Exciting times in treatment of systemic sclerosis

It is an exciting time for the treatment of systemic sclerosis (SSc) with better disease understanding, many new effective treatments, and more promising ones in the pipeline, attendees at the Irish Society for Rheumatology 2026 Spring Meeting heard.

Noted US expert and researcher in this area, Prof Julie Paik, Associate Professor of Medicine, Johns Hopkins School of Medicine, US, gave a talk entitled ‘Systemic sclerosis beyond the skin: Multisystem disease in clinical practice’.

She noted that SSc is a rare complex, multisystem autoimmune disease characterised by vasculopathy, immune dysregulation, and progressive fibrosis, leading to significant morbidity and mortality.

Prof Paik discussed the various subtypes/phenotypes and their association with more severe disease. SSc-associated myopathy, for example, is associated with a higher mortality, more severe disease risk, and myocardial disease risk. Careful ongoing screening and categorising of SSc patients is therefore key to identifying and managing their individual manifestations.

Treatment-wise, mycophenolate mofetil (MMF) is now the standard of care over cyclophosphamide for managing key manifestations of SSc, particularly associated interstitial lung disease (SSc-ILD) and skin manifestations, with rituximab being increasingly used in more refractory disease in line with emerging US data, Prof Paik said.

The most significant recent strides made in the treatment of SSc have been in the cardiopulmonary manifestations of the disease – specifically SSc-ILD and pulmonary arterial hypertension (SSc-PAH), which can develop in 10–15 per cent of SSc patients.

In recent years, the US Food and Drug Administration approved two drugs (tocilizumab and nintedanib) to treat SSc-associated ILD.

These are the first approved medications in the history of the treatment of SSc, “but we still have nothing for the skin,” she noted.

Ongoing trials are also showing promise with other agents, such as CAR T-cell therapy in early diffuse cutaneous SSc, and sotatercept – a novel, first-in-class activin signalling inhibitor – in treating SSc-PAH.

“Scleroderma is not the disease that it was 10–20 years ago. There has been an advent of medications to target the worst organ manifestations like cardiopulmonary disease and pulmonary hypertension ILD, that can now be treated,” Prof Paik told the Medical Independent.

“It is not a disease that we dismiss – and I have certainly had patients who were told there was no treatment for scleroderma. But now it is a very exciting time where there are multiple medications that can help our patients improve their quality-of-life and improve mortality [rates].”