What are the therapeutic options for progressive SSc-ILD?
Based on retrospective data and randomized controlled trials (e.g., Scleroderma Lung Studies I and II and Fibrosing Alveolitis Trial), either cyclophosphamide (CYC) CYC or mycophenolate mofetil (MMF) MMF are the drugs of choice for patients with progressive SSc-ILD. CYC can be given by monthly intravenous route or daily oral therapy. Both MMF and CYC are associated with modest improvements in FVC, degree of fibrosis on HRCT, health-related quality of life, and mRSS. In Scleroderma Lung Study II, treatment with MMF for 2 years was as effective as treatment with oral CYC for 1 year. MMF was safer and better tolerated. The lower toxicity profile of MMF makes it a more favorable option in general. Furthermore, in contrast to CYC, MMF is a suitable long-term immunosuppressive agent. In refractory cases, rituximab, azathioprine, or calcineurin antagonists (cyclosporine or tacrolimus) may be considered. Treatment with autologous HSCT may have beneficial effects on the ILD component of SSc but is typically reserved for those with dcSSc who have failed conventional therapies.
Pirfenidone and nintedanib were each approved by the Food and Drug Administration (FDA) in 2014 for the treatment of idiopathic pulmonary fibrosis (IPF). Both drugs were associated with slowing of lung function decline in those with IPF. Large, multicenter clinical trials are underway with each agent for the treatment of SSc-ILD (and other forms of ILD). In September of 2019 the FDA approved the use of nintedanib (either alone or in combination with MMF) for the treatment of SSc-associated ILD.
