The FDA ( Food and Drug Administration ) has approved Actemra ( Tocilizumab ), given alone or in combination with Methotrexate, for the treatment of active systemic juvenile idiopathic arthritis ( SJIA ) in children ages 2 years and older.

Systemic juvenile idiopathic arthritis, or Still’s disease, is a rare, potentially life-threatening disorder in children that causes severe inflammation throughout the body. is Systemic juvenile idiopathic arthritis distinguished from other forms of juvenile idiopathic arthritis ( JIA ) by the prominence of systemic and inflammatory features, including spiking fevers; rash; swelling and inflammation of lymph nodes, liver, and spleen; and high white blood cell and platelet counts.
The prevalence of juvenile idiopathic arthritis is an estimated 1 to 2 per 1,000 children, and systemic juvenile idiopathic arthritis affects about 10 percent of all juvenile idiopathic arthritis patients.

Actemra is an interleukin-6 ( IL-6 ) receptor blocker approved by the FDA in 2010, for treatment of adults with moderately to severely active rheumatoid arthritis who have had an inadequate response to other approved therapies.

An international, multicenter controlled trial demonstrated the safety and effectiveness of Actemra, in which 112 patients received either Actemra infusions or placebo infusions every two weeks. Study participants included patients with systemic juvenile idiopathic arthritis aged 2 to 17 years old who had inadequate response to or who were unable to take nonsteroidal anti-inflammatory drugs ( NSAIDs ) and corticosteroids.

Eighty-five percent of those receiving Actemra responded to treatment, compared with 24% of patients receiving placebo. Response was defined as at least 30% improvement in the American College of Rheumatology’s juvenile idiopathic arthritis efficacy variables, along with absence of fever in the preceding seven days. In the long-term, follow-up period of the trial there were three cases of macrophage activation syndrome among systemic juvenile idiopathic arthritis patients receiving Actemra.
Macrophage activation syndrome is a potentially fatal complication of childhood systemic inflammatory disorders, thought to be caused by excessive activation and proliferation of certain immune cells.

Actemra carries a Boxed Warning for serious infections. Patients treated with Actemra who develop a serious infection should stop Actemra treatment until the infection is controlled.

Changes in certain laboratory test results such as liver tests, blood counts, and cholesterol are not uncommon with Actemra and should be monitored with regular blood tests. The most common side effects in trial participants with systemic juvenile idiopathic arthritis included upper respiratory tract infection, headache, sore throat, and diarrhea.

Source: FDA, 2011

XagenaMedicine2011


Link: Xapedia - Medical Encyclopedia