Disconnect Exists Between PAH Study Outcomes and Patients’ Priorities

Patients who have pulmonary arterial hypertension (PAH) often do not feel better after treatment, even if they meet clinical endpoints for improvement.

The outcomes by which pulmonary arterial hypertension (PAH) treatments are judged by patients are often different from those used to assess therapies in clinical trials, according to a new report published in Pulmonary Circulation. This study argues that a greater focus on patient-centered outcomes would improve future trials and ultimately help patients better achieve outcomes that directly improve their lives.

The study authors said hemodynamics and scores on the 6-minute walking test have traditionally been used as the primary outcomes of clinical trials in PAH therapies, but they said such measures do not necessarily reflect the priorities of patients.

Recent studies have included other end points, but both have limitations, the authors said.

“HRQOL [health-related quality of life] has only been included as a secondary outcome and is commonly plagued by informative data missingness,” they wrote. “Clinical worsening has been used as a primary outcome, however, with varying components and without clear or consistent impact on patients.”

The investigators decided to conduct a qualitative study to gain insights from patients and clinicians about their own treatment priorities, in hopes that such data would inform the design of future trials. They recruited 11 patients and 8 clinicians and conducted individual semi-structured interviews. Each interview lasted 20 to 40 minutes. Eight of the patients were women, and the median age was 60 years. Two of the patients were Black. The etiologies of the patients’ disease varied, and 54% had functional class 3 disease according to the World Health Organization’s classification. The clinicians had a median age of 45 and had practiced for at least 5 years.

In the interviews, patients said their goals for PAH therapy would be an improvement in—or at least maintenance of—their ability to go about their daily activities without being sidelined by fatigue and breathlessness. They also said the disease affected their psychosocial well-being, in part because they felt socially isolated when they were unable to participate in activities with friends or when they required assistance in order to participate.

The 8 clinicians interviewed tended to focus more on clinical outcomes, including right ventricular function, 6-minute walk distance, hospitalization, and PAH risk prediction scores, the authors noted. However, the providers also conceded that such end points did not reflect the priorities of patients. One clinician quoted in the study said patients often do not feel better despite clinical improvement.

“All these objective tests we do are what the physician wants to see,” the provider said. “The quality of life is what patients want to see.”

The clinicians said personalized physical activity assessments might be a better way to link clinician and patient outcomes, such as by having patients use a wearable fitness tracker to monitor their activity levels. Clinicians also said quality of life needs to be a measured outcome, although it can be hard to accurately assess.

The study investigator said many of the factors identified in the interviews are reflected in HRQOL scores, but they said effective measurement of quality of life needs to be disease-specific.

“Lack of consensus around the optimal PAH-specific HRQOL instrument demonstrates the need for future comparative assessments and refinement of existing tools,” they said.

Likewise, they said the use of a composite end point like “clinical worsening” could be improved if it also included metrics like daily activity levels and psychosocial well-being.

The authors noted that their interviews were based on patients from a single academic medical center and did not reflect a diverse patient population. However, they said the findings offer important perceptions they said ought to inform future studies.


Auriemma CL, Minhas J, Blue R, Lapatra T, Kawut SM, Courtright KR. "It's that invisible illness": patient and clinician perspectives on outcomes in pulmonary arterial hypertension treatment. Pulm Circ. 2023;13(2):e12236. Published 2023 May 8. doi:10.1002/pul2.12236

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