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

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.

Study Overview

• Status: Completed
• Conditions: Heart Failure; Pulmonary Arterial Hypertension; Pulmonary Hypertension; Lung Transplantation; Chronic Lung Disease-Associated Pulmonary Hypertension
• Intervention / Treatment: Drug: Subcutaneous Treprostinil

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 Type: Interventional
• Enrollment (Actual): 46
• Phase: Phase 4

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Subcutaneous Treprostinil Group; 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.	Drug: 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in NYHA Functional Class	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).	Baseline to 3-6 months after treatment initiation
Improvement in 6-Minute Walk Distance (6-MWD)	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.	Baseline to 3-6 months after treatment initiation

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Guangzhou Institute of Respiratory Disease

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2015
• Primary Completion (Actual): September 1, 2024
• Study Completion (Actual): September 1, 2024

Study Registration Dates

• First Submitted: September 18, 2024
• First Submitted That Met QC Criteria: September 18, 2024
• First Posted (Actual): September 20, 2024

Study Record Updates

• Last Update Posted (Actual): September 23, 2024
• Last Update Submitted That Met QC Criteria: September 19, 2024
• Last Verified: September 1, 2024

More Information

Terms related to this study

• Keywords: Subcutaneous Treprostinil; Retrospective Study; 6-Minute Walk Distance; NYHA Functional Class; Primary Graft Dysfunction (PGD); Single-Arm Study
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Respiratory Tract Diseases; Hypertension; Lung Diseases; Pulmonary Arterial Hypertension; Hypertension, Pulmonary; Antihypertensive Agents; Treprostinil
• Other Study ID Numbers: 2022K-48

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: There is no plan to share individual participant data (IPD) from this study. Due to privacy concerns and the sensitive nature of the health data collected, the research team has decided not to make individual-level data available to external researchers. All data will remain confidential and will be used solely for the purposes of this study, in compliance with institutional and ethical guidelines.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No