Children suffering the agony of juvenile arthritis – which can leave many wheelchair-bound and result in permanent deformity – have been offered hope from a new drug therapy.
The National Institute for Health and Clinical Excellence, the body that provides prescribing guidelines, has approved RoActemra (tocilizumab) to treat systemic juvenile idiopathic arthritis (sJIA). Currently incurable, sJIA is the most severe type of juvenile arthritis and affects about 2,500 under-16s in the UK.
It is an extremely debilitating inflammatory condition causing extreme joint pain, a distinctive salmon-coloured skin rash and a constant high fever. It can also lead to problems with the spleen, liver and heart that can be life-threatening.
Dr Eileen Baildam, a paediatric rheumatologist at Alder Hey Children’s Hospital in Liverpool, says nobody knows the causes but it is thought to be an autoimmune condition, with a genetic component. ‘About a third of patients get better spontaneously, but two-thirds will continue to have serious flare-ups,’ she says.
One patient to have benefited already is 12-year-old Tiffany Robertson, who was five when she developed the condition. It left her in agony and bedridden for months. Her mother Kate, 34, from Southport, Merseyside, recalls: ‘The joints all over her body would swell up and stiffen and it could happen in one area or in several places at once: her hips, wrists, neck, ankles, fingers and toes.
‘She couldn’t get herself to the bathroom and if I touched her, she would scream. At some points she needed a wheelchair. She was put on steroids, with terrible side effects. By the time she was seven, she was so bloated that she was wearing clothes for 11-year-olds. People stared at her, which made her very self-conscious. She missed so much school they put a bed for her in the classroom, so she could at least be around other children.’
Tiffany tried various medications but none worked for long. Until now, the commonly used treatments for sJIA were anti-inflammatory drugs – ibuprofen or corticosteroids and methotrexate – which often do not slow the progression of the disease.
RoActemra, previously used to treat adult rheumatoid arthritis, works by blocking an immune system messenger called IL-6, which causes fever and inflammation in joints and organs.
IL-6 is meant to protect the body against infection but malfunctions and attacks the body. The more damage that occurs, the more the immune system’s white cells produce IL-6 to fight the unknown invader.
RoActemra was licensed for use following a five-year trial. Dr Baildam says the results were astounding. ‘After a year, two-thirds of patients had a 90 per cent improvement in their symptoms, which is extraordinary – three times as good as we had expected. For a lot of patients it switches off the condition. And it’s well tolerated with virtually no side effects. It gives thousands of children hope of restoring normality to day-to-day life.’
Eighteen months ago, just as she was about to start secondary school, Tiffany was allowed to try RoActemra, which is administered in hospital every two weeks by drip. ‘We were desperate,’ says Kate. ‘We didn’t want Tiffany to go on the trial because we were worried she would get a placebo and deteriorate even more, so she was prescribed the drug on compassionate grounds. After nine months we started to notice a difference and now you wouldn’t know she has sJIA.
‘She gets the odd niggle but she’s able to go to school every day and she even does morris dancing, travelling all over the North West. She’s a lot happier. The drug has made a huge difference to all our lives.’
Tiffany says: ‘I used to be very poorly, on and off, and miss school and going out with my friends, but now I can do everything I want to do, just like everybody else.
‘I am very thankful to my doctors and nurses for getting me the new medicine and looking after me.’