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

NCT06605326 | Completed Phase 4

• 1 other identifier: 2022K-48
• Study Type: interventional
• Target: 46
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Pulmonary Hypertension; Pulmonary Arterial Hypertension; Chronic Lung Disease-Associated Pulmonary Hypertension; Lung Transplantation; Heart Failure

Keywords

Subcutaneous Treprostinil; NYHA Functional Class; 6-Minute Walk Distance; Primary Graft Dysfunction (PGD); Retrospective Study; Single-Arm Study

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (1)

• Change in NT-proBNP Levels

  Baseline to 3-6 months after treatment initiation

Study Arms (1)

Subcutaneous Treprostinil Group

EXPERIMENTAL

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

Interventions

Subcutaneous Treprostinil DRUG

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.

Subcutaneous Treprostinil Group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Guangzhou Institute of Respiratory Disease: lead

MeSH Terms

Conditions

Hypertension, Pulmonary; Pulmonary Arterial Hypertension; Heart Failure; Primary Graft Dysfunction

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Hypertension; Vascular Diseases; Cardiovascular Diseases; Heart Diseases; Reperfusion Injury; Postoperative Complications; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Model Details: This is a single-arm, retrospective study in which all enrolled patients received subcutaneous treprostinil as a bridge therapy for lung transplantation. The study evaluates the efficacy and safety of the treatment in patients with severe pulmonary hypertension, including both PAH and CLD-PH. No randomization or control group is included in the study.

Study Record Dates

• First Submitted: September 18, 2024
• First Posted: September 20, 2024
• Study Start: January 1, 2015
• Primary Completion: September 1, 2024
• Study Completion: September 1, 2024
• Last Updated: September 23, 2024

Record last verified: 2024-09

Data Sharing

• IPD Sharing: Will not share