Ongoing recruitment
The Natural History and Outcome of Patients with
Scleroderma at High Risk for or with Early Pulmonary Hypertention
Study sponsor:
Scleroderma Foundation, Sibley Foundation and Actelion Pharmaceuticals
Coordinating_center:
Georgetown Universtiy Medical Center Division of Rheumatology, Immunology and
Allergy
Principal Investigator: Virginia D. Steen, MD
Project Coordinator: Suria Yesmin
Phone: (202) 687-6317
Email: sy86@georgetown.edu
Other centers:
Boston University
Principal Investigator: Robert W. Simms, MD
Project Coordinator: Kim Tobin
Phone: (617) 638-4310
Email: kimtobin@bu.edu
Not yet recruiting
University of Pittsburgh Division of Rheumatology
Principal Investigator: Tom Medsger, MD
Project Coordinator: Jennifer Jablon
Phone: (412)383-8674
Email: JablonJ@dom.pitt.edu
Not yet recruiting
John Hopkins University
Principal Investigator: Fredrick Wigley, MD
Project Coordinator: Adrianne Woods
Research Nurse Coordinator: Edrick Forbes
Phone: (410) 550-8582
Email: eborbes1@jhmi.edu
The University of Texas Health Science Center, Houston
Principal Investigator: Maureen D. Mayes, MD MPH
Project Coordinator: Marcia Kerr, RN
Phone: (718) 500-7118
Email: Marcia.L.Kerr@uth.tmc.edu
Medical University of South Carolina Division of Rheumatology & Immunology
Principal Investigator: Marcy Bloster, MD
Project Coordinator: Trisha Sturgill
Phone: (843) 792-5290
Email: sturgilt@musc.edu
Northwestern University Division of Rheumatology
Principal Investigator: John Varga, MD
Project Coordinator: Anabel Garza
Phone: (312) 503-0377
Email: a-garza@northwestern.edu
Center for Rheumatology, Albany
Principal Investigator: Lee Shapiro, MD
Project Coordinator: Tanya Sommers, BS RN CCRC
Phone: (518) 533-1326
Email: tsommers@joint-docs.com
University of Medicine and Dentistry, New Jersey
Principal Investigator: Vivien Hsu, MD
Project Coordinator: Julianne Wilson, BSN RN
Phone: (732) 235-7583
Email: wilsonj6@umdnj.edu
The Medical College of Wisconsin Department of Medicine & Rheumatolgy
Principal Investigator: Mary Ellen Csuka, MD
Email: mecsuka@mcw.edu
University of Michigan Division of Rheumatology
Principal Investigator: James R. Seibold, MD
Project Coordinator: Ann Julie Impens, PhD
Phone: (734) 936-1439
Email: aimpens@med.umich.edu
Not yet recruiting
Thomas Jefferson University
Principal Investigator: Chris Derk, MD
Email: chris.derk@jefferson.edu
University of California, Los Angeles Division of Rheumatology
Principal Investigator: Daniel Furst, MD
Project Coordinator: Tina Burger
Phone: (310) 825-3061
Email: CBurger@mednet.ucla.edu
University of Connecticut
Principal Investigator: Naomi Rothfield, MD
Email: rothfield@nso.uchc.edu
Stanford University Division of Rheumatology and Immunology
Principal Investigator: Lorinda Chung, MD
Phone: (650) 493-5000
Email: shauwei@stanford.edu
Not yet recruiting
Study design:
Systemic Sclerosis (SSc) is a rare, often fatal idiopathic disease, which has no
effective therapy. One of the major complications of systemic sclerosis is
pulmonary hypertension (PHT), which is now the cause of all scleroderma related
deaths. In light of this, PHAROS (Pulmonary Hypertension Assessment Registry of
Scleroderma) was developed to determine the timeline of pathologic progression
of pulmonary hypertension from pre-pulmonary hypertension to diagnosable
pulmonary hypertension, to clinical worsening of disease. The purpose is to
determine the best risk factors for detecting pulmonary arterial hypertension (PAH)
in scleroderma patients. 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 pulmonary functional test,
echocardiogram, 6 minute walk test 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 finding and record
specific predetermined events in the clinical course of disease. Over the next
several years, 400 patients will be entered by SCTC/PHAROS participating
centers.
Inclusion criteria:
Eligible patients at high risk for PAH (or with ‘pre’ PAH) will have the
following: 1. An increased resting pulmonary artery pressure on echocardiogram
>35mmHg, or 2. A decreased DLCO <55% predicted (without severe interstitial
fibrosis) or 3. FVC/DLCO ratio > 1.6 Patients with early PAH will have a
documented mean PAP >25mmHg on right heart catheterization within 6 months of
entry.
Exclusion criteria:
Diagnosis and treatment of pulmonary hypertension for >6 months.
Primary outcome measure:
The time from "early" pulmonary hypertension to diagnosable pulmonary
hypertension will be expressed in terms of the standard deviation and mean
number of days from diagnosis of "early" PHT to diagnosable PHT.
Secondary outcome measures:
The time from diagnosable pulmonary hypertension to occurance of clinical
worsening will be expressed in terms of the standard deviation and mean number
of days from diagnosis of PHT to the event documentation of clinical worsening.
Recruitment period:
Ongoing
Completion:
2010
Date of expected analysis:
Data analysis will begin at the completion of this observational clinical
research study.