Pediatric Pulmonary Hypertension Network (PPHNet) Informatics Registry

Pulmonary Hypertension (PH) is a syndrome characterized by vasoconstriction and abnormal
growth and function of endothelial and smooth muscle cells and other components within the
pulmonary vessels, which leads to elevation of the pulmonary artery pressure. PH may be
idiopathic (primary) without any known cause. Some cases of PH are familial. PH may also be
secondary to a specific disease process such as portal hypertension, congenital heart
disease, chronic lung disease, thromboembolic disease, connective tissue disease, human
immunodeficiency virus (HIV), and use of anorexigens. Left untreated, PH is often progressive
and fatal. There is no cure for PH. Therapy focuses upon treatment of secondary causes if
present, and reduction of the pulmonary artery pressure through medical therapy. There have
been many new developments within the past few years in the management of patients with PH.
While there is no cure for PH early detection and treatment are important for survival of
patients. Limited data is available that describes the etiologies, clinical course and
prognosis of pediatric pulmonary hypertension.

Objectives

Aim 1: Clinical Research

1. To provide a mechanism to store information about newborns, infants and children with
PH;

2. To determine the incidence and natural history of the various etiologies of pediatric
PH;

3. To define the investigator current diagnostic and therapeutic approaches to the diverse
conditions associated with pediatric PH;

4. To determine the response of children with PH to chronic therapies.

Aim 2: Research Infrastructure To create a robust scalable data architecture, to combine
traditional registry data, electronic Health Record (EHR), and PRO (Patient Reported Outcome)
data in a single resource.

Aim 3: Informatics Address three classes of unanswered questions crucial for the
characterization and management of PH, comparing the information value of registry vs. EHR
vs. fused data across registry/EHR/PROs, in the domains of spectrum of PH comorbidities, PH
indicators and endpoints of morbidity and mortality, and response to therapies in PH.

Inclusion Criteria:

- The subject's age of onset of pulmonary hypertension must be prior to age 18 years

- The person providing consent must be able to read either Spanish or English.

- The subject (and/or parent/legal guardian) must be able to provide informed consent

Exclusion Criteria:

- Diagnosed with pulmonary hypertension after age 18

- Refusal to sign informed consent