The Drug Abatacept Appears Beneficial in Systemic Sclerosis Despite Missing Primary Endpoint

According to a story from Medpage Today, a recent study testing the medication abatacept (marketed as Orencia) as a treatment for diffuse cutaneous systemic sclerosis concluded with rather mixed results. The bad news from this phase 2 trial was that the drug had failed to meet the primary endpoint. This endpoint was the Rodnan Skin Score, and the changes were not significantly meaningful. However, the good news was that the treatment offered meaningful improvements in a couple of other measures.

About Systemic Sclerosis

Systemic sclerosis, which is also referred to as scleroderma, describes a group of autoimmune diseases that can cause system-wide effects in the most severe cases. Diffuse cutaneous systemic sclerosis is a form that mostly impacts the skin but can still have impacts in other body systems. 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 systemic sclerosis, click here.

About The Study

Patients in the trial saw meaningful improvements on both the Health Assessment Questionnaire Disability Index (HAQ-DI) and the American College of Rheumatology Combined Response Index in Systemic Sclerosis (ACR-CRISS). These measures can effectively evaluate the involvement of vital organs such as the kidneys, the heart, and lungs. As a result, changes in these tool scores can reflect significant changes to the patient’s condition and quality of life. As this was a placebo controlled study, it was clear that abatacept was responsible for these improvements. With these mixed results, the future of abatacept as a treatment for diffuse cutaneous systemic sclerosis remains unclear and more research is necessary. 

Check out the original study here.


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