Pulmonary hypertension associated with sickle cell disease: pathophysiology and rationale for treatment.

Approximately one third of patients with sickle cell disease (SCD) have pulmonary hypertension (PH), which increases their risk of death. Endothelin (ET)-1 is elevated in SCD and appears to play a key role in many of the pathologic processes in this disease, including PH, suggesting that endothelin receptor antagonists such as bosentan may be effective in treating patients with SCD, particularly those with PH. Other possible treatments include sildenafil, epoprostenol, and oral arginine. Data from controlled clinical trials are needed to establish the most effective treatment of patients with PH associated with SCD.