Registry of Sarcoidosis Associated Pulmonary Hypertension (RESAPH)

Sarcoidosis is a multi systemic disease of unknown etiology with a highly
variable clinical course that pathologically is characterized by non-caseating
granulomas. Any organ may be involved, but the lungs and draining lymphnodes
are most commonly affected. In the Netherlands nearly 5000 people suffer from
sarcoidosis. Treatment options are limited and mainly aimed at preventing organ
damage. The majority of patients with sarcoidosis do well, but up to 9,7% of
the patients will eventually die of sarcoidosis.

The development of pulmonary hypertension is a severe complication of
sarcoidosis. The cause of pulmonary hypertension in sarcoidosis is
multi-factorial, including diastolic dysfunction, vessel compression by
adenopathy, lung destruction by fibrosis, and granulomatous vasculitis.
Sarcoidosis Associated Pulmonary Hypertension (SAPH) is an important risk
factor for early mortality in sarcoidosis patients. Prospective studies have
suggested that five to ten percent of all sarcoidosis patients have pulmonary
hypertension.

There is limited information about the management of pulmonary hypertension in
sarcoidosis. Most sarcoidosis centers have diagnosed few cases and no standard
therapy has evolved. Clinical trial for treatment of SAPH have been limited to
small series. Some patients improve when treated with pulmonary vasodilators.
Thus the optimal management of these patients remains unclear.

We propose to develop a multicenter registry of sarcoidosis associated
pulmonary hypertension (SAPH). With this registry, we will characterize the
demographics, clinical course, hemodynamics, pulmonary physiology, and disease
management of sarcoidosis associated pulmonary hypertension in the United
States. We will also compare these features to non-US sites.

An open label registry consisting of two populations of patients with SAPH. One
group will be the patients with known SAPH who are under care at the study
center. The second group will be a prospective study of newly diagnosed cases
of SAPH. the goal of the registry will be to include approximately 150 patients
in both groups for a total of 300 patients. A total of 6 centers from the
United States will be used and one of Europe (The Netherlands)

Next to the regular outpatient visits and investigations that are part of the
regular care for patients with SAPH, the additional burden associated with
participation in this study consists of:
- Filling in questionnaires: Patients will be asked to complete 3
questionnaires (St george*s respiratory questionnaire, FAS fatigue score and
SF-36 short-form health survey) that should take no more than 20-30 minutes of
their total time per visit.
- Next to the regular yearly six minute walk test, one additional six minute
walk test is performed per year in the first two years by the patient with
walking as far as they can in six minutes.
- During regular phlebotomy, additional blood is drawn

Information about the patient's underlying health will be collected. That
information will be stored in a secure database and identifiers will be used
(REDCap). There will be no specific information about a participant published
or otherwise made available.

The risk of the study is minimal.

There is no direct benefit for the patient in participating in this study. The
benefit would be to provide more information regarding this complication of
sarcoidosis.