TranspulmonarY Estrogen Gradient and Estrogen Receptors (TYEGER) in PAH
Status:
Recruiting
Trial end date:
2024-10-01
Target enrollment:
Participant gender:
Summary
Pulmonary arterial hypertension (PAH) is a disease characterized by elevated pressures in the
blood vessels of the lungs that is not caused by another disease processes. More
specifically, it is defined by a mean pulmonary artery pressure > 25 mm Hg, a pulmonary
vascular resistance > 3 Wood Units (WU), and a normal pulmonary capillary wedge pressure in
the absence of other etiology of pulmonary hypertension. The underlying mechanism of the
disease in still unknown, but marked changes to the small arteries in the lungs have been
observed. These changes include thickening of vessel walls and clot formation -- making the
vessels less capable of gas exchange. Currently, PAH therapies focus on dilating the "good"
remaining vessels that haven't been altered by this disease process; however, this therapy
does not cure the disease. Survival remains low despite progress.
There is growing human and experimental evidence supporting the concept that estrogens and
estrogen receptors in the lungs are involved in the process that leads to PAH.
As mentioned above, no current therapies attack the cause of PAH; they only act to dilate
remaining "good" vessels which can reduce the burden of the disease, but not cure it. Thus,
there is a critical need for novel therapeutics, as recently highlighted by a National
Institute of Health workshop on pulmonary vascular diseases which called for the exploration
of novel therapeutic approaches. None of the current FDA-approved treatments for PAH target
estrogen or estrogen receptors.
Despite the evidence supporting the concept that estrogens and estrogen receptors in the
lungs contribute to PAH, no human studies investigate the estrogen level and the amount of
estrogen receptors within the lungs of patients with PAH and their potential associations
with current disease severity or 1 year outcomes including survival after 1 year, functional
status, etc. Investigators hypothesize that a subset of PAH patients will have higher levels
of estrogen and estrogen receptors in their lungs which would make them good candidates for
novel therapies that block estrogen in hopes of halting the disease process.