Future of systemic sclerosis treatment will likely be combination therapy
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SAN DIEGO — Patients with systemic sclerosis will likely require multidisciplinary management with combination therapy to treat the myriad complications that may arise, according to a speaker at the Congress of Clinical Rheumatology West.
To ensure the kind of comprehensive care that these patients need, Elizabeth R. Volkmann, MD, MS, of the University of California, Los Angeles, recommended a multidisciplinary approach that involves specialists in rheumatology, pulmonology, gastroenterology, cardiology and radiology, among others.
Additionally, the multiple extracutaneous manifestations of SSc likely means that just one drug may not be enough to manage the disease.
“It may not be that we have one treatment for scleroderma,” Volkmann said. “Likely, moving forward, we will be giving patients combinations of therapies.”
In addition to affecting skin and nails, SSc can impact several organ systems, including the upper and lower gastrointestinal (GI) system, the musculoskeletal system, and the heart, lungs and kidneys.
The musculoskeletal system is involved in 97% of people with scleroderma, the skin in 95% and the GI system in 90%, according to Volkmann. The lungs, kidney and heart are less involved, at 65%, 50% and 23%, respectively, she added.
The symptoms can be confounding, and patients may be thought to have lupus. The early signs and symptoms of scleroderma can include fatigue, Raynaud's phenomenon, swollen hands and positive antinuclear antibodies.
“We have to do a better job of getting the diagnosis right early on,” Volkmann said.
It is for this reason, she added, that diagnostic efforts targeting all of these organ systems are needed in suspected scleroderma.
“There is no way to know what is going on inside their organ systems without doing testing,” Volkmann said.
For upper GI involvement, endoscopy is often required, while high-resolution CT scan, pulmonary function testing and echocardiogram are recommended for suspected lung complications.
Meanwhile, patients with suspected cardiac involvement should undergo echocardiogram.
“Even if the patient is not complaining of cardiac symptoms, this will give you a nice baseline,” Volkmann said.
For renal involvement, blood pressure measurement, urinalysis and glomerular filtration rate should be obtained.
“This seems like a lot, but it is really important to do this in all of your patients that have systemic sclerosis,” Volkmann said.
Treatment approaches vary depending on which common manifestation of scleroderma is present: cutaneous disease, interstitial lung disease, GERD or Raynaud’s phenomenon.
Mycophenolate mofetil is Volkmann’s first-line option for cutaneous disease.
“The reason I put mycophenolate above cyclophosphamide on this list is because it is much better tolerated,” she said. “I will rarely use cyclophosphamide in these patients if I have other options available.”
In severe cases of cutaneous scleroderma, autologous hematopoietic stem cell transplantation is an option. Volkmann said it can have a profound effect on patient’s skin disease. However, because it is a “risky procedure,” she said this is not a first- or even second-line strategy.
For SSc-ILD, cyclophosphamide and mycophenolate mofetil are also recommended, along with rituximab (Rituxan, Genentech).
“Some studies have looked at rituximab as rescue therapy for patients who have failed cyclophosphamide or mycophenolate,” Volkmann said. “That is generally how it is used in the United States.”
Nintedanib (Ofev, Boehringer Ingelheim) also may be used in SSc-ILD. According to Volkmann, studies have investigated this drug with mycophenolate as background therapy.
“This is important to consider as you think about combination therapy for your patients,” Volkmann said.
Most rheumatologists should be familiar with the dietary and lifestyle modifications that can improve GERD, she added. Removing fried or spicy foods, alcohol and coffee can be beneficial. Volkmann also suggested recommending additions to the patient’s diet, such as yogurt, avocados and spinach, which can have positive psychological benefit.
Raynaud’s phenomenon can be treated first with vasodilators and calcium channel blockers, according to Volkmann. Second-line therapies include PDE-5 inhibitors and endothelial receptor antagonists.
“In severe cases, we have to use the prostacyclins,” she said, but noted that gaining approval to use these medications can be challenging.
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Volkmann EA. Update on the pathogenesis and treatment of systemic sclerosis. Presented at: The Congress of Clinical Rheumatology West; Oct. 20-23, 2022 (hybrid meeting).
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