with arthritis, it is commonly prescribed "off label" for JRA, particularly in Europe.
Methods: This ongoing Phase IV registry is focused primariliy on drug safety. We report here data gathered through November, 2002. On-study visits occur every 6 months.
Results: 339 pts have been enrolled (489 pt yrs of follow-up) who have had 1171on-study visits. 54% had systemic, 32% had polyarticular, and 14% had pauciariticular onset JRA. Median time on drug at the time of the last reported visit was 1.3 yrs (range 0.1-7.7 yrs). Median dose was 3.2 mg/kg/d. Safety: 178 (53%) have dc/ed the agent after a median time on drug of 1.4 yrs (range 1-92 mos.) Chief reason for d/c was inefficacy (108). Adverse events (AEs) were listed as the 1 or 2 reasons for d/c in 40 pts, including 21 for hypertension and/or elevated creatinine, 6 for hirsutism, 4 for GI intolerance, 2 for seizures, 2 for thrombocytopenia, and 5 for other reasons. Effectiveness: At the last reported visit among those still receiving Neoral (161), the MD assessment of disease activity was classified as mild or moderate in 112, severe in 35, inactive in 14. Continuing disease activity was the most frequent reason for continuing Neoral. Clinical course after d/c (more than one answer allowed) included little change in clinical picture (79), decrease in disease activity (34), return of disease activity (32), resolution of AEs (26), persistence of AEs (2), unknown or alternate therapy (26).
Conclusions: Neoral is given for JRA for relatively short periods before d/c chiefly due to inefficacy. Renal complications occur in a substantial proportion of patients, although resolve quickly upon d/c of the agent.