Thalidomide, the drug that caused a spate of birth defects back in the 60s, may redeem itself as a treatment for children with a severe form of arthritis - called systemic onset juvenile rheumatoid arthritis, or SoJRA - according to a new study.

“Children with SoJRA often have a poor outcome because of chronic inflammation and corticosteroid side effects,” Dr. Thomas J. A. Lehman, of the Hospital for Special Surgery, in New York, and colleagues write in the Journal of Pediatrics.

“Thalidomide is a unique anti-inflammatory agent,” they explain, that suppresses several inflammatory factors.

The team studied 13 children who had not responded to conventional therapy and were given thalidomide and followed for 6 months. Eleven of the patients had a sustained response to the drug with adequate disease control. Improvement was observed within 4 weeks in most patients.

On average, the children were able to reduce their dose of steroids by about two-thirds over the 6-month period, and six of them were able to discontinue prednisone.

The team saw a decrease in the average number of affected joints - from 19 to 6 - and none of the patients had an increase in their affected joint count.

Only minor side effects were reported. Numbness and tingling were common at the start of treatment, but all the participants were able to continue on treatment at the same or a decreased dosage.

“The risks of the low dosage of thalidomide used in this study are acceptable” when compared with the permanent physical and psychological consequences of prolonged high-dose steroid therapy, Lehman’s group says.

“Further,” they add, “the possible toxicities of thalidomide are small when compared with those of autologous stem cell transplantation and other proposed salvage therapies for severe SoJRA.”

SOURCE: Journal of Pediatrics, December 2004.