The Efficacy of the Drug Epoprostenol in the Treatment of Pulmonary Hypertension Associated with Scleroderma FREE

Pulmonary hypertension (high blood pressure in the arteries that go to the lungs) can produce disabling shortness of breath and often causes death. It can occur on its own (primary pulmonary hypertension) but is usually the result of an underlying lung disease, such as scleroderma (secondary pulmonary hypertension). No proven therapies for pulmonary hypertension are known, but epoprostenol, a drug that can dilate narrowed arteries, has shown promise in the treatment of patients with primary pulmonary hypertension.

The researchers wanted to see if epoprostenol would be useful in treating pulmonary hypertension that is secondary to scleroderma.

One hundred eleven patients from 17 pulmonary hypertension centers in the United States with pulmonary hypertension associated with scleroderma.

The researchers randomly assigned patients to receive either usual treatment or usual treatment plus epoprostenol. For 12 weeks, a pump delivered small amounts of epoprostenol continuously through a small tube inserted into a vein. The researchers measured the degree to which shortness of breath limited patients' activities by observing how far each patient could walk in 6 minutes, both at the beginning of the study and again after 1, 6, and 12 weeks of treatment.

The 56 patients who got epoprostenol increased the distance they could walk in 6 minutes from 270 to 316 meters. The 55 patients who received only usual therapy could walk 240 meters at the start of the study but only 192 meters at the end. Four epoprostenol patients and 5 usual therapy patients died during the course of the study. Frequent side effects of epoprostenol were jaw pain, diarrhea, nausea, and loss of appetite. Depression also occurred, but less commonly. In addition, patients who got epoprostenol had important side effects, including infection (4 patients) and bleeding (3 patients), that were related to the intravenous tube.

This study included too few patients to show whether epoprostenol improved survival among patients with scleroderma-associated pulmonary hypertension. Patients might find that the side effects and inconvenience of receiving epoprostenol continuously through an intravenous tube are not worth the improvement in exercise ability. It is also unclear whether the benefit observed in the 12 weeks of this study would continue over a longer period.

Intravenous epoprostenol appears to improve exercise ability in patients with pulmonary hypertension due to scleroderma. However, associated side effects and the need for intravenous administration may limit the drug's usefulness.