Not uncommonly, some children with systemic-onset juvenile rheumatoid arthritis (JRA) have persistently active disease with joint destruction and profound growth delay despite maximum treatment with known medications. Based on previous observations of improvement in synovitis following intravenous (I.V.) cyclophosphamide (CYC) and methylprednisolone (MP) treatments, a group of children with severe systemic-onset JRA was treated in an attempt to control active synovitis and to allow tapering of corticosteroids.
Four patients with systemic-onset JRA were continued on a daily regimen of nonsteroidal antiinflammatory agents and prednisone, with a weekly subcutaneous dose of methotrexate (1 mg/kg). In addition, 1 patient continued receiving sulfasalazine and 1 patient remained on a regimen of sulfasalazine and hydroxychloroquine. Patients received 6-10 monthly treatments of I.V. CYC (500-1,000 mg/m2) and MP (30 mg/kg; 1 gm maximum) accompanied by I.V. mesna and large amounts of I.V. fluids. Subsequent treatments were given once every 2-3 months.
After 12-20 I.V. pulses of CYC, all patients showed improvement, and 3 achieved remission of disease. All were able to discontinue corticosteroid use and all had an increase in linear growth.
Monthly I.V. pulse CYC treatments can be useful to control disease in selected children with severe, destructive JRA.