Stanford Scleroderma Center of Excellence

Lorinda Chung, MD, MS
Stanford University

David Fiorentino, MD, PhD
Stanford University

Project Summary:

Dr. Chung: The primary goal of the Stanford Scleroderma Center is to provide outstanding multi-specialty care for patients with scleroderma, with experts from Rheumatology, Dermatology, Pulmonology, Gastroenterology, Cardiology, Immunology, and Hand/Vascular Medicine working together to take care of each patient as a whole. At our Scleroderma Clinic held at the Stanford Redwood City Outpatient Center, each patient is seen by a team of physicians from Rheumatology, Dermatology, and Internal Medicine all at the same time. Our Center has grown substantially over the past several years, with over 650 scleroderma patients evaluated and treated. We are also actively carrying out state-of-the-art research using tissue samples from patients with scleroderma and working with basic scientists to better understand what causes scleroderma, and to find markers in the skin or blood that can tell us which patients will go on to develop serious complications, like pulmonary arterial hypertension (PAH). In particular, we hope to develop a simple blood test to identify scleroderma patients who will ultimately develop PAH.

Research Update:

We have collected a large number of blood samples from scleroderma patients who have developed PAH over time from Scleroderma Centers throughout the US. In collaboration with the Stanford Human Immune Monitoring Core, we are evaluating these blood samples for 62 different markers of immune dysfunction. Over the course of the year, we hope to identify the most promising blood markers of PAH in scleroderma patients. We will then confirm our results in a completely separate group of scleroderma patients.

How This Work Will Impact Patients:

PAH affects about 10% of patients with scleroderma. Luckily, over the past two decades, multiple treatments have been developed that are effective for the treatment of PAH. However, scleroderma patients tend to do worse than other patients with PAH, in large part, because once the patients are found to have PAH, their disease is already very advanced. We hope to develop a simple blood test to identify, at the time of their initial clinic visit, scleroderma patients who will ultimately develop PAH. This will enable doctors to start effective treatments as early as possible to prevent the onset and progression of this potentially deadly complication.

Dr. Fiorentino on the Future of Scleroderma Research:

There’s finally a critical momentum, a critical mass of researchers that is becoming involved in trying to understand and cure this disease. As a result, we now have large international registries of patients that are key to studying scleroderma. This, along with the development of new tools we can apply that are allowing us to be able to look at human beings in a much deeper fashion molecularly, at a cost that is no longer prohibitive, bodes well for the future of scleroderma research.

Dr. Chung on Why Research Matters:

“Research, through collaborative efforts among patients, clinicians, and scientists, is the only road to new discoveries. Research discoveries result in better understanding of diseases, and allow us to know who is at risk, what to expect, how best to treat, and ultimately to allow us to develop cures for diseases like scleroderma.”

 

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