Clinical trial NCT01729611: Endothelial Function in Patients With Scleroderma or Cirrhosis With and Without Pulmonary Hypertension
Official title: Endothelial Function in Patients With Scleroderma or Cirrhosis With and Without Pulmonary Hypertension
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Pulmonary Hypertension
Keywords: Scleroderma
Cirrhosis
Lead sponsor: The Cleveland Clinic
Sponsor class: OTHER
First posted: 2012-11-20
Enrollment: 119
Sex: ALL
Minimum age (years): 17
Maximum age (years): 90
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with scleroderma or cirrhosis. These patients may or may not have known PAH or be on treatment of this condition.

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.

Eligibility criteria: Inclusion Criteria:

* Patients with scleroderma or cirrhosis.

Exclusion Criteria:

* Exclusion criteria include individuals younger than 16, etiologies of PAH other than scleroderma or cirrhosis.

Primary outcomes:
measure: Base Line
timeFrame: up to 3 months
Secondary outcomes:
Other outcomes:
Locations:
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
Study officials:
affiliation: The Cleveland Clinic
name: Adriano Tonelli, MD
role: PRINCIPAL_INVESTIGATOR
References: