“Steroid-toxicity is a huge problem amongst our patients… it sounds simple to just give people steroids, but the types of side effects they get when they have to be on these drugs for one to two years, sometimes many more years, to keep the disease suppressed produce many, many complicated side effects which can kill the patient.”
Dedee Murrell, MD DSc is a leading international expert on chronic inflammatory blistering diseases - both genetic and autoimmune. She is the Head of the Department of Dermatology at St George Hospital, University of New South Wales, Sydney, Australia, and Professorial Fellow at The George Institute for Global Health.
Professor Murrell was drawn to the complexity of diagnosing diseases of the skin, and uncovering their underlying causes - as well as investigating safer and more efficacious treatments. This has led to her developing new outcome measures that can be used in clinical trials to demonstrate whether new therapies are more effective and safer than the existing steroid treatments. Her work in this area also led to her being invited to join the international team that developed the Glucocorticoid Toxicity Index.
While training as a dermatologist at the University of North Carolina at Chapel Hill she was inspired by Dr. Al Briggaman to study the pathogenesis of blistering diseases. It was only later that she became involved in treatment and now runs regular special clinics for patients with these diseases.
Professor Murrell explains that dermatologists generally use topical therapies for most diseases, but for severe disease, systemic steroids have been used for many years. These treatments were life-saving for these patients because the mortality used to be around ninety percent, and when steroids were discovered and developed the mortality rates then gradually reduced to about ten or fifteen percent.
“But those patients nowadays are usually dying more from complications of being on long-term steroids than they are from the disease itself. Nowadays, we're trying to minimize the use of steroids and find cleverer ways of suppressing the immune system without off-target effects.”
“Steroid-toxicity is a huge problem amongst our patients… it sounds simple to just give people steroids, but the types of side effects they get when they have to be on these drugs for one to two years, sometimes many more years, to keep the disease suppressed produces many, many complicated side effects which can kill the patient.”
One of the challenges Professor Murrell experienced was when she initially reviewed the literature on the different definitions and outcome measures for a Cochrane Review. There were more outcome measures used in studies of pemphigus than there were patients. This meant the data couldn't be pulled together because in every country, and trial center, they were using different definitions and measures.
So the first thing she did was to get dermatologists all around the world at different venues together, to agree upon a standardized language. Since then Professor Murrell’s focus has been to develop validated tools to score the disease. As well as the quality of life of patients treated for the disease.
This led to her being invited to join the group of interdisciplinary experts led by Professor John Stone at Harvard, to contribute to the Glucocorticoid Toxicity Index as a representative of the dermatological aspects.
“The GTI has been great for me to be able to have a way to quantify the side effects that my patients are getting from steroids.”
Professor Murrell is now using the GTI in all the trials run in her clinical trial center, and she recently published a paper in JAAD International, one of the most respected dermatology journals. This paper presented data showing that using the GTI, they could detect detrimental effects within only three months of initiating steroid treatment, especially on the psychiatric well-being of the patients.
Her team is also now using the GTI in regular clinics, “dermatologists aren’t normally people who go around measuring people's blood pressure. But there are a few simple things like that that we've added to our safety follow-up so that we can include the data in the GTI scoring sheets.”
Professor Murrell also highlighted; “the GTI is a great way of showing the cost of all these side effects that these patients are getting from the steroid treatments and help get new drugs not only approved on the terms of efficacy but also funded by payers.”