|Portfolio||Paediatric, foetal and maternal health|
To investigate the effectiveness and safety of mycophenolate mofetil (MMF), versus standard cyclophosphamide (CYC) for treatment of polyarteritis nodosa (PAN), a serious inflammatory blood vessel disease (vasculitis) in children. We have concerns regarding toxicity of CYC in children that include excessive infection, bladder complications, infertility, and malignancy. MMF is believed to be a safer therapeutic option than CYC.
- Bell, S. A., & Tudur Smith, C. (2014). A comparison of interventional clinical trials in rare versus non-rare diseases: an analysis of ClinicalTrials.gov.. Orphanet journal of rare diseases, 9, 170.
- Hampson, L. V., Whitehead, J., Eleftheriou, D., Tudur-Smith, C., Jones, R., Jayne, D., . . . Brogan, P. A. (2015). Elicitation of Expert Prior Opinion: Application to the MYPAN Trial in Childhood Polyarteritis Nodosa. PLOS ONE, 10(3).
- Hee, S. W., Willis, A., Tudur Smith, C., Day, S., Miller, F., Madan, J., . . . Stallard, N. (2017). Does the low prevalence affect the sample size of interventional clinical trials of rare diseases? An analysis of data from the aggregate analysis of clinicaltrials.gov.. Orphanet journal of rare diseases, 12(1), 44.
- Tudur Smith, C., Williamson, P. R., & Beresford, M. W. (2014). Methodology of clinical trials for rare diseases. Best Practice and Research: Clinical Rheumatology, 28(2), 247-262.