The Natural History and Outcome of Scleroderma Patients at High Risk or with Early Pulmonary Hypertension

Study Status

Open to Enrollment

Study Description

The purpose of this study is to determine the timeline of progression from pre-pulmonary hypertension to diagnosable pulmonary hypertension based on right heart catheterization. Moreover, the study aims to determine the timeline for progression from diagnosable pulmonary hypertension to clinical worsening of disease — defined as death, hospitalization or worsening of pulmonary hypertension symptoms.

Systemic sclerosis is a rare, often fatal idiopathic disease that has no effective therapy. One of the most major complications of systematic sclerosis is pulmonary hypertension (PHT), which is now the cause of all scleroderma-related deaths. New therapeutic advances have improved short-term management of pulmonary hypertension in scleroderma, but long-term outcomes are unknown. With this in mind, the investigators have developed the Pulmonary Hypertension Assessment Registry of Scleroderma (PHAROS), a preventive, multicenter, web-based observational study that looks at the natural history and outcome of scleroderma patients who are at high risk for or have early pulmonary hypertension. Patients entered into the registry will be followed in prospective fashion, noting the clinical course of disease by both scheduled and event-driven follow-up. A thorough baseline history will be collected to determine key prognostic and correlative factors for both disease prevalence and progression. Yearly follow-up consisting of questionnaires, pulmonary function tests, echocardiogram, six-minute walk test and predefined patient characteristics will also be conducted to further understand and note the progression of scleroderma-related pulmonary arterial hypertension. Event-driven follow-up will occur to record findings and record specific predetermined events in the clinical course of disease.

Sponsor

Georgetown University

Key Eligibility

Inclusion criteria:

  • Patients over the age of 18 who have a clinical diagnosis of systemic sclerosis (ACR criteria or the LeRoy criteria for limited or diffuse scleroderma)
  • Diagnosis of pre-pulmonary hypertension defined as one of the following:
    • echocardiogram with a resting SPAP of > 40 mmHg
    • pulmonary function test with FVC > 70% and a DLCO < 55% of predicted or a FVC/DLCO ratio > 1.6
    • diagnosis of definite pulmonary hypertension
    • symptomatic pulmonary hypertension with a right heart catheterization showing a mean PA pressure > 25 mmHg and diagnosed in the past six months.
Exclusion criteria:
  • Diagnosis and treatment of pulmonary hypertension for six months or longer

Principal Investigator

Evelyn M. Horn, MD

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Contact Us

For general inquiries, or if you need assistance finding a study, please contact:

Robert Hagerty
Subject Recruitment Manager
Tel: (646) 962-9340
[email protected]

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