Highlights from the European Congress of Internal Medicine

FLORENCE, ITALY—Early treatment can help patients with systemic sclerosis (SSc) take advantage of a growing number of medication options, said Luc Mouthon, MD, PhD, head of the Internal Medicine department and Reference Center for Rare Systemic Autoimmune and Autoinflammatory Diseases of Ile de France, East and West, Cochin Hospital and Université Paris Cité.

Dr. Mouthon was one of several speakers who addressed topics related to rheumatology at the European Congress of Internal Medicine (ECIM) in Florence, Italy, in March.

Systemic Sclerosis

Dr. Mouthon

Diagnosing diffuse vs. limited SSc and determining organ involvement can have an impact on treatment selection, according to Dr. Mouthon.

Although many SSc-related deaths are associated with pulmonary arterial hypertension and interstitial lung disease (ILD), cardiac, gastrointestinal, renal or skin involvement often contributes to mortality, Dr. Mouthon explained. The failure to treat concurrent organ involvement or ILD has a negative impact on a patient’s survival.

“Unfortunately, the prognosis is the worst if we consider all the connective tissue diseases. … To treat early and efficiently, we have to get a better understanding of the pathophysiology, and identify biomarkers,” said Dr. Mouthon.

Treatment

Dr. Mouthon gave an overview of the available and upcoming treatments for SSc. For example, early diffuse SSc can be treated with low-dose glucocorticoids; immunosuppressants, including methotrexate, mycophenolate mofetil and cyclophosphamide; classical support; and early rehabilitation.

Autologous stem cell transplantation—a treatment being investigated for SSc—may be indicated for more severe cases, such as for patients with early diffuse disease, those with a severe increase on the modified Rodnan skin score or those with additional organ involvement, said Dr. Mouthon. However, it should be avoided in patients who have had SSc for more than five years or who have severe organ involvement, such as respiratory insufficiency, severe renal insufficiency or cardiac insufficiency. Patients with an inflammatory or fibrotic profile based on transcriptomics in peripheral blood mononuclear cells may respond to stem cell transplantation better than those with a sub-normal transcriptomic profile.

Abatacept is being investigated as a treatment for diffuse cutaneous SSc. Although a 2020 phase 2 trial with 88 participants found that abatacept did not lead to a significant change in the modified Rodnan skin score, the drug was well-tolerated, Dr. Mouthon said.¹

Nintedanib, an anti-fibrotic agent that targets fibroblasts and is authorized in primary pulmonary fibrosis, is a U.S. Food & Drug Administration-designated breakthrough treatment for ILD.