According to a story from Scleroderma News, findings from the recent Phase 2 EDITA clinical trial suggested that ambrisentan (marketed as Letairis), which is approved to treat pulmonary arterial hypertension (PAH), could also be effective in preventing the condition in people living with systemic scleroderma. PAH is a frequent complication found in scleroderma patients.
The findings were recently presented at this year’s European Alliance of Associations for Rheumatology (EULAR) Congress. The results could have significant implications for the standard of care for people with systemic scleroderma.
Letairis is already an FDA-approved therapy for PAH, but there’s little data on its efficacy specifically in people with scleroderma. In the EDITA study, 38 scleroderma patients were participating, and the drug was measured against placebo against the endpoint of prevention of worsening mean pulmonary arterial pressure (mPAP). While the initial results showed promising indicators of heart and lung health, the drug didn’t meet this endpoint in the study period.
But 19 patients continued treatment with the drug and agreed to continued monitoring. Long term data showed that mPAP increased by 1.91 mmHg on average in the placebo group but went down by an average of 1.53 mmHg in the Letairis group—a statistically important difference. Additionally, four patients in the control group would go on to develop PAH, defined as over 20 mmHg.
Overall, the researchers concluded that early treatment could prevent or impede the development of PAH in these patients.
About Scleroderma
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.
About Pulmonary Arterial Hypertension (PAH)
Pulmonary arterial hypertension is a condition in which the blood pressure in the arteries of the lungs is abnormally high. The cause of pulmonary arterial hypertension is often unknown in many cases. However, there are a variety of potential causes, such as certain heritable genetic mutations, exposure to certain toxins, and drug use (ex. methamphetamine). It can also appear as a symptom or complication in a number of other diseases, such as heart disease, connective tissue disease, and infection with HIV. The arteries in the lungs are often inflamed. Symptoms of this condition include rapid heartbeat, poor exercise tolerance, shortness of breath, fainting, leg swelling, fatigue, and chest pain. Treatment may include a number of medications and surgical operations, including lung transplant. A transplant can cure the condition, but it can cause many complications. Survival rate is often only about two or three years without treatment, but the latest drugs can prolong life by several years or more. Click here to learn more about pulmonary arterial hypertension.
