Research

The Natural History and Outcome of Scleroderma Patients at High Risk or with Early Pulmonary Hypertension: a Multicenter Study Performed by from the Scleroderma Clinical Trials Consortium (PHAROS)

IRB Number: 2014-265

Institutional Review Board, Hospital for Special Surgery

October 06, 2016

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Principal Investigator

Jessica K. Gordon, MD

Co-Investigators

Robert F. Spiera, MD
Annel Fernandez  
Emily Bakaj
Alexandra Morquette
Jaclyn Szymonifka

Summary

Systemic sclerosis (SSc) is a rare, often fatal disease with an unknown cause, which has no effective therapy. One of the most serious complications of systemic sclerosis is pulmonary hypertension (PHT), which is the most frequent cause of death from a scleroderma complication. New therapeutic advances have recently improved the short-term management of pulmonary hypertension in scleroderma, but the long-term outcomes are unknown.

This is a multicenter, longitudinal, prospective registry where we collect clinical and biological information on scleroderma patients with a high risk of developing pulmonary artery hypertension (PAH) or with recently diagnosed PAH. There are currently 5 enrolled patients. The study consist of a baseline visit and a 6 month follow up.

Inclusion/Exclusion Criteria

Inclusion:

Patient > 18 years with a clinical diagnosis of SSc (ACR criteria or the LeRoy criteria for limited or diffuse scleroderma)
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
Patients entered as a 'pre'-pulmonary arterial hypertension who then undergo right heart catheterization and are found to have pulmonary artieral hypersion, 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

 Exclusion:

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 group but do qualfy for the definite pulmonary hypertension group if they have a right heart catheterization showing a mean PAH of >25mmHg.

 

Contact Information

Annel Fernandez
Research Coordinator
212.774.2123
Fernandeza@hss.edu