An Addenbrooke's team is leading a new, national trial to look at the best treatment for vasculitis - and has recruited its first patient, 10 year old Eliska.
Vasculitis is a rare and potentially life threatening 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.
Steroids are commonly used to help treat the disease, however many people experience side effects or find no relief at all.
The new trial will investigate if three licensed, biologic drugs - that directly target the immune system - are more effective to treat vasculitis in patients of all ages.
Eliska is the first patient in the UK to take part in the trial at the NIHR Cambridge Clinical Research Facility (CRF) at Cambridge University Hospitals Trust (CUH).
Vasculitis has a range of symptoms from fatigue and weight loss to skin rashes, numbness, pain and shortness of breath.
By taking part in the new trial, Eliska hopes to make a difference.
I’m hoping it will help me and also other children with vasculitis
Eliska, CUH patient
Eliska added: "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.”
Vasculitis can be life-threatening and currently we don’t have enough information which medication is the most suitable.
Dr Kate Armon, consultant paediatric rheumatologist at CUH
Dr Kate Armon, a paediatric rheumatologist at CUH, is overseeing the paediatric part of the research, know as the BIOVAS trial.
She said:
"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."
The trial hopes to recruit 140 adults and children in total from around the UK, all 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.
Patients with rare inflammatory disorders, such as vasculitis, are often denied access to newer therapies due to a lack of evidence in their specific disorder.
Prof David Jayne, trial chief investigator and CUH consultant
The trial chief investigator is Professor David Jayne, a NIHR Cambridge Biomedical Research Centre (opens in a new tab) researcher and consultant in nephrology and vasculitis at CUH.
He said:
"BIOVAS is an NIHR funded national trial including 9 rare vasculitis disorders, which will produce evidence for three biologic therapies already in widespread use in other inflammatory conditions.
"Co-ordinated by the Vasculitis Research Group in Cambridge, we expect BIOVAS to change the standard of care and bring improvements to the health of patients suffering from these disorders."