# Mentored Patient-Oriented Research in Pulmonary Arterial Hypertension

> **NIH NIH K24** · UNIVERSITY OF PENNSYLVANIA · 2020 · $119,427

## Abstract

Steven M. Kawut, MD, MS is a Professor of Medicine and Epidemiology at the University of Pennsylvania
School of Medicine (Penn) who has committed his career to the research and care of patients with pulmonary
vascular disease (PVD). He heads a federally-funded research program based on the epidemiology and
treatment of PVD and right ventricular dysfunction. He has also mentored more than fifty trainees to success in
patient-oriented research (POR), resulting in publications and funding and in many cases their own
independent academic careers. The candidate's immediate- and long-term career goals during this competitive
renewal center on acquiring skills in performing comparative effectiveness research (CER) in rare PVDs and
developing his mentorship abilities, specifically in transitioning his funded mentees to independence and in
becoming mentors of their own trainees. The environment at Penn is ideal to develop the applicant as a mentor
and to foster the academic growth of mentees in these areas.
 Pulmonary arterial hypertension (PAH) is a rare disease which affects people of all ages, leading to
significant morbidity and a high risk of mortality. However, many questions about the study and therapeutic
approach to PAH remain unanswered. The minimally important difference (MID) for the six-minute walk
distance (6MWD) has not been clearly established in PAH. The use of the 6MWD in early phase studies and
as a component of clinical outcomes makes this an important area of research even in the era of long-term
studies in PAH. With the proliferation of new therapies for PAH, CER of different treatments in PAH in terms of
health-related quality of life and clinical worsening is a major area of interest. Dr. Kawut is involved in two new
initiatives which can address these critical questions in PAH and serve as training vehicles for himself and his
mentees. First, the FDA has provided Dr. Kawut with individual patient-level data (IPD) from all submitted
randomized clinical trials (RCTs) in PAH. Second, Dr. Kawut chairs the Steering Committee of the Pulmonary
Hypertension Association Registry (PHAR), which is a prospective registry of newly-diagnosed patients with
PAH at Pulmonary Hypertension Care Centers. We aim to derive and validate the MID in the change in 6MWD
in PAH and to determine the comparative effectiveness of different drug classes on health-related quality of life
and time to clinical worsening in patients with PAH.
 The long-term goal of this proposal is to refine the conduct of RCTs and clinical care in PAH by validating
the MID of the 6MWD and studying the comparative effectiveness of different treatment approaches. These
aims provide not only training opportunities for the mentor, but also valuable training and research experiences
for mentees at all levels. The opportunities for direct patient involvement in the CER being proposed and direct
patient contact make this project ideal for this K24 renewal.

## Key facts

- **NIH application ID:** 9993642
- **Project number:** 5K24HL103844-10
- **Recipient organization:** UNIVERSITY OF PENNSYLVANIA
- **Principal Investigator:** Steven M Kawut
- **Activity code:** K24 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $119,427
- **Award type:** 5
- **Project period:** 2011-05-01 → 2022-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9993642

## Citation

> US National Institutes of Health, RePORTER application 9993642, Mentored Patient-Oriented Research in Pulmonary Arterial Hypertension (5K24HL103844-10). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/9993642. Licensed CC0.

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