In a nutshell
This study assessed the safety and effectiveness of tocilizumab (TCZ, Actemra) in patients with systemic juvenile idiopathic arthritis (sJIA). The study concluded that TCZ was safe and effective.
Some background
Arthritis causes pain, stiffness, swelling and restricted movement in the joints. sJIA is a severe form of arthritis that affects children under 16 years old. sJIA affects the whole body, causing fever, rash and painful joints. It also causes inflammation in some organ tissues.
Some steroid drugs used to treat sJIA patients can cause, over the long term, adverse effects such as weight gain, reduced growth, and osteoporosis (weak and brittle bones).
TCZ is a drug that works by suppressing the immune system. It was approved for treatment of sJIA in Japan in 2008 and in the European Union and USA in 2011. However, the effects of this treatment in patients outside of a controlled clinical trial are still not clear.
Methods & findings
This study examined the effects of TCZ in patients with sJIA outside of clincial trials.
The study followed 417 children (52% males and 48% females) for 52 weeks or until they stopped taking TCZ. These patients received 8 mg/kg doses once every two weeks. Doses were changed depending on the severity of the illness. 86.6% of patients took TCZ until the end of the 52 weeks.
Fever symptoms reduced from 54.6% to 5.6% over the year, and rash symptoms reduced from 43% to 5.6%. After 52 weeks, 99% of patients had normal C-reactive protein levels (a protein indicating there is inflammation in the body).
69.3% of patients had adverse events, which were mainly infections (41%) and respiratory conditions (19.7%). 28.1% of patients had severe adverse events, which were mainly infections (13.2%) and blood conditions (8.2%).
The bottom line
The study concluded that TCZ was safe and effective in patients with sJIA. The high levels of serious adverse events may be due to differences between the patients from previous studies and those in this study (medication use and other illnesses).
The fine print
This observational study was not a clinical trial and did not have a control group for comparison.
What’s next?
If you have sJIA, or have a child with sJIA, you should discuss the results of this study with your doctor.
Published By :
Annals of the rheumatic diseases
Date :
Dec 07, 2015
