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

IRB Number: 2014-265

December 13, 2012

Institutional Review Board, Hospital for Special Surgery

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Principal Investigator

Jessica Gordon, MD

Co-Investigators

Robert F.Spiera, MD
Elana Bernstein, MD 
Stephen Lyman, PhD 
Wei-Ti Huang, MS 
Uzunma Udeh, BA
Nina Paddu, BA

Summary

This is a study of scleroderma patients to determine the time of pathologic progression of pulmonary hypertension from pre-pulmonary hypertension, to diagnosable pulmonary hypertension, to clinical worsening of disease.  Patients entered into the study will be followed in prospective fashion noting the clinical course of disease by both scheduled and event driven follow up. A total of 8-10 patients will be enrolled at this site and followed for a minimum period of 3-5 years (Approximately 15 sites will enroll 300-400 patients over the next 3 years).

Inclusion/Exclusion Criteria

INCLUSION CRITERIA
• Global Inclusion Criteria
o Eligible patients must meet all of the following inclusion criteria:
 Patient ³ 18 years with a clinical diagnosis of SSc (ACR criteria or the LeRoy criteria for limited or diffuse scleroderma
• Specific Inclusion Criteria
o 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.
o 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.
o 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 or a mean PA pressure > 30 with exercise.
o Patients who have a mean PA pressure > 25mmHg (on right heart catheterization)  who have severe interstitial fibrosis  will be entered as a definite pulmonary hypertension,  and classified and followed as WHO Group III even though they  would not have qualified for a ‘pre’ pulmonary hypertension.
o Patients who have a mean PA pressure > 25 with a wedge pressure which is > 15 will be entered and followed  and classified as  WHO Group II.

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.

Contact Information

Nina Paddu, BA
paddun@hss.edu
212-774-7194