Long-Term Treatment With Subcutaneous Treprostinil in Patients With Severe Inoperable Chronic Thromboembolic...

Area of Science:

• Cardiology
• Pulmonology
• Pharmacology

Background:

• Chronic thromboembolic pulmonary hypertension (CTEPH) is a severe condition requiring effective long-term management.
• Multimodal treatment strategies are crucial for patients with inoperable CTEPH.
• Subcutaneous treprostinil is a treatment option for pulmonary hypertension.

Purpose of the Study:

• To evaluate the long-term safety and tolerability of subcutaneous treprostinil in patients with severe CTEPH.
• To assess changes in functional class and exercise capacity over 24 months in the open-label extension (OLE) of the CTREPH study.
• To characterize multimodal treatment approaches used alongside treprostinil in this patient population.

Main Methods:

• Open-label extension (OLE) study following a 24-week blinded phase (CTREPH).
• Patients received high-dose (approx. 30 ng/kg/min) or low-dose (approx. 3 ng/kg/min) treprostinil.
• Treatment decisions (treprostinil dose, additional therapies) were based on standard of care and physician discretion.

Main Results:

• No new safety signals were detected during the 24-month OLE.
• Adverse events related to treprostinil were more frequent in the former low-dose group compared to the former high-dose group.
• Adverse events were less frequent over 24 months of OLE compared to the initial 6 months of the blinded trial, particularly infusion site pain.

Conclusions:

• Long-term subcutaneous treprostinil is a safe and effective component of multimodal therapy for severe CTEPH.
• Sustained clinical benefits in functional class and exercise capacity were observed over 24 months.
• Patients tolerating treprostinil initially are likely to maintain tolerability and clinical benefit long-term.