In an article published in The American Journal of Managed Care (AJMC), researchers provided an overview of the current treatment and management paradigm for systemic-sclerosis (also called scleroderma) with interstitial lung disease (ILD). The discusses the impacts of the most common therapies as well as challenges that the medical field continues to face when treating the rare disease. ILD, a frequent complication in scleroderma, is also the most common cause of death for patients.
Scleroderma, which is also referred to as systemic sclerosis, describes a group of autoimmune diseases that can cause system-wide effects in the most severe cases. The mechanism of this disease is believed to be an autoimmune response in which the immune system mistakenly attacks body tissue. Some factors that may contribute to triggering the autoimmune response include mutations of the HLA genes and exposure to certain materials, such as certain solvents, white spirits, ketones, and silica. Symptoms are broad-ranging and systemic, including kidney failure, erectile dysfunction, fatigue, stroke, headaches, facial pain, congestive heart failure, skin abnormalities, high blood pressure, chest pain, indigestion, and many more. Treatments are varied and depend on the symptoms, but most patients take medications in an attempt to suppress the autoimmune response. In severe cases, life expectancy is around 11 years from onset. To learn more about scleroderma, click here.
Compared to many related diseases, scleroderma carries a higher risk of mortality and the presence of ILD has been associated with worse outcomes based on a variety of different measures. The most common treatments for scleroderma are immunosuppressive agents, such as mycophenolate mofetil (MMF). This has become the preferred therapy in this class for this disease as it has been demonstrated to potentially halt the decline of lung function that occurs with ILD and has a better tolerability and safety profile compared to cyclophosphamide, for example.
The US Food and Drug Administration (FDA) also approved a drug of the tyrosine kinase inhibitor class for scleroderma in 2019. This drug is called nintedanib. This approach can slow the rate of lung function decline and is also effective in combination with MMF. The most recently approved therapy for the disease is tocilizumab, which is classified as an IL-6 inhibitor.
Future approaches under investigation include further tyrosine kinase inhibitors, antifibrotic therapy, B-cell depletion, and stem cell transplant. The authors note the lack of codified guidelines for the treatment of scleroderma with ILD. While each case is different and has independent considerations, rules on different therapy options, when to begin treatment, and so on could help improve outcomes for patients. While there are some useful treatments, there is still a significant unmet need for therapies with stronger effects and a better safety profile.