Projects: Priority Setting Partnership
A group of medical specialists instigated by Dr Karen Harman of the Nottingham University based Centre of Evidence Based Dermatology and a Consultant Dermatologist in Leicester, and led by the James Lind Alliance (JLA), were involved in a study to identify the top 10 priorities for future research into blistering diseases in the UK.
Many PEM Friends contributed to the prioritisation process and will, we believe, benefit from the focus it offers to anyone seeking funding for research into pemphigus and pemphigoid. It was a long journey, largely due to interruptions due to covid-19, and the outcomes were made public during 2023 in an article published in the British Journal of Dermatology.
The paper is the culmination of discussions, led by the JLA, between a group of medical professionals from different disciplines and a group of patients to agree the top priorities for blistering autoimmune conditions.
Six PEM Friends were on the steering committee and many more PEM Friends were involved in the input to the list of questions and the process of filtering these down, along with similar inputs from clinicians, nurses, GPs, etc. In September 2022, a workshop was held at which we further ranked a set of 17 questions to produce a Top 10. These will be the basis of deciding future priorities for research and will influence future grant applications.
Medical professionals and patients were largely in agreement.
The top 10 priorities voted for by medical specialists and patients are:
How effective, safe and cost-efficient is rituximab (or similar biologics) in BP/PV/MMP compared to standard steroid/immunosuppressant use, when should it be started and should it be a 1st line treatment?
Are outcomes for patients with BP/MMP/PV better if treatment is started earlier and with 'stronger' treatments, such as an immunosuppressant or biologic, rather than escalating from 'milder' treatments if they do not work?
How should persistent mouth lesions be best treated in pemphigus and pemphigoid?
What is the best treatment for preventing and repairing scarring in MMP (medical and surgical)?
Is it possible to identify drugs that block the specific immune pathways for BP/MMP/PV rather than treat them with broad immunosuppressive drugs?
What are the risks and benefits of the different tablet and injection treatments used to treat BP/MMP/PV? (such as azathioprine, mycophenolate mofetil, methotrexate, cyclophosphamide, chlorambucil, nicotinamide, dapsone, intravenous immunoglobulin, plasmapheresis)
What factors predict relapses in BP/MMP/PV, how can the risk of relapse be reduced and how are relapses best treated?
What is the best/most effective dose to prescribe for steroid tablets in BP/MMP/PV including the starting dose, when and how quickly to reduce the dose, and when to stop?
Can we predict the response to treatment in BP/MMP/PV and what factors affect this?
What is the best way to treat skin wounds in BP/MMP/PV including how should blisters/ erosions be best washed and managed and does treatment vary according to body site?
Further information is available on the Nottingham University website or the James Lind Alliance website.