Endothelial Function in Patients With Scleroderma or Cirrhosis With and Without Pulmonary Hypertension

NCT ID: NCT01729611

Last Updated: 2021-04-02

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 119 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2013-12-31
• Study Completion Date: 2021-04-30

Brief Summary

Pulmonary arterial hypertension (PAH) is a condition characterized by an increased pulmonary vascular resistance that can lead to right heart failure and death. Several diseases are known etiologies of PAH including scleroderma and cirrhosis. The presence of PAH in the context of systemic sclerosis or cirrhosis has a dramatic impact on prognosis and survival of the connective tissue or liver disease.

Despite advances in the diagnosis of PAH, echocardiography remains a necessary test for screening PAH in patients with scleroderma or cirrhosis. However, echocardiography is less than ideal for diagnosing PAH and predicting treatment response. Thus, there is a pressing need to identify methodologies that can accurately and non-invasively recognize the presence of PAH in patients with scleroderma and cirrhosis.

Hypothesis:

1. To measure endothelial function and exhaled gases in patients with scleroderma and cirrhosis. To assess whether they correlate with the presence or the development of PAH.

2. The degree of local (forearm) capillary vasodilation during treprostinil iontophoresis identifies patients who will develop PAH and in those already diagnosed PAH predicts response to PAH-specific therapies.

Detailed Description

Patients with scleroderma are known to have endothelial dysfunction and limited data suggested an association between the degree of endothelial function in scleroderma and the presence of PAH. However, these data is preliminary and has not been used to predict response to PAH-specific therapy or the development of PAH. We will test patients with cirrhosis because they tend to have PAH in the context of a hyperdynamic instead of a hypodynamic state as observed in scleroderma and PAH.

Aims:

1. To measure endothelial function and exhaled gases in patients with scleroderma or cirrhosis to assess whether they correlate with the presence or the development of PAH.

2. To evaluate the degree of endothelial response to local treprostinil iontophoresis and determine if this test can predict the development of pulmonary hypertension or response to PAH-specific therapies.

Conditions

Pulmonary Hypertension

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Scleroderma

60 patients with scleroderma - 30 with and 30 without Pulmonary Arterial Hypertension

No interventions assigned to this group

Cirrhosis

60 patients with cirrhosis - 30 with and 30 without Pulmonary Arteria Hypertension

No interventions assigned to this group

Eligibility Criteria

Inclusion Criteria

• Patients with scleroderma or cirrhosis.

• Minimum Eligible Age: 17 Years
• Maximum Eligible Age: 90 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

The Cleveland Clinic

OTHER

Sponsor Role: lead

Responsible Party

Adriano Tonelli

Staff Physician

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Adriano Tonelli, MD

Role: PRINCIPAL_INVESTIGATOR

The Cleveland Clinic

Locations

Cleveland Clinic

Cleveland, Ohio, United States

Countries

United States

Other Identifiers

Tonella1

Identifier Type: -

Identifier Source: org_study_id