Multi-center, Web Based Observational Study of Pulmonary Hypertension in Scleroderma Patients
Multi-center, Web Based Observational Study of Pulmonary Hypertension in Scleroderma Patients
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, to determine the timeline for progression from diagnosable pulmonary hypertension
 to clinical worsening of disease as defined as death, hospitalization, or worsening of PHT
 symptoms.
Systemic sclerosis (SSc) is a rare, often fatal idiopathic disease, which 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, Dr. Steen has developed Pulmonary
 Hypertension Assessment Registry of Scleroderma (PHAROS), a preventive, multi-center, web
 based observational study that looks at the natural history and outcome of scleroderma
 patients who are at high risk 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, 6
 minute walk tests and predefined patient characteristics will also be conducted to further
 understand and note the progression of scleroderma related PAH. Event driven follow up will
 occur to record findings and record specific predetermined events in the clinical course of
 disease.
1. Global Inclusion Criteria

 -  Eligible patients must meet all of the following inclusion criteria:

 -  Patient ≥ 18 years with a clinical diagnosis of SSc (ACR criteria or the LeRoy
 criteria for limited or diffuse scleroderma

 2. Specific Inclusion Criteria

 -  Diagnosis of "pre" pulmonary arterial hypertension defined as:

 -  Echocardiogram with a resting sPAP of ≥ 40mmHg Or

 -  Pulmonary function test with FVC >70% and a DLCO <55% of predicted or a FVC/DLco
 ratio >1.6. or

 -  Right heart catheterization which shows or a mean PA pressure > 30mmHg with
 exercise (with a mPAP < 25mmHg at rest)

 Patients entered as a 'pre'-pulmonary arterial hypertension who then undergo right heart
 catheterization and are found to have pulmonary arterial hypertension, pulmonary venous
 hypertension or diastolic dysfunction or pulmonary hypertension secondary to interstitial
 lung disease will be followed as a definite PH patient and classified into the appropriate
 category.

 -  Diagnosis of definite pulmonary hypertension Patients with pulmonary hypertension with
 a right heart catheterization showing a mean PA pressure > 25mmHg, diagnosed in the
 past 6 months.

 Classification of PH Group 1 PAH - Patients with mPAP ≥ 25mmHg with a wedge < 15mmHg Group
 2 PVH - Patients who have a mean PA pressure ≥ 25mmHg with a wedge pressure which is > 15
 mmHg Group 3 PH-ILD Patients who have a mean PA pressure ≥ 25mmHg (on right heart
 catheterization) who have moderate to severe interstitial fibrosis on HRCT scan with a FVC
 and TLC < 65% predicted

 b. Exclusion Criteria

 -  Diagnosis and treatment of pulmonary hypertension for > 6 months

 -  Patients with known severe interstitial fibrosis, pulmonary thrombotic disease, heart
 failure, cardiomyopathy,history of coronary artery disease or other cardio-pulmonary
 problems which could cause pulmonary hypertension are not eligible for the
 'pre'-pulmonary hypertension but do qualify for the definite pulmonary hypertension
 group if they have a right heart catheterization showing a mean PAH >25mmHg.