PAH Greatly Impairs Survival of Scleroderma Patients in First Years After PAH Diagnosis

Scleroderma patients diagnosed with pulmonary arterial hypertension (PAH) are at a high risk of PAH-related death, especially in the four years after the PAH diagnosis, a study shows. Also, being a man, having diffuse scleroderma, and worse scores on respiratory- and heart-related tests were associated with a higher risk of death due to PAH.

The findings support the need to identify early markers and improve treatment strategies for this high-risk patient population.

The study, “Long-term Outcomes in Systemic Sclerosis Associated Pulmonary Arterial Hypertension from the Pulmonary Hypertension Assessment and Recognition of Outcomes in Scleroderma Registry (PHAROS),” was published in the journal Chest.

Scleroderma, or systemic sclerosis, is a chronic autoimmune disease in which the body recognizes its own connective tissue — the tissue that supports and holds organs together — as foreign and attacks it. Scleroderma patients are at high risk of developing PAH, which is a leading cause of death and a predictor of early death in these patients.

Although some studies have assessed the short-term clinical course of scleroderma patients with PAH, as well as markers of high risk of death in the first years after PAH diagnosis, a thorough description of the long-term outcomes and predictors of mortality is lacking.

To fill this gap, researchers investigated survival rates, cause of death in the short-term (less than four years) and long-term (more than four years), and predictors of high risk of death in scleroderma patients with newly diagnosed PAH, using the Pulmonary Hypertension Assessment and Recognition of Outcomes in Scleroderma (PHAROS) registry.

PHAROS was designed to follow scleroderma patients at high risk for PAH, or with newly diagnosed pulmonary hypertension. Enrolled patients are registered in institutions that actively screen for PAH, and PAH diagnosis is confirmed using right heart catheterization, a medical procedure that assesses how well the heart is pumping and measures the pressure in the heart and lungs.

The study included 160 scleroderma patients with newly-diagnosed PAH (142 women), with a mean age of 60, and most them had no or slight limitation in daily activities. Patients were followed-up for a median of 7.1 years.

The survival rate at one year was 95%, at three years 75%, at five years 63%, and at eight years 49%.

During follow-up, 56 patients died — 44 in the first four years, and 12 after four years. Of those, 29 patients (52%) died from PAH-related deaths — 27 in the first four years, and two after four years. This suggests that PAH greatly impairs patients’ survival in the first years after diagnosis.

When researchers looked at PAH-related deaths only, the survival rates of patients at one and three years were 97% and 83%, and at five and eight years 76%. Patients who died in the first four years after PAH diagnosis showed a more severe disease at enrollment.

Clinical features associated with high risk of PAH-related death included male gender, diffuse scleroderma (the more aggressive form of scleroderma), elevated pressure inside the pulmonary artery (the blood vessel that moves blood from the heart to the lungs), lower exercise capacity (measured through the six-minute walk test), and reduced lung gas transfer capacity.

Based on the results, the team concluded: “Overall survival in PHAROS was higher than other SSc-PAH cohorts. PAH accounted for more than half of deaths and primarily within the first few years after PAH diagnosis.”