Cambridge recruits first patient to national inflammation study

A new trial taking place in UK to look at the best treatment for vasculitis has recruited its first patient– a child aged 10

A national trial led by chief investigator Professor David Jayne, NIHR Cambridge Biomedical Research Centre researcher and supported by the NIHR Cambridge Clinical Research Facility (CRF), is testing which biologic drugs – those that directly target the immune system – are most effective to treat vasculitis in all ages.

Vasculitis is a rare disease where inflammation occurs in the blood vessels. The immune system attacks the vessels, which become swollen, limiting or blocking blood supply to organs in the body.

Biologic drugs are licensed medicines and have been used to treat vasculitis and are expensive. Currently, there is not enough data to help clinicians choose which licenced drugs are most effective for patients, which is what the Cambridge team are investigating.

National trial begins

Vasculitis is potentially a life-threatening condition, yet the causes are unknown. Symptoms can range from fatigue and weight loss to numbness, pain and shortness of breath. Steroids are commonly used to help treat the disease, however, many people experience side effects or find no relief at all.

Cambridge researchers have now begun recruiting 140 adults and children around the UK with a rare vasculitis known as ANCA negative, who have not responded well to current treatments and cannot reduce their steroid dose to a safe level.

Patients will be randomised to either one of three biologic drugs or a placebo as well as answer questionnaires on their symptoms or if they have noticed any changes. They will then be monitored through follow-up appointments over a course of four to six months. The hope is to show which biologic medication is more effective.

Eliska, pictured top right with her mother, is the first child in the UK to take part in the trial at the NIHR Cambridge CRF at Addenbrooke’s Hospital. She said: “I’m hoping it will help me and also other children with vasculitis. I enjoy spending the time with the research nurses and Dr Armon.”

Kamila, Eliska’s mum, said: “I’m pleased that Eliska has a very special and experienced research team, and also good care here. I’m pleased they can help her and we hope she will be healthy soon.”

Dr Kate Armon, a paediatric rheumatologist, pictured left, who is overseeing the paediatric part of the trial in Cambridge said: “Vasculitis can be life-threatening and currently we don’t have enough information which medication is the most suitable. When we try to bring the steroid dose down for children and young people with vasculitis, the condition can flare, causing a fever, a rash and pain and stopping them from living a normal life.

“Our team agreed that the best option is to trial a biologic medication to see if it can control the disease better. On this trial, patients like Eliska will be randomised to a biologic drug as well as steroids and immune suppressive medication to control the illness, we will then monitor any changes to their condition.

“We hope to understand further how the biologic treatments affect patients like Eliska and find out which have better outcomes on the patient’s health and quality of life.

“I am delighted that Eliska has been able to participate in this important study to potentially treat her very rare disease. To have access to this trial in the NIHR Cambridge CRF has been perfect for the family and for the study.”