See clinical trial results for patients who received continuous intravenous (IV) epoprostenol plus conventional therapy*

*Compared to those who received conventional therapy alone.

Study Design

These results are from a 12-week, prospective, open-label, randomized trial. All patients were either New York Heart Association (NYHA) Functional Class III or IV, except for 5 NYHA Functional Class II patients.

Epoprostenol plus conventional therapy
(n=56)

Conventional therapy alone
(n=55)

In a 12-week, prospective, open-label, randomized trial:

The primary endpoint was a change from baseline in exercise capacity as measured by the 6-Minute Walk Test
Other major study objectives were changes in cardiopulmonary hemodynamics from baseline, including cardiac index, mean pulmonary arterial pressure, and pulmonary vascular resistance1
Conventional therapy could include anticoagulants, oral vasodilators, supplemental oxygen, digoxin, and/or diuretics.
Exercise Data in PAH-SSD
Statistically significant improvement was observed in exercise capacity, as measured by the 6-Minute Walk Test
Improvements were apparent in some patients at the end of the first week of therapy

No statistical difference in survival over 12 weeks was observed in PAH-SSD patients treated with epoprostenol as compared to those receiving conventional therapy alone.

Baseline ValuesMean Change From Baseline
at End of Treatment Period
ParameterEpoprostenol Plus
Conventional Therapy
(n=56)
Conventional
Therapy Alone
(n=55)
Epoprostenol Plus
Conventional Therapy (n=50)
Conventional
Therapy Alone (n=48)
CI (L/min/m2)1.92.20.5-0.1
mPAP (mmHg)5149-51
mRAP (mmHg)1311-11
PVR (Wood units)1411-51
mSAP (mmHg)9389-8-1
Baseline Values
CI (L/min/m2)
Epoprostenol Plus
Conventional Therapy (n=56)
1.9
Conventional Therapy
Alone (n=55)
2.2
mPAP (mmHg)
Epoprostenol Plus
Conventional Therapy (n=56)
51
Conventional Therapy
Alone (n=55)
49
mRAP (mmHg)
Epoprostenol Plus
Conventional Therapy (n=56)
13
Conventional Therapy
Alone (n=55)
11
PVR (Wood units)
Epoprostenol Plus
Conventional Therapy (n=56)
14
Conventional Therapy
Alone (n=55)
11
mSAP (mmHg)
Epoprostenol Plus
Conventional Therapy (n=56)
93
Conventional Therapy
Alone (n=55)
89
Mean Change From Baseline at End of Treatment Period
CI (L/min/m2)
Epoprostenol Plus
Conventional Therapy (n=50)
0.5
Conventional Therapy
Alone (n=48)
-0.1
mPAP (mmHg)
Epoprostenol Plus
Conventional Therapy (n=50)
-5
Conventional Therapy
Alone (n=48)
1
mRAP (mmHg)
Epoprostenol Plus
Conventional Therapy (n=50)
-1
Conventional Therapy
Alone (n=48)
1
PVR (Wood units)
Epoprostenol Plus
Conventional Therapy (n=50)
-5
Conventional Therapy
Alone (n=48)
1
mSAP (mmHg)
Epoprostenol Plus
Conventional Therapy (n=50)
-8
Conventional Therapy
Alone (n=48)
-1
CI: cardiac index; mPAP: mean pulmonary arterial pressure; mRAP: mean right atrial pressure; mSAP: mean systemic arterial pressure; PVR: pulmonary vascular resistance. Denotes statistically significant difference between epoprostenol and conventional therapy groups (n is the number of patients with hemodynamic data).2
Epoprostenol plus conventional therapy§
Increased
Cardiac Index by 26%
Decreased
Mean Right Atrial Pressure by 8%
Mean Pulmonary Arterial Pressure by 10%
Pulmonary Vascular Resistance by 36%
Mean Systemic Arterial Pressure by 9%
Hemodynamic Data in PAH-SSD

In a 12-week, prospective, open-label, randomized trial, statistically significant differences in hemodynamic parameters were observed in patients who received epoprostenol plus conventional therapy.§

These hemodynamic parameters generally worsened in the group receiving conventional therapy alone.

§Compared with those who received conventional therapy alone.

Common Adverse Events
in PAH-SSD

The most common and dose-limiting adverse events during dose initiation and escalation (≥1%) were:

Flushing (58%)
Headache (49%)
Nausea/vomiting (32%)
Hypotension (16%)
Anxiety/nervousness/agitation (11%)
Chest pain (11%)
Dizziness (8%)
Bradycardia (5%)
Abdominal pain (5%)
Musculoskeletal pain (3%)
Dyspnea (2%)
Back pain (2%)
Sweating, dyspepsia, hypesthesia/paresthesia, tachycardia (1%), respectively

Adverse events occurring in patients with PAH-SSD with ≥10% difference between epoprostenol and conventional therapy alone were:

Flushing (23% vs 0%)
Hypotension (13% vs 0%)
Anorexia (66% vs 47%)
Nausea/vomiting (41% vs 16%)
Diarrhea (50% vs 5%)
Jaw pain (75% vs 0%)
Pain/neck pain/arthralgia (84% vs 65%)
Headache (46% vs 5%)
Skin ulcer (39% vs 24%)
Eczema/rash/urticaria (25% vs 4%)

Thrombocytopenia has been reported during uncontrolled clinical trials in patients receiving epoprostenol.

Potential adverse events from postmarketing evaluations include anemia, hypersplenism, pancytopenia, splenomegaly, and hyperthyroidism.

Although the relationship to epoprostenol administration has not been established, pulmonary embolism has been reported in several patients taking epoprostenol and there have been reports of hepatic failure.

References: 1. Badesch DB, Tapson VF, McGoon MD, et al. Continuous intravenous epoprostenol for pulmonary hypertension due to the scleroderma spectrum of disease: a randomized, controlled trial. Ann Intern Med. 2000;132:425-434. 2. VELETRI® (epoprostenol) for Injection Full Prescribing Information. Actelion Pharmaceuticals US, Inc.