Pediatric Rheumatology Online Journal → June 2003 → Medical Treatment → Miscellaneous Medical Treatment→ Abstract #64

THALIDOMIDE: AN OLD DRUG FOR AN OLD PROBLEM

S. K. F. Oliveira

¹Pediatric Rheumatology, Instituto de Puericultura e Pediatria Martagao Gesteira - Universidade Federal do Rio de Janeiro, Rio de Janeiro, RJ, Brazil

Background: Some patients with juvenile idiopathic arthritis are refractory to all medication and follow a troublesome course that impairs a normal physical and emotional development. For many years, thalidomide was banned due to its teratogenic effects, but in recent years, there has been a renewed interest in this drug due to its potential usefulness in the treatment of various diseases. The mechanism of action in these diseases remains unclear, although researchers suspect that effectiveness is due to its inhibitory activity on tumor necrosis factor-alpha (TNF-alpha), modulation of interleukins and/or altered expression of adhesion molecules. Last year, a paper showed that some systemic JIA patients were able to benefit from this drug, opening a new perspective for these refractory cases. Our aim is to report a new patient successfully treated with this drug.
Patient: The patient is 10 years old and has had systemic JIA since the age of 18 months. Her disease was very aggressive in terms of systemic and polyarticular involvement and followed a continuous course. On many occasions she had to take high doses of steroids (oral, pulses) that fortunately didn't seriously compromise her growth or bone density but caused necrosis of her hips. Previously, she had received nonsteroidal anti-inflammatory drugs, methotrexate (doses up to 25 mg/week), ciclosporine (3 mg/kg/day), cyclophosphamide pulses (22 IV pulses), etanercept (4mg/Kg/dose for 15 months), infliximab (4 to 6 mg/Kg/dose - 4 doses in 3 months) without a good control of the disease. The decision to prescribe thalidomide came as the last option before performing an autologous stem cell transplantation (ASCT). The drug was prescribed in a dose of 3 mg/kg/day (100mg/day). Clinical and laboratorial response were excellent and there has been no side effect so far.
Conclusion: We agree that thalidomide is a powerful drug in the treatment of refractory systemic JIA and should be considered before more hazardous procedures such as ASTC.