Project: Paediatric Drug Trial for Treatment of Polyarteritis Nodosa (PAN) – UCL Institute of Child Health & Great Ormond Street Hospital.
Duration: 1 Year – Commencing July 2013
LCTF Funding Award: £15,000
The trial will take place at selected Centres of excellence in the UK and across Europe, with a track record in treating children with Polyarteritis Nodosa (PAN), and will be done in association with UK clinical networks and the Paediatric Rheumatology International Trials Organisation (PRINTO). This drug trial is funded in collaboration with Arthritis UK, Vasculitis UK and The Lauren Currie Twilight Foundation.
Currently, children with PAN are treated with Cyclophosphamide (CYC) which has been the main treatment for more than 30 years, unfortunately, this treatment requires to be administered intravenously which requires a hospital stay. It also has a number of long-term, life-changing side effects, which include malignancy, infertility, sickness, haemorrhagic cystitis (bleeding from the bladder), low white cell counts and infections.
The alternative drug to be tested is Mycophenolate Mofetil (MMF), although there is limited clinical experience with this drug, its side effects are less severe.