# Anastrozole in Pulmonary Arterial Hypertension (AIPH2) - CCC (Lead)

> **NIH NIH R01** · UNIVERSITY OF PENNSYLVANIA · 2020 · $1,094,913

## Abstract

Project Summary
From the earliest modern description, pulmonary arterial hypertension (PAH) has been recognized as a
“female” disease. Female sex is the strongest risk factor for idiopathic and heritable PAH. Despite the
decades-old recognition of this sex predilection, surprisingly few human and experimental studies have
focused on the mechanism of female sex in causing PAH and the use of treatment targeting sex hormones.
While estrogen has been traditionally been considered protective from cardiovascular disease, the strong
female sex predominance in PAH implicates estrogen in the pathophysiology of the disease. Aromatase
converts androgens to estradiol (E2) in the periphery and is responsible for most of the E2 production in post-
menopausal women and men. Our research group and others have performed several studies which suggest
that aromatase and estrogen production may play an important role in the risk of PAH. Anastrozole is a generic
aromatase inhibitor which is has been FDA-approved for breast cancer for 20 years and has an excellent
safety profile. In these clustered Clinical Coordinating and Data Coordinating Center applications, we propose
a Phase II randomized, double-blind, placebo-controlled trial in 84 post-menopausal women and men with PAH
for one year to determine if anastrozole increases the six minute walk distance, improves right ventricular
function, improves quality of life, and has an acceptable side effect and safety profile.

## Key facts

- **NIH application ID:** 9846235
- **Project number:** 5R01HL134905-04
- **Recipient organization:** UNIVERSITY OF PENNSYLVANIA
- **Principal Investigator:** Steven M Kawut
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $1,094,913
- **Award type:** 5
- **Project period:** 2017-01-01 → 2021-12-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9846235

## Citation

> US National Institutes of Health, RePORTER application 9846235, Anastrozole in Pulmonary Arterial Hypertension (AIPH2) - CCC (Lead) (5R01HL134905-04). Retrieved via AI Analytics 2026-05-21 from https://api.ai-analytics.org/grant/nih/9846235. Licensed CC0.

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