LCTF Support PAN Paediatric Drug Trial
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, therefore 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, but include diarrhoea, low white cell count and infections, a lower risk of malignancy, but possibility of skin cancer increases, but there is no association to infertility.
Grant Currie, Trustee of The Lauren Currie Twilight Foundation says: “Lauren was still a child when we lost her to Vasculitis, the fact that we can support Great Ormond Street, to trial drugs that make children’s lives easier once they have been diagnosed means her death was not in vain. We have seen many child Vasculitis sufferers since we lost Lauren, anything we can do to make their lives easier is one of the charities main goals.
Dr Paul Brogan, Reader in Paediatric Vasculitis and Consultant Paediatric Rheumatologist , added: “At the moment the standard drug used to treat severe PAN in children is CYC; we believe that MMF is a safer alternative, potentially with fewer serious side effects. MMF has been trialled in tests with adults with vasculitis and has shown promising results; to change the current system we must show the same positive effect on children with PAN.”
You can read about this collaborative research and other LCTF research funded projects at our Research page on this website.