Dataset from Treatment of Pulmonary Hypertension and Sickle Cell Disease With Sildenafil Therapy

This study will examine whether the drug sildenafil can lower blood pressure in the pulmonary artery (the blood vessel that leads from the heart to the lungs) in patients with sickle cell disease and pulmonary hypertension (high blood pressure in the lungs). It will see if this treatment can reduce disease complications, such as shortness of breath, pain crisis, pneumonia, and increase survival. Patients 12 years of age and older with sickle cell disease and pulmonary hypertension may be eligible for this study. Participants are randomly assigned to receive sildenafil or placebo (sugar pill) for 16 weeks. Before starting treatment, patients have baseline studies, including a pregnancy test for females of childbearing age; a chest x-ray; pulmonary function tests to measure how much air the patient can breathe in and out; an echocardiogram (heart ultrasound); a 6-minute walk test to measure exercise capacity; a quality-of-life assessment and a pain inventory. Patients with moderate to severe pulmonary hypertension undergo heart catheterization to evaluate the severity of hypertension before beginning sildenafil therapy. During treatment, patients are monitored with the following: - Blood tests: weeks 6, 10 and 16. - Echocardiogram: weeks 6 and 16. - 6-minute walk test: weeks 6, 10 and 16. - Measurements of weight, blood pressure and heart rate: weeks 6, 10 and 16. - Pregnancy test for women of childbearing age: weeks 6, 10 and 16. - Pain questionnaire once a day for a week: weeks 6 and 1.0 - Quality-of-life questionnaire: week 16. - Heart catheterization: week 16 for patients with moderate to severe hypertension. At the end of the 16-week period, patients may opt to continue to receive sildenafil and monitoring in an open-label phase of the study for up to 1 year.