Treprostinil Improves Exercise Performance, Lowers Blood Pressure in Sickle-cell Patients

Treprostinil Improves Exercise Performance, Lowers Blood Pressure in Sickle-cell Patients

Treprostinil improves exercise performance and lowers blood pressure in sickle-cell disease (SCD) patients with pulmonary hypertension (PH), a study shows.

The therapy was also well tolerated by the patients, the researchers said.

The findings were reported in the study “Prostacyclin-analog therapy in sickle cell pulmonary hypertension,” published in Haematologica, the journal of the European Hematology Association.

PH is a serious complication in SCD patients, causing high morbidity, or disease severity, and mortality.

Doctors were concerned that although treprostinil controls PH, it might cause cardiac problems in SCD patients. This research addressed treprostinil’s effectiveness in controlling PH associated with SCD.

Eleven SCD patients with critical hemodynamic — or blood flow — values received treprostinil in three forms: inhaled, by injection or intravenously.

They were followed up for a median period of 13 months. Ten experienced a 35% decrease in pressure in their right ventricle, or heart chamber. Nine experienced a 20% increase in exercise capacity as measured by a six-minute walking distance test.

Patients who took treprostinil by injection responded the best, followed by those who received it intravenously and those who inhaled it.

The study also revealed that the decrease in right-ventricle pressure peaked after the first two months of therapy. Also, the higher the dose of treprostinil, the more improvement, researchers found.

Side effects were typical with treprostinil: swelling in the inguinal or groin area, headaches, dizziness, throat symptoms, jaw pain, pain around injection sites, bacteria in blood or tissue.

Two patients in the study died — a reflection of the high mortality rates among SCD patients with PH.

“In this cohort of 11 SCD patients, parenteral, especially subcutaneous, prostacyclin-analog therapy was well tolerated and appeared to be effective. Although additional experience is needed, there is no evidence that clinicians should avoid prostacyclin therapy when clinically indicated in patients with SCD and PH,” the researchers concluded.


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