New Rheumatoid Arthritis Drug Works for Adults, Children

In the second report, Japanese researchers, led by Dr. Shumpei Yokota, of the department of pediatrics at Yokohama City University School of Medicine, started 56 children, ages 2 to 19, on tocilizumab. These children all had systemic-onset juvenile idiopathic arthritis, which did not respond to the usual arthritis treatment. This is a common problem with this type of arthritis, the researchers noted.

After six weeks, children who had achieved a 30 percent reduction in their arthritis symptoms were randomly assigned to continue to receive tocilizumab or a placebo.

Of the 43 children in that phase of the study, 16 out of 20 who received tocilizumab continued the improvement they had made in the first phase of the trial, compared with only four of 23 children receiving a placebo, the researchers found.

During an additional 48 weeks in which 48 children continued to receive tocilizumab, 47 children achieved a 30 percent reduction in their symptoms, 45 children achieved a 50 percent reduction in their symptoms, and 43 children ultimately achieved a 70 percent reduction in their symptoms, according to the report.

Side effects included gastrointestinal bleeding, bronchitis, and gastroenteritis.

"The results of this placebo-controlled and open-label extension study with tocilizumab in children with systemic-onset juvenile idiopathic arthritis show a sustained clinical improvement and a favorable risk-benefit profile," the Japanese team wrote. "The findings of this study might represent a step forward in the control of a disease that has previously proved to be difficult to manage."

The study was funded by Chugai Pharmaceuticals.

Bongartz said tocilizumab may be worth a try with pediatric patients.

"The treatment options are pretty sparse, and children usually don't respond very well to methotrexate or other drugs, so here I think it's easier to decide in favor of initiating tocilizumab therapy," Bongartz said. "My threshold for initiating tocilizumab therapy would be lower, because I can't offer these children a lot of other choices."

New treatment options against rheumatoid arthritis are always welcome, Bongartz said. Still, he is cautious about using tocilizumab.

"It's another opportunity to offer treatment to patients who don't respond to first-, second- or third-line therapy, but I don't know if it works better than other established therapies. Based on the data available, it's almost impossible to make an informed decision about its benefits or potential harm," Bongartz said.

More information

For more on arthritis, visit the Arthritis Association.

SOURCES: Tim Bongartz, M.D., department of internal medicine, division of rheumatology, Mayo Clinic College of Medicine, Rochester, Minn.; March 22, 2008,The Lancet