Subcutaneous Treprostinil as a Bridge to Lung Transplantation in Severe Pulmonary Hypertension: A Single-Arm Retrospective Study

Phase 4

Completed

• Conditions: Pulmonary Hypertension; Pulmonary Arterial Hypertension; Chronic Lung Disease-Associated Pulmonary Hypertension; Lung Transplantation; Heart Failure
• Interventions: Drug: Subcutaneous Treprostinil

• Registration Number: NCT06605326
• Lead Sponsor: Guangzhou Institute of Respiratory Disease

Brief Summary

This single-arm, retrospective study aims to evaluate the safety and efficacy of subcutaneous treprostinil as a bridge therapy for lung transplantation in patients with severe pulmonary hypertension (PH). A total of 46 patients with either pulmonary arterial hypertension (PAH) or chronic lung disease-associated PH (CLD-PH) were included, all of whom were referred for lung transplantation between January 1, 2015, and September 1, 2024. The study examines changes in clinical, functional, and hemodynamic parameters following 3-6 months of treprostinil therapy.

Detailed Description

This study evaluates the use of subcutaneous treprostinil as a bridge therapy for lung transplantation in patients with severe pulmonary hypertension (PH), including both pulmonary arterial hypertension (PAH) and chronic lung disease-associated PH (CLD-PH). This retrospective, single-center study included 46 patients treated between January 1, 2015, and September 1, 2024. All patients experienced significant clinical and hemodynamic impairment at the time of treprostinil initiation. The primary aim was to assess improvements in NYHA functional class and 6-minute walk distance (6-MWD) following 3-6 months of therapy. Secondary outcomes included NT-proBNP levels, echocardiographic parameters, and post-transplantation survival rates. The study also analyzed the incidence of primary graft dysfunction (PGD) and the continued use of treprostinil post-transplantation.

Study Timeline

• Study Start: 2015-01-01
• Status Verified: 2024-09
• Primary Completion: 2024-09-01
• Study Completion: 2024-09-01
• Study First Submitted: 2024-09-18
• Study First Submitted QC: 2024-09-18
• Last Update Submitted: 2024-09-18
• Study First Posted: 2024-09-20
• Last Update Posted: 2024-09-20

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 46

Inclusion Criteria

• Patients diagnosed with severe pulmonary hypertension (PAH or CLD-PH) requiring lung transplantation
• Age 18 years or older
• Able to provide informed consent

Exclusion Criteria

• Patients with PH caused by left heart disease
• Patients unable to undergo lung transplantation due to medical conditions
• Patients with a history of severe drug allergies

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Subcutaneous Treprostinil Group	Subcutaneous Treprostinil	Patients in this group received subcutaneous treprostinil, starting at a dose of 1.25 to 4 ng/kg/min and gradually titrated according to patient response. The target dose was over 20 ng/kg/min after 3 months of treatment. All patients were referred for lung transplantation during or after treprostinil treatment.

Primary Outcome Measures

Name	Time	Method
Change in NYHA Functional Class	Baseline to 3-6 months after treatment initiation	The change in NYHA functional class will be assessed after 3-6 months of treatment with subcutaneous treprostinil. Improvement will be defined as a reduction in class (e.g., from class IV to class III or II).
Improvement in 6-Minute Walk Distance (6-MWD)	Baseline to 3-6 months after treatment initiation	The 6-minute walk distance (6-MWD) will be measured at baseline and after 3-6 months of subcutaneous treprostinil treatment to evaluate the improvement in exercise capacity.

Secondary Outcome Measures

Name	Time	Method
Change in NT-proBNP Levels	Baseline to 3-6 months after treatment initiation	NT-proBNP levels will be measured before and after treatment to assess changes in cardiac function.