Orencia (abatacept) — an approved medication for juvenile idiopathic arthritis and other inflammatory conditions — is a safe and effective treatment for localized scleroderma in both children and adults, according to a case series and review study.
“This is the first published paper on abatacept as a treatment for localized scleroderma in children in the world, and the first on either children or adults in the United States,” Ioannis Kalampokis, MD, PhD, assistant professor at the University of New Mexico’s division of pediatric rheumatology and the study’s lead author, said in a news story.
The study, “Abatacept in the treatment of localized scleroderma: A pediatric case series and systematic literature review,” was published in the journal Seminars in Arthritis and Rheumatism.
Severe cases of localized scleroderma are often treated with a combination of corticosteroids and methotrexate — widely used for treating inflammatory types of arthritis — but not all patients respond to this approach.
“Once you put out the inflammatory response with steroids early in the treatment, the challenge is to actually keep it down so it never comes back,” Kalampokis said.
Orencia, developed by Bristol Meyers Squibb, is a recombinant or laboratory-made protein that interferes with the activity of T-cells. T-cells are specialized cells of the immune system that help the body fight off bacteria and other invaders. In autoimmune diseases, such as scleroderma, these cells induce chronic inflammation.
Kalampokis and other researchers at the University of New Mexico (UNM) had found abatacept effective in treating inflammation in scleroderma. To further assess the therapy’s benefits in a more systematic approach, the team analyzed eight cases of children treated with Orencia as part of a combo with corticosteroids and mycophenolate or methotrexate. Available literature on Orencia’s use in other cases of localized scleroderma also were examined.
All of the children, who were ages 6 to 18, had been treated with systemic corticosteroids prior to starting on Orencia. “They had basically failed all other treatments,” Kalampokis said.
Five of the eight patients also documented their treatment responses using the Localized Scleroderma Cutaneous Assessment Tool, the modified Localized Scleroderma Skin Severity Index, and the Localized Scleroderma Skin Damage Index.
Over a maximum of 30 months of treatment, all of the participants responded well to Orencia, and showed clinically inactive disease. No new skin lesions formed and none of the existing lesions progressed. In addition, patients whose lesions had caused a loss of motion in the elbows, shoulders, and wrists experienced improvements.
No treatment-related side effects were reported.
The team noted that the lack of a control group in the study — all of the patients received the treatment — makes it impossible to determine if the sustained therapeutic responses were due to Orencia alone or to the combination with methotrexate and mycophenolate.
As for the literature review, the researchers identified three studies on the treatment of localized scleroderma with abatacept, involving a total of 18 adults. In all 15 of the participants were able to complete those studies, which indicates that Orencia was generally well-tolerated, the investigators said.
Among the three patients who did not complete the studies, one had an initial good response followed by a progressive tightening of the skin, fatigue, and tingling sensation in the legs,. The other two stopped treatment due to a diagnosis of breast cancer, deemed unrelated to the therapy, and ulcerative colitis.
As with the UNM patients, the participants who completed the prior studies saw improvement in their scores of skin health.
The fact that the results among the children treated at UNM largely mirrored those of the previously published cases in adults supports the use of Orencia to treat localized scleroderma, the researchers concluded.
“This study is truly a landmark, in that it will share our experience at UNM successfully treating children with scleroderma in this safe and novel way, and will likely help children with this rare disorder around the country,” Kalampokis said.
Still, a double-blind clinical trial using a control group is required to better assess Orencia’s therapeutic value for this indication, according to the scientists.
“Such a future clinical trial will require the collaboration of researchers from multiple centers that will enable a study with adequate power to provide robust conclusions on the effectiveness of abatacept in [localized scleroderma],” they wrote.
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