Immersive website targets knowledge gap, unmet therapy needs in systemic sclerosis
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A disease like systemic sclerosis demands a multifaceted approach. Comprehensive clinical interventions are required because the disease affects the skin, lungs, heart and kidneys, among other systems. But education is also necessary. While systemic sclerosis impacts between 60,000 and 75,000 Americans and can shorten life expectancy by one or two decades, it often gains far less attention than more high-profile conditions.
The recent launch of TotalSSc.com, a website dedicated to the disease, aims to rectify this situation. The website was originally planned to make its debut during the sixth iteration of the Systemic Sclerosis World Congress in March, prior to the event being postponed to July due to COVID-19.
Despite the setback of the postponed meeting, Tracy M. Frech, MD, MS, director of the Systemic Sclerosis Clinic and director of clinical trials for the division of rheumatology at the University of Utah Hospital, remains optimistic. She spoke with Healio Rheumatology about educational opportunities available through TotalSSc.com as well as recent developments in systemic sclerosis, offering a soup-to-nuts overview of current therapies and future directions.
Individualized Treatment
“The most important thing to consider is that no two systemic sclerosis patients are alike,” Frech, who serves as point person for the TotalSSc.com website, said in an interview.
Patients may present with skin or internal organ complications and their disease course can be vastly different from one to the other. This diversity has dictated treatment paradigms in the past.
“Treatments for systemic sclerosis have traditionally been directed at end-stage organ disease, including disease in the skin, lungs, joints and gastrointestinal tract,” Frech said.
Most patients will have Raynaud’s phenomenon, in which case vasodilators such as calcium channel blockers and phosphodiesterase-5 inhibitors can be used for treatment, according to Frech.
Physicians may treat skin fibrosis with immunosuppression such as methotrexate. If a patient has lung fibrosis, physicians may prescribe cyclophosphamide (Cytoxan, Chemocare), mycophenolate mofetil (Cellcept, Genentech) or nintedanib (Ofev, Boehringer Ingelheim). Patients with pulmonary hypertension are indicated to receive phosphodiesterase-5 inhibitors along with endothelial antagonists and/or prostacyclin analogs. If a patient has arthritis, methotrexate or tocilizumab (Actemra, Genentech) are options, whereas, gastrointestinal involvement is treated with reflux medications.
There are many exciting treatments on the horizon and these are just the highlights, Frech noted. Clinicians are encouraged to visit the TotalSSc site for information about the full treatment armamentarium for all manifestations of systemic sclerosis. “It is most important to think through treatments that are more preventive,” Frech said. “The key is to get patients on treatment before these end-stage manifestations occur.”
Teamwork and Communication
This raises the question of who might end up seeing these patients first. “If skin thickening or digital ulcers are the presenting features, a dermatologist may be the first to see the patient,” Frech said. “If pulmonary hypertension is seen on a screening echocardiogram, it may be cardiology.” It follows, then, that a pulmonologist may be the first to see the patient if interstitial lung disease occurs, or a gastroenterologist if the patient has severe gastrointestinal reflux disease.
All of this underscores the importance of having a rheumatologist on the team to direct care. “Rheumatologists are trained to care for this autoimmune disease,” Frech said. “Specifically, they will assess the skin, joints, muscles and tendons for need of immunosuppression. They will educate on the risks of steroid use and scleroderma renal crisis, the importance of internal organ screening with pulmonary function tests and echocardiogram, and important ancillary services such as occupational therapy and wound care, and they can understand the specifics of diet and exercise that may be effective in systemic sclerosis.”
Unfortunately, the journey from first presentation to a comprehensive care plan from rheumatology plus cardiology, pulmonary, gastroenterology or dermatology is often a long one for patients.
“This question about the patient journey is an important one that highlights the value of each patient being seen in a scleroderma specialty center where experts know and understand the value of treating each patient as an individual,” Frech said. “Typically, this will be accomplished by approaching disease pathogenesis as a progressive abnormality of the vasculature, which results in fibrosis and immune dysregulation and taking time to explain each aspect of treatment to the patient.”
In short: education works.
When a rheumatologist is at the center of treatment decisions, they can help other specialists understand that treatment may include a vascular approach for Raynaud’s phenomenon, scleroderma renal crisis and pulmonary hypertension; an immune approach for skin thickening; and both an immune and anti-fibrotic approach for interstitial lung disease. She added that supportive therapies such as reflux management, wound care, and hand therapy are also important for many patients. “All of this highlights the necessity of meticulous assessment of patients,” she said.
Hope on the Horizon
If there is one other point that Frech would like to stress, and that both the TotalSsc site and the World Congress aim to address, it is the importance of getting patients into clinical trials.
“The real take-home message is to focus on early recognition, early screening, active therapy and clinical trials options for these patients,” she said. “A main unmet need in this disease is that the previous approach of responding to damage rather than actively searching for disease manifestations is not the way to optimize results.”
Frech recommends “aggressively treating disease activity” and every manifestation in each of these patients.
“Getting back to the clinical trials, physicians should be aware that there are many exciting new targeted therapies on the horizon,” Frech said. “Immune therapies get a lot of attention, but these new therapies are not just targeting the immune system. They are addressing immune dysregulation in order to target fibrosis. The endocannabinoid system is an especially exciting area.”
Clinicians and researchers in every specialty are facing big challenges with COVID-19. But Frech remains undaunted. “There are a lot of reasons to be optimistic about systemic sclerosis,” she said. “It is an exciting time to be a rheumatologist.” – by Rob Volansky
For more information:
Tracy Frech, MD, can be reached at 50 N Medical Dr, Salt Lake City, UT 84132.
Disclosures: Frech reports no relevant financial disclosures.