Researchers have made a breakthrough in the treatment of a rare autoimmune disease that affects children.
A UK study has revealed that tumour necrosis factor inhibitor (anti-TNF) treatment is effective at improving muscle and skin health in children with juvenile dermatomyositis (JDM).
JDM, a rare chronic autoimmune disease that causes the body to attack the immune system, causes rash, skin ulceration and muscle weakness.
Although many of the youngsters who are diagnosed with the disease are able to lead normal lives, some fail to respond to treatments and that could result in complications, such as scarring, trapped nerves, and shortening of the muscles causing joints to stay bent, or an increased risk of death.
Presenting the findings at the European League Against Rheumatism Annual Congress (EULAR 2016), Dr Raquel Campanilho-Marques of the Institute of Child Health, University College London, UK, reported that 66 patients with JDM who were treated with anti-TNF agents showed significant improvements in muscle and skin involvement, as well as in overall disease activity.
She told delegates that there were significant changes in the median values of two standard muscle measurements: the Childhood Myositis Assessment Scale and Manual Muscle Testing (p<0.0001 and p=0.0097 respectively).
There were also significant improvements in skin involvement assessed using the modified skin Disease Activity Score (p<0.0001). Global disease activity also improved significantly (p<0.0001).
“High levels of the cell signalling protein TNF have been reported in JDM patients with a long disease course, suggesting this immune cell regulator may play a significant role in refractory disease,” she said.
“There are no published clinical trials (only case reports) of this therapy, but some are in progress. Our study is one of the largest to explore the efficacy and safety of anti-TNF therapy in a large independent cohort of JDM patients."
Of the cohort involved in the study, about a quarter switched their anti-TNF treatment. Of these, just under two-thirds occurred because of therapy failure, one quarter was because of bad reactions and one eighth switched because they preferred subcutaneous administration.
A total of 21 adverse reactions were reported, of which seven were considered severe (anaphylactic reactions on infliximab infusion).
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